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 ...

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

KaDee Troop is the mother of seven adopted children, four of whom have a rare genetic disorder that causes their skin to blister and tear at the slightest touch. Wounds heal slowly — if at all — and ...

In an interview ahead of Advanced Therapies Week, Anna Catalanotto of Cardinal Health discussed growth in cell and gene ...

"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 ...

(ORLANDO, Dec. 6, 2025) Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell ...

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.

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger ...

Results from a three-year trial suggest an experimental gene therapy for Huntington's disease can slow the progression of the deadly condition by 75%. When you purchase through links on our site, we ...

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 ...

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 ...