

FDA Regulation On Gene Therapy | The Brainstorm EP 96
15 snips Aug 6, 2025
Shea Wihlborg, a Multiomics Research Analyst at ARK Invest with a PhD in biochemistry, discusses the FDA's controversial halt of a Duchenne muscular dystrophy treatment. The conversation dives into how AI is revolutionizing drug discovery and clinical trials, paving the way for innovative gene therapies. Shea shares insights on potential regulatory changes and their impact on the biotech industry, alongside a look at how technologies like CRISPR could transform treatments for both rare and common diseases.
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Gene Therapy Safety Concerns
- A gene therapy for Duchenne muscular dystrophy caused two teenage patient deaths due to acute liver failure.
- The FDA halted treatment shipments for older patients but allowed it for younger, ambulatory patients initially.
FDA's Innovation-Friendly Vision
- The FDA aims to recalibrate standards to approve drugs faster and reduce costs using AI and big data.
- The agency wants to be an ally to industry innovation and patients’ needs.
AI Enhances Clinical Trials
- AI can optimize clinical trials by better patient targeting and trial design.
- Both industry and regulators are exploring AI to streamline drug development.