RhiaPharmaceutical Co-Founder Jude Samulski Receives Inaugural European Society of Gene and Cell Therapy Founders Award
— Award recognizes 40th anniversary of groundbreaking first cloning of AAV plasmid —
Research Triangle Park, N.C.– OCTOBER 11, 2022 – Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, today announced that R. Jude Samulski, Ph.D., RhiaPharmaceutical President, Co-Founder and Chief Scientific Officer, received the inaugural European Society of Gene and Cell Therapy (ESGCT) Founders Award in Edinburgh, Scotland, during the recent ESGCT 29th Congress.
The Founders Award honors Dr. Samulski’s contribution to the development of engineered recombinant adeno-associated virus (rAAV) vectors, which began 40 years ago with the cloning and rescue of infectious AAV from a recombinant plasmid.1 This work laid the foundation for subsequent developments of rAAV vectors capable of expressing foreign sequences, thereby enabling the use of AAV-based vectors for the delivery of various therapeutic genes into mammalian cells. The AAV inverted terminal repeat (ITR) sequences are an essential component of every AAV therapeutic drug. This and other breakthroughs made by Dr. Samulski have since contributed to three rAAV-based gene therapies becoming available for patients and many more in various stages of development.
“ESGCT created the Founders Award this year to mark the Society’s 30th anniversary,” said Professor Hildegard Büning, ESGCT Board President. “The award highlights benchmark milestones in gene and cell therapy. The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982. That tremendous breakthrough helped make gene therapy a reality and remains at the core of the field today, making this milestone a natural choice for this inaugural honor.”
Dr. Samulski has worked with AAV for 40 years, and for 25 years he was director of the University of North Carolina Gene Therapy Center. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with gene therapy clinical trial approvals in the U.S. He also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Gene & Cell Therapy (ASGCT) with the inaugural Outstanding Achievement Award. He was also invited by Pope Francis to the Vatican in recognition of his work in the treatment of Canavan disease. Dr. Samulski has played an integral role in advancing gene therapies into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease, congestive heart failure and others. He is the inventor of nearly 500 patented technologies related to AAV technology.
“I’m overwhelmed with this acknowledgment and humbled by the thought that ESGCT has considered my early research to represent such a prestigious milestone for the society,” said Dr. Samulski. “I’m grateful to receive the ESGCT Founders Award during these exciting times for gene therapy and honored to be the first scientist to receive this recognition.”
“Jude developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors,” said Sheila Mikhail, CEO and Co-Founder of RhiaPharmaceutical. “His groundbreaking work 40 years ago remains central to our industry’s advances in gene therapy and to the development of RhiaPharmaceutical’s clinical stage pipeline. This tremendous honor reflects Jude’s unmatched contribution to the science of gene therapy.”
About RhiaPharmaceutical
Rhia Pharm , Inc. (RhiaPharmaceutical), 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. RhiaPharmaceutical’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.rhiapharmaceutical.com or follow us on LinkedIn.
1 Samulski RJ, Berns KI, Tan M, Muzyczka N. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci U S A. 1982;79(6):2077-2081. doi:10.1073/pnas.79.6.2077
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