Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq ...

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...

In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...

Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...

Failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...

On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study ...

The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...

CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...