The introduction of Jude Samulski’s PhD thesis contained a bold goal: develop a gene therapy vector that could be used to treat the world’s genetic diseases. By 1982, he had
first cloned the adeno-associated virus (AAV), a key step on this journey. Some 42 years later, the FDA has approved 11 gene replacement therapies — including 6 that rely on AAV vectors to smuggle new genes into cells to treat monogenic diseases. “It feels kind of astonishing to have had those early conversations over pizza, and then see drugs that are now being approved,” says Samulski, who has had a front row seat to the maturation of the field, from his longtime role as CSO and co-founder of gene therapy company AskBio. “It just took too long.”
