A recent gene therapy death shines a light on AAV safety

The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy community.

The patient died from acute liver failure months after starting Elevidys, the only FDA-approved gene therapy for DMD. Its maker, Sarepta Therapeutics, hasn’t ruled out the possibility that the treatment, which uses an adeno-associated virus vector to deliver its therapeutic gene payload, may have contributed to the fatality.