Clinical trial on lead compound RST-001 to assess the safety and, potentially, efficacy of novel gene therapy application of optogenetics in patients with retinitis pigmentosa

DETROIT - A Wayne State University startup company announced today the first successful dosing of a patient in a clinical trial that is a major step forward for patients with vision challenges.

RetroSense Therapeutics LLC, a privately held biopharmaceutical company, successfully dosed their first patient in the first clinical trial to evaluate the safety of RST-001. The study is titled "Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Uniocular Intravitreal RST-001 in Patients with Retinitis Pigmentosa (RP)." RST-001 is designed to restore some vision in patients with RP, a genetic condition that leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina that sense light), resulting in severe vision loss and blindness.

"Successfully dosing our first patient with RST-001 represents a significant milestone in the development program," said Sean Ainsworth, CEO of RetroSense Therapeutics.  "Treating the first human patients should provide key insights into the potential for optogenetics in vision restoration and beyond. We hope to establish the power of using a gene therapy application of optogenetics to improve vision in individuals with currently incurable blindness."

David G. Birch, Ph.D., chief scientist and executive officer of the Retina Foundation of the Southwest  and the principal study investigator, added, "Patients enrolling in the trial understand that we are exploring brand-new territory, but are excited about the possibility of restoring some vision."

The initiation of this clinical study results from the culmination of several years of research and collaboration with researchers at leading institutions, including Zhuo-Hua Pan, Ph.D, at the Ligon Research Center of Vision in Wayne State University's Kresge Eye Institute and Richard Masland, Ph.D., at Massachusetts Eye and Ear Infirmary,  early pioneers in optogenetics for vision restoration.

The study is composed of two parts. An initial dose-ranging study is proposed whereby three dose levels of RST-001 will be studied in three separate groups of adult patients with advanced disease. This first part of the study is aimed at determining a single dose of the experimental agent, which is safe and well tolerated, to further evaluate in a fourth group of patients. The second part is aimed at obtaining additional safety data at the highest tolerated dose and providing important additional clinical data to guide the design of future efficacy studies.

"This is an exciting breakthrough for RetroSense," said Joan Dunbar, associate vice president for technology commercialization at Wayne State University. "The extraordinary efforts of Dr. Pan and his collaborators are now one step closer to finding a way to help restore sight because of the important work that RetroSense is doing. It is a long process, but their vigilance and commitment to restoring sight may one day soon give hope to many people."

In August 2015, the company's Investigational New Drug (IND) application for RST-001 received clearance from the United States Food and Drug Administration (FDA). The clinical trial tracker is NCT02556736. For more information on the clinical trial, please visit clinicaltrials.gov.

RetroSense Therapeutics is developing RST-001 as a first-in-class gene therapy application of optogenetics. Optogenetics refers broadly to means of conferring light sensitivity to cells that were not previously, or natively, light sensitive. By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is working to confer new light sensitivity to the retina, with the expectation of some degree of improved or restored vision for affected patients.

In 2014, the FDA granted Orphan Drug designation for RST-001 based on its development as a treatment of RP, a rare disease that affects an estimated 100,000 people in the United States. As a designated Orphan Drug, RST-001 is eligible for various development incentives under the Orphan Drug Act, including a potential waiver from FDA's application user fees, certain tax incentives and Orphan Drug exclusivity.    

About RetroSense Therapeutics

RetroSense Therapeutics is a privately held biotechnology company developing life-enhancing gene therapies designed to restore vision in patients suffering from blindness due to retinitis pigmentosa and advanced dry age-related macular degeneration. There are currently no FDA-approved drugs to improve or restore vision in patients with these retinal degenerative conditions. The company's approach to using optogenetics in vision restoration is based on pioneering, proprietary research conducted at Wayne State University and Massachusetts General Hospital. RetroSense has worldwide exclusive rights to the relevant intellectual property from both institutions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information about RetroSense, visit retro-sense.com.

About Retina Foundation of the Southwest

The Retina Foundation of the Southwest, located in Dallas, is a nonprofit eye research institute dedicated to preventing and restoring vision loss through innovative research and treatment. For over 30 years, the Retina Foundation has focused on developing treatments for age-related macular degeneration, pediatric eye disorders, and inherited eye diseases. The foundation is comprised of seven laboratories and a staff of scientists who to strive to improve the quality of life for people with retina related eye diseases.

About Wayne State University

Wayne State University is one of the nation's pre-eminent public research universities in an urban setting. Through its multidisciplinary approach to research and education, and its ongoing collaboration with government, industry and other institutions, the university seeks to enhance economic growth and improve the quality of life in the city of Detroit, state of Michigan and throughout the world. For more information about research at Wayne State University, visit research.wayne.edu.