358: FDA's gene therapy turmoil, and an alternative model for funding research episode artwork

EPISODE · Jun 19, 2025 · 30 MIN

358: FDA's gene therapy turmoil, and an alternative model for funding research

from The Readout Loud · host STAT

We discuss the tragic news that a second teenage boy with Duchenne muscular dystrophy died after taking Sarepta Therapeutics' gene therapy, raising renewed questions about the FDA's standards for approving drugs.We look at the continued turmoil within the FDA, including the forced dismissal of top gene therapy regulator Nicole Verdun, as well as the announcement of a controversial drug-review voucher program that seems to be steering the agency into politics.We also talk about a private equity firm's decision to fund a research lab at Harvard University, filling a void created by government spending cuts. And we look at the approval of a groundbreaking HIV prevention medicine from Gilead Sciences, as well as a recap of the big BIO convention this week and a preview of the upcoming American Diabetes Association meeting this weekend.

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358: FDA's gene therapy turmoil, and an alternative model for funding research

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