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FDA reversal sets up high-stakes hearing on Sarepta gene therapy

March 17, 2023


Sarepta’s treatment could become the first gene therapy for Duchenne, a devastating genetic disorder for which the biotechnology company has successfully developed three other drugs. The benefit of those medicines is seen as limited at best, however, and they are only cleared for use in limited groups of Duchenne patients. SRP-9001 promises to more significantly alter the disease’s course, replacing the defective gene that’s behind it with a functional version.

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