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 non-human primates and AstraZeneca steps up R&D in China with new drug, vaccine, and healthcare partnerships in the billions of dollars.
Listed below are links to the GEN stories reference 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
