The brain drug developer plans to slash R&D expenses by about 60% in the wake of the FDA surprisingly rejecting its rare disease drug Vyglxia.

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

A CRISPR-based gene therapy showed improvement in lipids in difficult-to-treat patients, but one study raised flags after a ...

The alliance extends a deal streak in genetic medicine for Lilly and hands the company rights to an experimental treatment ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the ...

A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...

Opal Sandy underwent a gene therapy injection to her right ear in 2023. A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...