SCIENCE
R&D PLATFORMS
  • Gene
    Targets/Indications optimally chosen for Gene Therapy
    Application with High Success Rate and Low Risk

    10 years of dedication in ophthalmic gene therapy

    Deep understanding in ophthalmic disease and molecular/cellular biology of gene therapy

    Choice of indications best-suited for gene therapy

    Clinical-driven R&D

  • Vector
    Mature AAV-based Platform with In-House
    Genetic Engineering Technology

    AAV2 vector already proven for Neuroophthalmological application

    Optimal vector design to minimize dosage

    Allotopic expression technology to restore function of impaired mitochondira

  • Delivery
    Efficient and Safe Outpatient Intravitreal Injection
    Special Clinical Methods to Reduce Local Immunogenicity

    Intravitreal injection: <30min procedure, minimally invasive

    Proprietary formulation to enhance efficacy

    Proprietary clinical procedure to reduce ocular immune response

Fully-Integrated R&D Platform for Ocular Gene Therapy
AAV platform has been validated by IIT1/IIT2 in 168 LHON patients following up nearly 8 years
  • 1
    Interdisciplinary knowledge system
  • 2
    Standardized technical methods
  • 3
    Personnel experience
  • 4
    Advanced instruments and equipment
  • 5
    Self-contained animal models for ophthalmic research