Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...

The FDA has approved Capsida Biotherapeutics' investigational new drug (IND) application for CAP-003 to treat Parkinson’s ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007 for Stargardt disease.

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

SpliceBio's lead in-house pipeline project is in Stargardt disease, an IRD with no approved therapies that is caused by ...

This is a developing news story. Please check back soon for updates. The FDA granted VG801 rare pediatric disease designation ...

The FDA’s accelerated approval pathway plays a vital role, and scrapping it or reversing course on already-approved therapies ...