This federal 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) aims to develop improved gene replacement and editing vectors for the treatment of Duchenne muscular dystrophy (DMD). The $671,240 award will fund research at the University of Washington over a 5-year period from January 2025 to December 2029. The key objectives are to: 1) Refine technologies...
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, totaling $1,601,933 and 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), supports research into therapeutic approaches for muscular dystrophy caused by mutations in the dysferlin gene (DYSF). The primary awardee, the University of Maryland, Baltimore, is conducting research to optimize engineered constructs of the DYSF-C2A domain...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $525,048 Project Grant (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) to Children's Hospital Medical Center in Cincinnati, OH. The grant is titled "Development and Optimization of Skeletal Muscle Delivery Vehicles" and runs from July 8, 2024 to May 31, 2029. The project aims to develop novel gene therapy delivery vehicles, including lentiviruses and extracellular vesicles,...
This Project Grant award of $1,292,847 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 the development of a novel therapeutic agent called ADA011 by Advertent Biotherapeutics, Inc. The goal is to inhibit muscle fatty degeneration and preserve muscle function in patients with limb girdle muscular dystrophy 2B (LGMD2B), a severe genetic disorder characterized by...
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), aims to develop enabling technologies for safe and effective gene editing in skeletal muscle. The $531,109 grant awarded to the University of Florida will fund research to address key challenges in applying gene editing to skeletal muscle diseases, including maximizing delivery and spread of editing...
This $1,557,819 Project Grant award from the National Institute of Child Health and Human Development (CFDA 93.865 - Child Health and Human Development Extramural Research) supports muscular dystrophy research at the Research Institute at Nationwide Children's Hospital in Columbus, Ohio. The award funds three key research projects: Project 1 (PI: Paul Martin, PhD) aims to utilize a novel bicistronic AAV vector to both prevent muscle disease and build new muscle function in patients with limb...
This federal Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA 93.350, provides $110,000 to Myogene Bio LLC to conduct preclinical research on the long-term durability of a gene editing therapy for Duchenne muscular dystrophy (DMD). The gene editing therapy is designed to remove DMD exons 45-55 to restore the reading frame for 50% of DMD patients while retaining 87% of the protein coding sequence. The funded studies will assess molecular 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 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 of $2,123,500.00 from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) aims to develop a novel gene therapy approach to treat muscular dystrophies. The key products and services to be delivered under this grant include:
Demonstrating the feasibility of using a dual adeno-associated viral (AAV) vector system to deliver full-length dysferlin, a protein deficient in dysferlinopathies, through intramuscular injections.
Evaluating the functionality and efficacy of the intein-generated full-length dysferlin in murine and human-derived myoblast models.
Optimizing the systemic delivery of the AAV-based intein/dysferlin therapy in mice, including dose-finding studies and biodistribution analysis in large animal models.
This research seeks to expand the use of AAV gene transfer technology to treat genetic disorders with large coding sequences that exceed the typical cargo capacity of these vectors, such as muscular dystrophies. The award period runs from September 2024 through May 2026.