Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

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

RegenXBio Inc. (($RGNX)) announced an update on their ongoing clinical study. RegenXBio Inc. is conducting a clinical study titled ‘AFFINITY ...

A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...

CAR T-cell therapy uses re-engineered immune cells to attack diseases. After proving its effectiveness on cancer patients, it ...

To solve this critical challenge, he founded Nephrogen in 2022, a biotech startup that uses AI and advanced screening to ...

Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program. North Carolina-based Opus ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their high cost limits ...