Nanoscope Therapeutics Takes Major Step for Gene-Agnostic Therapy in Retinitis Pigmentosa

Nanoscope Therapeutics Launches Submission for Revolutionary Gene-Agnostic Therapy

Nanoscope Therapeutics Inc., a pioneering biotechnology firm, has made a significant move in the field of ocular therapies by beginning a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its flagship investigational treatment, MCO-010. This marks a critical milestone as MCO-010 is poised to become the first gene-agnostic therapy specifically for patients afflicted by severe vision loss caused by retinitis pigmentosa (RP).

Understanding Retinitis Pigmentosa

Retinitis pigmentosa is a degenerative eye disease that leads to progressive vision loss, ultimately resulting in legal blindness in many patients. This condition is associated with a wide range of genetic mutations—over 100 genes and more than 1,000 different mutations—and it typically worsens with age, creating a pressing need for effective treatment options.

A Game-Changer for Patients

MCO-010 represents a disruptive approach in gene therapy because it is designed to function regardless of the genetic mutation responsible for the condition. The FDA has already granted fast-track designation for this application, allowing for priority review. The implications of this therapy are profound, offering a glimmer of hope to those who face a future of darkness due to RP.

According to Sulagna Bhattacharya, CEO and Co-Founder of Nanoscope, "For the first time, patients who are on the verge of permanent blindness have a chance to regain their sight. We express our deep appreciation to the FDA for their guidance in this endeavor."

How Does MCO-010 Work?

Deliverable through a simple one-time intravitreal injection, MCO-010 has the potential to restore vision by activating remaining intact retinal cells that can still respond to light, despite the loss of photoreceptors. This methodology not only bypasses the need for genetic testing—a hurdle for many patients—but integrates seamlessly into existing ophthalmological practices, maximizing patient accessibility and comfort.

Samarendra Mohanty, PhD, President and Chief Scientific Officer at Nanoscope, reflected on the decade-long effort to develop this innovative treatment: "Witnessing the transformative potential of MCO-010 in both our lab work and clinical trials is truly inspiring. The vision of making this therapy available to all who suffer from RP is now within reach."

Clinical Trial Success

The pivotal RESTORE Phase 2b clinical trial demonstrated promising results, with MCO-010 meeting its primary endpoints for Best Corrected Visual Acuity after 52 weeks, showing significant visual improvement compared to a sham control. The ongoing assessment over three years has continued to exhibit these visual gains without serious adverse effects, indicating a robust safety profile.

Dr. Allen C. Ho, MD, a leading retina specialist and Chief Medical Advisor at Nanoscope, expressed his optimism: "This therapy could fundamentally change the paradigm of care for patients with RP. For years, we had no restorative options to offer; now we have a potential breakthrough that could improve countless lives."

The Future of Treating Retinitis Pigmentosa

With the application for MCO-010 already underway, Nanoscope Therapeutics aims to secure approval by early 2026, as they navigate through the regulatory process. The potential impact of this therapy goes beyond just a treatment option; it aims to set a new standard of care for vision impairment caused by RP.

The broader vision for Nanoscope includes expanding their innovative therapeutic framework for other retinal diseases, promising a future where more patients can hope for restored or improved vision. As Glenn Sblendorio, Chairman of Nanoscope’s Board, aptly noted, "We are on the brink of a new era in retinal care. Our objective is to rekindle hope for RP patients and explore further applications across retinal degenerative diseases."

For further details regarding Nanoscope Therapeutics and MCO-010, visit their website at www.nanostherapeutics.com.