Bedford biotech’s gene therapy improves sight for 11 patients with a rare genetic retina disorder
A clinical study of Bedford-based Nanoscope Therapeutics’ one-size-fits-all gene therapy for a rare genetic retina disorder meaningfully improved sight for 11 blind patients, according to the company.
The proprietary therapy delivers multi-characteristic opsin — an optically activated protein similar to a molecular solar cell — to retinal cells, and can be used to treat an array of diseases.
Nanoscope said it now believes its trial results are long-lasting, possibly improving vision for the patients’ entire lives. The optimism is based on observing the 11 patients’ progress for 52 weeks following the trial.
Classic gene therapy can only treat one disease at a time — and there are hundreds of genes responsible for causing blindness. Optogenetic gene therapy can target hundreds of different diseases, including retinitis pigmentosa, age-related macular degeneration and Stargardt disease, all with a single product.
The therapy requires no retinal implants, invasive surgeries or goggles. Instead, the gene therapy is administered in a doctor’s office by a single injection through the eye.
Samarendra Mohanty, co-founder and president of Nanoscope Therapeutics, explained that genes are delivered to the retina, “not photoreceptors, because in this instance photoreceptors like rods and cones are already destroyed.”
The gene therapy transforms retinal cells into new photoreceptors.
“It’s like installing a solar panel on your house, on top of your roof … you get [a] current generated for using in a home,” Mohanty said. “[It’s the] same way the eye needs the current to send to [the] brain.”
The year-long study began in 2018. Patients who had no vision at all were selected to participate in 2019. Initial results from the study were reported by the company last year, revealing that all patients experienced improved visual acuity at 16 weeks. All also saw a greater than 90% improvement in accurately describing shapes.
“They used to come with their family member or chaperone,” said Sulagna Bhattacharya, co-founder and CEO, about patients in the clinical trial. By 16 weeks, “people start[ed] coming all by themselves. Of course, not driving the car, but taking public transportation,” she said.
Patients were able to take over daily chores and activities that they could not do alone prior to the gene therapy treatment, the company said.
Nanoscope Therapeutics can now begin studying its gene therapy through a randomized test, “which has potential to become pivotal,” Mohanty said. The company’s goal is to make the treatment available globally at an affordable price.
Current gene therapies can cost $3 million or more, Bhattacharya said. “We want to make sure that regular people can afford it because it’s one drug applicable for so many patients,” she said.
The drug’s wide range of applications means it can be mass-produced, increasing affordability, Bhattacharya said.
Nanoscope Therapeutics is a spin-off from Nanoscope Technologies, a Bedford company founded in 2009 that has gotten backing from the National Institutes of Health. Nanoscope Therapeutics received a $2 million grant from the National Eye Institute in June 2020, and landed an undisclosed funding round a month later.