Project Grant R43AR082726

Award Date 9/1/23
Completion Date 8/31/24
Dollars Obligated $295K
Federal Grant Program
93.846
Assistance Type
Project Grant
Place of Performance
California, USA
Similar Awards
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 Children's Hospital Medical Center in Cincinnati, OH. The grant is titled "Development and Optimization of Skeletal Muscle Delivery Vehicles" and runs from July 8, 2024 to May 31, 2029. The project aims to develop novel gene therapy delivery vehicles, including lentiviruses and extracellular vesicles,...
This $671,240 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 to develop improved gene therapy approaches for Duchenne muscular dystrophy (DMD). The key objectives are to: 1) Refine technologies for expressing larger and more functional dystrophin or utrophin proteins using adeno-associated virus (AAV) vectors, 2) Develop enhanced...
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), aims to develop enabling technologies for safe and effective gene editing in skeletal muscle. The $531,109 grant awarded to the University of Florida will fund research to address key challenges in applying gene editing to skeletal muscle diseases, including maximizing delivery and spread of editing...
This federal 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), aims to develop novel genetic medicine approaches to treat orphan diseases. The $2,123,500 award to Kinea Bio Inc. will fund research to overcome the limited cargo capacity of adeno-associated viral (AAV) vectors, which has excluded many disorders like dystrophies and dysferlinopathies...
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...
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...
This $692,292 federal 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 will fund research to understand the regulation of pediatric muscle regeneration and its implications for treating muscle diseases such as Duchenne muscular dystrophy (DMD). The 5-year project, running from April 2025 to March 2030, will be conducted by the Children's...
This federal 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 $1,601,933.00 to the University of Maryland, Baltimore to support research into developing C2 domain therapeutics for muscular dystrophy. The research aims to optimize engineered constructs of the DYSF-C2A domain to rescue deficits in calcium signaling and membrane repair in...
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...

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 proprietary "GigaLenti" lentiviral vector technology to specifically deliver the large dystrophin gene payload to muscle cells, overcoming limitations of current adeno-associated virus (AAV) gene therapy approaches. This 1-year project period runs from September 2023 to August 2024, and is part of NIAMS' broader research program on arthritis, musculoskeletal, and skin diseases.

Generated 7/16/24, 11:18 AM