DRUG DISCOVERY ‘15 TELFORD INTERNATIONAL CONFERENCE CENTRE 2ND - 3RD SEPTEMBER 2015 www.elrig.org #ELRIGDD15 CELL AND GENE THERAPY Thursday September 3rd Room: Beckbury Gene- and cell-based therapies are fast-growing forms of personalized medicine that rely on steady advances in gene transfer technologies, stem cell manipulation and the growing capacity for genetic reprogramming of mammalian cells. They may fulfill the promise of safe and effective therapies for currently untreatable diseases. The session will describe the latest developments and the crucial challenges for this field, touching on basic science, pre-clinical and clinical studies, with an emphasis on gene therapy approaches for blood, ocular and neurodegenerative disorders, and with a special insight in novel approaches aimed to generate transplantable neuronal populations from induced pluripotent stem cells. CHAIRS: Aniz Girach, NightstaRx. Angela Gritti, San Raffaele Telethon Institute for Gene Therapy (TIGET) TIME 9:10 - 9:15 SPEAKER Session Chairs TITLE Introduction and Welcome 9:15 - 10:00 Keynote Speaker: Stefano Pluchino Wellcome Trust-MRC Stem Cell Institute, University of Cambridge (UK) Gabor Foldes National Heart and Lung Institute, Imperial College London How Stem Cells Signal to Host Immune Cells 10:00 - 10:30 Modelling Cardiovascular Disease with Pluripotent Stem Cells: a High-Content Approach 10:30 - 11:00 COFFEE BREAK 11:00 - 11:30 Vania Broccoli Italian Research Council (CNR) and Ospedale San Raffaele 11:30 - 12:00 Accelerating Human iPSC in vitro Disease Modeling by Introducing Targeted Gene Mutations in Isogenic Cell Lines Through the CRISPR/Cas9 Nuclease System POSTER TASTERS 12:00 - 12:30 SNAPSHOT PRESENTATIONS 12:30 - 14:00 LUNCH - EXHIBITION - POSTERS PLENARY KEYNOTE: ROOM - IRONBRIDGE The Development of New Therapeutics for respiratory 14:00 - 15:00 Professor Tracy Hussell inflammation Manchester Collaborative Centre for Inflammation Research 15:00 - 15:30 Martin Pule Autolus Title TBA 15:30 - 16:00 Alessandra Biffi TIGET, IRCCS San Raffaele Scientific Institute Hematopoietic Stem Cell Based Gene Therapy for the Treatment of Lysosomal Storage Disorders 16:00 - 16:30 Alun Barnard Oxford University Gene Therapy for Choroideremia, an Inherited Cause of Blindness 16:30 - 17:00 POSTER SESSION