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. 

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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.

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Awards

Institution	Investigator	Grant Title	Award Value
Stanford University	Sergiu P Pasca	A targeted antisense oligonucleotide therapeutic strategy for Timothy syndrome	$5,596,629
University of California, Irvine	Dr. Michael Demetriou	GlyTR2 CAR T cell translation: safe pan-cancer killing via velcro-like density-dependent targeting of cancer glycans	$4,581,144
University of California, San Francisco	Gregory Maness Allen	FM-IL2 CAR T cells for Pancreatic Cancer	$5,644,776
Entos Pharmaceuticals	John Lewis	Lep001 – gene therapy for the treatment of congenital lipodystrophy	$4,000,000
Fate Therapeutics, Inc.	Martin Hosking	Development of an off-the-shelf iPSC derived CAR T cell therapy for the treatment of solid tumors	$4,000,000
Navega Therapeutics	Ana Maria Moreno	Development of an AAV Epigenetic Gene Therapy for Gain-of-Function SCN9A Disorders and Chronic Pain	$3,982,633
University of California, Los Angeles	Roger Hollis	Evaluation of an ex vivo lentiviral gene therapy for the treatment of Angelman syndrome	$5,276,765
ReviR Therapeutics	Dr Paul R August	Genetic Therapy Targeting mHTT mRNA and Somatic Expansion to Treat Huntington’s Disease	$4,618,687
University of Southern California	David D. Tran	A Novel Gene Therapy to Target Glioblastoma via Custom-Engineered Adenovirus-Associated Viral Vectors	$5,927,454
NysnoBio GT Neurology	Jennifer Johnston	Parkin Gene Therapy for Parkinson’s Disease	$2,450,510
Maro Bio	Akanksha Chhabra	A novel non-genotoxic antibody conditioning therapeutic to expand access to and safety of HSCT and CD34+ gene therapies	$4,043,427
University of California, Los Angeles	Dr. Donald B. Kohn	Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia	$5,028,351
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	$0
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	$0
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	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. Caroline Y. Kuo	Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA)	$4,304,883
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	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
University of California, Irvine	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	$4,769,410
VxBiosciences inc.	Dr. Christina Chuong	Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients	$730,000
City of Hope, Beckman Research Institute	Lei Tian	Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1α	$1,266,592
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	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: $374,690,696.80