Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the ...

The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Fractyl Health (Nasdaq:GUTS) wants to focus more on certain therapies, leading the company to cut nearly a fifth of its ...

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approval On track to dose first patient in Phase 3 trial in second half of 2025 LONDON, Feb. 03, 2025 (GLOBE NEWSWIRE) ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

MALVERN, Pa. - Ocugen, Inc. (NASDAQ: NASDAQ:OCGN), a biotechnology company with a current market capitalization of $212 million, announced today that the European Commission has endorsed the European ...

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program to ...

NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...

The bill would implement a moratorium for a decade. “I am not against vaccination in any way,” the freshman lawmaker said.