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 ...

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 ...

"This was the first proof that 'gene therapy in a box' could work." Gene therapies or cell therapies that involve genetically modified cells today are available at only a limited number of research ...

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 ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

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.

What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...

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 ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

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 ...