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.
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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 | Detail |
|---|---|---|---|---|---|---|
| Age-related macular degeneration | Mark Salman Humayun | University of Southern California | Phase 1 | Closed | 16 | |
| Age-related macular degeneration | Jane Stephanie Lebkowski | Regenerative Patch Technologies LLC | Phase 2 | Recruiting | N/A | |
| Age-related macular degeneration | Dr. Jeffrey H Stern | Luxa Biotechnology | Phase 1/2 | Launching | N/A | |
| Alpha Thalassemia Major | Dr. Tippi C. MacKenzie | University of California, San Francisco | Phase 1 | Enrolling by invitation | 10 | |
| Amyotrophic Lateral Sclerosis | MD Ralph Kern | BrainStorm Cell Therapeutics | Phase 3 | Completed | 196 | |
| Amyotrophic Lateral Sclerosis | Professor Clive Niels Svendsen | Cedars-Sinai Medical Center | Phase 1/2 | Completed | 18 | |
| Amyotrophic Lateral Sclerosis | Professor Clive Niels Svendsen | Cedars-Sinai Medical Center | Phase 1 | Launching | N/A | |
| 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 | |
| Arthritis Osteoarthritis |
Dr. Kristen A Johnson | Calibr | Phase 1 | Completed | 60 | |
| B cell cancers Leukemia |
Thomas J Kipps | University of California, San Diego | Phase 1/2 | Active, not recruiting | 102 | |
| B cell cancers Leukemia |
Dr. David B. Miklos | Stanford University | Phase 1 | Active, not recruiting | 56 | |
| B cell cancers Leukemia Leukemia, Acute Myeloid (AML) |
Dr. Matthew H Porteus | Stanford University | Phase 1 | Recruiting | 22 | |
| B cell cancers | Dr. Paul W Finnegan | Angiocrine Bioscience, Inc. | Phase 3 | Closed | 130 | |
| Beta Thalassemia | Bettina Cockroft | Sangamo BioSciences, Inc. | Phase 1/2 | Completed | 5 | |
| Bladder or Urinary Tract Disorder | James Yoo | Wake Forest University Health Sciences | Phase 1 | Not yet recruiting | 10 | |
| Blood Cancer | Edward Kavalerchik | Angiocrine Bioscience, Inc. | Phase 1 | Completed | 10 | |
| Blood Cancer | Thomas J Kipps | University of California, San Diego | Phase 1 | Completed | 26 | |
| Blood Cancer Solid Tumors |
Professor Irving L Weissman MD | Stanford University | Phase 1 | Completed | 88 | |
| Blood Cancer | Edward Kavalerchik | Angiocrine Bioscience, Inc. | Phase 1/2 | Completed | 42 | |
| 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 | |
| Bone or Cartilage Disease | Dr. Gayatri Rao | Rocket Pharmaceuticals, Inc. | Phase 1 | Closed | 1 | |
| Bone or Cartilage Disease Hearing Loss Intellectual Disability Metabolic Disorders Vision Loss |
Dr Robert Hayes | Immusoft Corporation | Phase 1 | Launching | N/A | |
| Brain Cancer | Dr. Christine E. Brown | City of Hope, Beckman Research Institute | Phase 1 | Active, not recruiting | 82 | |
| Brain Cancer | Anthony J. Gringeri | ImmunoCellular Therapeutics | Phase 3 | Suspended | 234 | |
| Brain Cancer Breast Cancer |
Dr Jana Portnow | City of Hope, Beckman Research Institute | Phase 1 | Recruiting | 39 | |
| Brain Cancer | Dr. Leo D Wang | City of Hope, Beckman Research Institute | Phase 1 | Recruiting | 18 | |
| Brain Cancer | Crystal Mackall | Stanford University | Phase 1 | Recruiting | 54 | |
| Brain Cancer | Dr Jana Portnow | City of Hope, Beckman Research Institute | Phase 1 | Launching | N/A | |
| Brain Cancer | Dr. Reena Parada Thomas | Stanford University | Phase 1 | Launching | N/A | |
| Brain Cancer | Dr. Hideho Okada | University of California, San Francisco | Phase 1 | Launching | N/A | |
| Brain Cancer | Noriyuki Kasahara | University of California, San Francisco | Phase 1 | Launching | N/A | |
| Colon Cancer | Mark Chao | Forty Seven Inc. | Phase 1/2 | Completed | 78 | |
| Colon Cancer | Ivan King | Tachyon Therapeutics, Inc | Phase 1 | Launching | N/A | |
| Corneal Damage | Dr. Sophie X Deng | University of California, Los Angeles | Phase 1 | Recruiting | 20 | |
| Corneal Damage | R. Kim Brazzell | Combangio, 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 | |
| COVID-19 | Dr. Michael Matthay MD | University of California, San Francisco | Phase 2 | Completed | 120 | |
| COVID-19 Respiratory Disorders |
William van der Touw | Celularity Inc. | Phase 1/2 | Closed | 86 | |
| Cystinosis Kidney Failure |
Dr. Stephanie Cherqui | University of California, San Diego | Phase 1/2 | Active, not recruiting | 6 | |
| Danon Disease Pediatrics |
Dr. Jonathan Schwartz | Rocket Pharmaceuticals, Inc. | Phase 2 | Launching | N/A | |
| Developmental Disorders Spina Bifida |
Dr. Diana L. Farmer | University of California, Davis | Phase 2 | Recruiting | 55 | |
| Epilepsy | Dr. Cory R Nicholas | Neurona Therapeutics | Phase 1 | Recruiting | 40 | |
| Epilepsy | Dr. Cory R Nicholas | Neurona Therapeutics | 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 |
University of California, Los Angeles Orchard Therapeutics |
Phase 2 | Completed | 10 | |
| Heart disease associated with Duchenne muscular dystrophy | Dr. Linda Marban | Capricor, Inc | Phase 2 | Completed | 25 | |
| Heart failure | Dr. Rachel Ruckdeschel Smith | Capricor, Inc | Phase 2 | Closed | 135 | |
| Heart failure | Dr. Joseph C. Wu | Stanford University | Phase 1 | Recruiting | 18 | |
| Heart failure | Dr. Matthew Pollman | Tenaya Therapeutics, Inc. | Phase 1 | Launching | N/A | |
| HIV-related Lymphoma HIV/AIDS |
Dr Mehrdad Abedi | University of California, Davis | Phase 1/2 | Active, not recruiting | 11 | |
| HIV/AIDS | Geoff Symonds | Calimmune, Inc. | Phase 1/2 | Completed | 13 | |
| HIV/AIDS | Prof. John A. Zaia | City of Hope, Beckman Research Institute | Phase 1 | Active, not recruiting | 12 | |
| HIV/AIDS Immune Disease |
Steven G. Deeks | University of California, San Francisco | Phase 1/2 | Recruiting | 18 | |
| HIV/AIDS | Mr. Daniel Dornbusch | Excision BioTherapeutics | Phase 1 | Active, not recruiting | 6 | |
| HIV/AIDS | Dr. Angelo Manuel Almeida Cardoso Ph.D., MD | City of Hope, Beckman Research Institute | Phase 1 | Launching | N/A | |
| Huntington's Disease | Dr. Vicki Wheelock | University of California, Davis | Phase 1/2 | Completed | 29 | |
| 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 |
Kinnari Patel | Rocket Pharmaceuticals, Inc. | Phase 1/2 | Completed | 9 | |
| Immune Disease | Dr. Jonathan Benjamin | ImmPACT-Bio, Inc. | Phase 1/2 | Launching | N/A | |
| Immune Disease | Maria Grazia Roncarolo | Tr1X Inc. | Phase 1 | Launching | N/A | |
| IPEX Syndrome | Dr. Rosa Bacchetta | Stanford University | Phase 1 | Recruiting | 30 | |
| Kidney Disease | Dr. Jeffrey L Veale | University of California, Los Angeles | Phase 1/2 | Launching | N/A | |
| Kidney Failure | Jeffrey Lawson | Humacyte, Inc. | Phase 3 | Active, not recruiting | 240 | |
| Kidney Failure | Jeffrey Lawson | Humacyte, Inc. | Phase 3 | Completed | 355 | |
| Kidney Failure | Robert Lowsky | Stanford University | Phase 1 | Active, not recruiting | 15 | |
| Kidney Failure | Daniel Brennan | Medeor Acquisition Corp. | Phase 3 | Completed | 30 | |
| Kidney Failure | Dr. Everett H. Meyer | Stanford University | Phase 1 | Recruiting | 22 | |
| Leukemia, Acute Myeloid (AML) | Dr. Colleen Delaney | Nohla Therapeutics Inc. | Phase 2 | Closed | 146 | |
| Leukemia, Acute Myeloid (AML) | Mark Chao | Forty Seven Inc. | Phase 1 | Closed | 258 | |
| Leukemia, Acute Myeloid (AML) | Joseph Paul Woodard | Immune-Onc Therapeutics | Phase 1 | Recruiting | 106 | |
| Leukemia, Acute Myeloid (AML) | Elizabeth Budde | City of Hope, Beckman Research Institute | Phase 1 | Launching | N/A | |
| Leukemia, Acute Myeloid (AML) | Rochelle Emery | Senti Biosciences, Inc. | Phase 1 | Launching | N/A | |
| Lung Cancer | Steven M. Dubinett | University of California, Los Angeles | Phase 1 | Active, not recruiting | 24 | |
| Lupus, Systemic Lupus Erythematosus (SLE) | Dr. Vaneet Sandhu | Fate Therapeutics, Inc. | Phase 1 | Launching | N/A | |
| Melanoma | Robert Dillman | Caladrius Biosciences | Phase 3 | Closed | 4 | |
| Melanoma Skin cancer |
Dr. Antoni Ribas MD/PhD | University of California, Los Angeles | Phase 1 | Closed | 5 | |
| Melanoma | Anusha Kalbasi | Stanford University | Phase 1 | Launching | N/A | |
| Multiple Myeloma | Rajesh Belani MD | Poseida Therapeutics, Inc. | Phase 1 | Closed | 105 | |
| Muscle Injury | Peter C Belafsky | University of California, Davis | Phase 1/2 | Recruiting | 62 | |
| Neurological Disorders | Amit Etkin | Alto Neuroscience | Phase 2 | Launching | N/A | |
| Neurological Disorders Other |
Prof. Olivia Kim-McManus | University of California, San Diego | Phase 1/2 | Launching | N/A | |
| Osteoarthritis | Dr. Thomas W Chalberg Jr. | Genascence Corporation | Phase 1 | Launching | N/A | |
| Osteonecrosis | Nancy E. Lane | University of California, Davis | Phase 1/2 | Completed | 58 | |
| Parkinson's Disease | Krystof S. Bankiewicz | Brain Neurotherapy Bio, Inc. | Phase 1 | Active, not recruiting | 11 | |
| Parkinson's Disease | Dr. Edward Wirth III | Aspen Neuroscience | Phase 1 | Launching | N/A | |
| Pediatrics Sickle Cell Disease |
Dr. Donald B. Kohn | University of California, Los Angeles | Phase 1 | Active, not recruiting | 4 | |
| Pulmonary Hypertension Vascular Disease |
Dr. Michael I Lewis | Cedars-Sinai Medical Center | Phase 1/2 | Completed | 26 | |
| Retinitis Pigmentosa | Dr. Henry John Klassen | University of California, Irvine | Phase 1/2 | Completed | 28 | |
| Retinitis Pigmentosa | Henry John Klassen | jCyte, Inc. | Phase 2 | Completed | 84 | |
| Retinitis Pigmentosa | Professor Clive Niels Svendsen | Cedars-Sinai Medical Center | Phase 1 | Recruiting | 16 | |
| Retinitis Pigmentosa | Henry John Klassen | jCyte, Inc. | Phase 2 | Completed | 84 | |
| Retinitis Pigmentosa | Paul Bresge | Ray Therapeutics, INC | Phase 1 | Recruiting | N/A | |
| Sarcoma | Theodore Scott Nowicki | University of California, Los Angeles | Phase 1 | Closed | 5 | |
| Severe Combined Immunodeficiency, Artemis deficient (ART-SCID) | Dr Morton Cowan | University of California, San Francisco | Phase 2 | 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 | |
| Solid Tumors | Dr. Dennis J Slamon | University of California, Los Angeles | Phase 1 | Completed | 46 | |
| Solid Tumors | Dr. Pamela Reilly Contag PhD | BioEclipse Therapeutics INC. | Phase 1 | Recruiting | 60 | |
| Solid Tumors | Pei Wang | Eureka Therapeutics, Inc. | Phase 1/2 | Launching | N/A | |
| Solid Tumors | Dr. John Le Gall | Allogene Therapeutics | Phase 1 | Launching | N/A | |
| Spinal Cord Injury | Dr. Jane Stephanie Lebkowski Dr. | Geron Corporation | Phase 1 | Completed | 5 | |
| Spinal Cord Injury | Francois Binette | Lineage Cell Therapeutics, Inc. | Phase 1/2 | Completed | 25 | |
| Stroke | Dr. Bijan Nejadnik | SanBio, Inc. | Phase 2 | Completed | 163 | |
| Stroke | Dr. Gary Steinberg | Stanford University | Phase 1 | Recruiting | 30 | |
| Type 1 diabetes | Howard Foyt | ViaCyte, Inc. | Phase 1/2 | Closed | 19 | |
| Type 1 diabetes | Manasi Jaiman | ViaCyte, Inc. | Phase 1/2 | Completed | 49 | |
| Type 1 diabetes | Dr. William Sietsema | Caladrius Biosciences | Phase 2 | Completed | 113 | |
| Type 1 diabetes | Peter Stock | University of California, San Francisco | Phase 1/2 | Recruiting | 8 |