First patient receives innovative gene therapy eye treatment from TechFW client

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Bedford-based Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for the treatment of degenerative diseases of the retina, administered optogenetic monotherapy to the first patient in its phase 2b clinical trial MCO-010 can be activated by ambient light to restore vision in patients with retinitis pigmentosa (RP).

Nanoscope, a client of TechFW, an incubator / accelerator in Fort Worth, made the announcement on July 19.

“I would like to congratulate the Nanoscope team, our clinical partners and the patients participating in the trial. The results of our Phase 2b trial may serve as the basis for the FDA’s consideration of expedited approval of MCO-010 for the treatment of RA. We look forward to working with the FDA and other regulatory agencies so that we can bring MCO-010 to RA patients. said Sulagna Bhattacharya, CEO of Nanoscope.

According to the World Health Organization, more than two billion people suffer from some form of visual impairment, including blindness, and the Centers for Disease Control and Prevention puts the economic costs in the United States at more than 51 billions of dollars. “If our optogenetic gene monotherapy is ultimately approved, it could provide relief for patients with several types of degenerative retinal disease,” Bhattacharya said.

Nasoscope Optogenetic Therapy Uses Intravitreal Solution AAV2 vector to deliver multi-characteristic opsin (MCO) genes into retinal cells, where they express polychromatic opsins allowing vision in different color environments. The therapy can be administered in a doctor’s office without the need for glasses or other procedures. Based on preliminary evidence from the company’s Phase 1 / 2a study, MCO-010 is potentially applicable not only for RP, but also for other degenerative diseases of the eye, regardless of mutations. genetic. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt’s disease.

Phase 2b will include 27 patients with advanced RP in a multi-center, randomized, double-blind, sham-controlled trial in the United States. in improving the vision and visual function of patients. The study is expected to complete recruitment by the end of 2021, with 12-month results available by the end of 2022.

“This is an important milestone for the Nanoscope and the field of ophthalmic research. We remain true to our commitment to bring safe and differentiated optogenetic monotherapy to the ophthalmic community, ”said Dr Mohamed Genead, Director of Development and Medical Director of Nanoscope. “As a result, we are excited about our MCO-010 development program, which we believe holds great potential for the treatment of a wide range of retinal diseases. “

A completed phase 1 / 2a open label study of MCO-010 in 11 patients with RP demonstrated that ambient light-activatable optogenetic monotherapy was well tolerated, with improved quality of life consistent with significant functional vision and improvement. of visual function in patients with advanced RP.

“We are excited about the tremendous interest in RP patients, caregivers and the clinical community. Our team is working hard to further expand the OLS platform for other retinal degenerations including Stargardt disease, dry age-related macular degeneration (AMD) and others, ”said Samarendra Mohanty, Ph .D., President and Scientific Director of Nanoscope. .


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