Gene therapy has changed what is possible for people living with sickle cell disease. New treatments have shown that the ...

Regulators have opened a one-time gene therapy to some of the youngest patients with sickle cell disease. On July 1, 2026, ...

The FDA expanded access to gene therapy for sickle cell disease, allowing children as young as 2 to receive the breakthrough ...

Digitized R&D workflows help cell therapy developers reduce manual data handling and accelerate biologics development.

Rare-disease families can now invest in Mahzi Therapeutics' gene therapy, giving patients a financial stake in developing ...

The Ara Parseghian Medical Research Fund will provide financial backing for the multi-year research effort.

Ocugen (NASDAQ:OCGN) outlined plans to advance three late-stage retinal disease programs, with company speaker Shankar saying ...

EpilepsyGTx, a biotechnology company developing cutting-edge gene therapies to treat refractory epilepsy, and ClearPoint ...

A gene therapy that instructs cells to produce more of an anti-ageing protein called klotho is about to be offered by a US company at overseas clinics to bypass FDA rules ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Sangamo Therapeutics ran out of cash. Eli Lilly and Astellas want the science it spent decades building.

Epilepsy affects more than 50 million people worldwide, making it one of the most common neurological disorders. Although ...