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:

  1. 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.
  2. 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.
  3. 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.
  4. 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.
  5. 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:
June 27, 2024
Total Awards:
49
Award Value:
$216,569,182

Awards

Institution Investigator Grant Title Award Value
Rejuvenate Bio Deborah D. Ascheim Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy $4,000,000
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
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
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
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
Cedars-Sinai Medical Center Dr. Eugenio Cingolani Extracellular Vesicles for Ventricular Tachycardia $5,999,441
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
Mahzi Therapeutics Dr. Yael Weiss Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE) $4,000,000
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
University of California, Davis Dr Mehrdad Abedi Superior forward-oriented b-globin vector for treating Sickle Cell Disease $4,619,455
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 $2,160,000
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. Dr 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:
$216,569,182.23