Project Grant R43AR078042

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 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 $671,240 to the University of Washington for developing improved gene therapy approaches to treat Duchenne muscular dystrophy (DMD). The research aims to refine technologies for expressing larger and more functional dystrophin and utrophin proteins using adeno-associated virus (AAV)...

The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $1,541,019 Cooperative Agreement (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to The Trustees of the University of Pennsylvania for a 3-year project to conduct IND-enabling studies of a non-immunogenic gene therapy for the ultra-rare subset of Duchenne muscular dystrophy (DMD) patients excluded from dystrophin gene therapy trials. The project aims to develop a safe and...

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 $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...

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 the Children's Hospital Medical Center in Cincinnati, Ohio. The grant supports research to develop and optimize novel skeletal muscle delivery vehicles, including lentiviruses and extracellular vesicles, that can effectively target and deliver gene therapies to treat genetic muscle diseases like Duchenne...

This federal Project Grant award from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), provides $236,250 to the University of California, Los Angeles (UCLA) to develop innovative antisense oligonucleotide (ASO) therapeutics targeting dystrophin exons 44 and 45 for the treatment of Duchenne muscular dystrophy (DMD). The key objectives are to design ASOs with enhanced binding affinity and...

This $661,501 federal Project Grant award was provided by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) to the University of California, Los Angeles (UCLA) for the period of September 1, 2024 through August 31, 2027. The award will fund research to develop AAV (adeno-associated virus) vectors targeted to muscle and muscle stem cells, which offer improved therapeutic efficacy and safety for treating Duchenne muscular dystrophy. UCLA, through...

This Project Grant award from the Department of Defense's Military Medical Research and Development program (CFDA 12.420) provides $572,306 in funding to the University of Washington for a 3-year research project to develop "ENHANCED GENE THERAPY FOR DMD USING LARGER DYSTROPHINS AND LOWER VECTOR DOSES". The aim is to advance gene therapy approaches for treating Duchenne muscular dystrophy (DMD) by utilizing larger dystrophin gene constructs and reducing the required vector doses. The...

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 $2,123,500.00 in funding to Kinea Bio Inc. in Mukilteo, WA. The award supports the development of a novel gene therapy approach using adeno-associated viral (AAV) vectors to treat muscular dystrophy disorders, such as dysferlinopathies, that have been challenging to address due to the large...