Gene Therapy Pioneer Jude Samulski Awarded for Breakthrough Innovations

The European Society of Gene and Cell Therapy (ESGCT), a nonprofit organization supporting scientists and clinicians, recognized gene therapy pioneer and entrepreneur Jude Samulski, Ph.D., with its inaugural Founders Award.

Samulski is a professor of pharmacology at the University of North Carolina’s School of Medicine, as well as the co-founder in 2001 and chief scientific officer of Asklepios BioPharmaceutical (AskBio), a gene therapy company based in North Carolina’s Research Triangle Park. He was also the director of UNC’s Gene Therapy Center for 25 years.

Early work by Samulski at AskBio and related entities was supported by grants and loans from the 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 whopping $4 billion buyout deal with German pharma giant Bayer but continues to operate as an independent scientific entity approaching 1,000 employees in the U.S. and abroad.

The ESGCT honored Samulski for his work in engineered recombinant adeno-associated virus (rAAV) vectors, which can transport gene therapies into cells to treat various diseases. He began his research in AAV 40 years ago as a graduate student at the University of Florida. There, he cloned and rescued AAV from plasmid in human cells, which laid the groundwork for various innovations in the gene therapy space. Today, he holds nearly 500 AAV-related technology patents and has helped advance therapies for a number of ailments, including hemophelia, Duchenne muscular dystrophy, and congestive heart failure. He was previously recognized by the American Society of Gene and Cell Therapy and Pope Francis.

The European organization created the Founders Award to commemorate its 30th anniversary. “The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982,” said Hildegard Büning, ESGCT’s board president. “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.”

Samulski responded by saying, "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." 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."