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