Project Grant R21HD116032
- Federal Project Grant Summary The National Institute of Child Health and Human Development (NICHD) awarded University of Massachusetts Medical School a $460,625 Project Grant under the Child Health and Human Development Extramural Research program (CFDA 93.865) for the period of June 1, 2026 through May 31, 2028. The award funds development of prime editing therapeutics targeting Limb Girdle Muscular Dystrophy R9 (LGMDR9), a progressive muscular disease caused by mutations in the FKRP gene....
- Federal Grant Award Summary Myogene Bio LLC, a woman-owned small business biotechnology firm, received a $605,000 Project Grant from the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865), effective September 1, 2025 through August 31, 2027. The award funds preclinical research to assess the restoration of an internally deleted dystrophin protein as a surrogate biomarker for gene editing...
- Federal Grant Award Summary The National Institute of Child Health and Human Development (NICHD) awarded the University of Miami a Project Grant totaling $664,101 under the Child Health and Human Development Extramural Research program (CFDA 93.865) for the period June 1, 2026, through May 31, 2031. The award funds research investigating how disease-associated mutations in the Polycomb system impair human development, with a specific focus on missense mutations in the RNF2 gene linked to...
- Grant Award Summary The University of Maryland, College Park received a $156,000 Project Grant from the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865), awarded on June 17, 2026, with a completion date of May 31, 2028. This research project investigates the intrinsic and extrinsic mechanisms underlying trigeminal nerve deficits in familial dysautonomia (FD), a rare hereditary neuropathy caused...
- Federal Project Grant Award Summary Carnegie Institution for Science received a $335,000 Project Grant from the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training program (CFDA 93.859), effective September 9, 2025, with completion anticipated by August 31, 2026. The grant supports fundamental research investigating the mechanisms by which lamins—type V intermediate filament nuclear proteins—regulate three-dimensional genome organization and...
- Federal Project Grant Award Summary The National Institute of Child Health and Human Development (NICHD) awarded the Regents of the University of Michigan a $386,100 Project Grant under the Child Health and Human Development Extramural Research program (CFDA 93.865) beginning August 4, 2025, with completion targeted for July 31, 2027. This award supports investigator-initiated biomedical research designed to advance understanding of human development and improve child health outcomes. The...
- Project Grant Summary The National Institute of Child Health and Human Development (NICHD) awarded $1,279,974 on August 14, 2025, to Washington University (through its Office of Sponsored Research Services) under the Child Health and Human Development Extramural Research program (CFDA 93.865) for preclinical testing of early-life anti-myostatin therapy for osteogenesis imperfecta. The project, extending through April 30, 2030, will evaluate the efficacy of pharmacological inhibition of...
- Federal Grant Award Summary The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $519,166 Project Grant on August 8, 2025, to the Research Institute at Nationwide Children's Hospital to support preclinical development of a vectorized exon skipping (VES) therapeutic approach for Duchenne Muscular Dystrophy (DMD) exon 44 mutations. Under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), the awardee will develop and...
- Federal Project Grant Award Summary The University of Texas Southwestern Medical Center received a Project Grant award of $151,040 from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), effective April 15, 2026, with a completion date of November 30, 2028. This research project focuses on developing muscle-optimized non-viral gene editors to improve therapeutic outcomes for...
- Federal Grant Award Summary Myogene Bio LLC received a $499,300 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), awarded September 1, 2025, with completion targeted for July 31, 2028. The award supports the optimization of manufacturing processes and analytical testing protocols for a dual adeno-associated virus (AAV) vector gene editing therapy targeting...
Grant Award Summary The National Institute of Child Health and Human Development (NICHD) awarded $424,743 to the University of Maryland, College Park under the Child Health and Human Development Extramural Research program (CFDA 93.865) on June 25, 2026. The project, titled "The Molecular Basis of R249W LMNA-Related Congenital Muscular Dystrophy," will deliver foundational research and proof-of-concept therapeutic evidence addressing a rare genetic disorder accounting for approximately 25% of reported cases. The award supports research through May 31, 2028, and will produce mechanistic insights into lamin biology and musculoskeletal development, including research tools and data intended to inform future in vivo therapeutic studies. The research deliverables encompass three primary objectives: (1) characterization of how the R249W mutation affects skeletal muscle differentiation; (2) structural analysis of lamin organization and protein interactions using computational modeling (AlphaFold, Rosetta), advanced microscopy techniques (FRAP, Airyscan, transmission electron microscopy), and biochemical methods (immunoprecipitation-mass spectrometry, chromatin immunoprecipitation-sequencing); and (3) design, delivery, and assessment of adenine base editor (ABE) constructs in patient cells to measure gene correction efficiency and rescue of identified cellular and molecular phenotypes. This work serves as proof-of-concept for a potential gene-editing therapy addressing a severe early-onset muscular dystrophy with no currently available treatments.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $424.7k | 6/25/26 |