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

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

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

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

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.

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

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

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

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

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

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing ...