Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 75/100

Failure Rate

16.7%

1 terminated/withdrawn out of 6 trials

Success Rate

80.0%

-6.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 4 completed trials have results

Key Signals

Enrollment Performance

Analytics

Phase 1
3(75.0%)
Phase 2
1(25.0%)
4Total
Phase 1(3)
Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (6)

Showing 6 of 6 trials
NCT04273269Phase 1Terminated

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Role: lead

NCT04310163Completed

Interviews and Video Capture in Patients With GM1 Gangliosidosis

Role: lead

NCT02746341Completed

Natural History Study of Patients With MPS IIIA

Role: lead

NCT03612869Phase 2Unknown

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Role: lead

NCT02053064Phase 1Completed

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Role: lead

NCT01474343Phase 1Completed

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Role: lead

All 6 trials loaded