#gene-therapy

A new Breakthrough Prize for the team behind the first gene therapy approved in the United States highlights how a once-experimental field became a clinical reality.

An Endpoints News report says five beta thalassemia patients treated with a Chinese gene-editing therapy no longer need regular transfusions, hinting at rising global competition in genetic medicine.

A new report says a novel gene-based therapy helped nerves heal better after severe peripheral injury, offering a potential advance for patients who often face lasting weakness, numbness, or loss of function.

Life Biosciences, a startup co-founded by Harvard biologist David Sinclair, has announced an $80 million financing round to support a clinical test of a one-time anti-aging gene therapy.

A correspondence in Nature Medicine argues that bespoke pediatric gene therapies are arriving faster than the traditional drug model can handle, and calls for a new regulatory framework built for individualized medicines

Prime Medicine reverses course to seek FDA approval for a previously sidelined rare immune disease therapy, citing the agency's evolving stance on rare diseases.