Nanoscope Therapeutics Publishes Breakthrough Clinical Data Demonstrating Significant Vision Restoration in Retinitis Pigmentosa with Novel AAV-based MCO-010 Gene Therapy

Nanoscope Therapeutics has announced the publication of highly encouraging Phase 1/2a clinical trial data in the esteemed journal Molecular Therapy, showcasing significant vision restoration in patients suffering from retinitis pigmentosa (RP), a debilitating inherited retinal disease. The peer-reviewed paper, titled “A synthetic opsin restores vision in patients with severe retinal degeneration,” details the successful application of the company’s innovative Multi-Characteristic Opsin (MCO-010), also known as Synthopsin-MCO-010, a synthetic opsin engineered to possess broad spectral sensitivity.

This novel gene therapy targets the ON-bipolar cells within the retina, a crucial component of the visual pathway. Delivered through a single, minimally invasive intravitreal injection utilizing a custom Adeno-Associated Virus serotype 2 (AAV2) vector, MCO-010 aims to restore light sensitivity in these degenerated retinal cells. Preclinical investigations in RP mouse models had previously demonstrated the potential of MCO-010 to restore visual behaviors, laying the groundwork for this promising human study. The Phase 1/2a trial enrolled four legally blind RP patients with confirmed ABCA4 gene variants, a specific genetic subtype of the disease. These patients, with severe vision impairment ranging from hand motion to mere light perception and a lack of independent mobility, represented a population with a high unmet medical need. The treatment protocol involved a short course of prophylactic oral steroids to mitigate potential immune responses following the single 100 µl intravitreal injection of AAV2-MCO-010 in the eye exhibiting worse visual acuity. Over a 52-week follow-up period, comprehensive assessments of vision and visual function were conducted.

Notably, using the standardized Freiburg Visual Acuity Testing (FrACT), all four patients demonstrated consistent and statistically significant improvements in Best Corrected Visual Acuity (BCVA), indicated by a reduction in logMAR values, by week 12, with these gains largely maintained through week 16. While statistical significance in BCVA was not sustained at the later time points of weeks 31 and 52, this was attributed to specific factors including a patient withdrawal due to COVID-19 related lockdown and another experiencing vitreous haze affecting visual acuity.

Furthermore, the study revealed improvements in the mean Visual Field Index (VFI) from weeks 8 to 52 post-treatment, suggesting a potential expansion of the functional visual field in some patients. Variability in both BCVA and VFI responses may be linked to differential expression patterns of MCO-010 across the central and peripheral retina, a factor discussed in the publication. Importantly, MCO-010 exhibited a favorable safety profile throughout the study, with no serious adverse events or unexpected safety concerns reported.

These findings represent a significant step forward in the development of a potential universal treatment for a wide range of inherited retinal degenerative diseases, offering hope for vision restoration through a single intravitreal injection.

Source: https://ophthalmologybreakingnews.com/nanoscope-therapeutics-publishes-clinical-data

https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(25)00205-9