Project Grant SB1AR084953
- 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 $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...
- The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) awarded a $302,500 Project Grant to Myogene Bio LLC, a woman-owned small business in San Diego, California. The grant, provided under NICHD's Child Health and Human Development Extramural Research program (CFDA 93.865), supports research to assess the use of a restored dystrophin protein as a biomarker for evaluating the efficacy of a gene editing therapy for Duchenne muscular dystrophy (DMD). The...
- 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 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 $313,148 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 support the development of an in vivo imaging platform for myotonic dystrophy type 1 (DM1) drug discovery. The project aims to create novel fluorogenic RNA aptamer-based reporters that can detect drug-induced changes in toxic RNA foci formation and sequestration of the MBNL splicing...
- 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), is focused on developing a novel nanovesicle-based therapy for myotonic dystrophy type 1 (DM1), a common adult-onset muscular dystrophy. The $306,859 award supports a two-phase effort to validate interfering RNA-loaded nanovesicles that can selectively modulate the toxic DMPK mRNA responsible for...
- 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 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...
- 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 $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 manufacturing process for a dual adeno-associated virus (AAV) vector gene editing therapy, using a Design of Experiment approach to improve yield and quality of the therapy materials; and 2) Developing and optimizing product-specific analytical assays and other release testing to support regulatory standards for manufacturing the gene editing therapy. This work is critical to enable the advancement of this DMD therapy candidate into clinical trials and eventual commercialization, as it targets a genetic deletion affecting 50% of DMD patients who currently have limited treatment options.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $499.3k | 8/28/25 |