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...
Boston-based Decibel Therapeutics, in collaboration with Regeneron Pharmaceuticals, has been granted orphan drug designation by the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) for its lead gene therapy product candidate, DB-OTO. This...
