Low Dose Cohort Safely Dosed In Retinitis Pigmentosa Initial Clinical Trial

RetroSense Therapeutics LLC announced today that the low dose cohort of patients safely dosed in the clinical trial for Retinitis Pigmentosa. This means the clinical trial can now proceed to mid dose cohort.


Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Uniocular Intravitreal RST-001 in Patients With Retinitis Pigmentosa (RP).

Retinitis Pigmentosa Low Dose Cohort


RST-001 is a gene therapy which is given as an injection into the eye and delivers a gene encoding a photo switch, channelrhodopsin-2, (optogenetics) to cells in the retina of the eye. When expressed, the channelrhodopsin-2 protein can depolarize in response to light thus generating a signal that is transmitted to the brain.

RST-001 is developed to restore some vision to patients with Retinitis Pigmentosa, which is a group of genetic conditions that lead to the progressive degeneration of rod and cone photoreceptors. (cells found in the retina that sense light are photoreceptors. The diseases results in severe vision loss and blindness.

Sean Ainsworth, CEO of RetroSense Therapeutics, stated,

We are quite pleased with the safety profile we have observed in this low dose cohort of patients. 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.

The success of the first cohort has created quite a hope for Retinitis Pigmentosa clinical trial. David G. Birch, Ph.D., scientific director of the Retina Foundation of the Southwest and principal investigator of the study, stated the success,

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 good news is that there were no signs of inflammation or ocular adverse events in the patients, and biological activity has been confirmed.

Source> BusinessWire

More info> RetroSense Therapautics website

Clinical Trial>

GW Editorial Staff

Editorial Staff at Generic Whiz.

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