Mar 30, 2023

Vision Improvements Reported for RP Patients in Phase 2B Clinical Trial of Nanoscope’s Optogenetic Therapy

Eye On the Cure Research News

Most participants with advanced retinitis pigmentosa (RP) in the trial had improved navigation and/or object discrimination in reduced lighting conditions

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Nanoscope Therapeutics has reported that 16 of 18 patients with advanced vision loss from retinitis pigmentosa (RP) in the RESTORE Phase 2b clinical trial for its optogenetic therapy were able to better complete at least one of two tests under low luminance: a simple Y mobility test or a shape recognition test. In the sham (placebo) group, 4 of 9 patients were able to better complete at least one of these tests.

The company also said that 7 of 18 participants in the treatment group had improved best corrected visual acuity (BCVA) of -0.3 LogMAR. In comparison, 1 person in the sham group had improved BCVA. LogMar is a test of visual acuity in which a lower number indicates better visual acuity. For example, a LogMAR of 0.0 is equivalent to 20/20 vision, and a LogMAR of 2.0 is 20/2000.

Baseline vision in the patient eyes to be treated was hand motion or counting fingers — i.e., worse than a LogMAR of 1.9 or 20/1600.

The company says that the improvements in vision are clinically meaningful.

The treatment was also well tolerated with no serious or adverse events in treated patients.

The treatment, MCO-010, is designed for people who have lost most or all of their photoreceptors, the cells that make vision possible. The treatment, a small drop of liquid delivered by an intravitreal injection, uses a human-engineered virus to deliver copies of a Multi-Characteristic Opsin (MCO) gene to bipolar cells — cells that don’t normally sense light but often survive after photoreceptors are lost to advanced retinal disease.

The company says that MCO-010 is enabling the recipient’s bipolar cells to sense ambient light, thereby working as a backup system for lost photoreceptors. The approach doesn’t use goggles or eyewear to enhance the visual information coming into the retinas.