Nanoscope Doses First Patient in Phase 2 Clinical Trial of its Optogenetic Therapy for Stargardt Disease
Eye On the Cure Research News
The emerging therapy is designed to restore vision for people with advanced retinal degenerative diseases
Nanoscope Therapeutics, a biotechnology company developing gene therapies for retinal degenerative diseases, has dosed the first participant in the Phase 2 STARLIGHT clinical trial of its optogenetic therapy for people with advanced Stargardt disease.
The company has also completed enrollment in the Phase 2b RESTORE clinical trial of its optogenetic therapy for people with advanced retinitis pigmentosa (RP). The company plans to report results from RESTORE in the first quarter of 2023.
Known as MCO-010, the optogenetic therapy 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 the 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 will enable 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.
Stargardt disease is the leading cause of inherited macular degeneration affecting approximately 30,000 people in the US. Usually diagnosed in childhood or young adulthood, the condition causes progressive loss of central vision.
Retinitis pigmentosa is an inherited retinal disease which is also often diagnosed in children or young adults. The condition, affecting about 100,000 people in the US, causes progressive night blindness and constriction of vision.
Both Stargardt disease and retinitis pigmentosa often lead to legal blindness by middle age.