ŇŹŇŠŇŤŇ
European Society of Gene and Cell Therapy French Society for Cell and Gene Therapy Collaborative Congress 2012
ESGCT 21st Anniversary Congress in collaboration with the SETGYC
European Society of Gene and Cell Therapy and Spanish Society of Cell and Gene Therapy will hold a joint meeting in Madrid
ESGCT and SFTCG Congress 2012 • Programme and Information Book
24-28 October 2013
25-29 October 2012 | Palais des Congrès de Versailles
www.esgct.eu • www.setgyc.es www.esgct.eu • www.sftcg.fr FR FRYHUVBLPSRVHG LQGG RYH HUVBLPSRVHG LQGG LQGG
COnGress OffiCe infOrMatiOn Contact names
Payment queries
Gaëlle Jamar – event Manager Vanessa sampson – Payment & Membership
Please go to the registration desk during the above opening hours
Registration desk opening hours
Membership queries
thursday 25 October 08.00-20.00 friday 26 October 07.45-18.30 saturday 27 October 08.00-13.00 sunday 28 October 08.30-19.00 Monday 29 October 08.30-13.30
Contact number in case of emergency
Please go to the registration desk during the above opening hours Gaëlle Jamar, event Manager +44 7766 475379
Wifi is aVailaBle in the Palais Des COnGres Wifi naMe: sePCV lOGin: esGCt2012 PassWOrD: esGCt2012
Cover image © Mairie de Versailles Gala dinner images © 2012 Bartabas Other images © Mairie de Versailles, unless otherwise indicated Design: www.catherinecharnock.co.uk
to landfill
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Partners – OVerVieW
PlaTinum ParTnErS
GOld ParTnErS
SilVEr ParTnErS
COnGrESS SuPPOrTErS
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
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Contents ii
Congress office information
1
Partners – overview
3
Boards
4
Welcome address
5
scientific committees
12
Programme at a glance
16
Partners
26
exhibitors
36
Programme
82
Congress social activities
84
travel information
85
Car parking near the Palais des Congrès
86
Restaurants near the Palais des Congrès
88
european society for Gene and Cell therapy Achievement Awards
88
esGCt evaluation
93
sFtCG Annual Congress 2013: Paris
93
Useful numbers
2
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
bOarDs ESGCT Board
Presidential Committee
seppo Ylä-herttuala, President luigi naldini, Vice-President Christof von Kalle, treasurer len seymour, general secretary robin ali Christopher baum Chiara bonini nathalie Cartier sarah ferber hidde haisma
nathalie Cartier, Chair Olivier boyer Odile Cohen-haguenauer anne galy salima hacein-bey David Klatzmann Philippe Moullier Marc Peschansky naomi taylor
SFTCG Board and local Organising Committee nathalie Cartier, Président anne Dubart Kupperschmitt, general secretary anne galy, treasurer Patrick Midoux, Vice-treasurer sophie gomez, Website Karim benihoud Pierre Cordelier alberto epstein sophie lebel-binay gilles Marodon Michel Pucéat anna salvetti
Organisers: Wats.on Consultancy: renée Watson, gaëlle Jamar, Vanessa sampson
With special thanks to albert tasteyre, DiM stempole biotherapie Mary Dean, asgCt freddy becher, local expert advisor stéphanie Yeunès, inserM Patrick Minari, inserM
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WelCOMe aDDress On behalf of the european society of gene and Cell therapy and the local organising committee, it is my pleasure to welcome you to Versailles for the annual Congress 2012, jointly organised with the french society for Cell and gene therapy. this 2012 edition is of particular importance, as we celebrate the 20th anniversary of the european society. With increasing numbers of clinical successes and promising new research leads, gene and cell therapy is a rapidly growing field in europe and worldwide. for this exceptional 2012 edition, we have worked to offer you an exciting programme, inviting 120 prestigious speakers and internationally acclaimed specialists in the fields of cell and gene therapy, immunotherapy, stem cells and regenerative medicine. 87 Oral presentations and almost 300 posters will be presented to offer you the latest data from around the world and to enable international scientific interactions. by holding the Congress in a small, focussed area, we hope to create a friendly and interactive community, allowing all members to discuss advances in cell and gene therapy, major concepts, and our vision for the future of a healthy world. Versailles is the ideal setting to come together and share in an important moment in france’s rich history. the Palais des Congrès de Versailles is resplendant in marble and oak, with frescoes by Chapelain-Midy, and bas-reliefs by Collarmarini. the Château de Versailles, a UnesCO world heritage site, is among the most beautiful achievements of 18th century french art. You will discover Versailles through pedestrian walkways, visits to the palace and its gardens, and the fabulous bartabas equestrian arts academy. i wish to warmly thank you for your participation and to thank our partners, sponsors and exhibitors for their major contributions. thanks also to the whole team that has assisted with the Congress preparation and management, without whose commitment, energy, enthusiasm and generosity this congress would never have been possible. nathalie Cartier
President of sftCg President of the esgCt 2012 Organising Committee
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
sCientifiC COMMittees Cardiovascular diseases
non-viral vectors
andy baker (Chair) seppo Ylä-herttuala (Chair) Mauro giacca Moshe flugelman Keith Channon Patrick Most
Daniel scherman (Chair) hidde haisma (Chair) ernst Wagner J.P. behr george Dickson Zoltan ivics
neurological and muscular diseases
infection, immune and vaccines
nicole Déglon (Chair) Maurilio sampaolesi (Chair) nathalie Cartier (Chair) nicholas Mazarakis Dominic Wells anders björklund
David Klatzmann (Chair) Mary Collins (Chair) naomi taylor ben berkhout Dorothee Von laer Zelig eshhar
Viral vectors
Stem cells and reprogramming
hildegard büning (Chair) luigi naldini (Chair) rob hoeben akseli hemminki anna salvetti alberto epstein els Verhoeyen
stefan Karlsson (Chair) Christopher baum (Chair) Juan bueren tim O’brien Catherine Verfaillie Katarina le blanc Marina radrizzani Willem fibbe
Genetic and metabolic diseases edvard smith (Chair) thierry VandenDriessche (Chair) fatima bosch Manuel grez robin ali beat thony
Ethics and regulatory affairs Odile Cohen-haguenauer (Chair) Klaus Cichutek (Chair) richard ashcroft alastair Kent gösta gahrton serge braun Otto Merten Martin schleef Klaus Kühlcke Michael fuchs
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Niveau Level
-1 BAR SALON 72 m²
ACCES / ACCESS Ascenseur / Lift Escalier / Stair Monte-charge / Goods lift
LULLI 350 m²
SERVICES Sanitaires / Restrooms TRAITEUR
Office Traiteur Caterers’ service area TRAITEUR
LES ESPACES / MEETING SPACE Salles / Rooms Entrées / Entrance
privé private
Niveau Level
ACCES / ACCESS
0 RACINE
MOLIÈRE
Bus RER
720 m²
Parkings / Car parks Ascenseur / Lift
MAZARIN
Escalier / Stair Monte-charge / Goods lift
-1
TRAITEUR
Ni
ve
au
SERVICES Sanitaires / Restrooms Vestiaires / Cloakroom TRAITEUR
Office Traiteur Caterers’ service area
HALL D'ACCUEIL lobby 300 m²
LES ESPACES / MEETING SPACE Salles / Rooms Entrées / Entrance Bureaux organisateurs
entrée
Privé
entrance
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Niveau Level
1
ACCES / ACCESS Ascenseur / Lift Escalier / Stair
SERVICES
AMPHITHÉÂTRE auditorium RICHELIEU 1 200 places
Sanitaires / Restrooms
LES ESPACES / MEETING SPACE Salles / Rooms Entrées / Entrance
SCÈNE stage SCÈNE VAUBAN 50 m²
Niveau Level
FOYER CONDÉ 100 m²
PASCAL 50 m²
2 Galerie LE NÔTRE 280 m²
ACCES / ACCESS
Balcon amphithéâtre Ba
Ascenseur / Lift
Auditorium balcony
300 places
Escalier / Stair
SERVICES Sanitaires / Restrooms
RICHELIEU 900 places
LES ESPACES / MEETING SPACE Salles / Rooms Entrées / Entrance
BOILEAU 50 m²
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COLBERT 130 m²
MONTESQUIEU 50 m²
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Please note: speakers and talk titles may change
ProgrAMMe At A gLAnce aSGct/ESGct EaRly phaSE clinical tRialS tRaininG couRSE Colbert/Montesquieu Sponsors: ASGCT, ESGCT, Généthon, Genosafe, AFM, Florida Biologix, Jennerex, Molmed, ReGenX Welcome
08.35
i: planning for an early phase clinical trial – Sponsor: Genosafe
10.30
Morning break
10.50
2: preclinical development – Sponsor: Genosafe
12.25
Lunch
13.25
3: manufacturing considerations – Sponsor: AFM/Généthon
15.20
Afternoon break
15.40
4: clinical trial compliance, monitoring and oversight – Sponsor: Genosafe
16.55
5: case studies in cross-atlantic rare disease trials – Sponsor: AFM
18.10
Adjourn
Sponsor: Université Paris Descartes 9.00
Gene transfer strategies
10.30
Morning break
10.45
controlling vector tropism, transgene expression or vector integration
12.15
Lunch
13.30
Gene therapy and immune responses
15.00
cell therapy
16.15
Afternoon break
16.45
Regulatory aspects of clinical trials in cell or gene therapy
17.30
Adjourn
ESGct and SftcG conGRESS opEninG cEREmony
8
18.30
opening symposium – Auditorium Richelieu
20.00
Welcome drinks – Mazarin Room
2_Prog_glance.indd 8
P. 40
Education day – Auditorium Richelieu
P. 38
08.30
Page 36
thursdAy 25 october
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
friDaY 26 OCtOber
1: major concepts for cell and gene therapy Auditorium Richelieu Sponsor: Cellectis
10.00
Morning break
10.30
1a: Gene and 1b: Cancer cell therapy for Auditorium Pid Richelieu Conde/Pascal Sponsor: CELL-PID
12.30
sftCg general assembly – Auditorium Richelieu
12.30
lunch and poster session 1* (P001-P148) – Gallerie Le Nôtre
14.30
2: Stem cells and regenerative medicine Auditorium Richelieu
16.00
afternoon break
16.30
2a: Overcoming genotoxicity Auditorium Richelieu Sponsor: PERSIST
18.30
Poster party 1 (P001-P148) – Gallerie Le Nôtre
19.00
speakers’ dinner – by invitation only
2b: immune response, immunoregulations, genetic vaccines Conde/Pascal
1c: neuromuscular Colbert/ Montesquieu Sponsor: AFM
2c: adult cell reprogramming Lulli Sponsor: Orgenesis
1d: regional talents Lulli Sponsor: IDF, Stempole
Page 42
08.30
2d: late breaking abstracts and diversified platforms of cell and gene therapy Colbert/ Montesquieu Sponsor: Lysogene
P. 48
registration open
Page 48
08.00
Page 42
main COnFErEnCE
Please note posters P001-P148 will only be displayed on Friday
*
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PLEASE NoTE: At 2am tonight the clocks will turn back one hour for the end of daylight Saving Time
ProGrAMME AT A GLANCE SATurdAy 27 oCTobEr 2012
3: current status of cell and gene therapy applications in man Auditorium Richelieu Sponsor: Généthon
10.30
Morning break
11.00
3a: cardiovascular Auditorium Richelieu Sponsor: ATMI
13.00
ESGCT General Assembly – Auditorium Richelieu
13.00
Lunch and poster session 2* (P149-P298) – Gallerie Le Nôtre
14.30
4: immunology Auditorium Richelieu Sponsor: Sigma-Aldrich
16.00
Afternoon break
16.30
4a: epigenetics Conde/Pascal
18.45
Poster party 2 (P149-P298) – Gallerie Le Nôtre
20.45
Projection film (Gattaca)
4b: GmP and bioprocess development for GT vectors Lulli Sponsor: Généthon
3d: Joint symposium Japanese and european Societies; cancer and oncolytic viruses Colbert/ Montesquieu Sponsors: Jennerex, JSGT
Page 60
3c: Brain gene therapy Lulli Sponsors: Braincav, ELA
4c: Joint Symposium Japanese and european Societies 2; Genetic diseases Colbert/ Montesquieu Sponsor: JSGT
4d: Targeting vector entry and expression Auditorium Richelieu Sponsor: SPP1230
Page 60
3b: Gene targeting Conde/Pascal Sponsor: PERSIST
Page 54
08.30
Page 54
main conference
Please note posters P149-P298 will only be displayed on Saturday
*
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Will Genetics create the perfect world ?
> Come and watch "GATTACA" by Andrew Niccol with Ethan Hawke, Uma Thurman and Jude Law
In the main auditorium, Palais des Congrès de Versailles * Free access to all congress attendees
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Š Sony Pictures/Inserm-P.Minary
Saturday 27th October at 20h45 (after the poster party)
01/10/2012 15:16
PLEAsE notE: At 2am last night the clocks turned back one hour for the end of daylight saving time
ProGrAMME At A GLAncE sundAy 28 octobEr 2012 main conference 5: Vision for the future Auditorium Richelieu Sponsor: AFM
10.30
Morning break
11.00
5a: ethical and regulatory issues in cell and gene therapy Conde/Pascal Sponsor: PERSIST
13.00
Lunch
13.30
career Session: european research council Lulli
14.30
6: ageing Auditorium Richelieu
16.30
Afternoon break
17.00
6a: eye Auditorium Richelieu Sponsor: Genzyme
19.00
End of Programme
19.30
Gala dinner – see page 83
6b: Bioprocess of cell therapy products Lulli Sponsor: bluebirdbio
Page 66
5d: Haematology: to insulate or not to insulate Auditorium Richelieu Sponsor: Généthon
P. 70
5c: Liver and metabolism Colbert/ Montesquieu Sponsor: ReGenX
6c: imaging and in vivo Studies Conde/Pascal
6d: non-viral gene delivery Colbert/ Montesquieu
Page 70
5b: Biotherapies institute Lulli Sponsors: Biotherapies Institute, AFM
Page 66
08.30
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
Monday 29 oCtobEr
7: Vision for a healthy world Auditorium Richelieu Sponsor: Sangamo
10.30
Morning break
11.00
Presidential symposium Auditorium Richelieu Sponsor: Genzyme outstanding achievement award Sponsor: Human Gene Therapy
Page 78
08.30
Page 78
main conference
Young investigator awards Sponsors: Human Gene Therapy, SFTCG, Molmed 13.00
End of the Congress
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Partners PlaTinum ParTnErS The DiM bioTHÉraPiES (DiM for Domaine d’intérêt Majeur) is a network of about 170 research teams working in the field of gene and cell therapy, regenerative medicine, stem cells, and transplantation, in the région ile-de-France (Paris region).The DiM was created in 2008 at the instigation of the regional council of ile-de-France to provide financial and organisational support to regional academic research teams: • PhD fellowships
• Post-doctoral fellowships
• Scientific event organisation
• Purchase of laboratory equipment
• Set up of infrastructure for common platforms Our research programme’s focus: develop and define applications of stem cells, of gene and cell therapy, and of transplantation in human medicine. Coordinator: nathalie Cartier www.stempole-idf.com 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 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 16
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
GOld 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.fondation.parisdescartes.fr
Genzyme has pioneered the development and delivery of transformative therapies for over 30 years. Founded in 1981 in boston, Massachusetts, Genzyme evolved from a tiny start-up with just a handful of employees to one of the world’s leading biotech companies. acquired by Sanofi in 2011, Genzyme now benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Genzyme has long been known for our expertise in the class of rare genetic diseases known as lysosomal storage disorders (lSDs). lSDs remain the heart of our company today, but we have also expanded – through both in-house development and strategic acquisitions and partnerships – to other disease areas such as thyroid cancer and multiple sclerosis. Driven by our commitment to patients, we strive to develop strong relationships with patient communities and listen to their perspectives so that we can truly understand their needs. These collaborations guide us as we continue to push the boundaries of medicine and technology to develop new and better therapies where none existed before. www.genzyme.com 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 17
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SilVEr ParTnErS 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 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 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 third year. For more information, see www.braincav.eu or contact Dr. E J Kremer at institut de Génétique Moléculaire de Montpellier (eric.kremer@ igmm.cnrs.fr) or Dr. anton ottavi: +33 (0)4 72 13 89 82. www.braincav.eu The Cellectis Group is based on a highly specific Dna engineering technology. its application sectors are human health, agriculture and bio-energies. Co-created by andré Choulika, its Chief Executive officer, Cellectis is today one of the world leading companies in the field of genome engineering. The Group has a workforce of 230 employees working on five sites worldwide. aFM, Dupont, baSF, bayer, Total, limagrain and novo nordisk are some of the Group’s clients and partners. www.cellectis.com 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 clinical pioneers, scientists and industrial partners in the advanced therapies field and aims to develop broad clinical application of safe cell-based therapies. http://cordis.europa.eu
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
SilVEr ParTnErS 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 an association of motivated and informed parents and patients who share responsibilities within Ela and have united their efforts in order to fight leukodystrophies and myelin diseases by establishing and respecting the following clear objectives: • to help and support families affected by leukodystrophy • to stimulate the development of research • to raise public awareness • to develop its work at an international level Ela is thus a bridge between all forms of leukodystrophies and a family solidarity network. www.ela-asso.com 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 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
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SilVEr 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 lYSoGEnE’s responsibility is to develop treatments for the rare disease community. lYSoGEnE is a clinical stage pioneering biotechnology company engaged in providing once in a lifetime, safe and efficient innovative gene therapy treatments for patients affected with severe neurological diseases. With its first product into the clinic, aiming at treating a fatal pediatric rare disease (Sanfilippo Syndrome), lYSoGEnE aims at expanding its extraordinary potential to other CnS indications with high unmet medical needs. www.lysogene.com 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 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
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
SilVEr ParTnErS SaFC is a leading developer and manufacturer of raw materials, providing services that support the biopharmaceutical industry. Using unrivaled expertise, SaFC’s scientists work to resolve the bioprocessing obstacles that can occur at every stage – from preclinical to commercialisation. SaFC is known for safe, productive and consistent raw materials and services. The SaFC portfolio includes proficiency in developing and manufacturing upstream and downstream solutions, single-use packaging and scale up services. it offers the widest selection of liquid media available to the market, encompassing custom media, supplements/feeds, buffers, water and reagents. www.safcglobal.com Sangamo bioSciences, inc. is developing novel zinc finger Dna-binding proteins (ZFPs), for therapeutic gene regulation and genome editing. Sangamo has ongoing Phase 2 clinical trials to evaluate safety and efficacy of a ZFP Therapeutic® for the treatment of Hiv/aiDS. other therapeutic programs are focused on monogenic diseases. Sangamo engineers sequence-specific ZFP nucleases (ZFns) for gene modification and ZFP transcription factors (ZFP TFs) for gene regulation. The company has strategic partnerships with Shire, Dow agroSciences and Sigma-aldrich Corporation. www.sangamo.com a reliable, single-source research partner providing reagents, technologies and industry-leading service to the life science and high technology markets. our strengths lie in scientific knowledge, innovation, high-quality products and reliable delivery to support consistent, predictable results and save researchers time. The Company operates in 35 countries with customers in 160 countries. our objective is to provide excellent service worldwide.
We provide a range of global solutions for research and manufacturing customers across our brands: Sigma® life Science; aldrich® Chemistry; Fluka® analytical; Supelco® analytical; SaFC®. www.sigma-aldrich.com
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COnGrESS SuPPOrTErS Human Gene Therapy is the premier journal covering all aspects of human gene therapy, including Dna, rna, and cell therapies. HGT has now expanded into two parts to include HGT Methods, a bimonthly journal focused exclusively on protocols, new tools, lab techniques, and procedures. The unique package of Human Gene Therapy and HGT Methods provides 18 issues of essential research, technologies, translation, and applications to promote the development of gene therapy products into effective therapeutics for treating human disease. The Journal publishes original investigations into the transfer and expression of genes and improvements in vector development, delivery systems, and animal models, including cancer, aiDS, heart disease, genetic disease, and neurological disease. www.liebertpub.com/hum Celogos is a privately-held biotech company that engineers innovative products, devices and services in cellular & tissular therapies for regenerative medicine. Celogos aims to identify, develop, register and commercialise cell therapy products which fill therapeutic gaps for unmet medical needs. Celogos will commercialise its products through collaboration with its mother company, Hra Pharma (www.hra-pharma.com). Celogos has entered a partnership with rouen University Hospital and inSErM U905 to develop a cellular therapy for fecal incontinence. Contact: Christelle Doucet, PhD, r&D Director, c.doucet@celogos.fr. www.celogos.fr new to nPG: Molecular Therapy-Nucleic Acids online. open access. HighQuality research. Molecular Therapy-Nucleic Acids is a sibling journal to Molecular Therapy. The journal will publish high quality basic, translational, and clinical research and cutting edge reviews and commentaries targeted to the advances in oligonucleotide- and gene-based therapies. Molecular Therapy-Nucleic Acids is a peer-reviewed, fully open access, online journal. Sign up today for a Molecular Therapy-Nucleic Acids alert. www.nature.com/mtna iTMo iHP (aviESan) The institute for immunology, Hematology, Pneumology and biotherapies is one of the 10 thematic institutes of aviesan. it combines basic, translational and clinical research in several fields: immunology, Hematology, Pneumology, Hemostasis, Gene and Cell Therapies and Dermatology. it is in charge of providing information about the associated research teams and of the scientific coordination of the research in those fields. www.aviesan.fr
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COnGrESS SuPPOrTErS orgenesis is a development stage company with a novel therapeutic approach in the treatment of diabetes by correcting malfunctioning organs with new functional tissues created from the patient’s own existing organs. orgenesis employs a molecular and cellular approach directed at converting liver cells into functional insulin-producing cells as a treatment for diabetes. This new therapeutic approach is called autologous insulin Producing (aiP) cell transplantation. www.orgenesis.com 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 reGenX bioSciences is leading the effort to translate promising gene delivery applications into a pipeline of next generation personalised therapies for a range of severe diseases with serious unmet needs. our proprietary nav™ technology is based on nav vectors, the next generation of recombinant adeno-associated virus (raav) vectors, including nav raav8, nav raav9, and nav raavrh.10. We believe that the nav technology represents the potential promise of curing the root cause of disease rather than the symptoms, and we are committed to establishing best in class standards for our nav vectors. www.regenxbio.com Founded in 1964, the French national institute of Health and Medical research (inSErM) is a public scientific and technological institute which operates under the joint authority of the French Ministry of Health and French Ministry of research. The institute’s Mission is to understand and improve human health. as the only French public research institute to focus entirely on human health, in 2008 inSErM took on the responsibility for the strategic, scientific and operational coordination of biomedical research. www.inserm.fr With its nine training and research departments (UFr) and its institute of Technology (iUT), Paris Descartes University encompasses all the fields of knowledge of human and health sciences. it is the only university of the ile-de-France region to offer medical, pharmaceutical and odontological studies; its health department is renowned in Europe and in the whole world for the high quality of its training and the excellence of its research. www.univ-paris5.fr 23
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European Leukodystrophies Association (ELA) Founded in France in 1992, the European Leukodystrophies Association (ELA) is an association of motivated and wellinformed patients and parents who have united their efforts to fight leukodystrophies, the myelin genetic disorders. Its objectives are clear: - to help and support patients and their families - to fund research and inform health professionals - to raise public awareness - to develop its action at an international level So far, ELA funded research and patient support for 33 and 7 million Euros respectively. Since 2000, ELA helped the creation of branches in Switzerland, Belgium, Luxembourg, Spain and Italy.
ELA - 2 rue Mi-les-Vignes - 54521 Laxou Cedex - France 00 33 3 83 33 48 59 - ela@ela-fondation.com www.ela-asso.com/en
The biggest innovation in cell culture production is also the smallest. Introducing the compact iCELLis® disposable bioreactor. The iCELLis® 500 is a scalable and easy-to-use disposable bioreactor for virus production that achieves and maintains high cell densities. The iCELLis system combines advantages of single-use technologies with the benefits of a fixed-bed system. By providing up to 500m2 – equivalent to 3,000 Roller Bottles of 1,700cm2 each – iCELLis is designed to intensify and simplify anchoragedependent cell processes. To arrange an iCELLis trial with bench-top iCELLis nano, please contact us via info@atmi-lifesciences.com. The iCELLis system – the world’s first single-use, high-cell-density bioreactor © 2012 ATMI, Inc. All rights reserved. ATMI, the ATMI logo and iCELLis are trademarks or registered trademarks of Advanced Technology Materials, Inc. in the U.S., other countries, or both.
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CY TOK IN
CY TOK INE S
UPERFAMILY • STEM CELL PR ORS • TNF S ODUCT TH FACT S GROW • S E IN K O • AN M E H C TIBOD KITS • IES • AN IMAL-FREE PRODUCTS • ELISA
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exhibitOrs
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exhibitOrs booth 1: GenoSafe: see page 19 www.genosafe.org booth 2: GE Healthcare life Sciences provides tools for drug discovery, biopharmaceutical manufacturing and cellular technologies, so research scientists and specialists around the world can be more productive, effective and creative. our vision is to be the start-to-finish bioprocessing solution provider, the partner of choice in cell and protein research, and the leader in life sciences services. building on our broad expertise across life sciences, we are firmly committed to help researchers around the world discover new ways to predict, diagnose and treat disease. www.gelifesciences.com booth 3: Sigma-aldrich: see page 21 www.sigma-aldrich.com booth 3: SaFC: see page 21 www.safcglobal.com booth 4: CellGenix is an international leading manufacturer and supplier of high quality cytokines and serum-free medium for the ex vivo cell culture of DC, T-Cells, nK-Cells, Hematopoietic Stem Cells, MSC, Chondrocytes, ESC and iPS. CellGenix products are used world wide in clinical trials in academia, commercial trials, production of vaccine and in translation, validation and testing or assay development by biotechnology partners. The manufacturing is in accordance with GMP guidelines and USP. www.cellgenix.com booth 5: PeproTech: see page 20 www.peprotechec.com
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exhibitOrs booth 6: With a background of 30 years, Quality assistance holds a unique position on the market for the development and validation of analytical methods. Thanks to our scientific expertise and six laboratories on one site (Mass Spectrometry, Physico-chemistry, bioanalysis, Cell Culture, Molecular biology and Microbiology), we assess your analytical development over the course of the cell expansion process for product characterisation, raw materials analysis, stability studies and bioanalysis. our multi-disciplinary team, combined with the highest quality standards, ensures the best management of your projects, including troubleshooting and respect of timelines. it makes sense – and it is cost effective – to concentrate all analytical expertise on one site. www.quality-assistance.com booth 7: The vector Core at the University of north Carolina, Chapel Hill (UnC vector Core) operates as a full-service viral vector production organisation, producing nearly 1000 lots per year. We have extensive experience in vector design, process development, as well as manufacturing of research and clinical grade vectors and we utilise our unique expertise to ensure your project is on time and on budget. our goal is to deliver the highest quality vectors to our academic, government, foundation, and biotech industry clients. We also offer process development, assay development, and other support services to accelerate the research and development process. http://genetherapy.unc.edu/jvl.htm booth 8: biooutsource: see page 18 www.biooutsource.com
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exhibitOrs booth 9: ark Therapeutics’ viral manufacturing services operations provide process development and manufacturing for clients at a variety of stages of therapeutic development in ark’s “State of art” flexible facility. our scientists have developed in-house technologies ranging from vectors to platform processes which therefore can be applied to client projects to reduce costs and time for viral products. The capability includes: • Three self-contained manufacturing suites, all able to accommodate products requiring bSl-2 • Process development, cGMP manufacture of bulk drug substance, fill and finish and release testing • access to ark’s unique platforms in adenoviral and lentiviral vector production www.arktherapeutics.com booth 10: aTMi: see page 18 www.atmi-lifesciences.com booth 11: based in Evry on the Genopole, Texcell is a world-renowned service company that provides viral safety, mycoplasm tests and immunology services in full compliance with GlP and GMP standards. With its French and international presence, TEXCEll has business dealings with india, Korea and Japan. They also have a majority share in a similar company in the USa at the end of 2010. This company is now called TEXCEll n.a. (north america). www.texcell.fr
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exhibitOrs booth 12: Cayla/invivoGen provides ultra-pure antibiotics made entirely without animal components, novel anti-mycoplasma treatments, and the largest collection of Prr agonists. our collection of innovative blue™ reporter Cells is the result of the intensive study of the innate immune system combined with our vector design capabilities and cell culture experience. We offer novel pFUSE plasmids and a unique set of vaccine adjuvants including our preclinical vacciGrade™ Prr ligands. invivoGen remains the only company devoted to marketing CpG-free plasmid Dna to facilitate a better understanding of how CpGs affect gene expression, which we believe is important for the future of gene therapy. www.invivogen.com booth 13: 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 14: The Center for Cellular and Molecular Therapeutics is dedicated to the understanding, development and application of gene and related cell and nucleic acid therapies and the promotion of professional and public education in the field. The Center fosters a multidisciplinary approach among researchers and scientists working to discover new gene and cell therapies in search of cures for debilitating and life threatening childhood disorders. based on its core mission, the Center established a state-of-the-art cGMP clinical vector manufacturing suite for both adeno associated viral vectors and lenti viral vectors. www.research.chop.edu/programs/ccmt
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exhibitOrs booth 15: life Technologies is a global biotechnology tools company dedicated to improving the human condition. our systems, consumables and services enable researchers to accelerate scientific exploration, driving discoveries and developments that make life better. life Technologies customers work across the biological spectrum, to advance personalised medicine, regenerative science, molecular diagnostics, agricultural and environmental research and 21st century forensics. life Technologies was created by the combination of invitrogen Corporation and applied biosystems inc. www.lifetechnologies.com booth 16: The institut Clinique de la Souris (iCS) is an infrastructure of excellence in translational research and functional genomics. Genetic research is conducted and supported combining generation and validation of mutant mouse models and comprehensive phenotypic analysis (physiological-behavioral-anatomical). iCS provides scientists with a broad set of highly specialised mouse services. iCS is a reference center of animal models for human pathologies. it contributes to better understanding and generation of novel therapeutics. www.ics-mci.fr booth 17: Cobra biologics is a leading international clinical and commercial manufacturer of biologics and pharmaceuticals with three GMP approved facilities. We offer a broad range of integrated and stand-alone development services, stretching from cell line development through to the commercial supply of investigational medicinal product. We take pride in manufacturing excellence and being a trusted provider, delivering what we promise and helping our customers to develop drugs for the benefit of patients. Cobra biologics provides manufacturing solutions to the biologics and pharmaceutical industry. www.cobrabio.com booth 18: PlasmidFactory: see page 20 www.PlasmidFactory.com
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exhibitOrs booth 19: lonza offers world class technology platforms in the areas of GMP cell culture and viral-based therapeutic manufacturing, custom biotherapeutic culture media, a large selection of primary and stem cells and a full line of custom bioassays. our extensive experience in cell therapy process optimisation and scale-up innovation helps clients to safely and effectively advance their products through all phases of the commercial pipeline and maximise their return on investment. our new viral-based Therapeutics group provides viral vaccine manufacturing as well as viral vector mediated gene therapies. our staff can design, develop, and implement a manufacturing process that meets your autologous or allogeneic therapeutic applications. www.lonza.com booth 20: Charles river: see page 19 www.criver.com booth 21: Miltenyi biotec “From bench to bedside� Miltenyi biotec is Germany‘s largest independent, privately owned biotech company. Since pioneering MaCS magnetic cell separation technology in 1990, we have grown into a vibrant, multinational team of more than 1200 biomedical scientists, physicians, engineers, and support groups. We develop and manufacture a portfolio of outstanding products ranging from unique cell labeling reagents, through sophisticated cell separation and analysis devices, to innovative systems for clinical applications. From research tools to GMP reagents for sophisticated applications, such as cellular therapy, the creativity of our interdisciplinary teams is reflected in the excellence of our products. www.miltenyibiotec.com booth 22: 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 cells to grow into complex, sophisticated 3-D models in vitro. www.cellon.lu
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exhibitors booth 23: Diamond Pharma Services is a leading technical and scientific consulting group with an emphasis on the following areas: • Regulatory affairs: product concept to registration andbeyond • Product development: nonclinical, CMC and clinical aspects • Pharmacovigilance: clinical trial, post-marketing and QPPV services • Compliance: GLP, GMP, GCP and QP services. Our specialised team of more than 27 professionals includes those with a broad and varied industry experience and former regulators, offering both breadth and depth of expertise, from which our clients can benefit. Specifically, we have significant experience with regards to Advanced Therapy Medicinal Products. www.diamondpharmaservices.com booth 24: In 2010, Merck acquired Millipore. This U.S. life science company was founded in 1954 and rose to become one of the globally leading providers of filter technology, bioprocessing and bioanalytical services. With this acquisition, Merck is now a leading life science company. The Merck Millipore division offers products for life science research such as assays, biomarkers and target solutions, as well as bioprocessing, lab water purification and filtration. Additionally, the division supplies speciality chemicals, for example the pharmaceutical, cosmetics and food industries. www.millipore.com booth 25: Since 1998, Laboratoires Genévrier has been strongly involved in cell therapy, with the creation of a Biotechnology centre in Sophia-Antipolis, France. Biotherapies, and more particularly cell therapy, are innovative medical treatments targeting unmet medical needs to improve millions of patients’ lifespans and quality of life. Autologous cell therapy is a personalised and physiological type of treatment able to reproduce tissue reconstruction and cell interactions. Laboratoires Genévrier develops innovative solutions of cell therapy products in their fields of expertise: fertility, dermatology, rheumatology and traumatology. www.laboratoires-genevrier.com
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ReseaRch PRioRity PRogRam 1230 (sPP1230) MECHanISMS oF GEnE VECToR EnTRy anD PERSISTEnCE
Increasing numbers of gene therapy studies have demonstrated therapeutic efficacy, in particular for the treatment of rare monogenetic diseases. However, the broader use of gene therapy 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 the 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 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 connectivity of this multidisciplinary research consortium are ensured by extensive internal and external networking, and through organising and supporting national and international congresses.. For further information on the Research Priority Program SPP1230 visit www.schwerpunktprogramm1230.de
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edit any gene at any position in any genome Custom TALEN™ and exclusive support for all your gene editing projects only available at Cellectis bioresearch With over twelve years expertise in genome customization Cellectis bioresearch is the dedicated service provider in Custom TALEN™ design, production, validation and comprehensive project support.
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Dosage d’endotoxines en 15 minutes C’est bien le temps qu’il vous faudra maintenant pour la réalisation de vos tests LAL avec la sécurité d’une méthode conforme pharmacopée et validée FDA Nous sommes tellement convaincus des bénéfices que le PTS et le MCS apporteront à votre laboratoire que nous vous invitons à en tester un gratuitement. www.stopwastingtimeandmoney.net
frendo@eu.crl.com 00.800.15.78.97.43
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Programme thursday 25 october 2012 ASGCT/ESGCT EArly PhASE CliniCAl TriAlS TrAininG CourSE Colbert/Montesquieu aim: clinical gene transfer and cell therapy studies are becoming increasing complex due to ongoing developments in the field itself, together with the growing number of international and trans-atlantic clinical trials. this course will provide an education forum for clinicians and scientists to expand their knowledge about the clinical trial process in countries across europe as well as trans-atlantic manufacturing considerations. speakers will include representatives from regulatory bodies in germany, Italy, France, and the united Kingdom. Participants include basic sciences and translational researchers, clinical investigators, physicians, postdoctoral fellows, graduate students, government employees, and regulators. organising committee: alessandro aiuti robin ali barrie carter helen heslop Sponsors
BIOSCIENCES
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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Programme thursday 25 october 2012 Education day Auditorium Richelieu 09.00
Gene transfer strategies Chair: Alberto Epstein INV001 adenovirus Florian Kreppel, Ulm University INV002 gene trans fer us ing retrovirus -derived vectors : current technology and applications Cecilia Frecha, International Agency for Research on Cancer, Lyon INV003 cancer virotherapy, the oncolytic virus approach to treat tumours Alberto Epstein, University of Lyon INV004 Non viral gene delivery techniques Pascal Bigey, Université Paris Descartes
10.30
morning break
10.45
controlling vector tropism, transgene expression or vector integration Chair: Christof von Kalle INV005 controlling vector tropis m by trans ductional targeting Els Verhoeyen, Université de Lyon INV006 exploiting microrNa regulation for genetic engineering Bernhard Gentner, San Raffaele Telethon Institute for Gene Therapy, Milan INV007 retroviral and lentiviral integration s ites s tudy Christof von Kalle, German Cancer Research Center, Heidelberg INV008 clonality s tudies in gene therapy Christopher Baum, Hannover Medical School
12.15
Lunch – Mazarin Room
13.30
Gene therapy and immune responses Chair: Hildegard Büning INV013 Weal and woe of anti-aaV immune res pons es Hildegard Büning, University of Cologne INV014 Immune res pons es to recombinant adenovirus Karim Benihoud, University Paris-Sud, Orsay INV14a retrovirus Anne Galy, Généthon, Evry
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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Programme thursday 25 october 2012 Education day Auditorium Richelieu 15.00
cell therapy Chair: Christian Jorgensen inv009 methods for iPs cell generation and quality control assessment Olivier Féraud, Ingestem Consortium, Villejuif inv010 application of induced Pluripotent stem cells in hematological genetic diseases José C. Segovia, CIEMAT-CIBERER, Madrid inv011 adiPoa project: adipose derived stroma cells for osteoarthritis Christian Jorgensen, University of Montpellier inv012 treg based cell therapy David Klatzmann, Pitié Salpêtrière Hospital, Paris
16.15
afternoon break
16.45
Regulatory aspects of clinical trials in cell or gene therapy Chair: Nicolas Ferry inv015 safety testing of high titre adenovirus Daniel Galbraith, BioOutsource, Glasgow inv016 dealing with regulatory authorities Nicolas Ferry, ANSM, Saint Denis, Paris
17.45
adjourn
ESGct and SftcG conGRESS opEninG cEREmony Auditorium Richelieu 18.30
opening symposium Chairs: Nathalie Cartier and Seppo Ylä-Herttuala Key note speakers: Jules Hoffman, National Center of Scientific Research in Strasbourg; Nobel Prize, Medicine, 2011 Jean Claude Ameisen, Université Paris Diderot
20.00
Welcome drinks – Mazarin Room
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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Programme friday 26 october 2012 main conference 08.00
registration open
08.30
1: major concepts for cell and gene therapy
Auditorium Richelieu
Chair: Luigi Naldini iNV017 the clonal repertoire of gene modified cells in humans Christof von Kalle, German Cancer Research Center, Heidelberg iNV018 In vivo genome editing as an approach to the treatment of genetic disease Katherine A. High, University of Pennsylvania, Philadelphia iNV019 targeted gene correction in a mouse disease model Toni Cathomen, University Medical Center, Freiburg
10.00
morning break
10.30
Parallel sessions 1a, 1b, 1c, 1d
Conde/Pascal
1a: Gene and cell therapy for PiD Chairs: Alessandro Aiuti, Donald Kohn iNV020 gene therapy for primary immunodeficiencies Alessandro Aiuti, HSR-TIGET, Milan iNV021 gene therapy for ada-deficient severe combined immune deficiency Donald Kohn, University of California, Los Angeles iNV022 development of gene therapy for HLH due to perforin deficiency and for XLP1 Bobby Gaspar, University College London Proffered papers or001 Lentiviral vector mediated gene therapy for the treatment of Wiskott-aldrich Syndrome Samantha Scaramuzza, San Raffaele Telethon Institute for Gene Therapy, Milan or002 correction of the X-cgd phenotype by a self-inactivating alpharetroviral vector in human and murine models Kerstin B. Kaufmann, Georg-Speyer-Haus, Frankfurt or003 gamma-retroviral gene therapy for X-cgd: differential outcome of single mdS1-evi1 integration vs double mdS1-evi1/Stat3 integration Janine Reichenbach, University Children’s Hospital, Zßrich
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or004 efficient generation of gene-corrected and reprogramming factorfree iPS cells from Scid-X1 patients Angelo Lombardo, San Raffaele Telethon Institute for Gene Therapy, Milan
02/10/2012 11:25
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe friDaY 26 OCtOber 2012 Or005 high-level clustering of integration sites in proto-oncogenes and leukemogenesis in gamma-retroviral Was gene therapy Anna Paruzynski, German Cancer Research Center, Heidelberg Auditorium Richelieu
1b: Cancer Chairs: Pierre Cordelier, Ian McNeish inV023 P hase 1 gene therapy trial for pancreatic cancer: from bench to bedside Louis Buscail, Hôpital de Rangueil, Toulouse inV024 O ncolytic Vaccinia induces programmed necrotic cell death in ovarian cancer Iain McNeish, Queen Mary University of London inV025 a novel rnai-based treatment for pancreatic cancer (PC): preclinical & clinical results Eithan Galun, Hadassah Hebrew University Hospital, Jerusalem Proffered papers Or006 safety and efficacy of repeated infusions of CelYVir in children with metastatic neuroblastoma Javier Garcia-Castro, Instituto de Salud “Carlos III”, Madrid Or007 lentiviral vector-based insertional mutagenesis identifies new liver cancer genes and molecular networks that have a pivotal role in human hepatocarcinogenesis Marco Ranzani, San Raffaele Telethon Institute for Gene Therapy, Milan sPP1230 award “Mechanisms of vector-host interactions” Or008 redirection of th17 cells with an iCOs-based Car enhances function, antitumor activity and persistence of th17 cells Sonia Guedan, University of Pennsylvania, Philadelphia Or009 lymfactintM (lx-1101): an adenovirally-delivered Vegf-C gene therapy in combination with lymph node transfer for the treatment of patients with secondary lymphoedema associated with breast cancer Alan Boyd, Laurantis Pharma, Turku, Finland
Colbert/ Montesquieu
1c: neuro-muscular Chairs: Thomas Voit, George Dickson inV026 gene therapy for Duchenne Muscular Dystrophy: microdystrophins, oligonucleotides and endonucleases George Dickson, Royal Holloway University of London
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe friDaY 26 OCtOber 2012 inV027 rna-modulating therapeutics for Duchenne Muscular Dystrophy Judith van Deutekom, Prosensa Therapeutics, Leiden inV028 gene therapy for spinal muscular atrophy: perspectives and problems Thomas Voit, Institut de Myologie, Paris Proffered papers Or010 M onitoring by serum mirna of a gene transfer treatment in a g-sarcoglycanopathy mouse model David Israeli, Généthon, Evry Or011 r aaV9-mediated gene transfer in the spinal cord of a feline model of motor neuron degeneration Thomas Bucher, Université de Nantes Or012 human mesoangioblasts can elicit alloreactive t-cell immune responses: implications for allogeneic cell therapy of DMD Maddalena Noviello, San Raffaele Scientific Institute, Milan Or013 aaV-mediated gene replacement therapy for x-linked myotubular myopathy Anna Buj-Bello, Généthon, Evry Lulli
1d: regional talents Chair: Nathalie Cartier introduction by the Chair inV029 M ajor concepts for cell and gene therapy Jude Samulski, University of North Carolina, Chapel Hill inV030 M eganucleases for genome engineering Philippe Duchateau, Cellectis Therapeutics, Paris Proffered papers Or014 sustained stimulation/expansion of regulatory t cells treat autoimmune disease without impairing effector immune responses to infection, vaccination and cancer Guillaume Churlaud, Pitié Salpêtrière Hospital, Paris Or015 a aV gene therapy for alzheimer disease: consequences of aaVmediated cholesterol 24-hydroxylase overexpression in thY-taU22 mouse model Marie-Anne Burlot, Université Paris Descartes Or016 exon skipping gene therapy for Dystrophic epidermolysis bullosa Sandrina Turczynski, Université Paris Descartes
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe friDaY 26 OCtOber 2012 Or017 sMn rescue by using oligonucleotides of tricyclo-Dna to induce exon 7 inclusion in sMn2 mrna Valérie Robin, Institut de Myologie, Paris 12.30
sftCg general assembly – Auditorium Richelieu
12.30
lunch – Mazarin Room; Poster session 1 (P001-P148) – Gallerie Le Nôtre
14.30
2: Stem cells and regenerative medicine
Auditorium Richelieu
Chair: Marc Peschanski inV031 human embryonic stem cells and iPs for regenerative medicine: two real prospects Marc Peschanski, I-Stem, Evry inV032 M esenchymal stromal cells: a new paradigm for cellular immune modulation Willem Fibbe, LUMC, Leiden inV033 skeletal muscle stem cells in regenerative biology Shahragim Tajbakhsh, Institut Pasteur, Paris
16.00
afternoon break
16.30
Parallel Sessions 2a, 2b, 2c, 2d
Auditorium Richelieu
2a: Overcoming genotoxicity Chairs: Christopher Baum, Eugenio Montini inV034 engineering mammalian cells for therapeutic applications Martin Fussenegger, Swiss Federal Institute of Technology, Basel inV035 the future of the retros Christopher Baum, Hannover Medical School inV036 integration site analysis in a clinical trial of lentiviral vector based hematopoietic stem cell gene therapy for Metachromatic leukodystrophy Eugenio Montini, San Raffaele Telethon Institute for Gene Therapy, Milan Proffered papers Or018 lentiviral vectors designed for liver-directed gene therapy do not display detectable genotoxicity in sensitive in vivo assays Marco Ranzani, San Raffaele Telethon Institute for Gene Therapy, Milan Or019 high-throughput monitoring of bone marrow clonality in pre-clinical and clinical gene therapy studies Stephanie Laufs, National Center for Tumor Diseases, Heidelberg Or020 tracking t-memory stem cells in humans by retroviral tagging Serena Scala, HSR-TIGET, Milan
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe friDaY 26 OCtOber 2012 Or021 identification of a domain responsible for the dosedependent antiproliferative and apoptotic effects of Sleeping Beauty transposase Melanie Galla, Hannover Medical School sPP1230 award “Mechanisms of vector-host interactions” Conde/Pascal
2b: immune response, immuno-regulations, genetic vaccines Chairs: Olivier Boyer, Gilles Marodon inV037 regulating dendritic cell signalling to improve vaccination Mary Collins, University College London inV038 nK cells and innate immunity Eric Vivier, Centre d’Immunologie de Marseille-Luminy (CIML) inV039 C himpanzee ad vector technology platform for genetic vaccine applications Stefano Colloca, Okairos, Pomezia Proffered papers Or022 engineering lentiviral vectors to target dendritic cells: the nanobody display technology Cleo Goyvaerts, Vrije Universiteit Brussels Or023 gene transfer in naive-derived memory stem t-cells: a novel promising platform for cancer immunotherapy Nicoletta Cieri, San Raffaele Scientific Institute, Milan Or024 I n vitro and in vivo modulation of aaV capsid CD8+ t cell responses with igg-derived MhC class ii epitopes Federico Mingozzi, Children’s Hospital of Philadelphia, Pennsylvania Or025 incorporation of antigens into viral capsids augments efficacy of adeno-associated Viral (aaV) vector-based vaccines Angela Nowag, University Hospital Cologne
Lulli
2c: adult cell reprogramming Chairs: Sarah Ferber, Hidde Haisma inV040 transdifferentiation: autologous cell replacement therapy Sarah Ferber, Sheba Medical Centre, Tel Hashomer inV041 reprogramming through the pluripotente state as a new paradigm for human cell rejuvenation Jean Marc Lemaitre, Institute of Fuctional Genomics, Montpellier inV042 D irect generation of functional dopaminergic neurons from human fibroblasts Vania Broccoli, San Raffaele Scientific Institute, Milan
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe friDaY 26 OCtOber 2012 Proffered papers Or026 i PsC-based gene therapy of congenital erythropoietic porphyria Aurélie Bedel, Université Bordeaux Segalen Or027 impaired epithelial differentiation of induced pluripotent stem cells from ectodermal Dysplasia eeC patients is rescued by aPr-246/ PriMa-1Met Daniel Aberdam, Université Paris Descartes Or028 generation of disease-free iPs cells from fanconi anemia mice with a hypomorphic mutation in Brca2. Victoria Moleiro, CIEMAT/CIBERER, Madrid Or029 M itotic correction of trisomy 21 by disomic cell segregation occurred in human pluripotent stem cells from Down syndrome Lucie Tosca, Université Paris-Sud, Villejuif Colbert/ Montesquieu
2d: late breaking abstracts and diversified platforms of cell and gene therapy Chairs: Amina Zinaï, Sophie Gomez Proffered papers Or030 intracerebral gene therapy for sanfilippo syndrome Amina Zinaï, Lysogene, Paris Or031 C ancer regression and neurologic toxicity following anti-Mage-a3 tCr gene therapy Richard A. Morgan, National Cancer Institute, Bethesda, MD Or032 self-inactivating lentiviral vectors for correction of rag1 severe Combined immunodeficiency Karin Pike-Overzet, Leiden University Or033 a prb-responsive, rgD-modified, and hyaluronidase-armed canine oncolytic adenovirus as a therapeutic veterinary agent and a model for human virotherapy Eduardo Laborda, Institut d’Investigacions Biomèdiques de Barcelona Or034 lMO2 perturbs human t cell development in vivo by two different mechanisms Anna-Sophia Wiekmeijer, Leiden University Or035 an alpharetroviral vector platform suitable for permanent and transient cell modifications Julia D. Suerth, Hannover Medical School
18.30
Poster party 1 (P001-P148) – Gallerie Le Nôtre
19.00
speakers’ dinner – by invitation only
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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Please nOte: at 2am tonight the clocks will turn back one hour for the end of Daylight saving time
PrOgraMMe satUrDaY 27 OCtOber 2012 08.30
3: Current status of cell and gene therapy applications in man
Auditorium Richelieu
Chair: Fulvio Mavilio inV043 1 3 years of experience with gene therapy of primary immuno deficiencies Alain Fischer, Hôpital Necker, Paris inV044 D evelopment of a gene therapy pipeline for inherited retinal dystrophies Robin Ali, University College London inV045 C art19 induces sustained functional Car t cell persistence and b cell aplasia Carl June, University of Pennsylvania, Philadelphia inV046 stable factor ix activity following aaV-mediated gene transfer in patients with severe hemophilia b. Amit Nathwani, University College London
10.30
Morning break
11.00
Parallel sessions 3a, 3b, 3c, 3d
Auditorium Richelieu
3a: Cardiovascular Chairs: Michel Puceat, Christian Buchholz inV047 C ell type specific gene delivery in the vascular system Christian Buchholz, Paul-Ehrlich-Institut, Langen inV048 genetically-targeted therapies for heart failure: steps to clinical practice Patrick Most, University Hospital Heidelberg inV049 regenerative medicine: transforming health care solutions André Terzic, Mayo Clinic, Rochester, MN Proffered papers Or036 aaV based gene therapy rescues the murine cardiac phenotype associated with friedreich ataxia Morgane Perdomini, Université de Strasbourg, Illkirch Or037 stimulation of transmural capillary endothelialization of smalldiameter synthetic vascular grafts through local overexpression of a novel recombinant Vegfr2-ligand Vegf-a109 Jarkko Hytonen, University of Eastern Finland, Kuopio Or038 P luripotent stem cells carrying a mutated lamina/C: a cell model of laminopathies for the search for new therapeutic targets. Anne-Claire Guénantin, Université Paris Descartes
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe satUrDaY 27 OCtOber 2012 Conde/Pascal
3b: Gene targeting Chairs: Chiara Bonini, Diana Schenkwein inV050 t-cell engineering for adoptive immunotherapy using tal-effector nucleases (talens) Andrew Scharenberg, Cellectis Therapeutics, Paris inV051 tCr gene editing to treat malignancies Chiara Bonini, San Raffaele Scientific Institute, Milan inV052 M odifying the hiV-1 integration machinery to yield safer vector integration Diana Schenkwein, University of Eastern Finland, Kuopio Proffered papers Or039 retargeting of lentivector integration by leDgf/p75 chimera functionally rescues a cell culture model for x-linked CgD Rik Gijsbers, KU Leuven Or040 D eveloping engineered zinc finger transcriptional repressors of huntingtin as a potential therapy for huntington’s disease Philip Gregory, Sangamo BioSciences, Richmond, CA Or041 C orrection of hemophilia b phenotype following Zfn mediated genome editing in adult mice Michael Holmes, Children’s Hospital of Philadelphia, Penns Or042 D e novo and in silico design of regulatory elements for targeted and robust tissue-specific expression in gene therapy Marinee Chuah, Free University of Brussels
Lulli
3c: Brain gene therapy Chairs: Nathalie Cartier, Alessandra Biffi inV053 C linical trial of hsC gene therapy for metachromatic leukodystrophy Alessandra Biffi, San Raffaele Scientic Institute, Milan inV054 hematopoitic stem cell gene therapy for x-linked adrenoleukodystrophy Nathalie Cartier, Université Paris Descartes inV055 gene therapy for Cns disorders Ron Crystal, Weill Medical College of Cornell University, New York inV056 therapy for schwannomas using aaV-P0-caspase and exosomemediated delivery of therapeutic rna/protein Xandra Breakefield, Massachusetts General Hospital, Boston, MA
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I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe satUrDaY 27 OCtOber 2012 Proffered papers Or043 M ir-155 silencing decreases neuroinflammation in aβ-stressed astrocytes and microglia cells Joana Guedes, University of Coimbra Or044 lentiviral delivery of human full-length wild-type tau protein mediates progressive spatio-temporal and cortico-cortical propagated neurofibrillary degeneration in rat brain Morvane Colin, Université de Lille Or045 W idespread vector distribution following intra-Csf delivery of aaV9 vectors in a large animal model Virginia Haurigot, CBATEG, Barcelona Colbert/ Montesquieu
3d: Joint symposium Japanese and European Societies; Cancer and oncolytic viruses Chairs: Len Seymour, Yasufumi Kaneda inV057 a non-replicating oncolytic viral particle as a novel therapeutic tool against cancers Yasufumi Kaneda, Osaka University, Suita inV058 C oload1 a group b oncolytic adenovirus: pre-clinical assessment of potency, safety and selectivity Kerry Fisher, University of Oxford inV059 P rostate cancer gene therapy in Japan Yasutomo Nasu, Okayama University Hospital inV060 targeted oncolytic and immunotherapeutic vaccinia Jx-594: clinical proof-of-concept for systemic efficacy David Kirn, Jennerex, San Francisco, California inV061 bioKnifetM, a uPa activity-dependent fusogenic sendai virus, as a new class of oncolytic bio-device to treat malignancies Yoshikazu Yonemitsu, Kyushu University, Fukuoka Proffered papers Or046 C ombining oncolysis with genetic immunornase delivery as innovative strategy for cancer therapy Ines Fernández-Ulibarri, DKFZ, Heidelberg Or047 O ncolytic adenovirus with temozolomide induces autophagy, immunogenic cell death and antitumor immune responses preclinically and in cancer patients Ilkka Liikanen, University of Helsinki
13.00
esgCt general assembly – Auditorium Richelieu
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I never said it was possible. I only said it was true. —Charles Richet
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lunch – Mazarin Room; Poster session 2 (P149-P298) – Gallerie Le Nôtre
14.30
4: immunology
Auditorium Richelieu
Chair: Naomi Taylor inV062 high-throughput identification of antigen-specific tCrs by tCr gene capture Ton Schumacher, The Netherlands Cancer Institute, Amsterdam inV063 tbC John Gribben, Barts Cancer Centre, London inV064 advancing hsC and t lymphocyte therapies by manipulating cell metabolism Naomi Taylor, IGMM, Montpellier
16.00
afternoon break
16.30
Parallel sessions 4a, 4b, 4c, 4d
Conde/Pascal
4a: Epigenetics Chairs: Seppo Ylä-Herttuala, Anna Salvetti inV065 C ancer epigenetics: new paradigms and drugs Frank Lyko, German Cancer Research Center, Heidelberg inV066 P olycomb complexes co-associate with a specific rna polymerase ii variant ines cells Ana Pombo, Imperial College School of Medicine, London inV067 epigenetic regulation of Vegf expression Seppo Ylä-Herttuala, University of Eastern Finland, Kuopio Proffered papers Or048 a ubiquitous chromatin opening element (UCOe) prevents transgene silencing in murine pluripotent cells and their differentiated progeny Mania Ackermann, Hannover Medical School Or049 D eciphering the molecular mechanisms of reprogramming using mirna screens Nils Pfaff, Hannover Medical School Or050 lentiviral transduction of CD34+ cells induces genome-wide epigenetic modifications Daniel Stockholm, Généthon, Evry
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I never said it was possible. I only said it was true. —Charles Richet
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4b: GmP and bioprocess development for GT vectors Chairs: Frédéric Revah, Manuel Carrondo inV068 P rocess development in single use to facilitate cgMP in gene therapy Manuel Carrondo, IBET, Oeiras inV069 large scale aaV production for clinical trials for the treatment of neuromuscular diseases Otto Merten, Généthon, Evry inV070 lentiviral vectors for in vivo clinical applications: current and future production strategies James Miskin, Oxford BioMedica Proffered papers Or051 P lasmid Dna for indirect clinical applications – how much gMP is necessary? Marco Schmeer, PlasmidFactory, Bielefeld Or052 D evelopment of a manufacturing process for an oncolytic vaccine Maria Fe C. Medina, McMaster University, Hamilton, Ontario Or053 inverted terminal repeat read-through and reverse packaging: mechanisms of occurence and strategies to minimise Dna impurities in recombinant aaV. John Fraser Wright, Children’s Hospital of Philadelphia, Penns
Colbert/ Montesquieu
4c: Joint Symposium Japanese and European Societies 2; Genetic diseases Chairs: Yoshikatsu Eto, Nicole Déglon inV071 applications of induced Pluripotent stem (iPs) cells for the pathogenesis of lyosomal storage Diseases (lsD) Yoshikatsu Eto, Tokyo Jikei University inV072 generation of disease-free fanconi anemia iPsC-derived hematopoietic progenitors with Zfn-mediated targeted addition of FANCA to the AAVS1 locus Paula Rio, CIEMAT/CIBERER, Madrid inV073 W hat we learnt from the gene therapy for aDa deficiency Masafumi Onodera, National Center for Child Health and Development, Tokyo inV074 toward the treatment of huntington’s disease with rnai Nicole Déglon, CEA, Institute of Biomedical Imaging (I2BM) and Molecular Imaging Research Center (MIRCen), Fontenay-aux-Roses
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I never said it was possible. I only said it was true. —Charles Richet
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Programme saturday 27 october 2012 Proffered papers or054 Lentiviral sgsH expression from the human cd11b promoter in transplanted haematopoietic stem cells fully corrects behaviour and neuropathology of mucopolysaccharidosis IIIa mice Alexander Langford-Smith, University of Manchester or055 aaV9 mediated gene therapy of mLd model mice Noriko Miyake, Nippon Medical School, Tokyo or056 Neonatal intracerebral gene delivery to correct cNs pathology in a mouse model of globoid cell Leukodystrophy Annalisa Lattanzi, San Raffaele Scientic Institute, Milan Auditorium Richelieu
4d: Targeting vector entry and expression Chairs: Hildegard Büning, Thierry VandenDriessche INV075 re-directing vector fate by capsid engineering Hildegard Büning, University of Cologne INV076 modulation of adenovirus vector - host interactions Florian Kreppel, Ulm University INV077 solving the t-cell problem: generation of immune stealth aaV Thierry VandenDriessche, Vrije Universiteit Brussels Proffered papers or057 a lentiviral vector pseudotyped with a baboon retrovirus envelope glycoprotein outperforms VsV-g-LVs for gene transfer into hematopoietic stem cells and lymphocytes Anaïs Girard Gagnepain, Université de Lyon or058 microrNa-150-regulated vectors for cell type-specific transgene expression in hematopoietic gene therapy Sebastian Brennig, Hannover Medical School or059 displaying high affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transfer Robert Münch, Paul-Ehrlich-Institut, Langen or060 regulated lentiviral vector gene transfer for X-cgd Valentina Capo, University of Rome
18.45
Poster party 2 (P149-P298) – Gallerie Le Nôtre
20.45
Projection film (gattaca) – Auditorium Richelieu
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nOtes
I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe sUnDaY 28 OCtOber 2012 08.30
5: Vision for the future
Auditorium Richelieu
Chair: David Klatzmann inV078 genetic engineering of human hematopoiesis for treating inherited diseases and cancer Luigi Naldini, San Raffaele Scientific Institute, Milan inV079 neural plasticity and neuronal diversity in the adult mammalian brain Fred H. Gage, The Salk Institute for Biological Studies, La Jolla, CA inV080 P ulp fictions David Klatzmann, Pitié Salpêtrière Hospital, Paris
10.30
Morning break
11.00
Parallel sessions 5a, 5b, 5c, 5d
Conde/Pascal
5a: Ethical and regulatory issues in cell and gene therapy Chairs: Odile Cohen-Haguenauer, Jude Samulski inV081 U ltra-rare orphan diseases and need for platform gene delivery Jude Samulski, University of North Carolina, Chapel Hill inV082 D evelopment of gene and cell therapy products: regulatory expectations Jean Hugues Trouvin, Université Paris Descartes inV083 O verlapping ethical issues in cell and gene therapies Nancy M. King, Wake Forest School of Medicine, Winston-Salem, NC Panel discussion
Lulli
5b: Biotherapies institute Chair: Marc Peschanski inV084 M ulticenter international gene therapy clinical trials for Wiskott-aldrich syndrome Anne Galy, Généthon, Evry inV085 an exon-skipping strategy using aaV8-U7snrna vectors for the treatment of Duchenne Muscular Dystrophy – results in grMD dogs and clinical perspectives Caroline Le Guiner, Université de Nantes inV086 human pluripotent stem cells for hD cell therapy: hope or hype? Anselme L. Perrier, I-Stem/AFM, Evry
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I never said it was possible. I only said it was true. —Charles Richet
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Programme sunday 28 october 2012 inv087 restoration of vision in the Pde6ß-deficient dog, a large animal model of rod-cone dystrophy Lolita Petit, Université de Nantes Colbert/ Montesquieu
5c: Liver and metabolism Chairs: Karen Kozarsky, Anne Dubart Kupperschmitt inv088 tbc Fatima Bosch, Universida Autònoma de Barcelona
BIOSCIENCES
inv089 Pancreatic beta cell development: experimental models to transfer data from rodent to human Raphael Scharfmann, Faculté Necker, Paris inv090 Helper-dependent adenoviral vectors for inborn errors of liver metabolism Nicola Brunetti-Pierri, Telethon Institute of Genetics and Medicine, Naples Proffered papers or061 redifferentiation of expanded human pancreatic β-cell-derived cells by inhibition of the notcH pathway Yael Bar, Tel Aviv University or062 Hepatic gene transfer of tFeb, a master autophagy regulator, results in clearance of mutant alpha-1-antitrypsin Nunzia Pastore, Telethon Institute of Genetics and Medicine, Naples or063 optimisation of liver gene transfer for hemophilia b Xavier M. Anguela, The Children’s Hospital of Philadelphia, Penns or064 integration-defective lentiviral vectors expressing engineered hyperactive Factor ix improve vector performance following hepatic transduction Nisha Nair, Free University of Brussels Auditorium Richelieu
5d: Haematology: to insulate or not to insulate Chairs: Salima Hacein-Bey, Karim Benihoud inv092 When insulation is not a solution: posttranscriptional deregulation of gene expression induced by vector integration Fulvio Mavilio, Généthon, Evry inv093 new chromatin insulators for gene therapy George Stamatoyannopoulos, University of Washington, Seattle inv091 tbc Philippe Leboulch, Brigham and Women’s Hospital, Harvard University, MA
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I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe sUnDaY 28 OCtOber 2012 Selected abstracts Or065 hla-universal platelets infusions prevent platelet refractoriness in a mouse model Christiane Gras, Hannover Medical School Or066 C D34+ cells mobilised by Plerixafor treatment in thalassemic patients are enriched in stem/progenitor cells endowed with high reconstitution potential and gene transfer susceptibility Maria Rosa Lidonnici, San Raffaele Telethon Institute for Gene Therapy, Milan Or067 targeting CD133+ cells to improve long-term gene marking in hematopoietic stem cell based gene therapies Christian Brendel, Georg-Speyer-Haus, Frankfurt Or068 P rotection of hematopoietic stem cells from stress-induced functional impairment by very low-dose interleukin-1 stimulation Makoto Otsu, University of Tokyo 13.00
lunch – Mazarin Room
13.30 Lulli
Career Session: European research Council funding Opportunities in 2013 Dr. Fiona Kernan, Scientific Department, ERC Executive Agency
14.30
6: ageing
Auditorium Richelieu
Chair: Nicolas Levy inV094 glial cell regulation of hematopoietic stem cell hibernation in the bone marrow niche Hiromitsu Nakauchi, University of Tokyo inV095 antisense based therapeutic approaches in hutchinson-gilford Progeria Nicolas Levy, Children’s Hospital La Timone, Marseille inV096 effect of ageing and muscle origin on human satellite cells Vincent Mouly, Institut de Myologie, Paris
16.30
afternoon break
17.00
Parallel sessions 6a, 6b, 6c, 6d
Auditorium Richelieu
6a: Eye Chairs: Robin Ali, Sam Wadsworth inV097 successful aaV-mediated gene therapy of rod-cone and cone-rod dystrophies in dogs: great promise for the treatment of rapid degenerative photoreceptor defects Fabienne Rolling, Université de Nantes
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I never said it was possible. I only said it was true. —Charles Richet
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Programme sunday 28 october 2012 inv098 optogenetic vision restoration strategy Jens Duebel, Institut de la Vision, Paris inv099 transplantation of es cell-derived photoreceptors Emma West, University College London Proffered papers or069 silencing HLa class i expression in human corneas to decrease the risk of graft rejection after keratoplasty Constanca Figueiredo, Hannover Medical school or070 use of a short hPde6b promoter sequence to drive efficient gene expression specifically in rods in a model of severe retinal dystrophy, the Rd10 mouse Yvan Arsenijevic, Jules-Gonin Eye Hospital, Unil, Lausanne or071 intravitreal delivery of aav-ndi1 for the treatment of Leber Hereditary optic neuropathy Naomi Chadderton, Trinity College Dublin or072 alterations of epithelial stem cell marker patterns in human diabetic corneas and effects of c-met gene therapy Graziella Pellegrini, University of Modena and Reggio Emilia, Modena Lulli
6b: Bioprocess of cell therapy products Chairs: Mitch Finer, Gabor Veres, Luc Douay inv100 In vitro generation of red blood cells: perspectives for transfusion medicine Luc Douay, Université Pierre et Marie Curie, Paris inv101 Large scale, high efficiency transduction of human cd34+ cells with lentiviral vectors for β-thalassemia and ccaLd clinical applications Gabor Veres, bluebirdbio, Cambridge, MA inv102 Progress towards the cgmP production of hesc derived rbcs Joanne Mountford, University of Glasgow/SNBTS Proffered papers or073 cd70 increases survival and avidity of cytotoxic t lymphocytes generated for adoptive cell immunotherapy strategies Mohamad Hamieh, University of Rouen Hospital or074 co-expression of a suicide gene in car t cells enables the safe targeting of cd44v6 for leukemia and myeloma eradication Monica Casucci, San Raffaele Hospital, Milan or075 clinical-grade human myoblasts for cell therapy: a chromosomal stability analysis Olivier Boyer, University of Rouen
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I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe sUnDaY 28 OCtOber 2012 Or076 integrated bioprocesses for scalable production, purification and cryopreservation of iPsC-derived cardiomyocytes Margarida Serra, Universidade Nova de Lisboa, Oeiras Conde/Pascal
6c: imaging and in vivo studies Chairs: Anne Galy, Serge Charpak inV103 the sodium iodide symporter (nis) and its role as a theranostic gene Christine Spitzweg, University of Munich inV104 imaging brain activity by monitoring Po2 transients in capillaries Serge Charpak, Université Paris Descartes inV105 M ultiparametric spinal Mri in degenerative motor neuron diseases Pierre-François Pradat, Pitié Salpêtrière Hospital, Paris Proffered papers Or077 P ositive molecular imaging of endogenous microrna regulation in pathophysiological conditions in mice Patrick Baril, Université d’Orléans Or078 imaging of mesenchymal stem cell recruitment into the stroma of hepatic colon cancer metastases using the sodium iodide symporter (nis) Kerstin Knoop, University Hospital of Munich Or079 Q uantitative nMr 1h imaging and 31P spectroscopy evaluation of locoregional high venous pressure raaV8-U7- ese6-ese8 exonskipping therapy in the grMD Jean-Laurent Thibaud, Ecole Nationale Vétérinaire d’Alfort, Paris Or080 C hanges in the progression of amyloid deposition in aPP/Ps mice after overexpression of different aPOe isoforms through intraventricular injection of an adeno-associated virus serotype 4 Eloise Hudry, Harvard Medical School, Mass
Colbert/ Montesquieu
6d: non-viral gene delivery Chairs: Daniel Scherman, Patrick Midoux inV106 new avenues in non viral gene delivery and therapy Daniel Scherman, Université Paris Descartes inV107 U pdate on the UK Cf gene therapy Consortium multi-dose, nonviral, gene therapy trial Eric Alton, Imperial College, London Proffered papers Or081 non viral gene transfer via Sleeping beauty transposon for Collagen Vii delivery in human primary keratinocytes Maria Carmela Latella, University of Modena and Reggio Emilia, Modena
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I never said it was possible. I only said it was true. —Charles Richet
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PrOgraMMe sUnDaY 28 OCtOber 2012 Or082 I n vivo mrna introduction to central nervous system using polyplex nanomicelles Keiji Itaka, University of Tokyo Or083 enhancement of Zorro-lna efficiency in huntingtin gene silencing via combination with bislna Eman M. Zaghloul, Karolinska Institutet, Stockholm Or084 non-invasive imaging of pulmonary gene transfer: from pretty images to product development Peggy Richard-Fiardo, Laboratoire TIRO, CEA, Nice Or085 I n vivo expression of nuclear triggered Pegylated polyacridine peptide polyplexes Kevin Rice, University of Iowa 19.00
end of Programme
19.30
gala dinner – meet in the lobby of the Palais des Congrès at 19.15, or in the Académie du Spectacle Equestre, Avenue Rockfeller, (opposite the Palais des Congrès) at 19.30
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I never said it was possible. I only said it was true. —Charles Richet
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Programme monday 29 october 2012 08.30
7: Vision for a healthy world
Auditorium Richelieu
Chair: Philip Gregory inv108 a holistic approach towards restoring vision in retinitis pigmentosa, between prosthetics, optogenetics and rehabilitation José-Alain Sahel, Institut de la Vision, Paris inv109 ZFn-edited cd4+ t cells for Hiv/aidS therapy: phase 1 trials of Sb728-t in Hiv-infected subjects Philip Gregory, Sangamo BioSciences, Richmond inv110 the role of partnerships in regenerative medicine and gene therapy – a pharma perspective Jason Gardner, GlaxoSmithKline, Philadelphia, Penn Proffered papers or087 ProSavin® a gene therapy approach for the treatment of Parkinson’s disease: a phase i clinical trial update Stéphane Palfi, CEA, Fontenay aux Roses or086 transcription activator-Like effector nucleases (taLens) for targeted inactivation of Hepatitis b virus cccdna Kristie Bloom, University of the Witwatersrand, Johannesburg
10.30
morning break
11.00
Presidential symposium
Auditorium Richelieu
Chairs: Seppo Ylä-Herttuala, Luigi Naldini new President’s welcome: Luigi naldini guest Speaker: odile cohen-Haguenauer, Hôpital St-Louis, Univ Paris-Diderot, Sorbonne Paris-Cité & UMR 8113, ENS de Cachan Outstanding Achievement Award addressing the bottlenecks in gene therapy through synthetic biology and de novo vector design Thierry VandenDriessche, Free University of Brussels
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I never said it was possible. I only said it was true. —Charles Richet
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Programme monday 29 october 2012 Young Investigator Awards restoration of vision by photoreceptor transplantation Dr Rachael Pearson, University College London assessing the Integration Profile of Lentiviral Vectors in gene therapy for X-adrenoleukodystrophy Dr Cynthia Bartholomae, German Cancer Research Center and National Center for Tumor Diseases, Heidelberg aaV8 gene delivery for organic acidemias: a promising therapy for two orphan diseases Dr Randy Chandler, National Institutes of Health Bethesda Maryland
13.00
end of the congress
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I never said it was possible. I only said it was true. —Charles Richet
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Congress soCial aCtivities Guided tour of the historic city of Versailles, passinG by the palace Duration 2h30. Times to be arranged for each group: meet with your guide at the entrance of the Palais des Congrès. the city of versailles was laid out as a setting for the Palace and is rich in buildings of great interest to visitors. the saint-louis quarter, built on the site of louis Xiii’s former deer park, is today one of the city’s historic focal points. laid out somewhat in the form of a chessboard around its magnificent cathedral, it is also remarkable for its “carrés”, small houses with typically Xviiith century mansard roofs, and for the royal Kitchen garden created by the garden designer de la Quintinie between 1678 and 1683. the notre-Dame quarter was a new town created at the instigation of louis Xiv in his desire to embellish the approaches to the Palace. visitors enjoy leisurely strolls here in the heart of the city, the streets lined with interesting shops and buildings such as the théâtre Montansier, the Church of notre-Dame, the statue in the emblematic Place Hoche, the Hôtel du Baillage in the antiques area and the market hall in the Place du Marché.
Guided tour of the palace of Versailles: the KinG’s and Queen’s apartments and the hall of mirrors Duration 90 minutes every lunch time, starting at 12h30. Meet with your guide at the entrance of the Palais des Congrès. enter the private residence of the sun King and discover the King’s apartments dedicated to the theme of the planets. enjoy sumptuous surroundings in which gold and marble form a harmonious decor. Walk the length of the Hall of Mirrors, now more dazzling than ever before. the recently restored glittering mirrors and candelabras reinforce the magical and prestigious spectacle of the long line of scintillating giltwork. Continue through the elegant Queen’s apartments and the bedchamber of Marie-antoinette. For more information on both tours please visit the Tourism Office Booth in the entrance hall of the Palais des Congrès.
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© GillES TarGaT
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
La Voie de l’écuyer Gala dinner—Sunday 28 October 2012—19.30
A
n unforgettable evening in the Royal Stables at the Château de Versailles. Choreographed by Bartabas, La Voie de l’Ecuyer evokes the Académie: learning and appropriating tradition and passing on expertise. Carrousel of the Lusitanians, sorraias with long reins, fencing on horseback and equestrian improvisations are performed to the sound of selected passages of music by Bach. The show is followed by drinks and a visit to the Royal Stables. Finished by Jules Hardouin Mansart in 1683 and restored in 2003, the
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stables are in perfect harmony with the architecture of the time. And finally, a delicious standing dinner in The Ring, a showcase of wood and mirrors, designed like an opera house and subtly lit by Murano glass chandeliers.You will just have experienced the show sat on the wooden tiers in this very same place and you are now having dinner on the golden sand where the horses were dancing less than one hour earlier. 50 Euros/person for tickets purchased on site www.acadequestre.fr
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traVel infOrMatiOn COnFErEnCE VEnuE: Palais des Congrès de Versailles 10 rue de la Chancellerie, 78000, Versailles tel: 01.40.68.22.22
• from Paris Saint lazare or Paris la défense train station: go to Versailles rive droite train station, then it is about a 15 minute walk to the Palais des Congrès.
By PlanE (WWW.adP.Fr ) • FrOm Orly: from Orly the best way to get to Versailles is by taxi, it is a 20 minute taxi ride and should cost around 40 to 50 euros.
• from Central Paris, rEr C line: go to Versailles rive Gauche train station, then it is about a 5 to 7 minute walk to the Palais des Congrès. (be careful, make sure you buy “Paris-Versailles rive gauche” ticket. t+ ticket is not valid for this journey).
• FrOm CharlES dE GaullE (rOiSSy) On ThE rEr (taxi would be very long and very expensive): the airport train station is a long walk from the terminals (could be as much as 15 minutes).
By BuS • the ratP no. 171 bus runs between the Pont de sèvres metro station and the Château de Versailles (Palais des Congrès just next to the Château).
hOW TO GET ThErE...
• buy a ticket to your final destination, Versailles rive gauche. (there are three different stations in Versailles, but Versailles rive gauche is around the corner from the Palais des Congres). • Catch rer b (direction saint remy les Chevreuse). • Change at saint Michel notre Dame (after about one hour), take rer ligne C (direction Versailles rive gauche). • from Versailles rive gauche it is a five minute walk. By Train www.transilien.com, www.ratp.fr • from Paris montparnasse train station: go to Versailles Chantier train station, then it is about a 10 minute walk to the Palais des Congrès.
By Taxi • richard taxi: +33 6 66 65 10 39 http://www.taxi-versailles.com • list of all taxis in Versailles: http://www.taxis-paris.fr/versailles.htm By Car • from Paris, autoroute a13 towards Versailles/saint Quentin-en-Yvelines/rouen to exit no. 5 Versailles centre/Versailles Château. head toward Versailles, then take avenue des etats-Unis, and, finally, avenue de saint Cloud. Versailles can also be reached by: a86, and n10. • Parking: there are many parking lots available in all key areas of the city: next to the Palais des Congrès, avenue de sceaux, and on the Place d’armes
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
Car parking near the palais des Congres Parking Place d’Armes
Parking Saint-Cloud
place du Château – F – 78000 Versailles distance from palais des Congrès: 200m tel: +33 (0)1 39 53 74 60 number of spaces: 640 open seven days per week
33 bis, avenue de st-Cloud – F – 78000 Versailles distance from palais des Congrès: 700m tel: +33 (0)1 30 21 75 00 number of spaces: 581 open seven days per week
Parking Europe 66 avenue de l’europe – F – 78000 Versailles distance from palais des Congrès: 450m tel: +33 (0)1 39 50 22 91 number of spaces: 100
Parking Marché Notre-Dame halles de Versailles – F – 78000 Versailles distance from palais des Congrès: 800m tel: +33 (0)1 39 50 22 91 number of spaces: 577 open 24 hours per day, seven days per week
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restaUrants near the Palais Des COngres restaurant
Telephone
a la Coiffe bretonne – 10 avenue du général de gaulle
01-30-21-78-22
au Chapeau gris – 7 rue hoche
01-39-50-10-81
au Chien qui fume – 72 rue de la Paroisse
01-39-53-14-56
au fou du roy – 6 rue de la chancellerie
01-30-21-80-81
au Petit Marquis – 6 rue andré Chenier
01-39-50-47-40
brasserie du théâtre – 15 rue des réservoirs
01-39-50-03-21
buffalo grill – 7 avenue de sceaux
01-30-21-32-03
Café de la Place d’armes – 1 avenue de saint Cloud
01-39-50-03-60
Chez tiouiche-au bon accueil – 4 rue st Julien
01-30-21-56-66
la flottille – parc du château
01-39-51-41-58
la Marée de Versailles – 22 rue au Pain
01-30-21-73-73
la taverne de Maître Kanter – 5 rue Colbert
01-39-50-02-50
le limousin – 1 rue de satory
01-39-50-21-50
le londres – 7 rue Colbert
01-39-50-05-79
le resto du roy – 1 avenue de st Cloud
01-39-50-42-26
le Valmont – 20 rue au Pain
01-39-51-39-00
lebanon – 11 rue de satory
01-30-21-23-13
les Manèges – 2bis avenue de Paris
01-39-07-46-46
les trois Marches – 1 boulevard de la reine
01-30-84-52-00
Pizza evi – 15 rue des etats généraux
01-39-50-16-53
restaurant l’entrecote – 18 bis rue neuve notre Dame
01-39-53-09-53
sister’s Café – 15 rue des réservoirs
01-30-21-21-22
M
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PRIX GALIEN 2012
GENE THERAPY FOR RARE DISEASES Advancing Therapies From Research to Patient Treatment
Created in 1990 by AFM-Telethon and funded through the donations to the French annual Telethon, Genethon’s mission is to design gene therapy products for rare diseases, to ensure their pre-clinical and clinical development as well as their production in order to provide patients with these innovative treatments.
FOR MORE INFORMATION and details:
www.genethon.fr
Š C.Hargoues - L.Audinet J.P.Pouteau - fotolia
Member of
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FlyerOr
eUrOPean sOCietY fOr gene anD Cell theraPY aChieVeMent aWarDs Outstanding achievement award: in collaboration with human gene therapy and Mary ann liebert Publishers, esgCt presents one award for an established researcher who has made a long-term, outstanding contribution to the field. €2000 honorarium and 30 minute presentation during the annual congress. young investigator awards: €1000 and a 15 minute presentation during the annual congress for up to four researchers who are showing exceptional promise. 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 details are available at www.esgct.eu/awards note: eligibility criteria applies
esgCt eValUatiOn We do hope you have enjoyed the esgCt/sftCg Collaborative Congress 2012. 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.
ED
For y In pan yH gen yR sta yH me yH cel yA cur yN an yH sta yP y Im yB yC
y Et y In
https://www.surveymonkey.com/s/ESGCT2012 a link is also available from the esgCt website.
OR
if you enjoyed this meeting, would you consider hosting/organising an esgCt or sftCg congress in 2015? if so, please forward your proposal to the esgCt board as soon as possible at office@esgct.eu, or the sftCg board at office@sftcg.fr
Firs
E-M
Stre
thank you in advance for your time.
City
ESGCT and SFTCG Team
Qua
Qua
n
Car
Exp
n 17,
To r
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FlyerOrderA5_Mise en page 1 24/09/12 19:20 Page1
The CliniBook
Clinical Gene transfer: state-of-the-art
EC-FP6
EDITED BY ODILE COHEN-HAGUENAUER, MD-PHD, ON BEHALF OF THE CLINIGENE NETWORK OF EXCELLENCE FOR THE ADVANCEMENT OF CLINICAL GENE TRANSFER AND THERAPY, EC-FP6, LSHB-CT-2006-018933 Foreword y Introduction including prospect for a pan-european infrastructure for GT vectors y Highlights on AAV mediated gene transfer y Retrovirus mediated gene transfer state-of-the-art y Highlights on lentivirus mediated gene transfer y Highlights on gene-modified cell therapy y Adenovirus mediated gene transfer: current developments y Non-viral based gene transfer: a new era y Highlights on iPS induction, genetic stability and emerging technologies y Preclinical assessment tools y Immune responses and Immunotoxicity y Biosafety platform state-of-the-art y Clinical trials y Ethical and regulatory issues y Integration and dissemination issues
L. Luzzatto & I. Verma ➤ O. Cohen-Haguenauer ➤ F. Bosch with R. Ali, A. Auricchio, E. Ayuso, V. Blouin, G.Dickson,
M. Giacca, O. Merten, P. Moullier
➤ P. Cruz & M. Carrondo with C. Baum, C. Buchholz, K. Cichutek,
H. Hauser, A. Schambach
➤ K. Cichutek & M. Schweizer with P. Alves, O. Cohen-Haguenauer,
F.L. Cosset, N. Déglon, E. Verhoeyen, D. Wirth
➤ G. Gahrton with E. Alici, O. Cohen-Haguenauer, P. Cruz,
W. Lindermaier
D.Klatzmann,
➤ S. Kochanek with P. Alves, M. Anton, M. Carrondo, B. Gänsbacher,
S. Ylä-Herttuala
➤ G. Dickson with C. Baum, J. Bode, Z. Itzvak, Z. Ivics, C. Marie,
M. Schleef, M. Schmeer, D. Sherman
➤ O. Cohen-Haguenauer with E. Bayart, T. Cathomen, M. Chuah,
F. Pâques, J. Smith, T. Vandendriessche
➤ A. Panet & A. Jacobs with C. von Kalle, C. Richard, M. Schmidt ➤ D. Klatzmann with B. Bellier, A. Galy, F. Mingozzi ➤ C. von Kalle with A. Aiuti, C. Baum, E. Montini, M. Schmidt ➤ B. Gänsbacher with R. Ali, A. Aiuti, N. Cartier, M. Eriksdotter-Jönhagen, A. Nathwani, S. Palfi, S. Ylä-Herttuala ➤ O. Cohen-Haguenauer, A. Kent, N. King ➤ R. Draghia-Akli with G. Dickson, B. Gänsbacher, A. Kent, D. Klatzmann, L. Naldini, T. Vandendriessche and the CliniGene Coordinator
ORDER FORM – Public price: 95 € (125 $) – Special price at ESGCT meeting up until November 30th: 80 € (105 $) First name - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - Last name and title - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - E-Mail - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - Street address and building - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - City - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - Zip Code
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Date
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The Premier Journal on Gene Therapy with Exclusive Coverage of Methods
Human Gene Therapy Methods including DNA, RNA, and Cell Therapies
www.liebertpub.com/hgtb
Editor-in-Chief: James M. Wilson, MD, PhD www.liebertpub.com/hum
Methods Editor: Thierry VandenDriessche, PhD
The Official Journal of:
European Society of Gene and Cell Therapy
www.liebertpub.com/hum
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Safety
and
e fficacy
of biotherapeutic productS
From research to clinical trials, we meet our client’s specific needs by performing custom studies in strict compliance with regulatory requirements • Development and validation of specific assays and analytical methods • Custom samples analysis Molecular assays • Biodistribution and shedding studies • Custom qPCR and RT-qPCR assays Viral vectors • Titration of viral particles and infectious genomes • Detection of recombinant particles • Detection of cell or plasmid-specific sequences Immunology
a ddreSS GenoSafe 1 rue de l’internationale BP 40064 91002 Evry Cedex France www.genosafe.com S hareholderS Genethon (60%) AFM (40%) Founded 2003
Characterization of: • Inflammatory responses • Humoral responses • Cell-mediated responses Cell lines • Customized cell line engineering • Stem cell characterization Clinical trials • Patient Follow-up
contact Vincent Zuliani Phone: +33(0)1.69.47.11.57 Fax: +33(0)1.69.47.11.61 contact@genosafe.com
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VIRAL SAFETY & IMMUNOLOGY GMP / GLP Services
• Characterization of cells
• Central Lab
• Safety Testing
• Biomarkers
• Biodistribution Studies
• Immunogenicity
• Viral Clearance
• Potency / Impurities
• Customized Cell Culture
• Protein expression
To get further information
Stand n°11
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2013 SFTCG AnnuAl ConGreSS 2013: PAriS
unique opportunities for young scientists to: • shape the programme • chair sessions • present data
Sthae dveate!
22 March 2013
Faculté de médecine de l’université Paris-Sud, Hôpital du Kremlin-Bicêtre
www.sftcg.fr
UsefUl nUMbers local police: vehicle removal ______________________________________ 01 39 50 25 48 Poison control centre ____________________________________________ 01 40 05 48 48 Police station __________________________________________________ 01 39 24 70 00 Car pound _____________________________________________________ 01 39 53 08 99 gendarmerie ___________________________________________________ 01 39 67 50 00 Duty pharmacies => police station _________________________________ 01 39 24 70 00 Phébus – urban transport ________________________________________ 01 39 20 16 20 fire service ______________________________________________________________ 18 emergency services _______________________________________________________ 17 emergency medical service __________________________________ 15 or 01 30 84 96 00 snCf (national rail operator) ______________________________________ 0 892 35 35 35 Yvelines emergency doctors’ service ________________________________ 01 39 58 58 58
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COnGress OffiCe infOrMatiOn Contact names
Payment queries
Gaëlle Jamar – event Manager Vanessa sampson – Payment & Membership
Please go to the registration desk during the above opening hours
Registration desk opening hours
Membership queries
thursday 25 October 08.00-20.00 friday 26 October 07.45-18.30 saturday 27 October 08.00-13.00 sunday 28 October 08.30-19.00 Monday 29 October 08.30-13.30
Contact number in case of emergency
Please go to the registration desk during the above opening hours Gaëlle Jamar, event Manager +44 7766 475379
Wifi is aVailaBle in the Palais Des COnGres Wifi naMe: sePCV lOGin: esGCt2012 PassWOrD: esGCt2012
Cover image © Mairie de Versailles Gala dinner images © 2012 Bartabas Other images © Mairie de Versailles, unless otherwise indicated Design: www.catherinecharnock.co.uk
to landfill
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ŇŹŇŠŇŤŇ
European Society of Gene and Cell Therapy French Society for Cell and Gene Therapy Collaborative Congress 2012
ESGCT 21st Anniversary Congress in collaboration with the SETGYC
European Society of Gene and Cell Therapy and Spanish Society of Cell and Gene Therapy will hold a joint meeting in Madrid
ESGCT and SFTCG Congress 2012 • Programme and Information Book
24-28 October 2013
25-29 October 2012 | Palais des Congrès de Versailles
www.esgct.eu • www.setgyc.es www.esgct.eu • www.sftcg.fr FR FRYHUVBLPSRVHG LQGG RYH HUVBLPSRVHG LQGG LQGG