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
Pediatrics
Sickle Cell Disease		 Dr. Donald B. Kohn PhD University of California, Los Angeles Phase 1 Active, not recruiting 4
Cystinosis
Kidney Failure		 Dr. Stephanie Cherqui University of California, San Diego Phase 1/2 Active, not recruiting 6
COVID-19
Respiratory Disorders		 William van der Touw Celularity Inc. Phase 1/2 Closed 86
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
Blood Cancer
Solid Tumors		 Professor Irving L Weissman MD Stanford University Phase 1 Completed 88
Solid Tumors Dr. Dennis J Slamon University of California, Los Angeles Phase 1 Completed 46
Pulmonary Hypertension
Vascular Disease		 Dr. Michael I Lewis Cedars-Sinai Medical Center Phase 1/2 Completed 26
Arthritis
Osteoarthritis		 Dr. Kristen A Johnson Calibr Phase 1 Completed 60
Immune Disease
Leukocyte Adhesion Deficiency		 Dr. Kinnari Patel Rocket Pharmaceuticals, Inc. Phase 1/2 Completed 9
Bone or Cartilage Disease
Hearing Loss
Intellectual Disability
Metabolic Disorders
Vision Loss		 Dr Robert Hayes Immusoft Corporation Phase 1 Launching N/A
Neurological Disorders
Other		 Prof. Olivia Kim-McManus University of California, San Diego Phase 1/2 Launching N/A
Immune Disease DR Jonathan Benjamin ImmPACT-Bio, Inc. Phase 1/2 Launching N/A
Lupus, Systemic Lupus Erythematosus (SLE) Bob Valamehr Fate Therapeutics, Inc. Phase 1 Launching N/A
Danon Disease
Pediatrics		 Dr. Jonathan Schwartz Rocket Pharmaceuticals, Inc. Phase 2 Launching N/A
COVID-19
Respiratory Disorders		 Prof. John A. Zaia City of Hope, Beckman Research Institute Phase 1 Launching N/A
Bladder or Urinary Tract Disorder James Yoo Wake Forest University Health Sciences Phase 1 Not yet recruiting 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
Pediatrics
Severe Combined Immunodeficiency, X-linked (X-SCID)		 Dr. Judith A Shizuru
Wendy Pang		 Stanford University
Jasper Therapeutics, Inc.		 Phase 1/2 Recruiting 40
HIV/AIDS
Immune Disease		 Steven G. Deeks University of California, San Francisco Phase 1/2 Recruiting 18
Developmental Disorders
Spina Bifida		 Dr. Diana L. Farmer University of California, Davis Phase 2 Recruiting 55
Muscle Injury Peter C Belafsky University of California, Davis Phase 1/2 Recruiting 62
Immune Disease
Severe Combined Immunodeficiency, X-linked (X-SCID)		 Stephen Gottschalk St. Jude Children’s Research Hospital Phase 1/2 Suspended 28