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.

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
Sickle Cell Disease	Dr. De-Fu Zeng	City of Hope, Beckman Research Institute	Phase 1	Active, not recruiting	3
Sickle Cell Disease	Dr. David Williams M.D.	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	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	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)	Dr Morton 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