All CIRM Grants

Institution Researcher name Grant Type Grant Title Award Value
University of California, Los Angeles Caroline Kuo Progression Award - Discovery Stage Research Projects Evaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8 $234,000
ExCellThera Inc. Pierre Caudrelier Cure Sickle Cell Initiative Clinical Trial Stage Projects A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease. $2,000,000
Stanford University Rosa Bacchetta Late Stage Preclinical Projects IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome $5,002,496
University of California, Los Angeles Caroline Kuo Therapeutic Translational Research Projects Ex Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome $4,896,628
University of California, San Francisco Mark Walters Late Stage Preclinical Projects Curing Sickle cell Disease with CRISPR-Cas9 genome editing $2,242,805
Rocket Pharmaceuticals, Inc. Kinnari Patel Clinical Trial Stage Projects LADICell $6,567,085
Stanford University Judith Shizuru Clinical Trial Stage Projects A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants $3,426,902
Children's Hospital of Oakland Research Institute Henry Erlich Diagnostic Translational Research Projects Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions $1,721,606
University of California, San Francisco Morton Cowan Clinical Trial Stage Projects Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells $12,000,000
Sangamo BioSciences, Inc. Bettina Cockroft Clinical Trial Stage Projects A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia $8,000,000
City of Hope, Beckman Research Institute Joseph Rosenthal Clinical Trial Stage Projects Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant $5,742,180
City of Hope, Beckman Research Institute Saswati Chatterjee Quest - Discovery Stage Research Projects Genome Editing of Sinusoidal Endothelial Stem Cells for Permanent Correction of Hemophilia A $2,182,193
Stanford University Hiromitsu Nakauchi Inception - Discovery Stage Research Projects Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion $210,906
Children's Hospital of Los Angeles Michael Pulsipher Clinical Trial Stage Projects Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES) $4,825,587
Stanford University Matthew Porteus Late Stage Preclinical Projects Genome Editing of Autologous Hematopoietic Stem Cells to Treat Sickle Cell Disease $4,849,363
University of California, Los Angeles Caroline Kuo Quest - Discovery Stage Research Projects Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome $1,512,333
University of California, San Francisco Tippi MacKenzie Clinical Trial Stage Projects In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major $10,906,978
University of California, San Diego Tannishtha Reya Inception - Discovery Stage Research Projects Reprogramming human stem cells for blood cell generation $210,060
American Association of Blood Banks Naynesh Kamani Conference II 15th International Cord Blood Sympsium $30,000
St. Jude Children's Research Hospital Stephen Gottschalk Clinical Trial Stage Projects Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning $11,924,780
Stanford University Rosa Bacchetta Quest - Discovery Stage Research Projects GENE EDITING FOR FOXP3 IN HUMAN HSC $984,228
University of California, Los Angeles John Chute Quest - Discovery Stage Research Projects Protein tyrosine phosphatase - sigma inhibitors for hematopoietic regeneration $2,116,708
University of California, San Francisco Mark Walters Therapeutic Translational Research Projects Curing Sickle cell Disease with CRISPR-Cas9 genome editing $69,159
Children's Hospital of Oakland Research Institute Mark Walters Therapeutic Translational Research Projects Curing Sickle cell Disease with CRISPR-Cas9 genome editing $4,394,276
University of California, Los Angeles Donald Kohn Clinical Trial Stage Projects Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects $10,156,925
Orchard Therapeutics plc Andrea Spezzi Clinical Trial Stage Projects Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects $8,465,745
University of California, San Diego Dionicio Siegel Inception - Discovery Stage Research Projects New Methods for the Chemical Expansion of Hematopoietic Stem and Progenitor Cells $232,200
Vitalant Research Institute Marcus Muench Inception - Discovery Stage Research Projects Modulating Liver Sinusoidal Endothelial Cell Permeability to Enhance Engraftment of Endothelial Cell Progenitors for the Treatment of Hemophilia A $180,000
International Society for Experimental Hematology David Traver Conference II International Society for Experimental Hematology, 45th Annual Scientific Meeting $25,000
American Association of Blood Banks Naynesh Kamani Conference II 14th International Cord Blood Symposium: Give Life Twice $15,000
University of California, San Francisco Jennifer Puck Late Stage Preclinical Projects 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
Stanford University Joseph Wu Conference II Drug Discovery & Stem Cell Models for Cardiovascular Disease Conference $7,500
University of California, Los Angeles Donald Kohn Clinical Trial Stage Projects A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease $7,083,364
Stanford University Matthew Porteus Preclinical Development Awards Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1 $874,877
University of California, Los Angeles Hanna Mikkola Tools and Technologies III A suite of engineered human pluripotent stem cell lines to facilitate the generation of hematopoietic stem cells $1,382,400
Stanford University Irving Weissman Tools and Technologies III Identification and isolation of transplantable human hematopoietic stem cells from pluripotent cell lines; two steps from primitive hematopoiesis to transplantable definitive cells, and non-toxic conditioning of hosts for hematopoeitic stem cell transp... $1,271,952
University of California, San Francisco Andrew Leavitt Tools and Technologies III Small molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use $1,416,600
University of California, Los Angeles John Chute Research Leadership Niche-Focused Research: Discovery & Development of Hematopoietic Regenerative Factors $4,645,297
University of California, Los Angeles Lili Yang Basic Biology V Differentiation of Human Hematopoietic Stem Cells into iNKT Cells $614,400
University of California, San Diego Dianne McKay Basic Biology V Role of intracytoplasmic pattern recognition receptors in HSC engraftment $615,639
University of California, Los Angeles Donald Kohn Disease Team Therapy Development III Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease $13,145,465
University of California, Los Angeles Donald Kohn Early Translational IV Beta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells $1,652,076
Salk Institute for Biological Studies Inder Verma Early Translational IV Development of a cell and gene based therapy for hemophilia $2,298,634
Sangamo BioSciences, Inc. Fyodor Urnov Strategic Partnership II A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells $2,760,540
Stanford University Hiromitsu Nakauchi Research Leadership Generation of functional cells and organs from iPSCs $5,427,358
University of California, San Francisco Ann Zovein New Faculty Physician Scientist Human endothelial reprogramming for hematopoietic stem cell therapy. $2,197,683
University of California, San Francisco Tippi MacKenzie New Faculty Physician Scientist In Utero Embryonic Stem Cell Transplantation to Treat Congenital Anomalies $2,661,742
University of California, Davis Alice Tarantal Conference 11th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases $16,850
Stanford University Judith Shizuru Disease Team Therapy Development - Research A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants $19,068,382
University of California, Davis Alice Tarantal Conference 10th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases $18,300
Stanford University Irving Weissman Tools and Technologies II Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs $1,463,814
University of California, Davis Alice Tarantal Conference 9th Annual Gene Therapy Symposium for Heart, Lung and Blood Diseases $12,000
Stanford University Steven Artandi Basic Biology II Self-renewal and senescence in iPS cells derived from patients with a stem cell disease $931,285
Scripps Research Institute Nicholas Gascoigne Transplantation Immunology Role of Innate Immunity in hematopoeitic stem cell-mediated allograft tolerance $1,705,554
University of California, San Francisco Tippi MacKenzie Transplantation Immunology Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation $1,230,869
University of California, Berkeley David Raulet Transplantation Immunology Inactivating NK cell reactivity to facilitate transplantation of stem cell derived tissue $952,896
Stanford University Judith Shizuru Transplantation Immunology Purified allogeneic hematopoietic stem cells as a platform for tolerance induction $1,233,275
University of California, Los Angeles Donald Kohn Disease Team Research I Stem Cell Gene Therapy for Sickle Cell Disease $8,833,695
University of California, Los Angeles Luisa Iruela-Arispe Basic Biology I Molecular Characterization and Functional Exploration of Hemogenic Endothelium $1,371,477
Salk Institute for Biological Studies Inder Verma Early Translational I Curing Hematological Diseases $5,979,252
University of California, San Francisco Brian Black Conference Weinstein Cardiovascular Development Conference $35,000
Salk Institute for Biological Studies Fred Gage New Cell Lines Development of Induced Pluripotent Stem Cells for Modeling Human Disease $1,737,720
Children's Hospital of Los Angeles Donald Kohn Disease Team Planning STEM CELL GENE THERAPY FOR SICKLE CELL DISEASE $12,131
University of California, Los Angeles Hanna Mikkola New Faculty I Mechanisms of Hematopoietic stem cell Specification and Self-Renewal $2,286,900
Stanford University Irving Weissman Comprehensive Grant Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells $2,471,386
University of California, San Diego Cornelis Murre SEED Grant Generation of long-term cultures of human hematopoietic multipotent progenitors from embryonic stem cells $473,952
University of California, Los Angeles Hanna Mikkola SEED Grant Improving microenvironments to promote hematopoietic stem cell development from human embryonic stem cells $550,241
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