Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...
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 ...
Passage Bio, Inc. (($PASG)) announced an update on their ongoing clinical study. Study Overview: Passage Bio, Inc. is conducting a Phase 1b ...
Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program. North Carolina-based Opus ...
RegenXBio Inc. (($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 ...
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 ...
DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...
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