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, 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 Sponsor: HUMAN GENE THERAPY FRIDAY 24 OCTOBER 2014 08:30-10:00 PLENARY SESSION 2: Highlights of Clinical Progress II Chairs: David Kirn, Len Seymour D. Kirn K. Fisher T. Schumacher 10:00-10:30 10:30-12:30 S2a: AAV Vectors Sponsor: 4D Molecular Therapeutics Chair: Hildegard Büning, D. Schaffer 12:30-14:30 14:30-16:00 RECEPTION D. Schaffer L. Vandenberghe R. Münch OR084 LA. Zhang OR85 A. Majowicz OR086 B. Cogné OR087 COFFEE BREAK S2b: Innate Immunity and Stem Cell S2c: Muscle Gene and Cell Therapy Therapy Sponsor: Avalanche Biotechnologies Sponsor: AFM Chair: Federicco Mingozzi, Axel Chair: Hidde Haisma, John Huard Schambach J. Huard A. Buj-Bello G. Dickson who will do the talk!!! M. Ressing F. Mingozzi D. Hoffmann G. Farinelli HT. Lin T. Moritz Chair: Seppo Ylä-Herttuala, Sarah Ferber MJ. Goumans H. Puccio J. Laakkonen L. Rodriguez-Borlado C. Lang N. Lachmann PLENARY SESSION 3: Emerging Technologies in Hematopoietic Stem Cell Gene Therapy Chair: Luigi Naldini Keith Joung K. Joung F. Zhang M. Fusseneger S3a: Genetic Vaccines and Ad Vectors Sponsor: Omnia Biologics Chair: Kerry Fisher, Dale Vanderputten J. Wilson S. Kochaneck V. Cerullo C. Bauche S. van de Wall W. Dong 18:30-20:00 P. Spitali C. Le Guiner S2d: Cardiovascular and Pulmonary Diseases ESGCT Assembly LUNCH 16:00-16:30 16:30-18:30 Sponsor: SILLAJEN COFFEE BREAK S3b: Epigenetic Regulation S3c: Gene And Cell Therapy for and Gene Editing II Ocular Diseases Sponsor: Supersist Sponsor: Oxford Biomedica Chair: Didier Trono, Hidde Chair: Robin Ali, Kyriacos Haisma Mitrophanous B. van Steensel D. Trono K. Zhang B. Roska P. Genovese Y. Cai U. Mock MJ. Pino-Barrio D. Fischer C. Gras I. Trapani S. Ellis POSTER SESSION 2 Sponsor: EDITAS S3d: Genetic Instability Syndromes and Vector Genotoxicity Sponsor: EUROFANCOLEN Chair: Juan Bueren, Christof von Kalle N. de Wind R. Heilbronn A. Calabria K. Petri S. Wilkening J. Bueren ESGCT and NVGCT Collaborative Congress, 23 - 26 October 2014 SATURDAY 25 OCTOBER 2014 8:30-10:00 PLENARY SESSION 4: Gene and Cell Therapy for Cancer Chairs: Rob Hoeben, Victor van Beusechem H. Abken R. Morgan S. Russel 10:00-10:30 10:30-12:30 S4a: RNA Therapeutics Sponsor: in CellArt Chair: Annemieke Aartsma-Rus, Bruno Pitard A. Aartsma-Rus M. Gait T. Beissert K. Breckpot D. Grimm K. Itaka COFFEE BREAK S4b: Gene and Cell Therapy for S4c: Blood Coagulation Diseases Metabolic Diseases Sponsor: UNIQURE Sponsor: Dimension Therapeutics Chair: Gerard Wagemaker, Harald Chair: Sam Wadsworth, Z. Ivics Petri S. Waddington Niek van Til A. Nathwani S. Qizen G. Gonzalez-Aseguinolaza V. Haurigot M. Kuroda R. Yadak 12:30-14:00 Sponsor: GE Healthcare Chair: Mirjam Heemskerk, Atilio Bondanza A. Bondanza M. Heemskerk S. Guerdan Carrio S. Mastaglio R. Mezzarda M. Pont PLENARY SESSION 5: New Approaches to Engineering Vectors and Cells Chair: Luigi Naldini, Phil Gregory C. Mummery P. Gregory M. Kay S5a: Primary Immune Deficiencies Sponsor: Net4CGD, CELLPID Chair: Alessandro Aiuti, Harry Malech A. Thrasher D. Williams B. Davis E. Almarza K. Pike-Overzet AS. Wiekmeijer 17:00-17:30 17:30-19:30 S4d: Immunotherapy of Cancer I General Assembly Dutch Society LUNCH 14:00-15:00 15:00-17:00 B. Nijmeijer E. Verhoeyen A. Follenzi Sponsor: BlueBird Bio S6a: Regenerative Medicine Sponsor: Pall Life Science Chair: Joost Verhaagen, Nathalie Cartier or PALL TBC M. Parmar J. Verhaagen M. Machluf D. D’Avola M. Quattrocelli S5b: Gene and Cell Therapy for Neural Diseases Sponsor: Oxford Biomedica Chair: Joost Verhaagen, Nathalie Cartier N. Cartier-Lacave A. Gritti MG. Biferi K. Kozarsky K. Mitrophanous M. Barkats S5c: RNA Vectors Chair: Sarah Ferber, Manfred Schmidt S5d: Bioprocessing of Cell &Gene Therapy Products Sponsor: Oxford Biomedica Chair: Otto Merten, James Miskin M. Hebben G. Towers M. Schmidt A. Cooper D. Gill S. El Ashkar C. Levy COFFEE BREAK S6b: Inflammatory & Autoimmune S6c: Oncolytic Therapies Diseases Sponsor: Arthrogen Sponsor: SAFC Chair: Margriet Vervoordeldonk, Chair: Len Seymour, Victor van Rosa Bacchetta Beusechem R. Vries R. Bacchetta A. Hemminki C. Dirven S. Casacuberta Serra S. Snoek F. van de Loo Sponsor: SANGAMO C. Engeland D. van den Wollenberg LA. Rojas Exposito R. Hernandez K. Kühlcke E. Kotsopoulo A. Sauer I. Johnston H. Lesch S6d: Immunotherapy of Cancer II Chair: Zoltan Ivics, Laurence Cooper L. Cooper M. Casucci A. Rodriguez Garcia J. Suerth W. Uckert C. Wiek D. Yu ESGCT and NVGCT Collaborative Congress, 23 - 26 October 2014 9:00-11:00 S7a: Exosomes and Non-Viral Delivery Sponsor: ARTHROGEN Chair: Fons van de Loo, Enrico Mastrobattista M.Wauben Pieter Vader M. Gayral U. Griesenbach P. Müller K. Raemdonc 11:00-11:30 11:30-13:30 SUNDAY 26 OCTOBER 2014 S7b: Infectious Diseases Sponsor: BD Biosciences Chair: Ben Berkhout, Luc van der Laan R. Fouchier B. Berkhout E. Senis Herrero D. Cesana M. Holmes S7c Gene Editing I Sponsor: Supersist, Blue Bird Bio Chair: Toni Cathomen, Christof von Kalle T. Cathomen A. Lombardo K. Börner P. Fatouros I. Maggio R. Torres S7d: Haematopoietic Stem Cell and HSC Gene Therapy Sponsor: Bluebird Bio Chair: Juan Bueren, Nathalie Cartier S. Hacein-‐ Bey-‐ Abina L. Maouche-Chrétien A. Aprile M. Fernandez-Garcia S. Kohlscheen MR. Lidonnici V. Poletti COFFEE BREAK PRESIDENTIAL SYMPOSIUM AWARDS CEREMONY Sponsor: Innovative Medicines Initiative (IMI), EBISC, Stembancc Chair: Luigi Naldini, Gerard Wagemaker, Fatiha Sadallah PRESIDENTIAL CONFERENCE Shinya Yamanaka Introduction about IMI (TBC), Stembancc (Martin Graff), EBISC (Tim Allsopp) (30 minutes) Volker Busskamp (Young Investigator Award) Vincenzo Cerullo (Young Investigator Award) SPONSORED BY GENETHON Manuel Grez (Outstanding Achievement Award) SPONSORED BY HUMAN GENE THERAPY Presentation of new president and 2015 Congress