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 |
|---|---|---|---|---|---|---|
| Sickle Cell Disease | Dr. De-Fu Zeng | City of Hope, Beckman Research Institute | Phase 1 | Active, not recruiting | 3 | |
| Sickle Cell Disease | David Williams | Boston Children’s Hospital | Phase 2 | Recruiting | 25 | |
| Sickle Cell Disease | Mark C. Walters M.D. | University of California, San Francisco | Phase 1 | Not yet recruiting | 9 | |
| 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 | |
| Anemia | Dr. Matthew H Porteus | Stanford University | Phase 1/2 | Launching | N/A | |
| Immune Disease Leukocyte Adhesion Deficiency |
Dr. Kinnari Patel | Rocket Pharmaceuticals, Inc. | Phase 1/2 | Completed | 9 | |
| 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 | |
| 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 | |
| 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 | |
| Immune Disease Pediatrics X-linked Chronic Granulomatous Disease |
Dr. Donald B. Kohn | University of California, Los Angeles | Phase 1/2 | Recruiting | 16 | |
| Pediatrics Sickle Cell Disease |
Dr. Donald B. Kohn | University of California, Los Angeles | Phase 1 | Active, not recruiting | 4 | |
| Immune Disease Severe Combined Immunodeficiency, X-linked (X-SCID) |
Stephen Gottschalk | St. Jude Children’s Research Hospital | Phase 1/2 | Suspended | 28 | |
| Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) | Mort Cowan | University of California, San Francisco | Phase 2 | Recruiting | 25 | |
| Beta Thalassemia | Bettina Cockroft | Sangamo BioSciences, Inc. | Phase 1/2 | Completed | 5 | |
| Sickle Cell Disease | Dr Pierre Caudrelier | ExCellThera Inc. | Phase 1 | Withdrawn | N/A | |
| Anemia Bone Marrow Transplant and Viral Infection |
Alice Bertaina | Stanford University | Phase 1/2 | Launching | N/A | |
| IPEX Syndrome | Dr. Rosa Bacchetta | Stanford University | Phase 1 | Recruiting | 30 |
