Therapeutic Translational Research Projects

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Objective

The mission of California Institute for Regenerative Medicine (CIRM) is to accelerate stem cell treatments to patients with unmet medical needs.
The objective of this initiative is to create a highly competitive opportunity for promising stem cell-based projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use for any one of the following product types:

  • TRAN 1: Stem cell-based therapeutic candidate
  • TRAN 2: Diagnostic based on stem cells, or critical for stem cell based therapy development or use
  • TRAN 3: Medical device (non-diagnostic) for a stem cell-based therapy or critical for stem cell based therapy development or use
  • TRAN 4: Novel tool that addresses a critical bottleneck to the discovery or development of stem cell-based therapy

Project outcomes, allowable activities, and what qualifies for each of the above product types are described in the corresponding Appendices: A, B, C, and D.  The overall application and review process and general requirements for all product types are described in main document.

Please see the Partnering Opportunity for Translational Research 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“. Under 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:
May 30, 2024
Total Awards:
81
Award Value:
$332,936,162

Awards

Institution Investigator Grant Title Award Value
Neurona Therapeutics Dr. Cory R Nicholas Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. $3,828,714
City of Hope, Beckman Research Institute Lei Tian Targeting pancreatic cancer with Allogeneic Off-the-Shelf PSCA-CAR NK cells $6,036,000
University of California, Los Angeles Dr. Lili Yang Combating Ovarian Cancer with Stem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy $5,601,600
Stanford University Natalia Gomez-Ospina Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells $4,696,296
Replay Holdings, Inc Anthony Conway Hypoimmunogenic iPSC-derived TCR-NK cells for oncology $4,107,571
AcuraStem Incorporated Mr. Samuel V. Alworth MS, MBA Development of AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies $4,012,325
Axent Biosciences Inc. Abhay Joshi A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation $3,999,241
University of California, Los Angeles Dr. Gerald Lipshutz DDS, MD, MS Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND $5,145,825
Ray Therapeutics, INC Paul Bresge Optogenetic Therapy for Treatment of Geographic Atrophy $3,998,930
University of California, San Francisco Sarah Monica Knox Neurogenic hydrogel stimulation of stem cells to regenerate radiation-damaged salivary glands $2,312,021
Siren Biotechnology, Inc. Dr. Nicole K Paulk Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma $3,997,919
Cedars-Sinai Medical Center Dr. Ahmed Gamal Ibrahim Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis $2,590,224
University of California, San Diego Dr Karen Christman Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction $4,353,438
University of California, Los Angeles Zulema Romero Garcia Adenine Base Editing for Autologous Hematopoietic Stem Cell Gene Therapy of CD3δ SCID $5,966,928
University of California, Los Angeles Dr. Donald B Kohn Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA) $4,309,973
University of California, San Diego Dr. Eric D. Adler MD Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease $5,180,389
University of California, Los Angeles Dr. Gerald Lipshutz DDS, MD, MS Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting $5,266,504
City of Hope, Beckman Research Institute Dr Michael A. Caligiuri Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody $6,036,001
University of California, Los Angeles Anthony J Aldave AAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations $4,338,166
ImmunoVec Luke Riggan Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome $3,999,899
BrainXell Therapeutics Maria Daniela Cirnaru Development of Autologous Cell Replacement Therapy for Parkinson’s Disease: Path to Personalized Treatment $3,841,110
University of California, Los Angeles Dr. Sophie X Deng Extracellular Vesicle-Based Therapy for Corneal Scars $5,779,276
University of California, Los Angeles Dr. Scott G Kitchen Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection $6,140,723
Rejuvenation Technologies, Inc. John Ramunas Telomerase mRNA for short telomere related pulmonary fibrosis $3,984,942
University of Southern California Dr. Rongfu Wang Novel T cell receptor-STEM T cell immunotherapy in lung cancer $5,689,540
Stanford University Walter Park Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy $6,054,165
VxBiosciences inc. Dr. Christina Chuong Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients $3,977,180
City of Hope, Beckman Research Institute Dr Jianhua Yu Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1α $6,036,002
Mahzi Therapeutics Dr. Allyson Berent Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting $3,800,760
University of California, Davis Dr. William J Murphy Overcoming resistance to standard CD19-targeted CAR T using a novel triple antigen targeted vector $3,869,061
University of California, San Diego Stephanie Cherqui CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia $4,846,579
University of Southern California Preet M Chaudhary Next generation affinity-tuned CAR for prostate cancer $5,805,144
University of California, Los Angeles Dr. Donald B. Kohn PhD Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension $4,751,297
Ray Therapeutics, INC Paul Bresge Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases $3,999,553
University of California, Irvine Dr. Aileen J Anderson A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury $4,950,024
University of California, San Francisco Arun P Wiita CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia $3,330,801
MyoGene Bio LLC Dr Courtney Young Development of MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy $3,400,000
Immusoft Corporation Dr Robert Hayes MPS II: Plasma cell delivery of iduronate sulfatase $3,994,676
Regencor, Inc. PILAR RUIZ-LOZANO IND-enabling Studies of Wearable Evolve-FSTL1 for Cardiac Regeneration after MI $3,923,191
University of California, San Francisco Dr. Hideho Okada Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma $4,556,536
ImmunoVec Luke Riggan Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome $3,551,332
University of California, Los Angeles Dr. Arjun Deb Targeting stromal progenitors to prevent the development of heart failure $4,714,824
Palo Alto Veterans Institute for Research Irene Lorenzo Llorente Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia $5,149,913
University of California, Los Angeles Dr. Irene L. Llorente Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia $707,754
Juvena Therapeutics Inc. Jeremy D O’Connell Clinical Translation of hESC-derived protein therapy that positively regulates the regenerative capacity of post-natal muscle for treating DM1  $3,906,376
University of California, Los Angeles Cristina Puig Saus CAR-Tnm cell therapy for melanoma targeting TYRP-1 $5,904,462
University of California, Los Angeles Dr. Lili Yang HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma $5,281,199
University of California, San Francisco Dr. Hideho Okada Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma $525,000
Sanford Burnham Prebys Medical Discovery Institute Dr. Evan Y. Snyder Human Neural Stem Cells (hNSCs) for neuroprotection in perinatal hypoxic-ischemic brain injury (HII)-Pre-IND-enabling Studies $4,963,684
Neurona Therapeutics Dr. Cory R Nicholas Development of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy $4,848,505
University of California, San Diego Mark H Tuszynski Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI) $6,235,897
University of California, Los Angeles Yvonne Y. Chen BCMA/CS1 Bispecific CAR-T Cell Therapy to Prevent Antigen Escape in Multiple Myeloma $3,176,805
University of California, Irvine Dr. Brian J. Cummings An optimized human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) $4,804,737
City of Hope, Beckman Research Institute Prof. Karen S Aboody M.D. Neural Stem cell-mediated oncolytic immunotherapy for ovarian cancer $2,873,262
University of California, Los Angeles Dr. Caroline Y. Kuo Ex Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome $4,896,628
University of Southern California Dr. Mark S Humayun PRPE-SF, polarized hESC-derived RPE Soluble Factors, as a Therapy for Early Stage Dry Age-related Macular Degeneration $3,697,935
Stanford University Dr. Theodore Leng NeuBright, a purified allogeneic cell therapy product for treatment of Dry Age-related Macular Degeneration $4,235,758
University of California, Los Angeles Steven Schwartz Clinical Translation of Autologous Regenerative Cell Therapy for Blindness $5,068,026
University of California, San Francisco Dr. Karin Lindgren Gaensler Developing engineered autologous leukemia vaccines to target residual leukemic stem cells $4,171,728
University of California, Irvine Dr. Magdalene J Seiler Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models $4,769,039
Stanford University Dr. Bertha Chen Autologous iPSC-derived smooth muscle cell therapy for treatment of urinary incontinence $5,977,155
MAX BioPharma, Inc. Dr. Farhad Parhami Phd, MBA Therapeutic development of Oxy200, an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis $1,400,000
University of California, San Diego Dan S Kaufman Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment $3,260,000
University of California, San Diego Catriona Jamieson A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia $2,511,767
Stanford University Dr. Anthony E. Oro DEBCT: Genetically Corrected, Induced Pluripotent Cell-Derived Epithelial Sheets for Definitive Treatment of Dystrophic Epidermolysis Bullosa $5,107,353
University of California, San Diego Ezra Cohen Development of ROR1 CAR-T cells to target cancer stem cells in advanced malignancies $5,795,584
University of California, San Diego Dr Karen Christman Injectable pro-regenerative scaffold for treating symptomatic peripheral artery disease $2,839,317
Gladstone Institutes, J. David Dr. Yadong Huang Human iPSC-derived GABAergic Progenitors for Alzheimer’s Disease Treatment $1,900,000
University of California, San Francisco Mark C. Walters M.D. Curing Sickle cell Disease with CRISPR-Cas9 genome editing $60,635
UCSF Benioff Children’s Hospital Oakland Dr. Mark C. Walters M.D. Curing Sickle cell Disease with CRISPR-Cas9 genome editing $4,394,276
University of Southern California Dr. Denis A Evseenko Dr. Pluripotent stem cell-derived chondrocytes for articular cartilage repair $2,503,104
Ankasa Regenerative Therapeutics, Inc. Ying Zhu An autologous somatic stem cell therapy for the treatment of osteonecrosis $2,088,780
University of California, Davis Dr. William J Murphy Placental Derived Natural Killer Cells to Target Solid Tumor Cancer Stem Cells (CSC) $0
University of California, Los Angeles Dr. Peter Cawood Butler Personalized Cell Therapy for Diabetes $1,494,896
Semma Therapeutics Dr. Felicia Pagliuca Personalized Cell Therapy for Diabetes $597,333
University of California, San Diego Dr. Lawrence S. B. Goldstein Human Embryonic Stem Cell-Derived Neural Stem Cell Transplants in Amyotrophic Lateral Sclerosis $1,790,000
University of California, Los Angeles Dr. Lili Yang Stem Cell-Based iNKT Cell Therapy for Cancer $6,956,775
City of Hope, Beckman Research Institute Dr. Yanhong Shi Ph.D. Process development for establishing an iPSC-based therapeutic candidate for Canavan disease $7,377,384
Stanford University Dr. Albert J Wong 2nd Generation Vaccine for the Treatment of Glioblastoma $2,929,889
University of California, Davis Dr. Joseph S Anderson Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease $883,174
Children’s Hospital of Los Angeles Dr. Mark R Frey ASCENT- Advanced Stem Cell Enteric Neuropathy Therapy $7,077,352
Total:
$332,936,161.80