Third patient dies after receiving a Sarepta gene therapy

A 51-year-old man has died after receiving an experimental gene therapy developed by Sarepta Therapeutics.

The patient was treated with SRP-9004 (patidistrogene bexoparvovec), a gene therapy developed to treat non-ambulant limb-girdle muscular dystrophy.

This follows after two teenage boys died earlier this year after receiving a different Sarepta gene therapy, Elevidys, for the treatment of Duchenne muscular dystrophy (see BioNews 1282).

All patients died from acute liver failure. Liver toxicity is a known risk of gene therapies that use adeno-associated viruses as vectors to deliver modified genes.

Please see Genetic Engineering and Biotechnology News for more information.