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

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

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

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

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

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 ...

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

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

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 ...

Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, today announced that the first patient has ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

An experimental genetic therapy can cure most cases of the 'Bubble Boy' disease, ADA-SCID. In the rare disorder, children are ...