FA Europe Scientific Meeting, Genoa, May 22 -23, 2025
The FA Europe Scientific Meeting, May 22nd and 23rd, 2025 at the Gaslini Academy in Genoa, brought together over 100 healthcare professionals, researchers and patient support group representatives from 13 European countries (Austria, Czech Republic, Denmark, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain and the UK) to share experiences, provide a comprehensive overview of current clinical and translational developments in Fanconi Anaemia and to encourage new collaborations within Europe.
Day 1 opened with a welcome from Prof. Carlo Dufour, followed by powerful personal stories from two adults living with FA, setting a context for subsequent sessions on haematopoietic stem cell transplantation, clonal evolution management, and Eltrombopag use with and without gene therapy. Further sessions examined pharmacologic stabilisation of bone marrow failure, translational readthrough-inducing drugs (TRIDs), and evolving gene therapy approaches. Flash talks showcased a wide array of preclinical findings, from mitochondrial metabolism and DNA repair modulation to novel animal models and ENT surveillance strategies. The day ended with a review of European psychosocial activities, followed by a buffet meal in the evening to encourage further collaborative discussions.
Day 2 shifted focus to FA Registry-driven insights from five European countries, followed by presentations on marrow evolution, epigenetics, mitochondrial dysfunction post-HSCT, and nascent DNA-protein crosslinks. Nutritional interventions were also explored as potential modifiers of genomic instability. Cancer-focused sessions covered head and neck oncology, AI-based screening, and proton therapy. Novel immunotherapies included FA-adapted CAR-T platforms targeting squamous carcinomas and myeloid malignancies. An update on the European Virtual Tumor Advisory Board was given, with feedback after the first 4 monthly meetings.
The meeting concluded with a review of the event from Prof. Hanneke Takkenberg, in which she said: “Let us carry forward the momentum of these days. Let us share data more openly, centre patient voices more consistently, and pursue not just longer life—but better life—for everyone living with Fanconi Anaemia. As was wisely said: If you want to go fast, go alone. But if you want to go far, go together. Thank you for going together with us—further than ever before”.
We are indebted to the Gaslini Institute, the EHA and European FA Patient Support Groups for sponsoring the event.
More photos from the event can be found here.