FDA panel suggests longer monkey trials of AAV gene therapies

2021-09-08

An FDA advisory committee said adeno-associated virus vector gene therapies should undergo long-term testing in animals to uncover any liver toxicity risk. AAV gene therapies for spinal muscular atrophy and X-linked myotubular myopath have been associated with acute liver failure in clinical trials, and following macaques for longer could reveal more about the mechanisms of acute and long-term effects, says Caroline Zeiss, a researcher at Yale University's Department of Comparative Medicine.

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