Project Grant R41HL151043

This federal Project Grant award, funded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), aims to study the role of phosphorylated connexin-43 (CX43) as a potential therapeutic target for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The primary grant recipient is Rutgers, The State University of New Jersey, which will receive $2,096,171 over the project period from July 2024 to May 2028. The research...

This $1,612,134 federal Project Grant award, provided by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, aims to investigate methods for enhancing the efficiency and safety of in vivo gene editing in muscle tissue as a potential therapeutic approach for Duchenne muscular dystrophy (DMD). The research project, led by the Joslin Diabetes Center, Inc., will test...

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 to Vivreon Biosciences LLC, a life sciences company in San Diego, California. The funding 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...

The National Heart, Lung, and Blood Institute (NHLBI), a division of the National Institutes of Health (NIH), awarded a $1,358,179 Project Grant (CFDA 93.837 Cardiovascular Diseases Research) to Revidia Therapeutics, Inc. to conduct IND-enabling safety and toxicity testing of a subcutaneous formulation of the PTP1B inhibitor MSI-1436. This novel small molecule therapy aims to treat fatal cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), a rare and debilitating genetic...

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 Cooperative Agreement 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) federal grant program, provides $1,541,019 to support preclinical research and development of a non-immunogenic gene therapy for a rare subset of Duchenne muscular dystrophy (DMD) patients. The research will focus on conducting IND-enabling studies for an innovative AAV vector encoding a...

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 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) program, provides $692,292 to the Children's Research Institute in Washington, D.C. to conduct research on understanding the regulation of pediatric muscle regeneration and its implications for treating muscle diseases like Duchenne muscular dystrophy (DMD). The research aims to address knowledge...

This federal Project Grant award, funded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), provides $2,282,515.00 to The University of Texas Southwestern Medical Center to develop non-invasive imaging biomarkers for detecting early metabolic changes associated with muscular dystrophy and cardiomyopathy. The award, effective June 15, 2024 through April 30, 2028, aims to leverage hyperpolarized carbon-13 MRI to assess...

This $236,250 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) supports the development of highly effective antisense oligonucleotide (ASO) therapeutics to treat Duchenne muscular dystrophy (DMD). The project aims to create novel ASO drugs that can induce exon skipping and partially restore dystrophin expression in DMD patients, with a focus on targeting exons 44...