Sarepta Under Scrutiny, AstraZeneca’s Big Bet, and AI vs. IDRs

Sarepta has temporarily paused shipments of Elevidys, its FDA-approved treatment for Duchenne muscular dystrophy. In this episode of GEN's Touching Base, we discuss the company’s response to the tragedies associated with its DMD therapy as well as with a new therapy for limb-girdle muscular dystrophy. Also in this episode, big updates from AstraZeneca including a $50 billion investment in U.S. manufacturing and R&D, a heartwarming story about preventing mitochondrial disease involving eight babies from the U.K., and from the lab of Nobel Prize winner David Baker, PhD, AI that designs drugs for previously “undruggable” proteins.

Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news.

Listed below are links to the GEN stories referenced in this episode of Touching Base:

About Face: Sarepta to Pause Elevidys Shipments Temporarily

By Alex Philippidis, GEN Edge, July 21, 2025

StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient Dies

By Alex Philippidis, GEN Edge, July 20, 2025

Sarepta Axes 500, 36% of Workforce, in Restructuring after DMD Patient Deaths

By Alex Philippidis, GEN Edge, July 16, 2025

AstraZeneca Commits $50B More to U.S. Manufacturing, R&D Projects

By Alex Philippidis, GEN Edge, July 22, 2025

AstraZeneca’s New $300M Plant Provides Control of Cell Therapy Production

By Gareth John Macdonald, GEN, May 14, 2025

Beyond Baby KJ: Next Steps in Manufacturing Genome Editing Cures

GEN Live, July 30, 2025

Mitochondrial Disease Milestone: Eight Babies Born Free of Disease via Pronuclear Transfer

By Julianna LeMieux, PhD GEN, July 16, 2025

Undruggable No More: AI Hits Disordered Proteins, Unlocks Therapy Targets

By Fay Lin, PhD GEN, July 18, 2025

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