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

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

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

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

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

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

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

High cost is a major challenge. The global cell and gene therapy market is projected to expand by $6.56b, reflecting an ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

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

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

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