The Readout Loud 358: FDA's gene therapy turmoil, and an alternative model for funding research
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Jun 19, 2025 A tragic turn of events unfolds as two teenage boys die after gene therapy, stirring controversy over the FDA's approval standards. Leadership turmoil at the FDA raises questions about safety and political influence in drug approvals. Meanwhile, a private equity firm steps in to fund Harvard research, highlighting concerns over corporate impact on academia. In brighter news, a groundbreaking HIV prevention drug gains approval, and insights from a massive biotech convention provide a glimpse into the future of healthcare.
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Duchenne Gene Therapy Deaths
- Two teenage boys with Duchenne muscular dystrophy died after receiving Sarepta's gene therapy, Alevitis, due to liver failure complications.
- Both were older, non-ambulatory patients, a group with limited safety data supporting the therapy's use.
FDA Gene Therapy Division Turmoil
- FDA's gene therapy division CBER faces turmoil with the forced dismissal of top regulator Nicole Verdun, who opposed Sarepta's accelerated approval.
- Her exit creates uncertainty for the future regulatory direction of gene therapies at FDA.
New CBER Chief's Balancing Act
- Vinay Prasad, a previously critical FDA critic, now heads CBER and balances accelerating rare disease treatments with scientific rigor.
- His conciliatory tone contrasts his prior stringent views, mixing uncertainty about future FDA gene therapy policies.