Quest – Discovery Stage 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 the Discovery Program is to support exploratory research leading to the discovery of novel stem cell technologies to improve patient care.
 

DISC 2: The Quest Awards

The Quest Awards Program promotes the discovery of promising new stem cell-based technologies that could be translated to enable broad use and ultimately, improve patient care. Projects funded through the Quest Awards should propose technology that is uniquely enabled by human stem/progenitor cells or directly reprogrammed cells, or uniquely enabling for the advancement of stem cell-based therapies. 

Please see the Partnering Opportunity for Discovery Stage Research Projects page for the Quest 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, select the tab labeled “Open Programs”. Under the section labeled “RFAs and Programs Open for Applications”, click on the applicable link to Start a DISC Grant Application.
  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:
September 28, 2023
Total Awards:
144
Award Value:
$207,885,189

Awards

Institution Investigator Grant Title Award Value
Palo Alto Veterans Institute for Research Irene Lorenzo Llorente Inhibitory interneurons derived from human induced pluripotent stem cells to treat stroke $2,140,122
Novoglia, Inc. Dr. Sunil Gandhi Ph.D Gene-corrected human microglia for the treatment of adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) $1,417,948
University of California, San Francisco Faranak Fattahi Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids $1,954,367
University of Southern California Dr. Justin K Ichida Development of a VAV2 antisense oligonucleotide (ASO) treatment for ALS $2,072,560
Cedars-Sinai Medical Center Dr. Ritchie Ho C9orf72 repeat expansion-tuned allelic suppression by CRISPRi as an ALS therapy $2,274,768
University of California, Davis Ricardo Anibal Maselli MD Treatment of Myasthenic Syndrome due to Choline Acetyltransferase Deficiency Using AAV9-mediated Gene Therapy $1,500,000
Adverum Biotechnologies Cameron Keast Baker Development of an Optogenetic Vision Restoration Gene Therapy Using an Engineered Form of Melanopsin $1,150,820
Stanford University Dr. Robert Negrin MD Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application $1,920,652
University of California, San Francisco Michael Wilson A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering $2,182,396
Stanford University Dr. Michael F Clarke Developing a breast cancer stem cell drug $2,053,727
Cedars-Sinai Medical Center Dr. Ke Liao RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis $2,264,509
University of Southern California Linda Sher Assessing the Functional, Immunologic and Microbiologic Characteristics of Human Livers Created in Chimeric Pigs $2,736,590
City of Hope, Beckman Research Institute Dr Jianhua Yu Reprogramming Somatic Cells into iPSCs Engineered with an Anti-PSCA CAR to Develop Allogeneic Off-the-Shelf Cell Therapy to Treat Pancreatic Cancer $2,263,500
Vertuis Bio, Inc. Lukas Bachmann Expanded Capacity AAV Retinal Gene Therapy Enabled by Efficient RNA-Joining Technology $0
Gladstone Institutes, J. David Lyandysha Viktorovna Zholudeva Excitatory spinal interneurons from human pluripotent stem cells to treat spinal cord injury $2,942,198
University of California, Los Angeles Prof. Song Li Vax-CT to promote formation of cancer-specific T memory stem cell for personalized cancer immunotherapy $2,267,714
University of California, Los Angeles Dr. Richard Joseph Pietras Reversal of dysregulated myelopoiesis in breast cancers and cancer stem cells to boost antitumor immunotherapy $2,327,680
Greenstone Biosciences, Inc. Paul D Pang Drug Discovery for Duchenne Muscular Dystrophy Using Patient-Derived Human iPSCs $675,000
University of California, Berkeley Mr. Fyodor Urnov A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells $1,809,372
University of California, Davis Aijun Wang PhD In Utero Treatment of Duchenne Muscular Dystrophy with Non-viral Gene Editing $2,035,544
Rubedo Life Sciences Inc. Marco Quarta Pharmacological regenerative treatment of idiopathic pulmonary fibrosis targeting the senescent niche of lung progenitor cells. $1,450,876
University of California, Los Angeles Dr. Gerald Lipshutz Gene Therapy for SLC6A8 Creatine Transporter Disorder $2,296,920
University of California, Los Angeles Frank Pajonk Development of novel small molecules against cancer stem cells in solid cancers $2,340,000
Scripps Health Dr. Darryl D. D’Lima Pluripotent Stem Cells for Tendon Tissue Engineering $2,734,163
Cedars-Sinai Medical Center Dmitriy Sheyn Microgel encapsulated iPSC-derived notochordal cells to treat intervertebral disc degeneration and low back pain $2,020,166
Lundquist Institute for Biomedical Innovation at Harbor – UCLA Medical Center Michelina Iacovino A Novel Therapy for Sanfilippo B $2,297,884
University of California, Berkeley Dr. Kevin E. Healy Novel Lipid Nanoparticles for Enhancing eNOS Synthesis for Cardioprotection Post Myocardial Infarction $2,060,248
BrainXell Therapeutics Matthew Goodus Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI) $2,025,000
University of California, San Diego Professor Alysson R Muotri A treatment for Rett syndrome using glial-restricted neural progenitor cells $1,402,240
Gladstone Institutes, J. David Dr. Deepak Srivastava Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation $1,565,784
The Scintillon Institute Brian Lawson An hematopoietic stem-cell-based approach to treat HIV employing CAR T cells and anti-HIV broadly neutralizing antibodies. $1,143,600
University of California, Irvine Dr. Michael Demetriou CAR T cells targeting abnormal N-glycans for the treatment of refractory/metastatic solid cancers $1,414,800
University of California, Los Angeles Dr. Lili Yang Combating Ovarian Cancer Using Stem Cell-Engineered Off-The-Shelf CAR-iNKT Cells $1,404,000
Minutia, Inc. Katy Digovich Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients $1,198,550
Nanotools Bioscience Dr. Alex Savtchenko PhD Enabling non-genetic activity-driven maturation of iPSC-derived neurons $675,000
University of California, Los Angeles Dr. Xian-Jie Yang Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease models $1,345,691
University of California, San Diego Dr. Joseph G. Gleeson Novel antisense therapy to treat genetic forms of neurodevelopmental disease. $1,180,654
Scripps Research Institute Stuart A Lipton M.D., Ph.D. Drug Development of Inhibitors of Inflammation Using Human iPSC-Derived Microglia (hiMG) $1,658,123
University of California, Los Angeles Dr Dinesh S Rao Novel methods to eliminate cancer stem cells $1,384,347
University of California, San Diego Dr. Michael Karin Targeting pancreatic cancer stem cells with DDR1 antibodies. $1,425,600
Cedars-Sinai Medical Center Dr. Robert Barrett A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease $776,340
Scripps Research Institute Dr Michael J Bollong Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS) $1,244,160
University of California, Davis Dr. Kyle D Fink PhD AAV-dCas9 Epigenetic Editing for CDKL5 Deficiency Disorder $1,429,378
University of California, Los Angeles Dr. Caroline Y. Kuo Defining the Optimal Gene Therapy Approach of Human Hematopoietic Stem Cells for the Treatment of Dedicator of Cytokinesis 8 (DOCK8) Deficiency $1,386,232
University of California, Los Angeles Dr. Donald B. Kohn Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia $1,177,739
Stanford University Dr. Judith A Shizuru Targeted Immunotherapy-Based Blood Stem Cell Transplantation $1,341,910
Tenaya Therapeutics, Inc. Timothy Hoey Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure $1,017,000
Intact Therapeutics, Inc. Jeffrey Linhardt Modulation of oral epithelium stem cells by RSpo1 for the prevention and treatment of oral mucositis $942,050
Cytonus Therapeutics, Inc. Dr. Edward Filardo Development of a novel stem-cell based carrier for intravenous delivery of oncolytic viruses $899,342
Salk Institute for Biological Studies Ronald Mark Evans Extending Immune-Evasive Human Islet-Like Organoids (HILOs) Survival and Function as a Cure for T1D $1,523,285
Stanford University Ansuman Satpathy Preclinical development of an exhaustion-resistant CAR-T stem cell for cancer immunotherapy $1,420,200
Stanford University Dr. Bertha Chen iPSC-derived smooth muscle cell progenitor conditioned medium for treatment of pelvic organ prolapse $1,420,200
University of California, San Francisco Brian Feeley Matrix Assisted Cell Transplantation of Promyogenic Fibroadipogenic Progenitor (FAP) Stem Cells $1,179,478
University of California, Los Angeles Prof. Song Li iPSC Extracellular Vesicles for Diabetes Therapy $1,354,928
Scripps Health Dr. Darryl D. D’Lima Meniscal Repair and Regeneration $1,361,775
The Stone Research Foundation for Sports Medicine and Arthritis Dr. Kevin Stone A Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste Grafting $1,180,309
University of California, San Diego Gene Wei-Ming Yeo RNA-directed therapy for Huntington’s disease $1,253,110
University of California, Irvine Dr. Mathew Mark Blurton-Jones Transplantation of genetically corrected iPSC-microglia for the treatment of Sanfilippo Syndrome (MPSIIIA) $1,083,379
University of California, San Francisco Dr. Julie Deane Saba Providing a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy $1,463,400
Scripps Research Institute Luke L Lairson Improving the efficacy and tolerability of clinically validated remyelination-inducing molecules using developable combinations of approved drugs $1,554,126
University of California, San Francisco Dr Julia Carnevale Generating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineering $1,463,368
Stanford University Dr. Sarah C Heilshorn Injectable, autologous iPSC-based therapy for spinal cord injury $789,000
Navega Therapeutics Ana Maria Moreno Optimization of a gene therapy for inherited erythromelalgia in iPSC-derived neurons $1,157,313
Cedars-Sinai Medical Center Dr Eduardo Marban New noncoding RNA chemical entity for heart failure with preserved ejection fraction. $1,397,412
Stanford University Dr. Stanley Qi A novel hybrid CRISPR tool for gene network perturbation and hiPSC engineering $704,661
University of California, San Diego Ziwei Huang Development of a new therapeutic for directing target specific stem cell migration and treatment $1,129,512
University of California, San Diego Dan S Kaufman Human iPSC-derived chimeric antigen receptor expressing macrophages for improved cancer treatment. $1,256,332
Stanford University Dr. Marius Wernig MD, PhD A universally applicable skin sheet for Dystrophic Epidermolysis Bullosa via next-generation gene editing, iPS cell technology and tissue engineering $1,229,040
University of California, Davis Dr. David J Segal PhD AAV9-Cas13 gene therapy for Angelman syndrome $1,364,903
Stanford University Dr. Phillip Chung-Ming Yang Hypoxia-specific Production of Exosomes from iPSC-derivatives for Myocardial Repair $1,418,023
Stanford University Kathleen M Sakamoto Small Molecules to inhibit Nemo-like Kinase for Treatment of Diamond Blackfan Anemia $848,098
University of California, Irvine Munjal M Acharya Stem cell-derived extracellular vesicles to reverse radiation-induced brain injury $1,064,724
University of California, San Francisco Dr Marisa Medina iPSCs as a screening tool to predict risk of nonalcoholic fatty liver disease $813,000
University of California, San Diego Dr. Tannishtha Reya Targeting Critical Regulators of Cancer Stem Cells $1,148,264
University of California, Davis Dr. Deborah K Lieu Building a hiPSC-based biopacemaker $1,260,827
City of Hope, Beckman Research Institute Professor Yanhong Shi Combating COVID-19 using human PSC-derived NK cells $249,998
University of California, San Diego Tariq M Rana Development of COVID-19 Antiviral Therapy Using Human iPSC-Derived Lung Organoids $250,000
University of California, Los Angeles Dr. Thomas Carmichael M.D., Ph.D. Human-induced pluripotent stem cell-derived glial enriched progenitors to treat white matter stroke and vascular dementia. $250,000
University of California, Irvine Robert F. Hunt Optimization of a human interneuron cell therapy for traumatic brain injury $250,000
University of Southern California Dr. Justin K Ichida Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS $222,300
University of California, Irvine Dr. Mathew Mark Blurton-Jones Transplantation of Pluripotent Stem Cell Derived Microglia for the Treatment of Adult-onset Leukoencephalopathy (HDLS/ALSP) $214,668
Vitabolus, Inc. Aline Betancourt The First Orally Delivered Cell Therapy for the Treatment of Inflammatory Bowel Disease $249,000
Lundquist Institute for Biomedical Innovation at Harbor – UCLA Medical Center Dr Eiji Yoshihara PhD Dual angiogenic and immunomodulating nanotechnology for subcutaneous stem cell derived islet transplantation for the treatment of diabetes $250,000
University of California, Los Angeles Dr. Donald B. Kohn Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia $219,230
University of California, San Diego Dan S Kaufman Human iPSC-derived chimeric antigen receptor-expressing macrophages for cancer treatment $222,200
Stanford University Alan G Cheng Modulation of the Wnt pathway to restore inner ear function $1,389,416
University of California, San Diego Prof James S Hagood Mesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosis $0
University of Southern California Dr. Neil Segil A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs $741,574
Salk Institute for Biological Studies Ronald Mark Evans Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes $1,637,209
Gladstone Institutes, J. David Li Gan Develop iPSC-derived microglia to treat progranulin-deficient Frontotemporal Dementia $1,547,157
University of California, Los Angeles Dr. Lili Yang Preclinical Development of An HSC-Engineered Off-The-Shelf iNKT Cell Therapy for Cancer $1,404,000
University of California, San Diego Dr. Eric D. Adler MD Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease $1,393,200
Stanford University Dr. Katja Weinacht Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome $1,251,720
City of Hope, Beckman Research Institute Dr Saul J Priceman Chimeric Antigen Receptor-Engineered Stem/Memory T Cells for the Treatment of Recurrent Ovarian Cancer $1,351,174
Stanford University Dr. Philip Arden Beachy PhD Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer $1,265,436
Scripps Research Institute Stuart A Lipton M.D., Ph.D. Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs $1,827,576
University of California, San Francisco Dr. Hideho Okada Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas $900,000
Children’s Hospital of Los Angeles Dr. Mark R Frey Universal Pluripotent Liver Failure Therapy (UPLiFT) $1,297,512
University of California, San Diego Dr. Jonathan H Lin Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases $1,153,937
University of California, Irvine Dr. Aileen J Anderson Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy for chronic cervical spinal cord injury. $1,575,613
University of California, San Francisco Dr. Shuvo Roy Silicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1 Diabetes treatment $1,113,000
University of California, Los Angeles Professor Scott G Kitchen Engineering Lifelong Cellular Immunity to HIV $1,701,178
University of California, San Diego Dr. Tannishtha Reya Targeting Cancer Stem Cells in Hematologic Malignancies $1,960,560
University of California, Los Angeles Dr. Thomas Carmichael M.D., Ph.D. iPS Glial Therapy for White Matter Stroke and Vascular Dementia $2,086,130
University of California, Los Angeles Dr. April Pyle Ph.D. Identification and Generation of Long Term Repopulating Human Muscle Stem Cells from Human Pluripotent Stem Cells $2,148,519
Stanford University Dr. Kyle M Loh Towards hepatocyte cell replacement therapy: developing a renewable source of human hepatocytes from pluripotent stem cells $1,968,456
University of California, San Diego Mark Tuszynski Neural Stem Cell Relays for Severe Spinal Cord Injury $1,638,900
Stanford University Dr. Helen M Blau Stimulating endogenous muscle stem cells to counter muscle atrophy $2,198,687
ViaCyte, Inc. Dr. Alan D. Agulnick Preclinical development of an immune evasive islet cell replacement therapy for type 1 diabetes $1,470,987
University of California, San Diego Dr Yang Xu Development of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCs $1,924,791
Neurona Therapeutics Dr. Cory R Nicholas Development of a Cellular Therapeutic for Treatment of Epilepsy $1,616,536
City of Hope, Beckman Research Institute Dr. Saswati Chatterjee Genome Editing of Sinusoidal Endothelial Stem Cells for Permanent Correction of Hemophilia A $2,182,193
University of California, Irvine Dr. Brian J. Cummings Identification and characterization of the optimal human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) 2.0. $1,671,213
University of California, Los Angeles Dr. Vaithilingaraja Arumugaswami PhD Immunization strategies to prevent Zika viral congenital eye and brain disease $2,128,867
Rumi Scientific CA Ali H. Brivanlou Discovery of therapeutics for Huntington’s Disease $1,399,800
University of California, Los Angeles UCLA Gay M Crooks Platform Technology for Pluripotent Stem Cell-Derived T cell Immunotherapy $965,636
X-Therma Inc. Dr. Xiaoxi Wei Non-Toxic, Highly-Effective Bioinspired Cryoprotectants for On-Demand Stem Cell Therapies $887,883
University of California, Los Angeles Dr. Caroline Y. Kuo Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome $1,512,333
University of California, Davis Dr. Deborah K Lieu Microenvironment for hiPSC-derived pacemaking cardiomyocytes $2,042,438
Stanford University Dr. Mark Mercola Multipotent Cardiovascular Progenitor Regeneration of the Myocardium after MI $1,809,234
University of California, San Diego Professor Yuan Chen A Novel Approach to Eradicate Cancer Stem Cells $279,977
City of Hope, Beckman Research Institute Professor Yuan Chen A Novel Approach to Eradicate Cancer Stem Cells $1,539,935
University of California, Berkeley Dr. Kevin E. Healy Human Cardiac Chip for Assessment of Proarrhythmic Risk $899,581
University of California, San Francisco Dr. Holger F Willenbring Preclinical development of AAV vector-mediated in vivo hepatic reprogramming of myofibroblasts as a therapy for liver fibrosis $1,638,389
Scaled Biolabs Inc. Dr Justin Cooper-White A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs $657,528
City of Hope, Beckman Research Institute Markus Müschen Lgr5-mediated self-renewal in B cell selection and leukemia-initiation $2,183,223
University of California, San Diego Professor Alysson R Muotri A treatment for Zika virus infection and neuroprotection efficacy $1,924,524
Stanford University Dr. Matthew H Porteus Genome Editing to Correct Cystic Fibrosis Mutations in Airway Stem Cells $1,968,456
University of California, San Francisco Dr. Julie B Sneddon Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells $2,006,076
University of California, Los Angeles Dr. John P Chute Protein tyrosine phosphatase – sigma inhibitors for hematopoietic regeneration $2,115,735
University of California, San Diego Dan S Kaufman Targeted off-the-shelf immunotherapy to treat refractory cancers $1,936,936
Parkinson’s Institute Dr. Birgitt Schuele CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease $1,288,415
Gladstone Institutes, J. David Dr. Deepak Srivastava Direct Cardiac Reprogramming for Regenerative Medicine $2,392,157
University of California, Irvine Leslie M Thompson hNSC-mediated delivery of ApiCCT1 as a candidate therapeutic for Huntington’s disease $1,650,263
Stanford University Dr. Roel Nusse Preclinical development of human hepatocyte progenitor cells for cell therapy $1,651,211
University of California, San Francisco Dr. Tejal Ashwin Desai Thin Film Encapsulation Devices for Human Stem Cell derived Insulin Producing Cells $1,092,063
Stanford University Dr. Rosa Bacchetta GENE EDITING FOR FOXP3 IN HUMAN HSC $984,228
California Institute of Technology Dr. David Baltimore Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells $1,569,193
Gladstone Institutes, J. David Dr. Deepak Srivastava Use of Human iPSC-derived Endothelial Cells for Calcific Aortic Valve Disease Therapeutics $2,400,048
Scripps Research Institute Dr. Jeanne F Loring Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons $2,299,786
University of California, Davis Dr. David J Segal PhD MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome $1,055,319
University of California, Berkeley David V. Schaffer Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury $1,627,099
Stanford University Professor Hiromitsu Nakauchi Novel Rejuvenated T Cell Immunotherapy for Lung Cancer $1,968,456
University of California, Los Angeles Dr. April Pyle Ph.D. CRISPR/Cas9 nanoparticle enabled therapy for Duchenne Muscular Dystrophy in muscle stem cells $2,150,400
Total:
$207,885,188.71