The National Eye Institute (NEI) awarded a $684,891 Project Grant (CFDA 93.867 Vision Research) to the Massachusetts Eye and Ear Infirmary in Boston, MA. The four-year grant, effective September 30, 2024, supports research to understand the functional consequences of rare genetic variants of unknown significance that contribute to inherited retinal degenerations, a leading cause of blindness. The key objectives are to: 1) Develop and apply a high-throughput splicing assay to identify rare coding...
This Project Grant award from the National Eye Institute (NEI), part of the Vision Research federal grant program (CFDA 93.867), provides $638,267 to the Schepens Eye Research Institute, Inc. to investigate therapies for optic neuropathy in familial dysautonomia, a severe neurodegenerative disorder. The research aims to develop retina-specific treatments, evaluate the efficacy of systemic and local therapeutic approaches to rescue visual function, and uncover the molecular mechanisms...
This federal Project Grant award, totaling $620,149, was provided by the National Eye Institute (NEI) under the Vision Research program (CFDA 93.867). The grant supports a multi-center research program focused on using advanced genome editing technologies, such as base editing and prime editing, to diminish the risk of age-related macular degeneration (AMD) by editing pathogenic alleles in the ARMS2/HTRA1 and CFH loci. The research will be conducted in human cells, including iPSC-derived retinal...
The National Eye Institute (NEI) awarded a $346,500 Project Grant (CFDA 93.867 Vision Research) to The Research Foundation for the State University of New York (RF SUNY) to develop an innovative mRNA-based gene therapy approach for treating inherited retinal dystrophies. The project aims to combine self-amplifying and self-splicing RNA technologies to increase the longevity, reduce the immunogenicity, and evaluate the potential in vivo side effects of this novel gene therapy approach. If...
The National Eye Institute (NEI) awarded a $2,790,977 Project Grant (CFDA 93.867 - Vision Research) to Oregon Health & Science University (OHSU) to optimize lipid nanoparticles (LNPs) for retinal gene editing in non-human primates. The key objectives are to: 1) determine LNP physicochemical features that facilitate photoreceptor expression of gene editors, 2) evaluate the immunogenicity of LNPs in the subretinal space, and 3) quantify in-vivo gene editing efficiency in photoreceptors....
This $1,789,542 Project Grant awarded by the National Eye Institute (NEI) under the Vision Research program (CFDA 93.867) supports research at St. Jude Children's Research Hospital to identify and characterize modular super-enhancers that regulate key transcription factor genes critical for retinal development in mice and humans. The research aims to provide a comprehensive map of promoter-super-enhancer interactions in the developing retina, which could help elucidate the genetic basis of...
The National Eye Institute (NEI) awarded a $592,457 Project Grant (CFDA 93.867 - Vision Research) to The Children's Hospital Corporation, doing business as Boston Children's Hospital, with a project period of September 30, 2024 to August 31, 2029. The funding will support research to characterize the response features of intrinsically photosensitive retinal ganglion cells (ipRGCs), which contain the photoreceptor melanopsin and influence processes such as the circadian clock, sleep, and pupil...
The National Eye Institute (NEI) has awarded a 5-year, $2,174,069 Project Grant (CFDA 93.867 - Vision Research) to The Trustees of the University of Pennsylvania to advance research on Fuchs' Endothelial Corneal Dystrophy (FECD). The key objectives are to elucidate the regenerative potential of corneal endothelial cells and develop innovative mRNA-based treatments to restore corneal transparency and visual function in FECD patients. The project involves a two-pronged approach. Aim 1 will use...
This federal Project Grant award from the National Eye Institute (NEI), under the Vision Research program (CFDA 93.867), supports the development of a novel genetic screening approach called "Mosaic in Vivo Perturbation (MVP)" to systematically assess gene function in the eye. The $1,387,142 award, effective September 1, 2024 through June 30, 2028, will be provided to the President and Fellows of Harvard College, doing business as Harvard Medical School. The key products and services...
The National Eye Institute (NEI) awarded a $360,897 Project Grant (CFDA 93.867 - Vision Research) to Human Cell Co, a for-profit biotechnology research company, with a period of performance from September 30, 2024 to August 31, 2025. The grant supports the development of a novel combination therapy approach for glaucoma, which involves topical application of an optimized nerve growth factor (NGF) mutein (HC201) and oral supplementation with nicotinamide (NAM). The goal of the Phase 1 study is to...