The Nanoscope team has developed Multi-Characteristic Opsins (MCOs) to sensitize cells toward low level of white light so that vision is improved at ambient room light.
"Not requiring any external stimulation device and sensitivity to broad range of colors makes our approach unique as there is least chance of phototoxicity and damage to retina", said Sulagna Bhattacharya, CEO of Nanoscope Therapeutics Inc. On the other hand, gene delivery by commonly used viral transfection may lead to unexpected inflammatory and immunological responses. Immune rejection poses a significant challenge for redosing the subjects with same or other therapeutic genes delivered using viral vectors. Further challenges remain in localized delivery of therapeutic genes such as MCO to targeted geographic atrophic area(s) in dry-AMD patients and size limit of the genes that can be packaged by the most-commonly used safe AAV vector.
To minimize collateral damage while inserting therapeutic genes, Nanoscope team uses a low power near-infrared (NIR) laser beam whose intensity is locally enhanced inside the organ of interest by use of gold nanoparticles bound to the targeted cell membranes. Nanoscope researchers have demonstrated the use of different laser wavelengths and parameters to deliver various therapeutic genes targeting multiple cell types. The gene delivery laser is integrated with different imaging devices such as fundoscope or an optical coherence tomography (OCT) system for real time image guidance to select the region(s) of interest and deliver the therapeutic molecule(s). Further, variety of lasers can be multiplexed in the delivery platform to target multiple cell types simultaneously. Nanoscope is also a recipient of Audacious Goal Initiative Grant from NIH to advance ultrafast laser based gene delivery to the retina. "Our optogenetic platform and laser delivery technologies are synergistic and will allow treatment for both fully and partially-degenerated retina", said Samarendra Mohanty, Ph.D., Principal Investigator of the grant and Chief Scientific Officer at Nanoscope Technologies. "This technology provides a unique therapeutic option for treating dry-AMD patients for which there is no approved therapy", said Vittorio Porciatti, D.Sc., Head of Research at Bascom Palmer Eye Institute.
Nanoscope is focused on advancing cell-gene therapy through various patented key platform technologies. "Through laser delivery, we have now overcome the limitation on size of the therapeutic genes that can be delivered", said Subrata Batabyal, Ph.D., co-investigator of the recently awarded grant. The Nanoscope team has integrated electrophysiology instrumentation with the OCT-guided laser gene delivery platform and Nanoscope Instruments is commercializing cutting-edge platform technologies for three-dimensional image guided delivery of therapeutic molecules to the diseased tissue regions. "OCT guided structural imaging integrated with physiology monitoring is allowing us to evaluate progression of retinal degeneration and efficacy of laser-based therapeutic gene delivery", said Sanghoon Kim, Ph.D., Senior Project Engineer at Nanoscope. "We discover the needs of researchers today and develop products that are multi-modal allowing researchers the capability to see, measure, functionalize and evaluate tissues" said Madhu Rao, Ph.D., VP of Sales & Marketing at Nanoscope Instruments, Inc.
Nanoscope Technologies, LLC, founded in 2009 by Samarendra Mohanty, PhD., has received several SBIR awards, National Institutes of Health (NIH) R01 grants, and patents which focus on optical stimulation, gene delivery, and imaging for neural activity monitoring.