Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA) — a rare, life-threatening disorder in which bone marrow ...

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

UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Discover various promising treatments in development to address the unmet needs of people with Charcot–Marie–Tooth disease.

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

An experimental vaccine showed promise in treating aggressive breast cancer, while a new gene therapy method advances ...

Proliferative vitreoretinopathy (PVR) is an eye condition that leads to blindness and can currently only be addressed with ...

The global macular degeneration treatment market is poised for significant growth, projected to expand from USD 9.1 billion ...

The U.S. Food and Drug Administration is weighing the need for a regulatory action on bluebird bio's gene therapy for a rare ...

Novartis's acquisition of Kate Therapeutics gives it a pipeline of gene therapies for three genetic neuromuscular diseases.

Wilson celebrates the first U.S. patient to complete gene therapy for sickle cell, but says advocacy must continue.

Novartis has acquired Kate Therapeutics for up to $1.1 billion, the companies said today, in a deal that expands the buyer’s ...