adeno-associated virus (AAV)-based gene therapy / Vertex, Affinia Therap - LARVOL DELTA

High-Yield, Pan-Serotype Plasmid System for Manufacturing Adeno-Associated Virus Gene Therapies: Cost and Efficiency Benefits for R&D and Commercial Processes (ASGCT 2025) - "A major challenge in development of adeno-associated virus (AAV)-based gene therapies is the low manufacturing yield and poor packaging efficiency of AAV capsids...In summary, this innovative plasmid system addresses critical manufacturing challenges, enabling more efficient production of AAV gene therapies and unlocking the potential of diverse capsid platforms for development, clinical, and commercial use. Disease Focus of Abstract:None"

Gene therapy • Gene Therapies

Intrathecal Gene Therapy for Giant Axonal Neuropathy. (PubMed, N Engl J Med) - P1 | "Intrathecal gene transfer with scAAV9/JeT-GAN for giant axonal neuropathy was associated with adverse events and resulted in a possible benefit in motor function scores and other measures at some vector doses over a year. Further studies are warranted to determine the safety and efficacy of intrathecal AAV-mediated gene therapy in this disorder. (Funded by the National Institute of Neurological Disorders and Stroke and others; ClinicalTrials.gov number, NCT02362438.)."

Gene therapy • Journal • Cardiovascular • CNS Disorders • Gene Therapies • Pain • Pediatrics