NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces presentations at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. There will be two oral presentations highlighting clinical data from the Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs as well as a poster presentation highlighting preliminary clinical data from the Pyruvate Kinase Deficiency (PKD) program. All three programs utilize Rocket's "Process B" manufacturing platform.

Details for Rockets presentations are as follows:

Oral Presentations Title:Gene Therapy for Fanconi Anemia, Complementation Group A: Updated Results from Ongoing Global Clinical Studies of RP-L102Session Title:Gene Editing, Therapy and Transfer IPresenter:Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineSession Date:Monday, December 7, 2020Session Time:11:30 a.m. - 1:00 p.m. (Pacific Time)Presentation Time: 12:15 p.m. (Pacific Time)

Title:Phase 1/2 Study of Lentiviral-MediatedEx-VivoGene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1Session Title:Gene Editing, Therapy and Transfer IPresenter:Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date:Monday, December 7, 2020Session Time:11:30 a.m. - 1:00 p.m. (Pacific Time)Presentation Time: 12:30 p.m. (Pacific Time)

Poster PresentationTitle: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric PatientsSession Title: Gene Editing, Therapy and Transfer: Poster IIPresenter: Jos Luis Lpez Lorenzo, M.D., Hospital Universitario Fundacin Jimnez Daz, Madrid, SpainSession Date: Sunday, December 6, 2020Session Time: 7:00 a.m. 3:30 p.m. (Pacific Time)

About Fanconi AnemiaFanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-ISevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase DeficiencyPyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

See the original post:
Rocket Pharmaceuticals to Present Data from its Fanconi Anemia, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency Programs at the 62nd...

Avantor executives joined other industry leaders at virtual Cell & Gene Therapy Bioprocessing & Commercialization Conference

Panels discussed critical topics including process efficiency and scaling solutions

RADNOR, Pa., Nov. 2, 2020 /PRNewswire/ --Executives from Avantor Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, recently provided expert insight at the Cell & Gene Therapy Bioprocessing & Commercialization Virtual Conference. Cell and gene therapy (C>) are two of the most revolutionary applications driving the biopharmaceutical industry.

In separate panel discussions with other global industry leaders, Dr. Ger Brophy, EVP, Biopharma Production at Avantor and Claudia Berrn, SVP, Business Development and Commercial Operations at Avantor, each addressed novel considerations and challenges facing the biopharmaceutical industry, including the impact of COVID-19. They highlighted innovation to help drive the creation and commercialization of life-changing, personalized C> treatments.

In a panel on the evolution of C> and the path toward scalability and manufacturability of these treatments, Dr. Brophy gave his perspective on this growing need for the bioprocessing industry.

"Genuine progress is being made in the long-standing battle to effectively treat and control disease, and cell & gene therapies will only continue to unlock new frontiers in medicine," said Dr. Brophy. "We're beginning to see more clearly the issues that need to be addressed, and know that if products are to scale and become more accessible to patients worldwide, there needs to be efficiency in operations. There is an absolute requirement for automation, both to reduce variability and to generate process efficiencies. At Avantor, we are ready to make the essential materials and technologies available to companies leading the charge and treating patients. Innovation and agility are central to how we're partnering with the industry to resolve these issues."

On a separate panel, Ms. Berrn highlighted how the industry is navigating the challenges and impact of the global pandemic, from the fragmentation of supply chains to clinical trial disruption.

"In this critical moment for the industry and the world, it is more important than ever for leaders in the cell & gene therapy community to come together to strategize and share ideas," said Ms. Berrn. "Avantor is actively working with the world's leading pharmaceutical and biotechnology companies to accelerate the production of novel treatments. Collectively, our goal is to mitigate any challenges in the process of taking a groundbreaking treatment from its initial scientific discovery to delivery in treating patients."

Avantor provides products for biopharma production workflows, including cell and gene therapy offering. Search 'Avantor biopharma' in your browser.

About AvantorAvantor, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. One of our greatest strengths comes from having a global infrastructure that is strategically located to support the needs of our customers. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries.We set science in motion to create a better world. For information, visit avantorsciences.com and find us on LinkedIn, Twitter and Facebook.

Robert DonohoeSenior Director, Corporate CommunicationsAvantorM: 484-688-4730[emailprotected]

Source: Avantor and Financial News

SOURCE Avantor and Financial News

http://avantorsciences.com

Continued here:
Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 - PRNewswire

While the US Food and Drug Administration (FDA) is still receiving investigational new drug applications (INDs) for cell and gene therapies, officials are concerned about the impact of the COVID-19 pandemic on clinical trials.Its clear that COVID-19 has adversely affected all aspects of development of cell and gene therapies, said Peter Marks, director of FDAs Center for Biologics Evaluation and Research (CBER), said at the Alliance for Regenerative Medicines Meeting on the Mesa. For some of the studies that are ongoing there are some real challenges to overcome in terms of endpoints that may have been missed.The pandemic also has disrupted global harmonization efforts around gene therapies, Marks said.We were on the cusp, in fact, working with global regulators trying to get towards more harmonization of gene therapy programs in different countries, he said. Were trying to keep it moving but its a challenge to do.Marks noted that before COVID-19 he spent about 75% of his time on cell and gene therapies, but the pandemic has forced him to shift priorities. Some things have less policy demands at this point in time. At this point in time its very much reversed and its probably 80% of my time on COVID-related activities.Marks also noted that CBERs Office of Tissues and Advanced Therapies (OTAT) has been struggling to keep up with its workload even before the pandemic. With the influx of applications for cell and gene therapies over the last five years, Marks said the office, Should have doubled in size and its only modestly larger, 15-20% larger in size.Marks said he is not satisfied with the level of dialogue the agency has been able to have with gene therapy developers. Especially early on, we should be able to have this dialogue that really facilitates setting things up well so that our knowledge of the entire fieldwe help leverage that for every sponsor.Weve been so strapped in terms of personnel that its hard to do that, Marks said, noting that COVID-19 has exacerbated things even further. Because the number of gene therapy applications hasnt fallen off dramatically, some of the trials may not be moving as quickly, but the applications keep coming in. Marks said that OTAT has also had to shift priorities during the pandemic and that he hopes the next user fee cycle will bring in the resources necessary to staff up further.Speaking on a separate panel with members of industry, OTAT Director Wilson Bryan echoed Marks sentiment.We were stretched thin before the pandemic, and with the flood of work that came in, it really had an impact, he said. Sometimes folks dont like to admit this, but we all know weve had delayed meetings, weve had to delay review of some applications because of giving priority to the pandemic.However, Bryan said the office is getting its balance and is working to catch up on some of its delayed activities.Bryan expressed some worry about the financial well-being of some of the smaller companies his office works with. Were hearing a lot about their struggles to stay afloat and continue and finish off their development programs and whether or not those development programs are going to be sufficient to meet regulatory standards, he said.One of the challenges, said Timothy Schroeder, CEO of CTI Clinical Trial & Consulting, will be dealing with gaps in data from clinical trials. The question is going to be how do sponsors, how do regulatory authorities and how do companies such as ourselves fill those gaps?On the regulator side, Bryan said his office is working with companies on an individual basis to sort out those issues, which differ from one indication to the next.Bryan added that one positive to come of the pandemic is greater interest in remote outcome assessments in clinical trials. If we have an energy now to develop outcome measures and validate outcome measures that allow us to reliably capture information from patients in remote locations, that will ultimately facilitate development, he said.The pandemic also has significantly disrupted FDAs ability to conduct surveillance and preapproval inspections. While the agency has resumed some domestic inspections and mission-critical foreign inspections, it also is leveraging other sources of information, including inspection reports from other regulators, and requesting documents from applicants and facilities in lieu of on-site inspections where possible. (RELATED: FDA issues pandemic inspections FAQ guidance, Regulatory Focus 19 August 2020).Were considering virtual inspections, particularly for companies where the site has a track record, but if its a site that is brand new with no track record or if its a site with that has a bad track record, were hesitant to do that, Bryan said.Bryan also raised the prospect of FDA inspectors tagging along remotely for an inspection being conducted by other regulators. Is it possible that we could have an inspection by European inspectors and have US regulators going along for a virtual inspection at the same time? We think about those things, I dont know that weve done them yet, Bryan said, adding that he is not sure whether FDA inspectors would be comfortable with the information they would get.Curran Simpson, chief operations and technology officer at REGENXBIO, said he sees promise in virtual audits and believes the level of documentation a site provides can be indicative of its compliance.How often have I walked into a manufacturing facility thats well-run but has terrible documentation? Almost never. I think virtual audits, if you do a risk-based approach and the audit partner has the ability to send documentation in an efficient way and you have experienced people doing this, I think youre going to get the same flavor of an audit very quickly from the level of the documentation, he said.Of course, youll want to accompany that to the extent possible with imaging of the facility, Curran said, To see if those practices are being followed, the overall cleanliness of the facility and the management of material movement If you dont get a good impression from the documentation that youre working through, its probably a bigger issue that you want to escalate.Amy DuRoss, co-founder and CEO of Vineti, an enterprise software company specializing in advanced therapies, expressed some doubts about the current potential for fully remote audits.Certainly our piece of the chain because were enterprise software is readily auditable remotely, but I would say that the overall system and in manufacturing, Im not sure weve evolved as a species yet to adapt our remote techniques to get a full picture I dont think were there yet, she said.

Read this article:
FDA officials, experts discuss impact of COVID-19 on cell and gene therapies - Regulatory Focus

SINGAPORE - "When will YOU win a Nobel Prize?"

Scientists receive regular reminders of how important this prize is in their relatives' (and the public's) mind. For many, the Nobel Prize is their idea of science.

Indeed these are the most famous awards in medicine, chemistry and physics. Scientists also pay attention to the Nobel Prizes because we love science.

It is only natural to chat and debate about the most significant recent discovery in our field.

This requires broad perspective and context.

With only one prize each year, how can a breakthrough in cancer therapy be compared with or ranked against a fundamental discovery about infections?

This must be particularly stressful for the group that meets regularly in Sweden to decide who is to win the most coveted prize in science.

Last week, the 2020 Nobel Prizes were announced for the discovery of the Hepatitis C virus (physiology or medicine), advancing our understanding of black holes (physics), and a technology for editing genomes (chemistry).

As a scientist studying the genomes or genetic make-up of bacteria, I want to provide a broader perspective on the latter to give some insight into where we might fruitfully invest in more research.

Dr Emmanuelle Charpentier and Dr Jennifer Doudna discovered one of gene technology's sharpest tools: the CRISPR/Cas9 genetic scissors.

Using these, researchers can change the DNA of animals, plants and micro-organisms with extremely high precision, reads the statement by the Royal Swedish Academy of Sciences, in explaining the decision to award them the prize.

"This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true," it said.

The two women used a system called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) to edit a genome.

A genome is the entire set of DNA in an organism.

French scientist Emmanuelle Charpentier is one of the two winners of the 2020 Nobel Prize in Chemistry. PHOTO: EPA-EFE

You may remember from school that your body is made up of individual cells; each cell has a nucleus, and inside the nucleus is DNA - the "instruction set for life". Your DNA is unique - these differences make you unique, specifying the colour of your eyes and your general height and weight, among many other traits.

Certain differences can influence your susceptibility to diseases such as diabetes, cancer or Alzheimer's.

Interestingly, we know that DNA differences do influence risk for these diseases, but we often don't know exactly what DNA changes are responsible, much less how this takes place.

But for some diseases, such as sickle cell anaemia, Duchenne muscular dystrophy or cystic fibrosis, we know exactly what DNA changes cause the disease.

If we could "edit" these specific changes, we would completely cure the disease.

Genome editing means making precise changes to the DNA of an organism, and it therefore has the potential to cure - not just treat - any disease caused by DNA differences.

Genome editing, therefore, has a huge range of applications.

The CRISPR system is a set of molecules that was originally discovered in bacteria.

Why did it win the Nobel prize? One key feature of CRISPR is that it is programmable and general.

There have been other technologies in the past that could be used to edit genomes; but CRISPR made it dramatically easier because it was the first technology that could be easily changed to target the sickle cell DNA mutation in one experiment, and then the cystic fibrosis mutation, with just a minor change, in the next experiment.

This programmability made it applicable to any genome edit we could ever want to make.

But though important, programmability was not quite enough.

What made CRISPR a front runner in Nobel Prize discussions in cafes across the world (or, more precisely, cafes across the street from research labs across the world) was that it was also a relatively small, independently functioning protein machine.

In other words, we could take just the CRISPR components, and they would work the same way in nearly any cell type - a heart cell, nerve cell, immune cell or even an insect cell, yeast cell or bacterial cell.

The system requires only a few components, then it performs its function in nearly any cell we put it in - it's a self-sufficient, all-in-one module.

Interestingly, the CRISPR system was extracted from bacteria, where it was used as a sort of immune system. In bacteria, the CRISPR system seems to have evolved to be self-contained, so that it could be transferred to other bacteria and still function properly.

These two features - programmability and modularity - are two advantages that CRISPR improved on, compared with other genome editing technologies.

This is why it has had such a broad impact on biology, and researchers are using CRISPR in nearly every imaginable cell type, to make better plants for farming, customised bacteria for biotechnology, modified mosquitoes for vector-borne diseases and fundamental research to find the DNA changes we'll later want to edit, to cure diseases.

Looking more broadly, we have seen these key features in other Nobel Prize-winning technologies.

There is a very strong parallel with restriction enzymes (another DNA-cutting system), the discovery of which garnered a Nobel Prize in 1978.

A revolution in biology has been already wrought by restriction enzymes, and scientists expect CRISPR will herald another biological revolution of similar scale.

The use of restriction enzymes allowed for recombinant DNA engineering and molecular cloning.

In fact, nearly all of molecular biology was advanced by the clever application of restriction enzymes.

Like CRISPR, restriction enzymes come from bacteria. They can also be transferred between different bacteria and still function - they have the property of self-contained modularity.

In fact, many of the foundational technologies that support modern biology are derived from bacteria.

Many are modular - this seems to be something that evolution particularly selects for in bacteria, perhaps because they can easily transfer DNA among themselves.

Modularity, in turn, makes these bacterial systems highly efficient and easily extracted, so that we can use them for our own purposes in test tubes and in other cells - whether bacterial, plant or human.

Other examples of bacterial-related Nobel Prizes are: streptomycin as a treatment for tuberculosis (1953); transfer of DNA between bacteria (1958); the protein that copies DNA (a polymerase) (1959); the details of how cells respond to changing conditions (1965); application of DNA polymerases in polymerase chain reaction (1993); and bacterial phages for directed evolution (2018).

Bacteria are an enormously diverse group of organisms; what we call a single species of bacteria has individuals that are more different than humans are from chimpanzees.

Therefore, bacterial research is amazingly rich - which is why they have contributed so much to all of biology, a tiny fraction of which is recognised by Nobel Prizes.

The diversity of bacteria means that, for nearly any process or function we might need - be it copying DNA or editing the genome - there is likely some bacteria that has highly optimised that process.

Bacteria are, in fact, the Olympic athletes of any biological process.

The fact that many bacteria can exchange DNA also means that many of their molecular machines are self-contained and modular, and we can reasonably expect them to still work when isolated or transplanted, allowing us to repurpose them for our own ends.

In Singapore, there is a strong community of people working on bacteria and their molecular systems.

Some are working downstream on optimisation and application of CRISPR.

Others, like my lab, are doing more foundational work, the discovery science that comes before these prizes: understanding the process of DNA transfer, characterising the diversity of bacteria and delving into the details of how these bacterial machines work.

There is also a community of bacteriologists (called the Bacterial Ultra Group, or BUG) looking directly at the details for how bacteria cause disease, the direct interaction interface between bacteria and humans.

Bacteria are endlessly fascinating, and we have much more to learn from them.

More Nobel Prize-worthy discoveries and technologies are certainly lying in wait for us within these diverse organisms.

Dr Swaine Chen is an associate professor of medicine in the Infectious Diseases Programme at the Yong Loo Lin School of Medicine at the National University of Singapore, and a group leader in bacterial genomics at the Genome Institute of Singapore, Agency for Science, Technology and Research.

His research uses genome sequencing and genome manipulation to understand bacterial diversity and how bacteria cause infections in humans, such as the yusheng-associated outbreak of Group B Streptococcus infections in Singapore in 2015.

The overall aim of his research is to enable faster detection and more effective treatment of bacterial diseases.

In 2012, he was one of the cofounders of the Bacterial Ultra Group (BUG), an active research community in Singapore that studies the mechanisms and spread of antibiotic resistance and details of the strategies that bacteria use to infect humans.

You can find out more about BUG and Dr Chen's research on Twitter (@swaine_chen) or at this website.

See original here:
Genetic scissors from bacteria, a tool to slice and dice code of life - The Straits Times

WALTHAM, Mass., Oct. 15, 2020 (GLOBE NEWSWIRE) -- OncXerna Therapeutics, Inc., a precision medicine company using an innovative RNA-based biomarker platform to predict patient responses for potentially first-in-class targeted oncology therapies, today announced that Laura Benjamin, Ph.D., Founder and CEO of OncXerna, will participate at the Jefferies Virtual London Healthcare Conference taking place November 17-19, 2020.

About OncXerna Therapeutics

OncXerna is aiming to deliver next-generation precision medicine for a larger group of cancer patients by leveraging the companys deep understanding of how to prospectively identify patients based on the dominant, RNA-based biology of their tumor microenvironments. This allows OncXerna to pair those patients with OncXernas clinical-stage therapies and known mechanism of action that directly address these biologies, to dramatically improve patient outcomes. For more information on OncXerna, please visit oncxerna.com/

About OncXernas RNA-based Biomarker Platform

Existing precision medicines target only approximately 10% of cancersthose with gene mutations or oncogenic drivers for a small number of genes. Using its proprietary biomarker platform, OncXerna is leveraging the companys deep understanding of tumor biology at the RNA level to identify the dominant biology underlying a patients cancer. OncXernas first biomarker panel is specific to the tumor microenvironment (TME Panel-1). Initial results from TME Panel-1 reveal 4 different dominant biologies, demonstrating the presence of specific patient subgroups and their predictive value in responding to treatment. OncXerna is further optimizing the biomarker platforms tumor microenvironment panel through multiple research collaborations, including a collaboration with Moffitt Cancer Center.

About Bavituximab

Bavituximab is an investigational antibody that reverses immune suppression by inhibiting phosphatidylserine (PS) signaling and is currently in Phase 2 clinical trials to treat a specific subset of patients with advanced gastric cancer to improve their response to anti-PD-1 treatment. The mechanism of action of bavituximab is to block tumor immune suppression signaling from PS to multiple immune cell receptor families (e.g., TIMs and TAMs). The dominant biology targeted by bavituximab may be relevant for patients with many types of solid tumors whose immune systems are too suppressed to benefit from currently available immune oncology therapies. OncXernas clinical trials currently combine bavituximab with KEYTRUDA to test the hypothesis that relieving immunosuppression can enhance responses to checkpoint inhibitors. Bavituximab is an investigational agent that has not been licensed or approved anywhere globally, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced gastric cancer.

About Navicixizumab

Navicixizumab is an investigational anti-DLL4/VEGF bispecific antibody that has demonstrated antitumor activity in patients who have progressed on Avastin (bevacizumab) in a Phase 1a/b clinical trial. The U.S. Food and Drug Administration granted Fast Track designation to navicixizumab for the treatment of high-grade ovarian, primary peritoneal or fallopian tube cancer in patients who have received at least three prior therapies and/or prior treatment with Avastin. OncXerna is targeting patients whose dominant tumor biology is driven by angiogenesis with a focus beyond VEGF to include broader anti-angiogenic pathways. Navicixizumab is an investigational agent that has not been licensed or approved anywhere globally, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced ovarian cancer.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Investor and Media Contact:

Ashley R. RobinsonLifeSci Partners, LLCarr@lifesciadvisors.com

Read the original here:
OncXerna Therapeutics to Participate at the Jefferies Virtual London Healthcare Conference - GlobeNewswire

While the FDA is still receiving investigational new drug applications (INDs) for cell and gene therapies, officials are concerned about the impact of the Covid-19 pandemic on clinical trials.

Its clear that COVID-19 has adversely affected all aspects of development of cell and gene therapies, said CBER director Peter Marks at the Alliance for Regenerative Medicines Meeting on the Mesa. For some of the studies that are ongoing there are some real challenges to overcome in terms of endpoints that may have been missed.

The pandemic also has disrupted global harmonization efforts around gene therapies, Marks said.

We were on the cusp, in fact, working with global regulators trying to get towards more harmonization of gene therapy programs in different countries, he said. Were trying to keep it moving but its a challenge to do.

Marks noted that before Covid-19 he spent about 75% of his time on cell and gene therapies, but the pandemic has forced him to shift priorities. Some things have less policy demands at this point in time. At this point in time its very much reversed and its probably 80% of my time on COVID-related activities.

Marks also noted that CBERs Office of Tissues and Advanced Therapies (OTAT) has been struggling to keep up with its workload even before the pandemic. With the influx of applications for cell and gene therapies over the last five years, Marks said the office, Should have doubled in size and its only modestly larger, 15-20% larger in size.

Marks said he is not satisfied with the level of dialogue the agency has been able to have with gene therapy developers. Especially early on, we should be able to have this dialogue that really facilitates setting things up well so that our knowledge of the entire fieldwe help leverage that for every sponsor.

Weve been so strapped in terms of personnel that its hard to do that, Marks said, noting that Covid-19 has exacerbated things even further. Because the number of gene therapy applications hasnt fallen off dramatically, some of the trials may not be moving as quickly, but the applications keep coming in. Marks said that OTAT has also had to shift priorities during the pandemic and that he hopes the next user fee cycle will bring in the resources necessary to staff up further.

Speaking on a separate panel with members of industry, OTAT Director Wilson Bryan echoed Marks sentiment.

We were stretched thin before the pandemic, and with the flood of work that came in, it really had an impact, he said. Sometimes folks dont like to admit this, but we all know weve had delayed meetings, weve had to delay review of some applications because of giving priority to the pandemic.

However, Bryan said the office is getting its balance and is working to catch up on some of its delayed activities.

Bryan expressed some worry about the financial well-being of some of the smaller companies his office works with. Were hearing a lot about their struggles to stay afloat and continue and finish off their development programs and whether or not those development programs are going to be sufficient to meet regulatory standards, he said.

One of the challenges, said Timothy Schroeder, CEO of CTI Clinical Trial & Consulting, will be dealing with gaps in data from clinical trials. The question is going to be how do sponsors, how do regulatory authorities and how do companies such as ourselves fill those gaps?

On the regulator side, Bryan said his office is working with companies on an individual basis to sort out those issues, which differ from one indication to the next.

Bryan added that one positive to come of the pandemic is greater interest in remote outcome assessments in clinical trials. If we have an energy now to develop outcome measures and validate outcome measures that allow us to reliably capture information from patients in remote locations, that will ultimately facilitate development, he said.

The pandemic also has significantly disrupted FDAs ability to conduct surveillance and preapproval inspections. While the agency has resumed some domestic inspections and mission-critical foreign inspections, it also is leveraging other sources of information, including inspection reports from other regulators, and requesting documents from applicants and facilities in lieu of on-site inspections where possible.

Were considering virtual inspections, particularly for companies where the site has a track record, but if its a site that is brand new with no track record or if its a site with that has a bad track record, were hesitant to do that, Bryan said.

Bryan also raised the prospect of FDA inspectors tagging along remotely for an inspection being conducted by other regulators. Is it possible that we could have an inspection by European inspectors and have US regulators going along for a virtual inspection at the same time? We think about those things, I dont know that weve done them yet, Bryan said, adding that he is not sure whether FDA inspectors would be comfortable with the information they would get.

Curran Simpson, chief operations and technology officer at Regenxbio, said he sees promise in virtual audits and believes the level of documentation a site provides can be indicative of its compliance.

How often have I walked into a manufacturing facility thats well-run but has terrible documentation? Almost never. I think virtual audits, if you do a risk-based approach and the audit partner has the ability to send documentation in an efficient way and you have experienced people doing this, I think youre going to get the same flavor of an audit very quickly from the level of the documentation, he said.

Of course, youll want to accompany that to the extent possible with imaging of the facility, Simpson said, To see if those practices are being followed, the overall cleanliness of the facility and the management of material movement If you dont get a good impression from the documentation that youre working through, its probably a bigger issue that you want to escalate.

Amy DuRoss, co-founder and CEO of Vineti, an enterprise software company specializing in advanced therapies, expressed some doubts about the current potential for fully remote audits.

Certainly our piece of the chain because were enterprise software is readily auditable remotely, but I would say that the overall system and in manufacturing, Im not sure weve evolved as a species yet to adapt our remote techniques to get a full picture I dont think were there yet, she said.

RAPS: First published in Regulatory Focus by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.

The rest is here:
FDA officials, experts discuss impact of Covid-19 on cell and gene therapies - Endpoints News

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Regenerative Medicine Market by Product, Application, Geography - Global Forecast to 2025" - https://www.reportlinker.com/p04700208/?utm_source=GNW However, the high cost of cell and gene therapies and ethical concerns related to the use of embryonic stem cells in research and development are expected to restrain the growth of this market during the forecast period.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market, by product, during the forecast periodBased on products, the regenerative medicine market is segmented into tissue-engineered products, cell therapies, gene therapies, and progenitor and stem cell therapies.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market in 2019.

The increasing adoption of tissue-engineered products for the treatment of chronic wounds and musculoskeletal disorders and the rising funding for the R&D of regenerative medicine products and therapies are the major factors driving the growth of this segment.

Oncology segment accounted for highest CAGRBased on applications, the regenerative medicine market is segmented into musculoskeletal disorders, wound care, oncology, ocular disorders, dental, and other applications.In 2019, the oncology segment accounted for the highest growth rate.

This can be attributed to the rising prevalence of orthopedic diseases, growing geriatric population, increasing number of stem cell research projects, growing number of clinical researches/trials, and the rich pipeline of stem cell products for the treatment of musculoskeletal disorders.

Europe: The fastest-growing region regenerative medicine marketThe global regenerative medicine market is segmented into North America, Europe, the Asia Pacific, and Rest of the World.The North America region is projected to grow at the highest CAGR during the forecast period in 2019.

The growth in the North American regenerative medicine market can be attributed to rising stem cell banking, tissue engineering, and drug discovery in the region; expansion of the healthcare sector; and the high adoption of stem cell therapy and cell immunotherapies for the treatment of cancer and chronic diseases.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 20%, Tier 2 - 45%, and Tier 3 - 35% By Designation: C-level - 30%, D-level - 20%, and Others - 50% By Region: North America - 36%, Europe - 25%, Asia Pacific - 27%, and Rest of the World 12%

Lits of companies Profiled in the Report: 3M (US) Allergan plc (Ireland) Amgen, Inc. (US) Aspect Biosystems (Canada) bluebird bio (US) Kite Pharma (US) Integra LifeSciences Holdings Corporation (US) MEDIPOST Co., Ltd. (South Korea) Medtronic plc (Ireland) Anterogen Co., Ltd. (South Korea) MiMedx Group (US) Misonix (US) Novartis AG (Switzerland) Organogenesis Inc. (US) Orthocell Limited (Australia) Corestem, Inc. (South Korea) Spark Therapeutics (US) APAC Biotech (India) Shenzhen Sibiono GeneTech Co., Ltd. (China) Smith & Nephew plc (UK) Stryker Corporation (US) Takeda Pharmaceutical Company Limited (Japan) Tego Science (South Korea) Vericel Corporation (US) Zimmer Biomet (US)

Research Coverage:This report provides a detailed picture of the global regenerative medicine market.It aims at estimating the size and future growth potential of the market across different segments, such as product, application, and region.

The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall regenerative medicine market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to position their business better and make suitable go-to-market strategies.

This report will enable stakeholders to understand the pulse of the market and provide them with information on the key market drivers, restraints, opportunities, and trends.

Read the full report: https://www.reportlinker.com/p04700208/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

See the original post:
The global regenerative medicine market is projected to reach USD 17.9 billion by 2025 from USD 8.5 billion in 2020, at a CAGR of 15.9% -...

-- Continued functional improvements were observed at 18 months in the low-dose cohort --

-- First look at functional outcomes in high-dose cohort found improvements 6 months after administration --

-- Results in both cohorts continue to reinforce safety and tolerability profile of SRP-9003 --

CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Companys investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included 18-month functional data from three clinical trial participants in the low-dose cohort and 6-month functional data from three participants in the high-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

There are currently no approved treatments for people with LGMD2E a disease that causes significant disability in children and often leads to early mortality. Its very encouraging that we continue to see consistent, positive data from our investigational gene therapy SRP-9003 across several measures, as we know the community needs more options, said Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy, Sarepta Therapeutics. The improvements in functional measures at 18- and 6- months in participants from both cohorts who received SRP-9003 are distinctly different from what an age-matched, natural history group would predict with LGMD2E. This sustained durability of the response in functional outcomes reinforces that SRP-9003 is getting to the muscle and suggestive of improvement against disease-mediated muscle damage. When coupled with the strong expression results and encouraging safety profile seen to date, todays results increase our confidence in the construct and provide additional evidence as we advance the higher dose of SRP-9003 into the next stage of clinical testing.

Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed at 90 days. Cohort-specific results as follows:

Cohort 1 (low dose), at 18 months:

Cohort 2 (high dose), at 6 months:

About SRP-9003 and the studySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 5x1013vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 2x1014vg/kg. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days.

Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-Girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding future clinical testing for SRP-9003, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases, the potential benefits of SRP-9003 and potential market opportunities.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

InternetPosting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com.Weencourageinvestorsandpotentialinvestorsto consult our website regularly for important information about us.

Source:Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

Originally posted here:
Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional...

Lysosomal Therapeutics went into JP Morgan 2017 with a sale all but sealed. With $100 million upfront and a $48 million cash infusion to execute on the R&D plan, Allergan lined up an exclusive option to buy the Cambridge, MA-based biotech once the Phase I safety and proof-of-concept data for its lead drug are in.

The team delivered the results by the end of 2018, CEO Kees Been recalled, and after several months of negotiations they had shaken hands over a June buyout.

We had agreed to be acquired by Allergan, he told Endpoints News. And of course just a few days before they were supposed to acquire us, they announced their acquisition by AbbVie which basically terminated the deal.

At the urging of investors, he spent the next year scouting a new buyer for LTIs slate of experimental Parkinsons disease treatment, led by a glucocerebrosidase enzyme activator. The company finally settled on BIAL, a Portuguese pharma company thats looking to set up a new research center in the US.

Been is keeping the upfront under wraps, but disclosed that milestones would add up to $130 million.

All six staffers at LTI, including Been and CSO Peter Lansbury, have been retained to run the newly established BIAL Biotech.

Although the name has changed, the emphasis remains on the programs that LTI already has going, which are all driven by genetic targets.

The lead program, BIA 28-6156 or LTI-291, focuses on activating one enzyme thats lacking in Parkinsons disease patients with certain GBA mutations.

By normalizing enzyme activity you restore the lysosomal function in the cell, he said. With that you restore the glycosphingolipids lipid metabolism in the cells of the brain, which should lead to a disease modifying effect which hopefully translates into reducing the progression of the disease.

The plan for BIAL Biotech is to go straight into Phase II in the second half of 2021 once they nab an IND in the US (the early clinical work was done in the Netherlands), set up registries at clinical sites to identify the right patients and complete long-term tox work.

One of the ways the parent BIAL stood out from other pharma suitors, Been added, was that they agreed with this plan while others insisted on doing an extra study with a brain imaging agent.

While this marks BIALs first official US footprint, the Portuguese drugmaker also boasts of two out-licensed drugs available in the country: Neurocrines Ongentys, an add-on treatment to levodopa/carbidopa in patients with Parkinsons disease experiencing off episodes; and the partial seizures drug Aptiom now marketed by Sunovion.

But its keen to go deeper into Parkinsons by leveraging growing knowledge of genetic mutations to craft a targeted approach, Been said.

They want to become a leader or a major player in the field of Parkinsons disease focused on all these subsegments of Parkinsons disease, he said.

That could also mean partnering on geographies where BIAL, whose neurological portfolio is already available in Europe, isnt quite as active.

Co-founded by Henri Termeer and Bob Carpenter, LTI has accrued a stellar slate of investors over the years, including Hatteras Venture Partners, Atlas Venture, Lilly Ventures, Sanofi-Genzyme BioVentures, Roche Venture Fund and Partners Innovation Fund.

Read this article:
Its sale to Allergan was called off at the last minute. One year later, a biotech co-founded by Henri Termeer finds a new buyer - Endpoints News

This phase 1/1b, multi-centre, observer-blinded, dose-ranging, randomized, placebo-controlled trial will assess the safety, reactogenicity and immunogenicity of different doses of the vaccine given to healthy participants aged 18 to 75 years old in three states (NSW, SA and WA) across Australia.

As a phase 1 trial, the key goal is to examine the safety of two doses of the vaccine given one month apart. If the trial is successful, then a phase 2 trial will be undertaken in a larger number of participants.

Through this network of key experts and partners, this collaborative approach promises to accelerate the development and access to this COVID-19 candidate vaccine in Australia.

The Vax4COVID specialised expertise focuses on rapid planning, recruitment, ethics and genetically modified organism (GMO) approvals, and execution of SARS-CoV-2 vaccine trials in healthy individuals and/or at-risk populations, including healthcare workers and older adults, with the capacity to pre-recruit cohorts of interested individuals.

This project embodies the important role the University of Sydneys health and medical researchers play in rising to the health challenges of our time. In what has been a difficult year, it is the collaborations between our leading scientists and industry, such as this, that will provide the solutions and pave the way forward, said Professor Duncan Ivison, Deputy Vice-Chancellor (Research) at the University of Sydney.

Mr Laurent Dapremont, Chief Executive Officer of Technovalia said: We are very pleased and proud to be able to contribute to the development of an effective solution to the crisis we have been living for many months. This is a unique collaborative effort with outstanding partners and clinicians, and most importantly, plans are being put in place with BioNet and LuinaBio to rapidly develop a vaccine againstCOVID-19 which can be produced in Australia.

Dr Pham Hong Thai, Chief Executive Officer of BioNet-Asia, said: We are glad to continue our collaboration with Technovalia as this is the second vaccine to be evaluated in Australia, first Pertagen and now this vaccine, two recombinant vaccines against highly contagious respiratory diseases, respectively pertussis (whooping cough) and COVID-19. We are also delighted to join A/Prof. Wood and his colleagues from Vax4COVID in the fight against the COVID-19 pandemic in Australia.

Link:
University of Sydney to advance COVID 19 DNA vaccine to human trials - News - The University of Sydney

[co-authors: Dan Meckley, Preston Tran, and Heather Hatcher, Ph.D.]

Photo by National Cancer Institute on Unsplash

Innovative technologies are being deployed to address the Western worlds major killer: cancer. Traditionally, cancer treatment has included surgery, chemotherapy, and radiation, but recently, the development of targeted immunotherapies such as monoclonal antibodies and immune checkpoint inhibitors (e.g., PD-1, PD-L1 and CTLA-4 inhibitors) are showing considerable promise in immunooncology.

The fields of immunology and oncology have been linked since the late 19th century, when studies showed that killed and denatured bacteria injected into sites of sarcoma (a tumor that starts in the bone or muscle) resulted in tumor shrinkage. The intersection between immune surveillance and tumor biology has led to broad therapeutic advances, including the search for a cancer vaccine.

Traditional prophylactic vaccines work to prevent disease by preparing the bodys immune system against a pathogenic infection such as influenza or polio. Over the last decade, the US Food and Drug Administration (FDA) has approved prophylactic vaccines that prevent development of cancer by protecting against cancer-causing pathogens such as human papillomavirus (HPV) (GARDASIL 9; Merck Sharp & Dohme Corp., Whitehouse Station, NJ) and hepatitis B virus (HEPLISAV-B; Dynavax Technologies Corp., Emeryville, CA).

A cancer vaccine is a therapeutic vaccine that targets pre-existing tumors in cancer patients who have a fundamentally different immune response relative to that of healthy individuals. Cancer is characterized by an accumulation of genetic alterations, and every tumor has its own unique composition of mutations and novel surface antigens, or neo-antigens, with only a small fraction shared between patients. Not surprisingly, therapeutic vaccines have been challenging to develop; however, tumor neo-antigens present an antigenic target for pharmaceutical companies to design and develop cancer vaccines.

Within the past several years, there has been an explosion in early-stage clinical activity in gene-modified and cell-based immunooncology, which now encompasses about 58% of Phase I trials. The FDAs Center for Biologics Evaluation and Research (CBER) provided sponsors with guidance on Clinical Considerations for Therapeutic Cancer Vaccines (October 2011) to determine optimal dosing, potential biological and clinical activity, and safety profile during early phase clinical trials, as well as endpoint selection in late phase clinical trials to support a subsequent Biologics License Application (BLA) for marketing approval. Many trials have shown potent therapeutic responses in a proportion of patients with late stage cancer, but it has been rare for trials to obtain more than a 510% partial or complete response. However, this limited success has not lessened the enthusiasm for development of potential cancer vaccines. In 2019, there were nearly 700 oncology clinical trials utilizing specific regenerative medicine and advanced therapy technologies to treat leukemia, lymphoma, and cancers of the brain, breast, bladder, cervix, colon, esophagus, ovaries, pancreas and others (ARM 2019 Annual Report, https://alliancerm.org/sector-report/2019-annual-report/). The 2010 FDA approval of the first cancer vaccine (Provenge (sipuleucel-T); Dendreon Corp., Seattle, WA), was supported by clinical trials showing that the vaccine prolongs survival in patients with metastatic, castration-resistant prostate cancer, though the effect was modest. In 2015, the FDA approved a therapeutic cancer vaccine for the treatment of advanced melanoma (IMLYGIC or T-VEC, talimogene laherparepvec; Amgen, Thousand Oaks, CA).

Despite the challenges, each translation of cancer vaccines to the clinical setting has yielded a deeper understanding of the immunologic response produced by cancer.

Several platforms for cancer vaccination are being tested, including peptides, proteins, antigen presenting cells, tumor cells, and viral vectors. Prior clinical trials have shown that cancer vaccines are well tolerated, target tumor neo-antigens and induce antigen cascade. Current trials seek to improve cancer vaccine efficacy either by targeting novel tumor antigens or employing vaccines in combination with other therapeutic approaches. Additionally, provisions in the 21st Century Cures Act have allowed the FDA to use an accelerated approval pathway for cancer vaccines that have been designated as regenerative medicine advanced therapy (RMAT).

Cancer vaccination comprises an array of approaches that seek to generate, amplify, or skew (or a combination thereof) antitumor immunity. Cancer immunotherapy may ultimately establish its position as the fourth pillar of anti-cancer therapy, complementing surgery, chemotherapy, and radiation.

[View source.]

Read the rest here:
Cancer Vaccines: The Fourth Pillar of Anti-Cancer Therapy? - JD Supra

Two Yale research teams will each receive approximately $9 million in grants from the Aligning Sciences Across Parkinsons (ASAP) initiative to study the underlying biology of Parkinsons disease.

The ASAP grants, to be distributed over three years, are part of a major international, multi-institutional effort to uncover the basic disease mechanisms that drive the progressive neurological disorder, which afflicts 7 to 10 million people worldwide. The initiative builds and leverages a network of leading investigators, which will ultimately serve to promote rapid access to data, enabling breakthroughs across scales that will accelerate benefits for patients.

A Yale team headed byPietro De Camilli, the John Klingenstein Professor of Neuroscience, professor of cell biology, and investigator for the Howard Hughes Medical Institute, will study how gene mutations linked to Parkinsons affect the function of brain cells during the course of the disease. De Camilli will team with scientists from Weill Cornell Medicine to study the impact of Parkinsons disease on the physiology and metabolism of synapses, with the goal of identifying new therapeutic targets.

A second Yale team led byDavid Hafler, the William S. and Lois Stiles Edgerly Professor of Neurology and professor of immunobiology, will investigate whether the progression of Parkinsons disease pathology in the brain is initiated by an autoimmune process triggered by the gut microbiome. The research, part of the Center for Neuroinflammation at Yale, will leverage long-standing collaborations with researchers from Massachusetts General Hospital and the Broad Institute to produce an unprecedented map of the neuro-immune-gut interactions, with the goal of identifying new treatments for the disease.

The awards to two Yale teams illustrate the universitys dedication to collaborative science and the growing role Yale neuroscientists are playing in elucidating fundamental mechanisms of the most intractable conditions afflicting the brain and central nervous system, said Nancy J. Brown, dean of the Yale School of Medicine. Without a more robust understanding of basic mechanisms we cannot make progress in the treatment of Parkinsonism, she added.

Other Yale members of the De Camilli team areKarin Reinisch, the David W. Wallace Professor of Cell Biology and of molecular biophysics and biochemistry;Shawn Ferguson, associate professor of cell biology and neuroscience; andKallol Gupta, assistant professor of cell biology.

Other Yale members of the Hafler team areLe Zhang, assistant professor of neurology;Sreeganga Chandra,associate professor of neurology and neuroscience;Rui Chang,assistant professor of neuroscience;Noah Palm,assistant professor of immunobiology;Brian KooandJesse Cedarbaum, members of the clinical Department of Neurology; andDavid van Dijk, assistant professor in the Department of Medicine and Genetics.

ASAP is a coordinated research initiative dedicated to fostering collaboration and resources to better understand the underlying causes of Parkinsons disease. The Michael J. Fox Foundation is ASAPs implementation partner and issued the grants.

View original post here:
Yale teams get multi-million-dollar awards to study biology of Parkinson's - Yale News

Sept. 23, 2020 12:00 UTC

Company appoints Isaac Veinbergs, Ph.D., an established leader with strong expertise in diseases of the central nervous system, as President and CEO

SAN DIEGO--(BUSINESS WIRE)-- Libra Therapeutics, Inc. launched today to develop novel drug candidates focused on restoring the cellular balance lost in neurodegenerative diseases. These include amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which are both characterized by disequilibrium caused by the production and decreased clearance of neurotoxic proteins. In conjunction with the companys launch, Libra Therapeutics has secured $29 million in Series A financing co-led by Boehringer Ingelheim Venture Fund (BIVF), Epidarex Capital, and Sant. The round was joined by Yonjin Venture, Dolby Family Ventures, and Sixty Degree Capital.

Libra Therapeutics was founded on breakthrough science generated by Axxam S.p.A., a leading discovery research organization, which provides Libra Therapeutics novel and proprietary chemical matter, cutting edge and exclusive assays, and robust drug discovery expertise. The financing proceeds will be used primarily to build the team and advance a pipeline of novel small molecule drug candidates for the treatment of neurodegenerative diseases.

My career has been dedicated to CNS drug discovery. I have seen firsthand the devastation diseases like ALS, Alzheimers, and Parkinson's disease have on patients and their families, said Isaac Veinbergs, Ph.D., President and CEO of Libra Therapeutics. We are delighted to build on Axxams pioneering foundational research to accelerate the development of novel therapies with the potential to slow or prevent disease progression to deliver meaningful improvements in patient outcomes across a wide range of neurodegenerative diseases.

The most frequent genetic cause of ALS and FTD is the expansion of the C9orf72 gene. Multiple studies have proposed that C9orf72 regulates intracellular trafficking and autophagy in neuronal cells, and thus Libra Therapeutics aims to increase autophagy to counter the loss of C9orf72 function. The companys therapeutic platform is uniquely positioned to discover and develop novel small molecule drugs that can both increase autophagy to more rapidly clear toxic proteins and attenuate the production of neurotoxic proteins.

Libra Therapeutics takes a unique approach to tackling neurodegenerative diseases with two distinct but complementary approaches that target key pathways that drive neurodegeneration, said Martn Heidecker, Ph.D., Managing Director, BIVF USA. By modulating scientifically and genetically validated targets to decrease neurotoxic proteins, theres a clearly defined development path with translational and clinical biomarkers.

We are delighted to have played a key role in the creation and funding of Libra Therapeutics as well as co-leading the financing. The science behind the company is outstanding and has enabled the recruitment of a highly experienced executive and an exceptional international investor syndicate, said Henning Steinhagen, Ph.D., Venture Partner, Epidarex Capital. By bringing together these key components, Libra Therapeutics has a highly differentiated platform to generate novel therapies that aim to improve the lives of patients with neurodegenerative diseases.

About BIVF

Created in 2010, the Boehringer Ingelheim Venture Fund GmbH (BIVF) invests in groundbreaking therapeutics-focused biotechnology companies to drive innovation in biomedical research. BIVF is searching for significant enhancements in patient care through pioneering science and its clinical translation by building long-term relationships with scientists and entrepreneurs. BIVFs focus is to target unprecedented therapeutic concepts addressing high medical needs in immuno-oncology, regenerative medicine, infectious diseases and digital health. For more information, visit http://www.boehringer-ingelheim-venture.com.

About Epidarex Capital

Epidarex Capital is a transatlantic venture capital firm with a track record of building exceptional life science companies in emerging hubs in the US and UK. Epidarex experienced team of early stage investors partner with entrepreneurs and leading research institutions to transform world-class science into highly innovative products addressing major unmet needs in the global healthcare market. For more information, visit http://www.epidarex.com.

About Sant

Founded in 2006, Sant is a specialized healthcare and life sciences investment firm with over a half-billion dollars in capital under management. The Firm invests in early-stage companies developing innovative new medical technologies, biotechnologies and digitally-enabled healthcare services. Since inception, the Firm has made nearly 40 portfolio investments, including Claret Medical (Boston Scientific), TVA Medical (Becton Dickinson), Millipede Medical (Boston Scientific), Molecular Templates (MTEM), AbVitro (Celgene) and Explorys (IBM Corp). Sant invests nationally and is headquartered in Austin, Texas, with additional offices in Houston. For more information, please visit http://www.sante.com.

About Libra Therapeutics

Libra Therapeutics is a biotechnology company focused on developing novel disease-modifying therapeutics that can restore the cellular balance disrupted in neurodegenerative diseases. The company is advancing three distinct and novel preclinical small molecule programs designed to increase autophagy and decrease production of neurotoxic protein aggregates. Libra Therapeutics was founded to capitalize on proprietary technologies and therapeutic assets from Axxam, a leading discovery research company. The company is based in San Diego. To connect with the company, visit http://www.libratherapeutics.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200923005096/en/

Visit link:
Libra Therapeutics Launches with $29 Million Series A to Develop Novel Therapeutics for Neurodegenerative Diseases - BioSpace

After 15 years of unrelenting work, a team of scientists from Gladstone Institutes has now discovered a potential drug candidate for heart valve disease that works in both human cells and animals and is ready to move toward a clinical trial. Their findings were just published in the journal Science.

"The disease is often diagnosed at an early stage and calcification of the heart valves worsens over the patient's lifetime as they age," says Gladstone President and Director of the Roddenberry Stem Cell Center Deepak Srivastava, MD,who led the study. "If we could intervene early in life with an effective drug, we could potentially prevent the disease from occurring. By simply slowing the progression and shifting the age of people who require interventions by 5 or 10 years, we could avoid tens of thousands of surgical valve replacements every year."

This also applies to the millions of Americansabout one to two percent of the populationwith a congenital anomaly called bicuspid aortic valve, in which the aortic valve only has two leaflets instead of the normal three. While some people may not even know they have this common heart anomaly, many will be diagnosed as early as their forties.

"We can detect this valve anomaly through an ultrasound," explains Srivastava, who is also a pediatric cardiologist and a professor in the Department of Pediatrics at UC San Francisco (UCSF). "About a third of patients with bicuspid aortic valve, which is a very large number, will develop enough calcification to require an intervention."

Srivastava's research into heart valve disease started in 2005, when he treated a family in Texas who had this type of early-onset calcification. All these years later, thanks to the family's donated cells, his team has finally found a solution to help them and so many others.

A Holistic Approach in the Hunt for a Therapy

Members of the family treated by Srivastava had disease that crossed five generations, enabling the team to identify the causea mutation in one copy of the gene NOTCH1. Mutations in this gene cause calcific aortic valve disease in approximately four percent of patients and can also cause thickening of valves that trigger problems in newborns. In the other 96 percent of cases, the disease occurs sporadically.

"The NOTCH1 mutation provided a foothold for us to figure out what goes wrong in this common disease, but most people won't have that mutation," says Srivastava. "However, we found that the process that leads to the calcification of the valve is mostly the same whether individuals have the mutation or not. The valve cells get confused and start thinking they're bone cells, so they start laying down calcium and that leads to hardening and narrowing of the valves."

In the hunt for a treatment, the group of scientists chose a novel, holistic approach rather than simply focusing on a single target, such as the NOTCH1 gene.

"Our goal was to develop a new framework to discover therapeutics for human disease," says Christina V. Theodoris, MD, PhD, lead author of the study who is now completing her residency in pediatric genetics at Boston Children's Hospital. "We wanted to find promising therapies that could treat the disease at its core, as opposed to just treating some specific symptoms or peripheral aspects of the disease."

When Theodoris first joined Srivastava's lab at Gladstone, she was a graduate student at UCSF. At the time, they knew the NOTCH1 gene mutation caused valve disease, but they didn't have the tools to study the problem further, largely because it was very difficult to obtain valve cells from patients.

"My first project was to convert the cells from the patient families into induced pluripotent stem (iPS) cells, which have the potential of becoming any cell in the body, and turn them into cells that line the valve, allowing us to understand why the disease occurs," says Theodoris. "My second project was to make a mouse model of calcific aortic valve disease. Only then could we start using these models to identify a therapy."

One Drug Candidate Rises to the Top

For this latest study, the scientists searched for drug-like molecules that could correct the overall network that goes awry in heart valve disease and leads to calcification. To do so, they first had to determine the network of genes that are turned on or off in diseased cells.

Then, they used an artificial intelligence method, training a machine learning program to detect whether a cell was healthy or sick based on this network of genes. They subsequently treated diseased human cells with nearly 1,600 molecules to see if any drugs shifted the network in the cells enough that the machine learning program would reclassify them as healthy. The researchers identified a few molecules that could correct diseased cells back to the normal state.

"Our first screen was done with cells that have the NOTCH1 mutation, but we didn't know if the drugs would work on the other 96 percent of patients with the disease," says Srivastava.

Fortunately, Anna Malashicheva, PhD, from the Russian Academy of Sciences, had collected valve cells from over 20 patients at the time of surgical replacement, and Srivastava struck up a fruitful collaboration with her group to do a "clinical trial in a dish."

"We tested the promising molecules on cells from these 20 patients with aortic valve calcification without known genetic causes," Srivastava adds. "Remarkably, the molecule that seemed most effective in the initial study was able to restore the network in these patients' cells as well."

Once they had identified a promising candidate in cells in a dish for both NOTCH1 and sporadic cases of calcific aortic valve disease, Srivastava and his team did a "pre-clinical trial" in a mouse model of the disease. They wanted to determine whether the drug-like molecule would actually work in a whole, living organ.

The scientists confirmed that the therapeutic candidate could successfully prevent and treat aortic valve disease. In young mice who had not yet developed the disease, the therapy prevented the calcification of the valve. And in mice that already had the disease, the therapy actually halted the disease and, in some cases, led to reversal of the disease. This finding is especially important since most patients aren't diagnosed until calcification has already begun.

"Our strategy to identify gene networkcorrecting therapies that treat the core disease mechanism may represent a compelling path for drug discovery in a range of other human diseases," says Theodoris. "Many therapeutics found in the lab don't translate well to humans or focus only on a specific symptom. We hope our approach can offer a new direction that could increase the likelihood of candidate therapies being effective in patients."

The researchers' strategy relied heavily on technological advancements, including human iPS cells, gene editing, targeted RNA sequencing, network analysis, and machine learning.

"Our study is a really good example of how modern technologies are facilitating the kinds of discoveries that are possible today, but weren't not so long ago," says Srivastava. "Using human iPS cells and gene editing allowed us to create a large number of cells that are relevant to the disease process, while powerful machine learning algorithms helped us identify, in a non-biased fashion, the important genes for distinguishing between healthy and diseased cells."

"By using all the knowledge we gathered over a decade and a half, combined with the latest tools, we were able to find a drug candidate that can be taken to clinical trials," he adds. "Our ultimate goal is always to help patients, so the whole team is very pleased that we found a therapy that could truly improve lives."

About the Research Project

The paper, "Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease,"was published online by Science on December 10, 2020.

Other authors include Ping Zhou, Lei Liu, Yu Zhang, Tomohiro Nishino, Yu Huang, Sanjeev S. Ranade, Casey A. Gifford, Sheng Ding from Gladstone; Aleksandra Kostina from the Russian Academy of Sciences; and Vladimir Uspensky from the Almazov Federal Medical Research Centre in Russia.

The work was funded by the California Institute of Regenerative Medicine; the National Heart, Lung, and Blood Institute; and the National Center for Research Resources. Gladstone researchers also received support from the Winslow Family, the L.K. Whittier Foundation, The Roddenberry Foundation, the Younger Family Fund, the American Heart Association, several programs and fellowships at UCSF, residency programs from Boston Children's Hospital and the Harvard Medical School, the Uehara Memorial Foundation, and a Howard Hughes Medical Institute Fellowship of the Damon Runyon Cancer Research Foundation.

About Gladstone Institutes

To ensure our work does the greatest good, Gladstone Institutes focuses on conditions with profound medical, economic, and social impactunsolved diseases. Gladstone is an independent, nonprofit life science research organization that uses visionary science and technology to overcome disease.

Media Contact: Julie Langelier | Assistant Director, Communications | [emailprotected] | 415.734.5000

SOURCE Gladstone Institutes

https://gladstone.org

See more here:
A Potential Therapy for One of the Leading Causes of Heart Disease - PRNewswire

HACKENSACK, N.J., Jan. 19, 2021 /PRNewswire/ --Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced a $1 million programmatic investment in Satellos Bioscience Inc. (Satellos) to support the development of a new regenerative medicine for the therapeutic treatment of Duchenne.

Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the dystrophin gene. In people living with Duchenne, it was discovered by Dr. Michael Rudnicki, the scientific founder of Satellos, that muscle stem cells are severely compromised in their ability to create muscle progenitor cells which repair injured muscle. As a result, people with Duchenne are unable to keep up with the continuous damage to their muscles throughout life.

Satellos's technology is based on this ground-breaking discovery into what controls muscle stem cell differentiation, and the significant impact its dysregulation has on the progressive nature of Duchenne. The company is developing a means to correct this problem through a novel therapeutic approach involving the administration of a small molecule drug, which mobilizes the body's own muscle stem cells, also known as satellite cells, to repair and regenerate muscles.

Such a small molecule drug could be used to treat all people living with Duchenne and furthermore, could be used throughout life to boost the continuous repair and regeneration of skeletal muscle ravaged by this disease, thereby enhancing and extending the lives of people with Duchenne.

"For over 25 years, PPMD has been committed to exploring and supporting every single therapeutic possibility. With this programmatic investment in Satellos, we continue our cutting-edge approach to accelerate finding treatments that have the potential to end Duchenne for every single person impacted by the disease," said Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation. "There is compelling proof of concept data showing that the Satellos technology can improve muscle quality and restore function in the mdxmouse model of Duchenne. This investment from PPMD will enable the Satellos team to build on their proof of concept by amplifying their efforts to invent and refine a drug development candidate suitable for testing in humans."

"We are delighted to be working with PPMD and proud to have the therapeutic potential of our novel science recognized by such a leading entity in the fight against Duchenne", said Frank Gleeson, Satellos founder and CEO. "Our profound discoveries into how muscle stem cells repair and regenerate muscle offers a new avenue for addressing a root cause of the progressive debilitation characteristic of Duchenne. Support from PPMD will enable us to accelerate the development of our new treatment approach which offers the promise of helping Duchenne patients of all ages and stages of disease."

To learn more about PPMD's robust Research Strategy, funding initiatives, and strategies for accelerating drug development,click here.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY:

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength.Parent Project Muscular Dystrophy (PPMD)fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won four FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne atEndDuchenne.org.Follow PPMD onFacebook,Twitter, Instagram, andYouTube.

ABOUT SATELLOS BIOSCIENCE INC.:

Satellos is a regenerative medicine company dedicated to developing novel therapeutics that stimulate or restore muscle regeneration in life threatening disorders. Our founding scientist, Dr. Michael Rudnickidiscovered that the dysregulation of stem-cell polarity a process that balances replenishment of stem cells and production of specialized tissue cells, including muscle can lead to the inability of the body to properly repair and regenerate muscle throughout life.

Satellos is initially applying these discoveries to our lead program to develop a new therapeutic treatment which restores muscle regeneration in Duchenne muscular dystrophy, a lethal degenerative disease. Defects in muscle regeneration are also causative in many chronic conditions which we plan to pursue including sarcopenia (muscle wasting with age), cachexia (muscle wasting from chemotherapy or smoking) and various dystrophies. Founded in 2018, Satellos is headquartered in Canada.For more information about Satellos' discovery platform and development programs please visit Satellos.com.

SOURCE Parent Project Muscular Dystrophy (PPMD)

Join the fight. End Duchenne.

Go here to read the rest:
Parent Project Muscular Dystrophy Invests $1 Million in Satellos Bioscience to Support New Regenerative Medicine Technology - PRNewswire

SALT LAKE CITY It's amazing Cinch Wright is alive.

The little boy got cutting edge gene therapy that replaced disease-causing genes with healthy ones.

It can knock out a gene that's not working right, or add a new gene to the body to help fight disease.

"He loves the dog and the horses and the cows," says Alex Wight about his son.

No doubt about it, Cinch Wight is going to be a cowboy just like his dad.

To date, the FDA has approved four types of gene therapy including one that was given approval just in time for Cinch.

A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy or SMA.

"That was the first time I'd ever even heard the term and what it was," said Cinch's mother, Amber.

SMA is a neuromuscular disorder that can paralyze a baby in the first few weeks of life.

"My first thought was, he's never going to be able to ride broncs or anything like that," Alex remembered.

Just one day after Cinch was born, the FDA approved a new gene therapy.

"We were pretty excited to get a phone call from the Department of Health, you know, and have this baby here who we can use this treatment on after its approval," recalled Dr. Russell Butterfield, pediatric neurologist with the University of Utah Health's Intermountain Primary Children's Hospital.

A critical gene in little Cinch was missing.

Dr. Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinch's nerve cells.

"It's like a delivery truck to deliver genes to where you want them to go," he explained.

"What that does do, is it stops the disease right where it is."

Just a few years ago, most children born with SMA didn't make it to their second birthday.

"The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that," said Dr. Butterfield.

"And understanding that how different this child's life will be with this new medicine."

Alex Wight has written an inspirational book about his son.

Some might say it's a true story about how real cowboys never give up.

"I wanted to let him know that no matter how hard it gets, as long as he keeps going, he'll be all right," said Alex.

Zolgensma is a new, working copy of a human SMN gene, and makes up for the missing or nonworking survival motor neuron 1 (SMN1) gene, which helps motor neurons work properly. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein, which motor neuron cells need to survive and support muscle functions.

Doctors don't know if the one-time infusion will last a lifetime or will have to be repeated and there could be a possible risk of inflammation to the liver that doctors will closely monitor.

The gene replacement therapy costs $2.1 million dollars.

Insurance paid for most of it but Alex hopes sales from his children's book will help pay the rest.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.comor Marjorie Bekaert Thomas at mthomas@ivanhoe.com.

See the rest here:
YOUR HEALTH: New gene therapy saves infant's life - WQAD.com

Then, Girondi claimed, Sloan Kettering mothballed his work to favor Bluebird, whose chief executivehad a prior business relationship with the cancer center's boss.

Girondis rage has been fueled by Bluebirds trajectory since then: its thalassemia treatment was approved last year by the European Union and at $1.8 million per patient will be among the most expensive.

In my neighborhood, theyd have gotten ball batted for similar behavior, said Girondi, a self-described former street tough from the South Side of Chicago whosbeen using such talk to describe his adversaries for years.

Now, Girondi is finally getting his day in court. Having survived years of legal challenges, which have portrayed his case as absurd and Girondi himself as erratic and ill-tempered, his trial began on Thursday.Errant is seeking hundreds of millions of dollars in damages, according to court filings.

The trial promises a rare glimpse into the not uncommonly messy marriage of medical researchers and for-profit companies, and it will showcase a slew of revealingdocuments and emails that have emerged in the court file, including one that Girondis lawyers described in court as the smoking gun.

Written in June 2010 by Nick Leschly, then interim president of Genetix Pharmaceuticals, which was renamed Bluebird Bio a few months later, the emailsaid:Pat Girondineed to shut him down.

The recipient of the email, another Genetix executive, responded by saying they need to be nice, suck up, etc. to Girondi, so they can review valuable data from a Sloan Kettering scientist with whom Errant was collaborating.

Both Sloan Kettering and Bluebird deny Errants allegations.

Sloan Kettering is vigorously defending itself in court, said Jorge Lopez, executive vice president and general counsel for Memorial Sloan Kettering Cancer Center, in a statement. We also disagree with EGTs characterizations of the case and of the Courts rulings. He declined further comment.

In court filings, Sloan Kettering has argued that Girondis company, called EGT for short,wasnt tricked but rather was short of money and repeatedly failed to meet obligations outlined in its deal.

The evidence shows that EGTs case is the fantasy of a defunct company and its founder that refuse to accept responsibility for their own failure, an attorney for Sloan Kettering Institute wrotein a July 8, 2019 filing.

Bluebirds attorney, Jeffrey Eilender, said the court record has disproved Errantsclaim of a conspiracy between Sloan Kettering Institute, referred to as SKI in court filings, and Bluebird, as well as an allegation that Bluebird gleaned secrets from Girondis company.

None of the evidence relied upon by EGT shows a material issue as to the ultimate fact: none suggests in any way that there was an agreement between Bluebird and SKI to defraud EGT, Bluebirds lawyers wrotein a July 8, 2019 court filing. In fact, EGT does not even explain how or why the facts it cites are relevant here (they are not); it just throws everything at the wall to see if it sticks.

As for Leschlys email saying that Girondi needed to be shut down, Eilender said it referred to Girondi,not the company. Why? Because with all due respect to Mr. Girondi, hes a nudnik, Eilender told the court at January 2019 hearing, explaining Girondi had becomea nuisance.

Leschly wasnt available for an interview, but he has previously expressed his opinions about Girondi and his firm.

Errant is toothless and the guy behind it is completely insane, truly, Leschly wrote in a 2012 email to an investment analyst.

Thalassemia is an inherited blood disorder in which the body doesnt produce enough hemoglobin, the substance in red blood cells that carriesoxygen. Moderate and severe, or beta,cases require frequent blood transfusions and can result in early death.

In 1992, Rocco Girondi was diagnosed with a more severe form of the blood disorder. He was two years old. The next year, Girondi retired from what he describes as a lucrative trading career to devote himself to finding a cure.

Girondi, 62, isnt your typical biotech entrepreneur. A high school dropout, he was listed as one of America's most eligible bachelors in Playgirl magazine in 1988 and appeared on the Oprah Winfrey Show in an episode on male chauvinists. (A copy of the show wasnt readily available; Girondi said he had defended both a mans and a woman's right to work, but believed one should stay home if they have children).A 1987 article about him in Chicago Magazine is entitled Fonzie Gets Rich. He left Chicago decades ago for Italy, where he occasionally performs in concert, playing blues and rock andthe occasional Italian ballad. But hestill speaks in the blunt, sometimes salty, manner of the Chicago neighborhood where he grew up.

He had some money at a time when few others showed interest in gene therapy. By 2000, Girondi began providing financial support to researchers at Sloan Kettering, including Dr. Michel Sadelain, who had brought thalassemia under control in mice, according to Errants complaint. Those researchers had developed a method to replace defective genes in thalassemia patients with a healthy copy. The plan used a modified virus known as a vector to deliver the genetic material into the cells.

At that time, gene therapy was relatively new and scarred by missteps, including a patient who had died after undergoing treatment. In 2005, Sloan Kettering granted a license to develop Sadelains potential gene therapy treatment to the only interested party, Errant Gene Therapeutics, according to the complaint.

Progress was slow and hampered by delays, according to Girondi and his lawyers. But by 2010, Errant had manufactured enough of the medicine to start clinical trials, his lawyers say in court papers.

Girondi said the relationship between his company and Sloan Kettering changedsoon after Craig Thompsons hiring as president and chief executive officer of Memorial Sloan Kettering Medical Centerwas announced in August 2010. By that fall, he said it was clear his company was on the outs.

It ended very strangely, Girondi said. I think thats the best way to say it.

Sadelain didnt respond to messages seeking comment.

Thompson, 67, had previously worked at the University of Pennsylvania, where he had been director of the Abramson Cancer Center. He also co-founded a company called Agios Pharmaceuticals in 2007with the goal of unlocking a new field of discovery in cellular metabolism.

Agiosreceived an infusion of $33 million from several venture capital firms in 2008 including Third Rock Ventures, where Leschly the future Bluebird CEO -- was a partner. Leschly also served as Agioss interim chief business officer,according to a 2010 Bluebird press release. A few months later, when Thompson began his job at Sloan Kettering, he was listed as being on Agioss scientific advisory board.

In September, 2010, Sloan Kettering asked Errant for physical possession of the vector to complete a study which it said was necessary to move forward with clinical trials, according to Errants lawyers. Errant delivered the vector and never got it back, the lawyers said.

Sloan Kettering said in court documents that by 2010, Errant had defaulted on its obligations, and that following arbitration and a new deal the following year, all rights granted to Errant in the 2005 deal reverted to Sloan Kettering.

Thompson started his job at Sloan Kettering in November, 2010. That same month,Sloan Ketteringmet with Bluebird and gave them a technical demonstration on Errants vector, sharing confidential information that served as a preludeto a more formal agreement the next year, according to Errants lawyers.

In November 2010, Bluebirds board of directors in 2010 weighedthe pros and cons of collaborating withSloan Kettering, according to Errants court filings. Among the positives? Eliminates the most threatening competitor, according to thepresentation, which is part of the court record.

Andrew Maslow, a former Sloan Kettering executive, said in an interview thathe made the decision to pursue a collaboration with Bluebird, not Thompson. One reason was the improving landscape for gene therapy, and another was Bluebirds capabilities, he said.

These guys are the real thing. They are totally capable, he said. They were just the opposite of Pat.

While Errant squabbled with Sloan Kettering, Bluebird continued to move toward commercialization of its treatment.

Bluebirds treatment for transfusion-dependent thalassemia patients, Zynteglo, was approved by the European Union last year, and the company plans to apply for U.S. approval in 2021. But thats just the beginning. The gene therapy is also intended foruseas a treatment for sickle cell disease(SCD). It could ultimately generate $1 billion a year in annual sales, according to Bloomberg Intelligence.

These therapies have the potential to transform the lives of patients with thalassemia and SCD,said Marc Engelsgjerd, a biotech analyst at Bloomberg Intelligence, who called the treatments groundbeaking.

Leschly, for one, has already benefited. He has pocketed roughly $78 million from stock sales since the company went public in 2013, according to data compiled by Bloomberg. Girondi and Sadelain have been left to contemplate what might have been.

We could have gotten an incredible product and a Nobel Prize, Sadelain said, in a May 2015 phone call that Girondi recorded, also part of the court file. And right now, we have nothing.

View post:
Sloan Kettering Institute, Bluebird Bio in court trial over gene therapy deal - Crain's Chicago Business

Basel, October 1, 2020 Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma (onasemnogene abeparvovec) that demonstrated patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials. SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement.1,2,3 These data as of December 31, 2019, and presented today during a virtual Clinical Trial Poster Session as part of the World Muscle Society (WMS) 2020 Virtual Congress, support the robust clinical evidence that has demonstrated a consistent, transformative benefit across Zolgensma clinical trials for the treatment of patients with SMA.

We are seeing further evidence of the potential of Zolgensma to effectively halt motor neuron loss following a one-time, intravenous infusion. In STR1VE-EU, patients achieved rapid improvements in motor function following treatment with Zolgensma, and most have already achieved motor milestones not observed in the natural history of SMA Type 1, said Professor Eugenio Mercuri, M.D., PhD., Department of Pediatric Neurology, Catholic University, Rome, Italy. These interim results are especially encouraging considering STR1VE-EU includes some patients with a more severe phenotype than in the START and STR1VE-US studies, further supporting the gene therapys positive benefit/risk profile, even in this more fragile population.

"These strong interim results from the STR1VE-EU clinical trial continue to demonstrate consistent and significant therapeutic benefit in patients with SMA Type 1, the most common form of the disease, adding to the robust body of clinical evidence for Zolgensma, said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies. With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients.

Phase 3 STR1VE-EU Data as of December 31, 2019STR1VE-EU is designed to evaluate the efficacy and safety of a single, one-time IV infusion of Zolgensma in patients with SMA Type 1 who are less than six months of age at the time of gene therapy, with one or two copies of theSMN2backup gene and who have bi-allelicSMN1gene deletion or point mutations. The mean age of dosing was 4.1 months and the mean age at the onset of symptoms was 1.6 months. The mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score at baseline was 28. Thirty-one of 33 patients (93.9%) were able to swallow thin liquids, and 10 patients (30.3%) required feeding support at baseline. Nine of thirty patients (27.3%) required ventilatory support at baseline. STR1VE-EU is distinct in its inclusion and exclusion criteria and baseline clinical characteristics of enrolled patients compared with START or STR1VE-US. Specifically, some patients in STR1VE-EU had a more severe disease phenotype at baseline, including lower CHOP-INTEND scores and the need for nutritional and ventilatory support.

At last visit before the data cutoff, patients in STR1VE-EU were between 6.9 and 18.6 months of age, and mean duration in the study was 10.6 (1.815.4) months. Thirty-one out of 32 (97%) patients in the intent-to-treat (ITT) population survived event-free, including 30 (93.8%) who could have reached 10.5 months of age and 18 (56.3%) who could have reached 13.6 months of age. An event is defined as the need for tracheostomy or the requirement of 16 hours of respiratory assistance per day (via non-invasive ventilatory support) for 14 consecutive days in the absence of an acute reversible illness, excluding peri-operative ventilation. Untreated natural history indicates that only 50% and 25% of babies with SMA Type 1 will survive event-free by the time they reach 10.5 months of age and 13.6 months of age, respectively.3

Twenty-one patients (65.6%) achieved motor milestones not observed in the natural history of SMA Type 1. This includes six patients (18.8%) who could sit independently for 10 seconds (the primary efficacy endpoint), 20 patients (66.7%) who gained head control, eight patients (25%) who were able to roll from back to sides and one patient who could stand with assistance, crawl and walk with assistance. The mean increase in CHOP INTEND from baseline was 5.9 points (n=31) which was observed as early as at one month post-dosing, 10.1 points at 3 months (n=29) post-dosing, and 13.3 points at six months (n=27) post-dosing. Twenty-one children (65.6%) enrolled in STR1VE-EU achieved and maintained a CHOP INTEND score of 40 points and 12 children (37.5%) were able to achieve a score of 50. According to natural history, untreated patients with SMA Type 1 almost never achieve a CHOP INTEND score 40.3,4

The majority (91.7%) of patients who were free of ventilatory support at baseline remained either completely free of ventilatory support or received prophylactic BiPAP support during the study for acute reasons. Two-thirds (66.7%) of patients in the ITT population were able to feed orally without the need for feeding support, an important indicator of stabilization/halting of disease progression.

As previously reported, one patient discontinued the study because of a serious adverse event of hypoxic-ischemic brain damage and respiratory distress that resulted in death. Novartis and the investigator considered the events and death to be unrelated to treatment with Zolgensma based on autopsy findings. Thirty-two of 33 patients were reported to have at least one adverse event (AE), of which six patients experienced serious adverse events that were considered by the investigator to be related to Zolgensma. Liver transaminase elevations, some of which were reported as adverse events, were experienced by 29 of 33 patients (87.9%), but all resolved with the use of prednisolone. Four patients had reported decreases in platelet counts <75,000, three of which were isolated laboratory abnormalities without adverse events reported. Overall, no new safety signals have been identified and the reported adverse events are consistent with the cumulative safety profile with Zolgensma.

Novartis Gene Therapies is grateful to the courageous patients and families who participate in clinical trials, enabling the company to further its efforts to make a meaningful difference in the lives of patients with rare genetic diseases.

About Zolgensma (onasemnogene abeparvovec)Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma was approved by the U.S. Food and Drug Administration in May 2019 and represents the first approved therapeutic in Novartis Gene Therapies proprietary platform to treat rare, monogenic diseases using gene therapy.5 In addition to the United States, Zolgensma is approved in Japan, Europe and Brazil. More than 600 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program. Novartis Gene Therapies is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, and South Korea in late-2020 or early 2021.5

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

About Spinal Muscular AtrophySMA is the leading genetic cause of infant death.1,2 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.3,4SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.1 It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.5 This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.6 More than 30% of patients with SMA Type 2 will die by age 25.7

About Novartis Gene TherapiesNovartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are turning promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). This therapy is now approved in the U.S., Japan, Europe and Brazil, and additional registrations are being pursued in close to three dozen countries, with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, Argentina and South Korea in late 2020 or early 2021. Our robust AAV-based pipeline is advancing treatments for Rett syndrome; a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene; and Friedreichs ataxia. We are powered by the worlds largest gene therapy manufacturing footprint of more than one million square feet, enabling us to bring these therapies to patients around the world at quality and scale.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, can, will, plan, may, could, would, expect, anticipate, seek, look forward, believe, committed, investigational, pipeline, launch, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact media.relations@novartis.com

References1. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15:895908.2. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. Available at: http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed October 29, 2019.3. Finkel RS, McDermott MP, Kaufmann P. et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83:8107.4. Kolb SJ, et al. Ann Neurol. 2017;82:88391.5. SolerBotija C, et al. Brain. 2002;125:162434.6. Wang CH, et al. J Child Neurol. 2007;22:102749.7. Darras BT, Finkel RS. Natural history of spinal muscular atrophy. In: Sumner CJ, Paushkin S, Ko CP, eds. Spinal Muscular Atrophy: Disease Mechanisms and Therapy, 2nd ed. London, UK: Academic Press/Elsevier;2017:399421.

# # #

Novartis Media RelationsE-mail: media.relations@novartis.com

Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

Excerpt from:
Zolgensma data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival,...

DUBLIN, Nov. 5, 2020 /PRNewswire/ -- The "Curative Oncology and Rare Disease Therapies Transforming the Global Biologics Market, 2020-2024" report has been added to ResearchAndMarkets.com's offering.

The forecast for the global biologics market until 2024 reveals an interesting trend and paradigm shifts that are set to take place in the industry. Importantly, the study also identifies actionable growth opportunities for industry participants to profit upon.

With this research service, the publisher's Transformational Health team provides critical insights into the global biologics industry highlighting the growth opportunities, market revenue segmentation, and technology trends influencing its growth. The global biologics market covered in this study includes 4 major segments - antibody therapies, recombinant proteins, vaccines, and regenerative medicine.

This research service identifies the largest revenue-generating segments and key therapeutic areas for the global biologics industry. Accelerated regulatory approvals for biologic products and the emergence of next-generation technologies are highlighted, in addition to their influence on market trends during the forecast period.

The study highlights the dynamics of the biologics industry and the sub-segments of the biologics market. Growth opportunities in antibody-drug conjugate (ADC), RNA therapy, and gene therapy platforms are also explored. Further, the study discusses the impact (such as the accelerated development of vaccines) of the COVID-19 pandemic on the biologics industry and the market. Current challenges facing the biologics industry such as the sluggish growth rate of vaccines, and recombinant proteins are discussed.

Most importantly, this research service discusses possible future market trends such as the accelerated development of mRNA-based vaccines and the increasing importance of speciality bio-CDMOs for the development and commercialization of regenerative medicine. Based on the market trends and revenue forecasts, the group of analysts highlight key growth opportunities in ADC, RNA, and gene therapy platforms through successful drug launches such as Belantamab Malfodotin, Inclisiran, and Zolgensma.

Research Highlights

Highlights of the report include:

Key Issues Addressed

Key Topics Covered:

1. Executive Dashboard

2. Market Overview - Biologics

3. Market Dynamics

4. COVID-19 Impact Analysis of the Biologics Market

5. Revenue Forecast - Total Biologics Market

6. Antibody Therapies Segment Analysis

7. Recombinant Proteins Segment Analysis

8. Vaccines Segment Analysis

9. Cell & Gene Therapy Segment Analysis

10. Companies to Action - Pipeline Analysis

11. Growth Opportunities

12. The Last Word - Strategic Considerations for Staying on the Growth Trajectory

13. Appendix

For more information about this report visit https://www.researchandmarkets.com/r/mwd43p

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

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

SOURCE Research and Markets

http://www.researchandmarkets.com

View original post here:
Global Biologics Market Report 2020-2024: Strategic Collaborations with Specialty Bio-CDMOs will Continue to Drive Growth Opportunities in...

NEW YORK, Sept. 23, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the awarding of 15 new MDA grants totaling more than$4 million toward research focused on a variety of neuromuscular diseases (NMDs), including Duchenne muscular dystrophy (DMD), Charcot-Marie-Tooth disease (CMT), Becker's muscular dystrophy (BMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). This round of grant funding reinforces MDA's unwavering commitmentin the face of declining income due to the COVID-19 pandemicto the progress of neuromuscular disease research and builds on the more than$1 billionMDA has already invested in research to uncover new treatments and cures for NMDs since its inception. Some grants will go into effect this year, while others will be awarded in 2021.

"We continue to fund the most innovative research that will lead us to cures for a range of neuromuscular diseases," saysSharon Hesterlee, PhD, executive vice president and chief research officer for Muscular Dystrophy Association. "We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable."

Dr. Hesterlee added, "Although COVID led the cancellation of MDA's spring review session, we are pleased to announce the funding of these projects, which were reviewed in 2019."

The newly funded projects will aim to advance research discoveries and new therapy development in multiple areas. The awarded grants will fund studies to further advance our understanding of genetic causes of and risk factors for NMDs, investigate new approaches to developing gene therapies and other innovative potential treatments, including stopping disease progression and improving genetic testing technologies.

For a complete list of individual awards for this grant cycle, visit MDA's website and explore theGrants at a Glancesection. Highlights from thegrant awards for this grant cycleinclude:

ALS grants will be announced separately later this month, as will grants being given jointly by MDA and other organizations.

About the Muscular Dystrophy AssociationFor 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this throughinnovations in scienceandinnovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures.Research we have supportedis directly linked to life-changing therapies across multiple neuromuscular diseases.MDA's MOVRis the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports thelargest network of multidisciplinary clinicsproviding best in class care at more than 150 of the nation's top medical institutions. OurResource Centerserves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence atsummer campand through recreational programs, at no cost to families.During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted atmda.org/COVID19. For more information, visitmda.org.

SOURCE Muscular Dystrophy Association

https://www.mda.org

Follow this link:
Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research - PRNewswire