Wayne State University

Technology developed by Dr. Zhou-Hua Pan and licensed by RetroSense gets "green light" for orphan drug designation for retinitis pigmentosa treatment

Posted: Thursday, October 30, 2014

Unique approach combines gene therapy and optogenetics with the intention to treat blindness

Technology to restore vision through the use of a component of green algae developed by Zhuo-Hua Pan, Ph.D., scientific director of the Ligon Research Center of Vision at the Kresge Eye Institute, the Edward T. and Ellen K. Dryer Endowed Professor in Vision and Blindness Research in the Department of Ophthalmology, and professor of anatomy and cell biology in Wayne State University's School of Medicine, was granted Orphan Drug designation for the treatment of retinitis pigmentosa (RP) by the U.S. Food and Drug Administration. RP, a genetic condition, leads to the progressive degeneration of rod and cone photoreceptors in the retina and results in severe vision loss and blindness. Currently there are no FDA-approved drugs to treat the disease.

RetroSense Therapeutics, LLC, a privately-held biopharmaceutical company, is developing RST-001 as a first-in-class gene therapy application of optogenetics - a means of conferring light sensitivity to cells that were not previously, or natively light sensitive - designed to restore vision to those affected by RP.

By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is conferring new light sensitivity to the retina, with the expectation of improved or restored vision. RST-001 is expected to have application to all forms of RP, independent of causative gene or mutation.

"We are pleased that the FDA has granted Orphan Drug status to our lead product, RST-001," said Sean Ainsworth, CEO of Retrosense Therapeutics. "This significant milestone will enable us to continue to develop new and innovative treatments for retinitis pigmentosa, a truly debilitating condition. We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa."

The FDA Office of Orphan Products Development supports the evaluation and development of products that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions. The FDA awards Orphan Drug designation as an incentive to develop drugs and biological therapeutics for diseases that affect fewer than 200,000 people in the United States. The benefits of Orphan Drug designation include a seven-year period of market exclusivity following FDA approval, certain tax credits for clinical testing expenses conducted after orphan designation is received, and reduced regulatory fees.

"RetroSense's Orphan Drug designation from the FDA is a major step forward in the development of RST-001," said Joan Dunbar, associate vice president for technology commercialization at Wayne State University. "Sean Ainsworth has dedicated years of extensive efforts to champion this technology to the next level, and because of his motivation and the extensive research of Dr. Pan and his research team, we are closer to a potential treatment for retinitis pigmentosa."