An article published by Ophthalmology Times Europe shared that the first patient had been dosed in the Phase 2 STARLIGHT clinical trial evaluating MCO-010 as a potential therapeutic option for Stargardt disease. Altogether, six patients will enroll within this study. Patients will receive an intravitreally injected dose of MCO-010 at 1.2E11gc per eye. Researchers hope to determine the safety, efficacy, and tolerability of this treatment.

According to drug developer Nanoscope Therapeutics, MCO-010, an optogenetic gene therapy using Multi-Characteristic Opsin (MCO):

utilizes a convenient and well-established intraocular injection for delivery of a gene that encodes for the ambient light-sensitive MCO protein into retinal cells. These therapies are intended to enable retinal cells to detect light so that patients with retinitis pigmentosa or Stargardt disease may see again.

Researchers hypothesize that an MCO-based approach could help those who have already lost much of their vision.

Outside of Stargardt disease, Nanoscope Therapeutics is also exploring MCO-010 as a treatment option for patients with retinitis pigmentosa (RP) within the Phase 2b RESTORE study. Data from this study should be available towards the beginning of 2023.

About Stargardt Disease

Stargardt disease is an inherited retinal disorder in which cones and rods within the retina die. ABCA4 gene mutations are the most common cause of Stargardt disease, though other gene mutations have been implicated in its development. Normally, ABCA4 helps to produce a protein which clears vitamin A bypoducts from photoreceptors. However, the mutation means that this process does not work correctly. As rods and cones die, patients begin to experience some vision loss. This often begins in childhood or adolescence though, in some cases, vision loss may not become noticeable until adulthood. Most people do not lose their full vision, but do progress to 20/200 vision. Symptoms can (but do not always) include:

• Vision loss in both eyes
• Loss of some peripheral vision
• Involuntary eye movements
• Sensitivity to bright light
• Color blindness
• Gray, black, or hazy spots in center of vision
• Difficulty adjusting when moving from light to dark environments