Leber congenital amaurosis

Foundation Fighting Blindness Applauds Phase 3 Study Results for Investigational Gene Therapy Treatment

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.

A Statement from Dr. Stephen Rose about Recent LCA Gene-Therapy Findings

The Foundation Fighting Blindness is aware of the most recent results of the University of Pennsylvania and Moorfields RPE65/LCA2 gene-therapy trials, which were recently published in The New England Journal of Medicine and presented before the Association for Research in Vision and Ophthalmology on Sunday, May 3. These dosing trials have shown that retinal gene therapy for LCA2 is safe and that there is substantial improvement of vision in the treated area of the retina.

Foundation Fellowship Awarded to Clinical Investigator

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.

U.S. Human Study for Choroideremia Gene Therapy Launched by Spark Therapeutics

The rapidly advancing field of vision-saving gene-therapies is taking a significant step forward with the launch of a U.S. clinical trial for a treatment for choroideremia, a blinding condition affecting males. Researchers believe a single dose of the therapy, which replaces mutated copies of the choroideremia gene, CHM, with healthy copies, will halt or possibly reverse the disease process for several years. The Foundation Fighting Blindness provided funding over two decades for lab studies that helped make the trial possible.

Several FFB-Funded Treatments Moving Closer to Human Studies

A few weeks ago, 15 of the world’s leading retinal researchers, all funded by  FFB’s Translational Research Acceleration Program (TRAP), gathered for an annual meeting in Las Vegas to discuss their progress in developing vision-saving treatments. Most are poised to move into clinical trials within the next two to five years.  
“It’s an exciting time in translational research for retinal diseases,” says Patricia Zilliox, Ph.D., chief drug development officer at the