| NCT ID | Title | Condition | Status |
|---|---|---|---|
| Phase 3 | |||
| NCT06971094 | Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the… | SMA - Spinal Muscular Atrophy | Recruiting |
| Phase 1/2 | |||
| NCT05824169 | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… | Spinal Muscular Atrophy | Recruiting |
| NCT06391736 | Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug | Pompe Disease (Late-onset) | Recruiting |
| NCT06421831 | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… | Spinal Muscular Atrophy Type 3 | Recruiting |
| NCT05793307 | Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug | Pompe Disease Infantile-Onset | Active, Not Recruiting |
| NCT05901987 | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… | SMA II | Active, Not Recruiting |
| NCT07173933 | Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310… | Wilson Disease | Not Yet Recruiting |
| Phase 1 | |||
| NCT05860569 | Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic… | Hypertriglyceridemia, Familial | Recruiting |