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NIH Funds Light-Aided Gene Therapy for Eye Disease

Eye measurements

(Sakuie, Pixabay)

Update. 2 June 2020. The NIH grant referenced in the text is incorrect. Nanoscope Technologies says it received a later, more comprehensive and multi-year award to advance a light-based non-viral gene delivery to treat retinal disorders. We will update the link when NIH publishes the grant document.

29 May 2020. A company creating a gene therapy for age-related macular degeneration activated with near-infrared rays received an NIH grant to advance its technology. National Eye Institute, part of National Institutes of Health, awarded Nanoscope Technologies in Arlington, Texas $77,600 for the six-month project.

Nanoscope Technologies develops gene therapies activated by light waves to treat degenerative eye diseases such as age-related macular degeneration, or AMD, a common disorder and the leading cause of vision loss in people over the age of 50. The condition occurs in the macula, the part of the retina with light-sensing cells at the back of the eye. When the macula is damaged, the retina’s ability to turn the sensed light into electrical signals for the brain is impaired.

Current therapies for AMD, says the company, are retinal implants requiring invasive surgery or gene therapies activated by high-intensity lasers, or treatments to slow progression rather than cure the disease. With Nanoscope’s technique, gold nanoscale rods are coated with light-sensitive proteins called opsins and injected into the center of the eye. Once the gold nanorods and proteins bind to the damaged retinal cells, a second injection adds the therapeutic genes carried by benign viruses, like most other gene therapies. Low-level near-infrared light waves, not visible in ambient light, are then beamed into the eye, where the gold nanorods concentrate the beams to heat the opsins and activate retinal cells that take up therapeutic genes.

Nanoscope Technologies successfully demonstrated the process with lab mice, reported last month in the journal Molecular Therapy: Methods & Clinical Development. The company says the technology can deliver gene therapies more precisely to damaged regions of the retina, which most other gene therapies cannot. In addition, current gene therapies can invoke an unwanted immune response in some cases. In the journal article, the researchers say they detected no toxicity to the eyes of test mice, nor did they find unwanted damage to their eyes or release of immune-system enzymes, and only minimal quantities of the delivery virus in other organs.

“Not requiring any external stimulation device and sensitivity to broad range of colors,” says Sulagna Bhattacharya, CEO of Nanoscope Technologies in a company statement released through EurekAlert, “makes our approach unique as there is least chance of phototoxicity and damage to retina.”

The new NIH award funds further development of the technique the company calls viral vector carried multi-characteristic opsin, or vMCO, as a treatment for the “dry” type of AMD, not involving blood vessel leakage in the eye, affecting 85 to 90 percent of people with the condition. Nanoscope plans to test vMCO treatments further in mice to determine its ability to restore long-term vision, as well as stability and safety. And company also plans to test the treatments in dogs induced with dry AMD. Nanoscope says if it’s successful, the company will be in a position to test vMCO in monkeys, and seek a partnership to advance the treatment to clinical trials.

The grant to Nanoscope is made under NIH’s Small Business Innovation Research, or SBIR, program that sets aside a portion of its overall research funding for small U.S.-based companies with science-based products. NIH says it invests more than $1 billion in SBIR and related Small Business Technology Transfer awards.

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