9:00-9:20

Challenges in the classification and nomenclature of different forms of microangiopathies
Marina Vivarelli (Italy)

9:20-9:40

Novel mechanism underlying the pathogenesis of immune thrombotic thrombocytopenic purpura (aTTP)
Long Zheng (US)

9:40-10:00

Anti von Willebrand factor therapies in immune thrombotic thrombocytopenic purpura (iTTP): now and then
Paul Coppo (France)

10:00-10:15

Discussion

10:15-10:40

The loss of tolerance in patients who previously underwent immune tolerance induction (ITI)
Sébastien Lacroix-Desmazes (France)

10:40-11:10

Coffee break

11:10-11:30

Bispecific antibodies in acquired haemophilia A
Robert Klamroth (Germany)

11:30-11:50

Experimental and clinical rationale for the use of bispecific antibodies in von Willebrand disease
Caterina Casari (France)

11:50-12:00

Discussion

12:00-13:00

Sponsored by Sanofi
PROGRAMME

13:00-14:30

Lunch

14:30-15:00

Sponsored by BioMarin
PROGRAMME

15:00-15:10

Immunogenicity assessment of efanesoctocog alfa: results from the phase 3 XTEND-1 study
Christoph Königs (Germany)

15:10-15:20

Evaluation of the effect of early prophylactic corticosteroid administration with adeno- associated viral gene therapy in the haemophilia A dog model
David Lillicrap (Canada)

15:20-15:30

Sustained high level expression of FVII following AAV-mediated gene delivery in vivo
Andrea D’Amico (The Netherlands)

15:30-15:40

Interindividual variability of response to gene therapy in haemophilia B: ranges of factor IX increase after 24 months in HOPE-B etranacogene dezaparvovec trial
Steven Pipe (US)

16:30-16:45

Coffee break

16:45-17:30

ROUND TABLE “Where are we and what will be the future of gene therapy?“
Chairs: Flora Peyvandi (Italy), Mike Makris (UK)
Discussants: Brian O’Mahony (Ireland), Glenn Pierce (US)

17:30

Wrap up and closing remarks
Flora Peyvandi (Italy), Pier Mannuccio Mannucci (Italy)