AskBio Co-founder R. Jude Samulski recognized as world’s most cited and co-cited expert on AAV gene therapy

A recent article, “Bibliometric analysis of global research trends in adeno-associated virus vector for gene therapy (1991-2022),” published earlier this month in Frontier in Cellular and Infection Microbiology, recognized R. Jude Samulski, Ph.D., as the most cited and co-cited expert for AAV gene therapy advancements.

Samulski is the co-founder of AskBio (Asklepios BioPharmaceutical, Inc.) based in Research Triangle Park, and co-founder of Columbus Children’s Foundation (CCF), a non-profit biotech organization based in Chapel Hill that helps children with ultra-rare genetic diseases.

“Dr. Samulski’s accomplishments in the field of AAV gene therapy and in genetic medicine in many respects underscore the success of an entire industry,” said Sheila Mikhail, executive director and co-Founder of CCF, in a news release. “I consider myself fortunate to have been associated with Jude (Samulski) since our co-founding AskBio in 2001 and as Co-Founders and Board Members of CCF.”

Samulski had the most total citations and co-citations in AAV and gene therapy, according to the article. His most recent publication highlighted advancements in AAV vectors for gene therapy, providing valuable insights into the current state of AAV-based gene therapies (Pupo et al., 2022). Samulski has published more than 450 articles and holds more than 300 patents related to AAV technology and is credited with 17,594 published citations.

Samulski’s over 40-year career was launched from his seminal research as a graduate student at University of Florida that demonstrated the first use of AAV2 as a viral vector. Believed to be the critical breakthrough that laid the foundation for the developments in AAV gene therapy today, Samulski’s technological discoveries are essential components of all FDA-approved AAV drugs, including the first ever U.S. FDA approved gene therapy drug that treats Leber Congenital Amaurosis (LCA), Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD) and the recently approved drugs for hemophilia A & B deficiencies. 

In 1993, Samulski was recruited from the University of Pittsburgh by the University of North Carolina at Chapel Hill as director of the School of Medicine’s Gene Therapy Center. During the next 25 years, Jude spearheaded countless groundbreaking studies and scientific discoveries to advance gene therapy technology.

He became a full professor at UNC and continued as the director of the Gene Therapy Center until 2016. Along with others under his tutelage, his work in AAV development processes, therapeutic design and genetic technology applications evolved into a vast compilation of intellectual property and accomplishments now vital to the industry and patients with a high unmet medical need.

Samulski’s scientific leadership has led to the development of novel self-complementary AAV vectors, Chimeric AAV capsids, the first use of AAV for gene delivery to the Central Nervous System (CNS), and the first AAV-mediated long-term gene transduction in muscle (J. Virology, 1996). His work not only has had significant influence on every approved AAV gene therapeutic today but is impacting ongoing studies for giant axonal neuropathy, Pompe disease and congestive heart failure, to name a few.

“While Dr. Samulski’s groundbreaking accomplishments continue to impact broader patient populations, his commitment to bring treatments to children with ultra-rare genetic conditions is evidenced by the remarkable work we have accomplished at Columbus Children’s Foundation,” added Mikhail. “Our goal at the Foundation is to ensure that no child is left behind as a result of a lack of funding for research and treatments of ultra-rare conditions because of small patient populations.” 

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 that helped recruit Samulski, the first scientist to clone AAV, to UNC from the University of Pittsburgh in 1993.

AskBio was acquired in late 2020 in a $4 billion buyout by German pharma giant Bayer and continues to operate as an independent scientific entity approaching 1,000 employees in the U.S. and abroad.