Razionale scientifico

Gene therapy is rapidly reshaping the management of inherited hemoglobin disorders, particularly β-thalassemia and sickle cell disease (SCD). The recent clinical implementation of gene-based therapeutic strategies represents one of the most important advances in hematology and rare diseases, opening new perspectives for potentially curative treatments. Several gene therapy approaches have now entered clinical practice, demonstrating significant efficacy in reducing transfusion burden, improving quality of life, and modifying disease progression. At the same time, important challenges remain regarding patient selection, access to therapies, long-term safety, sustainability, and integration into healthcare systems. This ECM webinar aims to provide clinicians, hematologists, pediatricians, transfusion specialists, researchers, and healthcare professionals with an updated overview of the current status of gene therapy for thalassemia and sickle cell disease, with a particular focus on clinical application, patient management, and future therapeutic developments. The programme will bring together leading Italian experts actively involved in translational research and clinical implementation of advanced therapies for hemoglobinopathies. Particular attention will also be dedicated to emerging next-generation technologies, including gene editing and innovative in vivo therapeutic platforms, which are expected to further expand treatment opportunities in the coming years. The webinar intends to promote multidisciplinary discussion and strengthen scientific collaboration in the field of advanced therapies for rare hematological diseases.

Responsabili del corso

• Aurelio Maggio

  Responsabile scientifico

  Specializzazione: Professore

Relatori e docenti

• Mattia Algeri

  Relatore

• Juan Antonio Bueren

  Relatore

• Marisanta Napolitano

  Relatore

Domande frequenti