Stem Cell and Gene Therapy working GROUP


  • To make equal access to Stem Cell and Gene Therapy a reality across Europe for patients affected by rare disorders of the immune system including auto-immune and auto-inflammatory conditions. This goal shall be reached by bringing together ongoing activities and studies in this field, by harmonizing data collection and analysis and finally enabling prospective studies to improve the treatment options for these patients across Europe.
  • List of current HSCT & GT Studies



Bénédicte Neven

Hôpital Necker-Enfants Malades, Paris