ESGCT Congress Book

The Brighton Centre, Brighton | 27-31 October 2011

www.esgct.eu/congress/2011 • www.bsgt.org

Collaborative Congress 2011

European Society of Gene and Cell TherapyBritish Society for Gene Therapy

Cover image © www.visitbrighton.com Other images © www.visitbrighton.comDesign: www.catherinecharnock.co.uk

to landfill

ESGCT/BSGT COLLABORATIVE CONGRESS 2011

PLATINUM PARTNERS

GOLD PARTNERS

SILVER PARTNERS

CONGRESS SUPPORTERS

2 | CONTENTS

CONTENTS1 Partners – overview

3-5 Brighton Centre floorplans

6 Welcome address

7 Committees

8-13 Programme at a glance

14-20 Partners

22-25 Exhibitors

26-46 Programme

47 Clinigene Industry Satellite: joint platform between industry and centres of excellence

48 Congress office information

49 Travel information

51 Emergency procedure: The Brighton Centre

52-53 Congress social activities

54 Gala evening (Fun of the Fair)

56 ESGCT evaluation

58 European Society for Gene and Cell Therapy Achievement Awards

61 Brighton map


BRIGHTON CENTRE FLOORPLAN | 3

BRIGHTON CENTRE GROUND FLOOR

North Lifts

South Lifts

Box Office

Information Desk

To seafront

Foyer

Meeting Room 1

Syndicate WingEntrance

To car parks and shopping centre

Syndicate 1 & 2 Syndicate Wing

4 | BRIGHTON CENTRE FLOORPLAN

BRIGHTON CENTRE FIRST FLOOR

Auditorium 1

EastBar

SecurityDesk

WestBar

Medical Room

Auditorium 2

Office 22Bar

To car parks and shopping centre

North Lifts

South Lifts


BRIGHTON CENTRE FLOORPLAN | 5

BRIGHTON CENTRE SECOND FLOOR

Syndicate 3 & 4Syndicate Wing

Auditorium 1

MeetingRoom14

MeetingRoom 3

Office 4 Meeting

Room 5

North Lifts

6 | WELCOME ADDRESS

WELCOME ADDRESSOn behalf of the European Society of Cell and Gene Therapy and the Local Organising Committee it is my pleasure to welcome you to Brighton for the XIXth Annual Congress, which is being held this year in collaboration with the British Society for Gene Therapy.

The continued success of the annual conference mirrors the exciting developments in the field: each year we aim to offer a scientific programme that fully represents the variety of science and medicine under the joint umbrella of gene- and cell-based therapies, and we are delighted to welcome so many leading authorities to this year’s meeting. The theme for Brighton 2011 is “Letting the Science do the Talking” so you can expect a focus on cutting-edge science, translational research and the latest data from around the world. The rich and stimulating programme sees the contribution of 80 invited speakers, more than 50 selected oral presentations, and almost 400 poster presentations, and the high number of registrants is once again testimony to the quality and content of the meeting, as well as the organisation and international dimension.

With additional events for students, young scientists, industry, clinicians and principal investigators complemented by a programme of exciting social activities in a quintessentially English seaside town – famed for its stunning Royal Pavilion, elegant regency architecture, award winning restaurants and galleries – this Congress will provide a superb platform for international scientific interaction and knowledge transfer, and a unique opportunity to network with all the stakeholders in our dynamic and exciting field of research. By holding the Congress in a small, focussed area – with all hotels close to the conference centre – we are hoping to create a very friendly and interactive community feeling, perfect for building friendly relationships and collaborations. I hope that together we can continue to advance our scientific knowledge and build upon our existing excellence.

I look forward to sharing with you an outstanding and memorable scientific event. I would like to send my thanks and appreciation to all our contributors, sponsors and exhibitors, and to everyone who assisted with the Congress preparation and management, without whose commitment, enthusiasm and generosity this meeting would definitely not have been possible.

Len Seymour, ESGCT Congress President


COMMITTEES | 7

COMMITTEESCardiovascular diseasesAndy Baker (Chair) Seppo Ylä-Herttuala (Chair) Mauro Giacca Moshe Flugelman Keith Channon Patrick Most

Neurological and muscular diseasesNicole Déglon (Chair) Maurilio Sampaolesi (Chair) Nathalie Cartier-Lacave (Chair) Nicholas Mazarakis Dominic Wells Anders Björklund

Viral vectorsHildegard Büning (Chair) Luigi Naldini (Chair) Rob Hoeben Akseli Hemminki Anna Salvetti Alberto Epstein Els Verhoeyen

Genetic and metabolic diseasesEdvard Smith (Chair) Thierry VandenDriessche (Chair) Fatima Bosch Manuel Grez Robin Ali Beat Thony

Non-viral vectorsDaniel Scherman (Chair) Hidde Haisma (Chair) Ernst Wagner J. P. Behr George Dickson Zoltan Ivics

Infection, immune and vaccinesDavid Klatzmann (Chair) Mary Collins (Chair) Naomi Taylor Ben Berkhout Dorothee Von Laer Zelig Eshhar

Stem cells and reprogrammingStefan Karlsson (Chair) Chris Baum (Chair) Juan Bueren Tim O’Brien Catherine Verfaillie Katarina Le Blanc Marina Radrizzani Willem Fibbe

Ethics and regulatory affairsOdile Cohen-Haguenauer (Chair) Klaus Cichutek (Chair) Richard Ashcroft Alastair Kent Gösta Gahrton Serge Braun Otto Merten Martin Schleef Klaus Kühlcke Michael Fuchs

8 | PROGRAMME AT A GLANCE

EDUCATION DAYTwo parallel sessions taking place in Syndicate Rooms 3 and 4 P

28

13.00 1a: Strategies for delivery and expression of transgenesSyndicate 3

1b: Hot topics in gene and cell therapy and tissue engineeringSyndicate 4

15.00 Afternoon break

15.30 2a: ImmunologySyndicate 3

2b: Transgene integrationSyndicate 4

17.30 Adjourn

PROGRAMME AT A GLANCE

Please note: speakers and talk titles may change

THURSDAY 27 OCTOBER

PUBLIC ENGAGEMENT DAY: GENE THERAPY AND STEM CELLS: 21ST CENTURY MEDICINESyndicate Rooms 1 and 2 Sponsors: Wellcome Trust, Fanconi Hope, CGD Trust, GSK, Blubird Bio, TAGTC Pa

ge 2

6-27

10.00 Registration

10.30 What is gene and cell therapy?

12.30 Hands-on activities and interactive talks

13.15 Lunch, meet the researchers, networking bingo, careers advice

14.00 Zombie Science show and conversations with an expert

15.00 Brain gene therapy for real

15.30 Interactive workshop with The Naked Scientists

17.00 Keynote speaker – Professor Lord Winston

17.15 Adjourn

PROGRAMME AT A GLANCE | 9


THURSDAY 27 OCTOBER

CAT-ESGCT SATELLITE WORKSHOP: ADVANCED THERAPY MEDICINAL PRODUCTS: FROM PROMISE TO REALITY Auditorium 1 Sponsor: Bioreliance Pa

ge 2

9-30

08.30 Registration

09.15 Welcome and opening

09.30 Translating academic research into commercial products – regulatory considerations

09.30 1: Cell-based medicinal products

11.00 Coffee break

11.30 2: Gene therapy

12.30 3: European and national regulatory path for ATMPs

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 examples

15.15 Coffee break

15.45 5: Cell therapy and cell-based medicinal products – practical examples

17.00 Closing remarks

17.15 Adjourn

ESGCT AND BSGT CONGRESS OPENING CEREMONY Auditorium 1 P

31

10.00 Registration open

17.30 Welcome cocktail

19.00 Opening symposium

20.30 Adjourn


MAIN CONFERENCE08.00 Registration open

08.30 Plenary session 1: Advances in clinical trialsAuditorium 1Sponsors: CGD, CELL-PID Pa

ge 3

2

10.15 Morning break

10.45 1a: Respiratory gene and cell therapySyndicate 3

1b: Imaging technologies and applicationsSyndicate 1Sponsor: Clinigene

1c: Genotoxicity #1Auditorium 1Sponsors: Clinigene, SPP1230, PERSIST Pa

ge 3

2-33

12.15 Lunch and poster session 1

13.30 ESGCT General Assembly

14.15 Plenary session 2: Overcoming the hurdles to successful gene therapyAuditorium 1Sponsors: SPP1230 Pa

ge 3

4


16.30 2a: Regulatory round table Auditorium 1Sponsor: Clinigene

2b: Careers workshopSyndicate 1

2c: Mechanisms of cellular differentiationSyndicate 3

2d: Open poster / networking sessionFoyer

Page

34-

35

18.00 Poster party with moderated posters

19.00 Speakers’ dinner by invitation only

FRIDAY 28 OCTOBER



MAIN CONFERENCE07.30 Themed breakfast meetings

Sponsor: Charles River LaboratoriesRestaurant

09.00 Plenary session 3: Stem cells in health and diseaseAuditorium 1Sponsor: PERSIST Pa

ge 3

6

11.00 Morning break

11.30 3a: The Eye – cell and gene therapySyndicate 3Sponsors: Clinigene, AAV Eye

3b: Cancer immunotherapy Auditorium 1

3c: iPS cells and control of differentiationSyndicate 1Sponsor: Clinigene Pa

ge 3

6-37


14.00 Update and perspectives on European advanced therapy researchAuditorium 1 P3

8

14.45 Plenary session 4: Cancer gene and virotherapy (ISCGT symposium)Auditorium 1Sponsors: PsiOxus, ISCGT, Jennerex Pa

ge 3

8


16.45 4a: Genetic diseaseSyndicate 3

4b: Cancer gene and virotherapyAuditorium 1Sponsor: Transgene

4c: Stem cells bioengineeringSyndicate 1Sponsor: Hyperlab

4d: Non-viral technologiesSyndicate 4

Page

38-

40

18.15 Sussex High tea and sparkling wine with moderated posters

20.00 Adjourn

SATURDAY 29 OCTOBER

PLEASE NOTE: At 2am tonight the clocks will turn back one hour for the end of Daylight Saving Time


MAIN CONFERENCE09.00 Plenary session 5 : Gene therapy in infectious disease

Auditorium 1 P 41

10.30 Morning break

11.00 5a: Manipulation of the vasculatureSyndicate 1Sponsor: Ark

5b: Cell and vector manufacturingSyndicate 3Sponsor: Généthon

5c: Neuromuscular gene therapyAuditorium 1Sponsor: Généthon

Page

41-

42


14.15 Plenary Session 6 : Emerging technologiesAuditorium 1Sponsors: Clinigene, PERSIST Pa

ge 4

3


16.30 Plenary session 7: Fairbairn AwardAuditorium 1Sponsor: The Paterson Institute Pa

ge 4

3

17.30-19.00 7a: Genetic vaccinesSyndicate 1Sponsor: Okairos

7b: CNS cell and gene therapySyndicate 3

7c: New therapiesSyndicate 4

7d: Genotoxicity #2Auditorium 1

Page

44-

45

19.30 Main congress “fun of the fair” evening including dinner, fairground games and bar

SUNDAY 30 OCTOBER



MAIN CONFERENCE09.00 Plenary session 8 : Clinical trials #2

Auditorium 1Sponsor: Clinigene Pa

ge 4

6

10.30 Morning break

11.00 Plenary session: Presidential symposiumAuditorium 1

Outstanding Investigator AwardYoung Investigator AwardsSponsors: PlasmidFactory, Molmed, Oxford Biomedica, Human Gene Therapy, BrainCAV, Sangamo

Page

46

12.30 Main congress adjourns

13.15 Clinigene Industry Satellite Workshop: Joint platform between industry and centres of excellenceThe RestaurantClinigene

Page

46

16.30 Adjourn

MONDAY 31 OCTOBER

14 | PARTNERS

PARTNERSPLATINUM PARTNERS

Clinigene is the European network for the advancement of clinical gene transfer and therapy: FP6-EC funded network of excellence fostering interaction of all stakeholders in the field in order to facilitate and help harmonise ethical, quality, safety, efficacy and regulatory issues.

www.clinigene.eu

MolMed is a medical biotechnology company focused on research, development and clinical validation of novel antitumor therapies, with key expertise in cell and gene therapies. MolMed’s service area, GMP Solutions, provides tailor made services for the manufacturing of patient-specific or genetically modified cells and active pharmaceutical ingredients for clinical use.

www.molmed.com

PERSIST project explores the use of highly innovative gene-modifying and delivery technologies, and capitalises on recent discoveries in gene expression control to develop radical solutions to the problem of precisely controlling the fate and expression of exogenous genetic information in gene therapy, with applications in these and other deadly diseases. The project combines more than 20 of Europe’s outstanding experts from eight countries in the field of genetic engineering for persisting gene expression.

www.persist-project.eu

BioReliance Corporation is a leading provider of cost-effective contract services to the pharmaceutical and biopharmaceutical industries, offering more than 1,000 tests or services related to biologics safety testing, specialised toxicology and animal health diagnostics. Our capabilities include a broad spectrum of cGMP- and GLP-compliant virological, immunological, molecular biology and microbial testing services.

www.bioreliance.com

PARTNERS | 15


GOLD PARTNERS

SPP1230 is an interdisciplinary, multicentric Research Priority Programme of the German Research Council (DFG) with the mission to investigate “Mechanisms of gene vector entry and persistence”. The projects investigate basic mechanisms of cell entry, episomal maintenance or chromosomal insertion of transgenes, and the cellular and systemic responses to genetic cell modification. The overall aim of this network is to improve efficiency, predictability and biosafety of genetic therapy with a focus on hematopoietic cells.

www.schwerpunktprogramm1230.de

Created in 1958 by a group of patients and their families, and recognised as being of public utility in 1976, AFM (French Muscular Dystrophy Association) has a single objective: to defeat neuromuscular diseases, which are devastating muscle-wasting diseases. It has set itself two missions: curing neuromuscular diseases and reducing the disabilities they cause.

www.afm-france.org

Généthon develops and manufactures gene therapy products for rare diseases with the goal of making these innovative treatments available to patients. To meet this challenge, Généthon has assembled the technical and human resources needed to accelerate the medical application of scientific discoveries arising from fundamental research. Strong translational research programmes (at preclinical and clinical stages) engage multi-disciplinary teams and are supported by a first-rate technological platform and cGMP capacity. Généthon currently sponsors several early-phase gene therapy clinical trials (including an international trial)

www.genethon.fr

Transgene is a publicly traded French bio-pharmaceutical company dedicated to the development of therapeutic vaccines and immunotherapeutic products in oncology and infectious diseases, and has four compounds in advanced clinical trials. Transgene has concluded strategic agreements for the development of two of its immunotherapy products, an option agreement with Novartis for the development of TG4010 to treat various cancers, and an in-licensing agreement with US-based Jennerex Biotherapeutics Inc., to develop and market JX594/TG6006, an oncolytic virus.Transgene has bio-manufacturing capacities for viral-based products.

www.transgene.fr

16 | PARTNERS

Jennerex, Inc. is a clinical-stage biotherapeutics company focused on the design, development and commercialisation of first-in-class, targeted oncolytic products for cancer. Our lead product JX-594 is currently in two mid-stage clinical trials in patients with liver cancer. Published studies of JX-594 have shown its ability to selectively target a variety of common cancer tumor types, with improved survival and an excellent safety profile.

www.jennerex.com

SILVER PARTNERS

Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in the development and commercialisation of innovative gene-based medicines. The Company has a platform of gene delivery technologies, which are predominately based on highly engineered viral systems.

www.oxfordbiomedica.co.uk

Sangamo BioSciences Inc. is developing zinc finger protein (ZFP) Therapeutics®, a new class of innovative medicines that function at the DNA level. Sangamo has ongoing clinical trials of treatments for diabetic neuropathy, HIV/AIDS, and recurrent glioblastoma multiforme. Other therapeutic programmes are focused on Parkinson’s disease, monogenic diseases and neuropathic pain.

www.sangamo.com

PlasmidFactory is Europe’s leading contract manufacturer for plasmid DNA. Production ranges from the research to the industrial scale. We produce plasmids in modern laboratories with high quality standards and according to your individual wishes.

www.PlasmidFactory.com

www.astrazeneca.com

PARTNERS | 17


The FP7-CELL-PID European project utilises genetically modified HSC and their descendants as immunotherapeutic cells to build a healthy immune system in Primary Immuno Deficiency patients. It gathers together pioneers clinical, scientists and industrial partners in the advanced therapies field and aiming at broad clinical application of safe cell-based therapies.

http://cordis.europa.eu/fetch?CALLER=FP7_PROJ_EN&ACTION=D&DOC=1&CAT=PROJ&RCN=96787

Okairos develops genetic vaccines for major infectious diseases and cancer, using a novel proprietary technology based on new, potent, replication-incompetent Adenovirus vectors. These vectors are not neutralised by human sera, and they hold promise for generating effective T cell responses, where existing vectors have failed.

www.okairos.it

PsiOxus Therapeutics Ltd develops novel therapeutics for serious diseases. We are an Oxford, London and Berlin based development stage biotechnology company with world leading scientists and a highly experienced management team. Our approach is to combine different elements including small molecules, viruses, polymers and other macro-molecules to produce novel patent protected therapeutics. We deliberately target diseases that result in significant associated global morbidity and mortality, with a particular focus upon cancer.

www.psioxus.com

The Hyperlab consortium gathers nine organisations – academia, research institutes and SMEs – from seven countries. Each of these partners has been carefully selected to bring particular expertise or facilities to the consortium. They share a common interest in advancing stem cell research by developing innovative technologies, and will collaborate in an integrated, synergistic approach that will lead to results that would not be achievable by any of the partners on its own.

www.hyperlab.eu

18 | PARTNERS

www.wellcome.ac.uk

Charles River is a full-service, global contract research organisation committed to delivering high-quality products and services to the pharmaceutical industry. Our broad range of capabilities spans each phase of discovery and development in every major therapeutic area, enabling you to enhance productivity and increase speed to market.

www.criver.com

CONGRESS SUPPORTERS

Cancer Research UK ‘s vision is to conquer cancer through world-class research. We are the largest volunteer supported cancer research organisation in the world and we support the work of 3,000 scientists working across the UK.

www.cancerresearchuk.org

The ultimate goal of the AAVEYE consortium is to develop gene therapy strategies for the treatment of inherited severe photoreceptor diseases, namely of RP and LCA. The results of this research programme will provide the knowledge and validation to further develop novel AAV-mediated therapeutic approaches with a broad potential application in the retina and central nervous system.

www.aaveye.eu

www.cgd.org.uk

Fanconi Hope is a registered national charitable trust set up by parents of Fanconi Anaemia (FA) affected children and clinicians with an interest in FA. To support a UK Fanconi Anaemia National Registry. To promote awareness and understanding of Fanconi Anaemia among affected families, the medical profession and the general public. To encourage research in the area of Fanconi Anaemia.

www.fanconi.org.uk

PARTNERS | 19


www.tocagen.com

BrainCAV is an FP7 consortium of scientists and clinicians (11 partners and 6 countries) developing tools to understand and treat brain diseases using the unique capacity of canine adenovirus (CAV-2) vectors. BrainCAV is now in its 3rd year. For more information, see www.braincav.eu or contact Dr. EJ Kremer at Institut de Génétique Moléculaire de Montpellier ([email protected]) or Dr. Anton Ottavi: +33 (0)4 72 13 89 82.

www.braincav.eu

www.avantilipids.com

GlaxoSmithKline (GSK) has an established history of successfully researching and developing orphan drugs to treat rare diseases. Recognising the size of the challenge, but also the opportunity to deliver new medicines to patients, we announced the creation of a dedicated rare diseases unit in February 2010. Initially focusing on 200 rare diseases, we are collaborating with organisations and institutions to develop medicines, including gene and cell therapies, quicker and more effectively than ever before.

www.gsk.com

www.liebertpub.com

20 | PARTNERS

bluebird bio is developing innovative gene therapies for severe genetic disorders. The company’s proprietary platform treats genetic diseases by placing a functional gene into the patient’s extracted bone marrow stem cells, and transplanting these corrected stem cells back into the patient. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy and beta-thalassemia/sickle cell anemia.

www.bluebirdbio.com

www.visitbrighton.com

www.arktherapeutics.com

Amsterdam Molecular Therapeutics (AMT) is the world leader in gene therapy. Our first product, Glybera, is a gene therapy for the treatment of lipoprotein lipase deficiency, an orphan genetic disorder. Glybera is filed for marketing approval with the European Medicines Agency; a decision is expected this summer. If approved, as expected, Glybera will become the first gene therapy available to patients in Europe. Subsequent filings and marketing approvals are expected in North America. Glybera approval validates gene therapy as therapeutic platform and opens the way for the development of many new gene therapy medicines. AMT is developing a pipeline of gene therapy products including treatments for hemophilia B, acute intermittent porphyria and Duchenne muscular dystrophy.

www.amtbiopharma.com

Foreword L. Luzzatto and I. Verma

y Introduction including prospect for a ➤ O. Cohen-Haguenauerpan-european infrastructure for GT vectorsy Highlights on AAV mediated ➤ F. Bosch with E. Ayuso, V. Blouin, N. Cartier, G.Dickson, M. Giacca,gene transfer O. Merten, P. Moullier

y Retrovirus mediated gene transfer ➤ P. Cruz & M. Carrondo with C. Baum, C. Buchholz, K. Cichutek, state-of-the-art O. Cohen-Haguenauer, H. Hauser

y Highlights on lentivirus ➤ K. Cichutek & M. Schweizer with O. Cohen-Haguenauer,mediated gene transfer P. Cruz, N. Deglon, K. Mitrophanous

y Highlights on gene-modified ➤ G. Gahrton with E. Alici, O. Cohen-Haguenauer, P. Cruz, cell therapy D.Klatzmann, W. Lindermaier

y Adenovirus mediated gene transfer: ➤ S. Kochanek with M. Anton, P. Cruz, B. Gansbacher, S. Ylä-Herttualacurrent developmentsy Non-viral based gene transfer: ➤ G. Dickson with C. Baum, J. Bode, F. Bosch, Z. Itzvak, D. Sherman, a new era M. Schleef, M. Schmeer

y Highlights on iPS induction ➤ A. L. Bennaceur-Griscelli with C. Baum, E. Bayart, M. Chuah,technologies and genetic stability O. Cohen-Haguenauer, T. Vandendriessche

y Preclinical assessment tools ➤ A. Panet with A. Jacobs, C. von Kalle, D. Sherman, M. Schmidt

y Immune responses and Immunotoxicity ➤ D. Klatzmann with A. Galy, F. Mingozzi

y Biosafety platform state of the art ➤ C. von Kalle with A. Aiuti, O. Cohen-Haguenauer, E.Montini,M. Schmidt, M. Wisher

y Clinical trials ➤ B. Gänsbacher with R. Ali, A. Auricchio, A. Boyd, N. Cartier,M. Eriksdotter-Jönhagen, C. von Kalle, D. Klatzmann, S. Yla-Herttuala

y Ethical and Regulatory Issues ➤ O. Cohen-Haguenauer with L. d’Apote, A. Boyd, K. Cichutek, A. Kent,N. King, L. Luzzatto, C. Schröder

y Integration and Dissemination issues ➤ CliniGene coordinator with S. Aymé, B. Gänsbacher, T. Vandendriessche

Edited by CliniGene, the European Network for the Advancement of Clinical Gene Transfer and Therapy (Project N°: LSHB-CT-2006-018933)Book coordinator: O. Cohen-Haguenauer

Clinical Gene transfer: state of the art The CliniBook

Pre-order form – Public price: 95 € / Special price for ESGCT participants until 31/01/12: 80 €

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EC-FP6

Annonce CliniGenebookBat_Mise en page 1 22/09/11 11:19 Page1

22 | EXHIBITORS

EXHIBITORS

EXHIBITORS | 23


EXHIBITORS

Booth 1: Polyplus-transfection SA develops and sells innovative solutions for the delivery of nucleic acids in research, bioproduction and therapeutics. Polyplus-transfection has been ISO 9001-certified since 2002 and supplies its proprietary range of reagents for the transfection of genes, oligonucleotides and siRNA through a worldwide distributor network.

www.polyplus-transfection.com

Booth 2: Caliper Life Sciences is a leading provider of in vivo imaging systems, multispectral microscopy systems and LabChip (R) technology for molecular biology. The range of systems includes the IVIS bioluminescence, fluorescence and micro CT systems which are seen as the gold standard for in vivo imaging.

www.caliperLS.com/IVIS

Booth 3: Demand Thermo Scientific products to accelerate discovery and move science forward. The Thermo Scientific portfolio includes a broad array of high-quality instruments, reagents, laboratory consumables, equipment, and services – designed to help you run your laboratory at peak performance, from start to finish. See the entire Thermo Scientific line up at www.thermoscientific.com

www.thermoscientific.com

Booth 4: BioOutsource is a Contract Microbiology Testing Laboratory offering an extensive range of GMP, GLP and GCP services to support safety testing and characterisation of Biologics & Vaccines. Our testing follows the guidelines provided by ICH, FDA, EMEA and various pharmacopoeias which enables BioOutsource to optimise your testing programme to comply with multiple regulatory environments.

www.biooutsource.com

www.arktherapeutics.com

Booth 5: ATMI, (Nasdaq: ATMI) is a global leader in enabling process materials and process technology for semiconductor, display and life science industries. At ATMI, process ingenuity unleashes new process possibilities for customers.

www.atmi-lifesciences.com

24 | EXHIBITORS

Booth 6: GenoSafe is a CSO specialised in the evaluation of the quality, efficacy and safety of gene and cell therapy products. We propose a real partnership from research stages to clinical phases. Study design, development/validation of analytical methods, and product testing; control of viral vectors batches (rAAV, rHIV, rMLV); preclinical evaluation; clinical trial: patients’ follow-up

www.genosafe.org

Booth 7: EUFETS, a German based company, supports the development and commercialisation of cell and gene therapies. Services include cGMP-compliant manufacturing of viral vectors, genetically modified cells and, as a new service, in vitro transcribed RNA. EUFETS offers process and assay development, validation, quality control, storage and QP release. EUFETS also supports preclinical product development (R&D/GLP studies) of biologics with customised in vitro bioanalytical programmes.

www.eufets.com

Booth 8: Clinigene is the European network for the advancement of clinical gene transfer and therapy: FP6-EC funded network of excellence, fostering interaction of all stakeholders in the field in order to facilitate and help harmonise ethical, quality, safety, efficacy and regulatory issues.

www.clinigene.eu

Booth 9: BioReliance Corporation is a leading provider of cost-effective contract services to the pharmaceutical and biopharmaceutical industries, offering more than 1,000 tests or services related to biologics safety testing, specialised toxicology and animal health diagnostics. Our capabilities include a broad spectrum of cGMP- and GLP-compliant virological, immunological, molecular biology and microbial testing services.

www.bioreliance.com

Booth 10: MolMed is a medical biotechnology company focused on research, development and clinical validation of novel antitumor therapies, with key expertise in cell and gene therapies. MolMed’s service area, GMP Solutions, provides tailor made services for the manufacturing of patient-specific or genetically modified cells and active pharmaceutical ingredients for clinical use.

www.molmed.com

EXHIBITORS | 25


Booth 12: PeproTech was established in 1988 by a group of scientists who decided to focus their efforts on the development and production of recombinant cytokines for life-science research. Today, PeproTech is a world leader in supplying high quality cytokine products including E. coli, insect, and mammalian cell-derived recombinant proteins, their monoclonal/polyclonal antibodies, ELI SA development kits, and other cytokine-related reagents.

www.peprotechec.com

Booth 13: CELLON S.A. The RollerCell 40 is suitable for all current roller bottle applications in research laboratories and large scale manufacturing sites. The CELL-tainer is the next generation of disposable mixing bioreactors. The Rotary Cell Culture System is the only device that efficiently creates an environment that enables extremely fragile cell cultures and co-cultures of human and animal cell to grow into complex, sophisticated 3-D models in vitro.

www.cellon.lu

Booth 14: PlasmidFactory is Europe’s leading contract manufacturer for plasmid DNA. Production ranges from the research to the industrial scale. We produce plasmids in modern laboratories with high quality standards and according to your individual wishes.

www.plasmidfactory.com

Booth 16: Corning Life Sciences, together with our subsidiary Axygen BioScience, is a global manufacturer of tools and equipment for cell culture, molecular biology and drug screening. Products include centrifuges, liquid handling, thermal cyclers, electrophoresis, shakers, plastic cell culture consumables, purification kits and automation-friendly robotic tips.

www.corning.com

Booth 18: AgileBio specialises in scientific IT solutions for life sciences, R&D biotech and other industries. Founded in 2002, AgileBio continuously develops LabCollector, the first collaborative Intranet software for the management of lab information. LabCollector allows each scientist in the lab to manage and share their data quickly with other lab members. Built around independent modules that can interact with each other, LabCollector will manage a variety of day-to-day information like sample storage, experimental results, quality records and SOPs.

www.agilebio.com

26 | PROGRAMME

PROGRAMME

THURSDAY 27 OCTOBER

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 Welcome and introduction Professor Adrian Thrasher, President of the BSGT

11.00 What is gene therapy?Dr Maria Limberis, University of Pennsylvania

11.30 What is stem cell therapy?Dr Tristan McKay, Queen Mary University of London

12.00 The life of a clinician doing gene therapyProfessor Iain McNeish, Cancer Research UK

12.30 DNA origami hosted by Lynda Coughlan and Co, Glasgow UniversityInteractive demonstration involving the audience as a method for demonstrating the complexity of DNA and how it is applied as a biomedicine

13.00 Short talk by Sense About Science and careers in science and the media

13.15 Lunch, meet the researchers, networking bingo, careers adviceA chance for attendees to quiz scientists directly about any aspect of their working lives or science

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

Sponsored by TAGTC

PROGRAMME | 27


THURSDAY 27 OCTOBER

15.00 Brain gene therapy for real Dr Ahad Rahim, University College London

15.30 The Naked Scientist hosts the DNA sequencing race, and the afternoon debate – scientists on the panelSchool 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 Keynote speech Professor Lord Winston

17.15 Adjourn

THURSDAY 27 OCTOBER

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


15.30 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 HBVFarzin 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

PROGRAMME | 29


THURSDAY 27 OCTOBER

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

30 | PROGRAMME

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 examplesGene therapy case study 1Gopalan Narayanan, CAT Member – UK – MHRAGene therapy case study 2: ADA-SCIDAlessandro Aiuti, HSR-Tiget, GTWP member Jonathan Appleby, GSKcGMP virus manufacture and evolving release testsEleanor 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 Coffee break

15.45 5: Cell therapy and cell-based medicinal products – practical examplesCell-based medicinal product case study 1Paula Salmikangas, CAT Vice-Chair – Finland – FimeaCell-based medicinal product study 2: 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 remarksSeppo Ylä-Herttuala, ESGCT PresidentLen Seymour, Congress PresidentChristian K. Schneider, European Medicines Agency – CAT Chair

17.15 Adjourn

THURSDAY 27 OCTOBER

PROGRAMME | 31


THURSDAY 27 OCTOBER

ESGCT AND BSGT CONGRESS OPENING CEREMONY Auditorium 1

10.00 Registration open

17.30 Welcome cocktail

19.00 Opening symposiumChair: Len SeymourThe potential of genetics researchProfessor Lord Winston, Imperial College LondonTumour viruses, rumour viruses and pseudo virusesRobin Weiss, University College London

20.30 Adjourn

32 | PROGRAMME

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

FRIDAY 28 OCTOBER

PROGRAMME | 33


FRIDAY 28 OCTOBER

Syndicate 1 1b: Imaging technologies and applicationsChair: Georges VassauxINV06 Monitoring gene therapy by non-invasive optical imaging

Kevin Francis, Caliper Life Sciences, Cheshire INV07 Molecular imaging of gene expression: NIS as a reporter gene

Georges Vassaux, Inserm U948, University of Nantes

Proffered papers:OR05 Multi-colour RGB marking to assess liver tumour development in vivo

Boris Fehse, University Medical Center Hamburg-EppendorfOR06 Capsid modifications with the potential for life cycle imaging and

retroviral protein transferTobias Maetzig, Hannover Medical School

OR07 Visualisation of active homing of mesenchymal stem cells into the tumor stroma of hepatocellular carcinoma (HCC) using the sodium iodide symporter as reporter geneKerstin Knoop, Ludwig-Maximilian-University Munich

Auditorium 1 1c: Genotoxicity #1Chair: Chris BaumINV08 Targeting integration to selected genomic

loci and in situ tailoring of cassette design allows robust transgene expression without perturbing endogenous transcriptionAngelo Lombardo, HSR-TIGET, Milan

INV09 Therapeutic strategies with integration-deficient lentiviral vectorsRafael Yanez, Royal Holloway University of London

Proffered papersOR08 Hepatic lentiviral gene transfer is associated with clonal selection, but

not with tumour formation in serially transplanted miceIna Rittelmeyer, Hannover Medical School

OR09 Large-scale rAAV integration site analysis in a preclinical mouse modelChristine Kaeppel, DKFZ/NCT, Heidelberg

OR10 Lentiviral vector-based insertional mutagenesis identifies new clinically relevant cancer genes involved in the pathogenesis of hepatocellular carcinomaMarco Ranzani, San Raffaele Scientific Institute, Milan

34 | PROGRAMME


Upstairs P011-P050 CNS, respiratory and genetic and metabolic disease

Downstairs P135-P184 New delivery technologies and non-viral vectors

13.30 ESGCT General Assembly

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

Auditorium 1 Chairs: Nathalie Cartier-Lacave, Charles Coutelle INV10 HSC gene therapy trial for metachromatic leukodystrophy

Alessandra Biffi, San Raffaele Scientific Institute, Milan INV11 Immune responses to capsids and transgene products in

pre-clinical and clinical trialsJim Wilson, University of Pennsylvania

INV12 Preclinical assays to assess genotoxicity and “phenotoxicity“Chris Baum, Institute of Experimental Hematology, Hannover Medical School

Proffered papersOR11 AAV capsids induce innate immune responses in human non-

parenchymal liver cellsHildegard Büning, University of Cologne


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

Auditorium 1 2a: Regulatory round tableChairs: Gösta Gahrton, Alastair Kent, Nancy KingRetroviral and lentiviral vectors for long-term gene correction; clinical challenges in vector and trial design: presentation of the report on the joint RAC-Clinigene meeting in bethesda1. Jacqueline Corrigan-Curray, NIH-OBA RAC, Maryland 2. Dan Takefman, FDA-CBER, MarylandFirst-in-human trial design and decisions: lessons from gene transfer researchNancy M. P. King, Center for Bioethics, Health, and Society, North CarolinaThe necessity for data-sharing towards advancement of clinical translation: building up sample IMPD and substantiating master filesKlaus Cichutek, Paul-Ehrlich-Institut, LangenEMA-CAT Focus group to facilitate navigation of ATMP regulatory frameworkChristian K. Schneider and Lucia d’Apote, EMA

FRIDAY 28 OCTOBER

PROGRAMME | 35


Syndicate 1 2b: Careers workshopChair: Lynda CoughlanINV13 Advancing your research career

Ross English, Vitae, South East Hub, UKINV14 An overview of scientific journals and publishing

Jim Wilson, University of PennsylvaniaINV15 European Research Council: supporting independent research

careersFiona Kernan, ERC, Brussels

INV16 Science communication Julia Wilson, Sense about Science, London

Syndicate 3 2c: Mechanisms of cellular differentiationINV17 Using adult tissues for generating new organs: autologous cell

replacement therapy for diabetesSarah Ferber, Sheba Medical Centre, Tel Aviv

INV18 Mesenchymal to epithelial transitioning enhances the reprogramming of human non pancreatic endocrine cells towards beta cells Kevin Docherty, University of Aberdeen

Proffered papersOR12 Enhanced myogenic potential of mesoangioblast-derived induced

pluripotent stem cellsMattia Quattrocelli, K.U. Leuven

Foyer 2d: Open poster / networking session

18.00 Poster party with moderated posters

19.00 Speakers’ dinner by invitation only

FRIDAY 28 OCTOBER

36 | PROGRAMME

07.30The Empress Suite, The Grand Hotel

Themed breakfast meetings

09.00 Plenary session 3: Stem cells in health and disease

Auditorium 1 Chairs: Chris Denning, Chiara Bonini INV19 Human induced pluripotent stem cells for cell

therapy against inherited metabolic disordersLudovic Vallier, University of Cambridge

INV20 Spinal cord regeneration using ES cellsJane Lebkowski, Geron Corporation, California

INV21 Cancer stem cells: a source of new therapeutic targets?Norman Maitland, University of York

INV22 Exploring and exploiting TALE nucleases (TALENs) for targeted genomeToni Cathomen, Hannover Medical School

11.00 Morning break


Syndicate 3 3a: The Eye – cell and gene therapyChair: Robin AliINV23 Development of stem cell therapy for the

treatment of retinal degeneration Robin Ali, University College London Institute of Ophthalmology

INV24 Utilisation of iPS cells for retinal degenerative diseasesMasayo Takahashi, RIKEN, Kobe

INV25 Challenges and successes of retinal gene therapy with AAV vectorsAlberto Auricchio, The Telethon Institute of Genetics and Medicine (TIGEM) and Federico II University, Naples

Proffered papersOR13 Gene replacement therapy in mouse models of inherited cone

photoreceptor disordersAlexander Smith, University College London

OR14 AAV8-mediated expression of VEGF antagonists in macaque eye: comparison of subretinal vs. intravitreal delivery of vectorMaria Limberis, University of Pennsylvania

SATURDAY 29 OCTOBER

PLEASE NOTE: At 2am tonight the clocks will turn back one hour for the end of Daylight Saving Time

PROGRAMME | 37


SATURDAY 29 OCTOBER

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

immunotherapiesDavid Klatzmann, Groupe Hospitalier Pitié Salpêtrière, Paris

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

Proffered papersOR15 Disruption of HLA expression to enable allogeneic cells to escape

immune recognitionMichael Holmes, Sangamo BioSciences, California

OR16 Targeting chemoresistant myeloid leukemia and multiple myeloma through genetic redirection of T cells against CD44 variant 6Monica Casucci, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

OR17 Editing human lymphocyte specificity for safe 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 stem 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 DiseaseChris Denning, University of Nottingham

Proffered papersOR18 Generation of fanconi anemia iPS cells with a targeted integration of

FANCA in the AAVS1 locusRocío Baños, CIEMAT/CIBERER, Madrid

OR19 Zinc-finger nuclease-mediated correction of the cellular RS-SCID phenotypeShamim Rahman, Hannover Medical School

OR20 Reprogramming of fibroblasts using mRNA encoding four reprogramming factorsGeertrui Tavernier, Gent University

SATURDAY 29 OCTOBER


Upstairs P051-P089 Stem cell bioengineering, Genotoxicity/targeted integration and neuro and muscle

Downstairs P185-P235 Viral 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 KasaharaINV30 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

INV31 Virotherapy in the UKKevin Harrington, Institute for Cancer Research, London

INV32 Molecular design of improved adenovirusesRamon Alemany, Catalan Institute of Oncology-IDIBELL, Barcelona


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

Syndicate 3 4a: Genetic diseaseChairs: Steve Howe, Suzy BuckleyINV33 Fetal and neonatal gene therapy

Simon Waddington, University College LondonINV34 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 VIII expression from a novel expression cassette with a potent codon-optimised FVIII constructJenny McIntosh, University College London

Sponsored by ISCGT

PROGRAMME | 39


SATURDAY 29 OCTOBER

Auditorium 1 4b: Cancer gene and virotherapyChairs: Iain McNeish, Len SeymourINV35 Targeting DNA damage repair and paclitaxel

resistance in ovarian cancer using oncolytic adenovirusesIain McNeish, Barts Cancer Institute, Queen Mary University of London

INV36 An ascending dose phase 1 trial of the safety and tolerability of Toca 511 in subjects with recurrent high grade gliomaDoug Jolly, Tocagen Inc., San Diego

Proffered papersOR24 Human data with a desmoglein 2 binding oncolytic adenovirus Ad3-

hTERT-E1AOtto Hemminki, University of Helsinki

OR25 Enhancement of antitumour efficacy of E3B-deleted oncolytic adenoviruses by targeting STAT1 that interacts with E3 14.7 protein of adenovirus in macrophageYaohe Wang, Barts Cancer Institute, Queen Mary University of London

OR26 Adenovirus with hexon Tat-PTD modification exhibits increased therapeutic effect in experimental neuroblastoma and neuroendocrine tumorsDi Yu, Rudbeck Lab Klinimm(IGP), Uppsala

Syndicate 1 4c: Stem cells bioengineeringChairs: Heiko Zimmermann, Paula Alves INV37 Technological strategies and microfluidic

concepts for stem cell procedures – the HYPERLAB approachHeiko Zimmermann, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

INV38 Embryonic stem cell lines: from biology to products Petter Björquist, Cellartis AB, Göteborg

Proffered papersOR27 Non-viral sonoporation gene therapy for orthotopic bone

regenerationGeorg Feichtinger, LBI Trauma, Vienna

OR28 Correction of artemis deficiency in murine hematopoietic stem cell by I-Sce 1 meganuclease and artemis recombination matrix mediated homologous recombinationJulie Rivière, Hôpital Necker, Paris

40 | PROGRAMME

Syndicate 4 4d: Non-viral technologiesChairs: Hidde Haisma, Matthew WoodINV39 Endosomal escape pathways for delivery of biologicals

Hidde Haisma, Groningen Research Institute of Pharmacy, Groningen University

INV40 Exosome-mediated RNAi delivery for neurological diseaseMatthew Wood, University of Oxford

Proffered papersOR29 Replicating minicircles: Overcoming the limitations of transient and

stable expression systemsJuergen Bode, Hannover Medical School (MHH)

OR30 Development of minicircle-DNA vectors as non-viral liver-directed gene therapy for phenylketonuria (PKU)Hiu Man Viecelli, University of Zurich

OR31 Orally administered chitosan nanoparticles for gene delivery of FIX to treat hemophilia BPatricia Quade-Lyssy, DRK-Blutspendedienst Baden-Württemberg, Hessen

18.15 Sussex high tea and sparkling wine with moderated posters

20.00 Adjourn

SATURDAY 29 OCTOBER

PROGRAMME | 41


SUNDAY 30 OCTOBER

09.00 Plenary session 5 : Gene therapy in infectious disease

Auditorium 1 Chairs: Uta Griesenbach, Mary Collins INV41 ZFN-edited CD4 T cells for HIV/AIDS Therapy: Phase 1 trials to

evaluate the safety and tolerability of SB-728-T in HIV-infected subjectsPhil Gregory, Sangamo BioSciences, California

INV42 Genetic vaccines for malariaAdrian Hill, University of Oxford

INV43 Restriction factors and gene therapy for HIVGreg Towers, University College London

10.30 Morning break


Syndicate 1 5a: Manipulation of the vasculatureChair: Stuart NicklinINV44 microRNAs in post-ischemic angiogenesis

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

INV45 Gene therapy of familial hypercholesterolemiaSeppo Ylä-Herttuala, A. I. Virtanen Institute, University of Eastern Finland

Proffered papersOR32 Hypoxia-regulated HO-1 expression improves post-ischemic blood

flow and skeletal muscle regenerationAgnieszka Jazwa, Jagiellonian University, Krakow

OR33 Reducing LDL cholesterol with exon-skipping antisense oligonucleotidesPetra Disterer, University College London

OR34 Role of antioxidants genes in angiogenesis: significance for endothelial progenitor cellsJozef Dulak, Jagiellonian University, Krakow

42 | PROGRAMME

Syndicate 3 5b: Cell and vector manufacturingChair: Olivier DanosINV46 TBC

Olivier Danos, University College LondonINV47 Purification and analysis of virus, VLP and pDNA vectors for gene

therapy with CIM® monolith chromatographyJohn Creedy, Progressive Research Systems Ltd on behalf of BIA Separations GmbH

INV48 Manufacture of clinical-grade lentiviral vectors for ex vivo useAnne Galy, Généthon, Paris

Proffered papersOR35 Improved manufacturing of measles virus for human oncolytic

virotherapy clinical trialsMark Federspiel, Mayo Clinic, Minnesota

SUNDAY 30 OCTOBER

Auditorium 1 5c: Neuromuscular gene therapyChair: George DicksonINV49 Systemic AAV9 gene therapy of spinal muscular atrophy

Martine Barkats, Inserm, Institut de Myologie, Paris INV50 Genetic therapies for duchenne muscular dystrophy

George Dickson, Royal Holloway University of LondonINV51 Effective limb transduction and phenotypic correction after injection

of rAAV8-U7snRNA in GRMD dogsPhilippe Moullier, Généthon, Paris

Proffered papersOR36 Tau reprogramming by RNA trans-splicing: a gene therapy strategy for

neurodegenerative diseasesMaria-Elena Avale, King’s College London


Upstairs P090-P134 Stem cells and reprogramming, ocular disease, manufacture, regulatory and ethics, immunological and vaccines and imaging)

Downstairs P236-P290 Cancer/vaccines/virotherapy, cardiovascular disease and late breaking news

PROGRAMME | 43


SUNDAY 30 OCTOBER

14.15 Plenary Session 6 : Emerging technologies

Auditorium 1 Chairs: Luigi Naldini, Christof von KalleINV52 Cellular Recognition and Restriction

of Viruses and VectorsMatthew Weitzman, The Children’s Hospital of Philadelphia

INV53 Correction of factor IX geneKathy High, Howard Hughes Medical Institute and The Children’s Hospital of Philadelphia

INV54 Target site selection of transposon-based vectors and strategies for targeted transgene insertionZoltan Ivics, Max Delbrück Center for Molecular Medicine, Berlin

Proffered papersOR37 Alpharetroviral SIN vectors: Favorable integration characteristics in

murine hematopoietic stem cells and decreased genotoxicity in the In Vitro Immortalization AssayJulia Debora Suerth, Hannover Medical School


16.30 Plenary session 7: Fairbairn Award

Auditorium 1 Proffered papers OR38 Genetic modification of cancer cells using non-viral,

episomal S/MAR vectors for in vivo tumour modelingSuet Ping Wong, Imperial College London

OR39 A novel gene- and drug-based combined intervention for stroke is neuroprotective in vitro and in vivo Emily Ord, University of Glasgow

OR40 A cluster of basic amino acids in the factor X serine protease mediate surface attachment of adenovirus:FX complexesMargaret Duffy, University of Glasgow

OR41 Sheep amniotic fluid derived CD34+ stem cells engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantationS. W. Steven Shaw, University College London

44 | PROGRAMME

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

Syndicate 1 7a: Genetic vaccinesChair: Kerry FisherINV55 Novel chimpanzee adenovirus vector vaccines inducing

adaptive cellular immunityAlfredo Nicosia, Okairos, Rome

INV56 Pre-immune models for improved vaccine designKerry Fisher, University of Oxford

Proffered papersOR42 T cells expressing gentically modified CD8 co-receptor have enhanced

T cell effector functionEmma Morris, University College London

OR43 Antigen-expressing immunostimulatory liposomes: a novel genetically-programmable vaccine platformEnrico Mastrobattista, Utrecht University

OR44 Improved systems for generating and evaluating adenovirus vaccine vectors reveal differences in immunogenicity between vectors of different adenoviral species Matthew Dicks, University of Oxford

Syndicate 3 7b: CNS cell and gene therapyChairs: Scott Ralph, Eric KremerINV57 Widespread long-term expression in primate CNS using helper-

dependent canine adenovirus vectorsEric Kremer, Institut de Génétique Moléculaire de Montpellier, Universités de Montpellier

INV58 Enhanced pseudotyping efficiency of HIV-1 lentiviral vectors by a rabies/vesicular stomatitis virus chimeric envelope glycoproteinNicholas Mazarakis, Imperial College London

INV59 Current advances in gene therapy for spinal muscular atrophyMimoun Azzouz, University of Sheffield

Proffered papersOR45 Silencing of mutant ataxin-3 rescues motor deficits and neurological

abnormalities in Machado-Joseph disease transgenic miceLuis Pereira de Almeida, Center for Neurosciences and Cell Biology, University of Coimbra

SUNDAY 30 OCTOBER

PROGRAMME | 45


Syndicate 4 7c: New therapiesChair: Mark Tangney INV60 Engineering bacteria for simultaneous imaging and therapy of cancer

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

INV61 Tumour-specific bacterial growth: a vector toolbox for localising cancer therapeuticsMark Tangney, Cork Cancer Research Centre, University College Cork

Proffered papersOR47 A new lentiviral vector pseudotype outperforms by far VSV-G-LVs for

gene transfer into hematopoietic stem cellsEls Verhoeyen, Inserm, University of Lyon

OR48 Ultrasound-enhanced delivery of polymer-coated oncolytic adenovirus for tumour growth inhibitionRobert Carlisle, University of Oxford

Auditorium 1 7d: Genotoxicity #2

Proffered papersOR46 Defining the lentiviral integrome in human hematopoietic cells

Alessandra Recchia, University of Modena e Reggio EmiliaOR50 Tracking hematopoietic stem cell fate in humans by retroviral tagging

Luca Biasco, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)OR51 Lentiviral vector common integration sites in the ALD and MLD

clinical trials and in a preclinical model reflect a benign integration bias and not oncogenic selectionEugenio Montini, The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)

OR52 Read-through/splicing-capture mechanism is the major determinant of enhancer genotoxicity of vectors with active LTRsDaniela Cesana, HSR-TIGET, Milan

19.30 Main congress “fun of the fair” evening including dinner, fairground games and bar

SUNDAY 30 OCTOBER

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


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

48 | CONGRESS OFFICE INFORMATION

CONGRESS OFFICE INFORMATIONContact namesClare Beach – Administration Manager Renée Watson – Business Manager

Registration desk opening hoursThursday 27 October 08.00-20.00

Friday 28 October 07.45-19.00

Saturday 29 October 08.00-15.00

Sunday 30 October 08.30-19.30

Monday 31 October 08.30-13.30

For information on BSGT and local attractions, please go to the BSGT and Visit Brighton information points, which will be located in the foyer and will be open from 10.00-16.30

Payment queriesPlease go to the registration desk during the above opening hours

Membership queriesPlease go to the registration desk during the above opening hours

Contact number in case of emergencyRenée Watson, ESGCT Business Manager, +44 7903 233064


TRAVEL INFORMATION| 49

TRAVEL INFORMATIONConference venue The Brighton Centre Kings Road, Brighton, East Sussex, BN1 2GR Tel: 01273 290131 Fax: 01273 779980 www.brightoncentre.co.uk

Getting around in BrightonWalking is one of the best ways to discover the city. People sometimes forget that Brighton is rich in heritage and simply bursting with stunning Regency architecture. Combine this with a compact, walkable city and you’ve got the perfect place for a walking tour.

You can download excellent walking maps and information from: www.visitbrighton.com/site/maps-guides-and-interactive/maps

The ‘Flavour of the Lanes’ walking tour gives a great taste of all that you should see, and is possible to do in chunks of time across the week: www.visitbrighton.com/site/maps-guides-and-interactive/walking-tours

LOCAL TAXISThe main city centre taxi ranks are in East Street, Queen’s Square and at the Rail Station.

Brighton and Hove Radio Cabs: 01273 204060City Cabs: 01273 205205Streamline Taxis: 01273 202020 / 747474

LOCAL BUSESBrighton and Hove Buses cover all areas of the city centre and beyond. ‘CitySAVER’ tickets offer unlimited travel on virtually all bus services in the city. If you wish to combine both train and bus travel, just ask for a ‘Plusbus’ ticket when buying your train ticket.

Increasing numbers of Gene Therapy studies report on therapeutic efficacy, in particular for the treatment of rare monogenetic diseases. However, the broader use of Gene Therapy concepts is currently precluded by the occurrence of adverse events. Understanding the basic mechanisms of vector-mediated gene delivery and cell modification will be key to improving the safety and also efficacy of Gene Therapy.

The Research Priority Program 1230 “Mechanisms of Gene Vector Entry and Persistence” of the German Research Council (DFG) was initiated in 2006 to address these core questions. Specifically, projects in the “Area Vector Fate” investigate aspects of early interaction between vectors and their target cells, such as vector entry, episomal persistence or chromosomal insertion of transgenes, while projects of the “Area Cell Fate” focus on deciphering the factors that determine the fate of gene-modified cells in the organism.

To comprehensively tackle these core research topics, projects belonging to the fields of virology, cell biology, haematology, bioinformatics and mathematical modelling were selected by an international board of reviewers. Coherence and timeliness of this multidisciplinary research consortium is further ensured by extensive internal and external networking, and through organisation and support of national and international congresses.

For further information on the Research Priority Program SPP1230 visit www.schwerpunktprogramm1230.de

RESEARCH PRIORITY PROGRAM 1230 (SPP1230)MECHANISMS OF GENE VECTOR ENTRY AND PERSISTENCE


EMERGENCY PROCEDURE | 51

EMERGENCY PROCEDURE: THE BRIGHTON CENTRE

1. UPON HEARING THE ALARM (INTERMITTENT)

a) The alarm will be silenced whilst the activated area is checked.

b) Standby and await further information.

2. UPON HEARING THE ALARM (CONTINUOUS RINGING)

a) LEAVE THE BUILDING by the nearest available exit – follow all instructions given by Brighton Centre Staff and Duty Manager.

b) DO NOT wait to collect personal belongings (e.g. coats, handbags) and DO NOT wait around for others. DO NOT USE THE LIFTS.

c) DO NOT run or panic – there is no need. Move swiftly but calmly.

d) CLOSE THE DOOR if you are the last to leave the room you are in.

e) PROCEED to the assembly point at the junction of RUSSELL ROAD/WEST STREET at the rear of the building.

f ) WAIT at the assembly point for further instruction from Brighton Centre staff.

g) DO NOT RE-ENTER THE BUILDING without permission.

3. IF YOU SHOULD DISCOVER A FIRE (AND NO ALARM HAS YET BEEN SOUNDED)

a) OPERATE THE ALARM from the nearest alarm point.

b) Proceed as per point 2 above.

52 | SOCIAL ACTIVITIES

CONGRESS SOCIAL ACTIVITIESSome of the greatest ideas come from discussions over a pint! In many ways the social activities can make the congress a real experience so we have incorporated the best of Britain and Brighton in the following social activities:

Whole Congress “Fun of the Fair” Gala eveningSunday 30 October, 19.30, Hilton Brighton Metropole Hotel (see page 54 for details).

Morning jog, bicycling and sea kayakingIf you fancy swapping your lab coat for a pair of trainers, then Sunday morning will give you a chance to test yourself against your fellow delegates in a morning jog along the beach. If you aren’t so keen on running but still want to get out on the beach there will be sea kayaks or bicycles available for you to have a try of with the help of some local experts to show you how it’s done.

BIKE RENTALBrighton Sports Company is pleased to provide a bike rental service for our Congress delegates on Brighton and Hove seafront. The service adds a new dimension to the services on offer in one of the busiest beach resorts in the UK. Located right next to the coastal bike path on Madeira Drive, Brighton, it is the perfect location to see the best of what Brighton and Hove has to offer.

Opening hours:07.00-09.00 – Pre booking only. 10.00-18.00 – Open bookings and turn up and ride subject to availability. 18.00-19.00 – Pre booking only

1 hour: £5 (normally £6) 2 hours: £8 (normally £9) 3 hours: £10 (normally £12) 4 hours plus: £14 (normally £16)

Bookings can be made by email [email protected] or by phone 07917 753794

Payment: cash only. Delegates will need to show conference ID to claim discounted rates.

Maps and advice on where to cycle are available with all bookings. All bikes come with locks and helmets if required.

mailto:mark%40brightonsports.co.uk?subject=bike%20rental


SOCIAL ACTIVITIES | 53

KAYAKINGHead on down to Brighton Watersports and experience Brighton in a way most others don’t! See Brighton’s West Pier up close from the far side; get that freedom of being out on the open waves without exerting the energy required by swimming; open your eyes to a new view of Brighton! We offer anything from kayaking, stand up paddle boarding, wakeboarding, water-skiing, ringo’s and more!

Session times: 07.30-09.30 12.00-14.00 17.00-18.30Sessions last 40 minutes and the first 100 people to book will get their session for free! You must quote the congress when you book and show your delegate badge when you arrive to get this offer.

Bookings can be made by phone on 01273 323160 (we will need 12 hours’ notice to secure your kit)

How to find us: we are situated on the seafront, down on the beach itself, half way between the palace and West Pier, in front of the Old Ship Hotel.

Contact details: The Brighton Watersports, 185 Kings Road Arches, Brighton Seafront, BN1 1NB [email protected]

Poster party and English afternoon teaIf there is one thing the English do well it is scones and tea! We couldn’t think of a better marriage than high quality scientific discussion of posters with an English Afternoon Tea. This is a great chance to get to chat with our exhibitors as well whose products can make a big difference to your work.

Good food, yes in England!We will be doing our best to bust those myths about English food by investing in a really delicious menu for the congress. We will be having bowl food most days which offers a selection of nutritious, hot meals and desserts including plenty of options for vegetarians. In addition there will be free coffee, tea, water and fruit available throughout the day to keep you going.

Fun of the FairSunday 30 October 2011

Hilton Brighton Metropole Hotel19.30

All congress participants are invited to a night of fun, food and festivities, at a subsidised price of €30 (payable at the registration desk).

We will be bringing all the fun of Brighton Pier inside our hotel with ice cream, funfair stalls such as hoopla, coconut shy, cork rifle game and tin can

alley, and giant scalextrics. There will be a full bar all washed down with a healthy portion of fish ‘n chips.

Please make your way to Hilton Brighton Metropole Hotel (see map opposite) at 19.30 and follow the signs to

The ESGCT/BSGT Fun of the Fair evening.

If you haven’t already registered for this event on our website please come to the registration desk.

Your first two drinks FREE!

GENE THERAPY TREATMENTS FOR RARE DISEASESFrom research to treatments for patients

Created in 1990 by AFM (the Association Française contre les Myopathies) and funded by the donations of the French Telethon.

FOR MORE INFORMATION and details: www.genethon.fr © C

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locationBy Road

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By Air

From LondonM23/A23 from London, 40mins from junction 7 off the M25

From Heathrow International AirportM25 to junction 7, M23/A23 1 hr and 30 minutes drive

From Gatwick International Airport30 minutes drive down the A23

From London49 minutes

From Gatwick International Airport30 minutes

DistancesFrom Brighton Train Station to Hilton Brighton Metropole – 1 mile.

Access on foot via Queens Road, West Street and then west along the seafront

Taxis available from the train station

Distances10 minutes on foot 2 minutes by car

Nearest AirportGatwick International Airport

Distances30 minutes by train or by car

Hilton Brighton Metropole Hotel

ESGCT EVALUATIONWe do hope you have enjoyed the ESGCT/BSGT Collaborative Congress 2011. We really value your feedback about all aspects of the Congress. We would be very grateful if you could take a few minutes to complete this online questionnaire either during or soon after the Congress. There are several workstations with interenet access available by the registration desk that you can use to complete the survey:

www.surveymonkey.com/s/LQBDSWN

A link is also available from the ESGCT website.

If you enjoyed this meeting, would you consider hosting/organising an ESGCT or BSGT congress in 2014? If so, please forward your proposal to the ESGCT Board as soon as possible at [email protected], or the BSGT Board at [email protected].

Thank you in advance for your time.

ESGCT and BSGT Team

www.bsgt.org

Friday 9 March 2012

BSGT ANNUAL CONFERENCE, LONDON 2012

University College London Institute of Child Health

Savedate!the

Unique opportunities for young scientists to:• shape the programme

• chair sessions• present data

Currently enrolling: toCa 511 & 5-FC in multiCenter, asCending-dose studies in patients with reCurrent hgg

Key entry criteria:

• prior surgery, rt, and tmZ

• age 18 and over

For more information, including a listing of study site locations, please visit www.tocagen.com/clinicalstudiesor visit www.clinicaltrials.gov and search for Toca 511

Toca 511 is an investigational product. An investigational product is one that has not been proven to be safe and effective for this use and is not licensed by the US Food and Drug Administration or any other government agency. An investigational product can only be administered in a research study.

A New Treatment Approach Under Investigation for

Recurrent High Grade Glioma

EUROPEAN SOCIETY FOR GENE AND CELL THERAPYACHIEVEMENT AWARDSOutstanding Achievement Award: one award for an established researcher who has made a long term, outstanding contribution to the field.

YoungInvestigator Awards: €1000 and a guest speaker invitation for up to four researchers (max age 40) who are showing exceptional promise.

Best Abstract Award: €500 for the top two abstracts.

Travel grants: up to 10 awards of €250 for PhD and first post docs. These will be awarded on the basis of abstract score.

Application and nomination forms are available at www.esgct.eu/awards

ISSN: 1946-6536

Human Gene Therapy MethodsPart B

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ESGCT 20th Anniversary Congressin collaboration with the SFTCG

www.esgct.eu • www.sftcg.fr

26-29 October 2012

Savedate!the

European Society of Gene and Cell Therapy and

French Society of Cell and Gene Therapy

Will Hold a Joint Meeting in Versailles

ESGCT Congress Book

Documents

Transcript of ESGCT Congress Book