RetroSense Therapeutics Completes Low Dose Cohort in Clinical Trial of Novel Gene Therapy Application of Optogenetics

Proceeding to mid dose cohort in assessment of safety and, potentially efficacy, of lead product RST-01 in patients with retinitis pigmentosa

ANN ARBOR, MI, USA I August 10, 2016 I RetroSense Therapeutics LLC, a privately-held biopharmaceutical company, today announced that the low dose cohort of patients has been safely dosed in the initial clinical trial to evaluate the safety of RST-001. No inflammation or ocular adverse events were seen in any of these patients, and biological activity has been confirmed.

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 being developed with the goal of restoring some vision to patients with RP, a group of genetic conditions that lead to the progressive degeneration of rod and cone photoreceptors (cells found in the retina that sense light), resulting in severe vision loss and blindness. RST-001 is genotype independent, and is expected to have application irrespective of which genetic defect causes the photoreceptors to degenerate.

“We are quite pleased with the safety profile we have observed in this low dose cohort of patients,” said Sean Ainsworth, CEO of RetroSense Therapeutics. “Going into our mid dose cohort with the strong safety profile we have seen early on suggests potential for a higher therapeutic index – or, potentially better safety and efficacy outcomes – for RST-001.”

David G. Birch, Ph.D., scientific director of the Retina Foundation of the Southwest and principal investigator of the study, added, “We are excited to progress to the second cohort in the initial dose ranging portion of the trial. There have been so few options to treat RP and due to the severity of the progressive nature of this disease, patients are excited to learn about a new approach which might improve sight.”

The Company’s Investigational New Drug (IND) application for RST-001 received clearance to initiate the clinical trial from the United States Food and Drug Administration (FDA) in late 2015. The study is composed of two parts. The initial dose-ranging study will examine three dose levels of RST-001 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 that is safe and well tolerated, to further evaluate in a fourth group of patients. The second part of the study 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. More information is available at The clinical trial tracker is NCT02556736.

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.

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 on Retro-Sense, please visit

SOURCE: RetroSense Therapeutics

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