Project Grant P01HL187309
- The National Heart, Lung, and Blood Institute awarded The Children's Hospital of Philadelphia $889,932 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to engineer and scale production of red blood cells derived from human induced pluripotent stem cells (iPSCs) for two high-impact applications: alloantibody detection and neonatal transfusion therapy. The project addresses urgent clinical needs in two vulnerable patient populations. Sickle cell disease...
- The National Institute of Diabetes and Digestive and Kidney Diseases awarded The Children's Hospital of Philadelphia $133,500 on May 5, 2026, under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) to investigate mechanisms of hemoglobin developmental switching as a therapeutic target for sickle cell disease and beta-thalassemia. The project, titled "Control of Hemoglobin Expression by the Chromatin Remodeler CHD7" (R03DK147724), will characterize...
- The National Heart, Lung, and Blood Institute awarded $1.834 million to St. Jude Children's Research Hospital Inc. on July 5, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to test senescence-targeting therapies for improving hematopoietic stem cell function and mobilization in sickle cell disease. The research addresses pathologic bone marrow stress in sickle cell disease patients, which damages and depletes hematopoietic stem and progenitor cells and selects for...
- The National Institutes of Health National Heart, Lung, and Blood Institute awarded The Children's Hospital of Philadelphia $1,741,543 on September 15, 2025, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate lung injury in pediatric hematopoietic cell transplant recipients. The research examines molecular mechanisms of pulmonary toxicity in pediatric HCT recipients, who experience lung injury in up to 40% of cases with mortality rates of 40% when requiring...
- The National Heart, Lung, and Blood Institute awarded The Trustees of The University of Pennsylvania $632,561 on August 20, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to identify and optimize signaling pathways that promote the maturation of pre-hematopoietic stem cells into long-term, multi-lineage adult-repopulating hematopoietic stem cells. The research focuses on transforming growth factor beta and bone morphogenetic protein signaling pathways, with emphasis on...
- The National Institutes of Health National Heart Lung and Blood Institute awarded Trustees of Boston University $100,228 on December 12, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate enhancement of engraftment potential in human hematopoietic stem cells. The research addresses the challenge of preserving hematopoietic stem cell functionality during the ex vivo culture steps required for gene editing therapies. The project employs multi-modal profiling of...
- The National Heart, Lung, and Blood Institute awarded The Children's Hospital Corporation, doing business as Boston Children's Hospital, $516,458 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) for Project Grant R01HL187595. The funded research investigates mechanisms of somatic rescue in Fanconi Anemia, a prototypical inherited bone marrow failure syndrome characterized by deficiency in DNA interstrand crosslink repair. The project examines how some...
- The National Heart, Lung, and Blood Institute awarded Duke University $238,516 on August 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to conduct admixture mapping of genetic modifiers in sickle cell disease. The research leverages the National Heart, Lung, and Blood Institute's TOPMed program, which includes multiple cross-sectional adult sickle cell disease cohorts totaling more than 4,000 participants with whole genome sequencing data and extensive clinical...
- The National Heart, Lung, and Blood Institute awarded The Children's Hospital of Philadelphia $850,577 on August 15, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate how translesion synthesis DNA polymerases mitigate replication stress in hematopoietic stem cells. The research examines the role of LNK deficiency in reducing DNA damage associated with replication stress in hematopoietic stem and progenitor cells, particularly in the Fanconi anemia...
- The National Heart, Lung, and Blood Institute awarded $1,137,435 to the University of Pennsylvania on March 20, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to advance therapeutic gene-editing approaches for cardiovascular and inherited disease. The recipient is developing and translating gene-editing therapies across three therapeutic domains. The first targets liver-centered, multiplex editing to prevent atherosclerotic cardiovascular disease through tunable...
The National Heart, Lung, and Blood Institute awarded The Children's Hospital of Philadelphia $2,712,905 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop safe, effective, and scalable curative therapies for hemoglobinopathies including sickle cell disease, alpha-thalassemia, beta-thalassemia, and Fanconi anemia by targeting hematopoietic stem cells in vivo using antibody-directed, lipid nanoparticle delivery systems. The research strategy employs lipid nanoparticles designed to deliver mRNA-encoded pro-apoptotic payloads or gene editors directly to diseased stem cells, coupled with advanced disease modeling platforms including humanized mouse models and preclinical large animal systems such as sheep and non-human primates to evaluate safety and efficacy. The ultimate goal is selective depletion of diseased stem cell populations or durable genetic correction of faulty hematopoietic stem cells to achieve transformative therapies that address limitations of current hematopoietic stem cell transplantation, which carries toxicities that limit patient eligibility and threaten fertility and long-term health. The program consists of three interconnected research projects supported by two specialized scientific cores and a central administrative core. Work is performed in Philadelphia, Pennsylvania, with a period of performance from September 1, 2026, through May 31, 2031. The award is a Project Grant under the assistance type classification.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $2.7m | 8/31/26 |