Project Grant R41AR087232
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Vecgen, Inc. $333,689 on September 1, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop and validate a diagnostic tool predicting immune responses to adeno-associated virus (AAV) gene therapies. Vecgen will clone and produce a comprehensive set of AAV vectors carrying FDA-approved gene therapy sequences and establish standardized immunogenicity benchmarks using...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded $151,040 to The University of Texas Southwestern Medical Center on April 15, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop muscle-optimized non-viral gene editors for Duchenne muscular dystrophy. The project addresses limitations in lipid nanoparticle (LNP) delivery of CRISPR gene editing to skeletal muscle by designing gene editors that improve myonuclear...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded the University of Florida $1.768 million on September 15, 2025, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to assess magnetic resonance measures of bioenergetics and microvascular function in Duchenne and Becker muscular dystrophy. The project evaluates whether MR-based measures of muscle energetics and blood vessel function can track disease progression and treatment...
- 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...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded the University of Florida $340,978 on August 11, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop microRNA-targeting strategies for tissue-restricted gene delivery in articular joints. The research addresses limitations in intra-articular biologic treatments for joint injury and disease by engineering adeno-associated vectors (AAV) to deliver growth factors such...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded the University of Arizona $578,565 on July 10, 2026, to investigate the role of skeletal myosin binding protein-C in distal arthrogryposis under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846). Distal arthrogryposis is a genetic skeletal muscle disorder characterized by congenital joint contractures, muscle weakness, and reduced mobility with significant clinical morbidity. No...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Ikaika Therapeutics Inc. $995,938 on September 1, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to fund preclinical development of anti-LTBP4 monoclonal antibodies for treating neuromuscular diseases. The project develops second-generation anti-LTBP4 antibodies targeting the hinge region of latent TGF-binding protein 4, which regulates pathological TGF-β activation...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Duke University $377,506 on June 5, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop a chimeric antigen receptor T cell (CAR-T) therapy targeting inflammation and fibrosis in Duchenne muscular dystrophy. The research will evaluate CAR-T cells engineered to eliminate pro-inflammatory macrophages and fibrogenic fibro-adipogenic progenitors in the dystrophic muscle...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded the University of Arizona $375,381 on September 2, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop a small-molecule drug discovery platform targeting MYBPC1 mutations that cause distal arthrogryposis. The project addresses the absence of available therapeutics for distal arthrogryposis, a congenital muscle disorder affecting approximately 1 in 3,000 live births...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Northeastern University $652,195 on June 17, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop and test surface-modified, cartilage-targeting exosomes as non-viral gene delivery vehicles for osteoarthritis treatment. The project engineers charge-reversed cationic exosomes derived from mesenchymal stem cells, anchored with arginine-rich peptide motifs, to...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Ventura Life Sciences, LLC $305,443 on September 1, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop novel AAV capsids for skeletal muscle gene therapy. The recipient will employ directed evolution methods, high-throughput screening, and immune assays to engineer and validate adeno-associated virus (AAV) capsid variants with improved transduction capability for skeletal muscle while maintaining a favorable safety profile. The work addresses safety concerns arising from high-dose AAV9 and AAVRH74 protocols in Duchenne's muscular dystrophy treatment by engineering next-generation AAV capsids based on the AAVRH74 serotype, leveraging triplet AAV library synthesis to generate up to 1 billion clonal variants and RNA-guided directed evolution coupled with long-read screening to identify candidates with enhanced muscle targeting and reduced hepatic toxicity. The assistance type is a Project Grant. Performance takes place in Gainesville, Florida, with a period of performance from September 1, 2026, through August 31, 2027.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $305.4k | 8/27/26 |