Nanoscope Therapeutics Advances Novel Gene Therapy for Retinitis Pigmentosa with FDA Rolling Submission
[City, State] – July 14, 2025 – Nanoscope Therapeutics Inc., a late-stage biotechnology company pioneering novel gene therapies for degenerative retinal diseases, today announced a significant milestone in its pursuit of treating retinitis pigmentosa (RP). The company has initiated a rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for MCO-010, a groundbreaking gene-agnostic therapy designed to address this debilitating inherited retinal disorder.
This marks a pivotal moment for patients and the broader ophthalmology community, as MCO-010 represents the first therapy of its kind to target a broad spectrum of RP patients, irrespective of the specific gene mutation responsible for their condition. Retinitis pigmentosa is a group of inherited diseases characterized by progressive vision loss, often leading to blindness, affecting millions worldwide. Currently, treatment options are limited and often specific to particular genetic subtypes.
The rolling submission process allows Nanoscope Therapeutics to submit portions of its BLA to the FDA as they are completed, rather than waiting for the entire dossier. This expedited approach reflects the FDA’s commitment to facilitating the review of promising therapies for serious conditions with unmet medical needs.
MCO-010 utilizes Nanoscope’s proprietary Multi-Characteristic Optic (MCO) gene therapy platform, which aims to restore vision by introducing a gene that encodes a light-sensitive protein into retinal cells. What sets MCO-010 apart is its unique gene-agnostic mechanism, meaning it does not require prior genetic diagnosis of the specific RP mutation. This broad applicability holds the potential to benefit a significantly larger patient population than gene-specific therapies.
The company’s decision to initiate the rolling submission is supported by compelling clinical trial data from its Phase 2b study. These trials have demonstrated statistically significant and clinically meaningful improvements in visual acuity and visual field in patients treated with MCO-010. The therapy has shown a favorable safety profile, further bolstering confidence in its therapeutic potential.
“We are incredibly proud to reach this important juncture in our development of MCO-010,” said [Name and Title of a Nanoscope Therapeutics spokesperson, e.g., Dr. Susan Chen, CEO of Nanoscope Therapeutics]. “This rolling submission to the FDA is a testament to the hard work of our dedicated team and the promising results we have seen in our clinical trials. We believe MCO-010 has the potential to be a transformative treatment for individuals living with retinitis pigmentosa, offering hope for restoring their sight and improving their quality of life.”
Nanoscope Therapeutics remains committed to collaborating closely with the FDA throughout the review process and looks forward to the potential availability of MCO-010 for patients. The advancement of this gene-agnostic therapy represents a significant step forward in the fight against inherited retinal diseases and underscores the innovative spirit driving progress in gene therapy.
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PR Newswire Policy Public Interest published ‘Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa’ at 2025-07-14 11:00. Please write a detailed article about this news in a polite tone with relevant information. Please reply in English with the article only.