Sonpiretigene Isteparvovec

The STARLIGHT study is the first and only clinical trial to demonstrate vision improvement in patients with Stargardt disease. MCO-010 demonstrated mean visual acuity improvement of 12 ETDRS letters over 48 weeks in patients with atrophy confined to the macula -with no reports of serious adverse events. Nanoscope has completed an end of phase 2 meeting with the FDA and plans to initiate a registration-enabling Phase 3 trial in Stargardt disease patients by the end of 2025. DALLAS, Aug. 14, 2025 /PRNewswire/ -- Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel gene-agnostic therapies for patients suffering vision loss from retinal degenerative diseases, today announced the publication of positive results from its STARLIGHT clinical trial in eClinical Medicine, part of The Lancet Discovery Science. STARLIGHT is the first and only clinical trial to demonstrate vision improvements in patients with Stargardt disease (SD). STARLIGHT is a Phase 2, open-label study designed to assess the safety and efficacy of a single intravitreal injection of gene-agnostic MCO-010 therapy in six participants with severe vision loss due to SD. Key findings include: Mean improvement in best corrected visual acuity (BCVA) from baseline to week 48 was: For all patients, +5.5 and +9.0 ETDRS letters without and with a wearable low-vision aid, respectively. In individuals with atrophy confined to the macula, +12.0 and +32.0 ETDRS letters, without and with a wearable low-vision aid, respectively. Some patients observed vision improvement as early as 4 weeks post-treatment that was maintained throughout the duration of the study. MCO-010 was well tolerated with no reported serious adverse events over 48 weeks. Stargardt disease, also known as juvenile macular dystrophy, is a major cause of vision loss in children, affecting more than 40,000 people in the US and leaving over 20,000 legally blind. In patients with SD, the light-sensing photoreceptors in the macula degrade, leading to loss of central vision. Currently, there are no approved treatments for SD. "While this was an open-label study, this is one of the first studies to demonstrate vision improvement in Stargardt disease patients. This is a remarkable finding as these patients typically experience irreversible central vision loss over their lifetimes.," added Byron Lam, MD, Professor of Ophthalmology at Bascom Palmer Eye Institute, lead author and principal investigator of the STARLIGHT study. "I am thrilled to be part of the upcoming Phase 3 trial of MCO-010 in Stargardt disease, helping advance this promising therapy towards potential approval." "We are grateful to the patients and investigators who participated in the trial," added Samarendra Mohanty, PhD, President / Chief Scientific Officer. "The results we have seen in STARLIGHT for SD patients reinforce the therapeutic potential of our gene-agnostic MCO-010 platform, building on positive outcomes in both Stargardt and retinitis pigmentosa populations." The published manuscript, titled "Safety and Efficacy of MCO-010 Optogenetic Therapy in Patients with Stargardt Disease in USA (STARLIGHT): an Open-label Multi-Center Ph2 Trial", is now available online and will appear in an upcoming print issue of The Lancet. About MCO-010 and the upcoming STARGAZE Phase 3 Study Nanoscope's proprietary MCO-010 (sonpiretigene isteparvovec) is a one-time, in-office intravitreal gene-agnostic therapy designed to restore vision in patients with advanced retinal degeneration. MCO-010 activates highly dense bipolar retinal cells to become light sensitive, utilizing remaining visual circuitry following photoreceptor death. MCO-010 therapy does not require genetic testing, surgical intervention, or repeat dosing, enabling broad patient applicability within existing retina office workflows. STARGAZE is a Phase 3, randomized, double-masked sham-controlled study designed to evaluate the efficacy and safety of MCO-010 in patients with Stargardt Disease. Following a successful End-of-Phae 2 meeting, and in alignment with FDA guidance, this registration-enabling study will enroll approximately 60 patients across multiple centers. The primary endpoint is the change in BCVA using a standard vision chart vs sham control. A key secondary endpoint will assess improvement in multi-luminance shape recognition. Top-line results are expected in 2027. For more information on MCO-010 and Nanoscope Therapeutics visit: . About Nanoscope Therapeutics Nanoscope Therapeutics is developing gene-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (NCT04945772), a rolling BLA submission to the FDA has been initiated. If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2025. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD. Preclinical programs include Leber congenital amaurosis (LCA), in IND-enabling studies, as well as an IND-ready asset for geographic atrophy (GA). Contact: Nanoscope Therapeutics (817) 857-1186 [email protected] SOURCE Nanoscope Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

126-week results from RESTORE follow-up study (REMAIN) to highlight the extended efficacy and safety of Nanoscope's MCO-010 optogenetic therapy for Retinitis Pigmentosa DALLAS, July 28, 2025 /PRNewswire/ -- Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel gene-agnostic therapies for patients suffering severe vision loss from retinal degenerative diseases, announced that its multi-characteristic opsin (MCO) gene therapy platform will be featured in a presentation by Dr. David S. Liao at the 2025 American Society of Retina Specialists Annual Scientific Meeting, taking place July 30-August 2, 2025 at the Long Beach Convention Center in Long Beach, California. Dr. Liao will present 126-week data on the strong efficacy and safety profile of MCO-010 in treating retinitis pigmentosa (RP) patients via a one time, in-office intravitreal injection without the need for genetic testing. Nanoscope recently initiated a rolling submission of a Biologics License Application (BLA) to the FDA for MCO-010 for the treatment of severe vision loss due to RP. The data, from Nanoscope's RESTORE follow-up study (REMAIN), form the backbone of Nanoscope's BLA rolling submission. Details for the presentation are as follows: "We are excited to have this 126-week data presented at ASRS, especially with our recent BLA announcement," said Dr. Samuel Barone, Chief Medical Officer for Nanoscope. "This will be a great opportunity for the ASRS community to discuss our data and its potential impact on RP patients." For more information on MCO-010 and Nanoscope Therapeutics visit: . About Nanoscope Therapeutics Nanoscope Therapeutics is developing gene-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (NCT04945772), a rolling BLA submission to the FDA has been initiated. If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2025. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD. Preclinical programs include Leber congenital amaurosis (LCA), in IND-enabling studies, as well as an IND-ready asset for geographic atrophy (GA). Contact: Nanoscope Therapeutics (817) 857-1186 [email protected] SOURCE Nanoscope Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED