Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine This voice experience is generated by AI. Learn more. This ...

NANJING, China — A groundbreaking clinical trial has achieved what many thought impossible: restoring meaningful hearing in people born profoundly deaf, including teenagers and young adults who were ...

OCGN's gene-agnostic eye therapy aims to treat multiple retinal mutations, with key filings and pivotal data in 2026???2027 set to test its broad platform.

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) on Saturday presented data from multiple studies demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in patients aged 5 years ...

Vinay Prasad, M.D., is out again as the chief medical and scientific officer for the U.S. Food and Drug Administration (FDA).

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends. It was a contrast to how Cole, 18, spent part of the 2024 holiday ...

RESEARCH TRIANGLE PARK, N.C., July 23, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (IRD) (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of ...