ORGANIZED BY

N. CIAVARELLA, Italy A.B. FEDERICI, Italy D. LILLICRAP, Canada S. LACROIX-DESMAZES, France P.J. LENTING, France J. VOORBERG, The Netherlands

P.M. MANNUCCI, Italy F. PEYVANDI, Italy

SCIENTIFIC COMMITTEE

10TH BICINTERNATIONALCONFERENCE

10TH BICINTERNATIONALCONFERENCE

Palazzo DucalePiazza Giacomo Matteotti, 9 - Genoa

27 /

06

/ 20

19

Replacement therapies in hemophilia A and B - Efficacy of extended half-life productsChairs: David Lillicrap (Canada), Pier Mannuccio Mannucci (Italy)

Real life data: real benefits. What have we achieved? Pratima Chowdari (UK)

How to optimize the use of extended half-life products (PK analysis)Alfonso Iorio (Canada)

Safety of replacement therapies (standard and novel) (Part I)Chairs: Nicola Ciavarella (Italy), Maria Elisa Mancuso (Italy)

Which bleeding phenotype can we expect in severe hemophilia A previously untreated patients treated with non replacement therapies?Karin Fijn van Draat (The Netherlands)

How safety of novel anti-hemophilic drugs should be approached. Known and unknown adverse effects Flora Peyvandi (Italy)

Are Fc – FVIII products less immunogenic? New clinical data? Christoph Königs (Germany)

Safety of replacement therapies (standard and novel) (Part II)Chairs: Giovanni Di Minno (Italy), Flora Peyvandi (Italy)

Long-term side-effects of pegylated products: facts or fancies?Silvio Garattini (Italy)

European pharmacovigilance of new products (with special emphasis on pegylated products)Anneliese Hilger (EMA, Germany)

Immunogenicity of subcutaneous and intravenous drugsDavid Lillicrap (Canada)

Bayer Lunch Satellite SymposiumTBA

Takeda Satellite SymposiumTBA

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Kedrion Satellite LectureTBA

Keynote Lecture “Choice of assays for novel anti-hemophilic therapies”Armando Tripodi (Italy)

The role of von Willebrand factor in factor VIII immunogenicityChair: Giancarlo Castaman (Italy), Augusto B. Federici (Italy)

FVIII peptide presentation to the immune system; modulation by von Willebrand factorJan Voorberg (The Netherlands)

High von Willebrand factor affinity FVIII and immunogenicity Peter Lenting (France)

Walking Poster Session

FRIDAY

SATURDAY

Non-replacement therapies in the hemophilias: UpdateChairs: Peter Lenting (France), Jan Voorberg (The Netherlands)

FitusiranSteve Pipe (USA)

APC SerpinJames Huntington (UK)

Anti-TFPI Alan Mast (USA)

Thrombotic microangiopathy associated with emicizumab: views from an insiderPaul Coppo (France)

Novel drugsChair: Paul Coppo (France)

Caplacizumab: results of phase II/phase III studiesMarie A. Scully (UK)

Novo Nordisk Satellite LectureTBA

Roche Satellite LectureTBA

Octapharma Lunch Satellite SymposiumTBA

Gene TherapyChair: Steve Pipe (USA), Amy Shapiro (USA)

New strategies for a better and safer gene transfer: less immunogenicLuigi Naldini (Italy)

Liver toxicity in gene therapyAmit Nathwani (UK)

Oral Communications – HemophiliaTargeting of hepatocyte subpopulation contributing to liver post-natal growth is crucial for maintenance of transgene expression in liver-directed gene therapyMichela Milani (Italy)

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Transplantation of fetal liver cells into newborn hemophilic mice for the treatment of hemophilia A without inhibitors formationSimone Merlin (Italy)

B-AMAZE, a Phase 1/2 trial of a novel investigational adeno associated virus (AAV) gene therapy (FLT180a) in subjects with severe or moderately severe hemophilia B (HB)Pratima Chowdary (UK)

Developing a novel Coagulation Factor VIII with Reduced Immunogenicity by a Direct Deimmunization ApproachKarina Winterling (Germany)

Recombinant, patient-derived FVIII-neutralising antibodies: a platform for research, product testing, and ex vivo modelling of haemophilia ACarmen Coxon (UK)

Towards the transplacental delivery of maternal FVIII to FVIII-deficient progeny for induction of active immune tolerance to therapeutic FVIIIAngelina Mimoun (France)

Immunotolerance induction in hemophilia(Part I)Chair: Maria Elisa Mancuso (Italy), Kate Pratt (USA)

How to maintain tolerance in hemophilia ASébastien Lacroix-Desmazes (France)

How to induce Treg responses in experimental hemophilia ANaro Biswas (USA)

Is it important to induce immune tolerance in the era of non-replacement therapies?Elena Santagostino (Italy)

Specific Treg therapy for hemophilia inhibitors: CARs versus BARs?David Scott (USA)

Walking Poster Session

SUNDAY

Von Willebrand factor and ADAMTS13 in hemostasis and thrombosisChairs: Ilaria Mancini (Italy), Karen Vanhoorelbeke (The Netherlands)

Von Willebrand factor self-association role in hemostasis and thrombosisJosé Lopez (USA)

Interactions of von Willebrand factor/ADAMTS13 Jim Crawley (UK)

Modulation of ADAMTS13 conformation (open/close) in TTP Karen Vanhoorelbeke (Belgium)

Impact of cohort studies on therapeutic approaches for von Willebrand diseaseJeroen Eikenboom (The Netherlands)

Update on 3WINTERSAugusto B. Federici (Italy)

Acquired von Willebrand diseaseGiancarlo Castaman (Italy)

Role of von Willebrand factor in the vessel wall: interplay with vascular smooth muscle cellsCécile Denis (France)

Oral Communications – von Willebrand Factor & ADAMTS 13Von Willebrand factor (VWF) production and secretion, and endothelial cell characteristics in healthy ECFCs; Generating a valid ex vivo model for von Willebrand disease (VWD)Suzan De Boer (The Netherlands)

Inhibition of ADAMTS13 prevents the loss of high molecular weight von Willebrand factor (VWF) multimers in an in vitro left ventricular assist deviceShannen Deconinck (Belgium)

Conformation of ADAMTS13 in the French cohort of child-onset thrombotic thrombocytopenic purpuraBérangère Joly (France)

Patient anti-ADAMTS13 autoantibodies induce an open ADAMTS13 conformation in immune mediated thrombotic thrombocytopenic purpuraElien Roose (France)

Tilting the balance: A trade-off between thrombotic thrombocytopenic purpura (TTP) patients autoantibody binding and proteolytic activity of ADAMTS13 variantsNuno Alexandre Gomes Graca (The Netherlands)

In-depth epitope mapping of anti-ADAMTS13 autoantibodies in immune-mediated thrombotic thrombocytopenic purpura patients using a large library of ADAMTS13 fragmentsKadri Kangro (Belgium)

Other clinical challenges of hemophiliaChairs: Massimo Morfini (Italy), Elena Santagostino (Italy)

Thrombosis in hemophilia: results from EUHASS Registry, including thrombotic microangiopathiesMike Makris (UK)

Ageing with hemophiliaPier Mannuccio Mannucci (Italy)

Eradication of HCV in EuropeMassimo Colombo (Italy)

Immunotolerance induction in hemophilia(Part II)Chairs: Sebastien Lacroix-Desmazes (France), Carmen Escuriola (Germany)

Gene-therapy-mediated tolerance inductionValter R. Arruda (USA)

A working group on new concepts for ITIKate Pratt (USA)

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