The future of haemophilia: From novel factor replacement to beyond-replacement strategies
FAD sincrona internazionale su terapia dell'emofilia: factor replacement (efanesoctocog alfa), strategie di rebalancing, gene therapy e gene editing. Relatori italiani e internazionali. 6 crediti ECM, 13 novembre 2026.
Fonte dati per questo evento: Agenas. Evento organizzato da provider terzo. Smart ECM riporta dati pubblici dell'evento a scopo informativo e non è responsabile né del corso né dei contenuti, né ha accordi commerciali o di collaborazione con il provider.
Razionale scientifico
The therapeutic landscape of hemophilia is undergoing a profound transformation. While the field is rapidly expanding beyond conventional replacement therapy toward rebalancing strategies, gene therapy, and gene editing, replacement treatment itself is also entering a new phase of innovation. In particular, the availability of novel factor replacement approaches, including high-sustained factor VIII therapies such as efanesoctocog alfa, is reshaping expectations in prophylaxis, bleed protection, treatment personalization, and quality of life. These advances are broadening the clinical possibilities within replacement therapy and are redefining how clinicians position factor-based strategies in relation to non-replacement options. At the same time, the emergence of rebalancing therapies, RNA interference approaches, AAV-based gene therapy, and gene editing is creating an increasingly complex decision-making environment for hemophilia treatment centers. Key issues now include treatment selection, patient profiling, monitoring, long-term safety, durability of response, access, sustainability, and future care pathways. Webinar Garden 2026 aims to provide a multidisciplinary update on the most recent advances, integrating clinical evidence, real-world data, international consensus recommendations, and the patient perspective, with the goal of outlining the future management of coagulation disorders in the era of advanced therapies.
Programma
The Future of Haemophilia: From Novel Factor Replacement to Beyond-Replacement Strategies· Online (piattaforma FAD sincrona – Webinar), Via Emilia 38, Palermo
- 14:00-14:10Opening & Welcome – Presentation of objectives; Overview of the 2025–2026 paradigm shiftM. Napolitano, A. Maggio
- 14:10-15:00The Real-World Evolution of Hemophilia CareM. Napolitano
- 14:10-15:00Beyond Factor Replacement: Clinical Trial Evidence and Real-World Challenges of Rebalancing StrategiesGuy Young
- 14:10-15:00Discussion
- 15:00-16:10Valoctocogene and Etranacogene in 2026: Durability, Immunogenicity and Patient SelectionSarina Levy
- 15:00-16:10Gene Therapy in the Real World: Building Sustainable Long-Term Follow-Up ModelsWolfgang Miesbach, Glenn Pierce
- 15:00-16:10Discussion
- 16:10-16:25Virtual Break
- 16:25-17:10Gene Editing for Hemophilia A: From Intron 22 Inversion to Base EditingM. Pinotti
- 16:25-17:10Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia AA. Srivastava
- 16:25-17:10Discussion
- 17:10-17:302026 International Consensus: Managing Hemophilia in the Era of Advanced TherapiesC. Hermans
- 17:30-18:10Final Round Table: Is Hemophilia Still a Rare Disease in 2030?C. Hermans, Guy Young, Sarina Levy, A. Srivastava
- 18:10Closing Remarks
Responsabili del corso
BIBA TOUR SRL
Provider
ID Provider: N° 1420
Città: Palermo (PA)
Tipologia: Società, Agenzie Ed Enti Privati
Stato accreditamento: Standard - 1° Rinnovo
Medico Chirurgo, Coagulation Disorders Cedric Hermans
Responsabile scientifico
Specializzazione: Emostasi, trombosi, emofilia
Struttura: Division of Haematology, Cliniques universitaires Saint-Luc, Catholic University of Louvain, Brussels, Belgium
Ruolo: Full Professor, Head Haemostasis and Thrombosis Unit
Medico Chirurgo, Ematologia Aurelio Maggio
Responsabile scientifico
Specializzazione: Ematologia
Struttura: Fondazione Franco e Piera Cutino ETS, Palermo
Ruolo: Retired
Medico Chirurgo, Ematologia Mariasanta Napolitano
Responsabile scientifico
Specializzazione: Emofilia, malattie rare del sangue
Struttura: Haematology and Rare Disease University Unit, Hospital V.Cervello, Palermo
Ruolo: Head of Haematology and Rare Disease University Unit; Associate Professor of Hematology; Director specialization school in hematology, University of Palermo, PROMISE Department
Con il contributo non vincolante di
- SOBI ITALIA SRL
- CHIESI IFARCEUTICI
Domande frequenti
L'evento è accreditato per: Medico Chirurgo (Farmacologia E Tossicologia Clinica); Farmacista (Farmacista Pubblico Del Ssn); Medico Chirurgo (Pediatria); Biologo (Biologo); Psicologo (Psicoterapia); Tecnico Sanitario Laboratorio Biomedico (Iscritto Nell’Elenco Speciale Ad Esaurimento); Medico Chirurgo (Genetica Medica, Patologia Clinica (Laboratorio Di Analisi Chimico-Cliniche E Microbiologia)); Farmacista (Farmacista Di Altro Settore); Medico Chirurgo (Medicina Interna, Laboratorio Di Genetica Medica); Farmacista (Farmacista Territoriale); Psicologo (Psicologia); Medico Chirurgo (Psicoterapia, Ematologia); Infermiere (Infermiere); Tecnico Sanitario Laboratorio Biomedico (Tecnico Sanitario Laboratorio Biomedico).
Fonte dati per questo evento: Agenas. Evento organizzato da provider terzo. Smart ECM riporta dati pubblici dell'evento a scopo informativo e non è responsabile né del corso né dei contenuti, né ha accordi commerciali o di collaborazione con il provider.