Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 65/100

Failure Rate

20.0%

1 terminated/withdrawn out of 5 trials

Success Rate

50.0%

-36.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

100%

1 of 1 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

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Clinical Trials (5)

Showing 5 of 5 trials
NCT02725580Phase 1Completed

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

Role: lead

NCT04273243Active Not Recruiting

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

Role: lead

NCT07173153Phase 1Enrolling By Invitation

Gene Therapy for SLC6A1 Neurodevelopmental Disorder

Role: lead

NCT04644549Terminated

Natural History Study of Batten Disease

Role: lead

NCT03285425Active Not Recruiting

Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

Role: lead

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