PRELIMINARY PROGRAMME

Gene therapy

Chairs: Flora Peyvandi (Italy), Antonia Follenzi (Italy)

09:00

Gene therapy in haemophilia A and B: summary
David Lillicrap (Canada)

09:20

FVIII-induced endoplasmic reticulum (ER) stress in the context of gene therapy
Randal J. Kaufman (US)

09:40

Lentiviral factor VIII gene therapy: where we are standing
Luigi Naldini (Italy)

10:00

Discussion

10:10

Round table “Gene therapy: calculating the unknown”
Chairs: Flora Peyvandi (Italy)
Discussants: Declan Noone (Ireland), Brian O’Mahony (Ireland), Glenn Pierce (US)

10:40

Coffee break

Haemophilia

Chairs: Nicola Ciavarella (Italy), Angiola Rocino (Italy)

11:10

Engineering of novel factor VIII molecules: how we could improve the next molecules
Lindsey George (US)

11:30

Nanobodies as diagnostic and therapeutic tools in haemostasis
Peter Lenting (France)

11:50

Discussion

Satellite Lecture

12:00

Roche

Satellite Lecture

12:30

Sanofi Genzyme

13:00

Lunch

Satellite Symposium

14:00

Takeda

Oral Communications

Chairs: Cecile Denis (France), Augusto B. Federici (Italy)

15:00

Transplacental delivery of maternal FVIII for induction of FVIII-specific immune tolerance
Angelina Mimoun (France)

15:10

N-glycan shielded rhADAMTS13 for treatment of immune thrombotic thrombocytopenic purpura
Nuno Graca (The Netherlands)

15:20

Anti-cysteine/spacer autoantibodies that open the conformation of ADAMTS13 are a common feature of the autoimmune response in immune-mediated thrombotic thrombocytopenic purpura
Laure De Waele (Belgium)

15:30

From coagulation to angiogenesis: possible role of factor VIII in endothelial functionality
Cristina Olgasi (Italy)

15:40

Syntaxin 5 is essential for Golgi integrity, Weibel-Palade body biogenesis and stimulus-induced von Willebrand factor secretion from endothelial cells
Marije Kat (The Netherlands)

15:50

Developing a model for studying von Willebrand disease with hiPSC-derived endothelial cells
Suzan De Boer (The Netherlands)

16:00

Coffee break

Non replacement therapies: novel drugs

Chairs: Mike Makris (UK), Jan Voorberg (The Netherlands)

16:30

Antithrombin knockdown: tipping the balance of haemostasis and thrombosis
Margaret V. Ragni (US)

16:50

Rebalancing haemostasis by inhibiting Activated Protein C (APC)
Jim Huntington (UK)

17:10

Discussion

17:20

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

17.30

E-POSTER SESSION