The US Food and Drug Administration (FDA) has placed a clinical hold on REGENXBIO’s investigational gene therapy, RGX-111, for the treatment of MPS I, following a single case of neoplasm (intraventricular CNS tumour) in a participant treated in its Phase I/II study. The FDA also placed a… Read More
AFFINITY DUCHENNE, a multi-centre, open-label trial of gene therapy RGX-202 for Duchenne muscular dystrophy has dosed its first patient, REGENXBIO has announced. The company also revealed new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data. Read More
New data has revealed a robust microdystrophin expression from a pivotal dose of gene therapy RGX-202 for Duchenne muscular dystrophy (Duchenne). RGX-202 is an investigational one-time AAV therapeutic designed to deliver a novel microdystrophin gene that includes key regions of the naturally occurring dystrophin gene. Read More
