Innovation Summit Fosters Researcher, Clinician, and Industry Collaborations to Advance Gene and Cell Therapies for Inherited Retinal Diseases

Foundation Fighting Blindness and Oregon Health and Science University Casey Eye Institute host event

(Columbia, MD) — The Foundation Fighting Blindness (FFB), a national nonprofit focused on funding cutting-edge retinal disease research, and the Casey Eye Institute at Oregon Health & Science University will co-host the fourth annual Retinal Cell and Gene Therapy Innovation Summit in Baltimore, May 4–5, 2017.

The summit will take place just prior to ARVO 2017 — the annual meeting of the Association for Research in Vision and Ophthalmology. It will include presentations by the retinal disease field’s leading experts on potential gene and stem-cell therapies and how best to deliver them to patients. The Summit begins at 1:00 p.m., Thursday, May 4, and runs through 5:00 p.m., Friday, May 5.

“The purpose of the Summit is to create visibility for the many treatment therapy projects in or entering the clinic based on a gene or cell therapy approach,” said Patricia Zilliox, FFB-CRI chief drug development officer. “These approaches, many of which were initiated or supported with funding from FFB, hold strong promise for the treatment of diseases of the retina such as AMD or retinal dystrophies. This progress is occurring due to the expertise and collaboration of companies, clinicians, and scientists, many of whom will be in attendance at the summit.”

Gene therapy, in particular, has shown good promise in the battle to treat inherited retinal diseases. The Foundation was instrumental in funding the preclinical and early clinical work that resulted in an investigational gene therapy targeting retinal diseases caused by mutations in the RPE65 gene. This therapy, which was the first gene therapy to successfully complete a Phase III clinical trial, is expected to be submitted to the U.S. Food & Drug Administration for review this year. If approved, it would be the first-ever approved gene therapy for an inherited retinal disease and the first for any inherited disease in the United States.

The summit will cover numerous gene and stem-cell therapies targeting multiple vision-robbing diseases, including sessions on preclinical RPE cell transplantation, cell therapy for age-related macular degeneration, subretinal transplantations, vascular and neuronal cell-based therapies, gene therapy, and vector design.

“Advances in cell and gene therapy continue to bring us closer to treatments for all inherited retinal degenerations. We are excited for the fourth annual Innovation Summit to be held in Baltimore this year prior to the ARVO meeting, which will gather some of the top speakers from around the world,” said Mark Pennesi, MD, PhD, Associate Professor, Chief, Ophthalmic Genetics Division, Casey Eye Institute, Oregon Health & Science University.

Summit registration is available online at


The Foundation Fighting Blindness was established in 1971. It has since raised more than $700 million for research aimed at preventing, treating, and curing blindness caused by retinal degenerative diseases. In excess of 10 million Americans, and millions more worldwide, experience vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and other inherited retinal diseases.

OHSU Casey Eye Institute is a world-recognized academic eye center eliminating blindness through education, innovation, and community service. Casey is one of the leading institutions for vision research in the nation known for developing and utilizing the very latest treatments and technologies and is a global leader in ophthalmic imaging and in clinical trials related to eye disease. From ground-breaking research and education to specialty care in genetics, retina, glaucoma, inflammatory eye disease, and refractive and plastic surgery — Casey’s focus is to provide the best possible eye health in its clinics and out in the community.

Spark Therapeutics is a sponsor of the Innovation Summit. Spark Therapeutics is a fully integrated company that strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal disease (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver.

Original Date: 
Monday, April 17, 2017