Recombinant adeno-associated virus (rAAV) technology pioneered by AskBio's Dr. Jude Samulski is key component of all FDA approved AAV gene therapeutics

BayerAsklepios BioPharmaceutical, Inc. (AskBio), a wholly-owned and independently-operated subsidiary of Bayer AG, today announced that the FDA's approval of a new adeno-associated virus (AAV) gene therapy to treat adults with hemophilia B marks another milestone for the advancement of AAV therapeutics and highlights the important research contributions made by AskBio Co-Founder, President and Chief Scientific Officer, R. Jude Samulski, PhD. The significance of Dr. Samulski’s groundbreaking AAV work is validated by the role his pioneering research and discoveries have played in the development of all currently approved AAV gene therapies and most that are in the clinic. Dr. Samulski’s early discovery of how to clone AAV led to its widespread use as a mechanism to deliver healthy genes to cells, and we believe this is the foundation for AAV genetic medicine as it stands today.

"The progress made in gene therapy to date is remarkable," said Jude Samulski, AskBio Co-Founder, President and Chief Scientific Officer. "It is humbling to see the impact of the research I completed over 40 years ago and how this is accelerating AAV therapeutic development. My ultimate vision, shared by all of us at AskBio, is to make gene therapy more accessible for everyone in need around the world. I believe we will be there soon."

Dr. Samulski led the UNC Gene Therapy Center from 1993 to 2016 and, in 2001, co-founded AskBio, where many advances in recombinant AAV (rAAV) and underlying AAV vector technology were, and are being, made. Since 2017, three AAV gene therapies have been approved for use by the FDA. Research performed at the UNC Gene Therapy Center, and advanced at AskBio, helped pave the way for the development of these therapeutics. Hemgenix®(1) (etranacogene dezaparvovec-drlb), for hemophilia B, uses the AAV vector with Padua variant R338L; Zolgensma®(2) (onasemnogene abeparvovec-xioi), for spinal muscular atrophy, uses self-complementary AAV vectors; and Luxturna®(3) (voretigene neparvovec-rzyl), for inherited retinal disease, uses the original AAV2 capsid. All are based on Dr. Samulski’s pioneering research or on technology that he developed in collaboration with others in the field, as are Duchenne muscular dystrophy AAV therapeutics in clinical development.

"We are incredibly fortunate for Jude’s scientific leadership at AskBio and for his continued contributions to genetic medicine, added AskBio’s CEO, Sheila Mikhail. "Every day, we see the faces of the people who are running out of time and can’t wait for answers. It is our responsibility to find solutions. I believe that Jude, our AskBio colleagues and others throughout the industry are providing hope for people with devastating diseases."

AskBio, co-founded by Dr. Jude Samulski and Sheila Mikhail, is a global leader in molecular medicine and serves as a key driver of Bayer’s gene therapy research, manufacturing and clinical advancements.

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease, and congestive heart failure. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.askbio.com or follow us on LinkedIn.

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses, before special items, amounted to 4.9 billion euros.

1. Hemgenix® is a registered trademark of CSL Behring LLC
2. Zolgensma® is a registered trademark of Novartis AG.
3. Luxturna® is a registered trademark of Spark Therapeutics, Inc.

Most Popular Now

Engineered bacteria find tumors, then alert the au…

Combining discoveries in cancer immunology with sophisticated genetic engineering, Columbia University researchers have created a sort of "bacterial suicide squad" that ...

Scientists reveal a potential new approach to trea…

Scientists at the National Institutes of Health and Massachusetts General Hospital in Boston have uncovered a potential new approach against liver cancer that could lead ...

Pfizer invests $43 billion to battle cancer

Pfizer Inc. (NYSE: PFE) and Seagen Inc. (Nasdaq: SGEN) today announced that they have entered into a definitive merger agreement under which Pfizer will acquire Seagen, a...

First nasal monoclonal antibody treatment for COVI…

A pilot trial by investigators from Brigham and Women's Hospital, a founding member of the Mass General Brigham healthcare system, tested the nasal administration of the ...

AstraZeneca launches call for entries to the 2023 …

AstraZeneca has announced the launch of the 2023 R&D Postdoctoral Challenge, an initiative designed to accelerate ideas to transform the treatment of some of the world’s ...

US FDA Advisory Committee votes to support effecti…

GSK plc (LSE/NYSE: GSK) announced that the US Food and Drug Administration (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted that the avail...

Tumour cells' response to chemotherapy is driven b…

Cancer cells have an innate randomness in their ability to respond to chemotherapy, which is another tool in their arsenal of resisting treatment, new research led by the...

Pfizer's ZAVZPRET™ (zavegepant) migraine nasal spr…

Pfizer Inc. (NYSE: PFE) today announced the U.S. Food and Drug Administration (FDA) has approved ZAVZPRET™ (zavegepant), the first and only calcitonin gene-related peptid...

Normalizing tumor blood vessels may improve immuno…

A type of immune therapy called chimeric antigen receptor (CAR)-T cell therapy has revolutionized the treatment of multiple types of blood cancers but has shown limited e...

Gene and cell therapies to combat pancreatic cance…

Pancreatic cancer is an incurable form of cancer, and gene therapies are currently in clinical testing to treat this deadly disease. A comprehensive review of the gene an...

DNA treatment could delay paralysis that strikes n…

In virtually all persons with amyotrophic lateral sclerosis (ALS) and in up to half of all cases of Alzheimer's disease (AD) and frontotemporal dementia, a protein called...

Novartis Tafinlar + Mekinist approved by FDA for p…

Novartis today announced the U.S. Food and Drug Administration (FDA) granted approval for Tafinlar® (dabrafenib) + Mekinist® (trametinib) for the treatment of pediatric p...