Funding Clinical Trials

Our Agency funds clinical trials testing promising stem cell or gene therapies for currently incurable diseases or disorders to help patients with unmet medical needs. For basic information about stem cell trials, visit our Clinical Trials 101 page. For a list of clinical trials that are a result of early-stage CIRM grants click here.

96

CIRM-FUNDED CLINICAL TRIALS

Disease AreasInvestigatorOrganizationPhaseTrial StatusTarget EnrollmentDetail
Alpha Thalassemia Major Dr. Tippi C. Mackenzie MD University of California, San Francisco Phase 1 Enrolling by invitation 10
Pediatrics
Sickle Cell Disease
Mr. Donald B. Kohn University of California, Los Angeles Phase 1 Recruiting 6
Sickle Cell Disease Dr. De-Fu Zeng City of Hope, Beckman Research Institute Phase 1 Active, not recruiting 3
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) Mort Cowan University of California, San Francisco Phase 1 Recruiting 25
Sickle Cell Disease Dr Pierre Caudrelier ExCellThera Inc. Phase 1 Withdrawn N/A
Sickle Cell Disease Mark C. Walters M.D. University of California, San Francisco Phase 1 Not yet recruiting 9
IPEX Syndrome Dr. Rosa Bacchetta Stanford University Phase 1 Recruiting 30
Anemia Ryotaro Nakamura City of Hope, Beckman Research Institute Phase 1 Launching N/A
Immune Disease
Pediatrics
X-linked Chronic Granulomatous Disease
Mr. 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
Blood Cancer
Bone Marrow Transplant and Viral Infection
Michael A. Pulsipher Children’s Hospital of Los Angeles Phase 1/2 Active, not recruiting 60
Beta Thalassemia Bettina Cockroft Sangamo BioSciences, Inc. Phase 1/2 Completed 5
Immune Disease
Leukocyte Adhesion Deficiency
Dr. Kinnari Patel Rocket Pharmaceuticals, Inc. Phase 1/2 Completed 9
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
Genetic Disorder
Immune Disease
Pediatrics
Severe Combined Immunodeficiency, Adenosine deaminase-deficient (ADA-SCID)
Dr. Donald B Kohn
Leslie Meltzer
Mr. Donald B. Kohn
University of California, Los Angeles
Orchard Therapeutics plc
Phase 2 Completed 10
Sickle Cell Disease David Williams Boston Children’s Hospital Phase 2 Active, not recruiting 10