Non-replacement therapies in the hemophilias: Update
Chairs: Peter Lenting (France), Jan Voorberg (The Netherlands)
09.00
Fitusiran
Steve Pipe (USA)
09.30
APC Serpin
James Huntington (UK)
10.00
Anti-TFPI
Alan Mast (USA)
10.30
Thrombotic microangiopathy associated with emicizumab: views from an insider
Paul Coppo (France)
11.00
Break
Novel drugs
Chair: Paul Coppo (France)
11.30
Caplacizumab: results of phase II/phase III studies
Marie A. Scully (UK)
Satellite Lecture:
Treatment of bleeding episodes in the era of non-factor replacement therapies
Sponsored by Novo Nordisk
12:00
(not accredited for CME)
Chair: Pratima Chowdary (UK)
Welcome & introduction
Pratima Chowdary (UK)
Concomitant use of rFVIIa and emicizumab in congenital haemophilia A with inhibitors: Safety data from the HAVEN clinical programme
Stephanie Seremetis (USA)
Subcutaneous prophylaxis with concizumab in patients with haemophilia A and haemophilia A/B with inhibitors: Phase 2 trial results
Giancarlo Castaman (Italy)
Q&A Discussion & final remarks
All
Satellite Lecture:
From trials to clinical practice: emergent treatments to overcome challenges in people with haemophilia A
Sponsored by Roche
12.30
(not accredited for CME)
Chair: Flora Peyvandi (Italy)
Speaker: Amy Shapiro (USA)
Lunch Satellite Symposium:
Can we improve inhibitor management in patients with haemophilia A? – Clinical approaches to inhibitor prevention and elimination
Sponsored by Octapharma
13.00
(not accredited for CME)
Chair: Jan Astermark (Sweden)
The challenge of inhibitors in haemophilia A – Introduction
Jan Astermark (Sweden)
Immune tolerance induction in haemophilia A – challenges, experience and future approaches
Carmen Escuriola (Germany)
ITI in the context of new therapies for haemophilia A: The Atlanta and MOTIVATE studies
Robert Sidonio (USA)
Final results from the NuProtect study of Nuwiq in previously untreated patients
Ri Liesner (UK)
Q&A, Concluding remarks
All
Gene Therapy
Chairs: Steve Pipe (USA), Amy Shapiro (USA)
14.00
Phagocytosis-shielded lentiviral vectors for hemophilia gene therapy
Alessio Cantore (Italy)
14.30
Liver toxicity in gene therapy
Edward Tuddenham (UK)
Oral Communications – Hemophilia
15.00
Targeting of hepatocyte subpopulation contributing to liver post-natal growth is crucial for maintenance of transgene expression in liver-directed gene therapy
Michela Milani (Italy)
Transplantation of fetal liver cells into newborn hemophilic mice for the treatment of hemophilia A without inhibitors formation
Simone 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 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)
Towards the transplacental delivery of maternal FVIII to FVIII-deficient progeny for induction of active immune tolerance to therapeutic FVIII
Angelina Mimoun (France)
16.00
Break
Immunotolerance induction in hemophilia (Part I)
Chairs: Maria Elisa Mancuso (Italy), Kate Pratt (USA)
16.30
How to maintain tolerance in hemophilia A
Sébastien Lacroix-Desmazes (France)
17.00
How to induce Treg responses in experimental hemophilia A
Naro Biswas (USA)
17.30
Is it important to induce immune tolerance in the era of non-replacement therapies?
Elena Santagostino (Italy)
18.00
Specific Treg therapy for hemophilia inhibitors: CARs versus BARs?
David Scott (USA)
18.30
Walking Poster Session
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