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 ...

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

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

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

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

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.

Understanding what to patent versus what to keep as a trade secret is an increasingly important and challenging decision ...

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...

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