Mucopolysaccharidosis II

FDA Delays Denali’s Hunter Syndrome Therapy Decision by Three Months

2 weeks ago talkbio0Tagged Accelerated, Alfa, amendment, Approved, Biologics License Application, Clinical pharmacology discipline, Deferred, Denali, Mucopolysaccharidosis II, review extension, tividenofusp, United States Food and Drug Administration

FDA delay; Denali Therapeutics; Hunter syndrome; tividenofusp alfa; MPS II; Biologics License Application (BLA); review extension; clinical pharmacology; major amendment; accelerated approval

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

2 months ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

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

9 months ago talkbio0Tagged Gene, gene therapy, Mucopolysaccharidosis II, Nippon, Regenxbio, RGX-111, RGX-121, Shinyaku, strategic partnership

RegenXBio, Hunter syndrome, Gene therapy, Nippon Shinyaku, Strategic partnership, Rare metabolic disorder, Mucopolysaccharidosis type II (MPS II), RGX-121, RGX-111