March 22, 2016
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RetroSense Therapeutics doses first patient in phase 1/2 clinical study of RST-001

The first patient has been dosed in a phase 1/2 clinical study of RST-001, a gene therapy designed to restore vision in patients with retinitis pigmentosa, according to a press release from RetroSense Therapeutics.

In the first part of the study, three groups of patients will be administered three dose levels of RST-001 to determine a single dose that is safe and well-tolerated, which will be then administered to a fourth group of patients. The second part of the study will obtain additional safety data at the highest tolerated dose and provide additional clinical data to guide the design of future efficacy studies.

“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 using a gene therapy application of optogenetics to improve vision in individuals with currently incurable blindness,” Sean Ainsworth, CEO of RetroSense, said in the release.