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.
0
CIRM-Funded Clinical Trials
Disease Areas | Investigator | Organization | Phase | Trial Status | Target Enrollment | Detail |
---|---|---|---|---|---|---|
Sickle Cell Disease | David Williams | Boston Children’s Hospital | Phase 2 | Recruiting | 25 | |
Bone Marrow Transplant and Viral Infection | Michael A. Pulsipher | Children’s Hospital of Los Angeles | Phase 1/2 | Active, not recruiting | 60 | |
Anemia | Ryotaro Nakamura | City of Hope, Beckman Research Institute | Phase 1 | Launching | N/A | |
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Sickle Cell Disease | Dr Pierre Caudrelier | ExCellThera Inc. | Phase 1 | Withdrawn | N/A | |
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 | |
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 |
University of California, Los Angeles Orchard Therapeutics plc |
Phase 2 | Completed | 10 | |
Immune Disease Leukocyte Adhesion Deficiency |
Dr. Kinnari Patel | Rocket Pharmaceuticals, Inc. | Phase 1/2 | Completed | 9 | |
Beta Thalassemia | Bettina Cockroft | Sangamo BioSciences, Inc. | Phase 1/2 | Completed | 5 | |
Immune Disease Severe Combined Immunodeficiency, X-linked (X-SCID) |
Stephen Gottschalk | St. Jude Children’s Research Hospital | Phase 1/2 | Suspended | 28 | |
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 | |
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 | |
Pediatrics Sickle Cell Disease |
Dr. Donald B. Kohn | University of California, Los Angeles | Phase 1 | Active, not recruiting | 4 | |
Alpha Thalassemia Major | Dr. Tippi C. MacKenzie | University of California, San Francisco | Phase 1 | Enrolling by invitation | 10 | |
Sickle Cell Disease | Mark C. Walters M.D. | University of California, San Francisco | Phase 1 | Not yet recruiting | 9 | |
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) | Mort Cowan | University of California, San Francisco | Phase 2 | Recruiting | 25 |