AAVS1 gene
Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy
Sarepta Therapeutics; limb-girdle muscular dystrophy; SRP-9004; gene therapy; patient death; acute liver failure; FDA clinical hold; AAV vector; Elevidys; safety concerns
AstraZeneca’s Alexion Signs $825M AAV Capsid Deal with JCR Pharma
AstraZeneca; Alexion; JCR Pharmaceuticals; JUST-AAV; gene therapy; AAV capsid; rare disease; licensing deal; genomic medicines
Neurogene Details New Safety Measures After Young Patient’s Death in Rett Syndrome AAV Gene Therapy Trial
Neurogene; AAV gene therapy; Rett syndrome; NGN-401; ASGCT; safety measures; HLH; clinical trial; MECP2 gene; FDA; dose adjustment
Neurogene’s Rett Syndrome Trial Halted After Patient Death Linked to High-Dose Gene Therapy
Neurogene, Rett syndrome, gene therapy, AAV, clinical trial, patient death, high-dose complications
Neurogene’s Rett Syndrome Gene Therapy Shows Efficacy but Raises Safety Concerns
Neurogene, Rett syndrome, gene therapy, NGN-401, AAV vector, clinical trial, safety concerns, efficacy results
Roche and Dyno Therapeutics Launch $1B+ Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Neurological Diseases
Roche, Dyno Therapeutics, AAV gene therapy vectors, Neurological diseases, AI-driven gene delivery, LEAP technology, Gene therapy collaboration
Roche Revisits Dyno for AAV Technology with $50M Upfront Payment
Roche, Dyno Therapeutics, AAV technology, gene therapy, neurological diseases, strategic partnership
Purespring Therapeutics Secures £80M ($105M) Series B Funding to Advance IgAN Gene Therapy into Clinical Trials
Purespring Therapeutics, IgAN gene therapy, kidney disease, Series B funding, clinical trials, AAV gene therapy, podocytes, nephrotic syndrome, glomerular kidney disease.