Nov. 5, 2020 06:00 UTC

TOKYO & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Modalis Therapeutics Corporation (Modalis) (TOKYO: 4883), a leading company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM epigenetic gene modulation technology, today reported financial results for the third quarter ended September 30, 2020, as well as recent operational highlights.

"Our goal is to create CRISPR based gene therapies for genetic disorders, most of which fall into the orphan disease category. There should be no disease that is ignored because of its small patient population, and our mission to develop disease modifying treatments for these diseases reflects our belief that Every Life Deserves Attention. We are proud to be a pioneer in CRISPR based gene modulation therapies and we are grateful to our investors and employees who are working to fulfill this important mission, said Haru Morita, Chief Executive Officer of Modalis.

Operational Highlights:

Third Quarter 2020 Financial Results:

About Modalis:

Modalis Therapeutics is developing precision genetic medicines through epigenetic gene modulation. Founded by Osamu Nureki and leading scientists in CRISPR gene editing from University of Tokyo, Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double-stranded DNA cleavage, gene editing or base editing. Modalis is focusing initially on genetic disorders caused by loss of gene regulation resulting in excess or insufficient protein production which includes more than 660 genes that are currently estimated to cause human disease due to haploinsufficiency. Headquartered in Tokyo with laboratories and facilities in Cambridge, Massachusetts. For additional information, visit http://www.modalistx.com.

Consolidated Financial Results for the Nine Months Ended September 30, 2020 [Japanese GAAP]

Company name: Modalis Therapeutics CorporationStock exchange listing: Tokyo Stock ExchangeCode number: 4883URL: https://www.modalistx.com/jp/ Representative: Haruhiko Morita, President and Representative DirectorContact: Naoki Kobayashi, CFO and Executive OfficerPhone: +81-3-6822-4584Scheduled date of filing quarterly securities report: November 13, 2020Scheduled date of commencing dividend payments: -Availability of supplementary briefing material on quarterly financial results: AvailableSchedule of quarterly financial results briefing session: -

(Amounts of less than one million yen are rounded down.)

1.

Consolidated Financial Results for the Nine Months Ended September 30, 2020 (January 1, 2020 to September 30, 2020)

(1) Consolidated Operating Results

(% indicates changes from the previous corresponding period.)

Operating revenue

Operating income

Ordinary income

Profit attributable toowners of parent

Nine months ended

Million yen

%

Million yen

%

Million yen

%

Million yen

%

September 30, 2020

340

-

168

-

209

-

214

-

September 30, 2019

-

-

-

-

-

-

-

-

(Note)

Comprehensive income:

Nine months ended September 30, 2020: 215 million [-%]

Nine months ended September 30, 2019: - million [-%]

Basic earnings

per share

Diluted earnings

per share

Nine months ended

Yen

Yen

September 30, 2020

8.34

-

September 30, 2019

-

-

(Notes)

1. The Company has not prepared the consolidated financial statements for the nine months ended September 2019. Accordingly, no figures are shown for the nine months ended September 30, 2019 and no percentage changes are shown for the nine months ended September 30, 2020.

2. Although the Company has dilutive shares, diluted earnings per share are not indicated because the Companys shares were not listed and the average share price is not available for the period under review.

(2) Consolidated Financial Position

Total assets

Net assets

Capital adequacyratio

Million yen

Million yen

%

As of September 30, 2020

6,480

6,428

99.2

As of December 31, 2019

3,938

3,842

97.6

(Reference)

Equity:

As of September 30, 2020: 6,428 million

As of December 31, 2019: 3,842 million

View source version on businesswire.com: https://www.businesswire.com/news/home/20201104005831/en/

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Modalis Therapeutics Reports Third Quarter 2020 Financial Results and Operational Highlights - BioSpace

THURSDAY, Dec. 10, 2020 (HealthDay News) -- Use of the ex vivo CRISPR-Cas9-based gene-editing platform to edit the erythroid enhancer region of BCL11A in hematopoietic stem and progenitor cells, producing CTX001, results in increased hemoglobin (Hb) among patients with transfusion-dependent -thalassemia (TDT) and sickle cell disease (SCD), according to a study published online Dec. 5 in the New England Journal of Medicine to coincide with the annual meeting of the American Society of Hematology, held virtually from Dec. 5 to 8.

Haydar Frangoul, M.D., from the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues presented safety and efficacy results from patients with at least three months of follow-up from two first-in-human studies of CTX001 for TDT and SCD. Data were included for seven patients with TDT and three with SCD.

The researchers found increases in total Hb and fetal Hb among all patients over time. Patients with TDT stopped receiving packed red blood cell transfusions soon after CTX001 infusion; the first patients with TDT who received CTX001 remained transfusion-free for more than 15 months. Since CTX001 infusion, the patients with SCD have had no vaso-occlusive crises (VOCs); the first patient to receive CTX001 remained VOC-free for more than one year. The safety profile after CTX001 infusion was generally consistent with busulfan myeloablation in all 10 patients. One patient with TDT had four serious adverse events related or possibly related to CTX001.

"By gene editing the patient's own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases," Frangoul said in a statement.

Several authors disclosed financial ties to pharmaceutical companies, including CRISPR Therapeutics and Vertex Pharmaceuticals, which sponsored the trial.

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ASH: CRISPR-Cas9 Gene Editing Promising in TDT, SCD - HealthDay News

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

Mutation for Huntingtons Disease Linked to Frontotemporal Dementia and ALS

Researchers with the National Institutes of Health/National Institute of Neurological Disorders And Stroke found that a mutation known to cause Huntingtons disease is linked to frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). Huntingtons disease and several other neurological disorders are associated with repeat expansion, a type of mutation that is kind of a genetic stutter, where certain amino acids in the DNA repeat abnormally. Huntingtons disease is the result of a sequence of three amino acids in the huntingtin gene repeats over and over again. The more repeats, the earlier the disease onset.

In a large international project, researchers screened the complete genomes from large cohorts of FTD/ALS patients and compared them to age-matched healthy individuals. Several patients had a well-established genetic marker for FTD/ALS appeared, but what caught everyone off guard was a small subset that had the same huntingtin mutation. They did not demonstrate classical symptoms of Huntingtons, but did for ALS or FTD.

None of these patients symptoms would have clued their physicians into thinking that the underlying genetic cause was related to the repeat expansion we see in Huntingtons disease, said Sonja Scholz, investigator, NINDS Intramural Research Program. Our patients simply dont match a textbook definition of disease when it comes to which mutation produces which symptoms. Here we have patients carrying a pathogenic huntingtin mutation but who present with FTD or ALS symptoms.

There is a theory that since a gene therapy for that mutation is currently in advanced clinical trials, it may have applications to FTD or ALS patients with this particular mutation. There are also possible applications for additional genetic screening for FTD and ALS. The research was published in the journal Neuron.

FDA Report Confirms Pfizer-BioNTechs COVID-19 Vaccine Efficacy

Although not exactly new research, the U.S. Food and Drug Administration (FDA), as part of the first day of its vaccines advisory committee meeting regarding emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 vaccine, released a 53-page report on Tuesday, December 8, 2020, that summarized data from their candidate vaccine trial. The data supports early data indicating the vaccine is safe and has an efficacy of 95%. The data also outlined how various diversity groups, including people over 65, those with pre-existing medical conditions like diabetes, and Black and/or Hispanic populations, were affected. The results show all appeared to be well protected. Approximately a third of the participants met the definition of obesity, which is lower than the general population of the U.S. The average age of volunteers in the trials was 51.

Eye Drop Designed to Deliver Drugs to the Retina and Other Back-of-the-Eye Tissues

Investigators with the Mass Eye and Ear of the Schepens Eye Research Institute developed an eye drop that can effectively deliver drugs to the retina and other tissues at the back of the eye. The experimental treatment is made up of nanoparticles called eNano-Ro5, and in their preclinical studies delivered a small molecule inhibitor of the transcription factor RUNX1 to the back of the eye. The excessive function of RNX1 has been linked by this same team of scientists to abnormal growth of blood vessels in people with proliferative diabetic retinopathy. In preclinical models reported in 2017, injection of the molecule curbed the aberrant vessel growth. In the new study, they packaged the drug into the nanoparticles and tested them in preclinical models of recurrent retinal detachment and proliferative vitreoretinopathy (PVR). eNano-Ro5 was effective in delivering the drug to the back of the eye, which decreased the severity of PVR.

COVID-19 Virus Particularly Well-Suited to Jump from Animals to Humans

Researchers at Duke University Medical Center studying the origin of SARS-CoV-2, the virus that causes COVID-19, found that it was particularly well-suited to jump from animals to humans. Genetic analysis found that its closest relative was a coronavirus that infects bats, but the ability to jump to humans was tied to a gene fragment from a coronavirus that infects the pangolin, a scaly mammal in Asia. The species-to-species ability to jump is caused by the viruss ability to bind to host cells via changes in its genetic code. However, they found that the typical coronaviruses that infect pangolins are too different from SARS-CoV-2 to have directly caused the pandemic. But they do contain a receptor-binding site that allows the viruses to attach to a cell surface protein common on human respiratory and intestinal epithelial cells, endothelial cell and kidney cells. Although the viral ancestor in the bat is closely related to SARS-CoV-2, its binding site is significantly different, meaning that on its own it cant efficiently infect human cells. They suggest that SARS-CoV-2 is a hybrid virus between bat and pangolin viruses.

Learning More About Which Immune Cells Offer COVID-19 Protection

With the Pfizer-BioNTech, Moderna and AstraZeneca-University of Oxford COVID-19 vaccines either authorized for emergency use or about to in different countries, researchers are still grappling with the exact types of immune responses needed to protect against the disease. Investigators from Beth Israel Deaconess Medical Center, working with monkeys, found that relatively low levels of antibodies offered protection in monkeys against the SARS-CoV-2 virus that causes COVID-19. They also investigated the role of CD8+ T-cells. They found that while antibodies alone can offer protection, including at relatively low levels, T-cells are also helpful if antibody levels are insufficient.

New Insulin Molecule Better Regulates Blood Sugar in Diabetes

Researchers at the University of Copenhagen and biotech company Gubra developed a new insulin molecule that they believe will eventually be able to better control type 1 diabetes. Currently, insulin on the market cannot tell the difference in type 1 diabetic patients if they need a small or large effect from the insulin. The new insulin molecule has a built-in molecular-binding capability that can sense how much blood sugar is in the body. As blood sugar increases, the molecule becomes more active and releases more insulin. As blood sugar decreases, less insulin is released. To date, it has been tested and been proven effective in rats. They are working to engineer the molecule so that it works more quickly and accurately before they can test it in humans.

New Form of Alzheimers Protein in CSF Identified

Investigators with Washington University School of Medicine discovered a novel form of tau, one of two proteins associated with Alzheimers disease (the other is beta-amyloid). The new type is MTBR tau, and the researchers believe it can be used to identify what stage of Alzheimers the person is in and track the progression of the disease. MTBR stands for microtubule-binding region tau. The disease starts when beta-amyloid begins forming plaques in the brain. This stage can last two decades or more without signs of cognitive decline. But soon afterward, tau tangles start to spread in the neurons, and the cognitive issues begin to appear and progress. The tau tangles can be detected by PET brain scans, but they are time-consuming and expensive. The study was published in the journal Brain.

The MTBR tau fluid biomarker measures tau that makes up tangles and can confirm the stage of Alzheimers disease by indicating how much tau pathology is in the brains of Alzheimers disease patients, said Randall J. Bateman, the Charles F. and Joanne Knight Distinguished Professor of Neurology at Washington School of Medicine in St. Louis. If we can translate this into the clinic, wed have a way of knowing whether a persons symptoms are due to tau pathology in Alzheimers disease and where they are in the disease course, without needing to do a brain scan.

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Research Roundup: Huntington's Mutation Linked to Frontotemporal Dementia and ALS and More - BioSpace

The approval of KYMRIAH, YESCARTA, Alofisel and Zyntelgo has increased the interest of pharma stakeholders in cell therapies; further, owing to the technical challenges in this field, outsourcing manufacturing operations has become a necessity

Roots Analysis has announced the addition of Cell Therapy Manufacturing Market (3rd Edition), 2019 2030 report to its list of offerings.

Owing to various reasons, the demand for cell therapies is anticipated to increase over the coming years. Therefore, both therapy developers and contract service providers may need to strengthen their capabilities and expand available capacity. In this context, automation is expected to be a key enabler within the cell therapy manufacturing and contract services industry.

To order this 500+ page report, which features 160+ figures and 250+ tables, please visit this link

Key Market Insights

More than 160 organizations claim to be engaged in cell therapy manufacturingThe market landscape is dominated by industry players, representing more than 60% of the total number of stakeholders. Amongst these, over 55 are large or mid-sized firms (having more than 50 employees).

100+ players focused on T-cell and stem cell therapiesMost of these players are focused on manufacturing T-cell therapies, including CART, TCR or TILs. It is worth highlighting that more than 35 organizations claim to have necessary capabilities for the manufacturing of both types of therapies.

Presently, 70+ companies have commercial scale capacityAs majority of the cell therapy products are in clinical trials, the demand is high at this scale. However, it is worth noting that several players (~50%) have already developed commercial scale capacity for cell therapies.

Europe is currently considered a current hub for cell therapy productionMore than 220 manufacturing facilities have been established by various players, worldwide; of these, 35% are in Europe, followed by those based in North America. Other emerging regions include Australia, China, Japan, Singapore, South Korea and Israel.

50+ facility expansions reported between 2015-2019More than 85% of the expansions are related to setting up of new facilities across different regions. Maximum expansion activity was observed in the US and in certain countries within the Asia Pacific regions.

20+ companies offer automated solutions to cell therapy developersPlayers that claim to offer consultancy services related to automation include (in alphabetical order) Berkeley Lights, Cesca Therapeutics, Ferrologix, FluDesign Sonics, GE Healthcare and Terumo BCT. Further, we identified 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.

Partnership activity has grown at an annualized rate of 16%, between 2014 and 2018More than 200 agreements have been inked in the last 5 years; majority of these were focused on the supply of cell-based therapy products for clinical trials. Other popular types of collaboration models include manufacturing process development agreements (16%), services agreements (12%) and acquisitions (10%).

By 2030, developed geographies will capture over 60% of the market shareAsia Pacific is anticipated to capture the major share (~36%) of the market 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, which is estimated to grow at a CAGR of ~26%.

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

Key Questions Answered

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 the manufacturing of cell therapies is largely being outsourced due to exorbitant costs associated with the setting-up of in-house expertise. 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 manufacturing services for cell-based therapies, featuring a company overview, information on manufacturing facilities, and recent collaborations.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html or email [emailprotected]

You may also be interested in the following titles:

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

Excerpt from:
Cell Therapy Manufacturing Market is estimated to reach close to USD 10 Billion by 2030, claims Roots Analysis - PRnews Leader

DUBLIN, Oct. 28, 2020 /PRNewswire/ -- The "Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations" report has been added to ResearchAndMarkets.com's offering.

This research identifies some of the key developments across CAR-T cell therapies and provides insights across technological, IP, and investment landscapes. The study also provides an analysis of the competitive landscape while highlighting the key growth opportunities within the CAR-T cell therapy platform.

Key Topics Covered:

1.0 Executive Summary1.1 Research Focus: Emerging Technologies Enabling chimeric antigen receptor (CAR) T-cell Therapies1.2 Analysis Framework: The Author's Core Value1.3 Research Methodology: Five Steps Toward Success1.4 Key Findings of Technology Breakthrough Driving Sepsis Diagnosis

2.0 Technology Snapshot2.1 Rising Pace of Cell and Gene Therapy Approvals2.2 Regulatory and Ethical Perspectives on Gene Therapy2.3 Rising Demand for Precision Medicine Strategies2.4 Manufacturing Continues to be the Key Bottle Neck2.5 II Generation Chimeric Antigen Receptors Likely to Dominate the Cell Therapy Landscape in the Future2.6 CD-19 is the Most Common Target Antigen for Allogeneic CAR-T Therapies

3.0 Emerging Patent Landscape3.1 Steady Increase in Patent Grants for CAR-T Cell Therapies3.2 University of Pennsylvania and Novartis Lead the Patent Landscape for CAR-T Cell Therapies3.3 China and the US Lead the Patent Landscape for CAR-T Cell Therapies3.4 Snapshot of Key Patent Grants: Novartis3.5 Snapshot of Key Patent Grants: Cellectis and BlueBird Bio

4.0 Analysis of the Investment Landscape4.1 Key M&A Trends Across the Global Life Sciences Sector4.2 Gene Therapy - Venture Capital Funding Assessment4.3 Gene Therapy - Big Pharma In-licensing Deals Assessment4.4 Strategic Insights: Cell Therapies and Gene Therapies, Viral Vector CMOS

5. Analysis of the Competitive Landscape5.1 Allogene Therapeutics5.2 Precision BioSciences Inc.5.3 CRISPR Therapeutics AG5.4 Cellectis S.A.5.5 Celyad5.6 Bristol-Myers Squibb (BMS)5.7 Gilead5.8 Novartis5.9 BlueBird Bio5.10 Summary of the Scoring Methodology5.11 Competitive Analysis of CAR-T Participants

6.0 CAR-T Cell Therapies: Growth Opportunity Universe6.1 Growth Opportunity: CAR-T for Solid tumors, 2020

7.0 Industry Influencers

For more information about this report visit https://www.researchandmarkets.com/r/dgf6f3

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Analysis of Recent Cell and Gene Therapy Innovations: 2020 Competitive Landscape Report - CD-19 is the Most Common Target Antigen for Allogeneic CAR-T...

News Release

Tuesday, October 27, 2020

Researchers from the National Institutes of Health (NIH) have discovered a new inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS), which is caused by mutations in the UBA1 gene. VEXAS causes symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in myeloid cells. The scientists reported their findings in the New England Journal of Medicine.

Nearly 125 million people in the U.S. live with some form of a chronic inflammatory disease. Many of these diseases have overlapping symptoms, which often make it difficult for researchers to diagnose the specific inflammatory disease in a given patient.

Researchers at the National Human Genome Research Institute (NHGRI), part of the NIH, and collaborators from other NIH Institutes took a unique approach to address this challenge. They studied the genome sequences from more than 2,500 individuals with undiagnosed inflammatory diseases, paying particular attention to a set of over 800 genes related to the process of ubiquitylation, which helps regulate both various protein functions inside a cell and the immune system overall. By doing so, they found a gene that is intricately linked to VEXAS, a disease which can be life-threatening. So far, 40% of VEXAS patients who the team studied have died, revealing the devastating consequences of the severe condition.

Usually, researchers discover a previously unknown disease by studying several patients with similar symptoms, then searching for a gene or multiple genes that may play a role in causing the disease. However, this was not a viable option for the NIH research team.

We had many patients with undiagnosed inflammatory conditions who were coming to the NIH Clinical Center, and we were just unable to diagnose them, said David B. Beck, M.D., Ph.D., clinical fellow at NHGRI and lead author of the paper. Thats when we had the idea of doing it the opposite way. Instead of starting with symptoms, start with a list of genes. Then, study the genomes of undiagnosed individuals and see where it takes us.

Out of the genome sequences of 2,560 patients with undiagnosed inflammatory conditions, over 1,000 patients had undiagnosed recurrent fevers and body-wide inflammation. The rest, part of the NIH Undiagnosed Diseases Network, had unusual and unclassified disorders.

Our objective was to see if any of the 2,560 patients shared variations in the same gene, said Daniel Kastner, M.D., Ph.D., scientific director of the Intramural Research Program at NHGRI and a senior author of the paper. Instead of looking at clinical similarities, we were instead taking advantage of shared genomic similarities that could help us discover a completely new disease.

Out of the 800 genes, one stood out. Three middle-aged males had rare and potentially damaging genomic variants in the UBA1 gene, but each of the three males appeared to have two copies of the UBA1 gene with one copy harboring the mutation, which was not unexpected because humans usually have two copies of every gene. However, the UBA1 gene resides in the X chromosome, and males have only one X chromosome (and one Y chromosome).

We were amazed to see this and wondered what it could mean. And thats when it clickedthis was only possible if there was mosaicism in these men, said Dr. Beck.

Mosaicism occurs when some people have groups of cells with mutations that are different from the rest of the body. The team predicted that there were specific cells in the patients bodies that carried the UBA1 gene in its normal form while other cells carried the gene in its mutated form.

Using DNA-sequencing methodologies, the researchers found that the mosaicism was indeed present in the patients myeloid cells, which are responsible for systemic inflammation and act as the first line of defense against infections.

The researchers then analyzed the genome sequences of additional individuals from various NIH cohorts and databases, which led to the discovery of an additional 22 adult males with the UBA1 gene mutations. Most of the individuals had symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in the myeloid cells.

Out of the combined 25 individuals, researchers were able to find a link between the various clinical rheumatologic and blood-related diagnoses made for the patients. Because these conditions exist in people with UBA1 mutations, the team grouped the various conditions into a new disease: VEXAS.

By using this genome-first approach, we have managed to find a thread that ties together patients carrying all of these seemingly unrelated, disparate diagnoses, Dr. Kastner said.

The researchers hope that this new genome-first strategy will help healthcare professionals improve disease assessments and provide appropriate treatments for thousands of patients who have various inflammation-related conditions. The study may also pave the way for a new and more appropriate classification of inflammatory diseases.

Additional research support for this study was provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Dental and Craniofacial Research, the National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, the National Cancer Institute and the NIH Clinical Center.

NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Extramural Research Program supports grants for research, and training and career development at sites nationwide. Additional information about NHGRI can be found athttps://www.genome.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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Scientists use clues in the human genome to discover new inflammatory syndrome - National Institutes of Health

Image: SARS-CoV-2 viral particles (blue) in a clinical isolate. (Photo courtesy of CDC)

Researchers at Yale University (New Haven, CT, USA) and the Broad Institute of MIT and Harvard (Cambridge, MA, USA) screened hundred of millions of cells exposed to the SARS-CoV-2 and MERS viruses, and have identified dozens of genes that enable the viruses to replicate in cells, as well as those that seem to slam the door on the virus. The pro-viral and anti-viral roles of these genes will help guide scientists in development of new therapies to combat COVID-19, according to the researchers.

Scientists have identified how SARS-CoV-2 attaches to and invades cells, but less is known about why some cells are more susceptible to infection. Understanding the genetics behind host cells susceptibility to infection may help explain why some people exposed to the virus experience few or no symptoms while others become extremely ill or die. Researchers performed a genome-wide screen of a line of green monkey cells, which are more sensitive to SARS-CoV-2 infection than commonly used human cell lines. The screens for the first time allowed researchers to simultaneously track interactions of virus and cells. The screens confirmed earlier findings that the ACE2 gene, which encodes a receptor on the cell surface, promotes infection by SARS-CoV-2.

However, the screens also identified two previously unknown pro-viral factors, as well as a third that seems to assist in preventing infection. They found that members of the SWI/SNF protein complex, which turns genes on and off, and HMGB1, which has a myriad of functions including regulation of inflammation, were linked to increased cell death after infection. The researchers then introduced small molecule drugs that inhibit function of two of the identified gene products, and found they could increase survival of cells after infection in a dish. By contrast, the histone H3 complex, which helps regulate expression of genes within the cell nucleus, seemed to provide a protective effect, inhibiting the ability of SARS-CoV-2 to infect and kill cells.

It is very important to understand wide variation of responses to COVID-19, for instance why advanced age makes it much more likely that people will die, said Yales Craig Wilen, assistant professor in laboratory medicine and immunobiology. We have identified both proviral and antiviral genes that may help us predict who is likely to get severely ill and what kind of drugs would be helpful or detrimental in treating patients.

Related Links:Yale University Broad Institute of MIT and Harvard

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Discovery of Helpful and Harmful COVID-19-Related Genes to Aid Development of New Therapies - HospiMedica

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that it will host a virtual R&D Day on Friday, October 30, 2020 at 11:30 AM Eastern time, to discuss the Companys AXO-Lenti-PD gene therapy for Parkinsons disease.

Axovants Parkinsons disease R&D Day will be moderated by Chief R&D Officer, Gavin Corcoran, M.D., and will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinsons disease from the following key opinion leaders:

In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:

Drs. Adler, Palfi, and Eberling will be joined by Dr. Corcoran to answer questions following the formal presentations.

AXO-Lenti-PD is the only investigational gene therapy for Parkinsons disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

To register for the R&D webcast, please click here.

A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.

Biographies of R&D Day Panelists:

Dr. Adler has received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adler has a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adlers main research interests are investigating tissue diagnostic tests for Parkinsons disease, biomarkers for an early diagnosis of Parkinsons disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adler was awarded the Mayo Clinic Distinguished Investigator of the Year Award.

Dr. Palfi has published extensively on trophic factor- and enzyme-based gene therapy in Parkinsons disease and Huntingtons disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors.

Dr. Eberling earned undergraduate and graduate degrees in biological psychology from the University of California at Berkeley, later moving to the Lawrence Berkeley National Laboratory where she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinsons disease.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523investors@axovant.com

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.commedia@axovant.com

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Axovant Gene Therapies to Host Virtual Parkinson's Disease R&D Day on October 30, 2020 - GlobeNewswire

Newswise PITTSBURGH, Oct. 26, 2020 James J. Collins, Ph.D., an innovator in synthetic biology whose ideas have contributed to novel diagnostics and treatments targeting infections and complex diseases, has been awarded the 2020 Dickson Prize in Medicine, the University of Pittsburgh School of Medicines highest honor.

The prize is given annually to an American biomedical researcher who has made significant, progressive contributions to medicine. The award consists of a specially commissioned medal, a $50,000 honorarium and an invitation to present the keynote lecture during the Universitys annual campus-wide showcase of scientific research. Due to the COVID-19 pandemic, both the annual showcase and Collins lecture have been postponed until 2021 at a date to be determined.

Dr. Collins is defining whats possible in the disciplines of synthetic and systems biology. His highly creative work applying engineering design principles to molecular biology has generated numerous new diagnostics and therapeutics with wide application to medicine, said Anantha Shekhar, M.D., Ph.D., Pitts senior vice chancellor for the health sciences and John and Gertrude Petersen Dean of Medicine. It is our honor to recognize him with the School of Medicines most prestigious award.

Im grateful to work with outstanding lab members and collaborators whose dedication and insight have been critical to what weve achieved, said Collins, who is the Termeer Professor of Medical Engineering and Science in the Department of Biological Engineering at Massachusetts Institute of Technology and is affiliated faculty with the Broad Institute of MIT and Harvard University, and the Wyss Institute at Harvard. I am thrilled and honored to receive the Dickson Prize in Medicine.

A seminal 2000 publication describing the successful creation of a stable, synthetic gene circuit in Escherichia coli bacteria has been cited more than 4,000 times and marked the arrival of an important new discipline in biomedicine. Collins later demonstrated that synthetic gene networks could be linked with a cells genetic circuitry as a regulatory mechanism to create programmable cells for biomedical applications.

More recently, Collins has created engineered microbes and whole-cell biosensors to serve as in vivo diagnostics and therapeutics. One innovative platform that he and colleagues developed embeds freeze-dried, cell-free synthetic gene networks onto paper and other materials with a wide range of potential clinical and research applications.

The resulting materials contain properties of a living cell, are stable at room temperature and can be activated by simply adding water. Collinss work on freeze-dried, cell-free synthetic biology has established a platform for a new class of rapid, programmable in vitro diagnostics for emerging pathogens, including drug-resistant bacteria and viruses. Collins and his team currently are developing a rapid self-activating COVID-19 face mask as a wearable diagnostic.

Collins earned an A.B. in physics at the College of the Holy Cross in Worcester, Mass., before completing a Ph.D. in medical engineering at the University of Oxford with the distinction of Rhodes Scholar. He has received a MacArthur Foundation Genius award, NIH Directors Pioneer Award and Sanofi-Institut Pasteur Award. Collins is an elected member of the National Academy of Sciences, National Academy of Engineering, National Academy of Medicine and the American Academy of Arts and Sciences. He is a charter fellow of the National Academy of Inventors.

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2020 Dickson Prize in Medicine Awarded to Pioneer Researcher in Synthetic Biology - Newswise

HARRIS COUNTY, Ala. (WRBL) A Harris County family is celebrating overcoming a major obstacle in treating their 8-year-old-son who has a rare form of Muscular Dystrophy, thanks to a village of supporters and a phone call from the Chief Medical Officer of a major insurance company.

8-year-old Jacob Saalman is like most kids. He loves being outdoors, riding four-wheelers, and playing baseball. This Halloween, he carved pumpkins with his family. Jacob loves his dad, a former Fort Benning Ranger, and his mom. Jacob adores his four-year-old brother, Hudson.

Jacob is very protective of Hudson. Hes a mother hen. Oftentimes I have to tell Jacob to stop parenting, shared Ryan and Brooke Saalman, Jacobs parents.

However, Jacob is not like most kids when it comes to his health. Jacobs handled more medical procedures, biopsies, and tests than many adults. Its heartbreaking. Jacobs mom and dad say hes resilient with a quiet strength.

I would say he is very strong and adaptable. All the things he has to do, all the medical stuff he has been through, pokes and prods and port placement and muscle biopsies. He has been through a lot, said Brooke.

Jacob is very sick. So is his brother Hudson. Both boys have Duchenne Muscular Dystrophy, a rare genetic disease, preventing their bodies from making a protein called Dystrophin that protects our muscles from breaking down with use.

Jacob plays ball now. By 12, he will most likely need a wheelchair. After that, the prognosis remains bleak for the brothers and the parents who love them.

The life expectancy is upper 20s and into the 30s. But you are not walking around or living a normal life. You are most likely on a ventilator and having a parent care of you 24/7, shared Ryan.

Jacobs medical team is convinced an FDA approved home infusion called Exondys 51 will significantly improve Jacobs quality of life by helping him produce partial Dystrophin proteins.

Its not a cure. It slows the rate of the disease, and also its been proven to help kids walk longer, and it is also shown to improve pulmonary function, and thats the issue later on down that causes a lot of problems and even death from this disease, said Brooke.

The Saalmans goal is to stave off the disease for as long as possible. They believe Exondys 51 can help keep Jacob as healthy as possible until a gene therapy is ready.

We still have hope. What gives us the most hope is there are gene therapy trials in phase three. So we are hoping within the next two or three years we will be able to have a gene therapy that will make this a much milder disease, said Brooke.

One of Jacobs physicians, Han C. Phan, MD, with Rare Disease Research LLC in Atlanta, Georgia, shared this statement with News 3:

Jacob has Duchenne Muscular Dystrophy, a slowly progressive condition for which without treatment could limit his life expectancy. Every child with DMD should receive the treatment he deserves and without such could be detrimental to his overall health.

The family says Horizon Blue Cross Blue Shield of New Jersey had twice denied Jacob Exondys 51, despite other major insurance companies approving similar patients treatment. The drug costs 300,000 a year. The company listed several reasons for denying coverage for the medicine in a letter sent to the Saalman family.

Ryan Saalman shared the familes heartbreak and frustration on his Facebook page earlier in the week. The status update was shared several thousand times and sent to News 3.

Monday, the Sallman family spoke with News 3 about their struggle since April to get the medication approved with their insurance company.

Its just frustrating to know there is a child that has this need, and we pay good money for our insurance. You think that if a doctor says this is medically necessary that they will get the medication, and thats not the case. These kids deserve that. Everything they go through. They deserve some help, said Brooke.

News 3 reached out to Horizon Blue Cross Blue Shield of New Jersey on Monday. Tuesday, News 3 worked with The Saalman family and Horizon BCBS of New Jersey to get the necessary medical privacy forms filled out to get information on the insurance companys case. However, before the process was complete, The Saalman family says they got a phone call from the insurance companys Chief Medical Officer. The family was thrilled to learn Exondys 51 has been approved for Jacob.

Today, I think we truly saw the power of community. In this fight against Duchenne Muscular Dystrophy, it takes a village, and I think our village got really big really fast. Our new village gave an 8-year-old boy a loud enough voice that Horizon BCBS has overturned last weeks denial. Jacob will be able to get the medication he needs. The Saalman Family wants to thank the thousands of people that shared our story; we truly believe it is because of all of you that this has happened,

The Saalman family is overjoyed. Time is not on their side as this disease doesnt slow down. With each delay comes another day Duchenne Muscular Dystrophy robs Jacobs muscles of their ability to function. The family hopes the newly approved infusion will be ready for Jacob within a few weeks. The treatment should help slow the diseases progression down and give the family the time they need to wait for a promising gene therapy they pray is on the horizon.

The day before The Saalman family learned the medication had been approved, Jacob hit the winning RBI for the final game of his baseball season in a tie game with two outs. Jacob got the game ball in the final game of what may be his last season of baseball.

And sometimes God shows up in the last game, in the last inning, with two outs and two strikes on the board, when the hope of a win is almost gone and He crushes our expectations. Man, did I ever need that reminder tonight, and maybe you do too, shared Jacobs father on Facebook.

For more information on Jacob and Hudsons battle with Duchenne MD and ways you can help, you can visit their blog: Saalman Strong. The family has been encouraged to establish a GoFundMe page: Jacob and Hudsons fight against Duchenne MD

News 3 will keep you updated on the Saalman family and the journey with their sons Jacob and Hudson.

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Parents win insurance appeal for 8-year-olds Muscular Dystrophy treatment - WWTI - InformNNY.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. today announced that the U.S. Food and Drug Administration (FDA) approved FoundationOneLiquid CDx for three new companion diagnostic indications to help match patients who may benefit from treatment with specific FDA-approved targeted therapies. The new indications are for Piqray (alpelisib) in advanced or metastatic breast cancer; Rubraca (rucaparib) in advanced ovarian cancer; and Alecensa (alectinib) in a certain type of metastatic non-small cell lung cancer (mNSCLC). The FDA also approved a label expansion for FoundationOne Liquid CDx to report additional select copy number alterations and genomic rearrangements.

FoundationOne Liquid CDx analyzes the largest genomic region of any FDA-approved comprehensive liquid biopsy test and was approved in August to provide tumor mutation profiling in accordance with professional guidelines for patients with any solid tumor. Concurrently, it was approved as a companion diagnostic for a poly (ADP-ribose) polymerase (PARP) inhibitor approved by the FDA for the treatment of metastatic castration-resistant prostate cancer patients with qualifying BRCA1/2 alterations, and for three first-line EGFR tyrosine kinase inhibitors (TKIs) for the treatment of non-small cell lung cancer patients.

FoundationOne Liquid CDx offers oncologists an important and minimally invasive tool to consider when making treatment decisions for their patients, regardless of the type of cancer they have, said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. These three additional companion diagnostic claims expand the tests clinical utility into breast and ovarian cancer, demonstrating our commitment to bringing precision medicine to more patients, and we plan to continue working with our biopharma partners to increase that reach.

Todays approval expands FoundationOne Liquid CDxs companion diagnostic indications to include the following targeted therapies:

PIK3CA is the most commonly mutated gene in HR+/HER2- breast cancer; approximately 40% of patients living with HR+/HER2- breast cancer have this mutation.1

An estimated one in four women with epithelial ovarian cancer have a mutation of the BRCA1 or BRCA2 gene.2

Using a blood sample, FoundationOne Liquid CDx analyzes over 300 cancer-related genes for genomic alterations. FoundationOne Liquid CDx results are delivered in an integrated report that identifies alterations matched to FDA-approved therapies. It also enables accelerated companion diagnostic development for biopharma companies developing precision therapeutics.

As a laboratory professional service which has not been reviewed or approved by the FDA, the FoundationOne Liquid CDx report delivers information about the genomic signatures microsatellite instability (MSI) and blood tumor mutational burden (bTMB), as well as single gene alterations, including NTRK fusions, to help inform the use of other therapies including immunotherapies. Also, as a laboratory professional service, the report provides relevant clinical trial information and includes interpretive content developed in accordance with professional guidelines in oncology for patients with any solid tumor.

About FoundationOne Liquid CDx

FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and mutation status confirmed using an FDA-approved tumor tissue test, if feasible. For the complete label, including companion diagnostic indications and complete risk information, please visit http://www.F1LCDxLabel.com.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

PIQRAY is a registered trademark of Novartis AG.

RUBRACA is a registered trademark of Clovis Oncology, Inc.

ALECENSA is a registered trademark of Chugai Pharmaceutical Co., Ltd., Tokyo, Japan.

Source: Foundation Medicine

1 The Cancer Genome Atlas Network. Comprehensive molecular portraits of human breast tumours. Nature. 2012;490(7418):61-70.2 Pennington et al, Clin Cancer Res. 2014; 20(3):764-7753 Dearden et al. Ann Oncol. 2013 Sep; 24(9): 23712376.

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FDA Approves New FoundationOneLiquid CDx Companion Diagnostic Indications for Three Targeted Therapies That Treat Advanced Ovarian, Breast and...

LAUSANNE, Switzerland--(BUSINESS WIRE)--Saphetor, a global precision medicine company and the creator of VarSome, the emerging global standard platform for human genome data, announced today a partnership with Roche Turkey. The collaboration will be centered around supporting Roches drug discovery initiatives into Spinal Muscular Atrophy (SMA).

The open genomics community VarSome.com brings together a vast global network of clinical and research genetic laboratories. Saphetor will facilitate connecting labs who wish to partner with Roche on SMA research. The goal will be to create and foster relationships as well as to accelerate scientific collaboration with SMA community.

Exploring new and more efficient way for scientific collaboration has long been an obstacle in the pharmaceutical industry. Saphetor is helping to avoid the loss of critical time through identifying the most relevant patients and facilitating the communication between involved parties such as labs, pharma and patients.

Anne Nijs, Transformation Leader, Rare Conditions for Pharma International at Roche said: We look forward to leveraging Saphetors innovative approach to bring together the global genomics community in order to connect with the right participants in the healthcare ecosystem of rare disorders.

Dr. Turgut Gkalp, SMA Rare Conditions Partner for Turkey, Azerbaijan and Georgia added: We are always looking for new ways to identify new stakeholders and therefore new collaboration opportunities. I believe our partnership with Saphetor will give us a better understanding of SMA testing landscape in Turkey.

Andreas Massouras, CEO of Saphetor commented: We are very excited to partner with Roche and contribute to their important effort in developing treatments for this debilitating and often lethal disease. VarSome.com is proving an extremely valuable resource for willing clinical and research labs as well as pharmaceutical companies to collaborate on a worldwide level.

About SMA

Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies. SMA leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. Depending on the type of SMA, an individuals physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.

About Roche

Roche 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.

Neuroscience is a major focus of research and development at Roche. The companys goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases.

Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimers disease, Huntingtons disease, Parkinsons disease, Duchenne muscular dystrophy and autism.

About VarSome and Saphetor SA

VarSome is the emerging global standard platform for human genome data. With more than 300,000 professional users and 80+ standardized and continuously updated major datasets freely searchable online, varsome.com is the world's largest community and knowledge base for human genome variant data. VarSome's premium, clinical and API tools further enable anyone and any organization, from individual healthcare professionals to hospitals and pharmaceutical companies, to harness and apply the power of this community and data to improve health and lives worldwide. VarSome is created by Saphetor, a Switzerland-based precision medicine company using bioinformatics to apply human genome data to benefit people.

VarSome Clinical is CE-IVD-certified and HIPAA-compliant, allowing fast and accurate variant discovery, annotation, and interpretation of NGS data for whole genomes, exomes, and gene panels, which helps clinicians reach faster and more accurate diagnoses and treatment decisions for genetic conditions.

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Saphetor Partners with Roche Turkey to Advance New Treatments for SMA - Business Wire

Oct. 27, 2020 12:00 UTC

The only tertiary analysis appliance on the market, CompStor Insight delivers 7x faster annotation in an easy to use solution with integrated workflows, multi-user support, needing no special IT skills

MILPITAS, Calif.--(BUSINESS WIRE)-- OmniTier Inc., an AI and multiomics application company whose bioinformatics solutions help deliver the promise of personalized medicine, debuted the CompStor Insight appliance for tertiary analysis in next-generation sequencing (NGS) applications. In benchmark results, CompStor Insight enables a 7x improvement in gene annotation performance allowing researchers to spend more time developing treatment innovations, and less time on data processing.

OmniTier made the announcement coincident with the ASHG 2020 virtual meeting, where it will showcase CompStor Insight and other members of the CompStor genomics analysis product family.

Our researchers are very busy because we need to analyze and interpret complex variants of many patients, said Kazuhiro Nitta, lecturer at Juntendo Universitys Graduate School of Medicine in Tokyo, and part of the Intractable Disease Research Center. He collaborated with OmniTier on CompStor Insight product development. It was an enjoyable experience to work with the innovative team at OmniTier. With the level of automation and sophisticated filtering that is supported by CompStor Insight, we estimate we could reduce the amount of time for downstream analysis of multi-genomic data sets dramatically.

Personalized medicine initiatives have put the spotlight on tertiary analysis as the most complex step in NGS, where scientists seeking better treatments for cancer or rare diseases investigate variants identified during secondary analysis. Competing open source solutions and internally developed applications in use today for tertiary analysis often exhibit low level of functional automation and require long data ingress-compute-egress cycles delay time to actionable data that does not scale with more server nodes.

CompStor Insight is the first tertiary analysis appliance on the market and enables faster time to results, and low per-subject cost. With an easy set up and straightforward Web browser interface, it provides push-button workflows for annotation, filtering, visualization and querying variant data. Utilizing OmniTiers proprietary MemStac tiered memory technology, CompStor Insight can process up to several thousand genome datasets at speed, enabling faster and more accurate interpretation of data in genome-wide-association studies (GWAS) and rare disease analysis. Because CompStor Insight is designed to interface to a wide range of standard and custom knowledge databases including ClinVar and gnomAD, teams have access to a wide set of reference data at their fingertips. Being a stand-alone appliance, CompStor Insight can store subject data locally in on-premise storage, or leverage cloud storage options.

In testing, CompStor Insight delivered a 7x reduction in run time to annotate typical WGS data, compared to Ensembl VEP. For NGS service businesses, where time is money, CompStor accelerates the annotation and filtering functions dramatically, providing an opportunity to grow their revenue and profit easily.

When used with OmniTiers secondary analysis appliance, CompStor Novos, organizations benefit from an end-to-end analytics solution, from sequencer output, advanced variant calling, and fast annotation, to state-of-the-art multisubject tertiary analysis. Pharmaceutical and biotech companies can take advantage of CompStor Insights intuitive GWAS workflows to identify biomarkers for drug development and develop subject selection strategies.

As the cost of gene sequencing decreases, many organizations are looking for faster, easier ways to learn from each patients data, and convert data to knowledge, said Christi Bird, principal consultant and fellow in Frost & Sullivan's Transformational Health Growth consulting team. OmniTier is addressing a key growth market as the bioinformatics industry works to enable the mass adoption of genomic medicine.

Researchers are straining to meet the explosive demand for personalized medicine with analytics that burden them with complexity and delays that impede the ability to turn data into knowledge and then treatments, said Hemant Thapar, CEO and founder of OmniTier. CompStor Insight is enabling the low per subject cost and faster turnaround time to actionable data that is critical to delivering better treatments for hundreds of diseases and genetic conditions.

CompStor Insight appliances will start shipping to pharmaceutical and research organizations in December 2020. For more information or to request pricing please visit http://www.omnitier.com.

About OmniTier Inc.

OmniTier develops AI and multiomics appliances and software for bioinformatics, scientific computing, and web services applications that deliver affordable real-time solutions to enrich everyday living. Its integrated appliance solutions accelerate data-intensive infrastructure applications, including genomic workflows and scientific analysis for machine learning and AI. Founded in February 2015, the company has R&D operations in Milpitas, CA and Rochester, MN.

CompStor Insight is for research purposes only. CompStor, CompStor Insight and MemStac are trademarks of OmniTier, Inc. CompStor Novos is a registered trademark of OmniTier, Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201027005319/en/

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OmniTier Streamlines Personalized Medicine Workflows with CompStor Insight for Next-Generation Sequencing Tertiary Analysis - BioSpace

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Senti Bio is at the forefront of using synthetic biology to engineer gene circuits that improve cell and gene therapy products. A gene circuit is an assembly of multi-component genetic constructs specifically designed to program cells to interact with the bodys complex environment using logic to perform desired therapeutic functions. Senti Bio uses these gene circuits to create smarter cell and gene therapies with enhanced therapeutic properties that aim to increase efficacy, precision and control.

Senti Bio is applying its gene circuit technology platform to develop an internal therapeutic pipeline of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells. Senti Bios lead development candidates include next-generation allogeneic CAR-NK cell therapies: SENTI-202 for acute myeloid leukemia (AML), SENTI-301 for hepatocellular carcinoma (HCC), and additional candidates for other undisclosed solid tumor targets.

Leaps by Bayers mission is to invest in breakthrough technologies that may transform the lives of millions of patients for the better, said Juergen Eckhardt, MD, Head of Leaps by Bayer. We believe that synthetic biology will become an important pillar in next-generation cell and gene therapy, and that Senti Bios leadership in designing and optimizing biological circuits fits precisely with our ambition to prevent and cure cancer and to regenerate lost tissue function.

In addition to potentially treating cancer with allogeneic CAR-NK cells, the Senti Bio gene circuit technology platform can be deployed into multiple other cell and gene therapy delivery modalities, across diverse therapeutic areas, such as immunology, neuroscience, cardiovascular disease, regenerative medicine and genetic diseases with the potential to move from treatment to cure.

We are grateful for the support of new and existing investors, including Leaps by Bayer, who believe in our mission of developing gene circuits to program smart cell and gene therapies to improve health outcomes for many people, said Tim Lu, MD, PhD, co-founder and chief executive officer of Senti Bio. Over the past two years, our team has designed, built and tested thousands of sophisticated gene circuits to drive a robust product pipeline, focused initially on allogeneic CAR-NK cell therapies for difficult-to-treat liquid and solid tumor indications. I look forward to continued platform and pipeline advancements, including starting IND-enabling studies in 2021.

Proceeds from the Series B financing will support development of preclinical oncology programs and expansion of the Senti Bio gene circuit technology platform across additional delivery modalities and therapeutic areas. Senti Bio also plans to scale up clinical manufacturing, including process development and design of a cGMP-compliant manufacturing facility for off-the-shelf allogeneic CAR-NK cell product candidates.

The Series B syndicate included existing and new investors as follows: 8VC, Alexandria Ventures Investments, Amgen Ventures, Gaingels, Intel Capital, KB Investment, Leaps by Bayer, LifeForce Capital, LifeSci Venture Partners, Lux Capital, Matrix Partners China, Menlo Ventures, Mirae Asset Capital, NEA, Nest.Bio, Noveus Capital, Pear VC, Ridgeback Capital and Smilegate Investment.

About the Senti Bio Gene Circuit Technology Platform

By combining disciplines from computer science and biology, Senti Bio has designed, built and tested thousands of sophisticated gene circuits that can be deployed into virtually any cell therapy or gene therapy modality. Senti Bios gene circuits are novel and proprietary combinations of DNA that enable cells to sense their environment, perform logic and instruct cells to produce therapeutic proteins for enhanced safety and efficacy. Senti Bio believes that its approach to programming gene circuits in living cells may enable drug developers to build optimal functionality into almost any cell- or gene-based medicine. Senti Bios proprietary platform includes specific gene circuit technologies such as logic gates, small-molecule regulators, combinatorial payloads and synthetic promoters that have the potential to confer greater efficacy, precision and control to cell and gene therapy products. By mixing and matching different gene circuits together, Senti Bio has the ability to create next-generation medicines with enhanced functionality to outsmart disease.

About Bayer and Leaps by Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of 43.5 billion euros. Capital expenditures amounted to 2.9 billion euros, R&D expenses to 5.3 billion euros. For more information, go to http://www.bayer.com.

Leaps by Bayer, a unit of Bayer AG, leads impact investments into solutions to some of todays biggest challenges in health and agriculture. The investment portfolio includes more than 30 companies. They are all working on potentially breakthrough technologies to overcome some specific challenges such as, e.g. regenerating lost tissue function, reducing the environmental impact of agriculture, preventing or curing cancer, and others. For more information, go to leaps.bayer.com

About Senti Bio

Senti Bio is a next-generation therapeutics company that is developing gene circuits and programming cells for tremendous therapeutic value. Senti Bios mission is to outsmart complex diseases with more intelligent medicines to transform peoples lives. By programming cells to respond, adapt and make decisions, Senti Bio is creating smarter therapies with computer-like logic, enhanced functionality and greater therapeutic control.

Senti Bio is developing a wholly-owned, gene circuit pipeline focused on allogeneic CAR-NK cells to address major challenges in cancer treatment. Senti Bios lead product candidates include SENTI-202 and SENTI-301. SENTI-202 is a logic-gated allogeneic CAR-NK cell therapy for the potential treatment of acute myeloid leukemia (AML) that more precisely targets and eliminates cancer cells while sparing healthy tissues. SENTI-301 is a combinatorial payload-armed allogeneic CAR-NK cell therapy for the potential treatment of hepatocellular carcinoma. Beyond oncology, Senti Bio plans to leverage its gene circuit technology platform to build other cell and gene therapies that may be of interest to strategic partners across diverse therapeutic areas, such as immunology, neuroscience, cardiovascular disease, regenerative medicine and genetic diseases. For more information, please visit the Senti Bio website at https://www.sentibio.com.

Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayers public reports which are available on the Bayer website at http://www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210106005074/en/

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Leaps by Bayer Leads USD 105 Million Series B Financing in Senti Bio to Develop Next-Generation Cell and Gene Therapies Using Advanced Gene Circuit...

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, and UT Southwestern Medical Center (UTSW) today announced the launch of an innovation fund to discover and develop novel gene therapy candidates and next-generation technologies for monogenic diseases of the CNS. This expanded partnership will support UTSWs discovery efforts to facilitate the translation of promising discoveries from bench to clinic. Taysha will have an exclusive option on new programs and intellectual property associated with, and arising from, the research conducted under this agreement.

A team of researchers from the gene therapy program at UT Southwestern will explore novel gene therapy targets in new disease areas and create next-generation gene therapy technology platforms to address some of the current limitations of this modality.

We are excited to expand our alliance with UTSW to accelerate the discovery and development of novel gene therapy candidates and next-generation technologies for patients with monogenic CNS diseases, said RA Session II, President, Founder and CEO of Taysha. We believe that the combination of UTSWs translational research expertise in gene therapy and strong track record of innovation and our experience in drug development and GMP manufacturing will create opportunities to reach more patients with unmet medical needs. Our relationship with the UTSW gene therapy program has produced over 18 novel product candidates, including TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1, which are currently in clinical development. We are pleased by the significant progress our partnership has achieved and are excited to build on that foundation and momentum to bring additional compelling innovation to the clinic.

About The University of Texas Southwestern Medical Center

UT Southwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes and includes 23 members of the National Academy of Sciences, 17 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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Taysha Gene Therapies and UT Southwestern Medical Center Launch Innovation Fund to Accelerate Advancement of AAV Gene Therapies for Monogenic Diseases...

New Covid-19 variants that appear to spread easier than the original strain are sparking concerns that the U.S. system for tracking the virus isnt keeping pace with mutations.

Some scientists say that even though testing works now, health officials need to be better prepared for variants down the road or risk not being able to properly detect the SARS-Cov-2 virus. Theyre calling on the Biden administration to invest more in genomic sequencing, a process that involves collecting the DNA blueprint of an organism.

A new mutation that can hide from a common testing procedure, like the variant that surfaced in the U.K. doesalbeit not significantlymarks a serious tremor before a quake, Fyodor Urnov, a professor in the University of California, Berkeleys Molecular and Cell Biology Department, said. While the variant does hide from one part of the test, most tests use multiple procedures to look for different parts of the SARS-Cov-2 virus, which for now negates the variants ability to avoid detection.

But without a national sequencing effort, the U.S. leaves itself vulnerable to more serious mutations down the line. Not just of SARS-Cov-2, but of any virus.

Sequencing Covid-19 variants is a piecemeal process in the U.S., typically happening in academic institutions or local labs. Some labs are forwarding suspected variant samples to the Centers for Disease Control and Prevention for sequencing. The CDC says its working to expand its ability to track Covid-19 and new variants.

This is a preview of what will be a recurrent problem, Urnov said. The United States of America has to have a federally supported effort to sequence a lot more virus, collect the data in a national database, and make those data available to all who work on molecular test development.

Urnov equates virus mutations to an outlaw changing their appearance and requiring law enforcement officials to update the sketch on their Most Wanted poster.

In this case, the poster is a molecular test. From a virus standpoint, the way to create the sketch is through genomic sequencing. Thats how scientists detect when a virus has mutated.

Right now, it doesnt appear the mutation seriously impacts Covid-19 tests used in the U.S. The Food and Drug Administration warned this month that three tests may be impacted by genetic variants of the virus, but that the impact does not appear to be significant. Covid-19 vaccines will still work too, doctors say.

But theres no way of knowing exactly what will come down the variant pipeline, and some scientists want to be better prepared so the nation isnt caught flat-footed.

This time, we got lucky, Urnov said.

The mutation that was originally detected in the U.K. is found on the viruss spike gene, Kimberly Hanson, a physician and professor at the University of Utah School of Medicine, said on a Covid-19 testing panel in early January.

Many tests that do target that gene, like a widely-used test by Thermo Fisher Scientific, typically also target two other parts of the SARS-Cov-2 virus.

Thermo Fishers test has three gene targets, and scientists only need to hit two out of three targets to determine a test is positive and that person has Covid-19, Hanson said. So even though one testing procedure doesnt pick up the new variant, there are two others that do and act as a safety net.

Antigen tests, or rapid tests, typically target something besides the spike protein, so the vast majority of our tests should be in good shape, Angela Caliendo, a physician and professor at Alpert Medical School at Brown University, said. She spoke at the Infectious Diseases Society of America panel with Hanson.

Eric Blank, the chief program officer for the Association of Public Health Laboratories, doesnt expect major Covid-19 variants to knock the U.S. testing strategy off kilter.

He said current tests will likely still be able to detect future variants, but it remains to be seen how much the virus will mutate.

We expect well see more and more of this over time and itll be a more prevalent thing as time goes on, Blank said of virus mutations. From a public health standpoint, that means we have got to get better about getting vaccines in arms.

The incoming administration says it supports a nationwide testing effort that includes genomic sequencing. Part of the president-elects pandemic strategy is to build better preparedness, according to his pandemic response outline released Jan. 14.

Biden wants to use $11 billion for a variety of pandemic preparedness efforts, including global efforts to build the capacity required to fight COVID-19, its variants, and emerging biological threats.

Scientists hope that means that going forward, the U.S. will see a more robust, nationwide sequencing system than the current piecemeal approach.

The new administration is perfectly positioned to have might of federal government to support the integration of the might of American science, Urnov said.

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'This Time We Got Lucky': Virus Variants Reveal Gaps in Tracking - Bloomberg Law

Kathryn Anderson, a developmental biologist at the Memorial Sloan Kettering Cancer Center known for her work detailing the genetics of early embryogenesis, died November 30 at age 68.

Throughout her scientific career, Anderson used rigorous genetic screening assays to identify mutations suspected of disrupting cell division and differentiation in model systems. Having identified a gene of interest, she would then turn to a technique known as forward genetics, creating model organisms such as fruit flies and mice with a particular phenotype to better understand its molecular underpinnings. Using these tools, Anderson made important contributions to scientists understanding of several genetic pathwaysmost notably the Toll and Hedgehog pathwaysrequired for proper development of these animals.

Kathryn was fearless and very open-minded, Tatiana Omelchenko, a senior research scientist in Andersons lab who uses confocal microscopy to do live imaging of mouse embryos, tells The Scientist. Every lab has its own environment and its own mood, and when you stepped into Kathryns lab, you immediately felt very focused.

Born in La Jolla, California, in 1952, Anderson became interested in science at a young age, stemming back to an article in LIFEthat included a detailed image of a human fetus, according to an interview released shortly after her death. She attended the University of California, Berkeley, where she earned her undergraduate degree in biochemistry before heading to a graduate program in neurodevelopment at Stanford University in 1973.

Anderson left that program after only two years, earning a masters degree in neuroscience, and spent the next several years looking for her scientific niche. She enrolled briefly in medical school at the University of California, San Diego, an experience that led her to realize her love of basic research. The clinical work wasnt my cup of tea, Anderson shared in a 2005 biography. The lab was where I felt most at home.

Ultimately, Anderson landed at the University of California, Los Angeles, studying the developmental genetics of Drosophilaunder the guidance of biologist Judith Lengyel. For her PhD work, Anderson showed that in the first two hours after fertilization, the development of Drosophilaembryos remains under maternal control, with maternal RNA and proteins directing cell division and differentiation within the egg.

Looking to further her study of fruit flies, Anderson next traveled to the Max Planck Institute for Developmental Biology in Germany as a postdoc to work with Drosophilageneticist Christiane Nsslein-Volhard. In 1995, Nsslein-Volhard would share a Nobel Prize for her work using mass screenings to identify mutations that disrupt embryonic development, and Anderson would continue studying a handful of the genes identified in these early screens throughout her career.

One such gene, known as Toll, turned out to play an important role in dorsal-ventral (D-V) differentiationdictating, as Anderson said in her biography, how a fly embryo knows its back from its belly. In addition to probing the function of Toll,Anderson continued building out the wider Toll pathway after returning to the University of California, Berkeley, as an assistant professor in 1985, and later in her own lab at the Sloan Kettering Institute, which she launched in 1996 at Memorial Sloan Kettering. During this time, Anderson and her team identified roughly a dozen genes involved in cell differentiation along the D-V axis, and she used similar screening methods to better understand Tolls role in innate immunity of Drosophila. Her findings were noted by geneticists Jules Hoffmann and Bruce Beutler, whose study of Toll-like receptors in both fruit fly and mammalian immunity would later earn them a Nobel Prize.

Kathryn Anderson

Memorial Sloan Kettering Cancer Center

After her successes in fruit flies, Anderson began thinking about applying her same methods to the study of mice. She spent a year on sabbatical in the lab of Rosa Beddington at the National Institute for Medical Research in the UK, where she showed that Toll had no analogous role in the D-V differentiation of mammals. It demonstrated, she said in a 2016 interview with Development, that there are things about early mammalian development that you cant figure out by extrapolating from flies.

Back at the Sloan Kettering Institute, Anderson began once again using mass genetic screenings, this time to identify mutations of interest in mice, and then studying them in fine detail. These were lengthy experiments that often took years to yield results. I think her major contribution is discovering the functions and roles of genes through this mutagenesis screen, Omelchenko says. This is amazing because . . . the mouse embryo model is quite complex, but she did the work.

Anderson and her team screened more than 12,000 mutations, selecting roughly 40 that produce obvious phenotypic disruptions midway through gestation. Working diligently over many years, Anderson identified previously unknown pathways that have since prompted new research directions in the field of developmental biology.

Through her screening, for example, Anderson identified a previously unknown relationship between ciliamicroscopic, hairlike structures on the outside of some cellsand proper signaling of the Hedgehog pathway that dictates cell differentiation in mammalian embryos. Further research showed that components of this pathway are enriched in cilia, while mice with certain mutations in genes involved in Hedgehog signaling lacked cilia altogether in a structure called the node that directs gastrulation in vertebrate embryos. That turned out to be pretty amazing, actually: theres this whole organelle required for Hedgehog signaling in vertebrates, but not in flies, Anderson said in her Developmentinterview. Its a geneticists dream, but raises the question of why organize the genome like this: there are so many weak points in Hedgehog signalingand Hedgehog is so vital.

For her contributions to the field of developmental biology, Anderson was inducted into the National Academy of Sciences in 2002 and elected as a member of the Institute of Medicine of the National Academies in 2008. In addition, she was awarded the Thomas Hunt Morgan Medal for lifetime contributions to the science of genetics in 2012, the Federation of American Societies for Experimental Biologys Excellence in Science Award in 2014, and the Society for Developmental Biologys Edwin G. Conklin Medal for distinguished and sustained research in 2016, among other honors.

Prior to her death, Anderson had spoken about the possible extension of her research into human genetics, as disruptions in hedgehog signaling have since been linked both to birth defects and to a series of diseases referred to as ciliopathies. It was, however, a line of questioning she planned to leave to other scientists, content to continue her methodical work exploring mutations in mice.

Many scientists are very quiet people, but contemporary society requires you to be very loud [so] that people will listen to you, Omelchenko says. Kathryn is such a great example of being quiet, being a very deep thinker, and at the same time becoming a very successful and bright scientist. I think I will keep learning from her even though she has passed away.

Anderson is survived by her husband, Timothy Bestor, a geneticist at Columbia University.

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Developmental Biologist Kathryn Anderson Dies at 68 - The Scientist

PENNINGTON, N.J. and SAN DIEGO, Jan. 8, 2021 /PRNewswire/ -- OncoSec Medical Incorporated (NASDAQ:ONCS) (the "Company" or "OncoSec") today announced the first patient was dosed in OMS-104, an investigator-initiated Phase 2 trial evaluating TAVO (tavokinogene telseplasmid), the Company's intratumoral DNA plasmid-based interleukin-12 (IL-12) therapy administered using its gene delivery platform (gene electrotransfer), in combination with the anti-PD-1 checkpoint inhibitor OPDIVO (nivolumab) as a neoadjuvant therapy prior to surgery in patients with operable, locally or regionally advanced melanoma. The trial is designed to evaluate if the addition of TAVO can improve clinical outcomes already observed when using nivolumab alone as a neoadjuvant therapy.

Anti-PD1 checkpoint inhibitors, when administered as a neoadjuvant therapy, have shown encouraging clinical results, but rapid recurrence remains an issue for many patients. TAVO in combination with OPDIVO may drive deep anti-tumor immune responses and complete elimination of tumors prior to surgery, leading to improved long-term clinical outcomes for a significant proportion of treated patients. TAVO in combination with another anti-PD-1 checkpoint inhibitor, KEYTRUDA (pembrolizumab), has already been shown to enhance overall response rate and partial tumor responses in patients with anti-PD-1 checkpoint-refractory metastatic melanoma in OncoSec's KEYNOTE-695 registration directed Phase 2 clinical trial.

"While studies have shown relapse and overall survival advantages when checkpoint inhibitors are given alone following surgery, there is a need to investigate novel immunotherapeutic agents such as TAVO that can be given preoperatively in order to further enhance the clinical efficacy of immunotherapy in patients with advanced melanoma," said Armad A. Tarhini, M.D., Ph.D., Leader of the OMS-104 trial and Senior Member and Professor at the H. Lee Moffitt Cancer Center and Research Institute and the University of South Florida Morsani College of Medicine. "The neoadjuvant approach utilizing TAVO in combination with checkpoint inhibitors as being tested in this study may improve operability, pathologic tumor response and long-term disease control, which is highly desirable for these patients, who continue to have a high risk of recurrence and progression despite the use of standard therapy after surgery."

OMS-104 (NCT04526730) is a Phase 2 open-label, single arm study investigating intratumoral TAVO delivered by gene electrotransfer, or short electric pulses, plus nivolumab as neoadjuvant therapy in patients with operable locally-regionally advanced melanoma. The trial aims to enroll 33 patients and consists of three phases:

1) Neoadjuvant phase, where TAVO will be administered intratumorally using gene electrotransfer in three cycles on days one and eight every four weeks and nivolumab will be administered after TAVO on day eight of each cycle via 30-minute intravenous (IV) infusion;

2) Surgical phase consisting of a definitive surgery that will be scheduled 2-4 weeks after the last dose of nivolumab following radiologic and clinical assessment; and

3) Adjuvant phase, where nivolumab monotherapy will begin 2-4 weeks after surgery and will be administered for up to nine four-week cycles.

The primary endpoint is pathological complete response, estimated based on the proportion of participants with no viable tumor on histologic assessment at definitive surgery after the 12-week neoadjuvant period.

Daniel J. O'Connor, President and Chief Executive Officer of OncoSec, added, "TAVO delivers DNA plasmid-based IL-12 directly into the tumor using gene electrotransfer, which demonstrably enhances the immunogenicity of the treated tumors to yield productive 'in situ' vaccines. This principle has yielded striking results in post-PD-1 patients and is likely relevant in this earlier clinical setting. We look forward to exploring the utility of TAVO as a potential neoadjuvant therapy in a variety of solid tumor settings for patients in need of more effective treatment options."

About TAVOOncoSec's gene delivery technology combines TAVO(tavokinogene telseplasmid), a DNA plasmid-based interleukin-12 (IL-12), with an intra-tumoral gene delivery platform (gene electrotransfer) to achieve endogenous IL-12 production in the tumor microenvironment that enables the immune system to target and attack tumors throughout the body. TAVO has demonstrated a local and systemic anti-tumor response in several clinical trials, including the pivotal Phase 2b trial KEYNOTE-695 for metastatic melanoma and the KEYNOTE-890 Phase 2 trial in triple negative breast cancer (TNBC). TAVO has received both Orphan Drug and Fast-Track Designation by the U.S. Food & Drug Administration for the treatment of metastatic melanoma.

About OncoSec Medical IncorporatedOncoSec Medical Incorporated (the "Company," "OncoSec," "we" or "our") is a late-stage biotechnology company focused on developing cytokine-based intratumoral immunotherapies to stimulate the body's immune system to target and attack cancer. OncoSec's lead immunotherapy investigational product candidate TAVO (tavokinogene telseplasmid) enables the intratumoral delivery of DNA-based interleukin-12 (IL-12), a naturally occurring protein with immune-stimulating functions. The technology, which employs geneelectrotransfer, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body. OncoSec has built a deep and diverse clinical pipeline utilizing TAVO as a potential treatment for multiple cancer indications either as a monotherapy or in combination with leading checkpoint inhibitors; with the latter potentially enabling OncoSec to address a great unmet medical need in oncology: anti-PD-1 non-responders. Results from recently completed clinical studies of TAVO have demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach along with an acceptable safety profile, warranting further development. In addition to TAVO, OncoSec is identifying and developing new DNA-encoded therapeutic candidates and tumor indications for use with its new Visceral Lesion Applicator (VLA), to target deep visceral lesions, such as liver, lung or pancreatic lesions. For more information, please visitwww.oncosec.com.

TAVO is a trademark of OncoSec Medical Incorporated.

KEYTRUDAis a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

OPDIVO is a registered trademark of Bristol-Myers Squibb Company.

Risk Factors and Forward-Looking StatementsThis release, as well as other information provided from time to time by the Company or its employees, may contain forward-looking statements that involve a number of risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Forward-looking statements provide the Company's current beliefs, expectations and intentions regarding future events and involve risks, uncertainties (some of which are beyond the Company's control) and assumptions. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. You can identify forward-looking statements by the fact that they do not relate strictly to historical or current facts. These statements may include words such as "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "should," "will" and "would" and similar expressions (including the negative of these terms). Although we believe that expectations reflected in the forward- looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. The Company intends these forward-looking statements to speak only at the time they are published on or as otherwise specified and does not undertake to update or revise these statements as more information becomes available, except as required under federal securities laws and the rules and regulations of the Securities Exchange Commission ("SEC"). In particular, you should be aware that the success and timing of our clinical trials, including safety and efficacy of our product candidates, patient accrual, unexpected or expected safety events, the impact of COVID-19 on the supply of our candidates or the initiation or completion of clinical trials and the usability of data generated from our trials may differ and may not meet our estimated timelines. Please refer to the risk factors and other cautionary statements provided in the Company's Annual Report on Form 10-K for the fiscal year ended July 31, 2020 and subsequent periodic and current reports filed with the SEC (each of which can be found at the SEC's website http://www.sec.gov), as well as other factors described from time to time in the Company's filings with the SEC.

Company ContactKim Jaffe, Ph.D.Assistant Vice President, Business Development & Operations+1-858-210-7330 [emailprotected]

Media ContactPatrick Bursey LifeSci Communications+1-646-970-4688[emailprotected]

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http://www.oncosec.com

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OncoSec Announces First Patient Dosed in Phase 2 Trial of TAVO Plus OPDIVO as Neoadjuvant Therapy for Melanoma - PRNewswire

WASHINGTON, Dec. 15, 2020 /PRNewswire/ --Over the last 30 years, significant progress has been made in the fight against cancer. Researchers have expanded their understanding of how cancer develops and how to target medicines for specific cancer types. Since peaking in 1991, the death rate associated with cancer declined by 29%, which translates to 2.9 million fewer cancer deaths. The most recent data shows that between 2016 and 2017 alone, cancer death rates declined by 2.2%, the largest single-year drop ever recorded. Despite the challenges imposed by the COVID-19 pandemic, this momentum continues with biopharmaceutical companies focusing on research and development of innovative cancer therapies.

Still, cancer remains the second leading cause of death in the United States, accounting for 21% of all deaths. It is estimated that new cancer cases reached 1.8 million in 2020, increasing demand for earlier screening and diagnosis, as well as new treatments to address substantial unmet medical needs so patients can continue to live long and healthy lives.

To continue the progress and deliver hope to those battling cancer, biopharmaceutical research companies are working to develop more effective and better tolerated treatments.

A new report today from PhRMA finds that more than 1,300 medicines and vaccines for various cancers are currently in development, either in clinical trials or awaiting review by the U.S. Food and Drug Administration.

New medicines have played a key role in cancer survival gains, much of which are driven by advances in molecular and genomic research that have revealed the unique complexities of cancer and changed our understanding of the disease. Examples of the science behind potential new cancer treatments include:

The more than 1,300 medicines and vaccines in development represent an increased recognition among researchers that no two cancers are alike, which has led to further adoption of personalized medicine and the creation of treatments to target cancers specific to a single person. As researchers continue to explore life-saving methods and technologies to fight cancer, it is important we foster an innovation ecosystem that encourages ongoing research and development in this space.

To read the new report on medicines and vaccines in clinical testing for various cancers, click here.

Learn more about cancer at PhRMA.org/Cancer

SOURCE Pharmaceutical Research and Manufacturers of America (PhRMA)

http://phrma.org

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Report: More than 1,300 Medicines and Vaccines in Development to Help Fight Cancer - PRNewswire