Stanford University |
Matthew Porteus |
Clinical Trial Stage Projects |
Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients |
$10,642,420 |
University of California, Santa Cruz |
Camilla Forsberg |
Foundation - Discovery Stage Research Projects |
Determining how age-specific heterogeneity of human hematopoietic stem cells and megakaryocyte progenitors contribute to thrombotic disease upon aging |
$1,536,000 |
Stanford University |
Everett Meyer |
Conference II |
Cellular Immune Tolerance Symposium |
$32,751 |
University of California, Berkeley |
Fyodor Urnov |
Quest - Discovery Stage Research Projects |
A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells |
$1,809,372 |
Stanford University |
Alice Bertaina |
Clinical Trial Stage Projects |
Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant |
$11,998,188 |
ImmunoVec |
Ryan Wong |
Late Stage Preclinical Projects |
Hematopoietic Stem Cell Gene Therapy for XCGD |
$3,999,959 |
Scripps Research Institute |
Michael Bollong |
Quest - Discovery Stage Research Projects |
Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS) |
$1,244,160 |
Stanford University |
Judith Shizuru |
Quest - Discovery Stage Research Projects |
Targeted Immunotherapy-Based Blood Stem Cell Transplantation |
$1,341,910 |
University of California, Los Angeles |
Donald Kohn |
Quest - Discovery Stage Research Projects |
Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia |
$1,177,739 |
University of California, Los Angeles |
Caroline Kuo |
Quest - Discovery Stage Research Projects |
Defining the Optimal Gene Therapy Approach of Human Hematopoietic Stem Cells for the Treatment of Dedicator of Cytokinesis 8 (DOCK8) Deficiency |
$1,386,232 |
Stanford University |
Rosa Bacchetta |
Clinical Trial Stage Projects |
Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome |
$11,999,179 |
ImmunoVec |
Katelyn Masiuk |
Therapeutic Translational Research Projects |
Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome |
$3,551,332 |
Stanford University |
Kathleen Sakamoto |
Quest - Discovery Stage Research Projects |
Small Molecules to inhibit Nemo-like Kinase for Treatment of Diamond Blackfan Anemia |
$848,098 |
University of California, San Francisco |
Mark Walters |
Cure Sickle Cell Initiative Clinical Trial Stage Projects |
Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease |
$8,389,407 |
University of California, Los Angeles |
Donald Kohn |
Quest - Discovery Stage Research Projects |
Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia |
$219,230 |
Boston Children's Hospital |
David Williams |
Cure Sickle Cell Initiative Clinical Trial Stage Projects |
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease |
$8,333,581 |
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. |
$600,000 |
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 |
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, 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 |
$5,867,085 |
Jasper Therapeutics, Inc. |
Wendy Pang |
Clinical Trial Stage Projects |
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants |
$2,313,398 |
Stanford University |
Judith Shizuru |
Clinical Trial Stage Projects |
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants |
$1,113,487 |
University of California, San Francisco |
Steven Mack |
Diagnostic Translational Research Projects |
Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions |
$633,014 |
UCSF Benioff Children’s Hospital Oakland |
Henry Erlich |
Diagnostic Translational Research Projects |
Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions |
$1,074,177 |
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 |
$5,150,000 |
City of Hope, Beckman Research Institute |
De-Fu Zeng |
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 |
$4,352,180 |
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 |
$2,115,735 |
University of California, San Francisco |
Mark Walters |
Therapeutic Translational Research Projects |
Curing Sickle cell Disease with CRISPR-Cas9 genome editing |
$60,635 |
UCSF Benioff Children’s Hospital Oakland |
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 |
Leslie Meltzer |
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 |
$2,638,745 |
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 |
$5,827,000 |
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 |
$1,651,884 |
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 |
$5,426,135 |
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, San Francisco |
Ann Zovein |
New Faculty Physician Scientist |
Human endothelial reprogramming for hematopoietic stem cell therapy. |
$2,197,683 |
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 |
$18,990,683 |
University of California, Davis |
Alice Tarantal |
Conference |
10th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases |
$18,300 |
University of California, Los Angeles |
Gay Crooks |
Basic Biology III |
Forming the Hematopoietic Niche from Human Pluripotent Stem Cells |
$1,252,857 |
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 |