JLK-247 / Phoenix Nest - LARVOL DELTA

Gene Replacement Therapy for MPS IIIC with scAAV9/HGSNAT Vector (ASGCT 2025) - "The findings highlight that CNS-directed gene replacement therapy with JLK-247 is a promising strategy to treat MPSIIIC. Further studies will assess postmortem histological and biomarker changes in these mice. GLP toxicology studies will assess the safety of JLK-247 to advance this experimental gene therapy approach towards clinical application."

Alzheimer's Disease • CNS Disorders • Dementia • Developmental Disorders • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Psychiatry • Rare Diseases