sonpiretigene isteparvovec (VMCO-I) / Nanoscope Therap - LARVOL DELTA

Nanoscope Therapeutics Announces U.S. Food and Drug Administration Acceptance of Biologics License Application for MOGENRY for the Treatment of Retinitis Pigmentosa with Severe Vision Loss (PRNewswire) - "The BLA is supported by positive efficacy and safety data from the Phase 1/2a trial (NCT04919473), and the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial (NCT04945772)."

FDA filing • Retinitis Pigmentosa

Mechanism and clinical evidence of MCO-010 optogenetic therapy improving color discrimination in patients with outer retinal degeneration (ASGCT 2026) - P1/2, P2, P2a | "Introduction MCO-010 (sonpiretigene isteparvovec) is an AAV2-delivered optogenetic gene therapy encoding Multi-Characteristic Opsin (MCO) with a unique multi-domain architecture designed to enable broadband spectral responsiveness across the visible light spectrum...Clinical data from Ph1/2a and Ph2b/3 trials in RP and the Ph2 trial in Stargardt patients demonstrate that MCO-010 treatment may result in improvement in color vision, in addition to gains in visual acuity. These findings support the potential of MCO-010 to restore meaningful vision in patients with outer retinal degenerations, in a disease-agnostic manner."

Clinical • CNS Disorders • Gene Therapies • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Bilateral functional improvement in patients following unilateral AAV-based retinal cell delivery of a bioengineered, synthetic protein with broad-spectrum light sensitivity (ASGCT 2026) - "Vision improvements were also observed in contralateral eyes, consistent with robust and cell-type-specific bilateral retinal transduction across mice, dogs, and NHPs following unilateral IVT administration. The nonclinical biodistribution patterns indicate interocular transfer via optic-nerve-chiasmal pathways without off-target expression in central visual structures."

Clinical • Age-related Macular Degeneration • CNS Disorders • Dry Age-related Macular Degeneration • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • HSPA5

Contralateral Transfer of AAV-MCO-010 and Retinal Transduction Following Unilateral Intravitreal Delivery (ARVO 2026) - "Biodistribution patterns indicate interocular transfer via optic-nerve–chiasmal pathways without off-target expression in central visual structures. These findings demonstrate a conserved mechanism of contralateral transfer following a single intravitreal AAV injection, supporting the significant benefit of optogenetic treatment of retinal degenerative diseases, which are generally manifested in both eyes."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology • HSPA5

Optogenetic REMAIN 3-Year Study Data: Lasting Vision Improvement with MCO-010 in the RESTORE Phase 2b/3 Trial for Retinitis Pigmentosa (ARVO 2026) - "Layman Abstract:There are very limited treatment options for progressive and permanent vision loss from retinal degenerations like retinitis pigmentosa (RP). MCO-010 is a gene therapy with the potential to address multiple retinal degnerations, and in clinical trial or patients with RP has shown meaningful and durable vision improvements 3 years after a one-time, in-office administration procedure."

P2/3 data • P2b data • Achromatopsia • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Nanoscope Therapeutics Receives Orphan Drug Designation in Saudi Arabia for MCO-010, Expands Global Regulatory Leadership in Vision Restoration (PRNewswire)

Orphan drug • Ophthalmology

Japan's Ministry of Health, Labor and Welfare (MHLW) grants MCO-010 Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations, enabling an accelerated regulatory pathway to treat patients with severe vision loss from inherited retinal diseases (PRNewswire) - "Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (NCT04945772), a rolling BLA submission to the FDA has been initiated....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 early 2026....A Phase 2 program for MCO in geographic atrophy (GA) is expected to start in early 2026."

FDA filing • Japanese regulatory • New P2 trial • New P3 trial • Orphan drug • Inherited Retinal Dystrophy • Retinal Disorders • Retinitis Pigmentosa

Nanoscope Strengthens Global Regulatory Pathways for MCO-010 with FDA RMAT and Five EMA Orphan Designations (PRNewswire)

FDA event • Orphan drug • Retinal Disorders

Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial. (PubMed, EClinicalMedicine) - P2a | "To our knowledge, this is the first report of improving on(eye)-chart vision of SD participants utilizing optogenetics. Nanoscope Therapeutics Inc."

Journal • Age-related Macular Degeneration • Cardiovascular • CNS Disorders • Gene Therapies • Glaucoma • Hematological Disorders • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders

Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa (PRNewswire) - "Nanoscope Therapeutics...announced the initiation of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its lead investigational therapy, MCO-010, for the treatment of severe vision loss due to retinitis pigmentosa (RP)....The FDA has granted Nanoscope rolling review of its BLA, with the first modules already submitted and completion of the full BLA submission anticipated in early 2026."

FDA filing • Retinitis Pigmentosa

Human Retinal Cell Delivery of a Bioengineered, Synthetic Protein Sensitive to Multiple Wavelengths of Light in Patients with Stargardt Disease (ASGCT 2025) - "MCO-010 optogenetic phase 2 results support the further investigation for treatment SD. Disease Focus of Abstract:Eye Disorders"

Clinical • Cardiovascular • CNS Disorders • Glaucoma • Hematological Disorders • Inherited Retinal Dystrophy • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders

Forced Degradation Studies of Multi-Characteristic Opsin Gene Therapy Drug Product (ASGCT 2025) - "These findings support the use of these validated assays for quality control and stability monitoring during MCO-010 drug product storage and handling for future clinical use. Disease Focus of Abstract:Ophthalmic Diseases"

Gene therapy • Gene Therapies • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Development of Orthogonal Potency Assays for Multi-Characteristic Opsin Gene Therapy: Gene Expression and Light-Stimulated Activity (ASGCT 2025) - "The successful implementation of these potency assays supports product release and stability testing and establishes standards for optogenetic gene therapy products. Disease Focus of Abstract:Rare Diseases"

Gene therapy • Gene Therapies • Rare Diseases

Analysis of Patient-Reported Outcomes of MCO-010 Mutation Agnostic Optogenetic Therapy for Retinitis Pigmentosa: 12-Month Results from a Phase 2b Randomized, Sham-Controlled, Patient- and Assessor-Masked Clinical Trial (RESTORE) (ISPOR 2025) - "RESTORE data demonstrate improved patient-reported outcomes on vision-related quality of life at week 52 in MCO-010 treated patients."

Clinical • P2b data • Patient reported outcomes • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Perspectives and open questions in vision restoration with vMCO-010. (PubMed, Mol Ther) - No abstract available

Journal

A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection (clinicaltrials.gov) - P=N/A | N=11 | Enrolling by invitation | Sponsor: Nanoscope Therapeutics Inc. | Trial completion date: Dec 2024 ➔ May 2025 | Trial primary completion date: Dec 2024 ➔ May 2025

Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

126-Week Visual Acuity Outcomes Following Mutation-Agnostic Optogenetic Therapy, MCO-010, for Retinitis Pigmentosa (RP) (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

SUSTAIN: Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study (clinicaltrials.gov) - P=N/A | N=6 | Active, not recruiting | Sponsor: Nanoscope Therapeutics Inc. | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

REMAIN: Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study (clinicaltrials.gov) - P=N/A | N=18 | Active, not recruiting | Sponsor: Nanoscope Therapeutics Inc. | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Verana Health and Nanoscope Therapeutics Announce Collaboration to Accelerate Retinitis Pigmentosa Research (GlobeNewswire) - "Verana Health....and Nanoscope Therapeutics...today announced a partnership to accelerate research for retinitis pigmentosa (RP), a rare inherited retinal disease that affects approximately 100,000 people in the U.S....Through this collaboration, Nanoscope Therapeutics will utilize Verana Health’s Qdata Retinitis Pigmentosa, which is a research-ready, real-world dataset that can be used to analyze patients with RP and track their clinical outcomes....Such insights will support the development and commercialization of MCO-010, Nanoscope Therapeutics’ optogenetic gene therapy that is designed to restore vision and potentially slow neurodegeneration."

Commercial • Retinitis Pigmentosa

Nanoscope Therapeutics Announces Presentations at Upcoming Medical Meetings (PRNewswire) - "Nanoscope Therapeutics Inc....today announced presentations at two upcoming medical meetings....Nanoscope is developing restorative treatments. As part of this, Dr. Ho will discuss 126-week data from Nanoscope's REMAIN long-term follow-up study of advanced retinitis pigmentosa patients dosed in the RESTORE randomized controlled trial....Dr. Gonzalez will present and discuss long-term follow-up efficacy and safety data of Nanoscope's MCO-010 optogenetic therapy for vision restoration in patients with severe vision loss due to retinitis pigmentosa."

Observational data • Ophthalmology • Retinitis Pigmentosa

Efficacy of Intravitreal Multi-Characteristic Opsin (MCO-010) Optogenetic Gene Therapy in a Mouse Model of Leber Congenital Amaurosis. (PubMed, J Ocul Pharmacol Ther) - "It also significantly improved the vision in rd12 mice relative to control AAV2-injected mice. MCO-010 therefore represents a novel and efficacious optogenetic therapeutic to treat LCA and other IRDs irrespective of the genetic defect(s)."

Gene therapy • Journal • Preclinical • CNS Disorders • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Nanoscope Publishes Breakthrough Showing MCO-010 Treatment Arrests Neurodegeneration (PRNewswire) - "Nanoscope Therapeutics...announced a breakthrough discovery in Translational Vision Science & Technology (TVST, an ARVO journal) demonstrating that MCO-010 optogenetic treatment of the inner retina arrests further retinal degeneration in an animal model of retinitis pigmentosa (RP), as assessed by optical coherence tomography and immunohistochemical analysis. Following intravitreal MCO-010 treatment (sonpiretigene isteparvovec), approximately 80% of bipolar cells were transduced in the retina, and no alterations in retinal thickness were observed, unlike the control group. The results described in the publication provide evidence that the expression of MCO-010 in the retinas of mice experiencing photoreceptor degeneration halts further loss and prevents further disorganization of the retinal cell layers."

Preclinical • Ophthalmology • Retinitis Pigmentosa

Multi-Characteristic Opsin Therapy to Functionalize Retina, Attenuate Retinal Degeneration, and Restore Vision in Mouse Models of Retinitis Pigmentosa. (PubMed, Transl Vis Sci Technol) - "Intravitreal MCO-010 was well tolerated in rd1 and rd10 mice models of RP, and it appeared to attenuate retinal photoreceptor degeneration based on retinal structure and functional outcome measures. As reported here, optogenetic treatment of the inner retina attenuates further retinal degeneration in addition to photosensitizing higher order neurons, and this disease-modifying aspect should be evaluated in optogenetic clinical trials."

Journal • Preclinical • CNS Disorders • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Nanoscope Therapeutics Announces End-of-Phase 2 Meeting with U.S. FDA and Plan to Initiate a Phase 3 Clinical Trial of MCO-010 to Treat Stargardt Macular Degeneration (PRNewswire) - "The FDA provided clear input on the proposed Phase 3 registrational trial in patients with SMD intended to support safety and effectiveness of MCO-010. The proposed study design will consist of a single MCO-010 intravitreal dose group, randomized 1:1 to a control cohort receiving a sham injection; The FDA provided valuable input on statistical methods for the proposed primary and secondary endpoints that will inform further engagement with FDA to align on statistical analyses; The FDA offered continued discussion and assistance to plan further evaluation of the multi-luminance shape discrimination test to support use as a key secondary endpoint; The FDA agreed on the sufficiency of the current nonclinical package to support a future BLA submission."

FDA event • New P3 trial • Macular Degeneration • Ophthalmology