Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

0.0%

0 terminated/withdrawn out of 6 trials

Success Rate

100.0%

+13.4% vs industry average

Late-Stage Pipeline

0 trials in Phase 3/4

Results Transparency

50%

1 of 2 completed trials have results

Key Signals

1 recruiting1 with results

Enrollment Performance

Analytics

Phase 1

4(80.0%)

Phase 2

1(20.0%)

5Total

Phase 1(4)

Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (6)

Showing 6 of 6 trials

NCT07640815Unknown

Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

Role: lead

NCT05353647Phase 2Active Not Recruiting

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

Role: lead

NCT01129544Phase 1Completed

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector

Role: lead

NCT03282656Phase 1Active Not Recruiting

Gene Transfer for Sickle Cell Disease

Role: lead

NCT03311503Phase 1Recruiting

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Role: lead

NCT01410825Phase 1Completed

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

Role: lead

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