Nanoscope targets FDA approval following positive gene therapy trial results

Hopes rise for AMD as retinitis pigmentosa gene therapy demonstrates ‘significant improvements’ in both primary and secondary endpoints.

Gene therapy company Nanoscope Therapeutics has unveiled promising top-line findings from its Phase 2b RESTORE trial, showcasing encouraging results for its lead program, a gene therapy targeting retinitis pigmentosa (RP). The mutation-agnostic gene therapy, MCO-010, demonstrated significant improvements in both primary and key secondary endpoints in the treatment of RP, a genetis condition characterized by progressive degeneration of the retina leading to worsening vision loss. From a longevity perspective, Nanoscope is also using the same platform to develop programs targeting geographic atrophy (GA) due to age-related macular degeneration (AMD).

Nanoscope, which specializes in gene-agnostic optogenetic therapies for retinal diseases, claims that RESTORE is the first randomized, controlled trial of a mutation-agnostic gene therapy for a genetic disease. RP is estimated to affect approximately 100,000 people in the US, and an estimated 2 million worldwide. The trial, which spanned over two years, enrolled 28 subjects with severe vision loss due to RP. The company says that MCO-010 achieved statistically significant improvements in visual acuity compared to the control group, without any serious adverse events reported.

“We observed significant vision restoration in many patients with severe vision loss, including those who were completely blind,” said University of Southern California professor David Boyer, an investigator in the trial. “Many patients treated with MCO-010 derived a clinically meaningful benefit measurable on the primary visual function test, and this effect was confirmed by a parallel improvement in functional vision assessments. If approved, MCO-010 is poised to make a positive, meaningful impact on the lives of patients affected by this debilitating condition.”

The positive outcomes of the trial pave the way for Nanoscope to move forward with its regulatory objectives. The company intends to submit a Biologics License Application (BLA) to the US FDA in the latter half of 2024, as it now proceeds towards potential approval and commercialization.

“The compelling data from the most recent analyses of the RESTORE trial at week 76 provide additional validation of Nanoscope’s versatile MCO platform, which is driving our expanding pipeline of programs in both Stargardt disease and geographic atrophy (GA) due to age-related macular degeneration (AMD),” said Sulagna Bhattacharya, CEO of Nanoscope.

“This achievement marks a significant milestone in the field of mutation-agnostic gene therapy, firmly establishing the promise of optogenetics as a therapeutic modality,” said Dr Samarendra Mohanty, Chief Scientific Officer of Nanoscope.