Project Grant R01HL171282

Award Date 7/29/24
Completion Date 6/30/28
Dollars Obligated $611K
Federal Grant Program
93.837
Assistance Type
Project Grant
Place of Performance
Durham, NC 27705, USA
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This federal Project Grant award, provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), aims to develop improved gene therapy approaches for treating Duchenne muscular dystrophy (DMD). The $671,240 award, which runs from January 2025 to December 2029, will fund research at the University of Washington to refine technologies for expressing larger and more functional...
This $1,557,819 Project Grant award from the National Institute of Child Health and Human Development (CFDA 93.865 - Child Health and Human Development Extramural Research) to the Research Institute at Nationwide Children's Hospital supports a Muscular Dystrophy Specialized Research Center (MDSRC) focused on advancing gene therapy treatments for muscular dystrophy. The MDSRC has three key research projects: 1) Developing a bicistronic AAV vector to prevent muscle disease and build new muscle...
This Project Grant award for $762,974 from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), supports research to understand the impact of Pompe disease on alveolar (lung) cells and explore gene therapy as a potential treatment. The key objectives are to: 1) Determine if Pompe disease disrupts the ability of alveolar type 2 cells to repair lung damage, 2) Elucidate how Pompe disease affects autophagy and cellular...
The National Heart, Lung, and Blood Institute (NHLBI), a division of the National Institutes of Health (NIH), awarded a $1,358,179 Project Grant (CFDA 93.837 Cardiovascular Diseases Research) to Revidia Therapeutics, Inc. to conduct IND-enabling safety and toxicity testing of a subcutaneous formulation of the PTP1B inhibitor MSI-1436. This novel small molecule therapy aims to treat fatal cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), a rare and debilitating genetic...
This federal Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA 93.350, provides $110,000 to Myogene Bio LLC to conduct preclinical research on the long-term durability of a gene editing therapy for Duchenne muscular dystrophy (DMD). The gene editing therapy is designed to remove DMD exons 45-55 to restore the reading frame for 50% of DMD patients while retaining 87% of the protein coding sequence. The funded studies will assess molecular and...
This federal Project Grant award, funded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), aims to study the role of phosphorylated connexin-43 (CX43) as a potential therapeutic target for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The primary grant recipient is Rutgers, The State University of New Jersey, which will receive $2,096,171 over the project period from July 2024 to May 2028. The research...
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This $320,251 Project Grant awarded by the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), supports the development of a non-viral nanoparticle gene therapy delivery platform for treating cystic fibrosis and other chronic lung diseases. The project aims to further optimize the proprietary modular gene transfer technology, which uses DNA and RNA nanoparticles combined with uptake enhancers to improve the efficiency...
The federal Project Grant award of $2,368,153 from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) supports research to evaluate the safety and efficacy of using adeno-associated viral (AAV) vectors for cystic fibrosis (CF) gene therapy. The key objectives are to: Assess single-dose administration of an AAV1 vector containing a truncated CFTR gene (AAV1-D27-264CFTR) in rhesus macaques. Determine the therapeutic effects of...
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