Project Grant R44HL182563
- Kinea Bio Inc. received a $306,859 Project Grant Award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), effective September 15, 2025, with completion targeted for August 31, 2026. The award funds development of a scalable, non-viral gene delivery therapeutic platform using cell-derived nanovesicles to treat myotonic dystrophy type 1 (DM1), the most common adult-onset...
- 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 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 Greenstone Biosciences, Inc. $625,968 on August 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to conduct small-molecule drug discovery for cardiac fibrosis associated with Duchenne muscular dystrophy using artificial intelligence, machine learning, and induced pluripotent stem cell-derived organoids. The R61 phase will perform unbiased proteomics on DMD iPSC-derived cardiomyocytes and cardiac fibroblasts to...
- The National Heart, Lung, and Blood Institute awarded the University of California, San Diego $795,900 on August 25, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop gene therapy for FLNC haploinsufficiency cardiomyopathy. FLNC haploinsufficiency, caused by nonsense, frameshift, or splice-site mutations, drives inherited dilated and arrhythmogenic cardiomyopathies characterized by early-onset systolic dysfunction, high malignant arrhythmia risk, and sudden...
- The National Heart, Lung, and Blood Institute awarded Novomedix LLC $355,963 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop and test novel small-molecule anti-fibrotic compounds for prevention and treatment of Duchenne muscular dystrophy cardiomyopathy. The project addresses cardiac complications in DMD patients, where improved respiratory support has extended survival into the second and third decade of life but exposed an unmet need for...
- 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...
- The National Institutes of Health National Heart Lung and Blood Institute awarded Sanford Burnham Prebys Medical Discovery Institute $1,603,648 on July 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop a single-construct gene therapy for enhanced cardiac repair. The funded research aims to overcome barriers to cardiac reprogramming by creating a gene therapy that simultaneously induces overexpression of cardiac transcription factors MEF2C, GATA4, and TBX5 (MGT)...
- The National Heart, Lung, and Blood Institute awarded Case Western Reserve University, Office of Research Administration $1,532,398 on July 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop and validate a gene therapy delivery platform for cardiomyopathies. The project, titled "Bait and Switch Gene Therapy for Cardiomyopathies," addresses the clinical translation barrier in adeno-associated virus (AAV) gene therapy for genetic heart diseases including...
- The National Heart, Lung, and Blood Institute awarded Emmune Inc. $299,664 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop a non-viral gene therapy vector for treating and preventing atherosclerotic cardiovascular disease. Emmune will leverage lipid nanoparticle (LNP) technology to achieve non-immunogenic gene delivery of a therapeutic protein capable of treating and preventing atherosclerosis. The company has discovered a novel approach to...
The National Heart, Lung, and Blood Institute awarded $599,669 to Kinea Bio Inc. on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop and validate KNA-123, an innovative gene therapy for Duchenne muscular dystrophy targeting cardiac muscle degeneration. The award is classified as a Project Grant. Kinea Bio will leverage the split intein-mediated protein ligation for gene therapy (SIMPLI-GT) platform to enable delivery of a larger, more functional midi-dystrophin transgene using dual AAV vectors, addressing size limitations of current gene therapies. The research comprises three specific aims: evaluating cardiac biodistribution, dystrophin expression, and functional rescue following systemic delivery of AAVmyo-midi-dystrophin in dystrophin-deficient rats; demonstrating rescue of dystrophic phenotypes in human iPSC-derived DMD cardiomyocytes; and developing and qualifying a functional potency assay using 3D engineered heart tissue from DMD patients. The work integrates molecular tools and preclinical models to advance therapeutic strategies for genetic disorders involving large genes. The recipient is Kinea Bio Inc., located in Seattle, Washington. Performance extends through August 31, 2027.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $599.7k | 8/28/26 |