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 ...

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 ...

"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 ...

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 ...

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

A research team says that for the first time in history, it’s successfully treated Huntington’s disease, a devastating and inherited disorder that kills nerve cells and leads to rapid decline and ...

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 ...

The FDA has put a clinical hold on a Tenaya Therapeutics gene therapy study while the biotech works to standardize activities related to the immunosuppression regimen across trial sites. Tenaya said ...

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 ...

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.