gene therapy
FDA Grants Regenxbio Priority Review for MPS II Gene Therapy Regulatory Application
The FDA will issue a decision on the BLA for RGX-121 by November, which if positive, could make it the first gene therapy for Hunter syndrome.
India already has a homegrown CAR T-cell therapy, and other regions are exploring their own strategies, including developing them at the point of care.
FDA Clears Ensoma's IND to Launch Immune Disorder Gene Therapy Trial
EN-374 is a gene therapy designed to treat X-linked chronic granulomatous disease by modifying hematopoietic stem cells in vivo.
From decoy DNA sequences to plasmapheresis, researchers are testing strategies to get around anti-AAV antibodies that hinder gene therapy response.
Nuevocor Raises $45 Million in Series B Funding Round
The funds will support a Phase I/II trial of its gene therapy candidate in an aggressive form of dilated cardiomyopathy due to LMNA gene mutations.
Apr 24, 2025