GC101 / GeneCradle - LARVOL DELTA

Innovative Intrathecal AAV9-mediated Gene Therapy for Spinal Muscular Atrophy: Phase I/II Safety and Efficacy Topline Results (ASGCT 2025) - "GC101, an intrathecal AAV9-mediated gene therapy, is well-tolerated and shows promising efficacy in Type 2 SMA patients. Patients previously treated with Nusinersen and/or Risdiplam can safely benefit from GC101. These results support GC101's ongoing development as a potential Type 2 SMA treatment, with further studies needed to assess long-term safety and efficacy."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Genetic Disorders • Infectious Disease • Movement Disorders • Muscular Atrophy • Pediatrics • Rare Diseases • Respiratory Diseases • SMA4 • SMN1

Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study. (PubMed, Chin Med J (Engl)) - No abstract available

Gene therapy • Journal • Gene Therapies

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients (clinicaltrials.gov) - P1/2 | N=21 | Recruiting | Sponsor: GeneCradle Inc

Gene therapy • New P1/2 trial • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients (clinicaltrials.gov) - P1/2 | N=33 | Recruiting | Sponsor: GeneCradle Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients (clinicaltrials.gov) - P1/2 | N=33 | Not yet recruiting | Sponsor: GeneCradle Inc

Gene therapy • New P1/2 trial • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: GeneCradle Inc

Gene therapy • New P1/2 trial • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases