Nanoscope Therapeutics Plans to Submit BLA for Gene Therapy to Treat RP

Nanoscope Therapeutics has announced a successful meeting with the FDA regarding its lead clinical program, MCO-010, for the treatment of retinitis pigmentosa (RP). Following regulatory feedback from the FDA, the company plans to initiate a rolling submission of its biologics license application (BLA) for MCO-010 in Q1 2025.

During the meeting, the FDA acknowledged Nanoscope's proposed strategy for advancing its BLA submission for MCO-010, a novel gene therapy designed to treat severe vision loss caused by RP. Nanoscope’s Fast Track Designation for MCO-010 allows the company to proceed with a rolling submission, which enables sections of the BLA to be reviewed as they are completed rather than waiting for the full application.

"Preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP," Allen C. Ho, MD, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor for Nanoscope, said in a company news release. "The FDA’s feedback has been essential in shaping our BLA submission strategy, providing hope for a potential restorative treatment for RP patients."

In addition to advancing the BLA for MCO-010, Nanoscope is also making progress in its program for Stargardt macular degeneration, which recently concluded a successful end-of-phase 2 meeting with the FDA and is now moving into a phase 3 registrational trial. 

The improvements in visual acuity observed over three years in our RESTORE and REMAIN studies underscore our commitment to delivering transformative therapies to patients," said Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope.