Project Grant R01HL180680
- The National Heart, Lung, and Blood Institute awarded the Regents of the University of Michigan $167,400 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop nanoparticle-mediated delivery of CRISPR-Cas9 gene editing for sickle cell disease treatment. The project focuses on advancing non-viral platforms for therapeutic gene editing of hematopoietic stem and progenitor cells. Current gene therapy approaches using ex vivo viral transduction or...
- The National Heart, Lung, and Blood Institute awarded the Trustees of Indiana University $516,592 on July 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to conduct integrative genomic analysis and develop high-dimensional risk prediction models for congenital heart defects using random field methods. The research leverages existing biospecimens and data from the National Birth Defect Prevention Studies, the Pediatric Cardiac Genomics Consortium, and the Trans-Omics for...
- The federal Project Grant award, titled "ENHANCEMENT OF GENOME EDITING IN HEMATOPOIETIC STEM CELLS BY NUCLEIC ACIDS NANOPARTICLES DELIVERY OF CRISPR/CAS SYSTEM", was granted by the National Heart Lung and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837). The $486,291 award, effective February 1, 2025 through January 31, 2028, supports the development of a nucleic acid nanoparticle technology to efficiently deliver the CRISPR/Cas9 system into...
- 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 Institutes of Health National Heart, Lung, and Blood Institute awarded $735,071 to Indiana University Indianapolis on July 15, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop next-generation dystrophin gene therapy for Duchenne muscular dystrophy. The research addresses the limitations of currently approved adeno-associated virus (AAV) micro-dystrophin gene therapy by developing improved approaches to systemically deliver full-length, fully...
- 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 $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, a component of the Department of Health and Human Services National Institutes of Health, awarded $792,498 to the Trustees of Indiana University (doing business as Indiana University Indianapolis) on August 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate gene-environment interactions in congenital heart disease, specifically the role of maternal obesity in offspring cardiac development. The research...
- 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 Beth Israel Deaconess Medical Center, Inc. $852,908 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to design and optimize elastin-like polypeptide (ELP) nanoparticles for pulmonary gene therapy of cystic fibrosis. The funded work develops fourth-generation ELP nanoparticles engineered to self-assemble into pH-responsive micellar nanoparticles for cytosolic delivery of mRNA, pDNA, proteins, and RNPs to...
The National Heart, Lung, and Blood Institute awarded $792,498 to Trustees Of Indiana University (Indiana University Indianapolis) on August 17, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) for a Project Grant titled "Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thalassemia and Sickle Cell Disease." The award funds development of in vivo CRISPR delivery systems using biomimetic lipid nanoparticles (LNPs) to deliver gene-editing components to long-term hematopoietic stem cells (LT-HSCs). The research addresses the therapeutic potential of inducing fetal hemoglobin production as a substitute for adult hemoglobin through genetic modification of the BCL11A binding site on the G-globin gene, targeting treatment of beta-hemoglobinopathies. Current ex vivo approaches requiring CD34+ cell isolation and electroporation are time-consuming, lack specificity for LT-HSCs, and are limited to well-equipped facilities, restricting accessibility in resource-limited regions where most cases occur. The project develops alternative in vivo delivery approaches leveraging exosome interactions with LT-HSCs. Performance takes place in Indianapolis, Indiana. The period of performance runs through April 30, 2031. The assistance type is a Project Grant.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $792.5k | 8/19/26 |