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 AreasInvestigatorOrganizationPhaseTrial StatusTarget EnrollmentDetail
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Sickle Cell Disease Dr Pierre Caudrelier ExCellThera Inc. Phase 1 Withdrawn N/A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
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) Mort Cowan University of California, San Francisco Phase 2 Recruiting 25
Pediatrics
Sickle Cell Disease		 Dr. Donald B. Kohn University of California, Los Angeles Phase 1 Active, not recruiting 4
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
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
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
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
Beta Thalassemia Bettina Cockroft Sangamo BioSciences, Inc. Phase 1/2 Completed 5
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
Alpha Thalassemia Major Dr. Tippi C. MacKenzie University of California, San Francisco Phase 1 Enrolling by invitation 10