Key Highlights

Risk & Performance

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

0.0%

0 terminated/withdrawn out of 5 trials

Success Rate

100.0%

+13.5% 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

Phase 1

4(80.0%)

Phase 2

1(20.0%)

5Total

Phase 1(4)

Phase 2(1)

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Showing 5 of 5 trials

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

Role: lead

Gene Transfer for Sickle Cell Disease

Role: lead

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

Role: lead

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

Role: lead

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

Role: lead

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