Nanoscope Therapeutics announced a successful End-of-Phase 2 meeting with the FDA, securing plans to advance its gene therapy, MCO-010, to a Phase 3 trial for the treatment of Stargardt macular degeneration.

The therapy, which received FDA fast track and orphan drug designations, is being developed to restore vision in patients suffering from severe vision loss. “The current paradigm for managing Stargardt disease is one of diagnosis, followed by vision rehabilitation and a lifetime sentence of declining vision to severe blindness,” noted Samuel Barone, MD, chief medical officer, in a press release. However, MCO-010 offers a mutation-agnostic approach to treatment, making it a potential option for many patients who have vision loss.

Researchers will randomize Stargardt macular degeneration patients 1:1 to receive either an MCO-010 intravitreal injection or a sham injection. The primary efficacy endpoint will be a change in best-corrected visual acuity from baseline to week 52, assessed using ETDRS (Early Treatment of Diabetic Retinopathy Study) letters. Secondary endpoints will include the multi-luminance shape discrimination test. The trial will include legally blind patients with vision worse than 20/200 and those as young as 12 years old.