A Year of DMD Gene Therapy Trial Failures

The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.

The gene therapy landscape for Duchenne muscular dystrophy (DMD) has encountered major hurdles in 2023 and 2024, as 2 high-profile trials—Sarepta’s EMBARK and Pfizer’s CIFFREO—failed to meet their primary endpoints.