10TH BIC INTERNATIONAL CONFERENCE 10TH BIC INTERNATIONAL CONFERENCE AUSPICES COMUNE DI GENOVA ORGANIZED BY SCIENTIFIC COMMITTEE CHAIRED BY: P.M. MANNUCCI, Italy F. PEYVANDI, Italy N. CIAVARELLA, Italy A.B. FEDERICI, Italy S. LACROIX-DESMAZES, France P.J. LENTING, France D. LILLICRAP, Canada J. VOORBERG, The Netherlands 100% 08:30 Activity Feed Schedule Speakers Sponsors Maps About General Info Attendees Social Events Search FOLLOW US DOWNLOAD NOW ON FACEBOOK & TWITTER THE SMC MEDIA EVENTS APP BICinternationalconferencehemophilia BIC_hemophilia 10TH BIC INTERNATIONAL CONFERENCE The major innovations that were presented at the most recent events suggest that 2019 is a year of continuous development in treatment approaches and new drugs. Data from basic science research and clinical trials are outlining new scenarios, not only in gene therapy but also in the progress of replacement and non-replacement therapies with new drugs appearing in the market. This joint meeting is meant to take the best of BIC & INHIBITORS International Conferences and feature only the most novel advances of basic science and clinical research in hemophilia, rare inherited coagulation disorders, von Willebrand factor and von Willebrand disease, gene therapy, the thrombotic microangiopathies (TTP, HUS and aHUS) and, of course, factor VIII inhibitors. The most distinguished experts in the field will present their very latest data and offer their wisdom and insight into the current science developments, during a 3-day programme. We wish to welcome clinical and basic science researchers to the Joint 10th BIC & 3rd Inhibitors in Hemophilia International Conference and look forward to interacting with them throughout the whole 3-day period. The Scientific Committee P.M. Mannucci, F. Peyvandi N. Ciavarella, A.B. Federici, S. Lacroix-Desmazes, P. Lenting, D. Lillicrap, J. Voorberg 6TH SEPTEMBER Replacement therapies in hemophilia 12:00 European pharmacovigilance of new A and B - Efficacy of extended half-life products (with special emphasis on products pegylated products) Chairs: David Lillicrap (Canada), Anneliese Hilger (EMA, Germany) Pier Mannuccio Mannucci (Italy) 12:30 Immunogenicity of subcutaneous and 08:30 Real life data: real benefits. What intravenous drugs have we achieved? David Lillicrap (Canada) Pratima Chowdari (UK) 13:00 Lunch Satellite Symposium: 09:00 How to optimize the use of extended Evolution by design: advancing the half-life products (PK analysis) standard of care in hemophilia A Alfonso Iorio (Canada) Sponsored by Bayer (not accredited for CME) Safety of replacement therapies Chair: Erik Berntorp (Sweden) (standard and novel) (Part I) Chairs: Nicola Ciavarella (Italy), How evolution leads to revolution in Maria Elisa Mancuso (Italy) hemophilia care Erik Berntorp (Sweden) 09:30 Which bleeding phenotype can we expect in severe hemophilia A Advances driven by evolution: previously untreated patients treated investigating the role of factor VIII with non replacement therapies? (FVIII) in coagulation and beyond Karin Fijn van Draat (The Netherlands) Hermann Eichler (Germany) 10:00 How safety of novel anti-hemophilic Advances driven by design: using real- drugs should be approached. Known life practice to guide clinical studies and unknown adverse effects (results from PROTECT VIII) Flora Peyvandi (Italy) Elena Santagostino (Italy) 10:30 Are Fc - factor VIII (FVIII) products Q&A less immunogenic? New clinical data? Christoph Königs (Germany) 14:00 Satellite Symposium: ADAMTS13 and von Willebrand factor 11:00 Break (VWF): progressing patient care through protein innovation Safety of replacement therapies Sponsored by Takeda (standard and novel) (Part II) (not accredited for CME) Chairs: Giovanni Di Minno (Italy), Chair: Flora Peyvandi (Italy) Mauro Biffoni (Italy) Welcome and introduction 11:30 Long-term side-effects of pegylated Flora Peyvandi (Italy) products: facts or fancies? Silvio Garattini (Italy) The ADAMTS13-von Willebrand factor 16:00 Keynote Lecture “Choice of assays for (VWF) interaction: clinical implications novel anti-hemophilic therapies” & applications Armando Tripodi (Italy) Peter Lenting (France) Chair: Wolfgang Schramm (Germany) Congenital thrombotic The role of von Willebrand thrombocytopenic purpura (cTTP) as factor (VWF) in factor VIII (FVIII) a chronic disease: what is the long- immunogenicity term view to achieve more optimal Chairs: Giancarlo Castaman (Italy), outcomes? Augusto B. Federici (Italy) Paul Coppo (France) 16:30 Factor VIII (FVIII) peptide The role of von Willebrand factor (VWF) presentation to the immune system; in individualized care for patients with modulation by von Willebrand factor von Willebrand disease (VWD) (VWF) Andreas Tiede (Germany) Jan Voorberg (The Netherlands) Panel discussion 17:00 High von Willebrand factor (VWF) Facilitated by Flora Peyvandi (Italy) affinity factor VIII (FVIII) and immunogenicity Meeting summary and close Peter Lenting (France) Flora Peyvandi (Italy) 17:30 Walking Poster Session 15:00 Satellite Lecture: See ePoster programme for details Von Willebrand factor (VWF) and factor VIII (FVIII): an unending love affair Sponsored by Kedrion (not accredited for CME) Chair: Prasad Mathew (USA) Setting the stage Prasad Mathew (USA) Clinical implications of this love story for patients with hemophilia A Maria Elisa Mancuso (Italy) Q&A 15:30 Break Scientific session Oral communications and poster session Satellite symposium/lecture 7TH SEPTEMBER Non-replacement therapies in the Subcutaneous prophylaxis with hemophilias: update concizumab in patients with Chairs: Peter Lenting (France), haemophilia A and haemophilia A/B Jan Voorberg (The Netherlands) with inhibitors: Phase 2 trial results Giancarlo Castaman (Italy) 09:00 Fitusiran Steven Pipe (USA) Q&A 09:30 APC serpin 12:30 Satellite Lecture: James Huntington (UK) From trials to clinical practice: emergent treatments to overcome challenges in 10:00 Anti-TFPI people with haemophilia A Alan Mast (USA) Sponsored by Roche (not accredited for CME) 10:30 Thrombotic microangiopathy Chair: Flora Peyvandi (Italy) associated with emicizumab: views Speaker: Amy Shapiro (USA) from an insider Paul Coppo (France) 13:00 Lunch Satellite Symposium: Can we improve inhibitor management 11:00 Break in patients with haemophilia A? Clinical approaches to inhibitor Novel drugs prevention and elimination Chair: Paul Coppo (France) Sponsored by Octapharma (not accredited for CME) 11:30 Caplacizumab: results of phase II/ Chair: Jan Astermark (Sweden) phase III studies Marie A. Scully (UK) The challenge of inhibitors in haemophilia A - Introduction 12:00 Satellite Lecture: Jan Astermark (Sweden) Treatment of bleeding episodes in the era of non-factor replacement Immune tolerance induction therapies in haemophilia A - challenges, Sponsored by Novo Nordisk experience and future approaches (not accredited for CME) Carmen Escuriola (Germany) Chair: Pratima Chowdary (UK) ITI in the context of new therapies Welcome & introduction for haemophilia A: the Atlanta and Pratima Chowdary (UK) MOTIVATE studies Robert Sidonio (USA) Concomitant use of rFVIIa and emicizumab in congenital haemophilia Final results from the NuProtect study A with inhibitors: safety data from the of Nuwiq® in previously untreated HAVEN clinical programme patients Stephanie Seremetis (USA) Ri Liesner (UK) Q&A Gene Therapy Towards the transplacental delivery Chairs: Steven Pipe (USA), of maternal FVIII to FVIII-deficient Amy Shapiro (USA) progeny for induction of active immune tolerance to therapeutic FVIII 14:00 Phagocytosis-shielded lentiviral Angelina Mimoun (France) vectors for hemophilia gene therapy Alessio Cantore (Italy) 16:00 Break 14:30 Liver toxicity in gene therapy Immunotolerance induction in Edward Tuddenham (UK) hemophilia (Part I) Chairs: Maria Elisa Mancuso (Italy), 15:00 Oral Communications - Hemophilia Kathleen Pratt (USA) Targeting of hepatocyte 16:30 How to maintain tolerance in subpopulation contributing to liver hemophilia A post-natal growth is crucial for Sébastien Lacroix-Desmazes (France) maintenance of transgene expression in liver-directed gene therapy 17:00 How to induce Treg responses in Michela Milani (Italy) experimental hemophilia A Naro Biswas (USA) Transplantation of fetal liver cells into newborn hemophilic mice for the 17:30 Is it important to induce immune treatment of hemophilia A without tolerance in the era of non- inhibitors formation replacement therapies? Simone Merlin (Italy) Elena Santagostino (Italy) B-AMAZE, a Phase 1/2 trial of a novel 18:00 Specific Treg therapy for hemophilia investigational adeno associated inhibitors: CARs versus BARs? virus (AAV) gene therapy (FLT180a) David Scott (USA) in subjects with severe or moderately severe hemophilia B (HB) 18:30 Walking Poster Session Pratima Chowdary (UK) See ePoster programme for details Developing a novel coagulation factor VIII with reduced immunogenicity by a direct deimmunization approach Karina Winterling (Germany) Recombinant, patient-derived FVIII- neutralising antibodies: a platform for research, product testing, and ex vivo modelling of haemophilia A Carmen Coxon (UK) Scientific session Oral communications and poster session Satellite symposium/lecture 8TH SEPTEMBER Von Willebrand factor (VWF) and 14:00 Oral Communications - Von Willebrand ADAMTS13 in hemostasis and factor (VWF) and ADAMTS 13 thrombosis (Part I) Chairs: Ilaria Mancini (Italy), Von Willebrand factor (VWF) production Karen Vanhoorelbeke (Belgium) and secretion, and endothelial cell characteristics in healthy ECFCs; 09:00