Second patient dies after receiving Elevidys gene therapy

A second patient has died from acute liver failure after receiving Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD).

Sarepta Therapeutics, the company producing Elevidys, provided a safety update over the weekend.

In March a 16-year-old with DMD died from acute liver failure after treatment with Elevidys (see BioNews 1282).

Liver toxicity is a known risk of gene therapies that use adeno-associated viruses as vectors to deliver modified genes.

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