Pictured: Gene therapy illustration showing an injection targeting a specific DNA site/iStock, Ilya Lukichev The FDA on Monday accepted Orchard Therapeutics’ Biologics License Application for OTL-200, an investigational gene therapy for rare disease metachromatic...
While FDA panelists supported Alnylam Pharmaceuticals’ medicine in transthyretin amyloidosis cardiomyopathy, they recommended a limited FDA approval that would severely restrict the med’s addressable population. (FDA) After Alnylam Pharmaceuticals faced...
New Jersey’s Rocket Pharmaceuticals has successfully aligned with the FDA on the design of a pivotal Phase 2 trial for a rare disease, potentially hastening the approval of their gene therapy. This open-label study will enlist 12 patients for treatment with...
The Montreal Children’s Hospital has reported a significant breakthrough: a nine-day-old baby named Samuel has become the youngest patient in North America to receive gene therapy for a rare, degenerative disease called spinal muscular atrophy (SMA). SMA affects...
The first proof of an effective gene therapy for a metabolic disease of the liver is reported in patients with Crigler-Najjar syndrome. No serious adverse events were reported in the phase I/II study evaluating the safety and efficacy of a gene therapy for patients...