Project Grant R01AR085061
- 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...
- 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 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), provides $1,292,847.00 to Advertent Biotherapeutics, Inc. to develop a novel agent called ADA011 for preventing fatty degeneration of muscles in limb girdle muscular dystrophy 2B (LGMD2B). The goal is to demonstrate the long-term efficacy of ADA011 in inhibiting muscle fatty degeneration and...
- 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...
- This federal Project Grant award, funded 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 an improved adeno-associated virus (AAV) gene therapy for Pompe disease. The $540,712 award to Duke University will support research to enhance the efficacy and safety of an IGF2-tagged human acid alpha-glucosidase (IGF2-hGAA) gene therapy approach for correcting...
- The Project Grant awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), will fund a longitudinal assessment of molecular and imaging biomarkers for Limb Girdle Muscular Dystrophy Type R1 (LGMDR1). The $448,278 grant, awarded on September 18, 2025, aims to develop validated molecular and imaging biomarkers to facilitate clinical trials for LGMDR1, a common genetic...
- The University of Florida was awarded a $599,496 project grant by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research federal grant program (CFDA 93.846). The objective of this 5-year project, with a performance period from September 15, 2025 to August 31, 2030, is to evaluate the potential of magnetic resonance (MR) measures of bioenergetics and microvascular function to track disease progression and...
- This $1,464,751 Project Grant award from the Defense Health Agency's Military Medical Research and Development program (CFDA 12.420) supports research conducted by the University of California, Los Angeles (UCLA) to develop adeno-associated virus (AAV) vectors targeted to muscle and muscle stem cells. The goal is to improve the therapeutic efficacy and safety of treatments for Duchenne muscular dystrophy. The award period runs from September 1, 2024 to August 31, 2027. As the prime awardee, UCLA...
- This $661,501 federal Project Grant award was provided by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) to the University of California, Los Angeles (UCLA) for the period of September 1, 2024 through August 31, 2027. The award will fund research to develop AAV (adeno-associated virus) vectors targeted to muscle and muscle stem cells, which offer improved therapeutic efficacy and safety for treating Duchenne muscular dystrophy. UCLA, through...
- This federal Project Grant award from the National Institutes of Health's Research Infrastructure Programs (CFDA 93.351) provides $156,136 to Recombinetics Inc. to advance gene therapy for Mucopolysaccharidosis Type IVA (MPS IVA), a rare lysosomal storage disease. The key products and services to be delivered under this award include: 1) Generating a porcine model of MPS IVA to serve as a valuable tool for understanding the disease biology and developing genetic therapies; 2) Conducting a...
This federal Project Grant award of $1,091,199.00, funded 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 gene therapies for treating LAMA2-related dystrophy, a form of muscular dystrophy. The project, led by Rutgers, The State University of New Jersey, utilizes adeno-associated virus (AAV) delivery of genes encoding laminin-binding proteins to restore missing laminin functions and ameliorate the disease phenotype in mouse models. Specifically, the research will evaluate dose-response relationships, test late-stage AAV delivery, and optimize gene expression to improve outcomes. This multi-year effort seeks to advance the understanding and treatment of this congenital muscular dystrophy, with the goal of developing effective somatic gene therapies.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $1.1m | 8/7/25 |