GC301 / GeneCradle - LARVOL DELTA

Safety and Efficacy of AAV9-Mediated Gene Therapy GC301 in ERT-Stabilized Infantile-Onset Pompe Disease: An Open-Label, Single-Arm Study with Over 12 Months of Follow-Up (ASGCT 2025) - "This study suggests that AAV gene therapy has the potential to become a new therapeutic approach for IOPD. Disease Focus of Abstract:Rare Diseases"

Clinical • Gene therapy • Gene Therapies • Hepatology • Liver Failure • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Dosage Effects on Circulating GAA as a Pharmacodynamic Biomarker for systemically delivered rAAV9-coGAA Gene Therapy in Pompe Disease (ASGCT 2025) - "Early clinical trial results further support these findings, demonstrating that in IOPD patients treated with GC301, the increase in muscle GAA enzyme activity significantly exceeded that observed in the blood. Disease Focus of Abstract:Metabolic Disease"

Biomarker • Gene therapy • PK/PD data • Gene Therapies • Hematological Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug (clinicaltrials.gov) - P1/2 | N=33 | Recruiting | Sponsor: GeneCradle Inc

Gene therapy • New P1/2 trial • Gene Therapies • Pompe Disease

Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug (clinicaltrials.gov) - P1/2 | N=16 | Recruiting | Sponsor: GeneCradle Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Pompe Disease

Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug (clinicaltrials.gov) - P1/2 | N=16 | Not yet recruiting | Sponsor: GeneCradle Inc

Gene therapy • New P1/2 trial • Gene Therapies • Pompe Disease

Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease (clinicaltrials.gov) - P=N/A | N=6 | Recruiting | Sponsor: Seventh Medical Center of PLA General Hospital

New trial • Gene Therapies • Pompe Disease