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.
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CIRM-Funded Clinical Trials
Photo Credit: UCLA Broad Stem Cell Research Center
Funding Opportunities
Visit our Funding Opportunities page to apply for clinical trial research funding.
| 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 Disorders | Ilya Rachman MD PhD | Nexcella, Inc. | Phase 1 | Launching | N/A | |
| Bone Marrow Transplant and Viral Infection | Michael A. Pulsipher | Children’s Hospital of Los Angeles | Phase 1/2 | Active, not recruiting | 60 | |
| 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 |
Phase 2 | Completed | 10 | |
| 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 |
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 | University of California, Los Angeles | Phase 1 | Active, not recruiting | 4 | |
| Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) | Dr Morton Cowan | University of California, San Francisco | Phase 2 | 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 |
