Scientists develop novel opsin and delivery platform for blind patients
According to the World Health Organization, approximately 285 million people are estimated to be visually impaired worldwide. Age-related macular degeneration (AMD) is a major cause of vision loss in aging populations. The number of affected patients is expected to double by 2050. The degree of visual loss increases with age and is a major concern as demography shifts toward a more elderly population. Most of the current clinical treatments are focused on slowing down the progression of these retinal diseases, since there is neither a cure nor a therapy that can totally stop the degeneration.
Nanoscope Scientists have developed a highly photosensitive Virus carrying Multi-Characteristics Opsin (VMCO) that allows stimulation of retinal cells for restoring vision in patients with AMD and genetic retinal diseases who have lost their photoreceptors. NanoScope Technologies has been awarded a National Institutes of Health (NIH) Small Business Innovation Research (SBIR) Grant from the National Eye Institute (NEI) to develop and conduct preclinical evaluation of VMCO.
"We have very promising preclinical results in the use of VMCO for vision restoration " said Weldon Wright, Chief Medical Officer of Nanoscope Technologies and Principal Investigator of the NEI SBIR grant.
Currently employed methods for delivery of therapeutic opsins to retinal cells involve a viral vector. While this technique has proven very safe and effective, it has the disadvantage that all areas of the retina are affected. Since many of the disease entities to be treated have various types of spatial localization with some areas of the retina affected and other area spared, it would be very advantageous to more accurately treat only the affected areas.
Nanoscope Technologies has been awarded recently two additional NIH grants to explore two localized methods of delivery. The first method uses a femtosecond laser to insert Multi-Characteristics Opsin (MCO) into individual cells. This technique offers the ultimate cellular specificity, but has the disadvantage of only being able to treat one cell at a time. The second technique is called Nano-enhanced Optical Delivery (NOD) in which MCO is mixed with gold nanorods and entire areas of the retina can be selectively transfected by exposing to a continuous wave laser beam.
"Since the gold nanorods have negligible toxicity in the eye, they are ideal for enhancing the light effect used in NOD therapy. In addition, continuous wave light sources used for NOD are compact, easy-to-use, and have significant potential for clinical ophthalmological use", said Samarendra Mohanty, Chief Scientific Officer of Nanoscope Technologies and Principal Investigator/co-investigator on the NIH grants.
The NanoScope research team and company are dedicated to the goal of translating VMCO and NOD platform technologies into treatments for retinal and other neurological diseases.
Contact Ms. Sulagna Bhattacharya at [email protected] for further information.