Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy ...

The three deaths resulted from acute liver failure in patients treated with either Elevidys or investigational therapies using the same AAVrh74 viral vector technology. One fatality occurred during a ...

Scientists have made major progress in treating congenital deafness using gene therapy. This approach repairs hearing loss by fixing the root cause—a faulty gene known as OTOF. The OTOF gene creates ...

Sarepta Therapeutics lays off 493 workers amid FDA probe, stock drop, and concerns over its gene therapy treatment, Elevidys.

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

The death of a third patient tied to a Sarepta Therapeutics gene therapy this year sent the company’s shares nosediving 36% Friday, halting the momentum the stock generated just a day earlier after ...