New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

The need to change regulations around gene-editing treatments was endorsed in November by the head of the US Food and Drug ...

As CRISPR moves from lab success to real-world medicine, 2026 will test whether these revolutionary gene-editing therapies can reach the patients who need them most or remain costly, complex ...

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

David Liu and Richard Merkin of the Broad Institute of MIT and Harvard, along with Columbia professor Sam Sternberg, have developed a new, targeted means of inserting entire genes into human DNA ...

Beam Therapeutics Inc. pivots from platform story to execution-driven thesis, with regulatory clarity accelerating BEAM-302 ...

Gene-editing techniques such as CRISPR-Cas9 have many uses in the area of food and agriculture. They can combat persistent drought and disease, and improve the colour and nutritional content of food ...

The CRISPR gene-editing tool can be used to silence an important hepatitis B virus gene, a proof-of-concept in vitro study suggests. "It's the first time we've seen CRISPR editing done in a hepatitis ...

Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the R75W mutation. Moreover, this gene editing technology can ...

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with ...