Researchers at Bedford-based Nanoscope Technologies LLC have received a multi-year, $2-million plus Small Business Innovation Research Award from the National Eye Institute (NEI), a division of the National Institutes of Health (NIH), for its gene therapy treatment.
Nanoscope’s research is advancing the development of a therapeutic gene and delivery platform for treating degenerative eye diseases, including dry age-related, macular degeneration (dry-AMD). The projected deliverable is a non-viral, spatially-targeted gene delivery platform for AMD therapy. It would restore visual response with targeted delivery of ambient-light, activatable opsin into the retina.
“Our opto-genetic platform and laser delivery technologies are synergistic and will allow treatment for fully- and partially-degenerated retina,” said Samarendra Mohanty, Ph.D., Principal Investigator of the grant and Chief Scientific Officer at Nanoscope.
“This technology provides a unique therapeutic option for treating dry-AMD patients for which there is no approved therapy,” said Vittorio Porciatti, Head of Research at the Bascom Palmer Eye Institute in south Florida.
The Nanoscope team 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 less chance of phototoxicity and damage to the retina,” said Nanoscope CEO Sulagna Bhattacharya.
Gene delivery by commonly used viral transfection may lead to unexpected inflammatory and immunological responses. To minimize collateral damage while inserting therapeutic genes, Nanoscope’s process uses a low-power, near-infrared (NIR) laser beam whose intensity is locally enhanced by use of gold nanoparticles bound to the targeted cell membranes. This platform technology is being explored for delivery of genes for vaccination against diseases such as COVID-19.
Nanoscope is focused on advancing cell-gene therapy through various patented key platform technologies. Founded in 2009 by Mohanty, it has received multiple SBIR awards, National Institutes of Health R01 grants, and patents that focus on optical stimulation, gene delivery and imaging for neural activity monitoring.