Researchers at Cedars-Sinai Medical Center in Los Angeles used an innovative gene-editing technique to prevent vision loss in a rodent model of autosomal dominant retinitis pigmentosa (adRP). The emerging therapy specifically shuts down the S334ter-3 mutation in the gene rhodopsin (RHO), a common cause of adRP.
Hello! My name is Heather Napolitano. When I began losing my vision, my husband and I were scared and searching everywhere for answers. The sense of community and support we felt upon finding the FFB was indescribable. They lifted our hopes and instilled a fight within us to find a cure.
The Foundation for Retinal Research and the Foundation Fighting Blindness merged their two organizations on January 1
The Foundation Fighting Blindness and the biopharmaceutical company Vision Medicines are partnering to develop a drug known as VM200 for preserving vision in people with Stargardt disease, the leading cause of inherited juvenile macular degeneration. The Foundation is committing $7.5 million to co-fund VM200 development. Vision Medicines plans to launch a human study of the emerging therapy in 2016.
Foundation-funded researchers from the University of Pennsylvania have found that an emerging gene therapy for X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR preserves vision in canines with late-stage disease.
Spark Therapeutics today announced positive top-line results from a Phase 3 pivotal trial of SPK-RPE65, an investigational gene therapy product for the treatment of RPE65-mediated inherited retinal dystrophies (IRDs). This research was funded in part by the Foundation Fighting Blindness, the world’s largest private funder of early, translational and clinical-stage research for blinding retinal diseases.