Gene Therapy Against Metachromatic Leukodystrophy – A European Registration Case Study

Time: 10:15 am
day: Day One


  • OTL-200 is an ex vivo autologous hematopoietic stem cell gene therapy approved by the European Medicines Agency (EMA) in 2020 and is currently the only
    approved treatment for MLD
  • Appreciate the details of a successful EU registration procedure for a gene therapy to treat metachromatic leukodystrophy
  • Hear about the various types of interactions with EMA before, during and after the registration