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...
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...
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 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 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 $1,727,423 to investigate the role of the muscle-derived protein FGF21 in Duchenne muscular dystrophy (DMD) and its effects on bone health. The research aims to identify the cellular source and molecular mechanisms driving elevated FGF21 expression in dystrophic muscle, as well as...
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 $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 $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 Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), provides $151,471.00 to the University of California, Davis to conduct research on using collagenase to target collagen architecture in fibrotic skeletal muscle. The goal is to reduce muscle stiffness and promote tissue regeneration, particularly in the context of Duchenne muscular...