Mucopolysaccharidosis II
FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121
FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease
RegenXBio Secures $110M Upfront in Strategic Partnership with Nippon Shinyaku for Hunter Syndrome Gene Therapy
RegenXBio, Hunter syndrome, Gene therapy, Nippon Shinyaku, Strategic partnership, Rare metabolic disorder, Mucopolysaccharidosis type II (MPS II), RGX-121, RGX-111