Sarepta Therapeutics is developing gene therapies for DMD. The FDA has mandated a black box warning for ALF due to patient deaths linked to the compound in trials.
1 report, 1 independent
Updated Jul 21
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What happened
Sarepta Therapeutics is developing gene therapies for DMD. The FDA has mandated a black box warning for ALF due to patient deaths linked to the compound in trials.
Who's involved
What this event is mainly aboutKeep exploring
The entities involved
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DMD
Nothing else this week.
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Sarepta Therapeutics
biopharmaceutical company
Nothing else this week.
- FDA
Related events
- FDA placed a clinical hold on a gene therapy involving Hunter.
- Solid Biosciences received FDA approval for a gene therapy trial on July 9, 2025.
- The FDA reviewed gene therapy applications and provided milestone payments for dosing involving Regenxbio and AbbVie.
- Experimental therapies require approval from the FDA.
- The FDA reviewed the Biologics License Application (BLA) for MOGENRY, a gene therapy, involving Nanoscope Therapeutics, Inc.
Coverage
Newest first; wire copies grouped- ZacksSRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its experimenta