Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Low Risk

Score: 20/100

Failure Rate

11.1%

1 terminated/withdrawn out of 9 trials

Success Rate

85.7%

-0.8% vs industry average

Late-Stage Pipeline

56%

5 trials in Phase 3/4

Results Transparency

100%

6 of 6 completed trials have results

Key Signals

6 with results

Enrollment Performance

Analytics

Phase 3
5(71.4%)
Phase 1
2(28.6%)
7Total
Phase 3(5)
Phase 1(2)

Activity Timeline

Global Presence

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Clinical Trials (9)

Showing 9 of 9 trials
NCT03505099Phase 3Completed

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

Role: lead

NCT03306277Phase 3Completed

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Role: lead

NCT03461289Phase 3Completed

Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Role: lead

NCT03381729Phase 1Terminated

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

Role: lead

NCT03837184Phase 3Completed

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Role: lead

NCT05481164Completed

Newborn Screening for Spinal Muscular Atrophy

Role: collaborator

NCT04042025Phase 3Active Not Recruiting

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Role: lead

NCT03421977Active Not Recruiting

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Role: lead

NCT02122952Phase 1Completed

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

Role: lead

All 9 trials loaded