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PuBliC EnGaGEmEnT day: GEnE THEraPy and STEm CEllS: 21ST CEnTury mEdiCinEAn interactive day for anyone who wants to find out about gene and cell therapy. Talks from experts, hands-on activities and special guests with Professor Lord Winston, The Naked Scientists and Zombie Science.Syndicate Rooms 1 and 2

10.00 registration

10.30 WelcomeandintroductionProfessor Adrian Thrasher, President of the BSGT

11.00 Whatisgenetherapy?Dr Maria Limberis, University of Pennsylvania

11.30 Whatisstemcelltherapy?Dr Tristan McKay, Queen Mary University of London

12.00 thelifeofacliniciandoinggenetherapyProfessor Iain McNeish, Cancer Research UK

12.30 DnAorigamihostedbylyndaCoughlanandCo,GlasgowuniversityInteractive demonstration involving the audience as a method for demonstrating the complexity of DNA and how it is applied as a biomedicine

13.00 shorttalkbysenseAboutscienceandcareersinscienceandthemedia

13.15 lunch,meettheresearchers,networkingbingo,careersadviceA chance for attendees to quiz scientists directly about any aspect of their working lives or science

14.00 ZombiescienceshowDoctor AustinAn interactive show designed to highlight gene and cell therapy in a fun and popular stage format

sponsoredbytAGtC

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15.00 BraingenetherapyforrealDr Ahad Rahim, University College London

15.30 thenakedscientisthoststheDnAsequencingrace,andtheafternoondebate–scientistsonthepanelSchool students take part in an interactive race to see who can perform DNA fingerprinting the fastest and get hands on experience of manipulating DNA. This will demonstrate how genetics, disease and treatment are important on an individual basis and how personalised medicine is becoming a reality. The audience will be able to question a panel of the speakers and other scientists and debate the ethics, practicalities, reality and possibilities presented by the novel therapies shown throughout the event.

17.00 KeynotespeechProfessor Lord Winston

17.15 Adjourn

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Education daySyndicate Rooms 3 and 4

13.00 parallel sessions 1a, 1b

Syndicate 3 1a: Strategies for delivery and expression of transgenesed01 general overview of viral/non-viral gene therapy

Len Seymour, University of Oxforded02 Achieving reproducible and stable transgene expression for long-term

therapeutic benefitMike Antoniou, King’s College London

ed03 smAr based systemsRichard Harbottle, Imperial College London

Syndicate 4 1b: Hot topics in gene and cell therapy and tissue engineering ed04 stem cells for studying heart disease

Chris Denning, University of Nottinghamed05 gene therapy for heart failure

Roger Hajjar, Mount Sinai Medical Centre, New Yorked06 cell therapy for diabetes

Sarah Ferber, Sheba Medical Centre, Tel Aviv

15.00 Afternoon break

15.30 parallel sessions 2a, 2b

Syndicate 3 2a: immunologyed07 An overview of the immune system, the challenges and opportunities

it provides for gene therapyPeter Searle, University of Birmingham

ed08a development of therapeutic vaccines for cancer and chronic infections: from Aml to hbV�Farzin Farzaneh, King’s College London

ed08b successful engraftment and chemoselection of allogeneic hsc transduced with mgmtP140K /hsV�-tK lentivirus without myeloablation or post-transplantKarin Gaensler, University of California

ed09 Immune responses to vector and transgenesJim Wilson, University of Pennsylvania

Syndicate 4 2b: transgene integration ed10 Assessing and avoiding insertional mutagenesis

Christof von Kalle, National Centre for Tumor Diseases, Heidelberg ed11 manipulating insertion profiles of transposon-based vectors

Zoltan Ivics, Max Delbrück Center for Molecular Medicine, Berlin ed12 targeted genome engineering with designer nucleases

Toni Cathomen, Hannover Medical School

17.30 Adjourn

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cat-ESgct SatEllitE WorkSHop: advancEd tHErapy mEdicinal productS: From promiSE to rEality Auditorium 1

08.30 registration

09.15 Welcome and openingseppo ylä-herttuala, esgct Presidentlen seymour, congress Presidentchristian K. schneider, european medicines Agency – cAt chair

09.30 translating academic research into commercial products – regulatory considerations

09.30 1: cell-based medicinal productsmanufacturing of cell-based medicinal products: common issues and adviceJean-Hugues Trouvin, CAT Member – France – AFSSAPSnon-clinical development of cell-based medicinal productsEgbert Flory, CAT Member – Germany – PEIProgressing cell-based medicinal products to market authorisation and into the clinicGopalan Narayanan, CAT Member – UK – MHRAcell-based medicinal products for a global market: FdA perspectives on common us/eu guidelinesSteven Oh, US FDA – Office of Cellular, Tissue and Gene Therapies

11.00 coffee break

11.30 2: gene therapymanufacturing of gene therapy products: common issues and adviceMaria Cristina Galli, CAT Member – Italy – ISSnon-clinical development of gene therapy productsBeatriz Silva Lima, CAT Member – Portugal – INFRAMEDProgressing gene therapy products to market authorisation and into the clinicKyri Mitrophanous, Oxford Biomedica

12.30 3: European and national regulatory path for atmpsrole of european and national regulatory authorities: who advises on what?Lucia D’Apote, European Medicines Agency – CAT Scientific secretariat MHRA speaker to be confirmedround table discussion: transition from an academic concept to a clinical stage – what is the regulatory path to accelerate product development?Christian K. Schneider, European Medicines Agency – CAT Chair

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13.00 lunch

14.00 From regulation to reality – challenges in translation of gene therapy and cell-based medicinal products

14.00 4: Gene therapy – practical examplesGenetherapycasestudy1Gopalan Narayanan, CAT Member – UK – MHRAGenetherapycasestudy2:ADA-sCIDAlessandro Aiuti, HSR-Tiget, GTWP member Jonathan Appleby, GSKcGmPvirusmanufactureandevolvingreleasetestsEleanor Berrie, Virus Manufacturing QP, University of OxfordPanel discussion Panel chairs: Gopalan Narayanan, CAT Member – UK – MHRA Maria Cristina Galli, CAT Member – Italy – ISS Len Seymour, ESGCT Congress President

15.15 Coffeebreak

15.45 5: Cell therapy and cell-based medicinal products – practical examplesCell-basedmedicinalproductcasestudy1Paula Salmikangas, CAT Vice-Chair – Finland – FimeaCell-basedmedicinalproductstudy2:ChondroCelectWilfried Dalemans, CTO TIGENIX Panel discussionPanel chairs:Paula Salmikangas, CAT Vice-Chair – Finland – Fimea Egbert Flory, CAT Member – Germany – PEI Thierry VandenDriessche, ESGCT CAT member

17.00 Closing remarksseppoYlä-herttuala,esGCtPresidentlenseymour,CongressPresidentChristianK.schneider,europeanmedicinesAgency–CAtChair

17.15 Adjourn

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ESGCT and BSGT COnGrESS OPEninG CErEmOny Auditorium 1

10.00 registrationopen

17.30 Welcomecocktail

19.00 Opening symposiumChair: Len SeymourthepotentialofgeneticsresearchProfessor Lord Winston, Imperial College Londontumourviruses,rumourvirusesandpseudovirusesRobin Weiss, University College London

20.30 Adjourn

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main conference08.00 Registration open

08.30 Plenary session 1: clinical trials #1

Auditorium 1 Chairs: Andy Baker, Adrian ThrasherINV01 Gene therapy for the treatment of heart

failureRoger Hajjar, Mount Sinai Medical Centre, New York

INV02 Assessment of potential promoters for lentiviral gene therapy in DMDFrancesco Muntoni, University College London Institute of Child Health

INV03 Gene therapy for immunodeficiencyAdrian Thrasher, University College London Institute of Child Health

Proffered papers:OR01 Lentiviral vector transduced CD34+ cells for the treatment of Wiskott-

Aldrich Syndrome Alessandro Aiuti, Fondazione Centro San Raffaele del Monte Tabor, Milan

10.15 Morning break

10.45 Parallel sessions 1a, 1b, 1c

Syndicate 3 1a: respiratory gene and cell therapyChair: Simon WaddingtonINV04 Moving forward with cystic fibrosis gene therapy

Uta Griesenbach, Imperial College London on behalf of the UK Cystic Fibrosis Gene Therapy Consortium

INV05 Cell therapy approaches for lung diseasesDaniel Weiss, University of Vermont College of Medicine

Proffered papers:OR02 Large-scale screening identifies coxsackievirus B3 (CVB3) as a

promising oncolytic virotherapy agent against non-small cell lung cancerHiroyuki Inoue, Medical Institute of Bioregulation, Kyushu University, Fukuoka

OR03 AAV9 gene transfer to the nose for pharmacologically-regulated systemic expression of therapeutic proteinsMaria Limberis, University of Pennsylvania

OR04 Long-term follow up of pre- and postnatal pulmonary gene transfer with rAAV2/5 in a mouse modelMarianne Carlon, K.U. Leuven

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Syndicate 1 1b: imaging technologies and applicationsChair: Georges VassauxInV06 monitoringgenetherapybynon-invasiveopticalimaging

Kevin Francis, Caliper Life Sciences, Cheshire InV07 molecularimagingofgeneexpression:nIsasareportergene

Georges Vassaux, Inserm U948, University of Nantes

Proffered papers:Or05 multi-colourrGBmarkingtoassesslivertumourdevelopmentin vivo

Boris Fehse, University Medical Center Hamburg-EppendorfOr06 Capsidmodificationswiththepotentialforlifecycleimagingand

retroviralproteintransferTobias Maetzig, Hannover Medical School

Or07 Visualisationofactivehomingofmesenchymalstemcellsintothetumorstromaofhepatocellularcarcinoma(hCC)usingthesodiumiodidesymporterasreportergeneKerstin Knoop, Ludwig-Maximilian-University Munich

Auditorium 1 1c: Genotoxicity #1Chair: Chris BaumInV08 targetingintegrationtoselectedgenomic

lociandin situtailoringofcassettedesignallowsrobusttransgeneexpressionwithoutperturbingendogenoustranscriptionAngelo Lombardo, HSR-TIGET, Milan

InV09 therapeuticstrategieswithintegration-deficientlentiviralvectorsRafael Yanez, Royal Holloway University of London

Proffered papersOr08 hepaticlentiviralgenetransferisassociatedwithclonalselection,but

notwithtumourformationinseriallytransplantedmiceIna Rittelmeyer, Hannover Medical School

Or09 large-scalerAAVintegrationsiteanalysisinapreclinicalmousemodelChristine Kaeppel, DKFZ/NCT, Heidelberg

Or10 lentiviralvector-basedinsertionalmutagenesisidentifiesnewclinicallyrelevantcancergenesinvolvedinthepathogenesisofhepatocellularcarcinomaMarco Ranzani, San Raffaele Scientific Institute, Milan

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12.15 lunch and poster session 1

Upstairs P011-P050 Cns,respiratoryandgeneticandmetabolicdisease

Downstairs P135-P184 newdeliverytechnologiesandnon-viralvectors

13.30 esGCtGeneralAssembly

14.15 Plenary session 2: Overcoming the hurdles to successful gene therapy

Auditorium 1 Chairs: Nathalie Cartier-Lacave, Charles Coutelle InV10 hsCgenetherapytrialformetachromaticleukodystrophy

Alessandra Biffi, San Raffaele Scientific Institute, Milan InV11 Immuneresponsestocapsidsandtransgeneproductsin

pre-clinicalandclinicaltrialsJim Wilson, University of Pennsylvania

InV12 Preclinicalassaystoassessgenotoxicityand“phenotoxicity“Chris Baum, Institute of Experimental Hematology, Hannover Medical School

Proffered papersOr11 AAVcapsidsinduceinnateimmuneresponsesinhumannon-

parenchymallivercellsHildegard Büning, University of Cologne

16.00 Afternoonbreak

16.30 Parallel sessions 2a, 2b, 2c, 2d

Auditorium 1 2a: regulatory round tableChairs: Gösta Gahrton, Alastair Kent, Nancy Kingretroviralandlentiviralvectorsforlong-termgenecorrection;clinicalchallengesinvectorandtrialdesign:presentationofthereportonthejointrAC-Clinigenemeetinginbethesda1. Jacqueline Corrigan-Curray, NIH-OBA RAC, Maryland 2. Dan Takefman, FDA-CBER, MarylandFirst-in-humantrialdesignanddecisions:lessonsfromgenetransferresearchNancy M. P. King, Center for Bioethics, Health, and Society, North Carolinathenecessityfordata-sharingtowardsadvancementofclinicaltranslation:buildingupsampleImPDandsubstantiatingmasterfilesKlaus Cichutek, Paul-Ehrlich-Institut, LangenEma-CaT Focus group to facilitate navigation of aTmP regulatory frameworkChristian K. Schneider and Lucia d’Apote, EMA

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Syndicate 1 2b: Careers workshopChair: Lynda CoughlanInV13 Advancingyourresearchcareer

Ross English, Vitae, South East Hub, UKInV14 Anoverviewofscientificjournalsandpublishing

Jim Wilson, University of PennsylvaniaInV15 europeanresearchCouncil:supportingindependentresearch

careersFiona Kernan, ERC, Brussels

InV16 sciencecommunication Julia Wilson, Sense about Science, London

Syndicate 3 2c: mechanisms of cellular differentiationInV17 usingadulttissuesforgeneratingneworgans:autologouscell

replacementtherapyfordiabetesSarah Ferber, Sheba Medical Centre, Tel Aviv

InV18 mesenchymaltoepithelialtransitioningenhancesthereprogrammingofhumannonpancreaticendocrinecellstowardsbetacells Kevin Docherty, University of Aberdeen

Proffered papersOr12 enhancedmyogenicpotentialofmesoangioblast-derivedinduced

pluripotentstemcellsMattia Quattrocelli, K.U. Leuven

Foyer 2d: Open poster / networking session

18.00 Posterpartywithmoderatedposters

19.00 speakers’dinnerbyinvitationonly

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07.30The Empress Suite, The Grand Hotel

themedbreakfastmeetings

09.00 Plenary session 3: Stem cells in health and disease

Auditorium 1 Chairs: Chris Denning, Chiara Bonini InV19 humaninducedpluripotentstemcellsforcell

therapyagainstinheritedmetabolicdisordersLudovic Vallier, University of Cambridge

InV20 spinalcordregenerationusingescellsJane Lebkowski, Geron Corporation, California

InV21 Cancerstemcells:asourceofnewtherapeutictargets?Norman Maitland, University of York

InV22 exploringandexploitingtAlenucleases(tAlens)fortargetedgenomeToni Cathomen, Hannover Medical School

11.00 morningbreak

11.30 Parallel sessions 3a, 3b, 3c

Syndicate 3 3a: The Eye – cell and gene therapyChair: Robin AliInV23 Developmentofstemcelltherapyforthe

treatmentofretinaldegeneration Robin Ali, University College London Institute of Ophthalmology

InV24 utilisationofiPscellsforretinaldegenerativediseasesMasayo Takahashi, RIKEN, Kobe

InV25 ChallengesandsuccessesofretinalgenetherapywithAAVvectorsAlberto Auricchio, The Telethon Institute of Genetics and Medicine (TIGEM) and Federico II University, Naples

Proffered papersOr13 Genereplacementtherapyinmousemodelsofinheritedcone

photoreceptordisordersAlexander Smith, University College London

Or14 AAV8-mediatedexpressionofVeGFantagonistsinmacaqueeye:comparisonofsubretinalvs.intravitrealdeliveryofvectorMaria Limberis, University of Pennsylvania

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PleAsenOte:At2amtonighttheclockswillturnbackonehourfortheendofDaylightsavingtime

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Auditorium 1 3b: Cancer immunotherapy Chair : Viggo van TendelooINV26 Learning from maternal-fetal tolerance for developing cancer

immunotherapies�David Klatzmann, Groupe Hospitalier Pitié Salpêtrière, Paris

INV27 cell therapy with genetically modified lymphocytes� in onco-hematologyChiara Bonini, San Raffaele Scientific Institute, Milan

Proffered papersOr15 dis�ruption of HLa expres�s�ion to enable allogeneic cells� to es�cape

immune recognitionMichael Holmes, Sangamo BioSciences, California

Or16 targeting chemores�is�tant myeloid leukemia and multiple myeloma through genetic redirection of t cells� agains�t cd44 variant 6Monica Casucci, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

Or17 editing human lymphocyte s�pecificity for s�afe and effective adoptive immunotherapy of leukemiaPietro Genovese, HSR-TIGET, Milan

Syndicate 1 3c: iPS cells and control of differentiationChairs : Andy Baker, Chris DenningINV28 microrNa regulation of s�tem cells�: from biology to

application for targeted gene therapyLuigi Naldini, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

INV29 stem cell Models� for studying cardiotoxicity and cardiac dis�eas�eChris Denning, University of Nottingham

Proffered papersOr18 Generation of fanconi anemia iPs cells� with a targeted integration of

FaNca in the aaVs1 locus�Rocío Baños, CIEMAT/CIBERER, Madrid

Or19 Zinc-finger nucleas�e-mediated correction of the cellular rs-scId phenotypeShamim Rahman, Hannover Medical School

Or20 reprogramming of fibroblas�ts� us�ing mrNa encoding four reprogramming factors�Geertrui Tavernier, Gent University

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13.00 lunch and poster session 2

Upstairs P051-P089 stem cell bioengineering, genotoxicity/targeted integration and neuro and muscle

Downstairs P185-P235 V�iral vectors

14.00Auditorium 1

update and perspectives on European advanced therapy research David Gancberg, European Commission Research and Innovation Directorate General, Brussels

14.45 plenary session 4: cancer gene and virotherapy (iScgt symposium)

Auditorium 1 Chairs: Farzin Farzaneh, Nori KasaharaInV�30 demonstration of safety and antitumoral activity

of Jx-594, a targeted multi-mechanistic oncolytic poxvirus, following intravenous infusion and/or intratumoral injections in patients with refractory metastatic cancersDavid Kirn, Jennerex Inc., San Francisco

InV�31 V�irotherapy in the uKKevin Harrington, Institute for Cancer Research, London

InV�32 molecular design of improved adenovirusesRamon Alemany, Catalan Institute of Oncology-IDIBELL, Barcelona

16.15 Afternoon break

16.45 parallel sessions 4a, 4b, 4c, 4d

Syndicate 3 4a: genetic diseaseChairs: Steve Howe, Suzy BuckleyInV�33 Fetal and neonatal gene therapy

Simon Waddington, University College LondonInV�34 Zooming in on the target: transductional and transcriptional targeting

Thierry VandenDriessche, V. U. Brussels

Proffered papersor21 Inflammation converts immunologically inert human

mesoangioblasts into sensitizers and targets of the alloreactive response: implications for allogeneic cell therapy of dmdMaddalena Noviello, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

or22 development of a new strategy for x-cgd gene therapy using a mirnA regulated lentiviral vectorGiada Farinelli, Children’s hospital Bambino Gesù and Tor Vergata University, Rome

or23 AAV� mediated Factor V�III expression from a novel expression cassette with a potent codon-optimised FV�III constructJenny McIntosh, University College London

sponsored by Iscgt

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Auditorium 1 4b: Cancer gene and virotherapyChairs: Iain McNeish, Len SeymourInV35 targetingDnAdamagerepairandpaclitaxel

resistanceinovariancancerusingoncolyticadenovirusesIain McNeish, Barts Cancer Institute, Queen Mary University of London

InV36 Anascendingdosephase1trialofthesafetyandtolerabilityoftoca511insubjectswithrecurrenthighgradegliomaDoug Jolly, Tocagen Inc., San Diego

Proffered papersOr24 humandatawithadesmoglein2bindingoncolyticadenovirusAd3-

htert-e1AOtto Hemminki, University of Helsinki

Or25 enhancementofantitumourefficacyofe3B-deletedoncolyticadenovirusesbytargetingstAt1thatinteractswithe314.7proteinofadenovirusinmacrophageYaohe Wang, Barts Cancer Institute, Queen Mary University of London

Or26 Adenoviruswithhexontat-PtDmodificationexhibitsincreasedtherapeuticeffectinexperimentalneuroblastomaandneuroendocrinetumorsDi Yu, Rudbeck Lab Klinimm(IGP), Uppsala

Syndicate 1 4c: Stem cells bioengineeringChairs: Heiko Zimmermann, Paula Alves InV37 technologicalstrategiesandmicrofluidic

conceptsforstemcellprocedures–thehYPerlABapproachHeiko Zimmermann, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

InV38 embryonicstemcelllines:frombiologytoproducts Petter Björquist, Cellartis AB, Göteborg

Proffered papersOr27 non-viralsonoporationgenetherapyfororthotopicbone

regenerationGeorg Feichtinger, LBI Trauma, Vienna

Or28 CorrectionofartemisdeficiencyinmurinehematopoieticstemcellbyI-sce1meganucleaseandartemisrecombinationmatrixmediatedhomologousrecombinationJulie Rivière, Hôpital Necker, Paris

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Syndicate 4 4d: non-viral technologiesChairs: Hidde Haisma, Matthew WoodInV39 endosomalescapepathwaysfordeliveryofbiologicals

Hidde Haisma, Groningen Research Institute of Pharmacy, Groningen University

InV40 exosome-mediatedrnAideliveryforneurologicaldiseaseMatthew Wood, University of Oxford

Proffered papersOr29 replicatingminicircles:Overcomingthelimitationsoftransientand

stableexpressionsystemsJuergen Bode, Hannover Medical School (MHH)

Or30 Developmentofminicircle-DnAvectorsasnon-viralliver-directedgenetherapyforphenylketonuria(PKu)Hiu Man Viecelli, University of Zurich

Or31 OrallyadministeredchitosannanoparticlesforgenedeliveryofFIXtotreathemophiliaBPatricia Quade-Lyssy, DRK-Blutspendedienst Baden-Württemberg, Hessen

18.15 sussexhighteaandsparklingwinewithmoderatedposters

20.00 Adjourn

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09.00 Plenary session 5 : Gene therapy in infectious disease

Auditorium 1 Chairs: Uta Griesenbach, Mary Collins InV41 ZFn-editedCD4tcellsforhIV/AIDstherapy:Phase1trialsto

evaluatethesafetyandtolerabilityofsB-728-tinhIV-infectedsubjectsPhil Gregory, Sangamo BioSciences, California

InV42 GeneticvaccinesformalariaAdrian Hill, University of Oxford

InV43 restrictionfactorsandgenetherapyforhIVGreg Towers, University College London

10.30 morningbreak

11.00 Parallel sessions 5a, 5b, 5c

Syndicate 1 5a: manipulation of the vasculatureChair: Stuart NicklinInV44 micrornAsinpost-ischemicangiogenesis

Costanza Emanueli, Laboratory of Vascular Pathology and Regeneration, School of Clinical Sciences, University of Bristol

InV45 GenetherapyoffamilialhypercholesterolemiaSeppo Ylä-Herttuala, A. I. Virtanen Institute, University of Eastern Finland

Proffered papersOr32 hypoxia-regulatedhO-1expressionimprovespost-ischemicblood

flowandskeletalmuscleregenerationAgnieszka Jazwa, Jagiellonian University, Krakow

Or33 reducinglDlcholesterolwithexon-skippingantisenseoligonucleotidesPetra Disterer, University College London

Or34 roleofantioxidantsgenesinangiogenesis:significanceforendothelialprogenitorcellsJozef Dulak, Jagiellonian University, Krakow

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Syndicate 3 5b: Cell and vector manufacturingChair: Olivier DanosInV46 tBC

Olivier Danos, University College LondonInV47 Purificationandanalysisofvirus,VlPandpDnAvectorsforgene

therapywithCIm®monolithchromatographyJohn Creedy, Progressive Research Systems Ltd on behalf of BIA Separations GmbH

InV48 manufactureofclinical-gradelentiviralvectorsforex vivouseAnne Galy, Généthon, Paris

Proffered papersOr35 Improvedmanufacturingofmeaslesvirusforhumanoncolytic

virotherapyclinicaltrialsMark Federspiel, Mayo Clinic, Minnesota

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Auditorium 1 5c: neuromuscular gene therapyChair: George DicksonInV49 systemicAAV9genetherapyofspinalmuscularatrophy

Martine Barkats, Inserm, Institut de Myologie, Paris InV50 Genetictherapiesforduchennemusculardystrophy

George Dickson, Royal Holloway University of LondonInV51 effectivelimbtransductionandphenotypiccorrectionafterinjection

ofrAAV8-u7snrnAinGrmDdogsPhilippe Moullier, Généthon, Paris

Proffered papersOr36 taureprogrammingbyrnAtrans-splicing:agenetherapystrategyfor

neurodegenerativediseasesMaria-Elena Avale, King’s College London

12.30 lunch and poster session 3

Upstairs P090-P134 stemcellsandreprogramming,oculardisease,manufacture,regulatoryandethics,immunologicalandvaccinesandimaging)

Downstairs P236-P290 Cancer/vaccines/virotherapy,cardiovasculardiseaseandlatebreakingnews

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14.15 Plenary Session 6 : Emerging technologies

Auditorium 1 Chairs: Luigi Naldini, Christof von KalleInV52 Cellularrecognitionandrestriction

ofVirusesandVectorsMatthew Weitzman, The Children’s Hospital of Philadelphia

InV53 CorrectionoffactorIXgeneKathy High, Howard Hughes Medical Institute and The Children’s Hospital of Philadelphia

InV54 targetsiteselectionoftransposon-basedvectorsandstrategiesfortargetedtransgeneinsertionZoltan Ivics, Max Delbrück Center for Molecular Medicine, Berlin

Proffered papersOr37 AlpharetroviralsInvectors:Favorableintegrationcharacteristicsin

murinehematopoieticstemcellsanddecreasedgenotoxicityintheInVitroImmortalizationAssayJulia Debora Suerth, Hannover Medical School

16.00 Afternoonbreak

16.30 Plenary session 7: Fairbairn award

Auditorium 1 Proffered papers Or38 Geneticmodificationofcancercellsusingnon-viral,

episomals/mArvectorsforinvivotumourmodelingSuet Ping Wong, Imperial College London

Or39 Anovelgene-anddrug-basedcombinedinterventionforstrokeisneuroprotectiveinvitroandin vivoEmily Ord, University of Glasgow

Or40 AclusterofbasicaminoacidsinthefactorXserineproteasemediatesurfaceattachmentofadenovirus:FXcomplexesMargaret Duffy, University of Glasgow

Or41 sheepamnioticfluidderivedCD34+stemcellsengraftinnOD-sCIDgammamiceandinlambsafterprenatalautologoustransplantationS. W. Steven Shaw, University College London

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17.30-19.00 Parallel sessions 7a, 7b, 7c, 7d

Syndicate 1 7a: Genetic vaccinesChair: Kerry FisherInV55 novelchimpanzeeadenovirusvectorvaccinesinducing

adaptivecellularimmunityAlfredo Nicosia, Okairos, Rome

InV56 Pre-immunemodelsforimprovedvaccinedesignKerry Fisher, University of Oxford

Proffered papersOr42 tcellsexpressinggenticallymodifiedCD8co-receptorhaveenhanced

tcelleffectorfunctionEmma Morris, University College London

Or43 Antigen-expressingimmunostimulatoryliposomes:anovelgenetically-programmablevaccineplatformEnrico Mastrobattista, Utrecht University

Or44 ImprovedsystemsforgeneratingandevaluatingadenovirusvaccinevectorsrevealdifferencesinimmunogenicitybetweenvectorsofdifferentadenoviralspeciesMatthew Dicks, University of Oxford

Syndicate 3 7b: CnS cell and gene therapyChairs: Scott Ralph, Eric KremerInV57 Widespreadlong-termexpressioninprimateCnsusinghelper-

dependentcanineadenovirusvectorsEric Kremer, Institut de Génétique Moléculaire de Montpellier, Universités de Montpellier

InV58 enhancedpseudotypingefficiencyofhIV-1lentiviralvectorsbyarabies/vesicularstomatitisviruschimericenvelopeglycoproteinNicholas Mazarakis, Imperial College London

InV59 CurrentadvancesingenetherapyforspinalmuscularatrophyMimoun Azzouz, University of Sheffield

Proffered papersOr45 silencingofmutantataxin-3rescuesmotordeficitsandneurological

abnormalitiesinmachado-JosephdiseasetransgenicmiceLuis Pereira de Almeida, Center for Neurosciences and Cell Biology, University of Coimbra

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Syndicate 4 7c: new therapiesChair: Mark Tangney InV60 engineeringbacteriaforsimultaneousimagingandtherapyofcancer

Jung-Joon Min, Chonnam National University, Hwasun Hospital, South Korea

InV61 tumour-specificbacterialgrowth:avectortoolboxforlocalisingcancertherapeuticsMark Tangney, Cork Cancer Research Centre, University College Cork

Proffered papersOr47 AnewlentiviralvectorpseudotypeoutperformsbyfarVsV-G-lVsfor

genetransferintohematopoieticstemcellsEls Verhoeyen, Inserm, University of Lyon

Or48 ultrasound-enhanceddeliveryofpolymer-coatedoncolyticadenovirusfortumourgrowthinhibitionRobert Carlisle, University of Oxford

Auditorium 1 7d: Genotoxicity #2

Proffered papersOr46 Definingthelentiviralintegromeinhumanhematopoieticcells

Alessandra Recchia, University of Modena e Reggio EmiliaOr50 trackinghematopoieticstemcellfateinhumansbyretroviraltagging

Luca Biasco, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)Or51 lentiviralvectorcommonintegrationsitesintheAlDandmlD

clinicaltrialsandinapreclinicalmodelreflectabenignintegrationbiasandnotoncogenicselectionEugenio Montini, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

Or52 read-through/splicing-capturemechanismisthemajordeterminantofenhancergenotoxicityofvectorswithactiveltrsDaniela Cesana, HSR-TIGET, Milan

19.30 maincongress“funofthefair”eveningincludingdinner,fairgroundgamesandbar

sunDAY30OCtOBer

Brighton_2011.indd 45 07/10/2011 19:30

09.00 Plenary session 8 : Clinical trials #2

Auditorium 1 Chairs: Thierry VandenDriessche, Rafael YanezINV62   ProSavin® a gene therapy approach for the treatment of 

parkinson’s disease: phase I clinical trial updateStéphane Palfi, CEA-CNRS MirCen, France and Oxford BioMedica

INV63   OncoVEX GM-CSF: from bench, to bedside; to approved drugRobert Coffin, Biovex, Massachusetts

INV64   A Phase I/II clinical trial entailing peripheral vein administration of a novel self complementary adeno-associated viral vector encoding human FIX for Haemophilia B gene therapy Amit Nathwani, University College London

10.30 Morning break

11.00 Plenary session 9: Presidential symposium

Auditorium 1 Chair: Seppo Ylä HertuallaPresidential TalkSeppo Ylä Hertualla, A. I. Virtanen Institute, University of Eastern Finland

Outstanding Achievement AwardAdrian Thrasher, University College London Institute of Child Health

Young Investigator AwardsOR53    “Factor-free” murine induced pluripotent stem 

cells for monogenetic neutrophil disease modelsAxel Schambach, Medical School Hannover

OR54    Genome-wide analysis of zinc finger nuclease specificityRichard Gabriel, NCT Heidelberg

OR55   Developing safer gene therapy approaches by zinc finger nucleases-mediated gene editingAngelo Lombardo, HSR-TIGET, Milan

OR56   A Hematopoietic Stem Cell (HSC) specific microRNA renews gene therapy and gives novel insights into the regulation of HSC homeostasisBernhard Gentner, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

Poster and other presentations

12.30 Main congress adjourns

13.15 Clinigene Industry Satellite Workshop

The Restaurant

Joint platform between industry and centres of excellence

16.30 Adjourn

MONDAy 31 OCtOBER

Brighton_2011.indd 46 13/10/2011 09:31

ESGCT/BSGT CollaBoraTivE ConGrESS 2011

CLINIGENE INDUSTRY SATELLITE: JOINT PLATFORM BETWEEN INDUSTRY & CENTRES OF EXCELLENCE– 5TH EDITION

Monday October 31st 2011, Brighton (UK) 13:15 – 16:00

Industry-Academic centres of excellence collaboration in challenges for early-phase clinical trials

CHAIRMEN: Martin Wisher (BioReliance, UK) & Manuel Carrondo (IBET, PT)

13:15 Finger buffet

13:30 – INTRODUCTION 5’ by Martin Wisher (Chair of the CliniGene Industry Committee) & Manuel Carrondo

13:35 - PRESENTATIONS

• “The benefits and pitfalls of R&D collaborations with industry” 10+5’ Kerry Fisher (PsiOxus, UK)

• “Gene therapy development for orphan diseases requires more academics than trial subjects - illustration from the Glybera for LPLD case” 10+5’ Janneke de Wal (AMT, NL)

• “Merging Ad5 academic products and validated production processes” 10+5’ Robert Shaw (Ark Therapeutics, FI-UK)

• “An accessible academic translational infrastructure: our role and challenges after 10 years” 10+5’ Larry Couture (Beckman Research Institute of City of Hope, USA)

• "It takes a whole village to raise a child: Taking SB-728-T from concept to clinic” 10+5’ Philip Gregory (Sangamo, USA)

• Title pending 10+5’ Gabor Veres (BlueBird Bio, USA-FR)

• “Innovation is where you find it - one size does not fit all” 10+5’ Sam Wadsworth (Genzyme R&D Center, part of Sanofi Group)

15:20 - ROUND TABLE chaired by Alan Boyd (Alan Boyd Consultants, UK), Felicia Rosenthal (CellGenix, DE) & Jeffrey Ostrove (CereGene, USA):

• How do Academia & Industry collaborate with each other, and on which ground it is maintained,

• What are the main difficulties and successes,

• What are the bottlenecks (research, intellectual property, timelines, management, regulatory issues...),

• Suggestions to improve these kind of collaborations in the future, and based on past experience.

15:55 – MEETING WRAP-UP

Brighton_2011.indd 47 07/10/2011 19:31