Late Stage Preclinical Projects
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CLIN1: Late Stage Preclinical Projects
Objective
The mission of California Institute for Regenerative Medicine (CIRM) is to accelerate stem cell treatments to patients with unmet medical needs. With our Clinical Stage Programs, we intend to speed up support for clinical stage candidate stem cell treatments that demonstrate scientific excellence. Under this initiative, we will provide funding for eligible projects that are completing late stage preclinical development through any stage of clinical trial activity.
For our CLIN 1 Late Stage Preclinical Projects, the objective is to create a highly competitive partnering opportunity for promising stem cell-based projects to accelerate the completion of preclinical activities necessary to attain an active IND or IDE with the FDA and to initiate start-up activities of the proposed clinical trial. CIRM expects projects under this program to advance rapidly into the clinic and to be accomplished within two years unless adequately justified.
Under this program, CIRM will act not only as a funding agency, but will also devote significant internal resources and leverage its external team of world-class subject matter experts to actively advance the project. The result of a successful application will be the formation of a true partnership that both accelerates the program and gives it the greatest opportunity for success.
Please see the Partnering Opportunity for Late Stage Preclinical projects for the Program Announcement, application deadline and other details.
To Submit an Application:
- Go to the Grants Management Portal (https://grants.cirm.ca.gov) and log in with your existing CIRM Username and Password. If you do not have a Username, Click on the “New User” link and follow the instructions to create a CIRM Username and password.
- After logging in, Click on the Menu tab. Select the tab labeled “Open Programs“. Undert the section labeled “RFAs and Programs Open for Applications“, click on the “Start a Grant Application” link for your selected program.
- Complete each section of the Application by clicking on the appropriate link and following the posted instructions. Proposal templates can be located and submitted under the “Uploads” section.
- To submit your Application, click on the “Done with Application” button. The “Done with Application” button will be enabled when all of the mandatory sections have been completed. Please note that once this has been selected, you will no longer be able to make changes to your Application.
- To confirm submission of your Application, select the tab labeled “Your Applications” and check the table under the section labeled “Your Submitted Applications“. You will see your Application number and project title listed once the submission process has been completed.
ICOC Approval:
March 27, 2025
Award Value:
$231,656,546
Awards
| Institution |
Investigator |
Grant Title |
Award Value |
| Cure Rare Disease | Dr. Susan Perlman | Advancing a novel antisense oligonucleotide for the treatment of SCA3, a devastating neurodegenerative disease |
$5,692,538 |
| University of California, Davis | Dr. Joseph S Anderson | Expression of Ube3a by the hematopoietic system for the treatment of Angelman syndrome |
$4,487,656 |
| Rejuvenate Bio | Noah Davidsohn | Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy |
$570,000 |
| Amplo Biotechnology Inc | Patricio Sepulveda | Targeted DOK7 gene therapy for Congenital Myasthenic Syndromes |
$2,894,305 |
| University of California, San Diego | Mark H Tuszynski | Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI) |
$6,000,000 |
| University of California, Los Angeles | Dr. Arjun Deb | Development of a therapeutic monoclonal antibody for the treatment of myocardial infarction and heart failure |
$5,999,998 |
| Children’s Hospital of Los Angeles | Shahab Asgharzadeh | Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and AYA with Recurrent Solid Tumors |
$6,000,000 |
| Elpida Therapeutics | Mr Terry Pirovolakis | Pre-Clinical To Clinical Gene Therapy Development For CMT4J |
$3,930,964 |
| University of California, San Francisco | Thomas Martin | A 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1) |
$4,585,501 |
| Cedars-Sinai Medical Center | Dr. Eugenio Cingolani | Extracellular Vesicles for Ventricular Tachycardia |
$5,999,441 |
| AcuraStem Incorporated | Mr. Samuel V. Alworth MS, MBA | Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis |
$0 |
| City of Hope, Beckman Research Institute | Prof. Karen S Aboody M.D. | Neural stem cell delivered CRAd-S-pk7 oncolytic viro-immunotherapy for ovarian cancer |
$5,314,547 |
| Tr1X Inc. | Dr. Maria Grazia Roncarolo | TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies. |
$4,000,000 |
| Mahzi Therapeutics | Dr. Yael Weiss | Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE) |
$4,000,000 |
| University of California, Davis | Dr Mehrdad Abedi | Superior forward-oriented b-globin vector for treating Sickle Cell Disease |
$4,598,398 |
| University of Southern California | Dr. Mark S Humayun | IND-enabling program for PRPE-SF, the secretome from polarized stem cell-derived RPE cells, for the treatment of dry-age related macular degeneration |
$5,993,562 |
| Stanford University | Dr. Matthew H Porteus | Autologous Gene Corrected Sinus Basal Cells to Treat Serious Cystic Fibrosis Sinus Disease |
$6,000,000 |
| University of California, Irvine | Kyriacos A Athanasiou | Treatment of the TMJ disc complex |
$6,000,000 |
| Aspera Biomedicines, Inc. | Catriona Jamieson | Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor |
$3,200,000 |
| University of California, Los Angeles | Dr. Jerome A. Zack Ph.D. | Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness |
$6,000,000 |
| Kenai Therapeutics | Howard J Federoff | Allogeneic iPSC derived Dopaminergic Drug Product for Parkinson’s disease |
$4,000,000 |
| Rampart Bioscience | Jeffrey Bartlett | Ex vivo Engineering of Autologous Hematopoietic Stem Cells for the Treatment of Hypophosphatasia |
$3,999,980 |
| Nammi Therapeutics, Inc. | Dr. David R Stover | IND-Enabling activities for the masked immunocytokine, QXL138AM |
$3,999,113 |
| Calidi Biotherapeutics, Inc. | 8587949607 Boris Minev | Allogeneic mesenchymal stem cells loaded with oncolytic virus for cancer treatment |
$3,111,467 |
| Ossium Health, Inc. | Dr. Brian Johnstone | Development of OSSM-007, cryopreserved interferon-gamma primed allogeneic MSCs, for treatment of steroid refractory acute graft versus host disease |
$3,457,858 |
| University of California, Davis | Dr. Joseph S Anderson | Hematopoetic stem cell gene therapy for the treatment of Tay-Sachs disease |
$4,048,253 |
| Stanford University | Natalia Gomez-Ospina | Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome) |
$5,444,353 |
| University of California, San Francisco | Dr. Karin Lindgren Gaensler | Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells |
$6,000,000 |
| ImmunoVec | Dr. Ryan L. Wong | Hematopoietic Stem Cell Gene Therapy for XCGD |
$3,999,959 |
| University of California, Los Angeles | Dr. Antoni Ribas MD/PhD | Skin regeneration and wound healing with a topical BRAF inhibitor |
$5,005,126 |
| University of Southern California | Dr. Denis A Evseenko Dr. | Plurocart: a novel stem cell-based implant for articular cartilage restoration |
$5,999,782 |
| University of California, San Diego | Thomas J Kipps | Late Stage Pre-Clinical Development of a Cirmtuzumab Based CAR T-cell for the Treatment of ROR1+ Hematological Malignancies |
$1,630,360 |
| Stanford University | Dr. Rosa Bacchetta | IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome |
$5,002,496 |
| University of California, San Francisco | Mark C. Walters M.D. | Curing Sickle cell Disease with CRISPR-Cas9 genome editing |
$2,242,805 |
| University of California, Davis | Dr. Diana L. Farmer | Placental Mesenchymal Stem Cell Augmentation of Fetal Myelomeningocele Repair |
$5,615,207 |
| Ankasa Regenerative Therapeutics, Inc. | Ying Zhu | IND-enabling development of ART352-L, an endogenous stem cell reactivation therapy to enhance bone healing in the elderly |
$3,987,693 |
| City of Hope, Beckman Research Institute | Xiuli Wang | CMV-specific T cells expressing anti-HIV CAR and CMV vaccine boost as immunotherapy for HIV/AIDS |
$3,812,797 |
| University of California, San Francisco | Dr. Krystof Bankiewicz | MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s disease |
$5,757,078 |
| Poseida Therapeutics, Inc. | Devon J Shedlock | Late-Stage Preclinical Study of CAR-T Memory Stem Cells Targeting PSMA (P-PSMA-101) for the Treatment of Castrate-Resistant Metastatic Prostate Cancer |
$3,992,090 |
| City of Hope, Beckman Research Institute | Dr. Angelo Manuel Almeida Cardoso Ph.D., MD | Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients |
$3,684,259 |
| University of California, Irvine | Dr. Leslie M Thompson | An hESC-derived hNSC Therapeutic for Huntington’s Disease |
$5,635,393 |
| Fate Therapeutics, Inc. | Bob Valamehr | IND enabling development of FT516: A Natural Killer Cell Immunotherapy for Cancer Derived from a Human Inducible Pluripotent Stem Cell |
$4,000,000 |
| Stanford University | Dr. Matthew H Porteus | Genome Editing of Autologous Hematopoietic Stem Cells to Treat Sickle Cell Disease |
$4,849,363 |
| Cellerant Therapeutics, Inc. | Swapna Panuganti | Development of CLT030-ADC, a Leukemic Stem Cell Targeting Antibody-Drug-Conjugate, for Treatment of Acute Myeloid Leukemia |
$6,863,755 |
| Cellular Biomedicine Group, Inc. | Jack J. Wang | Allogenic human adipose-derived mesenchymal stem cells for the treatment of knee osteoarthritis |
$1,200,000 |
| Stanford University | Dr. Gary Steinberg | Intraparenchymal NR1 Stem Cell Therapy for Chronic Subcortical Ischemic Stroke |
$5,300,000 |
| University of California, San Diego | Dr. Stephanie Cherqui | Ex vivo transduced autologous human CD34+ hematopoietic stem cells for treatment of cystinosis |
$5,273,189 |
| University of California, Los Angeles | Dr. Sophie X Deng | Regeneration of a Normal Corneal Surface by Limbal Stem Cell Therapy |
$4,244,211 |
| ViaCyte, Inc. | Dr. Tim Kieffer | Stem cell-derived islet cell replacement therapy with immunosuppression for high-risk type 1 diabetes |
$3,544,721 |
| University of California, San Francisco | Dr Jennifer M Puck | Ex Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID) |
$4,268,865 |
| Angiocrine Bioscience, Inc. | Dr. Paul W Finnegan | Development of AB-110: genetically-modified endothelial cells plus expanded cord blood hematopoietic stem cells as a transplantation therapy |
$3,797,117 |
| Calibr | Peter G Schultz | Development of a Chondrogenic Drug Candidate Targeting Resident Mesenchymal Stem Cells for the Treatment of Osteoarthritis |
$1,667,832 |
| Cedars-Sinai Medical Center | Shaomei Wang | IND-enabling study of subretinal delivery of human neural progenitor cells for the treatment of retinitis pigmentosa |
$4,954,514 |
| | | Total:
$231,656,546.23 |
