Jude Samulski receives American Society of Gene + Cell Therapy award
R. Jude Samulski, Ph.D., was honored by the American Society of Gene + Cell Therapy (ASGCT) with its Founders Award at the organization’s annual meeting earlier this week in Boston.
Samulski received the award for his instrumental role in the original cloning of adeno-associated virus (AAV) and for his involvement in groundbreaking work as the field advanced, including the development of chimeric AAV capsids, the first use of AAV for gene delivery to the central nervous system, and the first AAV-mediated long-term gene transduction in muscle.
The ASGCT’s Founders Award recognizes a pioneering contributor to the field of gene and cell therapy.
Samulski is a co-founder of Durham-based Asklepios BioPharmaceutical (AskBio). Since the company was established in 2001, he was also the company's chief scientific officer until 2024, when the company announced that Samulski was leaving the CSO job but remaining on the company board. Bayer AG acquired AskBio for $4 billion in late 2020.
Samulski has been honored around the world for his work in engineered recombinant AAV vectors, which can transport gene therapies into cells to treat various diseases. He began his research in AAV some 45 years ago as a graduate student at the University of Florida. There, he cloned and rescued AAV from a plasmid in human cells, which laid the groundwork for various innovations in gene therapy.
Today, he holds some 500 AAV-related technology patents and has helped advance therapies for numerous genetic disorders, including hemophilia, Duchenne muscular dystrophy, and congestive heart failure.
Early work by Samulski at AskBio and related entities was supported by grants and loans from the North Carolina Biotechnology Center totaling about $1 million. The support included a $250,000 grant to the University of North Carolina at Chapel Hill that helped recruit Samulski there from the University of Pittsburgh in 1993. He served as the director of UNC’s Gene Therapy Center for 25 years.
Earlier this week, AskBio announced that the first participant had been dosed with investigational gene therapy AB-1009 as part of PROGRESS-GT LOPD, a Phase 1/Phase 2 clinical trial in participants with late-onset Pompe disease (LOPD).
LOPD is a rare, progressive, debilitating genetic disorder that is estimated to affect at least 5,000 to 10,000 people worldwide. While there are multiple approved enzyme replacement therapies (ERTs) with recombinant human acid alpha-glucosidase (rhGAA), there remains a strong level of unmet medical need, as some individuals receiving ERT may experience a decrease in clinical response over time, which can contribute to overall increased treatment demands, the company said.