Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 8 trials

Late-Stage Pipeline

13%

1 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed trials have results

Key Signals

5 recruiting

Enrollment Performance

Analytics

Phase 1
7(87.5%)
Phase 3
1(12.5%)
8Total
Phase 1(7)
Phase 3(1)

Activity Timeline

Global Presence

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Clinical Trials (8)

Showing 8 of 8 trials
NCT07173933Phase 1Not Yet Recruiting

Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)

Role: lead

NCT05793307Phase 1Active Not Recruiting

Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug

Role: lead

NCT06391736Phase 1Recruiting

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Role: lead

NCT05901987Phase 1Active Not Recruiting

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

Role: lead

NCT05824169Phase 1Recruiting

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Role: lead

NCT06421831Phase 1Recruiting

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Role: lead

NCT05860569Phase 1Recruiting

Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis

Role: lead

NCT06971094Phase 3Recruiting

Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III

Role: lead

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