current congress programme

Tuesday 22 October 2019
17:00-19:00	2019 Opening session Chairs: Hildegard Büning, Fàtima Bosch, Angel Raya Room 113-117 INV025: Andrea Ballabio Tigem, Naples The lysosome: main regulator of cell metabolism and attractive therapeutic target INV026: Masayo Takahashi Riken, Kobe The benefits and risks of iPSC-derived retinal cell therapy
19:00-20:00	Welcome reception

Wednesday 23 October 2019
08:30-10:30	Session 1a: Gene and cell targeting Chairs: Toni Cathomen, Thierry VandenDriessche Room 116-117 INV027: Els Verhoeyen EVIR/CIRI/INSERM U1111, Lyon Site specific genome editing in human T, B cells and HSCs using baboon envelope gp pseudotyped “Nanoblades” loaded with Cas9/sgRNA and specific knock-in in HSCs when combined with an AAV-6 encoding donor DNA. INV028: Pietro Genovese SR Tiget, Milan “One size fits all" strategy for T cell correction, selection and depletion as new Treatment for HIGM1 syndrome OR01: Laura Torella CIMA, Pamplona Paired-nickase S.aureus Cas9 system is an efficient and potentially safer in vivo treatment for Primary Hyperoxaluria type 1 OR02: Antonio Carusillo University of Freiburg HDR-CRISPR: a novel system to promote Cas9-mediated homology-directed DNA repair OR03: Alessia Cavazza University College London Targeted gene correction of human haematopoietic stem cells for the treatment of Wiskott-Aldrich Syndrome OR04: Tiziana La Bella Inserm U1138, Paris Adeno-associated virus in human liver: natural history and consequences in tumor development	Session 1b: Innovaties therapies in cancer Chairs: Vincenzo Cerullo, Ramon Alemany Room 113-115 INV029: Cristina Fillat Idibaps, Barcelona Pancreatic cancer: from target discovery to innovative therapies INV030: Chiara Bonini Ospedale San Raffaele, Milan Genome editing of memory stem T cells for cancer immunotherapy OR05: Sheena Smith University of Zurich Paracrine delivery of therapeutic biologics for cancer OR06: Olivier Humbert Fred Hutchinson Cancer Research Centerm Seattle, WA Base editor-mediated CD33 engineering to improve safety and efficacy of CD33-targeted cancer therapy	Session 1c: iPSC & organoids Chairs: Josep M Canals, Axel Schambach Room 112 INV032: Nico Lachmann Hannover Medical School The "new stem cell“: macrophages for disease modeling and cell based therapies INV033: Nuria Montserrat IBEC Barcelona Engineering human pluripotent stem cells for organoid applications in regenerative medicine INV034: Alfonso Martinez-Arias University of Cambridge Gastruloids: an ESC based model for mammalian gastrulation and axial organization OR07: Frank Staal Leiden University Medical Center De novo generation of functional human thymus organoids from induced pluripotent stem cells OR08: Patricia Peking Paracelsus Medical University, SCI-TReCS, Salzburg Self-assembly of human stem/progenitor cells creates neo-vascularized skin and skin organoids	Session 1d: ATMP manufacturing Chairs: Philippe Moullier, Francesc Godia Room 211 INV035: Richard Snyder Thermo Fisher Scientific, Cambridge, MA Viral vector manufacturing and testing INV036: Matthias Hebben LogicBio, Cambridge, MA AAV manufacturing: critical parameters influencing vector quality attributes. INV037: Hanna Lesch Kuopio Center for Gene and Cell Therapy, Translational research of AAV expressing VEGF-B: large scale manufacturing process development, analytical development and pre-clinical studies in ischemic porcine model INV038: Amine Kamen McGill University, Montreal Process intensification for manufacturing of viral vectors for cell and gene therapy INV039: Luca Alberici Molmed, Milan 20 years journey of viral vector manufacturing and cell engeneering INV040: Carol Knevelman Oxford BioMedica Efficient processes for the commercial manufacture of lentiviral vectors OR09: Ana Filipa Rodrigues iBET, Oeiras Cell- and vector-engineering approaches for manufacturing high-titer GaLV pseudotyped lentiviral vectors from stable and constitutive producer cell lines OR10: Vinicia Polito Bambino Gesù Children’s Hospital, Rome Generation of an automated GMP-grade protocol in a closed system for the expansion of polyclonal memory γδ-T cells for a “third party” cell bank.
10:30-11:00	Coffee break
11:00-13:00	Plenary session 2: From Pre-clinical to clinical gene therapy Chairs: Juan Bueren, Alessandro Aiuti Room 113-117 INV041: Steve Brown IMPC, Harwell The International Mouse Phenotyping Consortium (IMPC): Delivering a comprehensive catalogue of mammalian gene function and novel pre-clinical models INV042: Fàtima Bosch UAB, Barcelona Translational gene therapy approaches to treat Mucopolysaccharidosis INV043: Matthew Porteus Stanford University, CA Engineering of cells using genome editing to treat disease INV082: Angelo Lombardo SR Tiget, Milan Exploiting targeted epigenome editing for therapeutic and biological applications
13:00-15:00	Lunch & Poster session I
13:45 14:45	Meet the society leadership Registration area A chance to meet the leadership of all national and international societies
15:00-17:00	Plenary session 3: Gene therapy clinical trials I Chairs: Adrian Thrasher, Seppo Yla Herttuala Room 113-117 INV045: David Williams Harvard Medical School, MA Validation of BCL11A as molecular target to treat sickle cell disease: results of pilot and feasibility trial using shmiR technology INV046: Fulvio Mavilio Audentes Therapeutics, San Francisco, CA Gene therapy for X-Linked Myotubular Myopathy INV047: Alberto Auricchio TIGEM, Naples Gene therapy of mucopolysaccharidosis type VI INV048: Jude Samulski UNC Gene Therapy Centre, Chapel Hill The road from Canavan to Pompe - a 20 yr clinical journey
17:00-17:30	Coffee break
17:30-19:30	Session 2a: Skeletal muscle & bone gene therapy Chairs: Marinee Chuah, Ana Buj Bello Room 116-117 INV049: Thierry VandenDriessche VUB, Brussels Gene therapy flexes its muscles INV050: Pura Muñoz Universidad Pompeu Fabra, Barcelona Muscle stem cell aging and rejuvenating strategies INV051: Johan Richter Lund University Towards haematopoietic stem cell-targeted gene therapy of infantile malignant osteopetrosis OR11: Daniel Moore University College London Modelling skeletal muscle laminopathies with human ips cells and bio-engineered skeletal muscles: prospects for genetic therapies OR12: Magdalena Matloka Center of Research in Myology, Paris Decoy-based gene therapy for Myotonic Dystrophy	Session 2b: First in man gene therapy Chairs: Alessandra Biffi, Giuliana Ferrari Room 113-115 INV052: Claire Booth UCL, London Evolving haematopoietic stem cell gene therapy INV053: Alessandro Aiuti SR Tiget, Milan Haematopoietic stem cell gene therapy for MPSIH INV054: Terrence Flotte University of Massachusetts Medical School, MA First-in-human gene therapy for Tay-Sachs disease: Report of two infants treated on an expanded access clinical trial of rAAVrh8-HexA/HexB (AXO-AAV-GM2) OR13: Erika Zonari SR TIGET, Milan Shortened ex vivo transduction for haematopoietic stem cell gene therapy of Hurler disease: impact on haematopoietic reconstitution potential OR14: Gavin Corcoran Axovant Sciences, NYC The SUNRISE-PD Study, a clinical trial of AXO-LENTI-PD: a CNS-directed gene therapy for the treatment of Parkinson’s Disease	Session 2c: Next generation engineered T&NK cells Chairs: Sonia Guedan, Boris Fehse Room 112 INV055: John Maher UCL, London T4 CAR T-cell immunotherapy of head and neck cancer – Phase I trial update INV056: Pablo Menendez Josep Carrera Research Center, Barcelona Advancing adoptive cellular immunotherapy for acute leukemia IN0V57: Concetta Quintarelli Ospedale Bambino Gesù, Roma Harnessing NK lymphocytes: an alternative platform for allogeneic CAR therapy OR15: Frederic Thalheimer Paul-Ehrlich Institut, Langen In vivo generated human CAR T cells eradicate B cell leukemia in preclinical mouse models OR16: Arpad Szoor University of Debrecen Trastuzumab derived HER2-specific CAR for trastuzumab-resistant breast cancer: CAR T-cells successfully engage target epitopes that are not accessible to antibodies	Session 2d: AAV vector development Chairs: Els Verhoeyen, Jim Wilson Room 211 INV058: Hildegard Büning Hannover Medical School Advanced AAV vectors - capsid engineering and beyond INV059: Eric Kelsic Harvard Medical School, MA Machine-guided AAV capsid engineering for gene therapy INV060: David Schaffer University of California, Berkeley, CA Directed evolution of novel AAV vectors for clinical gene therapy OR17: Jihad El Andari Heidelberg University Hospital Semi-rational engineering of next-generation AAV capsids for muscle gene therapy OR18: Eric Pozsgai Sarepta Therapeutics, Cambridge, MA Systemic gene transfer with AAVrh74.MHCK7.SGCB increased β-sarcoglycan expression in patients with limb girdle muscular dystrophy type 2E

Thursday 24 October 2019
8:30-10:30	Session 4: Gene therapy clinical trials II Chairs: Virginia Haurigot, Fulvio Mavilio Room 113-117 INV061: Juan Bueren CIEMAT CIBERER, Madrid Gene therapy for patients with Fanconi anaemia INV062: Michel Sadelain Memorial Sloan Kettering Cancer Center, NY Clinical strategies to overcome resistance to CAR therapy INV063: Don Kohn UCLA, CA Haematopoietic stem cell gene therapy for sickle cell disease INV064: Steven Gray UT Southwestern, Dallas, TX Intrathecal AAV9 as a platform approach to treat multiple CNS disorders
10:30-11:00	Coffee break
11:00-13:15	Presidential symposium Chairs: Hildegard Büning, Angel Raya Room 113-117 INV065: Hildegard Büning Hannover Medical School Presidential address INV066: Hans Peter Kiem Fred Hutchinson Cancer Research Center, Seattle, WA Targeting distinct haematopoietic stem cell populations for ex vivo and in vivo gene therapy ESGCT Outstanding achievement award INV067: Nathalie Cartier Lacave Gene therapy strategies for neurodegenerative diseases : From genetic to complex diseases ESGCT Young Investigator award 0R51: Paula Rio CIEMAT CIBERER, Madrid Innovative haematopoietic gene therapy strategies in Fanconi anaemia ESGCT AGM
13:15-14:45	Lunch & Poster session II
13:45 14:45	Ethics in gene and cell therapy Room 112 Chair: Odile Cohen-Haguenauer, Bernd Gänsbacher Odile Cohen-Haguenauer, Hôpital Saint Louis, Paris Ethics of first-in-man clinical trials in gene therapy Nicholas Anagnou, University of Athens Ethical issues of unproven therapies and stem cell tourism Matthew Porteus, Stanford University, CA The strict criteria for germline editing proposed by the 2017 study committee François Moreau-Gaudry, University of Bordeaux CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations Roundtable discussion		Infectious diseases meet gene therapy Room 211 Chair: Hans Pieter Kiem, Monique Nijhuis Javier Martinez-Picado, IrsiCaixa, Barcelona The IciStem experience: learning from allogeneic stem cell transplantation to cure HIV Anne Galy, Genethon, Evry Vectored immunoprophylaxis of infectious diseases : Current efforts and future perspectives Toni Cathomen, University of Freiburg The prospect of genome and epigenome editing to cure HIV and other infectious diseases Roundtable discussion
14:45-16:45	Session 3a: Latest news from cardiovascular and pulmonary gene therapy Chairs: Seppo Yla Herttuala, Mauro Giacca Room 116-117 INV068: Mauro Giacca Kings College London Identification of novel cardioprotectors from AAV libraries encoding for the secretome INV069: Maria Blasco CNIO, Madrid Telomerase gene therapy as a novel treatment for pulmonary fibrosis INV070: Seppo Ylä Herttuala University of Eastern Finland, Kuopio Recent developments in cardiovascular gene therapy OR19: Rosario Perona CSIC/UAM, CIBER, Madrid GSE4, a Dyskerin derived peptide enhances pneumocyte growth, reduces apoptosis, DNA damage and lung fibrosis. OR20: Rafael Sànchez-Sànchez Instituto de Investigacion sanitaria La Fe, Valencia Cardioprotective effect of miRNAs derived from mesenchymal stem cells extracellular vesicles in doxorubicin-induced damage	Session 3b: Sensory and CNS gene therapy I Chairs: Alberto Auricchio, Françoise Piguet Room 113-115 INV071: Stylianos Michalakis Harvard Medical School, MA Munchen University INV072: Luk Vandenberghe Harvard Medical School, MA AAV discovery for neurosensory indications and beyond. INV073: Hinrich Staecker The University of Kansas, Lawrence, KS Strategies for translating inner ear gene therapy; lessons from the first in human clinical trial OR21: Szilárd Kiss Weill Cornell Medical College, NY Intravitreal gene therapy with ADVM-022 for neovascular age-related macular degeneration (phase 1 OPTIC Trial) OR22: Adrian Thrasher University College London Phase 2/3 trial to assess the safety and efficacy of Lenti-D autologous haematopoietic stem cell gene therapy for cerebral adrenoleukodystrophy	Session 3c: Immune responses to gene therapy Chairs: Federico Mingozzi, Anne Galy Room 112 INV074: Percy Knolle TUM, Munich Mechanisms of liver tolerance and implication for liver-targeted gene therapy INV075: Roland Herzog Indiana University, Bloomington, IN Immune response mechanisms against AAV vectors INV076: Anna Kajaste-Rudnitski SR Tiget, Milan Vector-host interactions and innate immunity in haematopoietic stem cell gene engineering OR23: Giorgia Squeri SR Tiget, Milan Targeting pre-existing anti-transgene T cell response for effective gene therapy of Mucopolysaccharidosis type-I OR24: Malo Journou University of Nantes INSERM 1089 Recombinant AAV-mediated gene transfer to the skeletal muscle is associated to immune modulation of transgene expression in the macaque model	Session 3d: Blood diseases Chairs: Guillermo Guenechea, Francisco Martin Room 211 INV077: Jose Carlos Segovia Sanz CIEMAT CIBERER Madrid Treatment of Pyruvate Kinase Deficiency: From the laboratory to the lentiviral gene therapy clinical trial INV078: Giuliana Ferrari SR TIGET Milan Gene therapy for transfusion dependent beta-thalassemia: A lesson from patients OR25: Yari Gimenez Martinez CIEMAT/CIBERER, Madrid Lentiviral-mediated Phenotypic correction of CD34+ cells from RPS-19-deficient Diamond-Blackfan aneamia patients OR26: Denise Klatt Hannover Medical School Competitive sgRNA screen identifies Mapk14 as a druggable target to improve HSPC engraftment in a proinflammatory environment OR27: Evangelia Yannaki G. Papanikolaou Hospital, Thessaloniki Interim results from the phase 3 Northstar-2 and Northstar-3 studies of LentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassaemia OR28: Serena Scala SR Tiget, Milan Haematopoietic reconstitution dynamics of MPB- and BM-derived haematopoietic stem/progenitor cells in Wiskott-Aldrich syndrome patients treated with lentiviral gene therapy
16:45-17:15	Coffee break
17:15-19:15 Room 113-117	Plenary session 5: Gene editing, toxicity Chairs: Robin Ali, Luigi Naldini Room 113-117 INV079:David Liu Harvard University, MA Prime Editing: Search-and-replace genome editing without double-strand breaks or donor DNA INV080: David Rawlings University of Washington, Seattle, WA Gene editing in human T and B cells for novel clinical applications INV081: Jim Wilson University of Pennsylvania, Philadelphia, PA Safety and efficacy of in vivo editing in primate liver INV044: Luigi Naldini SR Tiget, Milan Advanced genetic engineering of haematopoiesis to treat human diseases
20:00	Molecular Mingle MNAC, Barcelona Coaches from CCIB at 19.15

Friday 25 October 2019
09:00 11:00	Session 4a: Sensory and CNS gene therapy II Chairs: Nathalie Cartier, Jose Toledo Aral Room 116-117 INV083: Abdel Benraiss University of Rochester, NY Cell replacement strategies for striatal regeneration in Huntington’s disease INV084: Robin Ali UCL, London ES-derived cone photoreceptor transplantation for the treatment of retinal degeneration OR29: Bence Gyorgy Institute of Molecular and Clinical Ophthalmology Basel Allele-specific gene disruption through discrimination of a single base change by S. aureus Cas9-KKH prevents progressive hearing loss after AAV-mediated gene delivery OR30: Allison Bradbury University of Pennsylvania, Philadelphia, PA Intrathecal AAV9-GALC corrects both central and peripheral nervous system disease in canine Krabbe disease in a dose and time-dependent manner OR31: Pengcheng Zhou Voyager Therapeutics, Cambridge, MA Widespread, dose-dependent and durable huntingtin lowering in Huntington’s disease-relevant brain regions of large mammals with intraparenchymal administration of the AAV gene therapy VY-HTT01 OR32: Kevin Foust AveXis, San Diego, CA Intrathecal administration of AVXS-301 for amyotrophic lateral sclerosis (ALS): survival extension and SOD1 reduction in mice and nonhuman primates	Session 4b: New approaches in gene editing Chairs: Paula Rio, Jose Carlos Segovia Room 113-115 INV085: Juan Rodriguez-Madoz University de Navarra, Pamplona Disease modelling and genome editing for treatment of rare metabolic diseases. INV086: Claudio Mussolino University of Freiburg Advances in precision genome and epigenome editing for human gene therapy OR33: Edoardo Schneider IGCEB, Trieste Human microRNAs selected by high-throughput screenings enhance CRISPR/Cas9-based homologous recombination in post-mitotic hearts OR34 Laura Ugalde Díaz CIEMAT/CIBERER, Madrid Broad applicability of NHEJ-mediated gene editing to correct mutations in a variety of Fanconi anaemia genes.	Session 4c: Metabolic and genetic diseases Chairs: Steven Gray, Fatima Bosch Room 112 INV087: Federico Mingozzi, Spark Therapeutics, Philadelphia, PA Liver gene transfer as a new modality for enzyme replacement therapy, the example of Pompe disease OR35: Petr Ilyinskii Selecta Biosciences, Watertown, MA ImmTOR™ tolerogenic nanoparticles enhance transgene expression after both initial and repeat dosing in a mouse model of methylmalonic acidemia treated with Anc80 AAV-Mut vector OR36: Heather Gray-Edwards University of Massachusetts, Boston, MA Bicistronic AAV gene therapy for Tay-Sachs disease OR39: Maria Jose de Castro Hospital Universitario Santiago de Compostela Safety, tolerability, biopotency and neurocognitive data of ABO-102 in Transpher A, an open-label, multicenter, single-dose, dose-escalation, Phase 1/2 Clinical Trial in Sanfilippo Syndrome type A (Mucopolysaccharidosis IIIA OR40: Verónica Jiménez UAB, Barcelona AAV-FGF21 gene therapy mediates healthspan expansion in old mice	Session 4d: Regulatory session Chairs: Martina Schüssler-Lenz, Ilona Reischl Room 211 INV088: Martina Schüssler-Lenz Paul-Ehrlich-Institut, Langen The EU landscape of authorised advanced therapies: presence and future INV089: Heli Suila Finish Medicines Agency Quality considerations for gene therapies INV090: Anne Engedahl Pastoft Danish Medicines Agency Non-clinical 84 data supporting gene therapy clinical trials’ INV091: Jan Müller-Berghaus Paul-Ehrlich-Institut, Langen Scientific and regulatory support for gene therapy in european countries Q&A and panel discussion with chairs and speakers, Patrick Celis (European Medicines Agency), audience
11:00-11:30	Coffee break
11:30-13:30	Session 5a: Cancer gene therapy Chairs: Cristina Fillat, Javier Garcia-Castro Room 116-117 INV092: Juan Fueyo MD Anderson, Houston, TX Virus, immunology and gliomas: new paradigms INV093: Ramon Alemany ICO, Barcelona Potential and limitations of oncolytic adenoviruses INV094: Vincenzo Cerullo University of Helsinki Dressing viruses in tumor’s clothing: cloning-free platforms to trigger tumor-specific immune response OR41: Daniel Abate - Daga Moffit Institute, Tampa, FL TBC OR42: Maria Cristina Ballesteros CIMA, Pamplona Local expression of a PD-L1 blocking antibody from a self-replicating RNA vector induces potent antitumor responses	Session 5b: New delivery systems and technologies Chairs: Ander Izeta, Zoltan Ivics Room 113-115 INV095: Helmut Hanenberg University of Duisburg - Essen Genetic gold for the treatment of haemophilia OR43: Jason Wright Homology Medicines Inc. Bedford, MA Nuclease-free genome editing by AAVHSC vectors leads to in vivo genome correction and amelioration of disease phenotype in a mouse model of phenylketonuria (PKU) OR44: Jonathan Finn Codiak BioSciences, Cambridge, MA A versatile platform for precision exosome engineering OR45: Manel Llado TIGEM, Naples Homology-independent targeted integration to counteract toxic gain-of-functions and loss of transgene expression due to cell proliferation. OR46: Stefanie Gehrig-Giannini Bayer AG Preclinical Research, Köln Engineering potent, small, chimeric, synthetic, RNA-guided nucleases (sRGN) from four uncharacterized Cas9 genes OR47: Giandomenico Turchiano University College London CAST-Seq, a novel preclinical genotoxicity assay, enables qualitative and quantitative insights in chromosomal aberrations in gene-edited human haematopoietic stem cells OR48: Anthony Forget Intellia Therapeutics, Cambridge, MA In vivo gene knockout followed by targeted gene insertion results in simultaneous reduced mutant protein levels and durable transgene expression	Session 5c: Liver gene and cell therapy Chairs: Gloria Gonzalez-Aseguinolaza, Xavier Anguela Room 112 INV096: Paolo Martini Moderna Therapeutics, Cambridge, MA Messenger RNA therapy for inherited metabolic disorders INV097: Nick Weber Vivet Therapeutics, Pamplona Targeting the liver to fix the bile: Opportunities and challenges in gene therapy for inherited cholestasis OR37: Leah Venturoni National Human Genome Research Institute, Bethesda, MD Treatment of methylmalonic acidemia (MMA) by targeted integration of MMUT into Albumin with a promoterless AAV vector (GeneRideTM) confers a progressive hepatocellular growth advantage in mice OR38 David Rodriguez- Buritica The University of Texas, Dallas, TX Positive cohort 1 results from the phase 1/2, AAV8-mediated liver-directed gene therapy trial in glycogen storage disease type Ia (GSDIa) OR49: Ana Cristina Puga Ultragenyx, Novato, CA A phase 1/2 clinical trial of AAV8 gene therapy in adults with late-onset OTC deficiency: CAPtivate cohort 1 + 2 results OR50: Anais Amaya Children's Medical Research Institute, Westmead Inhibition of proliferation in primary human hepatocytes following in vivo AAV-mediated genome editing	Session 5d: Make gene therapy a clinical reality Chairs: Alessandro Aiuti, Hildegard Büning Room 211 INV098: Alessandro Aiuti SR TIget, Milan Turning blood stem cells into medicinal products: lessons learned and future challenges INV099: Serge Braun AFM, Paris Patients are not just patients, they are also pioneers. The AFM-Telethon perspective INV100: Sol Ruiz Spanish Medicine Agency, Madrid Regulation of ATMP in the EU: Take it easy INV101: Magda Papadaki Merck, London Reinventing the system for ATMPs: need for integrated development, assessment and business models
13:30-15:30	13:30 - 14:00 - SETGYC General Assembly (Room 113-115) Lunch
14:00 15:00	Current challenges of the AAV vectors Room 211 Chairs: Hildegard Büning, Dirk Grimm Tiziana La Bella, Genethon, Evry Genotoxicity - Is there a danger? Hildegard Büning, Hannover Medical School Pre-existing and de novo immune responses towards AAV - what do we know and how can we manage this challenge? Dirk Grimm, University of Heidelberg How to deal with AAVs lack in cell type specificity and efficiency - capsid engineering as possible solution Eduard Ayuso, University of Nantes How to produce clinical grade AAV vectors large scale Panel Discussion: Hildegard Buning, Dirk Grimm, Eduard Ayuso, Tiziana La Bella, Terry Flotte		Current challenges of Lentiviral vectors Room 112 Chairs: Els Verhoeyen, Axel Schambach Jose Carlos Segovia, CIEMAT/CIBERER, Madrid Expanding the range of gene therapy to challenging diseases Els Verhoeyen, EVIR/CIRI/INSERM U1111, Lyon The future: In vivo gene therapy targeting haematopoietic stem cells and T cells Pietro Genovese, Harvard Medical School Gene editing: Towards clinical applications Hans Pieter Kiem, Fred Hutchinson Cancer Research Center, Seattle, WA Challenges in translating gene therapy to more patients and countries Panel Discussion: Anne Galy, Paula Rio, Els Verhoeyen, Axel Schambach, Jose Carlos Segovia, Pietro Genovese, Hans Pieter Kiem
15:30-17:30	Plenary session 6: Challenges for the future Chairs: Fatima Bosch, Gerry McLachlan Room 113-117 INV102:Olivier Rabin WADA, Montreal Gene doping: The ultimate deviation of gene therapy INV103: Carl June University of Pennsylvania, Philadelphia, PA Updates in CAR T cell therapies Keynote INV104: Kathy High Spark Therapeutics, Philadelphia, PA Turning genes into medicines: Challenges for the next 5 years and beyond Closing remarks
17:30-18:00	Closing drinks