Having demonstrated the ability to offer improved treatment related outcomes and also enhance the quality of lives of patients suffering from a diverse range of clinical conditions, the demand for cell therapies is anticipated to increase in the near future

Roots Analysis has announced the addition of Cell Therapy Manufacturing Market (2nd Edition), 2018 2030 report to its list of offerings.

Given the commercial success of multiple cell therapies, such as RECELL (Avita Medical) and YESCARTA (Gilead Sciences), and an evolving clinical pipeline, the opportunity for contract development and manufacturing organizations (CDMOs) is anticipated to grow significantly. Over the years, many service providers have begun automating their operations, in order to eliminate chances of human error during manufacturing.

To order this 400+ page report, which features 125+ figures and 175+ tables, please visit this link.

Over 145 companies / organizations are actively involved in manufacturing cell-based therapiesThe market landscape is currently dominated by the presence of industry players, which represent more than 55% of the total number of players. Amongst these, over 45 are large or mid-sized firms (having more than 50 employees). It is also worth noting that this field has witnessed the entry of several start-ups.

50+ organizations claim to possess commercial manufacturing capabilities for cell therapiesAs most of the cell therapy products are under clinical evaluation, majority of the manufacturing facilities currently have the capacity to support clinical scale production requirements. At the same time, it is worth noting that several players (over 35%) have already developed / are developing commercial scale capacity for cell therapies.

Europe has emerged as a key region for the production of cell therapies with more than 40% of manufacturing facilitiesGlobally, 195 facilities have been established by various players for the manufacturing of the cell therapies; of these, 41% are located in Europe, followed by those based in North America (38%). Other emerging regions include Australia, China, Japan, Singapore, South Korea and Israel.

Close to 15 companies are presently offering automated solutions to cell therapy developersOver the years, automation has emerged as a key enabler of cell therapy manufacturing. Players that claim to offer consultancy services related to automation include (in alphabetical order) these include (in alphabetical order) Berkeley Lights, Cesca Therapeutics, Ferrologix, FluDesign Sonics, GE Healthcare and Terumo BCT. Further, we identified five players, namely (in alphabetical order) Fraunhofer Institute for Manufacturing Engineering and Automation IPA, Invetech, KMC Systems, Mayo Clinic Center for Regenerative Medicine and RoosterBio, that offer consultancy solutions related to automation.

150+ partnerships were inked between 2014 and 2018Of these, 32% were observed to be focused on the supply of cell-based therapy products for clinical trials. Other popular types of collaboration models implemented in this domain include additional services agreements (24%), joint ventures (9%) and acquisitions (3%).

North America anticipated to capture 50% of the cell therapy manufacturing market by 2030Owing to high venture capital funding and drug development activity, North America is anticipated to capture close to 50% of the total market share by 2030. It is also important to highlight that financial resources, technical expertise and established infrastructure is likely to drive cell therapy manufacturing market in Europe to grow at a CAGR of over 18%.

To request a sample copy / brochure of this report, please visit this link

The USD 10+ billion (by 2030) financial opportunity within the cell therapy manufacturing market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom manufacturing of cell therapies is largely being outsourced due to the exorbitant costs associated with setting-up in-house facilities. The report includes detailed transcripts of discussions held with the following experts:

The research covers profiles of key players (industry and non-industry) that offer contract manufacturing services for cell-based therapies, featuring an overview of the company, information on its manufacturing facilities, and recent collaborations.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing-market-2nd-edition-2018-2030/209.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Cell Therapy Manufacturing Market is projected to reach close to USD 11 Billion by 2030 - Eurowire

Sachin Mohan,1 3 Shaffer Mok,4 Thomas Judge5

1Department of Gastroenterology and Hepatology, University of Minnesota School of Medicine, St Paul, MN, USA; 2Regions Hospital, Department of Gastroenterology and Hepatology, St Paul, MN, USA; 3Health Partners Digestive Care Center, St Paul, MN, 55130, USA; 4Division of Gastroenterology and Hepatology, University Hospital Digestive Health Institute, Westlake, OH 44145, USA; 5Division of Gastroenterology and Liver Diseases, Cooper University Hospital, Mount Laurel, NJ 08054, USA

Correspondence: Sachin MohanRegions Hospital, Department of Gastroenterology and Hepatology, 435 Phalen Blvd, St Paul, MN 55130, USATel +1 651-254-8680Fax +1 651-254-8656Email sachinmohan01@gmail.com

Purpose: Utilization of genetic databases to identify genes involved in ulcerative colitis (UC), Crohns disease (CD), and their extra-intestinal manifestations.Methods: Protein coding genes involved in ulcerative colitis (3783 genes), Crohns disease (3980 genes), uveitis (1043 genes), arthritis (5583 genes), primary sclerosing cholangitis (PSC) (1313 genes), and pyoderma gangrenosum (119 genes) were categorized using four genetic databases. These include Genecards: The Human Gene Database (www.genecards.org), DisGeNET (https://www.disgenet.org/), The Comparative Toxicogenomics Database (http://ctdbase.org/) and the Universal Protein Resource (https://www.uniprot.org/). NDex, Network Data Exchange (http://www.ndexbio.org/), was then utilized for mapping a unique signal pathway from the identified shared genes involved in the above disease processes.Results: We have detected a unique array of 20 genes with the highest probability of overlay in UC, CD, uveitis, arthritis, pyoderma gangrenosum, and PSC. Figure 1 represents the interactome of these 20 protein coding genes. Of note, unique immune modulators in different disease processes are also noted. Interleukin-25 (IL-25) and monensin-resistant homolog 2 (MON-2) are only noted in UC, CD, pyoderma gangrenosum, and arthritis. Arachidonate 5-lipoxygenase (ALOX5) is involved in UC, CD, and arthritis. SLCO1B3 is exclusively involved with pyoderma gangrenosum, UC, and CD. As expected, TNF involvement is noted in CD, UC, PSC, and arthritis. Table 1 depicts the detailed result.Conclusion: Our work has identified a distinctive set of genes involved in IBD and its associated extra-intestinal disease processes. These genes play crucial roles in mechanisms of immune response, inflammation, and apoptosis and further our understanding of this complex disease process. We postulate that these genes play a critical role at intersecting pathways involved in inflammatory bowel disease, and these novel molecules, their upstream and downstream effectors, are potential targets for future therapeutic agents.

Keywords: inflammatory bowel diseases, IBD, ulcerative colitis, UC, Crohns disease, CD, arthritis, primary sclerosing cholangitis, PSC, uveitis, pyoderma gangrenosum

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Identification of Novel Therapeutic Molecular Targets in Inflammatory | CEG - Dove Medical Press

A study of some of the sickest COVID-19 patients, such as those placed on ventilators, has identified gene variants that put people at greater risk of severe disease.

By Jocelyn KaiserOct. 13, 2020 , 1:25 PM

Sciences COVID-19 reporting is supported by the Pulitzer Center and the Heising-Simons Foundation.

Its one of the pandemics puzzles: Most people infected by SARS-CoV-2 never feel sick, whereas others develop serious symptoms or even end up in an intensive care unit clinging to life. Age and preexisting conditions, such as obesity, account for much of the disparity. But geneticists have raced to see whether a persons DNA also explains why some get hit hard by the coronavirus, and they have uncovered tantalizing leads.

Now, a U.K. group studying more than 2200 COVID-19 patients has pinned down common gene variants that are linked to the most severe cases of the disease, and that point to existing drugs that could be repurposed to help. Its really exciting. Each one provides a potential target for treatment, says genetic epidemiologist Priya Duggal of Johns Hopkins University.

In a standard approach to finding genes that influence a condition, geneticists scan the DNA of large numbers of people for millions of marker sequences, looking for associations between specific markers and cases of the disease. In June, one such genomewide association study in The New England Journal of Medicine (NEJM) found two hits linked to respiratory failure in 1600 Italian and Spanish COVID-19 patients: a marker within the ABO gene, which determines a persons blood type, and a stretch of chromosome 3 that holds a half-dozen genes. Those two links have also emerged in other groups data, including some from the DNA testing company 23andMe.

The new study confirmed the chromosome 3 regions involvement. And because 74% of its patients were so sick that they needed invasive ventilation, it had the statistical strength to reveal other markers, elsewhere in the genome, linked to severe COVID-19. One find is a gene called IFNAR2 that codes for a cell receptor for interferon, a powerful molecular messenger that rallies the immune defenses when a virus invades a cell. A variant of IFNAR2 found in one in four Europeans raised the risk of severe COVID-19 by 30%. Baillie says the IFNAR2 hit is entirely complementary to a finding reported in Science last month: very rare mutations that disable IFNAR2 and seven other interferon genes may explain about 4% of severeCOVID-19 cases. Both studies raise hopes for ongoing trials of interferons as a COVID-19 treatment.

A more surprising hit from the U.K. study points to OAS genes, which code for proteins that activate an enzyme that breaks down viral RNA. A change in one of those genes might impair this activation, allowing the virus to flourish. The U.K. data suggest there is a variant as common and influential on COVID-19 as the interferon genetic risk factor.

Other genes identified by Baillies team could ramp up the inflammatory responses to lung damage triggered by SARS-CoV-2, reactions that can be lethal to some patients. One, DPP9, codes for an enzyme known to be involved in lung disease; another, TYK2, encodes a signaling protein involved in inflammation. Drugs that target those two genes proteins are already in useinhibitors of DPP9s enzyme for diabetes and baricitinib, which blocks TYK2s product, for arthritis. Baricitinib is in early clinical testing for COVID-19, and the new data could push it up the priority list, Baillie says.

The chromosome 3 region still stands out as the most powerful genetic actor: A single copy of the disease-associated variant more than doubles an infected persons odds of developing severe COVID-19. Evolutionary biologists reported last month in Nature that this suspicious region actually came from Neanderthals, through interbreeding with our species tens of thousands of years ago. It is now found in about 16% of Europeans and 50% of South Asians.

But the specific chromosome 3 gene or genes at play remain elusive. By analyzing gene activity data from normal lung tissue of people with and without the variant, the U.K. team homed in on CCR2, a gene that encodes a receptor for cytokine proteins that play a role in inflammation. But other data discussed at last weeks meeting point to SLC6Z20, which codes for a protein that interacts with the main cell receptor used by SARS-CoV-2 to enter cells. I dont think anyone at this point has a clear understanding of what are the underlying genes for the chromosome 3 link, says Andrea Ganna of the University of Helsinki, who co-leads the COVID-19 Host Genetics Initiative.

The U.K. genetics study did not confirm that the ABO variants affect the odds of severe disease. Some studies looking directly at blood type, not genetic markers, have reported that type O blood protects against COVID-19, whereas A blood makes a person more vulnerable. It may be that blood type influences whether a person gets infected, but not how sick they get, says Stanford University geneticist Manuel Rivas. In any case, O blood offers at best modest protection. There are a lot of people with O blood that have died of the disease. It doesnt really help you, says geneticist Andre Franke of the Christian-Albrecht University of Kiel, a coleader of the NEJM study.

Researchers expect to pin down more COVID-19 risk genesalready, after folding in the U.K. data plumbed by Baillies team, the COVID-19 Host Genetics Initiative has found another hit, a gene called FOXP4 implicated in lung cancer. And in a new medRxiv preprint posted last week, the company Ancestry.com reports that a gene previously connected to the effects of the flu may also boost COVID-19 susceptibility only in men, who are more likely to die of the disease than women.

Geneticists have had little luck so far identifying gene variants that explain why COVID-19 has hit Black people in the United States and United Kingdom particularly hard. The chromosome 3 variant is absent in most people of African ancestry. Researchers suspect that socioeconomic factors and preexisting conditions may better explain the increased risks. But several projects, including Baillies, are recruiting more people of non-European backgrounds to bolster their power to find COVID-19 gene links. And in an abstract for an online talk later this month at the American Society of Human Genetics annual meeting, the company Regeneron reports it has found a genome region that may raise the risk of severe disease mainly in people of African ancestry.

Even as more genetic risk factors are identified, their overall effect on infected people will be modest compared with other COVID-19 factors, Duggal says. But studies like the U.K. teams could help reveal the underlying biology of the disease and inspire better treatments. I dont think genetics will lead us out of this. I think genetics may give us new opportunities, Duggal says.

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Found: genes that sway the course of the coronavirus - Science Magazine

The immune response to infections is a delicate balance. We need just enough action to clear away the offending bacteria or viruses, but not so much that our own bodies suffer collateral damage.

Macrophages are immune cells at the front line, detecting pathogens and kicking off an inflammatory response when needed. Understanding how macrophages determine when to go all-out and when to keep calm is key to finding new ways to strike the right balance particularly in cases where inflammation goes too far, such as in sepsis, colitis and other autoimmune disorders.

In a study published October 14, 2020 in the Proceedings of the National Academy of Sciences , researchers at University of California San Diego School of Medicine discovered that a molecule called Girdin, or GIV, acts as a brake on macrophages.

When the team deleted the GIV gene from mouse macrophages, the immune cells rapidly overacted to even small amounts of live bacteria or a bacterial toxin. Mice with colitis and sepsis fared worse when lacking the GIV gene in their macrophages.

The researchers also created peptides that mimic GIV, allowing them to shut down mouse macrophages on command. When treated with the GIV-mimic peptide, the mices inflammatory response was tempered.

When a patient dies of sepsis, he or she does not die due to the invading bacteria themselves, but from an overreaction of their immune system to the bacteria, said senior author Pradipta Ghosh, MD, professor at UC San Diego School of Medicine and Moores Cancer Center. Its similar to what were seeing now with dangerous cytokine storms that can result from infection with the novel coronavirus SARS-CoV-2. Macrophages, and the cytokines they produce, are the bodys own immune-stimulating agents and when produced in excessive amounts, they do more harm than good.

Digging deeper into the mechanism at play, Ghosh and team discovered that the GIV protein normally cozies up to a molecule called Toll-like receptor 4 (TLR4). TLR4 is stuck right through the cell membrane, with bits poking inside and outside the cell. Outside of the cell, TLR4 is like an antenna, searching for signs of invading pathogens. Inside the cell, GIV is nestled between the receptors two feet. When in place, GIV keeps the feet apart, and nothing happens. When GIV is removed, the TLR4 feet touch and kick off a cascade of immune-stimulating signals.

Ghoshs GIV-mimicking peptides can take the place of the protein when its missing, keeping the feet apart and calming macrophages down.

We were surprised at just how fluid the immune system is when it encounters a pathogen, said Ghosh, who is also director of the Institute for Network Medicine and executive director of the HUMANOID Center of Research Excellence at UC San Diego School of Medicine. Macrophages dont need to waste time and energy producing more or less GIV protein, they can rapidly dial their response up or down simply by moving it around, and it appears that such regulation happens at the level of gene transcription.

Ghosh and team plan to investigate the factors that determine how the GIV brake remains in place when macrophages are resting or is removed to mount a response to a credible threat. To enable these studies, the Institute for Network Medicine at UC San Diego School of Medicine recently received a new $5 million grant from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. Ghosh shares this award with her colleagues Debashis Sahoo, PhD, assistant professor at UC San Diego School of Medicine and Jacobs School of Engineering, and Soumita Das, PhD, associate professor of pathology at UC San Diego School of Medicine.

Reference: Swanson L, Katkar GD, Tam J, et al.TLR4 signaling and macrophage inflammatory responses are dampened by GIV/Girdin. PNAS. 2020.doi:10.1073/pnas.2011667117

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Protein That Prevents Immune System Overreaction Could Have Therapeutic Applications - Technology Networks

The revolution of genetic testing has led to more accurate and widespread assays for patients with cancer; however, as more genetic variants are identified, it has become a greater challenge to determine the optimal treatment for an individual patient, according to GouthamNarla, MD, PhD.

As we sequence more genes, we will have more information, which is a good thing, said Narla. Of course, we will also find more variants that, at this time, we don't know whether they're pathogenic or benign. They get lumped into the uncertain category, which creates uncertainty for patients and for providers, as well.

In an interview withOncLiveduring the 2020 Institutional Perspectives in Cancer (IPC) webinar on Precision Medicine, Narla, an associate professor in the Department of Medicine; chief of the Division of Genetic Medicine, Department of Medicine; and associate director of the Medical Scientist Training Program, University of Michigan, further discussed the utility of genomic testing and updates in next generation sequencing (NGS).

OncLive: Could you discuss the key advances in cancer genetics? What are some of the mechanisms that have driven its development?

Narla: A couple of major advancements we've seen in cancer genetics is the identification of additional disease-causing variants. It used to be when I first trained as a medical geneticist, we really only knew about BRCA1/2 and some of the mismatch repair genes. Now, we know about other genes, including PALB2, and other members and genes in that family. That has expanded the testing opportunities for our patients.

The other aspect that has been very exciting is now some of these gene variants are predictive of response to therapies. We have therapies that can be specifically used and work for patients who harbor some of these germline variants. That has really changed the way in which we have treated patients who carry these variants.

What are some of the recent developments in NGS?

Previously, we were doing single-gene testing, oftentimes by Sanger sequencing. Now, we can do large panels of genes depending upon the company and the panel; these comprise anywhere from 60 to 70 genesin some cases, several thousand genes. It has allowed us to collect vast amounts of sequencing information. Some of it will not be directly actionable now, but it still fuels research opportunities for us at major academic medical centers, and when more knowledge [is] gained, we go back to some of those sequencing results to see if, in fact, there was something that is now actionable based upon new knowledge.

How are we using this information to develop targeting strategies?

A lot of the approaches that we are using now may not involve the directly targeting the defective gene or protein, but they are leveraging knowledge about how that defective gene or protein causes activation of targetable pathways. For example, when it comes to BRCA1 loss, that creates a unique opportunity to use a PARP inhibitor in a synthetic lethal interaction, where those cells become highly dependent upon that enzyme. Then, you can inhibit with small molecules [or perhaps] approved PARP inhibitors, such as olaparib (Lynparza), and others for which there are now [a number of approved drugs that can target] a range of BRCA-deficient metastatic tumors.

How else has genomic testing evolved?

The evolution has been both in the number of individuals that we test, as well as how many genes we test. [For example, we used to] test families in which there are numbers of individuals who have cancer and we had a strong pretest probability that they would have a germline variant. Now, in fact, every patient with metastatic ovarian cancer, regardless of family history, gets tested. This is because we have PARP inhibitors for them. It not only has implications for their family but it also has implications for their treatment choices.

What guidelines have been helpful to your practice as it relates to genomic testing?

There are a number of organizations from the American Cancer Society to National Cancer Institute and the National Comprehensive Cancer Network (NCCN) that have very robust guidelines on who to test. There is also a little bit of subjectivity in making an appraisal with a genetics professional, meaning a genetic counselor or a medical geneticist, because not every family will fit the structure or will even know the entirety of their family history. There is some nuance to this, but there are definitely very established guidelines that exist and that we use when making these types of decisions.

However, the NCCN guidelines are very good and are used by [our institution. Then we apply our own nuances when we see the patient on a case by case basis. But, [in terms of] informing who should be tested and who should not, and which individual in the family should be [tested], the NCCN guidelines are a very good [resource].

What challenges could be addressed with future research?

I would like to see more of an effort to share data across all institutions and testing companies to reclassify these variants. I would like to see more basic science and translational science around what we call variant reclassification, so that we can really make definitive calls about the sequence changes that we see. The more genes we sequence, the more variants we find, and on larger panels, [we can see these uncertain variants in up to] 20% of patients. We're finding something in a gene, but we don't know whether it's good or bad for the patient.

Are there any new capabilities or technologies emerging that you find particularly exciting?

From a technology perspective, the last 10 years in sequencing has been a revolution; the cost of sequencing has come down and the accuracy has gone up. I'm not sure that we're going to see that much more of a revolution in the sequencing technology; it will be more efficient and more cost effective. We're [going to see] the identification of new genes associated with disease [and will therefore] it will be in the variant reclassification space.

What testing or sequencing studies are of particular interest?

One type of study that has read-out recently comprise the effectiveness of immunotherapy in patients who have mismatch repair deficient tumors. That has been really game-changing for those patients. The other major study is the use of PARP inhibitors in BRCA-mutant tumorsoriginally in the second- and third-line settings of ovarian cancer. [PARP inhibitors] have now moved to maintenance [therapy], pancreatic cancer, prostate cancer, and others. That has changed the management of patients with BRCA-positive tumors.

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Identifying Genetic Variants, Matching With Targeted Therapies Serve as Next Great Challenge With Germline Testing in Oncology - OncLive

Its standard for drug companies to withhold details of clinical trials until after they are completed, tenaciously guarding their intellectual property and competitive edge. But these are extraordinary times, and now there is a growing outcry among independent scientists and public health experts who are pushing the companies to be far more open with the public in the midst of a pandemic that has already killed more than 193,000 people in the United States.

These experts sayAmerican taxpayers are entitled to know moresince the federal government has committed billions of dollars to vaccine research and to buying the vaccines once theyre approved. And greater transparency could also help bolster faltering public confidence in vaccines.

[September 8],nine pharmaceutical companies, including AstraZeneca and Pfizer,pledged to stand with science and rigorously vet any vaccine for the coronavirus an unusual pact among competitors. But the researchers said that missing from the joint statement was a promise to share more critical details about their research with the public and the scientific community.

Weve never had such an important clinical trial or series of clinical trials in recent history, said Dr. Eric Topol, a professor of molecular medicine at Scripps Research in La Jolla, Calif., and a longtime expert on clinical trials. Everything should be transparent.

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Are vaccines now in development safe? The companies making them uniformly won't release data - Genetic Literacy Project

Commenting on the Groups sales in the first nine months, Roche CEO Severin Schwan said: Roche is at the forefront of the fight against COVID-19 with a growing portfolio of diagnostics solutions, the development of new medicines and a number of partnerships across the industry. With the recent launch of the rapid antigen test, we further strengthened our position as a leading supplier of COVID-19 tests. At the same time, we continue to deliver solutions for patients suffering from other severe diseases. I am particularly pleased about the FDA approvals in the third quarter for three new medicines: Enspryng and Evrysdi for rare diseases, and the cancer medicine Gavreto. After the pandemic-related decline in the second quarter, sales stabilised in the third quarter due to continued strong demand for our new medicines and COVID-19 tests. Based on our current assessment, we confirm the outlook for the full-year.

Roches contributions to the fight against the COVID-19 pandemic in the third quarter:

Outlook confirmed for 2020Based on the current assessment of the COVID-19 impact, sales are expected to grow in the low- to mid-single digit range, at constant exchange rates. Core earnings per share are targeted to grow broadly in line with sales, at constant exchange rates. Roche expects to increase its dividend in Swiss francs further.

Group salesIn the first nine months of 2020, Group sales increased 1% to CHF 44.0 billion.

Sales in the Pharmaceuticals Division decreased 1% to CHF 34.3 billion. Sales grew strongly in the first quarter (+7%). As a result of COVID-19, they decreased in the second quarter (-6%) and since summer first signals of recovery are seen (-4% in the third quarter). Key growth drivers were the cancer medicine Tecentriq, the multiple sclerosis medicine Ocrevus, the haemophilia medicine Hemlibra, Actemra/RoActemra in immunology and Perjeta in breast cancer.

With a strong growth of 35% the new medicines generated sales of CHF 13.7 billion and grew by CHF 3.7 billion at constant exchange rates over 2019, more than offsetting the impact of the competition from biosimilars (sales reduction CHF 3.5 billion at constant exchange rates).

In the US, overall sales decreased 4%. While sales of Ocrevus, Hemlibra, Tecentriq, Actemra/RoActemra and Kadcyla increased, the competition from biosimilars for Herceptin, MabThera/Rituxan and Avastin affected total growth as expected. Ocrevus sales increased by 23% and were driven by both new and returning patient demand but partly impacted by COVID-19 effects. Hemlibra sales increased 68%, resulting from the ongoing rollout in the US. Tecentriq sales increased by 46%, driven by the launch in unresectable hepatocellular carcinoma (HCC) as well as the growth in the new indications extensive stage small cell lung cancer (ES-SCLC) and metastatic triple-negative breast cancer.

In Europe, sales increased 4% as the strong demand for Tecentriq, Ocrevus, Hemlibra, Kadcyla and Perjeta was able to offset the impact of lower sales of Herceptin (-32%), Avastin (-16%) and MabThera/Rituxan (-32%). The first biosimilar versions of Avastin were introduced in Europe in the third quarter of 2020.

In the International region (+6%), growth was mostly driven by Perjeta, Actemra/RoActemra, Alecensa, Tecentriq and Ocrevus, partially offset by the impact of the National Reimbursement Drug List update in China and COVID-19.

Sales decreased in Japan (-6%) as a result of the considerable competition from biosimilars, generics, COVID-19 and government price cuts. This decline was partially compensated by recently launched products including Tecentriq and Hemlibra.

The Diagnostics Division recorded very strong sales growth of 9% to CHF 9.7 billion, with particularly strong growth of 18% in the third quarter. After a 5% increase in the first quarter, momentum slowed to 2% growth in the second quarter as a result of the pandemic. The overall very strong sales growth is primarily due to the industry-leading portfolio of new COVID-19 tests. The Molecular Diagnostics business made the largest contribution (+77%) with PCR tests for COVID-19. Sales of diagnostic solutions for SARS-CoV-2 developed only this year clearly exceeded COVID-19 related declines in routine diagnostics sales. Additional product launches in the third quarter, including the SARS-CoV-2 antigen rapid test, further strengthened Roche's position as the world's leading supplier of COVID-19 tests.

Growth was reported in North America (+22%), EMEA3 (+9%), Latin America (+12%) and Japan (+5%). In the Asia-Pacific region (-4%), sales were heavily impacted by the pandemic, especially in China. Overall, demand was impacted by COVID-19 in all regions since the second quarter. Routine testing decreased significantly due to a decline in regular health checks while emergency and SARS-CoV-2 testing increased significantly.

Roches contributions to the fight against the COVID-19 pandemicIn September, the Elecsys Anti-SARS-CoV-2 S antibody test was launched for markets accepting the CE Mark. Roche has filed for Emergency Use Authorisation (EUA) from the FDA. The Elecsys Anti-SARS-CoV-2 S immunology test, which targets antibodies against the spike protein, can be used to quantitatively measure antibodies in people who have been exposed to SARS-CoV-2 and can play an important part in characterising a vaccine-induced immune response. The majority of current candidate vaccines aim to induce an antibody response against the spike protein of the virus.

In the same month, Roche received an EUA from the FDA for its cobas SARS-CoV-2 & Influenza A/B test for use on the cobas 6800/8800 Systems. This test is intended for the simultaneous qualitative detection and differentiation of SARS-CoV-2, influenza A and influenza B in patients suspected by their healthcare provider of having a respiratory viral infection; it is also available in markets accepting the CE Mark.

For urgent and emergency care settings, Roche also received EUA from the FDA in September for the cobas SARS CoV-2 & Influenza A/B test on the cobas Liat System, which provides results in 20 minutes.

Roche also launched the SARS-CoV-2 rapid antigen test in markets accepting the CE Mark and plans to submit files for an EUA with the FDA. The SARS-CoV-2 rapid antigen test is for use in point of care settings for symptomatic people. This can help healthcare professionals identify a SARS-CoV-2 infection in people suspected of carrying the virus with results typically ready in 15 minutes. In addition, it serves as a valuable initial screening test for individuals who have been exposed to SARS-CoV-2-infected patients or in a high-risk environment.

Roche also announced that it intends to launch a high-volume SARS-CoV-2 Antigen test as an aid in the diagnosis of SARS-CoV-2 infection. The test is planned to be made available at the end of 2020 for markets accepting the CE Mark. Roche also intends to file for EUA from the FDA. The test is performed by healthcare professionals and uses swab samples from patients with signs and symptoms suggestive of COVID-19, or people with either known or suspected exposure to SARS-CoV-2.

The portfolio of our recently developed SARS-CoV-2 tests as well as our existing diagnostics menu for critical care have become a significant factor in supporting patient management during the COVID-19 pandemic. Roche has already increased its overall production of tests fourfold over the usual volumes and has committed significant funds to continue expanding productions capacity for PCR tests over the coming year. Our investments are expected to result in more than 1,000 new jobs in the US and Europe.

The phase III Empacta study met its primary endpoint, showing that patients with COVID-19 associated pneumonia who received Actemra/RoActemra plus standard of care were 44% less likely to progress to mechanical ventilation or death compared to patients who received placebo plus standard of care. The cumulative proportion of patients who progressed to mechanical ventilation or death by day 28 was 12.2% in the Actemra/RoActemra arm versus 19.3% in the placebo arm. There was no statistical difference in mortality between patients who received Actemra/RoActemra or placebo by day 28. Approximately 85% of the 389 patients were from minority racial and ethnic groups. The trial was conducted in Brazil, Kenya, Mexico, Peru, South Africa and the USA.

In August, Roche and Regeneron joined forces in the fight against COVID-19 to develop, manufacture and distribute REGN-COV2, Regenerons investigational antiviral antibody cocktail. Regeneron has submitted a request to the FDA for an Emergency Use Authorization (EUA) for REGN-COV2. Initial data from the REGN-COV2 phase II portion of an ongoing study showed a reduction in viral load, an acceleration of symptom alleviation and a decrease in medical visits in non-hospitalised patients with COVID-19. Additional data from this study are expected by the end of 2020. We will be working with health authorities and global health institutions in a concerted, collective response, with the aim of achieving broad approvals.

Drug launches, filings, pivotal phase III trial readouts and pivotal trial starts planned by the Roche Group are largely on track.

The Covacta study of Actemra/RoActemra did not meet its primary endpoint of improved clinical status in hospitalised adult patients with severe COVID-19-associated pneumonia. In addition, the key secondary endpoints, which included the difference in patient mortality at week four, were not met; however, there was a positive trend in time to hospital discharge in patients treated with Actemra/RoActemra. This study generated robust information which will help physicians make decisions about the treatment of patients with this disease.

Overview of Roche Diagnostics COVID-19 diagnostic solutions developed in the first nine months of 2020

Regulatory achievements in the third quarterRegulators around the globe granted approvals for new Roche medicines, line extensions of existing medicines and new tests.

The FDA approved Evrysdi (risdiplam), an oral medication for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity, including types 1, 2, and 3 SMA. Evrysdi also improved survival without permanent ventilation at 12 and 23 months of treatment, compared to natural history.

FDA approval was granted for Enspryng (satralizumab-mwge) as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, lifelong and debilitating autoimmune disorder of the central nervous system, often misdiagnosed as multiple sclerosis, that primarily damages the optic nerve(s) and spinal cord, causing blindness, muscle weakness and paralysis. Enspryng demonstrated significant reduction in the risk of relapse compared with placebo as a monotherapy and when used concurrently with baseline immunosuppressant therapy.

The FDA also approved Gavreto (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer as detected by an FDA approved test. This indication was approved under the FDAs Accelerated Approval programme, based on data from the phase I/II ARROW study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

The European Commission granted conditional marketing authorisation for Rozlytrek for the treatment of adult and paediatric patients 12 years of age and older with solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior NTRK inhibitor and who have no satisfactory treatment options. The European Commission has also approved Rozlytrek for the treatment of adults with ROS1-positive, advanced non-small cell lung cancer (NSCLC) not previously treated with ROS1 inhibitors.

The FDA also granted approval for Tecentriq plus Cotellic and Zelboraf for the treatment of BRAF V600 mutation-positive advanced melanoma patients.

In Japan, Tecentriq in combination with Avastin, was approved by the Ministry of Health, Labour and Welfare for the treatment of unresectable hepatocellular carcinoma.

The FoundationOne Liquid companion diagnostic test (F1L CDx) received FDA approval. Furthermore, the comprehensive liquid biopsy service received the CE Mark in May. Both regulatory milestones allow for the new test to be commercialised in all markets that recognise the CE Mark and/or the FDA approval. F1L CDx is the most comprehensive pan-tumour liquid biopsy test for all solid tumours, incorporating multiple companion diagnostics. This test supports efforts to improve treatment results by helping oncologists optimise and personalise treatment for their patients with advanced cancer during all lines of therapy, and particularly for those where tissue-based testing is not possible.

Additional regulatory achievements in the third quarter of 2020:

Diagnostics key launches in the third quarter In addition to the new COVID-19 portfolio, Roche received FDA approval for the Ventana HER2 Dual ISH DNA Probe Cocktail assay for the detection of the HER2 biomarker in breast cancer and as a companion diagnostic for Herceptin therapy. HER2 - human epidermal growth factor receptor 2 - is an important biomarker sometimes found in breast cancers. Its detection and inhibition can help healthcare professionals manage this aggressive cancer more effectively. This new assay is designed to be completed within the same day, enabling clinicians to get results back faster than with other common methods of confirmatory testing for HER2. Results can be read using light microscopy, eliminating the need for a specialised fluorescence microscope.

The FDA also authorised the cobas EBV test, the first quantitative in vitro diagnostic test for Epstein-Barr virus (EBV) DNA in the US. The test meets World Health Organization standards for consistent result reporting among laboratories across the US, allowing for results to be easily comparable across hospitals and laboratories. Monitoring of Epstein-Barr virus DNA can help prevent the progression of life-threatening diseases such as cancer in transplant patients.

Roche also received FDA clearance for the BK virus quantitative test on cobas 6800/8800 Systems to support better care for transplant patients. The test provides standardised, high-quality results that can help healthcare professionals better assess the risk of complications caused by the BK virus in transplant patients and identify effective treatment options.

In August, the FDA approved the cobas HIV-1/HIV-2 qualitative test for use on the fully automated cobas 6800/8800 Systems in the US. The test provides healthcare professionals with a single result to confirm HIV diagnosis and differentiate HIV-1 and HIV-2, an important distinction needed to identify appropriate treatment options.

In September, Roche launched the Elecsys HIV Duo immunoassay in the US, following FDA approval in April 2020. Through separate measurement of the HIV p24 antigen (the virus) and anti-HIV antibodies (caused by immune reaction), this test can detect an acute HIV infection earlier than current methods. This approval enables a robust, comprehensive infectious diseases menu on the cobas e 801 system and a significant step towards bringing holistic value to the US market within the area of infectious diseases.

Roche announced FDA approval in September for the expanded use of CINtec PLUS Cytology, the first triage test based on biomarker technology for women whose cervical cancer screening results are positive for high-risk types of human papillomavirus (HPV). Additional information from this test supports clinical decisions on which women will benefit most from immediate follow-up. Laboratories can now use CINtec PLUS Cytology to triage positive results from the cobas HPV Test run on the fully integrated, automated and high-throughput cobas 6800/8800 Systems.

Key development milestones in the third quarter of 2020Regulatory filings and product launches for 2020 as well as pivotal trial read-outs and pivotal starts in 2020 are largely on track. We are making significant efforts to protect all studies with continued support from health authorities, but the ultimate outcome will depend on the length and severity of the pandemic.

Results from the phase III IMpassion031 study, evaluating Tecentriq in combination with chemotherapy (Abraxane, albumin-bound paclitaxel; nab-paclitaxel; followed by doxorubicin and cyclophosphamide) in comparison with placebo plus chemotherapy (including nab-paclitaxel), demonstrated a statistically significant and clinically meaningful improvement in pathological complete response (pCR) for the treatment of people with early TNBC, regardless of PD-L1 expression. The IMpassion031 study is the second positive phase III study from Roche to demonstrate the benefit of Tecentriq in TNBC and the first Tecentriq study to demonstrate a benefit in early TNBC.

The final overall survival (OS) analysis of the phase III IMpassion130 study, evaluating Tecentriq in combination with nab-paclitaxel, compared with placebo plus nab-paclitaxel, as a first-line treatment for patients with metastatic TNBC, was consistent with the first and second interim analyses. There was no significant difference in OS between the treatment groups in the ITT population. Clinically meaningful improvements of 7.5 months in median OS were seen with Tecentriq plus nab-paclitaxel in PD-L1-positive patients.

Detailed results from the phase III Archway study showed that 98.4% of Port Delivery Systems (PDS) patients were able to go six months without needing additional treatment and achieved vision outcomes equivalent to patients receiving monthly ranibizumab eye injections, a current standard of care. This study evaluates the investigational Port Delivery System with ranibizumab for the treatment of neovascular or wet age-related macular degeneration (nAMD), a leading cause of blindness globally.

New two-year data from part 1 of the pivotal Firefish study of Evrysdi in infants aged two to seven months with symptomatic Type 1 SMA showed that infants treated with the therapeutic dose of Evrysdi (17/21) continued to improve and achieve motor milestones.

Pharmaceuticals Division

Key pharmaceutical productsAvastin (-22%). For advanced colorectal, breast, lung, kidney, cervical and ovarian cancer, and relapsed glioblastoma (a type of brain tumour). Sales were impacted by the biosimilar competition in the US, Europe and Japan.

MabThera/Rituxan (-27%). For forms of blood cancer, rheumatoid arthritis and certain types of vasculitis. The sales decline was driven by all regions, due to the launch of biosimilars in the US and most EU markets and in Japan.

Herceptin (-31%). For HER2-positive breast cancer and HER2-positive metastatic gastric cancer. Sales were impacted by biosimilars in the US, Europe and Japan. In the US, the switch to Kadcyla in the adjuvant setting also impacted sales.

Actemra/RoActemra (+33%). For rheumatoid arthritis, forms of juvenile idiopathic arthritis and giant cell arteritis as well as CAR T cell-induced severe or life-threatening cytokine release syndrome. A number of countries included Actemra/RoActemra in their treatment guidelines for severe COVID-19 pneumonia. Actemra/RoActemra is not currently approved for this use; Roche is conducting several phase III clinical studies. The US and the International region were the major contributors to the sales increase.

Xolair (+2%, US only). For chronic idiopathic urticaria and allergic asthma. The sales increase was driven by the demand in both indications. Xolair remains the market leader in the larger allergic asthma indication.

Lucentis (-14%, US only). For eye conditions, including neovascular (wet) age-related macular degeneration, macular oedema following retinal vein occlusion, diabetic macular oedema, and diabetic retinopathy. Sales decreased in all approved indications and were especially affected by the COVID-19 pandemic due to disruptions in hospitals and ophthalmology practices and many patients delaying treatment during restrictions.

Highlights for medicines launched since 2012Ocrevus (first approved in 2017; CHF 3.3 billion, +29%). For the treatment of both the relapsing (RMS) and primary progressive (PPMS) forms of multiple sclerosis (MS). The strong demand for this treatment in both indications has continued, while the COVID-19 pandemic has had a certain negative impact. In the US, growth was driven both by new and returning patients, with a higher proportion of sales coming from returning patients. In Europe and the International region, Ocrevus continues to show strong initial uptake where launched.

Perjeta (first approved in 2012; CHF 2.9 billion, +17%). As therapy for HER2-positive breast cancer. Sales grew strongly in the International region. The increased patient demand for Perjeta for adjuvant early breast cancer therapy supports its continued strong growth.

Tecentriq (first approved in 2016; CHF 2.0 billion, +64%). Approved either alone or in combination with targeted therapies and/or chemotherapies in various forms of NSCLC, in small cell lung cancer (SCLC), certain types of metastatic urothelial cancer, and in PD-L1-positive metastatic TNBC. In the US and several other countries, Tecentriq in combination with Avastin is approved for people with unresectable or metastatic HCC and in the US and two other countries Tecentriq is approved in combination with Cotellic and Zelboraf for the treatment of people with BRAF V600 mutation-positive advanced melanoma. Strong sales growth was reported by all regions, driven mainly by the indications in ES-SCLC and TNBC. Sales in Japan increased due to robust uptake in first-line NSCLC and first-line ES-SCLC.

Hemlibra (first approved in 2017; CHF 1.6 billion, +79%). For treating people with haemophilia A with factor VIII inhibitors. It is also approved to treat people with haemophilia A without factor VIII inhibitors. Hemlibra is the only prophylactic treatment that can be administered subcutaneously and with multiple dosing options (once weekly, once every two weeks or once every four weeks). Sales continued to show a strong uptake in all regions, despite COVID-19 restrictions having some impact on potential new patients.

Kadcyla (first approved in 2013; CHF 1.3 billion, +37%). For treating HER2-positive breast cancer. The increased demand for Kadcyla was driven by its usage in the early breast cancer setting. Sales benefited from the positive read-out from the Katherine study and patients switching to the new standard of treatment.

Esbriet (first approved in 2014; CHF 844 million, +9%). For idiopathic pulmonary fibrosis. Sales continued to expand, driven by growth in the US and Europe.

Alecensa (first approved in 2015; CHF 841 million, +35%). To treat ALK-positive lung cancer. Alecensa showed continued sales growth across all regions.

Gazyva/Gazyvaro (first approved in 2013; CHF 472 million, +27%). For chronic lymphocytic leukaemia (CLL), rituximab-refractory follicular lymphoma and previously untreated advanced follicular lymphoma. Sales increased in all regions.

Polivy (first approved in 2019; CHF 126 million, +>500%). Part of combination therapy for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma.

Xofluza (first approved in 2018; CHF 28 million, +286%). For the treatment of acute, uncomplicated influenza, or flu, in people 12 years of age and older and people with high risk of developing flu-related complications.

Rozlytrek (first approved in 2019; CHF 15 million, +319%). For a specific form of NSCLC and for solid tumours expressing a specific gene fusion. In Japan, Rozlytrek was approved for treatment of ROS1 fusion- positive NSCLC.

Evrysdi (risdiplam, first approved in 2020; CHF 8 million*4). For the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older.

Phesgo (fixed-dose combination of Perjeta and Herceptin with hyaluronidase, first approved in 2020; CHF 7 million*). For the treatment of early and metastatic HER2-positive breast cancer by subcutaneous injection (SC) administered in combination with intravenous chemotherapy.

Enspryng (satralizumab, first approved in 2020; CHF 7 million*). For a rare neurodegenerative disease (neuromyelitis optica spectrum disorder).

Diagnostics Division

In the first nine months 2020, COVID-19 and emergency testing strongly increased while routine testing decreased as a result of continued declining or delayed regular health checks and medical appointments. Nevertheless, Roches broad, diversified test portfolio and its large number of instruments installed worldwide provided for a strong sales growth.

During 2020, Roche has increased its production capacity (reagents and consumables) for COVID-19 testing massively. This includes all our products used in fighting COVID-19 infections.

Centralised and Point of Care Solutions sales declined by 7%, its immunodiagnostics business (-8%) was strongly impacted by the COVID-19 impact on routine testing worldwide. COVID-19 related products such as the Elecsys Anti-SARS-CoV-2 test, Custom Biotech, Elecsys IL-6 test and the SARS-CoV-2 rapid antigen test partly offset the COVID-19 impact.

Sales in Molecular Diagnostics increased 77%, with 88% growth in the underlying molecular business. Growth was driven by virology (predominantly SARS-CoV-2), Quantitative PCR (to detect molecular/genetic targets) and Nucleic Acid Purification (to isolate and purify genetic material), Molecular Diagnostics systems, Molecular Point-of-Care (influenza viruses).

Diabetes Care sales decreased 2%, with the continued contraction of the Blood Glucose Monitoring (BGM) market due to patients switching to Continuous Glucose Monitoring (CGM) systems. The COVID-19 pandemic also had an impact. The decrease was reflected mainly in the EMEA region, notably in Germany, UK and Italy. The positive uptake of digital diabetes management solutions continued: AccuChek SugarView, RocheDiabetes Care Platform and mySugr.

Tissue Diagnostics sales increased 5%, supported by advanced staining, instrument sales and companion diagnostics business. However, overall sales were impacted by the COVID-19 pandemic.

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve peoples lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the worlds largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References[1] Unless otherwise stated, all growth rates in this document are at constant exchange rates (CER: average 2019).[2] Launched since 2012: Erivedge, Perjeta, Kadcyla, Gazyva, Esbriet, Cotellic, Alecensa, Tecentriq, Ocrevus, Hemlibra, Xofluza, Polivy, Rozlytrek, Phesgo, Enspryng, Evrysdi[3] EMEA = Europe, Middle East and Africa[4] recently launched, no growth figures available

Cautionary statement regarding forward-looking statementsThis Annual Report contains certain forward-looking statements. These forward-looking statements may be identified by words such as believes, expects, anticipates, projects, intends, should, seeks, estimates, future or similar expressions or by discussion of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the future from those reflected in forward-looking statements contained in this Annual Report, such as: (1) pricing and product initiatives of competitors; (2) legislative and regulatory developments and economic conditions; (3) delay or inability in obtaining regulatory approvals or bringing products to market; (4) fluctuations in currency exchange rates and general financial market conditions; (5) uncertainties in the discovery, development or marketing of new products or new uses of existing products, including without limitation negative results of clinical trials or research projects, unexpected side effects of pipeline or marketed products; (6) increased government pricing pressures; (7) interruptions in production; (8) loss of or inability to obtain adequate protection for intellectual property rights; (9) litigation; (10) loss of key executives or other employees; and (11) adverse publicity and news coverage.

The statement regarding earnings per share growth is not a profit forecast and should not be interpreted to mean that Roches earnings or earnings per share for 2020 or any subsequent period will necessarily match or exceed the historical published earnings or earnings per share of Roche.Roche Group Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com

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First nine months of the year with 1% sales growth at constant exchange rates, significant impact of COVID-19 pandemic - GlobeNewswire

Oct. 13, 2020 05:00 UTC

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- EdiGene, Inc., which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the successful completion of a RMB 450 million (approximately USD 67 million) Series B financing. 3H Health Investment led the round and other new investors included Sequoia Capital China, Alwin Capital and Kunlun Capital, along with continued support by previous investors, including IDG Capital, Lilly Asia Venture, Huagai Capital and Green Pine Capital Partners. Proceeds from the financing will be used to advance the companys pipeline into clinics and to expand the team.

Minchuan Wang, Ph.D., Partner of 3H Health Investment commented: As one of the most disruptive biomedical technologies, gene editing is rapidly moving toward clinics globally. EdiGene is leading the wave in China, as they have established outstanding research and development capabilities, and more importantly, they have developed an impressive portfolio of proprietary gene editing tools and product candidates. We are pleased to lead the Series B financing, and are excited to join force with EdiGene and other investors to bring life-changing innovative gene-editing therapies to patients in China and globally. We look forward to contributing our clinical, business, and policy resources to further strengthen the companys capabilities, and are confident that EdiGene is well-positioned to be a leader in gene-editing therapeutics.

We have raised approximately RMB 700 million (USD 100 million) in the past two years. We are delighted to add these top tier investors in closing of our Series B financing, and are grateful for the continuous support from the current investors, said Dong Wei, Ph.D.CEO of EdiGene, The round enables us to further scale up and transform our pipeline into clinical-stage, which is also a big step forward in building a globally competitive gene editing company. More importantly, we are closer to realizing our mission of bringing innovative and high-quality gene-editing therapies to patients in need.

We are very pleased to have the support and partnership from our investors, which propels the company to an exciting new stage, said Wensheng Wei, Scientific Founder of EdiGene, Together with the investors, we look forward to translating cutting-edge gene editing technologies into innovative therapies, bringing hope and health to patients and their families.

Founded in 2015, EdiGene has established four gene editing based platforms and is advancing its early stage programs into clinical development for patients with genetic diseases and cancer. The four platforms are ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. In addition, EdiGene has launched GMP manufacturing facility in 2018 in Guangdong Province.

About EdiGene, Inc

EdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with subsidiaries in Guangzhou, China and Cambridge, Massachusetts, USA. More information can be found at http://www.edigene.com.

About 3H Health Investment

3H Health Investment is a dedicated healthcare venture firm. We focus on unmet medical needs and invest in emerging fields of science and medicine to deliver breakthroughs to patients. Leveraging our extensive resources with clinical institutions, industry partners and policy institutes, we build leading innovative healthcare companies together with our partners. Please visit http://www.3hhinvestment.com to learn more about us.

About Sequoia Capital China

The Sequoia Capital team helps daring founders build legendary companies. In partnering with Sequoia Capital, companies benefit from our unmatched community and the lessons weve learned over 48 years. As The Entrepreneurs behind the Entrepreneurs, Sequoia Capital China focuses on four sectors: TMT, healthcare, consumer/service, and industrial technology. Over the past 15 years weve had the privilege of working with approximately 600 companies in China.

About Alwin Capital

Alwin Capital is a leading venture capital in China specializing in early stage life science investments. Driven by theme-focused research in life science, Alwin Capital has developed investment strategies that capture outstanding opportunities for innovative diagnostic and therapeutic developments. We are committed to the long-term investment in life science with the pursuit of the excess investment return based on the scientific insights. Founded in 2015, our management team has decades of experience in healthcare research and medical markets. We have invested in dozens of companies in life science and cultivated a bunch of leading companies in respective segments.

About Kunlun Capital

Kunlun Capital is a venture firm specializes on technological innovative companies investment. Founded in 2015 by Mr. Yahui Zhou, founder of Shenzhen-listed Kunlun Wanwei and NASDAQ-listed Opera, we now manage an investment fund of more than RMB 10 billion.

Our core is consumer value and we focus on technology, product or data-driven innovative business model. We identify the leading companies of different segments and look for founding teams with global potential and local capabilities. Our investment cases include Bellen, Obio Technology, Keya Medical, musical.ly, Dada Group, KK World, pony ai, Dreame, Leyan Technologies, etc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201012005376/en/

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EdiGene Raises Approximately USD 67 Million in Series B Financing to Advance Gene Editing Based Programs into Clinical Stage - BioSpace

Figure:

racial bias, algorithms

The New England Journal of Medicine recently published an article entitled Hidden in Plain SightReconsidering the Use of Race Correction in Clinical Algorithms about race and racism in medicine, specifically looking at algorithms and calculators that include race (or more often, Black v. non-Black) in their inputs. (June 17, 2020; https://bit.ly/3iqGYiT.)

We thought long and hard about how MDCalc (https://www.mdcalc.com/) should respond, and we eventually came up with a patient care-based policy statement that would support our goals: Help clinicians deliver the best quality care to patients and use evidence in their practices. (Read it at http://www.mdcalc.com/race.)

We decided to summarize briefly how race affects the results of an algorithm or score so clinicians can be better informed about the score they're using and make race an optional input when possible, allowing clinicians to opt in or out of including it. We will also specifically draw attention to race when it exists in a calculator in the instructions sections and evidence content to provide clearer, transparent information for our users.

As I looked deeper, I realized the whole thing is really, really messy. You've got:

While I won't cover all the specific examples in our statement, this got me wondering again about our heuristics in medicine. How and why have we decided to see certain trends and correlations and use those but not others? It's like my dog: I've trained her to sit on command, and she knows she's going for a walk when I put on my socks. She somehow also knows when I'm about to give her a bath, and she hides. There must be something I'm doing that she's picked up on as a pattern, but I'm completely oblivious.

Why is it fat fertile female 40 for cholecystitis when there are so many men with it? Or the worst headache of your life for subarachnoid hemorrhage, not sudden-onset headache? Or reheated rice for Bacillus cereus or clindamycin for Clostridioides difficile diarrhea? (Some of these may ring true in your experience, while others don't at all. I reheat my rice all the time and happily risk every bite.)

This is the challenge of our craft, which is part science, part art, part profession, and part trade. The problem is that some of these teachings and mantras are true, while others are stereotypes, some are old wives' tales, and some are based on one doctor's case report disseminated by a medical journal and taken as medical fact. What any doctor has in his head is a uniquely human mix of medical school textbook training, mnemonics, residency training, edicts issued by the attendings we respected and trusted, cases from residency and beyond that went well or poorly, trends and patterns our brains consciously and subconsciously absorb, heuristics and hunches, and probably least of all, evidence-based medicine. Often our brains turn that into a gut feeling, Spidey sense, or gestalt so we can generate a diagnosis and differential in less than a second. Pretty amazing but not without fault. Just like in any data analysis, including our brains, if it's garbage in, it's garbage out.

Take the disease-causing cystic fibrosis (CF) resulting from a mutation in the CFTR protein. CFTR mutations that can cause CF are present in up to one in 25 people of Northern European ancestry. One single mutation is the cause of about half of CF cases, but they have found more than 1500 other mutations in the gene. I definitely think of CF as a Northern European disease, to the extent that I probably don't consider it when I see certain children in the ED who don't look Northern European to me. That's my gestalt brain talking. The factual brain, however, knows that CF can present in people who don't look Northern European and that I've met people with CF who don't fit the stereotype. Yet my factual brain has to work really hard to correct the gestalt brain's error.

It takes a lot of effort to fix our gestalt brains, especially once they have formed. We need more evidence and better evidence in them as foundations in medical school and residency because once they get cemented, they are set. Like cement.

Similarly, we need to move away from race and toward ancestry as much as we can. The promise of cracking the genetic code is decades away, and most patients can't wait that long. Ancestry, for now, might help us while we wait for a bedside genetic decoder test. Race and racism will unfortunately be with us for a very long time, but we can do our part by trying to move research and evidence away from blunt pigeonholes like race.

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Comments? Write to us at emn@lww.com.

Dr. Walkeris an emergency physician at Kaiser San Francisco. He is the developer and co-creator of MDCalc (www.mdcalc.com), a medical calculator for clinical scores, equations, and risk stratifications, which also has an app (http://apps.mdcalc.com/), and The NNT (www.thennt.com), a number-needed-to-treat tool to communicate benefit and harm. Follow him on Twitter@grahamwalker, and read his past columns athttp://bit.ly/EMN-Emergentology.

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Emergentology: We Need to Take Race Out of Algorithms : Emergency Medicine News - LWW Journals

Overview

Genetic testing involves examining your DNA, the chemical database that carries instructions for your body's functions. Genetic testing can reveal changes (mutations) in your genes that may cause illness or disease.

Although genetic testing can provide important information for diagnosing, treating and preventing illness, there are limitations. For example, if you're a healthy person, a positive result from genetic testing doesn't always mean you will develop a disease. On the other hand, in some situations, a negative result doesn't guarantee that you won't have a certain disorder.

Talking to your doctor, a medical geneticist or a genetic counselor about what you will do with the results is an important step in the process of genetic testing.

When genetic testing doesn't lead to a diagnosis but a genetic cause is still suspected, some facilities offer genome sequencing a process for analyzing a sample of DNA taken from your blood.

Everyone has a unique genome, made up of the DNA in all of a person's genes. This complex testing can help identify genetic variants that may relate to your health. This testing is usually limited to just looking at the protein-encoding parts of DNA called the exome.

Genetic testing plays a vital role in determining the risk of developing certain diseases as well as screening and sometimes medical treatment. Different types of genetic testing are done for different reasons:

Generally genetic tests have little physical risk. Blood and cheek swab tests have almost no risk. However, prenatal testing such as amniocentesis or chorionic villus sampling has a small risk of pregnancy loss (miscarriage).

Genetic testing can have emotional, social and financial risks as well. Discuss all risks and benefits of genetic testing with your doctor, a medical geneticist or a genetic counselor before you have a genetic test.

Before you have genetic testing, gather as much information as you can about your family's medical history. Then, talk with your doctor or a genetic counselor about your personal and family medical history to better understand your risk. Ask questions and discuss any concerns about genetic testing at that meeting. Also, talk about your options, depending on the test results.

If you're being tested for a genetic disorder that runs in families, you may want to consider discussing your decision to have genetic testing with your family. Having these conversations before testing can give you a sense of how your family might respond to your test results and how it may affect them.

Not all health insurance policies pay for genetic testing. So, before you have a genetic test, check with your insurance provider to see what will be covered.

In the United States, the federal Genetic Information Nondiscrimination Act of 2008 (GINA) helps prevent health insurers or employers from discriminating against you based on test results. Under GINA, employment discrimination based on genetic risk also is illegal. However, this act does not cover life, long-term care or disability insurance. Most states offer additional protection.

Depending on the type of test, a sample of your blood, skin, amniotic fluid or other tissue will be collected and sent to a lab for analysis.

The amount of time it takes for you to receive your genetic test results depends on the type of test and your health care facility. Talk to your doctor, medical geneticist or genetic counselor before the test about when you can expect the results and have a discussion about them.

If the genetic test result is positive, that means the genetic change that was being tested for was detected. The steps you take after you receive a positive result will depend on the reason you had genetic testing.

If the purpose is to:

Talk to your doctor about what a positive result means for you. In some cases, you can make lifestyle changes that may reduce your risk of developing a disease, even if you have a gene that makes you more susceptible to a disorder. Results may also help you make choices related to treatment, family planning, careers and insurance coverage.

In addition, you may choose to participate in research or registries related to your genetic disorder or condition. These options may help you stay updated with new developments in prevention or treatment.

A negative result means a mutated gene was not detected by the test, which can be reassuring, but it's not a 100 percent guarantee that you don't have the disorder. The accuracy of genetic tests to detect mutated genes varies, depending on the condition being tested for and whether or not the gene mutation was previously identified in a family member.

Even if you don't have the mutated gene, that doesn't necessarily mean you'll never get the disease. For example, the majority of people who develop breast cancer don't have a breast cancer gene (BRCA1 or BRCA2). Also, genetic testing may not be able to detect all genetic defects.

In some cases, a genetic test may not provide helpful information about the gene in question. Everyone has variations in the way genes appear, and often these variations don't affect your health. But sometimes it can be difficult to distinguish between a disease-causing gene and a harmless gene variation. These changes are called variants of uncertain significance. In these situations, follow-up testing or periodic reviews of the gene over time may be necessary.

No matter what the results of your genetic testing, talk with your doctor, medical geneticist or genetic counselor about questions or concerns you may have. This will help you understand what the results mean for you and your family.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

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Genetic testing - Mayo Clinic

Lynparza is the only PARP inhibitor to demonstrate overallsurvival in metastatic castration-resistant prostate cancerFinal results from thePROfoundPhase III trial showed AstraZeneca and MSDsLynparza(olaparib) demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus enzalutamide or abiraterone in men with metastatic castration-resistant prostate cancer (mCRPC) with BRCA1/2 or ATM gene mutations, a subpopulation of homologous recombination repair (HRR) gene mutations.Patients had progressed on prior treatment with new hormonal agent (NHA) treatments (i.e. enzalutamide and abiraterone). Prostate cancer is the second-most common type of cancer in men, with an estimated 1.3 million new patients diagnosed worldwide in 2018.1Approximately 20-30% of men with mCRPC have an HRR gene mutation.2In the key secondary endpoint of OS,Lynparzareduced the risk of death by 31% versus enzalutamide or abiraterone (based on a hazard ratio [HR] of 0.69; 95% confidence interval [CI] 0.50-0.97; p=0.0175). Median OS was 19.1 months forLynparzaversus 14.7 months for enzalutamide or abiraterone, despite 66% of men on NHA treatments had crossed over to receive treatment withLynparzafollowing disease progression.An exploratory analysis also showed a non-statistically significant improvement in OS in the overall trial population of men with HRR gene mutations (BRCA1/2, ATM, CDK12 and 11 other HRRm genes), reducing the risk of death by 21% withLynparzaversus enzalutamide or abiraterone (based on a HR of 0.79; 95% CI 0.61-1.03). Median OS was 17.3 months versus 14.0 months for enzalutamide or abiraterone.Johann de Bono, one of the principal investigators of the PROfound Phase III trial, Head of Drug Development at The Institute for Cancer Research, and The Royal Marsden NHS Foundation Trust, said: Lynparzahas demonstrated significant clinical benefit across key endpoints in PROfound and the final overall survival results reinforce its potential to change the treatment standard for men with metastatic castration-resistant prostate cancer. The PROfound trial shows thatLynparzacan play an important role in this new era of precision medicine in prostate cancer, bringing a targeted therapy at a molecular level to patients with a historically poor prognosis and few treatment options.Jos Baselga, Executive Vice President, Oncology R&D, said: These results help to transform the treatment landscape for certain men with metastatic castration-resistant prostate cancer, where overall survival has been very difficult to achieve.Lynparzais the only PARP inhibitor to demonstrate overall survival versus enzalutamide or abiraterone for men with BRCA or ATM mutations. We look forward to continuing to bringLynparzato these patients around the world.Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: The PROfound trial is the first positive Phase III trial using molecular biomarker testing to help identify treatment options for certain men with metastatic castration-resistant prostate cancer. These results further underpin the importance of genomic testing for HRR gene mutations to identify this at-risk patient population and help physicians make treatment decisions. These results demonstrate the potential ofLynparzafor metastatic castration-resistant prostate cancer patients with certain HRR mutations.Final OS results from the PROfound Phase III trial were presented on Sunday 20 September during the Presidential Symposium at the 2020 European Society of Medical Oncology virtual congress, and published simultaneously inThe New England Journal of Medicine.Summary of OS resultsOS data cut-off date was 20 March, 2020

The most common adverse events (AEs) 20% were anaemia (50%), nausea (43%), fatigue/asthenia (42%), decreased appetite (31%), diarrhoea (21%) and vomiting (20%). The most common grade 3 AEs were anaemia (23%), nausea (2%), fatigue/asthenia (3%), decreased appetite (2%), and diarrhoea (1%). Twenty percent of patients onLynparzadiscontinued treatment due to AEs.ThePROfoundPhase III trialmet its primary endpoint in August 2019, showingLynparzasignificantly improved radiographic progression-free survival (rPFS) in men with BRCA1/2 or ATM genes, and met a key secondary endpoint of rPFS in the overall HRRm population, which formed the basis of theUS approval in May 2020. Regulatory reviews are ongoing in the EU and other countries.AstraZeneca and MSD are exploring additional trials in metastatic prostate cancer including the ongoingPROpelPhase III trial testingLynparzaas a 1st-line medicine for patients with mCRPC in combination with abiraterone versus abiraterone alone. Data are anticipated in 2021.Metastatic castration-resistant prostate cancer (mCRPC)Prostate cancer is associated with a significant mortality rate.1Development of prostate cancer is often driven by male sex hormones called androgens, including testosterone.3In patients with mCRPC, their prostate cancer grows and spreads to other parts of the body despite the use of androgen-deprivation therapy to block the action of male sex hormones.3Approximately 10-20% of men with advanced prostate cancer will develop CRPC within five years, and at least 84% of these men will have metastases at the time of CRPC diagnosis.4Of men with no metastases at CRPC diagnosis, 33% are likely to develop metastases within two years.4Despite advances in treatment for men with mCRPC, five-year survival is low and extending survival remains a key treatment goal.4HRR gene mutationsHRR genes allow for accurate repair of damaged DNA in normal cells.5,6HRR deficiency (HRD) means the DNA damage cannot be repaired, and can result in normal cell death.6This is different in cancer cells, where a mutation in HRR pathways leads to abnormal cell growth and therefore cancer.6HRD is a well-documented target for PARP inhibitors, such asLynparza. PARP inhibitors block a rescue DNA damage repair mechanism by trapping PARP bound to DNA single-strand breaks which leads to replication fork stalling causing their collapse and the generation of DNA double-strand breaks, which in turn lead to cancer cell death.6PROfoundPROfound is a prospective, multicentre, randomized, open-label, Phase III trial testing the efficacy and safety ofLynparzaversus enzalutamide or abiraterone in patients with mCRPC who have progressed on prior treatment with NHA treatments (abiraterone or enzalutamide) and have a qualifying tumour mutation in BRCA1/2, ATM or one of 12 other genes involved in the HRR pathway.The trial was designed to analyse patients with HRRm genes in two cohorts: the primary endpoint was rPFS in those with mutations in BRCA1/2 or ATM genes and then, ifLynparzashowed clinical benefit, a formal analysis was performed of the overall trial population of patients with HRRm genes (BRCA1/2, ATM, CDK12 and 11 other HRRm genes; a key secondary endpoint).LynparzaLynparza(olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in HRR, such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP withLynparzaleads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death.Lynparzais being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.Lynparzais currently approved in a number of countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in the US as a 1st-line maintenance treatment with bevacizumab for patients with homologous recombination deficiency (HRD)-positive advanced ovarian cancer.Lynparzais approved in the US, Japan, and a number of other countries for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. It is also approved in the US and several other countries for the treatment of germline BRCA-mutated metastatic pancreatic cancer.Lynparzais approved in the US for HRR gene-mutated metastatic castration-resistant prostate cancer. Regulatory reviews are underway in several countries for ovarian, breast, pancreatic and prostate cancers.Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 30,000 patients worldwide.Lynparzahas the broadest and most advanced clinical trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types.Lynparzais the foundation of AstraZenecas industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.The AstraZeneca and MSD strategic oncology collaborationIn July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialiseLynparza, the worlds first PARP inhibitor, andKoselugo(selumetinib), a MEK inhibitor, for multiple cancer types. Working together, the companies will developLynparzaandKoselugoin combination with other potential new medicines and as monotherapies. Independently, the companies will developLynparzaandKoselugoin combination with their respective PD-L1 and PD-1 medicines.AstraZeneca in oncologyAstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio ofnew medicines that has the potential to transform patients lives and the Companys future. With seven new medicines launched between 2014 and 2020, and a broad pipelineof small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers.By harnessing the power of four scientific platforms Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.AstraZenecaAstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visitastrazeneca.comand follow the Company on Twitter@AstraZeneca.References1. Bray et al. (2018). Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries.CA: A Cancer Journal for Clinicians, 68(6), pp.394-424.2. Mateo, J, et al (2015). DNA-repair defects and olaparib in metastatic prostate cancer.New England Journal of Medicine, 373(18), pp.1697-1708.3. Cancer.Net. (2019). Treatment of metastatic castration-resistant prostate cancer.www.cancer.net/research-and-advocacy/asco-care-and-treatment-recommendations-patients/treatment-metastatic-castration-resistant-prostate-cancer[Last Accessed: September 2020].4. Kirby, M. (2011). Characterising the castration-resistant prostate cancer population: a systematic review.International Journal of Clinical Practice, 65(11), pp.1180-1192.5. Li et al. (2008). Homologous recombination in DNA repair and DNA damage tolerance.Cell Research, 18(1), pp.99-113.6. Ledermann et al. (2016). Homologous recombination deficiency and ovarian cancer.European Journal of Cancer,60, pp.49-58.

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Lynparza reduced the risk of death by 31% in BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer in PROfound Phase III trial -...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that it presented the latest results from three Phase III studies from the Tecentriq (atezolizumab) clinical development program in triple-negative breast cancer (TNBC) at the European Society for Medical Oncology (ESMO) Virtual Congress 2020.

While we have made great progress in the treatment of many forms of breast cancer, TNBC remains an aggressive and difficult-to-treat disease, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. We are proud of our work to address challenges and advance scientific understanding of cancer immunotherapy in the context of distinct chemotherapy regimens and in various TNBC treatment settings. Although the IMpassion131 study did not reach its endpoint, we are pleased to bring new treatment options for some TNBC patients, and remain committed to improving the lives of all women with early and advanced stages of this disease.

Results from the Phase III IMpassion031 study, evaluating Tecentriq in combination with chemotherapy (Abraxane [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy (including nab-paclitaxel) demonstrated a statistically significant and clinically meaningful improvement in pathological complete response (pCR) for the treatment of people with early TNBC, regardless of PD-L1 expression. pCR was observed in 57.6% (95% CI: 49.7-65.2) of patients treated with Tecentriq in combination with chemotherapy, an increase of 16.5% from 41.1% (95% CI: 33.6-48.9) in patients treated with placebo plus chemotherapy (one-sided p=0.0044, significance boundary = 0.0184) in the intention-to-treat (ITT) population. The safety profile was consistent with the established profile of the individual medicines and no new safety concerns were identified.

The IMpassion031 study is the second positive Phase III study from Genentech demonstrating the benefit of Tecentriq in TNBC, and the first Tecentriq study to demonstrate benefit in early TNBC. Tecentriq in combination with nab-paclitaxel is currently approved in more than 70 countries worldwide, including the United States and across Europe, for the treatment of adults with unresectable locally advanced or metastatic TNBC in people whose tumors express PD-L1 (IC1%).

The final overall survival (OS) analysis of the Phase III IMpassion130 study, evaluating Tecentriq in combination with nab-paclitaxel compared with placebo plus nab-paclitaxel as a first-line treatment for patients with metastatic TNBC, was consistent with the first and second interim analyses. There was no significant difference in OS between the treatment groups in the ITT population. Clinically meaningful improvements in OS were seen with Tecentriq plus nab-paclitaxel in PD-L1-positive patients. The magnitude of OS improvements with Tecentriq in PD-L1-positive patients remained clinically meaningful, with an increase of 7.5 months in median OS with Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel (hazard ratio [HR]=0.67 [95% CI: 0.530.86]). However, this result could not be formally tested due to the prespecified statistical testing hierarchy. The cumulative safety of the Tecentriq plus nab-paclitaxel combination remains consistent with the previously reported safety data for this study and the known risks of individual study medicines. No new safety concerns were identified with longer follow-up.

Finally, results from the Phase III IMpassion131 study, evaluating Tecentriq in combination with paclitaxel compared with placebo plus paclitaxel as a first-line treatment for patients with metastatic TNBC, did not show significant improvement for progression-free survival (PFS) in the PD-L1-positive population (HR=0.82 [95% CI: 0.60-1.12]). The OS data showed a negative trend; however, the study was not powered for the secondary endpoint of OS, and OS data were immature at the time of analysis (initial HR=1.55 [95% CI: 0.86-2.80] in the PD-L1-positive population, based on 21% of patients with an event; updated HR=1.12 [95% CI: 0.76-1.65], updated analysis based on 41% of patients with an event). The safety profile of Tecentriq plus paclitaxel was consistent with the established safety profile of the individual study medicines and no new safety concerns were identified.

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies across several types of lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMpassion031 study

The IMpassion031 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy, in people with previously untreated, early TNBC. The primary endpoint is pCR using the American Joint Committee on Cancer (AJCC) staging system in the intention-to-treat (ITT) population and in the PD-L1-positive population. Secondary endpoints include overall survival (OS), event-free survival, disease-free survival and quality of life measures.

About the IMpassion130 study

The IMpassion130 study is a Phase III, multicenter, randomized, double-blind study evaluating the efficacy, safety and pharmacokinetics of Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel in people with unresectable locally advanced or metastatic TNBC who have not received prior systemic therapy for metastatic breast cancer. The study enrolled 902 people who were randomized equally (1:1). The co-primary endpoints are PFS per investigator assessment (RECIST 1.1), and OS in the ITT population and PD-L1-positive population. Secondary endpoints include objective response rate and duration of response.

About the IMpassion131 study

The IMpassion131 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq in combination with paclitaxel, in comparison to placebo plus paclitaxel, in people with previously untreated, inoperable, locally advanced or metastatic TNBC. The study enrolled 651 people who were randomized in a 2:1 ratio to receive Tecentriq or placebo plus paclitaxel. The primary endpoint is PFS per investigator assessment (RECIST 1.1) in the PD-L1-positive population, followed by intention-to-treat (ITT) population. Secondary endpoints include OS, objective response rate, and duration of response in the PD-L1-positive and ITT populations.

About triple-negative breast cancer

Breast cancer is the most common cancer among women worldwide. According to the American Cancer Society, close to 280,000 people in the United States will be diagnosed with invasive breast cancer, and more than 42,000 will die from the disease in 2020. Breast cancer is not one, but many diseases based on the biology of each tumor. In triple-negative breast cancer, tumor cells lack hormone receptors and do not have excess HER2 protein. Approximately 15 percent of breast cancers are triple-negative based on the results of diagnostic tests. It is an aggressive form of the disease with few treatment options.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indications (pronounced t-SEN-trik)

Tecentriq is a prescription medicine used to treat adults with:

A type of bladder and urinary tract cancer called urothelial carcinoma.

Tecentriq may be used in patients with urothelial carcinoma if their bladder cancer has spread or cannot be removed by surgery, and if they have any one of the following conditions:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

Tecentriq may be used alone as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines bevacizumab, paclitaxel, and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines paclitaxel protein-bound and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used alone in patients with lung cancer if:

A type of breast cancer called triple-negative breast cancer (TNBC).

Tecentriq may be used with the medicine paclitaxel protein-bound in patients with TNBC when their breast cancer:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called small cell lung cancer (SCLC).

A type of liver cancer called hepatocellular carcinoma (HCC).

Tecentriq may be used with the medicine bevacizumab when a patients liver cancer:

A type of skin cancer called melanoma.

Tecentriq may be used with the medicines cobimetinib and vemurafenib when a patients melanoma:

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

The most important information about Tecentriq is:

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life-threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

The most common side effects of Tecentriq when used in TNBC with paclitaxel protein-bound include:

The most common side effects of Tecentriq when used in hepatocellular carcinoma with bevacizumab include:

The most common side effects of Tecentriq when used in melanoma with cobimetinib and vemurafenib include:

Tecentriq may cause fertility problems in females, which may affect their ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects of Tecentriq.

Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Please visit http://www.Tecentriq.com for the full Tecentriq Prescribing Information for additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentechs approved PD-L1 checkpoint inhibitor, the companys broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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Genentech Presents New Data From Multiple Phase III Studies of Tecentriq in Triple-Negative Breast Cancer at ESMO Virtual Congress 2020 - Business...

CHICAGO, May 25, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this global regenerative medicine market report.

The regenerative medicine market is expected to grow at a CAGR of over 30% during the period 20202026.

Key Highlights Offered in the Report:

Key Offerings:

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Regenerative Medicine Market Segmentation

Regenerative Medicine Market by Application

Regenerative Medicine Market by Product

Regenerative Medicine Market by End-Users

Regenerative Medicine Market Dynamics

Regenerative medicine is expected to evolve and impact the overall healthcare industry in a positive way in the coming years. Among the global pharmaceutical companies, nearly 1000 companies are working on gene therapy, cell therapy, and tissue-engineering therapeutic products. Many companies worldwide have been developing a wide array of scaffolds that can be used in different tissue engineering applications, which cater to patients who require tissue and organ substitutes. The advances in scaffolds are attributable to several innovations in tissue scaffolds, bone scaffolds, and dental scaffolds. Tissue scaffolds basically act by integrating local cells in the desired shape of the scaffold after implantation. The scaffolds are of different types, such as cellusponge scaffolds in which cells are distributed in sponge pores and start growing. Collagen scaffolds have a unique porous network that allows diffusion of nutrients for cell growth, while hydrogel scaffolds have water content similar to natural tissue. Nanofiber scaffolds are transparent and ease cell imaging and quantification of cells.

Key Drivers and Trends fueling Market Growth:

Regenerative Medicine Market Geography

In 2020, North America accounted for a share of over 62% in the global regenerative medicine market. The region is expected to grow at a significant rate during the forecast period due to the highest number of RM companies in the world. The region has nearly 534 of the 987 RM companies worldwide. The growth is primarily attributable to the increasing incidence rates of different types of cancers such as non-Hodgkin lymphoma, Hodgkin lymphoma, melanoma of the skin, leukemia, and rare disorders, including Spinal muscular atrophy and multiple sclerosis. Cancer is the leading cause of death in North America. In 2018, nearly 1.9 million new cancer cases were reported in the North American region, along with 693,000 deaths. In the North American region, the US shows the highest prevalence rate for cancers such as non-Hodgkin lymphoma and other life-threatening rare diseases.

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Regenerative Medicine Market by Geography

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AriztonAdvisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

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Regenerative Medicine Market Size to Reach Revenues of over USD 27 Billion by 2026 - Arizton - PRNewswire

(UroToday.com)Dr. Juergen Gschwend summarized the three important abstracts in the setting of metastatic urothelial carcinoma (mUC) that were presented at the European Society of Medical Oncology (ESMO) 2020 Virtual Congress:

Dr. Gschwend began his talk stating that mUC is not a curable disease in most cases, and patients rarely survive beyond five years. Treatment of this disease is associated with a high therapy burden and has significant treatment-related adverse events (TRAEs) with increasing costs.

The evolution of the treatment options is shown in Figure 1, showing many new treatment options that are becoming available.

IMvigor130 was the first abstract discussed (figure 2). This was a positive randomized controlled trial, meeting the primary endpoint of progression-free survival (PFS), showing a . The analysis of overall survival (OS) is still pending. Atezolizumab + platinum-based chemotherapy was well-tolerated with no relevant toxicity added resulting from the addition of atezolizumab to chemotherapy.

IMvigor130 also provided an analysis of quality of life and patient-reported outcomes (PROs), which, according to Dr. Gschwend, provide an overall benefit. There was no significant difference in function and quality of life domains in both treatment arms. The PROs were not compromised by the addition of atezolizumab to the standard platinum-based chemotherapy.

Figure 1 Evolution of systemic treatment options in metastatic urothelial carcinoma:

Figure 2 Imvigor 130 trial NCT02807636 design:

Next, Dr. Gschwend discussed the JAVELIN bladder 100 phase three trial comparing the best standard of care (BSC) to Avelumab maintenance + BSC in mUC patients previously treated with platinum-based chemotherapy (figure 3).

Figure 3 JAVELIN bladder 100 trial design:

Dr. Gschwend continued and congratulated the authors of this study due to the very impressive biomarker analyses that were conducted in this study. However, it is still not clear if the work done with the suggested biomarkers will influence clinical decision making. The study shows that the impact of PD-L1 in the JAVELIN bladder 100 trial is questionable because both treatment arms did better in patients with positive PD-L1.

When assessing the impact of the tumor mutational burden (TMB) as a potential biomarker, it showed no clear prediction in this trial. In a similar manner, there was no clear prediction by either TMB or PD-L1 in the IMvigor 130 study. The other biomarkers that were identified and analyzed in the trial, including gene expression signatures, were shown to be potentially predictive, but they were more hypothesis-generating than clinically relevant. None of these potential biomarkers were shown to be predictive in the outcome of overall survival (OS) in the JAVELIN bladder 100 trial and IMvigor 130 trial.

An important limitation to remember is that the backbone of a biomarker test is the tumor tissue itself. However, it is not clear which tissue should be utilized whether tissue should be collected from the primary tumor or from a metastasis. The timing of tissue sampling is also not clear, and tumor heterogeneity within the same tumor also raises concern.

The last abstract discussed was the TROPHY-U-01 trial (figure 4), assessing the role of Sacituzumab Govitecan (SC), which is an anti-Trop-2 antibody-drug conjugate. This trial was compared to the EV-201 trial assessing the role of enfortumab Vedotin (EV), another antibody-drug conjugate, in the same patient population1. Both trials had similar results, as shown in figure 5.

The TROPHY-U-01 showed that SC has promising activity with an objective response rate of 27% compared to 44% for EV. There was a high median OS with 10.5 months for SG and 11.7 months for EV. There are currently ongoing phase three trials assessing the role of SG.

Figure 4 TROPHY-U-01 trial NCT03547973design:

Figure 5 Comparison of results from the TROPHY-U-01 trial and the EV-201 trial:

Dr. Gschwend ended his talk showing the currently available options for first-line treatment of mUC patients in 2020 and beyond (Figure 6).

Figure 6 First-line treatment options for metastatic urothelial carcinoma in 2020 and beyond:

Presented by: Prof. Dr. Jrgen Gschwend, PhD, Department of Urology, TUM School of Medicine, Munich, Germany

Written by: Hanan Goldberg, MD, MSc., Assistant Professor of Urology, SUNY Upstate Medical University, Syracuse, NY, USA, @GoldbergHananat the European Society for Medical Oncology Virtual Congress, ESMO Virtual Congress 2020 #ESMO20, 18 Sept - 21 Sept 2020.

References:

1. Rosenberg JE, O'Donnell PH, Balar AV, et al. Pivotal Trial of Enfortumab Vedotin in Urothelial Carcinoma After Platinum and Anti-Programmed Death 1/Programmed Death Ligand 1 Therapy. Journal of clinical oncology : official journal of the American Society of Clinical Oncology 2019; 37(29): 2592-600.

Related Content:ASCO 2020: JAVELIN Bladder 100 Phase III Results: Maintenance Avelumab + Best Supportive Case vs BSC Alone After Platinum-Based First-Line Chemotherapy in Advanced Urothelial CarcinomaJAVELIN Bladder 100: Avelumab for Previously Untreated Locally Advanced or Metastatic Urothelial Carcinoma - Thomas PowlesThe Dynamic Advancements in Personalized Treatments for Metastatic Urothelial Cancer - Andrea Apolo

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ESMO Virtual Congress 2020: Invited Discussant LBA24, 698O, 699O The Landscape is Moving in Metastatic Urothelial Carcinoma - UroToday

Gene therapy is a promising new method for treating non-small cell lung cancer (NSCLC). It allows doctors to target specific genes to prevent cancerous cells from growing and spreading.

NSCLC is a common form of cancer. It causes cancerous cells to form in the tissues of the lung. NSCLC is a serious condition. However, many people receive treatment and survive for years.

Treating NSCLC typically involves interacting with multiple specialists and receiving a combination of therapies. Specific treatment plans depend on factors that include the tumor size, type of NSCLC, and the extent of its spread to other organs.

Surgery, radiation or chemotherapy, and immunotherapy are examples of key techniques that doctors use to treat NSCLC.

Gene therapy is another promising treatment for NSCLC, which targets genes that contribute to the tumor.

There are two main approaches to using gene therapy to treat cancer:

This article focuses on the second approach to NSCLC gene therapy. Read on to learn more.

Getting genes into cells requires making vectors, which are vehicles that scientists engineer to deliver genetic materials. For example, viruses have a natural ability to deliver genetic material into cells and can act as vectors.

Scientists can deactivate parts of the virus that cause infectious diseases. They can then modify the virus to carry genetic material into cancerous cells.

One type of gene therapy for NSCLC targets tumor-suppressor genes, which are the most common gene mutation that contributes to the disease. Another approach involves restoring specific proteins to prevent disease progression.

Other possible applications include inserting genes that:

NSCLC gene therapy is a new form of treatment. However, early results are promising.

A 2017 review suggests that restoring a functional tumor-suppressing gene could slow the growth of cancer cells. Clinical trials have found that inserting tumor-suppressing genes into people who had not responded to other treatments reduced tumor size by up to 50%.

Another review in 2016 suggests that the treatment is more effective when combining NSCLC gene therapy with other therapies, such as chemotherapy or immunotherapy.

According to the American Cancer Society, doctors typically use gene therapy for advanced cancer cases.

NSCLC gene therapy is a new technique. However, it still has to meet rigorous Food and Drug Administration (FDA) standards for safety and effectiveness before a doctor can recommend it.

Gene therapies that the FDA approves are safe. However, they can have side effects, such as:

According to the FDA, gene therapies can transform medicine and provide options for people with illnesses that were previously without a cure. However, every treatment has limitations to its effectiveness.

Some limitations to gene therapy include:

Doctors will typically develop a treatment plan with people who have NSCLC depending on their health, age, and other relevant factors. Some common forms of NSCLC treatment include:

Doctors may combine these treatments to maximize their effectiveness. This will involve undergoing multiple treatments at once or back-to-back treatments, or both.

For example, doctors may use a therapy to treat cancer in one part of the body and another therapy to treat where it is spreading.

Doctors typically describe the outlook for people with cancer using the percentage of people alive at least 5 years after their diagnosis. This is the 5-year survival rate. They may further break down 5-year survival rates according to specific NSCLC diagnoses.

According to the American Cancer Society, the 5-year survival rate for people with NSCLC are:

NSCLC is a common form of lung cancer in the United States. Gene therapy for people with NSCLC is a promising new treatment that targets specific genes that contribute to disease progression. There is evidence that gene therapy can slow the growth of tumors in people with NSCLC.

Gene therapy is new, but has the potential to change the way doctors can treat cancer. Scientists and doctors must first overcome limitations, including finding reliable methods to deliver gene therapy.

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NSCLC gene therapy: Success rate, other options, and more - Medical News Today

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran. The two secondary endpoints were changes in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and gait speed assessed by the timed 10-meter walk test (10-MWT) compared to historical placebo. Vutrisiran met the primary endpoint (p less than 0.001) and achieved statistically significant results (p less than 0.001) for each of the Norfolk QoL-DN and 10-MWT secondary endpoints. In addition, vutrisiran treatment showed improvement compared to placebo on the exploratory cardiac biomarker endpoint, NT-proBNP (nominal p less than 0.05). Vutrisiran also demonstrated an encouraging safety and tolerability profile.

Based on these positive results, the Company plans to submit a New Drug Application (NDA) for vutrisiran with the U.S. Food and Drug Administration (FDA) in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan. The Company plans to submit a Marketing Authorisation Application (MAA) in the EU upon obtaining the results of the 18-month analysis expected in late 2021 as previously aligned with the European Medicines Agency (EMA).

We are excited to report positive topline results from the HELIOS-A study, which show that vutrisiran reduces neurologic impairment and improves quality of life in patients with hATTR amyloidosis with polyneuropathy as soon as 9 months, with an encouraging safety and tolerability profile. In addition, were very pleased to see evidence for reversal of polyneuropathy manifestations of disease and also favorable effects on the exploratory cardiac endpoint, NT-proBNP. We believe that vutrisiran, as a low-dose, once-quarterly, subcutaneously administered therapy, has the potential to be a highly attractive therapeutic option for patients living with this progressive, life-threatening, multi-system disease. We look forward to presenting the full 9-month results from HELIOS-A at a medical meeting in early 2021 and to announcing additional 18-month results, including additional exploratory cardiac endpoint data, in late 2021, said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. We would like to recognize and extend our profound gratitude to the patients, caregivers, investigators, and study staff who are participating in HELIOS-A and who, through their commitment during an especially difficult year, have helped make possible another potential advancement in the treatment of hATTR amyloidosis with polyneuropathy. We look forward to initiating our regulatory filings in early 2021 as we work to bring this investigational treatment one step closer to patients with this rare disease.

HELIOS-A (NCT03759379) is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran. The study enrolled 164 patients with hATTR amyloidosis with polyneuropathy at 57 sites in 22 countries. Patients were randomized 3:1 to receive either 25mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (as a reference comparator) for 18 months. The primary endpoint is the change from baseline in mNIS+7 score at 9 months1, relative to historical placebo. Secondary endpoints at 9 months are the change from baseline in the Norfolk QoL-DN score and the timed 10-MWT, relative to historical placebo. Changes from baseline in NT-proBNP were evaluated as an exploratory endpoint at 9 months. The efficacy results of vutrisiran in HELIOS-A are compared to historical placebo control data from the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in a patient population similar to that studied in HELIOS-A. Additional secondary endpoints at 18 months will be evaluated in the HELIOS-A study, including change from baseline in mNIS+7, Norfolk QoL-DN, 10-MWT, modified body mass index (mBMI), Rasch-built Overall Disability Scale (R-ODS), and serum transthyretin (TTR) levels. Additional exploratory cardiac endpoint data at the 18-month time point will be evaluated, including NT-proBNP, echocardiographic measures and cardiac amyloid assessments with technetium scintigraphy imaging. Following the 18-month study period, all patients are eligible to receive vutrisiran for an additional 18 months as part of an open-label extension study. Full 9-month results will be presented at a medical conference in early 2021 and topline 18-month results, including further exploratory cardiac endpoint data, are expected to be announced in late 2021.

Vutrisiran demonstrated an encouraging safety profile. There were two study discontinuations (1.6 percent) due to adverse events in the vutrisiran arm by Month 9, both due to deaths, neither of which was considered related to study drug. There were two serious adverse events (SAEs) deemed related to vutrisiran by the study investigator, consisting of dyslipidemia and urinary tract infection. Treatment emergent adverse events (AEs) occurring in 10 percent or more patients included diarrhea, pain in extremity, fall and urinary tract infections, with each of these events occurring at a similar or lower rate as compared with historical placebo. Injection site reactions (ISRs) were reported in five patients (4.1 percent) and were all mild and transient. There were no clinically significant changes in liver function tests (LFTs).

The HELIOS-A results reinforce our commitment to building an industry-leading franchise of medicines for the treatment of ATTR amyloidosis which began with the development and approval of ONPATTRO as a treatment for patients with hATTR amyloidosis with polyneuropathy. Indeed, the vutrisiran results from HELIOS-A now serve as a second example of the potential for RNAi therapeutics to have a meaningful impact for patients, showing the ability to halt and potentially even reverse polyneuropathy manifestations of the disease. Furthermore, our robust development program, including the APOLLO-B and HELIOS-B studies, investigates the potential of patisiran and vutrisiran, respectively, to treat the cardiac manifestations of disease across a broad spectrum of patients with ATTR amyloidosis, said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. We believe that our ATTR amyloidosis franchise will be a significant driver of Alnylams growth in the years to come, with the potential to position Alnylam as a top tier biopharma company.

Vutrisiran has been granted Orphan Drug Designation in the United States and the European Union for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast Track designation in the United States for the treatment of the polyneuropathy of hATTR amyloidosis in adults. The safety and efficacy of vutrisiran are being evaluated in the comprehensive HELIOS clinical development program and have not yet been evaluated by any health authority. The ongoing HELIOS-B Phase 3 clinical trial in patients with ATTR amyloidosis with cardiomyopathy was initiated in late 2019 and is currently enrolling at sites around the world. Together, the HELIOS-A and -B studies are intended to demonstrate the broad impact of vutrisiran across the multisystem manifestations of disease and the full spectrum of patients with ATTR amyloidosis.

Conference Call InformationAlnylam management will discuss the HELIOS-A results via conference call on Thursday, January 7, 2021, at 8:00 am ET. A webcast presentation will also be available on the Investors page of the Companys website, http://www.alnylam.com. To access the call, please dial 877-312-7507 (domestic) or +1-631-813-4828 (international) five minutes prior to the start time and refer to conference ID 4398564. A replay of the call will be available beginning at 11:00 am ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic) or +1-404-537-3406 (international) and refer to conference ID 4398564.

About hATTR AmyloidosisHereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis, represents a major unmet medical need with significant morbidity and mortality affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy.

About VutrisiranVutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylams Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that allows for infrequent subcutaneous injections. The safety and efficacy of vutrisiran have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authority.

About RNAiRNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as a major scientific breakthrough that happens once every decade or so, and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylams RNAi therapeutic platform, function upstream of todays medicines by potently silencing messenger RNA (mRNA) the genetic precursors that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam PharmaceuticalsAlnylam (Nasdaq:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylams commercial RNAi therapeutic products are ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), and, in Europe, Leqvio (inclisiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam exceeded the goals first established in 2015 under its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit http://www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

Alnylam Forward Looking StatementsVarious statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, expectations regarding the direct or indirect effects on Alnylams business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom and the success of Alnylams mitigation efforts, Alnylam's views and plans with respect to the potential for RNAi therapeutics, including vutrisiran and patisiran, expectations regarding the safety and efficacy of vutrisiran as a treatment for hATTR amyloidosis with polyneuropathy, and its potential to have a meaningful impact on the course of this disease, expectations regarding the potential of vutrisiran and patisiran to treat the cardiac manifestations of ATTR amyloidosis across a broad spectrum of patients, Alnylams prospects for building an industry-leading ATTR amyloidosis franchise and to become a top-tier biopharma company, the expected timing for the filing of regulatory submissions for vutrisiran the presentation of full 9-month results and the announcement of 18-month topline results, including exploratory cardiac endpoint data, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, the availability of safe and effective vaccine(s), material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylams business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylams ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates, including vutrisiran; the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates or its or its partner Novartis marketed products, including ONPATTRO, GIVLAARI, OXLUMO and Leqvio (in Europe); obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, including vutrisiran, and the success of its partner Novartis, in obtaining regulatory approval for inclisiran in the U.S. and elsewhere, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO, GIVLAARI, and OXLUMO, as well as its partner Novartis success obtaining pricing and reimbursement for Leqvio; progress in continuing to establish an ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO, GIVLAARI, and OXLUMO, and achieving net product revenues for ONPATTRO within its revised expected range during 2020; Alnylams ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's ability to manage its growth and operating expenses within the ranges of guidance provided by Alnylam through the implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylams ability to establish and maintain strategic business alliances and new business initiatives; Alnylam's dependence on third parties, including Novartis for the continued development and commercialization of Leqvio, Regeneron for development, manufacture and distribution of certain products, including eye and CNS products, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures; as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

1 In alignment with the EMA, the primary endpoint of change from baseline in mNIS+7 will be evaluated at 18 months to support an MAA.

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Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy - Business...

"Cancer is a disease of genetics, yet clinical practice has struggled to keep pace with rapid advancements in research, particularly with respect to the role of germline genetics. Testing guidelines and medical policy often codify barriers, further lengthening the path to adoption of widespread testing and in some cases restricting access to precision therapies and clinical treatment trials," said Ed Esplin, M.D., Ph.D., FACMG, FACP, clinical geneticist at Invitae. "Research presented at ASCO shows that cancer-linked genetic changes are common across cancer types and when patients do receive germline testing, over two thirds of those with positive results are eligible for changes to their treatment plans. It's clear that incorporating germline testing alongside tumor profiling can help oncologists better tailor treatment for each patient."

Data from 250 pancreatic cancer patients from the landmark INTERCEPT study conducted at the Mayo Clinic found that nearly one in six patients with pancreatic cancer (n=38) showed cancer-linked genetic changes and, importantly, receiving germline testing was associated with improved survival.

A separate study of prostate cancer patients confirmed similar findings in other cancer types that limiting testing deprives patients and clinicians of actionable information. In the first-ever presentation of the PROCLAIM study, which was conducted primarily in community urology clinics, of patients diagnosed with prostate cancer, a significant number of cancer-linked variants were missed if testing was done based on NCCN guidelines. Of the 532 patients with clinician-reported data, nearly half, 45% (n=239), did not meet NCCN criteria. Overall, 59 patients had a cancer-linked variant; one in 10 of them did not meet the criteria (9.6%, n=23), and 12.3% (n=36) of patients met the criteria. When a 12-gene panel was used, only 29 patients were found to have a cancer-linked variant and one third of these patients were missed by guidelines.

A third study showed simply changing medical policy is not enough to drive changes in clinician adoption. In a review of two independent datasets, including commercially insured and Medicare Advantage enrollees, only 3% (n=1,675) of the 55,595 colorectal cancer patients received germline genetic testing, despite medical policy recommending germline genetic testing for all colorectal cancer patients (consistent with the INTERCEPT colorectal cancer study). Of the patients who received testing, 18% (n=143) had a cancer-linked variant and two thirds, or 67% (n=96), of those patients were potentially eligible for precision therapy and/or clinical trials.

"The data have been available for years that show knowing what changes patients have in their genes is beneficial to treating their cancer. Yet the oncology community has been slower to adopt germline testing than tumor profiling, for reasons that are not entirely clear. These data presented at ASCO highlight the need for oncologists to embrace germline genetic testing as routine practice for all cancer patients," said Robert Nussbaum, M.D., chief medical officer at Invitae. "A positive germline genetic result may enable patients to enroll in clinical trials or gain access to new precision medicines. And equally important, the discovery of an inherited variant can alert relatives to seek out earlier cancer screening, helping avoid later-stage diagnoses and offering a treatment benefit if cancer develops."

Invitae aims to help overcome obstacles to the adoption of genetic testing by providing physicians with clinical consults to help interpret results and reducing cost as a barrier to genetic information. Invitae also provides patients direct access to genetic counselors, helping to integrate routine genetic testing into patient care with GIA, a HIPAA-compliant chatbot. Family members are also able to receive no-charge genetic testing if a positive result is found.

Details of the 2021 ASCO presentations:

Oral Abstract Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Discussion Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Gastrointestinal Cancer--GastroesophageaI, Pancreatic, and Hepatobiliary

About InvitaeInvitae Corporation(NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Safe Harbor StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the benefits of germline testing and genetic information; and that the data presented at ASCO highlight the need for increased germline testing in all cancer patients regardless of medical policy. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact:Laura D'Angelo[emailprotected](628) 213-3283

SOURCE Invitae Corporation

http://www.invitae.com

Originally posted here:
Germline genetic testing can benefit all cancer patients as a routine practice in cancer care - PRNewswire

Genetic medicines are genetic materials such as DNA and RNA delivered into the body as a therapeutic. They are a promising new class of medicine that was not possible even a short time ago.

Advancements in science and technology are changing the way we define disease, develop drugs and prescribe treatments with an explosion of insights into the role of genetics in infectious diseases, cancer and rare diseases. Genetic medicines are an emerging technology with the potential to be developed as personalized medicines and for mass administration, by teams with a wide range of capabilities.

2020 Nobel Laureate Dr. Michael Houghton on Lipid Nanoparticle RNA Vaccines

Genetic vaccines are a new class of medicine that introduces new genes to the body to produce key antigens that help the body fight infections. Genetic vaccines can be broken down into two broad categories, viral vectors and non-viral vectors that use synthetic methods such as lipid nanoparticles deliver genes into cells. Non-viral vectorsoffer some advantages of viral vectorsincluding the ability to deliver larger genes, reduced biosafety concerns, and simplified synthetic production.

Watch the Full Webinar

Particularly, mRNA vaccines are a recent innovation in genetic medicine but are now at the forefront of the many vaccine technologies for the COVID-19 pandemic. mRNA vaccines use non-viral vectors to deliver the drug into the body which leads to safer and fast-to-develop vaccines. The mRNA vaccines at the forefront of the COVID-19 pandemic show how genetic vaccines have not only the potential for high potency, low-cost manufacturing and safe administration but also the capacity for rapid development.

A promising application for genetic vaccines is self-amplifying mRNA (saRNA), a vaccine platform that uses the human body toamplifythe vaccine and produce vaccine antigens. saRNA can offer a potent vaccine in extremely small doses due to its ability to amplify itself inside the body.

All these advantages of the mRNA vaccines translate into the development of other genetic medicines. The search for a pandemic vaccine has accelerated all genetic medicines by highlighting their advantages on the world stage.

Learn More About Genetic Vaccines

Samuel Clark, PNI Director of R&D, on how an mRNA gene therapy can work.

Gene therapy is the introduction of genetic material into the body to modify how proteins are expressed to treat a disease. Gene therapy is a promising application for genetic medicines for a number of diseases such as inherited disorders, viral infection and cancers. This technique allows doctors to treat a disorder by delivering a gene into a patient instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy, including:

Replacing a disease-causing mutated gene with a healthy gene

Inactivating, or knocking out, a malfunctioning mutated gene

Introducing a new gene to help fight a disease

The amount of gene therapies being developed have continued to grow with 352 gene therapy in clinical trialsglobally at the end of last year. The FDA expects to see a doubling of new gene therapy applications each year. Scott Gottlieb, the former FDA commissioner, predicts that by 2025 the US would be approving between 10 and 20 gene therapies each year.

Learn More About Gene Therapy

In 1989, A National Institutes of Health (NIH) approved study provided evidence for the first time that human cells could be genetically modified and returned to the patient without harm. Since then, the industry has grown significantly with 22 gene and gene-modified cell therapies approved by regulatory bodies from various countries as of August 2019.

Precision NanoSystems can provide a customized, end-to-end pathway for your drug development programs, from lead candidate selection through early phase clinical trials to commercialization. Our proprietary technology platforms and comprehensive expertise enable researchers to work with a single, integrated partner to translate disease biology insights into non-viral genetic medicines. Minimizing handoffs throughout the process results in faster timelines and reduced costs.

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What is Genetic Medicine

No boy should have a last stretch of days. But Bertrand Might lived his as well as any boy could: There was a Star Trek marathon with his brother and sister, sunrises on the lakeshore, and visits with family in parks, beaches, and backyards anywhere they could safely gather during the pandemic.

His father, Matt Might, said it ended up being an unplanned farewell for 12-year-old Bertrand, whose health had always been precarious. He was the first person in the world diagnosed with a particular neurodegenerative condition that causes developmental delays, seizure-like activity in the brain, and frequent infections.

One of those infections, unrelated to Covid-19, led to his death on Oct. 23 after he spiraled into septic shock. But if his passing came too soon, it did not come before his life led to crucial discoveries for dozens of children with his condition.

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What he did with NGLY1 alone was pretty powerful, said Matt Might, referring to the gene involved in his sons disease. After years of research, it was the discovery of a double mutation in Bertrands NGLY1 gene, and the constellation of symptoms linked to it, that explained the cause of the illness and built a worldwide community around it.

There are 70 families on the patient mailing list right now for a disease that eight years ago didnt exist, Might said.

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Bertrand also inspired a quest by his father, an artificial intelligence expert and computer programmer, to employ precision medicine on a wider scale, using genetic data to help tailor treatments to patients with rare and hard-to-treat diseases like his sons.

Might began that work initially to help Bertrand, but it led to a stint on President Obamas precision medicine initiative and the creation of a new precision medicine institute (PMI) he now leads at the University of Alabama, Birmingham.

PMI was founded on this algorithm that Bertrand taught me, Might said. How do you try to therapeutically modulate a specific genetic target? There is a central game plan we use every time somebody comes in.

Might and his team examine what gene is involved in a persons condition and whether it is under-reactive, over-reactive, toxic, or missing altogether. The answers to those questions form the basis for a scientific process that often gives patients hope when conventional medicine has failed to provide an accurate diagnosis or effective treatments. A permanent endowment has been established at UAB in Bertrands name to fund advanced diagnostics and research to identify novel therapies for patients with no other options.

In Bertrands case, the double mutation in NGLY1 left him without an enzyme that facilitates the recycling of cellular waste. It severely limited his mobility, requiring him to use a wheelchair, and also impaired his liver function and ability to communicate.

Still, Bertrand drove the science of his condition while enduring countless hospitalizations, often due to infections that made it difficult to breathe.

Throughout his life, he developed a love for dolphins and an aquarium his parents set up in his bedroom. He spent hours learning words and reading with his father and mother, Cristina, and he bonded with his younger brother and sister over movies and video games.

Im proud of Bertrand in multiple ways, Might said. I would often tell people to imagine a being created without the ability to even feel malice. He was just a pure being, and I loved that about him.

In recent years, the science that led to his diagnosis has also begun to unravel the biology of NGLY1 deficiency and its impact on patients. A project sponsored by the National Institutes of Health is underway to screen hundreds of thousands of molecules for therapeutic potential against the illness, while Might has used computational methods to identify treatments that showed efficacy in animal subjects.

On Bertrands last day in the hospital, as his condition continued to deteriorate, his father read him an email from the father of another patient with his illness. It said that the Food and Drug Administration seemed pleased with pre-clinical studies of a gene therapy for NGLY1 and outlined a series of steps toward a clinical trial.

It was so meaningful to know the community that Bertrand formed has spawned efforts well beyond my own, Might said. And in the end, he died in a world where the hope of a cure existed.

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The life and death of a boy whose diagnosis brought hope to other patients - STAT

Cryptocurrency. Gene editing. 5G. Drones. We hear and report on these terms often.

But for the average person whos not well versed in these topics, they might just sound like tech-y jargon, or technology that exists solidly in the future. On the latter point: Yes, but not exclusively.

In a year when Bitcoin is much more than a buzzword, gene-editing tool CRISPR is influencing COVID-19research, 5G is frequently flashing on our screens and a kid you know probably got a drone for Christmas, these concepts must be widely understood.

Inspired by Explain like Im 5 genre of subreddit,Technical.ly spoke to four experts about what these technologies are and what they mean for our futures.

Dr. Eric Kmiec, the director of the ChristianaCare Gene Editing Institute in Newark, Delaware, says gene editing is a form of genetic medicine with the goal of correcting DNA mutations that lead to inherited disorders or cancer. Diseases such as sickle cell disease, cystic fibrosis and Pompes disease have been the early targets for clinical trials. In particular, the sickle cell disease trials going on have shown great promise.

Kmiec said gene editing could be looked at as a genetic spellchecker. Chromosomes are like the words in a sentence, and gene editing can help correct those words to make that sentence coherent, or in this case, healthy without the mutant or misspelled gene in the chromosome.

Lets say that the first word of the sentence is misspelled, that is the first gene along a string of genes in a chromosome, he said. We can design a CRISPR molecule to fix the misspelling of the gene in the chromosome with high precision and high efficiency. So far, the challenge has been that the CRISPR tool may occasionally act to aggressively and secondarily alter a single letter in the last word of the sentence, or a single base in the last gene along the string in the chromosome.

Dr. Eric Kmiec, director of the ChristianaCare Gene Editing Institute. (Courtesy photo)

The field of gene editing is more than 30 years old, but Kmiec said the emergence of gene-editing tool CRISPR has made gene editing more accessible to people of various backgrounds.

Previously, the efficiency and precision with which mutations were repaired in the human chromosome were at a level that was not clinically relevant, he said. Today, CRISPR is changing that and molecular geneticists now believe that this tool will help us translate gene editing into a clinical reality.

Kmiec sees gene editing as an opportunity to change how health care is delivered to people. With many genetic diseases and forms of cancer currently lacking successful treatments, repairing genes at their roots could be the future of healthcare. With gene editing, healthcare pros will be able to attack health issues at their core.

Making this service equitable for all members of society is central to Kmiecs work at the Gene Editing Institute.

One big challenge lies in making sure that all socioeconomic levels of patients have access to such breakthrough technologies and that it is not simply reserved for those who can pay the price, he said. At ChristianaCares Gene Editing Institute, we have integrated a strong diversity and ethical approach to patient care as we further develop these gene editing technologies. The future of gene editing should also include a humanistic side because it is not just about the technology. Its ultimately about the patient.

Many popular commercials from leading cellular service companies like AT&T tout 5G as the latest achievement in cellular technology. While it is an advancement over its predecessor, Connectify cofounder and CEO Alexander Gizis said its not as large of a step forward as may be advertised.

5G is the most current generation of cellular tech and standards, following 4G, which gave cell phone users LTE. LTE was an integral part of the process that gave us internet on our smartphones and tens of megabits to support phone calls and texting.

Alexander Gizis. (Photo via LinkedIn)

5G is taking it to the next level of new technology, said Gizis, whose Philly-based networking software company makes an app to boost your mobile connection. But its not a disruptive technology. The innovator dilemma is to create all these opportunities for startups and 5G is mostly sustaining tech to help Verizon, AT&T and Apple do smartphone things even better.

Gizis explained that while 5G allows for faster speeds and better latency the time between user action and response its success is predicated upon the certain number of frequencies that cellular companies are allowed to employ.

The good bands like 1800 megahertz are full, he said. When you run 5G there is only so much bandwidth.

Gamers and fans of video calls are most likely to notice the difference between 4G and 5G service. Gizis said that 5G sends big blocks of data, allowing for faster responses in gaming. He predicts that a lot more smart devices like the Apple Watch will emerge in the next couple of years as technologists better understand and have the technology capable of taking advantage of 5G.

Exyn Technologies COO Ben Williams defines drones by their independence relative to tech systems. Regular drones are remotely controlled systems with active pilots for all or most of its actions. The term drone generally refers to a non-thinking device reliant upon direct input from a human operator or pilot.

When systems gain the capability to respond to their environments is where things get interesting. According to Williams, as drones become more autonomous, they begin to take on the capabilities of a robot.

The more clear-cut case is where a system is completely autonomous and no pilot is involved at any point of the flight; this type of autonomous system is a robot, he said.

Williams, whose Pennovation-based company uses drones and ground-based robots to collect data from places where GPS isnt accessible, said the significance of drones in terms of the future of technology exists in their potential as autonomous systems. He considers these systems to be the next obvious success of software that will enable digital processing.

Many modern companies are digital-first companies that can take advantage of massive distributed computing resources and highly advanced algorithms and systems to utilize data from across every aspect of their companies, he said. However these benefits are only within reach for those companies (like Google, Facebook, etc.) that can get all of that data into a computer system, and where the actions they want to take are digital in nature.

An image of Exyns technology. (Courtesy photo)

While digital-first companies will be at an advantage in employing autonomous systems, Williams said more traditional and analog companies that mostly exist in the physical world will have a harder time making the adjustment. Getting input data is difficult and acting upon can be even harder.

Exyn uses autonomous systems to gather massive datasets for analog industries that include mining, construction and logistics. Autonomous warehouse robots and delivery systems are seeing significant growth as their technology becomes more efficient.

Williams expects to see commercial and industrial companies begin to acquire thousands of these autonomous systems to collect data and take action based on the data acquired. Beyond digital systems, Exyn is already using robots to perform in dirty and dangerous environments.

One common use case for Exyn here is in underground mines where survey teams simply cant access some of the very dangerous environments without autonomous robots, he said. These systems allow the survey teams to greatly expand their access to previously inaccessible or very dangerous areas of the mines.

Mike McCoy, associate director of emerging technologies at health insurance company Humana, is passionate about the rise of cryptocurrency for good: He lectures on blockchain for health care,organizes the local and is the former Philly-based campus engagement lead at Brooklyn blockchain company ConsenSys.

He defines cryptocurrency as a monetary, digital asset that can be exchanged for value, like the U.S. dollar. It is protected by cryptography, and anyone worldwide can access it with a digital tool like a mobile device, laptop or browser using a digital node, or server. It is the first decentralized currency with no owner to be exchanged for goods and services in human history, he said.

Mike McCoy. (Courtesy photo)

McCoy said that cryptocurrency is the internets currency and provides more freedom than fiat (government-issued) currency like the American dollar. Just as tech startups have built profitable enterprises off of intangible concepts, cryptocurrency allows anyone to exchange value regardless of their race, ethnic background, gender or national origin.

Because cryptocurrency is sovereign resistant and designed to be decentralized, it does not need the power of any state or geographic body to enforce its value. McCoy said this allows for it empower the people that use it.

It allows for trustless transactions between humans without some king or authority or government or corporation having to be the validator of a transaction, he said. Its able to distribute wealth creation, wealth storage, and wealth protection from the state. And thats what cryptocurrencies really enable.

Bitcoin is currently the worlds best-known form of cryptocurrency, and McCoy considers it the digital gold standard. According to McCoy, people can divide up shares of Bitcoin to as many people as possible, as opposed to gold, which has a physical limit of who and how many people can have access to it. Its possibilities as a programmable currency also make it more flexible in distributing to others.

Privacy is one of cryptocurrencys advantages but also brings its own issues, McCoy said. While parts of it are private, it is not completely private and is barely a decade old, still leaving much to the imagination for people interested in its use.

Its only from 2009, he said. People dont know if itll be around forever. But every year Bitcoin and cryptocurrency survives and goes through one of the various challenges facing it and the currency becomes more valuable as people entrust more of their time, computational power and native currencies into it.

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We asked 4 experts how cryptocurrency, gene editing, 5G and drones will influence the future - Technical.ly