Project Grant R61NS136653
- This federal Project Grant award of $671,240 from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) supports research to develop enhanced dystrophin gene therapies for Duchenne muscular dystrophy (DMD). The University of Washington will conduct this research from January 2025 through December 2029. The project aims to design and test micro-, mini-, and full-length dystrophin gene...
- This $499,300 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) supports research by Myogene Bio LLC, a woman-owned small business in San Diego, California, to advance manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy (DMD). The key products and services to be delivered include: 1) Optimizing the upstream...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $295,294 Phase I SBIR project grant (CFDA 93.846) to Gigamune, Inc. to develop a novel lentiviral gene delivery platform for in vivo delivery of the full-length dystrophin gene to muscle cells. The goal is to create an efficient and targeted approach for gene therapy to treat Duchenne muscular dystrophy, a severe genetic disorder causing muscle wasting. The project aims to leverage Gigamune's...
- This federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $692,292.00 to the Children's Research Institute in Washington, D.C. to conduct research aimed at understanding the regulation of pediatric regenerative myogenesis and its implications for muscle disease. The key products and services to be delivered under this 5-year award...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, will support preclinical testing of a novel gene therapy for a rare pediatric neurodegenerative disease. The $419,482 award to the University of Texas Southwestern Medical Center will fund studies to determine the minimal effective dose and post-symptomatic treatment efficacy of the...
- This $699,818 Project Grant awarded on July 8, 2025 by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) aims to develop an effective adeno-associated virus (AAV) gene therapy product for treating Mucopolysaccharidosis Type IIIC (MPS IIIC), a rare and devastating lysosomal storage disease. The project seeks to leverage extracellular vesicle-mediated mRNA trafficking to...
- This federal Project Grant award of $345,664.00 from the Defense Health Agency, under the Military Medical Research and Development (CFDA 12.420) program, supports an advanced biomedical research initiative led by Royal Holloway University of London. The research project aims to develop an AAV-based microdystrophin gene therapy for Duchenne muscular dystrophy, with a focus on enhancing immune protection through capsid bioengineering. The award period spans from December 1, 2024 to November 30,...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) will lay the foundation for developing a molecular therapy for ADNP syndrome. The $395,009 award to the University of California, Davis will fund research to: 1) test the mechanism of action of antisense oligonucleotides (ASOs) that can increase ADNP protein expression in human neurons, and 2)...
- This federal Project Grant award, funded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, aims to develop a non-viral gene-editing therapy using lipid nanoparticle (LNP)/mRNA complexes to correct UBE3A gene overexpression in neural stem progenitor cells during fetal brain development. The $654,339 award to the University of California, Davis will support research to...
- This $420,000 Project Grant awarded by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) supports research conducted by Myogene Bio LLC, a woman-owned small biotechnology company, to evaluate the efficacy of CRISPR gene editing technology in treating Duchenne muscular dystrophy (DMD). The research aims to correlate CRISPR efficacy with functional outcomes in preclinical DMD models, building on the company's previous federal grant awards focused...
This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, supports preclinical development of a gene therapy approach using adeno-associated virus (AAV) containing U7snRNA to skip exon 44 in the Duchenne muscular dystrophy (DMD) gene. The goal is to restore the reading frame and express a truncated but functional dystrophin protein, which could benefit 6-12% of DMD patients. The $519,166 award, effective August 8, 2025 through January 31, 2027, will allow the Research Institute at Nationwide Children's Hospital to compare their current vectorized exon skipping approach against alternative designs to identify the most robust exon 44 skipping response for potential therapeutic development.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $519.2k | 8/6/25 |