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  • Genotarget

    • Genotarget

    Developer of gene therapy drugs for the treatment of rare neuromuscular diseases.

    Genotarget focuses on developing drugs for the treatment of incurable, steadily progressing hereditary diseases using its own gene therapy platform. Their priority is neuromuscular diseases.

    The pipeline is based on gene transfer technology using AAV vectors.

    Genotarget

    Development of gene therapy drugs for the treatment of rare neuromuscular diseases.

    Drug
    Indication
    DSCVRY
    Preclinical
    Clinical
    Market
    • GT DF 100
      Indication:
      Dysferlinopathy
      Indication:

      Dysferlinopathy (AAV)

      Stage:
      Preclinical
      Description:
      AAV vector-based drug for the transfer of a fully functional copy of the DYSF gene for treating LGMD R2 (dysferlinopathy, Miyoshi myopathy).

    Developer: Genotarget