This Project Grant award from the Defense Health Agency's Military Medical Research and Development program (CFDA 12.420) provides $551,250.00 to the University of California, Los Angeles (UCLA) to develop 3D-Bridge antisense oligonucleotides and a stem cell-targeting modality for skipping exons of Duchenne muscular dystrophy. The award period is from July 1, 2024 to June 30, 2026. Through this grant, UCLA will leverage its research and administrative capabilities to advance novel therapeutic...
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), will fund research to develop improved gene replacement and editing therapies for Duchenne muscular dystrophy (DMD). The total award amount is $671,240 and the project period runs from January 22, 2025 to December 31, 2029. The key research objectives are to: 1) Refine technologies for expressing...
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 $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 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 $1,557,819 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...
The federal Project Grant award ID HT94252510342, totaling $405,341.00, was provided by the Defense Health Agency (DHA) under the Military Medical Research and Development (CFDA 12.420) program. The award supports the use of proresolution and restorative pharmacology approaches for Duchenne muscular dystrophy (DMD) therapy at the Children's Research Institute, a non-profit pediatric research organization in Washington, D.C. The award will fund advanced biomedical research and development focused...
This $420,000 Project Grant awarded by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) supports research conducted by Myogene Bio LLC, a woman-owned small biotechnology company, to evaluate the efficacy of CRISPR gene editing technology in treating Duchenne muscular dystrophy (DMD). The research aims to correlate CRISPR efficacy with functional outcomes in preclinical DMD models, building on the company's previous federal grant awards focused...
This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) provides $395,009 to the University of California, Davis to lay the foundation for developing a molecular therapy for ADNP syndrome. The key products and services to be delivered under this 2-year award include: Testing the presumed mechanism of action of antisense oligonucleotides (ASOs) that can...
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,...