Project Grant HT94252310787

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...

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,...

The National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research Federal Grant Program (CFDA 93.837), awarded Revidia Therapeutics, Inc. a $1,358,179 Project Grant to conduct IND-enabling safety and toxicity testing of a subcutaneous formulation of the PTP1B inhibitor MSI-1436. This novel therapy aims to treat fatal cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), a leading cause of mortality in this population. The proposed studies will...

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 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...

This Project Grant award from the National Institute of Allergy and Infectious Diseases (NIAID), under the Allergy and Infectious Diseases Research program (CFDA 93.855), will provide $817,161 to Mercer University to investigate the use of gut-derived mesenchymal stem cells (MSCs) as a potential autologous cell therapy for the treatment of perianal fistulas, a serious complication associated with Crohn's disease. The key objectives are to characterize the potency and immunosuppressive properties...

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...

The National Institute of Child Health and Human Development (NICHD) awarded a $295,923 Project Grant to Biostrategies LC under the Child Health and Human Development Extramural Research program (CFDA 93.865). The purpose of the award is to develop a "delivery-enhanced" gene therapy drug comprising an RTB:GALNS fusion protein for treating the rare metabolic disorder Mucopolysaccharidosis Type IVA (Morquio A Syndrome). The key objectives are to: 1) optimize the RTB:GALNS construct for...

The University of California, Davis was awarded a $478,417 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) federal grant program. The 5-year grant, effective September 1, 2024, will support the development of a multi-component, multi-functional extracellular vesicle-based nanotherapeutic for the treatment of multiple sclerosis (MS). The research aims to...

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...