FDA Meeting Helps Pave the Way for Clinical Trial of Cross-Cutting Gene Therapy

May 24, 2012
The clinical path for an innovative gene therapy designed to restore vision in people with a wide range of advanced retinal diseases is clearer thanks to a productive discussion with the U.S. Food and Drug Administration (FDA) about development plans. RetroSense Therapeutics, the company developing the gene therapy, and the Foundation Fighting Blindness, a funding partner, met with the FDA earlier this week to get feedback on plans to submit an Investigational New Drug (IND) application for launching a clinical trial of the treatment. Known as a pre-IND meeting, the discussion covered topics such as treatment manufacturing criteria and the scope and design of preclinical studies and the initial phases of a 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, the Foundation’s chief research officer. “Our pre-IND meeting was very helpful in determining the path that can give us the best opportunity for a timely and successful clinical trial.”

RetroSense’s gene therapy is designed to restore eyesight in people with advanced retinal diseases 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 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.

“We are very excited about the promise for bringing vision back to people who are completely blind,” says William T. Schmidt, chief executive officer of the Foundation. “Restoring vision in people with advanced degeneration is no small feat. But this solution is elegant and showing enormous potential.”

RetroSense’s treatment is optogenetic, meaning that 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 a variety of neurodegenerative conditions, including Parkinson’s disease. Nature magazine called it the “Method of the Year” in 2010.

On June 1, 2012, in Boston, the Foundation and Massachusetts Eye and Ear will host the Optogenetic Therapies for Vision workshop. The day-long forum will convene researchers, retinal specialists, companies interested in gene therapy, regulators and the National Eye Institute. They will examine the clinical path of optogenetic approaches in treating vision-robbing diseases such as macular degeneration, retinitis pigmentosa and related conditions.