Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 75/100

Failure Rate

25.0%

1 terminated/withdrawn out of 4 trials

Success Rate

75.0%

-11.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

33%

1 of 3 completed trials have results

Key Signals

1 with results

Enrollment Performance

Analytics

Phase 1
2(100.0%)
2Total
Phase 1(2)

Activity Timeline

Global Presence

Loading network data...

Clinical Trials (4)

Showing 4 of 4 trials
NCT04240314Phase 1Completed

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

Role: collaborator

NCT03223194Phase 1Terminated

Gene Transfer Clinical Study in Crigler-Najjar Syndrome

Role: lead

NCT02453152Completed

Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

Role: collaborator

NCT03078881Completed

Clinical Assessment Study in Crigler-Najjar Syndrome

Role: lead

All 4 trials loaded