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0 terminated/withdrawn out of 3 trials

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed trials have results

Key Signals

1 recruiting

Enrollment Performance

Analytics

Phase 1
3(100.0%)
3Total
Phase 1(3)

Activity Timeline

Global Presence

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Clinical Trials (3)

Showing 3 of 3 trials
NCT07070999Phase 1Recruiting

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Role: lead

NCT04771416Phase 1Suspended

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

Role: lead

NCT04713475Phase 1Active Not Recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

Role: lead

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