Research Articles - Retinitis Pigmentosa
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.
The U.S. Food and Drug Administration (FDA) has authorized the initiation of the first human study of a stem-cell treatment for people with retinitis pigmentosa (RP). ReNeuron, a stem-cell development company in the United Kingdom, will conduct the 15-particpant, Phase I/II trial of its emerging therapy at Massachusetts Eye and Ear Infirmary (MEEI).
Shire plc and the Foundation Fighting Blindness today announced a new agreement focused on furthering research for a novel treatment for autosomal dominant retinitis pigmentosa (adRP), a rare genetic disease that usually first occurs in late childhood or adolescence and is followed by the progressive loss of peripheral vision. There are no currently approved treatment options for adRP.
When it comes to restoring vision to the blind, researchers may soon bypass the eye entirely and go straight to the brain.
While driving research to treat and cure retinal diseases is at the core of the Foundation’s mission, enabling young clinician-scientists to develop promising therapies and move them through human studies is also critical to its commitment to eradicating blindness. Continuing its collaboration with the Howard Hughes Medical Institute (HHMI), which began in 2009, the Foundation has awarded annual fellowships to two medical students, who will take a year away from their clinical education to conduct retinal-disease research.
Rachel Huckfeldt, M.D., Ph.D., an up-and-coming clinical scientist, is receiving a one-year, $65,000 fellowship from the Foundation Fighting Blindness to gain the knowledge and experience to join the retinal clinical practice and launch a research program at the Massachusetts Eye and Ear Infirmary (MEEI). Her ultimate goal is to provide treatments and cures for people affected by retinal degenerative diseases.