Project Grant R01HL181220
- 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 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 $799,998 to The Trustees of the University of Pennsylvania on August 12, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop cardiotropic solid lipid nanoparticles (LNPs) as a drug and biologic delivery platform for cardiac therapeutics. The research focuses on engineering and understanding heart-targeting LNPs that accumulate in cardiac tissue at significantly higher concentrations than existing drug delivery...
- The National Heart, Lung, and Blood Institute awarded The Trustees of the University of Pennsylvania $632,081 on August 10, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop conformation-sensitive platelet-hitchhiking lipid nanoparticles for targeted delivery of mRNA and small interfering RNA to infarcted heart tissue following myocardial infarction. The project addresses therapeutic limitations in post-infarction recovery by engineering lipid nanoparticles to...
- The National Heart, Lung, and Blood Institute awarded Duke University $814,943 on July 15, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop non-viral gene therapy approaches for cardiac regeneration following myocardial injury. Duke University will develop cardiac-targeted lipid nanoparticles for in vivo transfection of cardiomyocytes and adapt a cell-state sensing platform (CELLREADR) to deliver therapeutic transgenes with cell-type precision to injured...
- The National Heart, Lung, and Blood Institute awarded The Trustees of the University of Pennsylvania $1,231,094 on September 15, 2025, under the Cardiovascular Diseases Research program (CFDA 93.837) to decipher the molecular mechanism of pediatric interstitial lung disease caused by surfactant protein C (SFTPC) gene mutations and advance targeted therapies. The University of Pennsylvania research team, led by Dr. Jeremy Katzen, will develop and leverage in vivo mouse models and human induced...
- The National Institute of Biomedical Imaging and Bioengineering awarded $645,265 to The Trustees of The University of Pennsylvania on June 1, 2026, under the Discovery and Applied Research for Technological Innovations to Improve Human Health program (CFDA 93.286) to develop DNA-loaded lipid nanoparticles (DNA-LNPs) for treating chronic diseases including atherosclerosis and emphysema. The research addresses toxicity barriers that have prevented DNA-LNP development for two decades. Prior...
- <p>The Defense Health Agency awarded the University of Pennsylvania $499,971 under the Military Medical Research and Development program (CFDA 12.420) on September 30, 2026, to support research repurposing a cancer-approved drug to ameliorate Duchenne muscular dystrophy pathology.</p> <p>The award funds a project grant with a period of performance extending from the award date through June 29, 2028, and performance takes place in Philadelphia, Pennsylvania.</p>
- The National Heart, Lung, and Blood Institute awarded The University of North Texas Health Science Center at Fort Worth $426,550 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop a bioengineering modular gene delivery system for lung-targeted transgene expression. The project addresses systemic gene therapy for monogenic lung diseases including cystic fibrosis, alpha-1-antitrypsin deficiency, and pulmonary arterial hypertension. The recipient...
- The National Heart, Lung, and Blood Institute awarded The Trustees of the University of Pennsylvania $2,031,250 on May 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop engineered CAR T-regulatory cells (CAR TREGs) targeting myocardial infarction-associated heart failure. The funded research aims to generate CAR TREGs that accumulate in fibrotic, damaged myocardial regions and suppress inflammation to promote healing and improve cardiac function. The work...
The National Heart Lung and Blood Institute awarded The Trustees of the University of Pennsylvania $623,909 on August 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to support rational design of gene therapy transgenes for Duchenne muscular dystrophy. The project addresses immunogenicity and incomplete functionality of miniaturized dystrophin transgenes in adeno-associated virus vector–based approaches to DMD gene therapy. The research employs a structure-guided approach to design improved synthetic nanotrophin transgenes. In Aim 1, investigators collaboratively design and develop novel transgene proteins and subfragments for in vitro production, purification, and biophysical analysis including X-ray crystallography to determine high-resolution three-dimensional structures. In Aim 2, the principal investigator's laboratory compares the efficacy of AAV vectors encoding a subset of these novel proteins to protect heart, diaphragm, and skeletal muscles from acute muscle injury, beginning with nanotrophin prototypes evaluated in small and large animal DMD models. The award funds work performed in Philadelphia, Pennsylvania, with performance extending through April 30, 2030. The project builds on prior evidence that central immunological tolerance for dystrophin-related protein (utrophin) enables high-level, durable expression in DMD models, contrasting with the robust cell-mediated immune response that destroys transduced myocytes expressing recombinant dystrophin. The assistance type is a Project Grant.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $623.9k | 8/3/26 |