Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

Passage Bio, Inc. (($PASG)) announced an update on their ongoing clinical study. Study Overview: Passage Bio, Inc. is conducting a Phase 1b ...

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

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

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

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

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

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 ...

Elania is one of 59 children born with ADA-SCID who have been cured using this gene therapy, which will soon be submitted for U.S. Food and Drug Administration (FDA) approval, researchers reported Oct ...

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

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 ...

The company intends to divest Roctavian and “remove it from our portfolio,” CEO Alexander Hardy said, after nearly three ...