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:
- 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“. Under 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:
April 27, 2023
Award Value:
$257,726,803
Awards
Institution |
Investigator |
Grant Title |
Award Value |
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 | Dr. Emmanuel E Baetge | 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 | Professor 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. | Leor Weinberger | 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 MD | 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 |
$4,841,428 |
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 |
$775,689 |
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 | 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 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: $257,726,802.64 |