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Nanoscope Therapeutics Inc., have announced the completion of enrolment for Phase 2 Stargardt disease study with optogenetic gene therapy 

Nanoscope Therapeutics Inc., a company based in Bedford, Texas, has reported the completion of enrollment for a Phase 2 study for Stargardt disease.  The study aims to use an ambient-light activatable “Multi-Characteristic Opsin” (MCO) optogenetic monotherapy, referenced to MCO-010, available under ClinicalTrials/gov identifies of NCT05417126.  According to the company, Sulagna Bhattacharya, CEO of Nanoscope stated that, “we are excited by the therapeutic potential of MCO-010, supported by robust data from previous preclinical and clinical studies. Completing the quick two-month enrollment of this Phase 2 trial, which will evaluate the safety and effects of a single intravitreal injection of MCO-010, brings us another major step forward in developing this novel therapy with broad therapeutic application.”


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, with trial results also expected in H1 2023.  Commenting on the achievement, Prof. Stephen Tsang, MD, PhD, at Columbia University, stated that, “Stargardt patients with severe degeneration typically lack sufficient light sensing photoreceptor neurons needed to qualify for experimental ABCA4gene specific therapies. Optogenetic therapy may be able to treat a wider group of patients with Juvenile macular degeneration regardless of the status of their light sensing photoreceptor neurons.”