FRATAXAV.
A first-in-class AAV-gene therapy for the curative treatment of Friedreich’s ataxia

Antoni Matilla Dueñas

  • PROJECT LEADER

    Antoni Matilla Dueñas

  • HOST ORGANIZATION,
    COUNTRY

    Fundación Instituto de Investigación Germans Trias i Pujol, Spain

  • DESCRIPTION

    We have developed FRATAXAV, a novel patented drug designed for the curative treatment of Friedreich’s ataxia (FRDA), a rare neurodegenerative disease. This first-in-class gene therapy is based on recombinant adeno-associated virus vector (rAAV9).

    Our asset is designed to supplement frataxin, a protein which is reduced in FDRA, to prevent, stop and reverse clinical symptoms.

    FRATAXAV has shown efficacy in vitro and in vivo, and it is currently the only product in development showing relevant impact in functional and anatomical readouts in the central neurological and peripheral cardiac features of the disease.