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...
This $882,971 Project Grant award from the National Eye Institute (NEI) under the Vision Research program (CFDA 93.867) supports the development of OPGX-RHO, a single-vector adeno-associated virus (AAV) gene therapy for the treatment of autosomal-dominant retinitis pigmentosa (ADRP). The key objectives are to: 1) Complete chemistry, manufacturing and controls of a clinical-grade batch of OPGX-RHO, including device compatibility and stability studies; and 2) Conduct efficacy and safety/toxicology...
This federal Project Grant award of $431,750.00 from the National Eye Institute (NEI), under the Vision Research federal grant program (CFDA 93.867), supports research to develop a novel, femtosecond (FS) laser-based approach for delivering gene therapy to the cornea. The key objectives are to create microchannels through the corneal epithelium to enhance transepithelial drug diffusion, and use subthreshold FS laser pulses to open temporary pores in stromal cells for plasmid-based gene delivery,...
This Project Grant award from the National Eye Institute (NEI), under the Vision Research federal grant program (CFDA 93.867), will support research focused on understanding the role of centriole linkage in retinal degeneration and hearing loss. The $462,430 award to the University of Utah will fund a 5-year study to investigate the function of the CNAP1 protein and its isoforms in maintaining the ciliary structure and the Usher Syndrome Type 2 (USH2) protein complex in photoreceptors and hair...
The Federal Cooperative Agreement award U01TR005352, funded by the National Institutes of Health (NIH) Office of the Director under the Trans-NIH Research Support program (CFDA 93.310), provides $3,314,560 to the Massachusetts Eye and Ear Infirmary to develop CRISPR-Cas-based gene editing therapeutics for two types of dominant genetic hearing loss: DFNA41 due to a P2RX2 mutation and DFNA2 due to a KCNQ4 mutation. The project aims to conduct IND-enabling studies, including evaluating off-target...
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 $103,860 Project Grant, awarded by the National Eye Institute (NEI) under the Vision Research federal grant program (CFDA 93.867), aims to advance the treatment and molecular understanding of oculocutaneous albinism (OCA) through gene therapy. The key objectives are to: Identify the optimal therapeutic window and delivery route for an adeno-associated viral vector-based OCA gene therapy (AAV-TYR). Administer the AAV-TYR therapy at critical time points during retinal development to rescue...
This $246,000 Project Grant award from the National Eye Institute (NEI) under the Vision Research program (CFDA 93.867) will support research by the University of Texas Southwestern Medical Center to identify and characterize gene/protein defects and molecular pathways that lead to early-onset glaucoma. The researchers will employ a high-throughput, unbiased forward genetics pipeline to screen for genes associated with inner retinal thinning, a hallmark of early-onset glaucoma. The project...
This federal Project Grant award from the National Eye Institute (NEI) under the Vision Research program (CFDA 93.867) provides $425,000 to the Massachusetts Eye and Ear Infirmary to conduct genetic profiling research on childhood glaucoma in India. The project aims to identify rare genetic variants that cause this debilitating eye disease, which can lead to irreversible blindness in children, particularly in developing countries like India where diagnosis and treatment options are limited....
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...