Project Grant R43AR086092
- 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 $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...
- 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 Project Grant awarded by 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 a longitudinal assessment of molecular and imaging biomarkers for Limb Girdle Muscular Dystrophy Type R1 (LGMDR1). The $448,278 grant, awarded on September 18, 2025, aims to develop validated molecular and imaging biomarkers to facilitate clinical trials for LGMDR1, a common genetic...
- This Project Grant award of $719,758 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 on the role of nuclear myotubularin (MTM1) in muscle development and the pathogenesis of X-linked myotubular myopathy (XLMTM). The research aims to characterize the localization and interactome of nuclear MTM1, define alterations in nuclear structure and genome...
- 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 Arthritis and Musculoskeletal and Skin Diseases (NIAMS), under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), aims to develop specialized cryo-electron microscopy (cryo-EM) techniques to study the structures of mitochondrial complexes implicated in muscular myopathies. The $444,109 award to the Scripps Research Institute in La Jolla, CA will support the creation of methods to directly visualize endogenous...
- The National Institute of General Medical Sciences (NIGMS) awarded a $700,000 Project Grant under the Biomedical Research and Research Training (CFDA 93.859) program to Mirimus, Inc., a self-certified small disadvantaged business located in Brooklyn, NY. The grant funding, with a period of performance from September 20, 2024 to April 19, 2025, will support the creation of next-generation RNA interference (RNAi) therapeutics and the development of an in vivo mouse model to evaluate their efficacy...
- This federal Project Grant award of $671,240 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 enhanced dystrophin gene therapies for Duchenne muscular dystrophy (DMD). The University of Washington will conduct this research from January 2025 through December 2029. The project aims to design and test micro-, mini-, and full-length dystrophin gene...
- 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...
This $313,148 Project Grant awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) will support the development of an in vivo imaging platform for myotonic dystrophy type 1 (DM1) drug discovery. The project aims to create novel fluorogenic RNA aptamer-based reporters that can detect drug-induced changes in toxic RNA foci formation and sequestration of the MBNL splicing factor in DM1 disease models. This non-invasive in vivo reporter system is intended to enable more efficient preclinical evaluation of potential DM1 therapeutic candidates by facilitating high-throughput screening and real-time monitoring of target engagement and pharmacokinetic properties, thereby reducing animal testing requirements. The awardee, Lucerna Inc., is a minority-owned and woman-owned small business specializing in developing advanced RNA-based technologies and diagnostic tools for various disease research applications.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $313.1k | 7/29/25 |