Uncut GEMS: Epigenetic Editing Aims to Refine Personalized Genetic Medicine

There was tragic biopharma news in mid-June 2025, when this episode was recorded. A second person treated with Elevidys, Sarepta Therapeutics' gene therapy for Duchenne Muscular Dystrophy (DMD), died from the drug. Just last week, Sarepta announced that a third clinical trial participant with limb-girdle muscular dystrophy died from liver failure caused by the experimental gene therapy, which is also suspected of Elevydis. Genetic medicines are still risky and in development, but difficulties like safety can spur innovation. This is where our special guest for this episode steps in.

After 25 years of leading drug programs in "big pharma," Amber Salzman left GSK after multiple family members were diagnosed with a hereditary neurodegenerative illness. She is currently the CEO of Epicrispr, a biotech company focused on epigenomic modulation, a promising area of genetic medicine. In this episode, Salzman discusses how Epicrispr is using their Gene Expression Modulation System (GEMS) to develop therapeutics for previously untreated genetic disorders, such as those that require finer tuning than simply turning genes on and off, as well as those that are too large for gene replacement, like DMD.

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Hosted by Jonathan D. Grinstein

Produced by Bill Levine

Audio mixed and mastered by David Mosely

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