Gene Therapy

The FDA’s decision to approve a one-time gene therapy for adults with moderate to severe hemophilia B could have a considerable impact on the treatment landscape, according to a researcher involved in the pivotal trial for the agent.

The FDA has granted regenerative medicine advanced therapy designation to an intrathecally delivered AAV9 gene transfer therapy to treat younger individuals with Rett syndrome, according to the manufacturer.

Gene therapy improved exercise capacity and blood flow and reduced chest pain occurrence and nitroglycerine use for patients with advanced ischemic heart disease, a speaker reported.

The American Association for the Advancement of Science elected orthopedic surgeon Jay R. Lieberman, MD, as a 2024 fellow for his research in gene therapy and joint replacement, according to a press release.

Ocugen has announced that dosing is complete in the second cohort of its phase 1/2 ArMaDa clinical trial to assess the safety of OCU410, a modifier gene therapy candidate for geographic atrophy.

The FDA approved fidanacogene elaparvovec-dzkt for treatment of adults with moderate to severe hemophilia B.

Exagamglogene autotemcel gave most patients with beta-thalassemia transfusion independence and drastically reduced the number of vaso-occlusive crises in those with sickle cell disease in a pair of phase 3 trials.

Bayer AG and its subsidiary Asklepios BioPharmaceutical announced the FDA granted fast track designation for its investigational gene therapy for the treatment of congestive heart failure.

In a video interview, Majda Hadziahmetovic, MD, retinal specialist and assistant professor of ophthalmology at Duke University discusses improving early detection and risk prediction for AMD patients.

In a video interview, Majda Hadziahmetovic, MD, retinal specialist and assistant professor of ophthalmology at Duke University discusses the necessary collaboration between ophthalmologists and optometrists on AMD care and management.