Dravet Gene Therapy Tested in Mice, Sarepta’s Setback, and AstraZeneca Updates

Gene therapies take center stage in this week’s episode. We discuss a potential gene replacement therapy for people with Dravet Syndrome that scientists have tested in mice with good results and highlight some recent progress from two editing companies. We also break down the recent news from Sarepta about the death of a patient after treatment with its gene therapy for Duchenne muscular dystrophy. In other stories, new data reveals a nonviral method for introducing transgenes into human primates and AstraZeneca steps up R&D in China with new drug, vaccine, and healthcare partnerships in the billions of dollars.

Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Julianna LeMieux, 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

Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome 

By Uduak Thomas, GEN, March 19, 2025 

DMD Patient Dies After Treatment with Sarepta Gene Therapy

By Alex Philippidis, GEN Edge, March 18, 2025 

PiggyBac Transposon System Creates Transgenic Cynomolgus Monkeys

By Julianna LeMieux, PhD, GEN, March 26, 2025 

From Tools to Trials: Editing Therapy Companies Pivot to Development

By Alex Philippidis, PhD, GEN Edge, March 20, 2025 

AstraZeneca Commits up to $11B+ to Chinese Collaborations, Beijing R&D Hub 

By Alex Philippidis, PhD, GEN Edge, March 21, 2025 

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