ESGCT/NVGCT COLLABORATIVE CONGRESS 2014 PROGRAMME PROGRAMME THURSDAY 23 OCTOBER 2014 THURSDAY 23 OCTOBER 2014 EDUCATION AND PATIENTS’ DAY 42 EDUCATION AND PATIENTS’ DAY 09.30 Amazon Opening lecture Chairs: Len Seymour, Sam Wadsworth Opening and welcome Sam Wadsworth, Dimension Therapeutics Overview of the current state of the art in gene and cell therapy Len Seymour, University of Oxford Amazon Education session 1 INV001 and INV002 Lessons learned for therapy development for Duchenne Muscular Dystrophy Annemieke Aartsma-Rus, Leiden University Medical Centre; Elizabeth Vroom, Duchenne Parent Project Netherlands 10.45 Morning break – Amazon foyer 11.15 Education session 2a: Technologies: general overviews evolutionary approaches Yangtze 2 Chair: Hildegard Büning Education session 2b: Technologies: selected topics Yangtze 1 Chair: Mirjam Heemskerk Education session 2c: Patient perspectives: hemophilia & liver diseases Amazon Chair: Piter Bosma INV003 Viruses with good intentions: developing oncolytic agents for therapy of cancer Rob Hoeben, Leiden University Medical Centre INV005 Redirecting T-cells by chimeric antigen receptors Hinrich Abken, University of Cologne INV007 Clinical advances in gene therapy from bench to bedside: a patient’s perspective Adam Jones, University of Sunderland INV004 Directed evolution approaches – a powerful technology to improve Adenoassociated viral vectors Hildegard Büning, University of Cologne INV006 Genetic information exchange by vesicles Michiel Pegtel, VU University, Amsterdam INV008 Gene therapy for hemophilia Frank Leebeek, Erasmus Medical Centre, Rotterdam 12.15 Lunch: round table discussions – Amazon foyer INV009 Towards a cure for Crigler-Najjar syndrome: a collaborate effort of researchers and patients Piter Bosma, AMC Hospital, Amsterdam 13.45 15.00 Amazon Education session 3a: Technologies: general overviews Yangtze 2 Chair: Angelo Lombardo Education session 3b: Technologies: selected topics Yangtze 1 Chairs: Axel Schambach, Claudia Waskow Education session 3c: Patient perspectives: acquired diseases Amazon Chair: Rob Hoeben INV010 Exploiting artificial nucleases for targeted genome editing in human cells Angelo Lombardo, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan INV012 Opening-up the stem cell niche for human hematopoietic stem cells in the mouse – an optimal model to study human hematopoiesis in vivo. Claudia Waskow, Technische Universität Dresden INV014 Oncolytic adenoviral therapy to enhance the cure rate for patients with localised prostate cancer Chris Bangma, Erasmus Medical Centre, Rotterdam INV011 Gene expression switches Ben Berkhout, University of Amsterdam INV013 Retro- and lentiviral vectors for gene therapy: from basic biology to clinical application Axel Schambach, Hannover Medical School INV015 Gene therapy for arthritis Margriet Vervoordeldonk, Arthrogen BV, Amsterdam Closing Chair: Len Seymour INV016 Cystic Fibrosis patients taking the lead Jacquelien Noordhoek, NCFS Dutch Cystic Fibrosis Foundation, Baarn; VU University, Amsterdam INV017 Organoid cultures for developing Cystic Fibrosis therapies Jeffrey Beekman, University Medical Centre, Utrecht INV018 European Union support to gene and cell therapy research in Horizon 2020 (2014–2020) David Gancberg, European Commission, Brussels INV019 Writing a Horizon 2020 application: do’s and don’ts Ellen Schenk, Erasmus Medical Centre, Rotterdam 16.30 Afternoon break – Amazon foyer 43