Foundation Fighting Blindness and RetroSense Therapeutics Make Progress Toward First-Ever Clinical Trial for Optogenetic Gene Therapy Treatment

FDA Meeting Clarifies Path to the Clinic for Innovative Gene Therapy to Restore Vision

After a productive meeting this week with the U.S. Food and Drug Administration (FDA), the Foundation Fighting Blindness acknowledges a clearer path to the clinic for an innovative gene therapy developed by RetroSense Therapeutics to restore vision in people with a wide range of advanced retinal degenerative diseases. With discussions focused on plans to submit an Investigational New Drug (IND) Application for launching a clinical trial of the treatment, the pre-IND meeting covered topics such as: treatment manufacturing criteria, scope and design of the preclinical studies, and the scope and design of initial phases of the clinical trial.

"It is important at this stage of treatment development to be heading in the right direction, and guidance from the FDA is critical," says Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. "Feedback from the FDA pre-IND meeting was very helpful in determining that the path RetroSense identified will give us the best opportunity to launch—to our knowledge—the first-ever clinical study using optogenetic technology."

RetroSense’s treatment approach is optogenetic because it enables cells that normally don’t respond to light to become light sensitive. While optogenetics is a relatively new field of research, it is showing great promise for treating blinding diseases and a variety of neurodegenerative conditions including Parkinson’s disease. RetroSense’s gene therapy is designed to restore eyesight in people with advanced retinal diseases, like macular degeneration and retinitis pigmentosa, who have lost most or all of their photoreceptors—the cells in the outer retina that convert light into electrical signals which our brains perceive as vision. The treatment harnesses ganglion cells, cells of the inner retina that do not normally provide vision or respond to light, but often survive in advanced disease. The RetroSense therapy delivers copies of a light-sensing algal gene to the ganglion cells thereby restoring vision.

Researchers have demonstrated the treatment’s effectiveness in preclinical studies, positioning them well for moving the treatment to humans. A key benefit of this form of gene therapy is that it should work independent of the underlying genetic defect causing the disease. The Foundation is a funding partner and granted $250,000 to Ann Arbor, Mich.-based RetroSense earlier this year to support the company’s lab research in preparation for a clinical trial.

On June 1, 2012, in Boston, the Foundation and Mass. Eye and Ear will host the Optogenetic Therapies for Vision workshop, a day-long forum convening researchers, retinal specialists, companies interested in gene therapy, regulators, and the National Eye Institute, to examine the clinical path of optogenetic approaches in treating vision-robbing retinal diseases.

Allie Gebhardt

Foundation Fighting Blindness

410-423-0643

AGebhardt@FightBlindness.org

About Foundation Fighting Blindness

The Foundation Fighting Blindness is a national non-profit organization driving research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect more than 10 million Americans. Since 1971, the Foundation has raised over $450 million as the leading non-governmental funder of retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a variety of retinal degenerative diseases, and proving a cure is in sight. With a network of 50 chapters, the Foundation also provides support, education and resources to affected individuals and their families in communities across the country.

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About Us

The Foundation Fighting Blindness is a national nonprofit driving blindness research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect 10 million Americans. In its 40-year history, the Foundation has raised more than $425 million as the leading non-governmental funder of retinal research. With a network of 50 chapters, the Foundation also provides free disease information and low vision resources to affected individuals and their families. Most recently, a breakthrough Foundation-funded study using gene therapy restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a wide variety of retinal degenerative diseases, and proving a cure is in sight.

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