Project Grant W81XWH2210949

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 $306,872 in funding to Vivreon Biosciences LLC, a San Diego-based life sciences company. The award supports the development of a novel small molecule therapeutic targeting the ORAI1 calcium channel to delay progressive muscle weakening associated with Duchenne muscular dystrophy (DMD) and...

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 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 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...

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 $563,484.00 Project Grant award from the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) supports research conducted by Duke University to regulate skeletal muscle fibrosis in Duchenne muscular dystrophy. The two-year award, effective June 1, 2024 through May 31, 2026, will enable Duke University's research team to advance understanding and potential treatments for this debilitating neuromuscular disorder affecting military service members and...

This is a $538,218 federal Project Grant awarded by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420). The grant supports research conducted by the University of Texas Health Science Center at Houston (UTHealth) to study the role of ERR-alpha in skeletal muscle regeneration and functional recovery for Duchenne muscular dystrophy. The project aims to advance scientific understanding and develop potential treatments for this debilitating...

The University of Florida Division of Sponsored Research received a $536,800 Project Grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846). The grant will fund a 3-year exploratory clinical trial to investigate the potential of the drug tadalafil, combined with structured exercise training, to improve muscle pathophysiology and function in individuals with Duchenne...

This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $610,819 to Duke University to conduct research aimed at optimizing gene therapy for respiratory insufficiency in Duchenne muscular dystrophy (DMD). The key objectives are to: Comprehensively examine breathing and respiratory motor units in novel humanized mouse models of DMD, and compare them to the established MDX DMD mouse...