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.

109

CIRM-FUNDED CLINICAL TRIALS

Disease Areas	Investigator	Organization	Phase	Trial Status	Target Enrollment
Alpha Thalassemia Major	Dr. Tippi C. MacKenzie MD	University of California, San Francisco	Phase 1	Enrolling by invitation	10
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
Beta Thalassemia	Bettina Cockroft	Sangamo BioSciences, Inc.	Phase 1/2	Completed	5
Blood Cancer 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
Genetic Disorder Immune Disease Pediatrics Severe Combined Immunodeficiency, Adenosine deaminase-deficient (ADA-SCID)	Dr. Donald B Kohn Leslie Meltzer Dr. Donald B. Kohn PhD	University of California, Los Angeles Orchard Therapeutics plc	Phase 2	Completed	10
Immune Disease Pediatrics X-linked Chronic Granulomatous Disease	Dr. Donald B. Kohn PhD	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
IPEX Syndrome	Dr. Rosa Bacchetta	Stanford University	Phase 1	Recruiting	30
Pediatrics Sickle Cell Disease	Dr. Donald B. Kohn PhD	University of California, Los Angeles	Phase 1	Active, not recruiting	4
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID)	Mort Cowan	University of California, San Francisco	Phase 1	Recruiting	25
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
Sickle Cell Disease	Mark C. Walters M.D.	University of California, San Francisco	Phase 1	Not yet recruiting	9