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    • Gene Therapy
    introduction

    Expertise in AAV gene therapy for incurable diseases

    Gene Therapy

    Pipeline Overview

    AAV Gene Therapy for Degenerative/Inherited Retinal Diseases
    ICM-322

    About ICM-322

    ICM-322 is a gene therapy targeting Age-related Macular Degeneration (AMD) as well as various retinal diseases and consists of AAV vectors carrying a therapeutic gene designed to support RPE (Retina Pigment Epithelium) cells. ICM-322 protects retina and maintain eyesight by enhancing RPE regeneration, suppressing blood vessel invasion and reducing inflammation. ICM has completed small-scale production of ICM-322, and the route of administration (ROA) has been established for therapeutic vector by SRI (Sub-Retinal Injection) and SChI (Supra-Choroidal Injection). Pre-clinical studies of ICM-322 are being conducted in various retina degeneration animal models.

    • What is Age-Related Macular Degeneration?
      • Age-related macular degeneration (AMD) is an eye disease that can blur central vision, and these symptoms are caused by age-driven damaged in macula, the part of the retina at the back of eye mediates visional signals to optic nerves.
      • Losing central vision accompanied by AMD progression can cause difficulties in recognizing faces, reading, driving as well as performing close-up work like cooking, and potentially results in blindness. In fact, AMD is one of the leading causes of vision loss for the aged people.
      • AMD can be developed slowly or fast depending on the individual patient. Since noticeable symptoms are not generally realized until the pathogenic progression reaches to the late-stage, it is advisable to have regular eye exams to monitor retina conditions to avoid serious AMD development.
      • Inherited retinal diseases are visual impairments caused by various genetic factors and include retinitis pigmentosa and congenital amaurosis. These diseases progress due to dysfunction or loss of photoreceptor cells caused by specific genetic mutations, and most of them cause progressive vision loss and blindness. In case of disease gene-specific treatments, there is a limitation that they can only be selectively applied to specific genetic mutations, and it is difficult to comprehensively apply to various diseases.
      • NIH National Eye Institute : Age-Related Macular Degeneration