CIRM Clinical Trials
Through its strategic funding of clinical trials, CIRM helps advance innovative stem cell and gene therapy discoveries toward future treatments for patients.

CIRM funds clinical trials across a broad range of diseases and conditions that include rare and genetic disorders, treatment-resistant and refractory diseases, and many forms of cancer. Our involvement in the clinical trial process is vital to transforming potential treatments into available therapies by accelerating pathways to commercialization. Visit our Funding Opportunities page to apply for clinical trial research funding.
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CIRM-Funded Clinical Trials
Disease Areas | Investigator | Organization | Phase | Trial Status | Target Enrollment | Detail |
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Sickle Cell Disease | Mark C. Walters M.D. | University of California, San Francisco | Phase 1 | Not yet recruiting | 9 | |
Sickle Cell Disease | Dr. De-Fu Zeng | City of Hope, Beckman Research Institute | Phase 1 | Active, not recruiting | 3 | |
Sickle Cell Disease | Dr Pierre Caudrelier | ExCellThera Inc. | Phase 1 | Withdrawn | N/A | |
Sickle Cell Disease | David Williams | Boston Children’s Hospital | Phase 2 | Recruiting | 25 | |
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) | Mort Cowan | University of California, San Francisco | Phase 2 | Recruiting | 25 | |
Pediatrics Sickle Cell Disease |
Dr. Donald B. Kohn | University of California, Los Angeles | Phase 1 | Active, not recruiting | 4 | |
IPEX Syndrome | Dr. Rosa Bacchetta | Stanford University | Phase 1 | Recruiting | 30 | |
Immune Disease Pediatrics X-linked Chronic Granulomatous Disease |
Dr. Donald B. Kohn | University of California, Los Angeles | Phase 1/2 | Recruiting | 16 | |
Immune Disease Severe Combined Immunodeficiency, X-linked (X-SCID) |
Stephen Gottschalk | St. Jude Children’s Research Hospital | Phase 1/2 | Suspended | 28 | |
Immune Disease Pediatrics Severe Combined Immunodeficiency, X-linked (X-SCID) |
Dr. Judith A Shizuru Wendy Pang |
Stanford University Jasper Therapeutics, Inc. |
Phase 1/2 | Recruiting | 40 | |
Immune Disease Leukocyte Adhesion Deficiency |
Dr. Kinnari Patel | Rocket Pharmaceuticals, Inc. | Phase 1/2 | Completed | 9 | |
Genetic Disorder Immune Disease Pediatrics Severe Combined Immunodeficiency, Adenosine deaminase-deficient (ADA-SCID) |
Dr. Donald B. Kohn Leslie Meltzer |
University of California, Los Angeles Orchard Therapeutics plc |
Phase 2 | Completed | 10 | |
Bone Marrow Transplant and Viral Infection | Michael A. Pulsipher | Children’s Hospital of Los Angeles | Phase 1/2 | Active, not recruiting | 60 | |
Blood Disorders | Ilya Rachman MD PhD | Nexcella, Inc. | Phase 1 | Launching | N/A | |
Beta Thalassemia | Bettina Cockroft | Sangamo BioSciences, Inc. | Phase 1/2 | Completed | 5 | |
Anemia Bone Marrow Transplant and Viral Infection |
Alice Bertaina | Stanford University | Phase 1/2 | Launching | N/A | |
Anemia | Dr. Matthew H Porteus | Stanford University | Phase 1/2 | Launching | N/A | |
Anemia | Ryotaro Nakamura | City of Hope, Beckman Research Institute | Phase 1 | Launching | N/A | |
Alpha Thalassemia Major | Dr. Tippi C. MacKenzie | University of California, San Francisco | Phase 1 | Enrolling by invitation | 10 |