#gene-therapy

A small clinical study suggests engineered immune cells may help some people with HIV maintain undetectable virus levels without ongoing medication, raising the prospect of longer-lasting control beyond daily antiretroir

Coverage from the American Society of Gene and Cell Therapy meeting suggests in vivo CAR-T is becoming one of the field's biggest themes, even though the work is still largely preclinical.

Encoded Therapeutics reported that an experimental gene therapy reduced seizures by 76% in children with Dravet syndrome, but the supplied source text indicates the result was observed in only three patients.

European regulators have cleared Fractyl Health to begin what the source describes as the first clinical test of a GLP-1 gene therapy. The decision signals how aggressively drug developers are pursuing alternatives to长期?

Researchers studying axolotls, zebrafish, and mice report a shared genetic program behind tissue regeneration, and say a zebrafish-inspired gene therapy partially restored regrowth in mice.

The FDA is rolling out a framework that could allow certain individualized gene therapies to reach patients with rare genetic disorders without human clinical trials, accelerating access while raising serious safety and信

The U.S. FDA has approved Regeneron’s Otarmeni, the first gene therapy cleared for hearing loss caused by OTOF mutations, marking a major milestone for genetic medicine and auditory research.

Researchers at the University of Trento report an mRNA-based genome-editing approach aimed at a cystic fibrosis mutation that affects about 10% of patients and does not respond to some current drug therapies.

Regeneron says it has secured the first FDA approval for a gene therapy targeting a rare inherited form of hearing loss and plans to offer the treatment free in the United States.

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.