Rapid Unfolding of Concerns in Gene Therapy Developments

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy Elevidys and growing scrutiny over the company’s transparency following multiple patient deaths. 

In this week’s episode of "The Top Line," we explore Sarepta’s brief standoff with the FDA over Elevidys, a short-lived marketing pause, and the regulator’s surprise reversal on the treatment. Fierce Pharma’s Fraiser Kansteiner and Angus Liu sit down with Fierce Biotech’s Gabrielle Masson to recap the story and discuss the broader implications the Elevidys saga may have for gene therapy, the FDA and the pharmaceutical industry.

To learn more about the topics in this episode: 

• Sarepta pivots to siRNA and lays off 500 staffers as Elevidys gets box warning
• Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
• Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys
• FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients
• Analysts demand transparency after Sarepta's roundabout disclosure of 3rd patient death
• Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe

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