Gene Therapy Setback: REGENXBIO’s RGX-121 Faces Regulatory Hurdles in Ultra-Rare Disease Treatment

Gene Therapy for Duchenne Muscular Dystrophy: Genethon Confirms Two-Year Efficacy in Patients Treated with Its Drug Candidate GNT0004 at Therapeutic Dose in the First Phase of Its Clinical Trial

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando

REGENXBIO reports positive data from Phase 1/2 trial of RGX-202 in DMD - BioTuesdays

REGENXBIO (NASDAQ: RGNX) has announced new positive interim data from the Phase 1/2 AFFINITY DUCHENNE trial of RGX-202—a potential best-in-class therapy for Duchenne muscular dystrophy (DMD). Trial investigator Carolina Tesi-Rocha, MD, clinical professor, neurology, Stanford School of Medicine, Stanford Children’s Health, will present the AFFINITY DUCHENNE trial data, including new functional, safety, biomarker, and cardiac MRI measures, at the …

REGENXBIO Reports New Positive Interim Data from Phase I/II AFFINITY DUCHENNE Gene Therapy Trial

REGENXBIO GENE THERAPYRGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key muscle damage biomarkers one year after treatment. Interim results from seven participants indicate improved functional performance and stable cardiac function, with strong microdystrophin expression, while Phase III topline data are expected in Q2…

Gene Therapy Setback: REGENXBIO’s RGX-121 Faces Regulatory Hurdles in Ultra-Rare Disease Treatment

REGENXBIO encountered a significant regulatory obstacle when the FDA issued a complete response letter regarding its biologics license application for RGX-121, an investigational gene therapy targeting mucopolysaccharidosis II (MPS II), commonly known as Hunter syndrome. As reported by Pharmaceutical Technology, the decision represents a setback for the biotech company’s decade-long development effort to bring this […]