Project Grant R01AR085087
- This Project Grant, awarded by the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research), aims to study alveolar injury and repair in Pompe disease. The $762,974 award, effective from January 29, 2025 to November 30, 2028, will support research at Duke University to investigate how the glycogen deficiency in Pompe disease disrupts alveolar type 1 and type 2 cells, leading to respiratory complications. The key objectives are to determine if these alveolar...
- 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...
- 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 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 $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 Project Grant award of $454,050.00, 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), will fund the development of a novel gene therapy for bone healing. The award recipient, Mayo Clinic, will use adeno-associated virus (AAV) to deliver bone morphogenetic protein-2 (BMP-2) to critical-sized bone defects in a validated rat model. The goal is to create an...
- This $667,125 Project Grant award from 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), aims to develop a long-lasting, bone-targeted gene therapy for Osteogenesis Imperfecta (OI), the most common bone fragility disease. The University of Massachusetts Medical School (UMMS) will use a novel recombinant adeno-associated virus (rAAV) vector with bone-specific...
- This Project Grant award of $3,192,404 from the National Institute of Allergy and Infectious Diseases (NIAID) under the Allergy and Infectious Diseases Research program (CFDA 93.855) supports research on immunomodulation in adeno-associated virus (AAV) gene therapy. The research aims to better understand, model, and manage immune responses that can arise from systemic dosing of recombinant AAV vectors, which pose a major challenge in the treatment of monogenic disorders using this gene...
- 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 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 skeletal muscle and brain deficits in a Pompe disease mouse model. The project seeks to address the limitations of current enzyme replacement therapy and gene therapy approaches by using site-specific mutagenesis to prevent off-target binding and associated adverse effects of IGF2-hGAA. The goal is to create a more effective and clinically translatable gene therapy for the treatment of Pompe disease, which causes progressive neuromuscular dysfunction.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $540.7k | 8/27/25 |