ESGCT and NVGCT Collaborative Congress, 23 - 26 October 2014 THURSDAY 23 OCTOBER 2014 17:00-18:30 ESGCT 2014 OPENING: Gerard Wagemaker Chairs: Luigi Naldini and Fulvio Mavilio Sponsor: GENETHON PLENARY SESSION 1: Highlights of Clinical Progress A. Biffi B. Gaspar M. Cavazzana A. Aiuti 18:30-20:00 WELCOME RECEPTION POSTER SESSION 1 FRIDAY 24 OCTOBER 2014 08:30-10:00 PLENARY SESSION 2: Highlights of Clinical Progress II Chair: Winald Gerritsen M. Grommeier J. Beadle T. Schumacher 10:00-10:30 10:30-12:30 S2a: Gene Editing Sponsor: TBC Chair: Toni Cathomen T. Cathomen A. Lombardo 12:30-14:30 14:30-16:00 RECEPTION Abstract Abstract Abstract Abstract 1 2 3 4 COFFEE BREAK S2b: Innate Immunity & S2c: Connective Tissue & Injury Tolerance Induction Repair Sponsor: Avalanche Sponsor: TBC Biotechnologies Chair: Hidde Haisma Chair: Hildegard Buning J. Huard T. Friedmann Abstract 1 M. Ressing F. Mingozzi Abstract Abstract Abstract Abstract Abstract 2 Abstract 3 Abstract 4 1 2 3 4 Sponsor: TBC S2d: Cardiovascular Diseases Sponsor: TBC Chair: Andy Baker MJ. Goumans H. Puccio Abstract Abstract Abstract Abstract 1 2 3 4 ESGCT Assembly LUNCH PLENARY SESSION 3: Emerging Technologies in Hematopoietic Stem Cell Gene Therapy Chair: David Williams Sponsor: TBC F. Zhang K. Joung M. Fusseneger 16:00-16:30 16:30-18:30 S3a: Genetic & Cellular Vaccines Sponsor: TBC Chair: TBC J. Wilson TBC Abstract Abstract Abstract Abstract 18:30-20:00 1 2 3 4 COFFEE BREAK S3b: Epigenetic Regulation S3c: Ocular Diseases Sponsor: TBC Sponsor: Oxford Biomedica Chair: TBC Chair: Robin Ali B. van Steensel D. Trono K. Zhang B. Roska Abstract Abstract Abstract Abstract 1 2 3 4 Abstract Abstract Abstract Abstract 1 2 3 4 POSTER SESSION 2 S3d: Ageing & Genetic Instability Syndromes Sponsor: EUROFANCOLEN Chair: TBC N. De Wind J. Bueren Abstract Abstract Abstract Abstract 1 2 3 4 ESGCT and NVGCT Collaborative Congress, 23 - 26 October 2014 20:00 SPEAKERS DINNER SATURDAY 25 OCTOBER 2014 8:30-10:00 PLENARY SESSION 4: Gene and Cell Therapy for Cancer Chairs: Len Seymour, Rob Hoeben H. Abken R. Morgan D. Kirn 10:00-10:30 10:30-12:30 S4a: RNA Therapeutics Sponsor: in CellArt Chair: Annemieke Aartsma-Rus A. Aartsma-Rus M. Gait Abstract Abstract Abstract Abstract 1 2 3 4 COFFEE BREAK S4b: Muscular & Metabolic S4c: Blood Coagulation Diseases Diseases Sponsor: Dimension Therapeutics Sponsor: UNIQURE Chair: Thierry VandenDriessche Chair: Gerard Wagemaker E. Tuddenham S. Qizen A. Buj Bello S. Waddington Abstract Abstract Abstract Abstract 12:30-14:00 S5a: Primary Immune Deficiencies Sponsor: Net4CGD Chair: Alessandro Aiuti, Harry Malech A. Thrasher D. Williams 1 2 3 4 17:00-17:30 S6a: Regenerative Medicine Sponsor: TBC Chair: Gerald de Haan J. Verhaagen M. Parmar Abstract Abstract Abstract Abstract Abstract Abstract Abstract Abstract 1 2 3 4 PLENARY SESSION 5: New Approaches to Engineering Vectors and Cells Chair: Gerald De Haan C. Mummery M. Kay P. Gregory Abstract Abstract Abstract Abstract 17:30-19:30 1 2 3 4 S4d: Immunotherapy of Cancer Sponsor: GE Healthcare Chair: Mirjam Heemskerk, Chiara Bonini A. Bondanza M. Heemskerk General Assembly Dutch Society LUNCH 14:00-15:00 15:00-17:00 Abstract Abstract Abstract Abstract 1 2 3 4 Sponsor: SillaJen 1 2 3 4 S5b: Neural Diseases Sponsor: Oxford Biomedica Chair: Joost Verhaagen N. Cartier-Lacave A. Gritti Abstract Abstract Abstract Abstract 1 2 3 4 S5c: Vectorology RNA Sponsor: TBC Chair: TBC G. Towers M. Schmitt Abstract Abstract Abstract Abstract 1 2 3 4 COFFEE BREAK S6b: Inflammatory & Autoimmune S6c: Oncolytic Therapy Diseases Sponsor: SAFC Sponsor: Arthrogen Chair: Len Seymour, Victor van Chair: Margriet Vervoordeldonk Beusechem R. Vries R. Bacchetta A. Hemminki C. Dirven Abstract Abstract Abstract Abstract 1 2 3 4 Abstract Abstract Abstract Abstract 1 2 3 4 Sponsor: SANGAMO S5d: Bioprocessing of C&G T Products Sponsor: Oxford Biomedica Chair: Otto Merten M. Hebben K. Kühlcke Abstract Abstract Abstract Abstract 1 2 3 4 S6d: Non-Viral Gene Therapy: Pharmacokinetics & Delivery Sponsor: TBC Chair: Zoltan Ivics L. Cooper M. Machluf Abstract Abstract Abstract Abstract 1 2 3 4 ESGCT and NVGCT Collaborative Congress, 23 - 26 October 2014 9:00-11:00 S7a: Exosomes and Microvesicles for Transfection Sponsor: ARTHROGEN Chair: Fons Vandeloo M. Wood TBC Abstract Abstract Abstract Abstract 11:00-11:30 11:30-13:30 1 2 3 4 SUNDAY 26 OCTOBER 2014 S7b: Infectious Diseases S7c: Vectorology, DNA Sponsor: TBC Sponsor: TBC Chair: Ben Berkhout, Luc van der Chair: Hildegard Büning Laan D. Schaffer S. Kochanek Abstract 1 B. Berkhout R. Fouchier Abstract Abstract Abstract Abstract 1 2 3 4 Abstract 2 Abstract 3 Abstract 4 S7d: Red Blood Cell Diseases Sponsor: Blue Bird Bio Chair: Juan Bueren M. Sadelain S. Hacein Bey Abstract Abstract Abstract Abstract 1 2 3 4 COFFEE BREAK PRESIDENTIAL SYMPOSIUM AWARDS CEREMONY Chair: Luigi Naldini ,Gerard Wagemaker, Fatiha Sadallah PRESIDENTIAL CONFERENCE Shinya Yamanaka Introduction about IMI (Fatiha Sadallah), Stembancc (speaker TBC), EBISC (speaker TBC) (30 minutes) Young Investigator Awards SPONSORED BY GENETHON Outstanding Achievement Award SPONSORED BY HUMAN GENE THERAPY Sponsor:IMI