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Light-Activated Therapy Improves Vision in Inherited Disease Trial

Retinal gene therapy

(National Human Genome Research Institute, NIH)

28 Apr. 2023. A clinical trial shows a light-activated protein from an injected gene improves visual abilities in people with advanced retinitis pigmentosa, an inherited eye disorder. Findings of the study assessing the light-activated gene therapy, developed by biotechnology company Nanoscope Therapeutics Inc., were presented on Wednesday at the annual meeting of Association for Research in Vision and Ophthalmology or ARVO in New Orleans.

Nanoscope Therapeutics, based in Dallas, creates treatments for degenerative eye diseases including retinitis pigmentosa, a rare inherited disease traced to a collection of genetic defects that cause a breakdown in cells in the retina, the layer of light-sensitive cells in the back of the eye. Retinitis pigmentosa is a progressive disease that can start in childhood, and advance from lack of night vision into loss of peripheral and eventually central vision leading to total blindness. At present, there are no cures for retinitis pigmentosa, and because no single gene defect is linked to the disorder, conventional gene therapies must address a specific faulty responsible gene.

The Nanoscope Therapeutics technology is a gene therapy, but in this case it delivers a gene that encodes for multi-characteristic opsins or MCOs. Opsins are light-sensitive G-protein-coupled receptor proteins found in the retina and other organs. The company’s lead product, MCO-010, is a single injection into the eye, with a gene coding for MCO proteins activated by ambient light, and delivered to retinal cells with an engineered benign adeno-associated virus, called AAV2. Nanoscope is also developing a non-viral delivered MCO gene therapy. The company says MCO-010 can correct for various inherited retinal conditions, independent of specific genetic defects.

All improved on at least one visual measure

At the ARVO meeting, Nanoscope reported on results from the company’s clinical trial testing MCO-010 in patients with advanced retinitis pigmentosa or RP. The mid-stage trial enrolled 27 patients at six sites in the U.S., randomly assigned two-to-one to receive MCO-010 in one of two dose levels or a sham treatment. The study team is looking primarily for participants’ scores on a standard vision-guided mobility test after one and two years, but also on tests of object recognition and shape discrimination, as well as visual acuity after one and two years.

The researchers report all 18 of the MCO-010 recipients in the trial show improvement after one year in at least one of the three visual ability measures — vision-guided mobility, object recognition and shape discrimination, or visual acuity — compared to five of the nine participants receiving the sham treatment. In addition, from 14 to 17 of the MCO-010 recipients show improved scores on two of the three visual ability indicators, compared to three or four sham treatment recipients. Plus, the company says participants report no serious or severe adverse effects, and rates of any treatment-related adverse effects are similar for both MCO-010 and sham therapy recipients.

Samarendra Mohanty, Nanoscope Therapeutics president and chief scientist, says in a company statement that “significantly more MCO-010 treated patients experienced clinically significant vision improvements.” Mohanty adds, “This randomized controlled trial provides compelling evidence that MCO-010 optogenetic therapy, as a mutation-agnostic treatment, can improve vision in patients with advanced RP.”

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