Ann Arbor Company Begins Clinical Trial to Treat Degenerative Eye Disease Condition


Ann Arbor-based RetroSense Therapeutics LLC, a biopharmaceutical company, has completed its first successful medicating of a patient in a clinical trial for its new drug for the treatment of retinitis pigmentosa, an inherited, degenerative eye disease that causes severe vision impairment. The company’s therapy is based off an approach pioneered at Wayne State University in Detroit.

“Successfully dosing our first patient with the RST-001 (drug) represents a significant milestone in the development program,” says 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.”

Ainsworth says RST-001 is designed to restore some vision in patients with retinitis pigments, which affects an estimated 100,000 people in the United States.

The study is broken down into two parts. The initial part of the study will look at three different dose levels of RST-001 in three separate groups of adult patients with the disease. Ainsworth says the second part is aimed at obtaining additional safety data at the highest tolerated dose.

He says the trial results came from several years of research by Zhuo-Hua Pan at the Ligon Research Center of Vision in Wayne State University’s Kresge Eye Institute and Richard Masland at Massachusetts Eye and Ear Infirmary.

“The extraordinary efforts of 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,” says Joan Dunbar, associate vice president for technology commercialization at Wayne State. “It’s a long process, but their vigilance and commitment to restoring sight may one day soon give hope to many people.”

RetroSense received approval from the U.S. Food and Drug Administration to proceed with human clinical trials in August.

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).​