Sarepta & what’s next for gene therapy. Plus: Leading FDA’s CDER

Third death puts Elevidys back in spotlight. George Tidmarsh named head of key FDA center. Trends in biotech venture money — a BioCentury podcast

Last week’s public disclosure that a gene therapy from  Sarepta Therapeutics Inc. (NASDAQ:SRPT) had caused a third death led FDA to ask the company to stop distributing its DMD gene therapy Elevidys delandistrogene moxeparvovec, a move the biotech has resisted. The deaths, and disputes between FDA and Sarepta, raise questions about the future of AAV gene therapies, as well as the future of FDA’s platform technology designation. On the latest BioCentury This Week podcast, BioCentury’s analysts unpack the events surrounding Sarepta’s gene therapies and discuss how FDA, industry and patient groups should come together to learn the lessons from the tragic, avoidable deaths. 

BioCentury’s analysts also assess Monday’s appointment of Stanford professor and biotech executive George Tidmarsh to lead FDA’s Center for Drug Evaluation and Research, and check in on the latest trends in venture financings.

This episode of BioCentury This Week is sponsored by IQVIA Biotech.