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 |
---|---|---|---|---|---|---|
Alpha Thalassemia Major | Dr. Tippi C. MacKenzie | University of California, San Francisco | Phase 1 | Enrolling by invitation | 10 | |
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 | |
Beta Thalassemia | Bettina Cockroft | Sangamo BioSciences, Inc. | Phase 1/2 | Completed | 5 | |
Blood Cancer 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 | |
Genetic Disorder Immune Disease Pediatrics Severe Combined Immunodeficiency, Adenosine deaminase-deficient (ADA-SCID) |
Dr. Donald B Kohn Leslie Meltzer Dr. Donald B. Kohn PhD |
University of California, Los Angeles Orchard Therapeutics plc |
Phase 2 | Completed | 10 | |
Immune Disease Pediatrics X-linked Chronic Granulomatous Disease |
Dr. Donald B. Kohn PhD | 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 | |
IPEX Syndrome | Dr. Rosa Bacchetta | Stanford University | Phase 1 | Recruiting | 30 | |
Pediatrics Sickle Cell Disease |
Dr. Donald B. Kohn PhD | University of California, Los Angeles | Phase 1 | Active, not recruiting | 4 | |
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) | Mort Cowan | University of California, San Francisco | Phase 1 | Recruiting | 25 | |
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 |