Project Grant R21TR005472
- 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...
- 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...
- 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...
- 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 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...
- 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 of $511,987.00 from the National Heart Lung and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) supports a comparative study on gene regulatory and structural mechanisms in lamin-related cardiomyopathy. The project aims to directly compare two prevailing hypotheses on the pathogenesis of lamin A/C-related dilated cardiomyopathy (LMNA-DCM) - the gene regulatory hypothesis and the structural hypothesis - to advance the mechanistic...
- This $306,872 Project Grant awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under CFDA 93.846 is supporting Vivreon Biosciences, LLC, a life sciences company in San Diego, California, to develop a novel small molecule therapeutic targeting the ORAI1 calcium channel for treating Duchenne muscular dystrophy (DMD) and other dystrophinopathies. The key objectives of this 2-year project are to assess the toxicity profile of the therapeutic candidate and...
- This $563,484.00 Project Grant award from the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) supports research conducted by Duke University to regulate skeletal muscle fibrosis in Duchenne muscular dystrophy. The two-year award, effective June 1, 2024 through May 31, 2026, will enable Duke University's research team to advance understanding and potential treatments for this debilitating neuromuscular disorder affecting military service members and...
This federal Project Grant award of $440,000 from the National Center for Advancing Translational Sciences (NCATS) CFDA program is funding the development of an anti-LTBP4 biologic to treat Limb Girdle Muscular Dystrophy 2C (LGMD 2C). LGMD 2C is a severe subtype of Limb Girdle Muscular Dystrophy caused by the loss of the G-sarcoglycan protein, resulting in progressive muscle weakness and impaired mobility. The project seeks to leverage the discovery that the LTBP4 protein is a genetic modifier that can modulate muscle membrane stability and fibrosis in LGMD 2C. The goal is to develop an anti-LTBP4 biologic that can reduce excess TGF-beta activation and delay disease progression. The project will be conducted by Northwestern University over a two-year period from August 1, 2025 to July 31, 2027.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $0 | 9/12/25 | ||
| Not listed | $440.0k | 7/24/25 |