Stargardt disease treatment gets FDA fast track

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stargardt disease blindness

Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation (FTD) for MCO-010. 

MCO-010 is an ambient-light activatable multi-characteristic opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for the treatment of Stargardt disease to improve visual function.

“We are pleased to receive the FDA’s decision to grant fast track designation for the second indication of our vision restoring therapy, and we look forward to sharing six-month data from the phase 2 STARLIGHT clinical trial of MCO-010 therapy in Stargardt patients this quarter,” said Sulagna Bhattacharya, CEO of Nanoscope Therapeutics. 

“Receiving fast track designation reinforces the urgency of our mission to find an effective solution for Stargardt patients and is an acknowledgement of MCO-010’s potential to address this unmet medical need.”

Study details of Nanoscope Therapeutics‘ MCO-010

In September 2022, enrollment was completed in the phase 2 open-label STARLIGHT clinical trial of MCO-010 in six patients with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease. In this study, all subjects received the same single intravitreal dose of 1.2E11gc/eye of MCO-010. Six-month data from the STARLIGHT trial are expected in Q1 2023.

The FDA designation is intended to facilitate the development and expedite the review of novel therapies to treat serious conditions for which there is unmet medical need. With the designation, Nanoscope Therapeutics is eligible for more frequent regulatory meetings and communications with the FDA.

In February 2022, enrollment was completed in the phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial of MCO-010. Top line data from the RESTORE trial are also expected in Q1 2023.

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