The National Eye Institute of the National Institutes of Health has awarded $1.5 million to Nanoscope Therapeutics for its ambient light-activatable optogenetic gene therapy.
Bedford-based Nanoscope, a TechFW client, was given the Phase 2b Small Business Research Innovation (SBIR) grant to advance its work in age-related macular degeneration (AMD). The company aims to use its therapy to relieve the millions suffering worldwide with AMD.
The patent-protected ambient light activatable protein is meant for restoration of vision in people with AMD.
“There is no treatment for the diseases that we’re working on,” Nanoscope’s co-founder, president, and chief scientific officer, Samarendra Mohanty, recently told Dallas Innovates. “So there’s a real unmet need that we are trying to fill.”
Mohanty, who launched Nanoscope Technologies in 2009, was joined by CEO Sulagna Bhattacharya as co-founder in 2013. Together, the duo leads the development of a suite of optogenetic therapies that could provide sight to blind individuals suffering from retinal degenerative diseases.
The biotech’s pipeline of products includes therapy for patients with retinitis pigmentosa, Stargardt disease, and other inherited retinal diseases, in addition to AMD.
“Open to going public”—and taking steps to prepare
In June this year, Mohanty and CFO Anthony Togba told Dallas Innovates they are open-minded to the option of going public with their company, and doing due diligence to prepare for that potentiality.
Togba said at the time that the company was “performing readiness activities, undertaking internal processes, and putting in place the structures to be ready to go public,” adding that he anticipates a better sense of the timeline by late Q4 or before.
Inside the MCO therapy
The leading MCO gene therapy (Multi-Characteristic Opsin) aims to re-sensitize the retina to detect low light levels.
The product, MCO-010, is at the moment in a late-stage Phase 2b trial for treating retinitis pigmentosa—a rare inherited degenerative eye disease that causes the breakdown of cells in the retina, leading to severe vision impairment—in the U.S.
MCO-010 has already been granted Orphan Drug designation by the US Food and Drug Administration for retinitis pigmentosa (RP) and Stargardt disease, a genetic eye disorder that also affects the retina.
Both retinal diseases lead to blindness. A Phase 1/2a trial of MCO-010 showed patients blinded by RP experienced clinically meaningful vision restoration, according to Nanoscope.
“Our preliminary data show MCO therapy can be applied in a mutation-independent manner for specific inherited retinal disorders and can serve as a retinal disease-agnostic platform therapy,” Mohanty, PhD, Nanoscope’s president and chief scientific officer, said in a statement.
Mohanty, who was the principal investigator of the recently awarded grant, plans to use the capital to further advance the MCO therapy. Specifically, the team wants to investigate geographic atrophies of the macula (a part of the retina at the back of the eye) for juveniles, as well as adults.
The optogenetic gene therapy uses Nanoscope’s proprietary AAV2 vector to deliver MCO genes into bipolar retinal cells. The MCOs are engineered to be fast and polychromatic, the company says, which can enable vision in various color environments.
The MCOs essentially reprogram bipolar cells to act like photoreceptor cells damaged by the full range of inherited retinal disorders, regardless of any underlying disease-causing gene mutations.
Nanoscope says the therapy involves a single eye injection that is administered in a medical office setting.
CEO Sulagna Bhattacharya said winning the NIH grant is a testament to the innovativeness and clinical significance of Nanoscope’s optogenetic therapy. It underwent a highly competitive review process.
Restoring vision with a single injection
In June, (RP). A single intravitreal injection produced positive results that persisted through one year.
Nanoscope Therapeutics announced in late June that it has received FDA approval of its Investigational New Drug application for its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP). The company’s randomized, double-blind, sham-controlled Phase 2b trial started in locations across the U.S.
Nanoscope closes Series A
Last year, Nanoscope completed an oversubscribed Series A round and announced it was adding Dr. Alvaro Guillem, co-founder of ZS Pharma, as its Chairman of the Board.
The undisclosed amount of funding was used to begin a clinical trial on inherited retinal disorders and other eye-related initiatives as part of Nanoscope’s mission to “give sight back to the blind.”
Guillem has almost 40 years of experience in bringing therapies to market and getting pharmaceutical manufacturing facilities ready for commercial production. His experience includes bringing Mucinex to market as part of a team at Adams Respiratory Therapeutics. The company was later sold to Reckitt Benckiser for $2.3 billion.
He also co-founded and worked as president and CEO at ZS Pharma, a fellow TechFW client that became one of the org’s greatest success stories when it was sold to Astra Zeneca in 2015 for $2.7 billion.
Guillem previously said he’s looking forward to guiding Nanoscope’s transition to the next stage as a leader in ocular gene therapy—noting that its MCO tech “has the potential to impact rare diseases.”
The biotech has received other SBIR awards and National Institutes of Health R01 grants, as well as patents for optical stimulation, gene delivery, and imaging for neural activity monitoring.
Quincy Preston contributed to this story. The story was updated September 15, 2021 at 11 p.m. with additional background information on the company.
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