Nanoscope eyes market after gene therapy restores vision for patients with retinal disease

Nanoscope Therapeutics is back with more data assessing its gene therapy that uses light-sensitive molecules to treat retinal disease. With data demonstrating vision improvement in hand, the biotech is now planning to submit the therapy for FDA approval later this year.

The two-year phase 2b study tested MCO-010 among patients with permanent and severe vision loss from advanced retinitis pigmentosa (RP), a term that encapsulates a group of rare genetic eye disorders. The new data build off early findings presented a year ago that found that 16 of the 18  (89%) patients injected with MCO-010 demonstrated an improvement of two or more luminance levels across two visual tests at 12 months, compared to four of the nine (44%) patients who received placebo.    

Now, the Texas-based biotech has shared that the trial, dubbed RESTORE, also demonstrated a statistically significant improvement in best-corrected visual acuity (BCVA) after 52 weeks for 18 patients split among a high-dose and low-dose group, compared to nine participants in the control arm. The measure was the trial’s primary endpoint.

“We observed significant vision restoration in many patients with severe vision loss, including those who were completely blind,” David Boyer, M.D., an investigator in the trial and ophthalmology professor at the University of Southern California Keck School of Medicine, said in a March 26 release. “Many patients treated with MCO-010 derived a clinically meaningful benefit measurable on the primary visual function test, and this effect was confirmed by a parallel improvement in functional vision assessments.”

A single intravitreal injection of MCO-010 also showed statistically significant BCVA improvement at Week 76 among the high-dose group. However, improvement in the low-dose arm was not statistically significant at the same extended time stamp.

The gene therapy was generally well tolerated, and no treatment-related serious or severe adverse events were reported, according to Nanoscope.   

With new data in hand, Nanoscope intends to apply for FDA approval in the second half of 2024. The biotech plans to use the higher dose of MCO-010 (1.2E11gc/eye) as the commercial dose.

Currently, there is no available cure for RP. MCO-010, which has already snagged fast-track and orphan tags from the FDA, is an ambient-light activatable multicharacteristic opsin optogenetic therapy. Unlike other eye-targeted gene therapies, which take aim at a specific gene mutation in the outer retinal cells, Nanoscope’s therapy is designed to target inner retinal neurons to restore vision lost as RP progresses.

Nanoscope is also testing out the mutation-agnostic gene therapy in Stargardt disease, another rare eye disease.