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Nanoscope Therapeutics Inc., have reported clinical trial results from a Phase 2 Stargardt disease study with optogenetic gene therapy. 

Nanoscope Therapeutics Inc., a company based in Bedford, Texas, has reported key results from a Phase 2 clinical trial, “STARLIGHT”, at the American Society of Retina Specialists (ASRS) annual meeting in Seattle last month.  The trial evaluated the effects of a novel therapeutic, MCO-010, for patients with Stargardt disease and key summaries of the data indicated clinically meaningful improvements in best-corrected visual acuity (BCVA), an approximate 3dB gain in mean sensitivity in visual field perimetry, and reporting no serious adverse events (SAEs) for the cohort. Commenting on the announcement, Dr. Allen Ho, Professor of Ophthalmology at Thomas Jefferson University and Attending Surgeon, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital stated that, “I appreciated the opportunity to present on the promising results of Nanoscope’s Phase 2 STARLIGHT clinical trial. With no current treatment options, MCO-010 may bring hope to those patients with severe vision loss due to Stargardt disease by restoring broadband light sensitivity”.


The study aims to use an ambient-light activatable “Multi-Characteristic Opsin” (MCO) optogenetic monotherapy, referenced to MCO-010, available under identifies of NCT05417126.  The phase 2 “STARLIGHT” study is an open-label trial which has enrolled six subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease.  All subjects received the same single intravitreal dose of 1.2 × 1011 VG per eye of MCO-010, as used in a previous Phase 2b retinitis pigmentosa (RP) study.  The optogenetics gene therapy strategy aims to turn bipolar cells into light-sensing activated neurons in response to light, making them the new photoreceptors of the retina. The strategy develops an optogenetic gene therapy using “multi-characteristic opsin” (MCO) to re-sensitize the retina to detect low light levels.  The outcomes of the clinical trial use a primary endpoint for ocular and systemic adverse events, with secondary outcomes of visual acuity (BCVA), light-guided mobility, and optical flow using Low Vision Multi-Parameter Test (LVMPT).


The company’s optogenetic therapy uses a proprietary AAV2 vector to deliver the genes into retinal cells to enable vision in different colour environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions. In addition, MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA and is concurrently being evaluated in a Phase 2b RESTORE trial in patients with RP.  The RESTORE trial results presented earlier this year showing that 100% (18/18) MCO-010 patients experienced a clinically meaningful improvement in vision-guided mobility (MLYMT), near-field object recognition (MLSDT) or visual acuity (BCVA) (p=0.007 vs sham) and 94.4% (17/18) MCO-010 patients showed vision improvement in the MLYMT or BCVA (p=0.008 vs sham).  In addition, 88.9% (16/18) of MCO-010 patients experienced a clinically meaningful 2 or more luminance level improvement in MLYMT or MLSDT (p=0.02 vs sham.  Reporting on this achievement, David Boyer, MD, Retina-Vitreous Associates Medical Group in Beverly Hills, CA and Nanoscope Clinical Advisory Board member stated that, “the fact that we see any gains in vision after a single intravitreal injection is remarkable. Some participants who were living with severe vision impairment due to RP have noticed improvement in visual function. In addition, MCO-010 had a favourable safety profile. It is an honour to have been a part of the first randomized controlled trial to show a visual improvement in a profoundly visually impaired population.”