Preliminary programme
Please, note that the Conference agenda may still undergo minor changes due to organizational reasons.
GENE THERAPY
Chairs: Wolfgang Miesbach (Germany), Glenn Pierce (USA)
09:00-09:20
Efficacy and safety of gene therapy with AAV
Paul Batty (UK)
09:20-09:40
Where we are with lentiviral vectors in bleeding disorders
Alessio Cantore (Italy)
09:40-10:00
Liver health and AAV gene therapy
Vincenzo La Mura (Italy)
10:00-10:20
Immune tolerance induction by gene therapy
Roland Herzog (USA)
10:20-10:35
Discussion
10:35-11:05
Coffee break
11:05-11:25
The prospects of genome editing to treat inherited coagulation disorders
Gregory Newby (USA)
11:25-11:45
Gene therapy in factor VII deficiency and long term data on FIX deficiency with AAV
Amit Nathwani (UK)
11:45-12:00
Discussion
SATELLITE SYMPOSIUM
12:00-13:00
Sponsored by Octapharma
PROGRAMME
13:00-14:00
Lunch
SATELLITE SYMPOSIUM
14:00-15:00
Sponsored by Takeda
PROGRAMME
ORAL COMMUNICATIONS
Chairs: Augusto B. Federici (Italy), Riitta Lassila (Finland)
15:00-15:10
Comparison of a newly-formulated DDAVP to Stimate in persons with type 1 von Willebrand disease
Amy Shapiro (USA)
15:10-15:20
Lentiviral vector-based innovative gene therapy approaches for hemophilia A
Michela Milani (Italy)
15:20-15:30
Phase I study of VGA039 in individuals with von Willebrand disease demonstrates sustained drug concentrations, increased thrombin generation, and decreased bleeding after single subcutaneous injection
Carolyn Millar (UK)
15:30-15:40
Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors: Results from a phase 1/2 trial
Konstantia-Maria Chavele (UK)
15:40-15:50
Targeting antigen-specific B cells in iTTP using modular CAR T cells
Sara Rovira Puig (Belgium)
15:50-16:00
Efficacy and safety update of giroctocogene fitelparvovec in adults with moderately severe to severe hemophilia A: results from the phase 3 AFFINE gene therapy trial
Davide Matino (Canada)
16:00-16:30
Coffee break
VWD – CLINICAL SESSION
Chairs: Giancarlo Castaman (Italy), Cécile Denis (France)
16:30-16:50
DDAVP in VWD: how to predict the response
Frank Leebeek (the Netherlands)
16:50-17:10
Personalized prophylaxis for VWD
David Lillicrap (Kingston, ON, Canada)
17:10-17:30
Type 1 VWD: do they bleed? How can we assess their bleeding tendency?
Omid Seidizadeh (Italy)
17:30-17:45
Discussion
17:45-18:45
E-POSTER SESSION
PROGRAMME