Nanoscope Therapeutics is developing optogenetic gene therapies using Multi-Characteristic Opsin (MCO) to re-sensitize the retina to detect low light levels. These therapies have the potential to restore vision in millions of visually impaired individuals suffering from retinal degenerative diseases, including Retinitis Pigmentosa, Stargardt Disease and Dry Age-related Macular Degeneration (AMD).
MCO-010 for RP and Stargardt Disease
Our lead product, MCO-010, is in clinical development for retinitis pigmentosa (RP) and Stargardt disease, which are two rare retinal diseases that cause blindness. MCO-010 has been granted Orphan Drug and Fast Track designation by the US Food and Drug Administration for both diseases.
MCOs have potential to address all forms of retinal degeneration and are applicable for patients suffering from complete or partial damage to the retina.
Nanoscope’s MCO-010 gene therapy 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.
How our proprietary technology differs from other gene therapies
Our therapy focuses on treating blindness regardless of the underlying genetic mutation
It is an ambient-light sensitive therapy in which a polychromatic Opsin is delivered to retinal cells enabling vision in different color environments
Simple and convenient office-based procedure, invasive ocular surgery not required
No external device or stimulation is required, eliminating risk of photo-toxicity
Nano-enhanced Optical Delivery – NOD
Imagine delivering new genetic material to retinal cells with no viruses at all. The company has strategic partnerships to create a unique non-viral alternative gene delivery platform designed to improve targeting precision with less immunogenicity. The non-viral technology involves a near-infrared laser-based spatially targeted nano-enhanced delivery (NOD) of therapeutic genes. This technology can be used to deliver competent genes to correct the underlying genetic defects that lead to vision loss, as well as optical gene therapies.