Project Grant R01HL180569
- 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...
- The National Heart, Lung, and Blood Institute awarded The Trustees of the University of Pennsylvania $718,991 on July 6, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to determine the role of red blood cells in sepsis heterogeneity. The funded research investigates how red blood cells capture and deliver cell-free bacterial DNA to immune cells and distant organs during sepsis, a dysregulated host response to infection that remains a leading cause of death. The project...
- 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 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 Children's Hospital of Philadelphia $889,895 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate the role of biological sex and the innate immune response in neonatal bronchopulmonary dysplasia. The award funds research examining how sex chromosomes and X-chromosome inactivation regulate neonatal lung macrophage polarization in response to hyperoxic injury. The project will establish the...
- The National Heart, Lung, and Blood Institute awarded Boston Children's Hospital $169,160 on December 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to engineer hypoimmunogenic induced pluripotent stem cell (iPSC)-derived vascular cells and map immune-vascular interactions that drive immune tolerance and vascular integration. The project addresses clinical barriers to iPSC-based vascular therapies: immune rejection of allogeneic transplants and low differentiation...
- 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 The Trustees of Columbia University in the City of New York $246,750 on August 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate red blood cell transfusion characteristics in intracranial hemorrhage patients. The research examines how variations in red blood cell component and donor characteristics affect transfusion efficacy and patient outcomes in intracranial hemorrhage, a life-threatening condition...
- The National Heart, Lung, and Blood Institute awarded Emory University $1,288,327 on September 15, 2025, under the Cardiovascular Diseases Research program (CFDA 93.837) to conduct research on red blood cell survival in recipients with sickle cell disease. The research evaluates the impact of donor and recipient factors on transfused red blood cell (RBC) survival in sickle cell disease patients and examines mechanisms contributing to RBC clearance. The study proposes an experimental,...
- 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 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 patients undergoing frequent transfusions face severe alloimmunization—the development of antibodies against donor red blood cell antigens—which limits transfusion options and delays care. Alloantibody identification requires access to reagent red blood cells with rare or variant antigen profiles that remain in extremely limited supply. In parallel, clinical evidence suggests that transfusing fetal-type red blood cells, such as cord blood red blood cells, may reduce complications including retinopathy of prematurity in very premature infants, but supply constraints prevent their clinical adoption. The funded work combines gene editing, erythroid differentiation, and transfusion biology to produce engineered red blood cells with rare blood group antigen profiles and fetal-type functional characteristics. Aim 1 defines the molecular and metabolic programs governing blood group antigen expression, maturation, and function in the engineered cells, and assesses their utility in antibody detection and in vivo survival. Aim 2 optimizes scalable production by enhancing hematopoietic stem and progenitor cell generation and erythroid maturation. The award funds work performed at The Children's Hospital of Philadelphia Research Institute in Philadelphia, Pennsylvania. The period of performance runs from September 1, 2026, through May 31, 2030.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $889.9k | 8/14/26 |