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When Roy began to lose his vision 20 years ago, he still hoped for a cure for his retinitis pigmentosa (RP).
But after being rejected for participation in a clinical trial, he received a call from his doctor saying there was another study he could join, providing much-needed hope.
What is retinitis pigmentosa?
RP is a genetic disease that affects the retina (the layer of photoreceptors cells in the eye), causing it to break down over time, eventually resulting in loss of vision.
Patients with RP begin showing symptoms in their childhood, and among the most common ones is loss of night vision and loss of peripheral vision.
RP affects an estimated 1.5 million people worldwide, and in the U.S., 1 in 4,000 people are known to suffer from hereditary retinal dystrophy, as RP is also known as, according to NIH.
According to a paper published by Investigative Ophthalmology & Visual Science (IOVS) “to evaluate the course of visual acuity and visual field defect” in patients with RP over time, “30% of patients had a visual acuity of less than 20/60, with 12% of patients legally blind.”
Currently, there’s no definitive cure for this disease, however, this biotech company is working hard on new gene therapies that can potentially restore patients’ eyesight.
Bringing back the light of hope
This is the motto of Nanoscope Therapeutics, a Biotechnology Innovation Organization (BIO) member company that is introducing an advanced “gene therapy, using light-sensitive molecules,” used to treat and eventually cure genetic diseases causing visual impairment and blindness.
Currently in its clinical stage, Nanoscope Therapeutics is developing “a proprietary platform technology, employing ambient light-sensitive Multi-Characteristic Opsins (MCO) that has potential to treat a broad range of genetic illnesses,” including RP, dry age-related macular degeneration (AMD), and Stargardt disease.
Phase 2 of their RESTORE clinical trial has already shown significant results and efficacy when using the MCO-010 drug in advanced RP.
The trial tested 27 patients, 18 of which “with severe vision impairment due to RP” were injected with MCO-010, while nine were given a placebo, per Fierce Biotech. Of the 18 patients who received MCO-010, 16 (89%) showed two or more levels of improvement in vision within one year, compared to four of the nine (44%) placebo patients.
At the beginning of the year, Nanoscope Therapeutics’ MCO-010 was granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA), making the company eligible for more frequent regulatory meetings and communications with the FDA.
