PROGRAMME

09:00 SESSION 1:
New discoveries on the mechanism of inhibitor development

Chairs: Pier Mannuccio Mannucci (Italy), Donna Di Michele (USA)
Roadmap for improved haemophilia treatment
Flora Peyvandi (Italy)
Speed bumps on the innovation highway
Donna Di Michele (USA)
Unexpected guests; Stabilin‐2 as a regulatory factor for inhibitor development
David Lillicrap (Canada)
IDO1 and inhibitor development in hemophilia A
Davide Matino (Italy)

10:40 Coffee break & Poster Session (view page)

11:00 SESSION 1 (continued)
Chairs: Jan Voorberg (The Netherlands), Christoph Königs (Germany)
Quantification and phenotyping of factor VIII-specific T cells in healthy donors
Bernard Maillère (France)
Antigen presentation in inhibitor development: new discoveries
Sébastien Lacroix-Desmazes (France)
To serve and protect: modulating effect of von Willebrand factor on FVIII peptide presentation
Jan Voorberg (The Netherlands)

12:30 ORAL COMMUNICATION SESSION
Industry research

Chairs: David Lillicrap (Canada), Elena Santagostino (Italy)
Emicizumab prophylaxis in adolescent/adult patients with haemophilia A previously receiving episodic or prophylactic bypassing agent treatment: updated analyses from the HAVEN 1 Study.
Maria Elisa Mancuso (Italy)
Role of rFVIIIFc in immune tolerance induction in severe hemophilia A patients with Inhibitors
Nisha Jain (USA)
Efficacy, safety, and pharmacokinetics of once-weekly emicizumab prophylaxis in paediatric persons with haemophilia A (PwHA) with inhibitors two years old or younger in the HAVEN 2 Study
Flora Peyvandi (Italy)
Low immunogenicity of human-cell-derived recombinant FVIII in previously untreated patients with non-null F8 mutations
Anna Pavlova (Germany)
Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of haemophilia
Torben Colberg (USA)
Real-World safety and benefit of early treatment with room temperature-stable recombinant activated Factor VII (rFVIIa) in patients with haemophilia A or B with inhibitors: SMART-7™ study results
Kaan Kavakli (Turkey), on behalf of the SMART-7™ steering committee

13:30 Lunch

14:30 SESSION 2:
New developments for tolerance induction

Chairs: Flora Peyvandi (Italy), Federico Mingozzi (France)
Gene therapy and new approaches for tolerance induction Federico Mingozzi (France)
Federico Mingozzi (France)
Liver induced tolerance
Percy Knolle (Germany)

15:30 Coffee break & Poster Session (view page)

16:00 SESSION 2 (continued)
Chairs: Luigi Naldini (Italy), David Lillicrap (Canada)
Engineering regulatory T cells: Driving CARs to BARs
David Scott (USA)
Perspectives of Immune Tolerance Induction (ITI) in the era of new haemophilia treatment options
Johannes Oldenburg (Germany)
Alternatives to human VIII – orthologs, ancestors and hybrids thereof
Christopher B. Doering (USA)

17:30 WALKING ORAL COMMUNICATIONS SESSION
Foyer in front of the Plenary Hall (with refreshments)

Chairs: Donna Di Michele (USA), Frits Rosendaal (The Netherlands)
Generation and characterization of humanized hemophilic mice expressing HLA-DRB1*0101
Jules Russick (France)
Recombinant FVIII product type and the risk of inhibitor development in previously treated patients with hemophilia A: a systematic review and meta-analysis
Shermarke Hassan (The Netherlands)
SIPPET effect on Turkisk previously untreated patients (PUP) with severe hemophilia A: National data
Kaan Kavakli (Turkey)
Identification and functional characterization of a novel consensus sequence splicing mutation of factor VIII (FVIII) coagulation gene associated with severe hemophilia A and inhibitors development
Rosella Famà (Italy)
Single center experience in the treatment of haemophilia A PUPs
Susan Halimeh (Germany)
Lot-matching decreases the time for Immune Tolerance Induction (ITI): Preliminary results of the ObsITI study
Jonathan Ducore (USA)
Severe acquired von Willebrand syndrome (type 3-like): a case report
Francesca Stufano (Italy)
Discussion and question and answers

19:00 End of sessions

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