Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...

On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...

Clinical Trial Success at Cincinnati Children's Supports Recent US FDA Approval for First Therapy to Directly Replace a Gene in the Brain ...

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.