| Phase 3 |
| NCT06971094 |
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the… |
SMA - Spinal Muscular Atrophy |
Recruiting |
2025-06-05 |
| Phase 1/2 |
| NCT05824169 |
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… |
Spinal Muscular Atrophy |
Recruiting |
2025-07-03 |
| NCT06391736 |
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug |
Pompe Disease (Late-onset) |
Recruiting |
2025-07-03 |
| NCT06421831 |
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… |
Spinal Muscular Atrophy Type 3 |
Recruiting |
2025-07-03 |
| NCT07173933 |
Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310… |
Wilson Disease |
Not Yet Recruiting |
2025-09-15 |
| NCT05793307 |
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug |
Pompe Disease Infantile-Onset |
Active, Not Recruiting |
2025-07-03 |
| NCT05901987 |
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… |
SMA II |
Active, Not Recruiting |
2025-07-03 |
| Phase 1 |
| NCT05860569 |
Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic… |
Hypertriglyceridemia, Familial |
Recruiting |
2025-07-03 |