Full programme
OTHER STRATEGIES FOR GENE THERAPY
Chairs: Peter Lenting (France), David Lillicrap (Canada)
9:00-9:20
Lentiviral vector transduced autologous hematopoietic stem cell-based gene therapy for haemophilia A
Alok Srivastava (India)
9:20-9:40
Lentivirus in haemophilia B
Luigi Naldini (Italy)
9:40-10:00
Non-viral strategies for gene therapy
David Lillicrap (Canada)
10:00-10:15
Discussion
10:15-10:45
Coffee break
JOINT AND BONE HEALTH IN HAEMOPHILIAS
Chairs: Roberta Gualtierotti (Italy), Jan Voorberg (The Netherlands)
10:45-11:05
Open questions for joint health
Pratima Chowdary (UK)
11:05-11:25
Artificial intelligence and telemedicine for joint health in haemophilia patients
Roberta Gualtierotti (Italy), Sergio Mascetti (Italy)
11:25-11:45
The role of factor VIII in bone physiology
Raimondo De Cristofaro (Italy)
11:45-12:00
Discussion
SATELLITE LECTURE
12:00-12:30
Sponsored by CSL Behring
PROGRAMME
12:30-14:00
Lunch
SATELLITE LECTURE
14:00-14:30
Sponsored by Roche
PROGRAMME
NEW CLINICAL ASPECTS
Chairs: Pier Mannuccio Mannucci (Italy), Mike Makris (UK)
14:30-14:50
The use of antithrombotic drugs in haemophilia
Roger Schutgens (The Netherlands)
14:50-15:10
Prophylaxis in infants with haemophilia A/B using bispecific antibodies
Karin Fijn van Draat (The Netherlands)
15:10-15:30
The clinician’s choice: which patient is suitable for which therapy?
Steven Pipe (US)
15:30-15:45
Discussion
15:45-16:10
Coffee break
KEYNOTE LECTURE
Chair: Augusto B. Federici (Italy)
16:10-16:30
Von Willebrand factor functions outside haemostasis
James O’Donnell (Ireland)
ORAL COMMUNICATIONS
Chairs: CécileDenis (France), Jan Voorberg (The Netherlands)
16:30-16:40
Estimating the population-based prevalence of congenital TTP using large-scale genetic databases
Omid Seidizadeh (Italy)
16:40-16:50
Poor FcRn-mediated recycling accounts for the limited increase in FVIIIFc half-life
Alejandra Reyes Ruiz (France)
16:50-17:00
Exploring red blood cells as a novel tolerogenic approach for factor VIII inhibitors employing immuno-dominant FVIII-derived peptides presented on MHC class II
Mariarosaria Miranda (The Netherlands)
17:00-17:10
Immune tolerance promotion by LSEC-specific lentiviral vector-mediated delivery of the FVIII transgene regulated by the stabilin-2 promoter
Antonia Follenzi (Italy)
17:10-17:20
Amelioration of a von Willebrand disease type 2B phenotype in vivo using allele-selective small interfering RNAs
Noa Linthorst (The Netherlands)
17:20-17:30
Lentiviral vectors bearing the baculovirus GP64 surface protein efficiently transduce liver endothelial cells in vivo and correct mouse haemophilia A
Michela Milani (Italy)
17:30-18:30
E-POSTER SESSION