Project Grant R41AR076871

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...

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 $499,300 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) supports research by Myogene Bio LLC, a woman-owned small business in San Diego, California, to advance manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy (DMD). The key products and services to be delivered include: 1) Optimizing the upstream...

The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) awarded a $302,500 Project Grant to Myogene Bio LLC, a woman-owned small business in San Diego, California. The grant, provided under NICHD's Child Health and Human Development Extramural Research program (CFDA 93.865), supports research to assess the use of a restored dystrophin protein as a biomarker for evaluating the efficacy of a gene editing therapy for Duchenne muscular dystrophy (DMD). The...

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 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, supports preclinical development of a gene therapy approach using adeno-associated virus (AAV) containing U7snRNA to skip exon 44 in the Duchenne muscular dystrophy (DMD) gene. The goal is to restore the reading frame and express a truncated but functional dystrophin protein, which could...

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 Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), has awarded a $444,838 project grant to the Trustees of Indiana University (doing business as Indiana University Indianapolis) to conduct research on the use of SGLT2 inhibitors for mitigating Duchenne cardiomyopathy, a severe complication of Duchenne muscular dystrophy. The project, titled "CHERISH (Duchenne Cardiomyopathy Mitigation SGLT2 Inhibitor),"...

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), is focused on developing a novel nanovesicle-based therapy for myotonic dystrophy type 1 (DM1), a common adult-onset muscular dystrophy. The $306,859 award supports a two-phase effort to validate interfering RNA-loaded nanovesicles that can selectively modulate the toxic DMPK mRNA responsible for...

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 $1,292,847.00 to Advertent Biotherapeutics, Inc. to develop a novel agent called ADA011 for preventing fatty degeneration of muscles in limb girdle muscular dystrophy 2B (LGMD2B). The goal is to demonstrate the long-term efficacy of ADA011 in inhibiting muscle fatty degeneration and...