Funding supports safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Rybrevant Faspro, the new subcutaneous version of amivantamab, was approved for patients with a specific form of lung cancer ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval. The FDA has approved the gene therapy ...

“Bubble boy disease” was once a death sentence. A scientific breakthrough changed that. By Simar Bajaj A common cold was enough to kill Cora Oakley. Born in Morristown, N.J., with virtually no immune ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

Shares of the gene therapy developer rose 12.5% to $9.55 in premarket trading on Monday after the news. Sign up here. Under the deal, MeiraGTx will get an upfront payment of $75 million and ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...

Opal Sandy underwent a gene therapy injection to her right ear in 2023. A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...