Gene Therapy
introduction

Expertise in AAV gene therapy for incurable diseases

Gene Therapy

Pipeline Overview

AAV Gene Therapy for Osteoarthritis (OA)
ICM-203

About ICM-203

ICM-203 is a gene therapy targeting osteoarthritis (OA) and consists of AAV vector carrying a therapeutic gene designed to effect both articular cartilage and synovial membrane. Since ICM-203 is intra-articularly injected , the risk of off-target exposure can be minimized. In the knee joint, ICM-203 ameliorates OA symptoms by inducing articular cartilage regeneration, suppressing synovial inflammation, and mitigating osteophyte formation. We have completed pre-clinical studies and cGMP production of ICM-203, and the Phase 1/2a clinical trial study is currently conducted in Australia and FDA IND application(P1/2a) was approved in October 2022.

  • Clinical Study Info.
    • Phase 1/2a in AU (ClinicalTrials.gov Identifier: NCT04875754)
  • What is Osteoarthritis?
    • Osteoarthritis (OA) is the most common form of arthritis, and often called as degenerative joint disease or “wear and tear” arthritis. It develops most frequently in the knees, hands, and hips.
    • Upon the onset of OA, breakdown of articular cartilage is elevated, and the underlying sub-chondral bone is affected. These degenerative changes are slowly developed and aggravated over time. OA can cause pain, stiffness, and swelling in the joint. Severe locomotive disabilities can occur to late-stage patients hindering normal daily life and routine work.