Nanoscope
Nanoscope co-founder and CEO Sulagna Bhattacharya
Nanoscope Therapeutics Inc., a biotechnology company focused on developing gene therapies for vision loss, said Thursday that it planned to start the process of applying for approval to sell its gene therapy after receiving positive regulatory feedback from the FDA.
The company’s experimental treatment, MCO-010, treats a condition called Retinitis Pigmentosa, a group of rare eye diseases that affect the retina and is a leading cause of blindness in working-age adults.
“Preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP.” said Allen C. Ho, director of retina research and co-director of the Retina Service at Wills Eye Hospital, and chief medical adviser of Nanoscope, in a statement. “Feedback from the FDA has informed Nanoscope on its BLA (Biologics License Application) submission plan, thereby presenting the potential for a viable restorative option for patients whose vision has been lost to the array of progressive retinal degenerations that comprise RP.”
Severe vision loss is often the outcome for individuals diagnosed with RP, ultimately leading to blindness. The current treatment paradigm focuses on vision rehabilitation, but patients still endure a lifetime of progressive vision impairment, thereby dramatically impacting quality of life.
MCO-010 recently showed promising results in a U.S. clinical trial. The trial involved multiple centers and used a rigorous approach to compare the treatment against a placebo. The therapy has also earned special recognition from the FDA, receiving "Fast Track" status to speed up the review process, as well as "orphan drug" designation, which encourages the development of treatments for rare diseases like retinitis pigmentosa and Stargardt disease.
Nanoscope Therapeutics, which has offices in Bedford and Dallas, is a former client of TechFW.
“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team,” said Sulagna Bhattacharya, Nanoscope co-founder and CEO. “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”