Nanoscope Therapeutics is on a mission to restore sight to those with age-related macular degeneration. And early trials of its therapy show it can do just that.
To help fuel its Phase 2b trials, the Bedford-based biotech startup announced landing a $1.5 million Small Business Research Innovation grant from the National Eye Institute as it looks to bring its optogenetic gene therapy to market.
“Our preliminary data show Multi-Characteristic Opsin ... can be applied in a mutation-independent manner for specific inherited retinal disorders and can serve as a retinal disease-agnostic platform therapy,” said Samarendra Mohanty, principal investigator of the recently awarded NIH grant and Nanoscope's president and chief scientific officer, in a statement. “With this grant, we can accelerate advancement of our MCO therapy for geographic atrophies of the macula for juveniles as well as adults.”
Nanoscope Therapeutics is a part of research and development and commercialization company Nanoscope Technologies, which also includes Nanoscope Diagnostics. For its part, Nanoscope Therapeutics uses an ambient light-activated protein injected into retinal cells that act similar to photoreceptors to help restore sight in patients.
The National Eye Institute, a part of the National Institutes of Health, previously awarded Nanoscope a $2 million grant for its work. It’s also raised equity funding, reporting a $12.8 million raise last July. At the time, the company was looking to raise a total of $16 million.
Nanoscope’s lead gene therapy, called MCO-010, focuses on treating retinitis pigmentosa. In January, it received an orphan drug designation from the FDA. In June, the company reported that all 11 patients in a 52-week Phase 1/2a clinical study showed “meaningful functional improvement.” That finding allowed the 12-person Nanoscope team to kick off Phase 2b clinical trials in July.
The company was launched in 2009 by Mohanty. It brought on Sulagna Bhattacharya as CEO in 2013. This year, while announcing a $2 million Series A from undisclosed investors, Nanoscope added Dr. Alvaro Guillem as its new board chairman. He is best known for serving as president and CEO of Dallas biopharmaceutical company ZS Pharma, which sold to Astra Zeneca for $2.7 billion in 2015.
The recent progress of Nanoscope’s therapy potentially shines a light on the company’s future plans. Earlier this year, when the company received FDA approval for its Phase 2b trials, CFO Anthony Togba told Dallas Innovates that Nanoscope was “putting in place the structures” to potentially go public and would have a more solid idea of the next steps by Q4 or earlier.
“The technology is poised to make long-lasting impact on patients suffering from retinal degeneration,” Mohanty previously said. “Combined with the track record of the highly skilled executive team and advisors, Nanoscope is going to drive the future market on ocular gene therapy.”
