For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...
Pharmaceutical Technology on MSN
Potentially “groundbreaking” FDA gene therapy pathway leaves key issues unresolved
A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.
SGT-212 has been granted FDA Rare Pediatric Disease and Fast Track designations –– SGT-212 is the only dual route gene therapy in development ...
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him. In 2022, London-based Orchard Therapeutics stopped ...
The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...
Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...
Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.
Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...
The first child in history has successfully been treated with a new genetic therapy for an ultra-rare developmental defect ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback