JLK-247 / Phoenix Nest - LARVOL DELTA

Favorable Efficacy and Safety in Preclinical Studies Pave the Way for AAV9-HGSNAT Gene Therapy for MPS IIIC (ASGCT 2026) - "Early postnatal intervention confers better therapeutic benefit, providing robust correction of neurological, behavioral, and biochemical abnormalities. These results strongly support the translational potential of AAV9/HGSNAT gene therapy, particularly for early intervention in patients with MPS IIIC, and highlight its promise as a disease-modifying treatment for this currently untreatable disorder."

Gene therapy • Preclinical • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

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