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ESGCT 21st Anniversary Congressin collaboration with the SETGYC
www.esgct.eu • www.setgyc.es
24-28 October 2013
European Society of Gene and Cell Therapy and
Spanish Society of Cell and Gene Therapy
will hold a joint meeting in Madrid
ESGC
T and SFTCG Congress 2012 • Program
me and Inform
ation Book
25-29 October 2012 | Palais des Congrès de Versailles
www.esgct.eu • www.sftcg.fr
Collaborative Congress 2012
European Society of Gene and Cell TherapyFrench Society for Cell and Gene Therapy
Cover image © Mairie de Versailles Gala dinner images © 2012 BartabasOther images © Mairie de Versailles, unless otherwise indicated
Design: www.catherinecharnock.co.uk
to landfill
COnGress OffiCe infOrMatiOn
Contact namesGaëlle Jamar – event Manager Vanessa sampson – Payment & Membership
Registration desk opening hoursthursday 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
Payment queriesPlease go to the registration desk during the above opening hours
Membership queriesPlease go to the registration desk during the above opening hours
Contact number in case of emergencyGaëlle Jamar, event Manager +44 7766 475379
Wifi is aVailaBle in the Palais Des COnGres
Wifi naMe: sePCVlOGin: esGCt2012PassWOrD: esGCt2012
covers_imposed2.indd 2 04/10/2012 12:54
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
1
PlaTinum ParTnErS
GOld ParTnErS
SilVEr ParTnErS
COnGrESS SuPPOrTErS
Partners–OVerVieW
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2
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
1_Frontmatter.indd 2 02/10/2012 12:47
3
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
3
ESGCT BoardseppoYlä-herttuala,Presidentluiginaldini,Vice-PresidentChristofvonKalle,treasurerlenseymour,generalsecretaryrobinaliChristopherbaumChiarabonininathalieCartiersarahferberhiddehaisma
SFTCG Board and local Organising CommitteenathalieCartier,PrésidentanneDubartKupperschmitt,generalsecretaryannegaly,treasurerPatrickMidoux,Vice-treasurersophiegomez,WebsiteKarimbenihoudPierreCordelieralbertoepsteinsophielebel-binaygillesMarodonMichelPucéatannasalvetti
Presidential CommitteenathalieCartier,ChairOlivierboyerOdileCohen-haguenauerannegalysalimahacein-beyDavidKlatzmannPhilippeMoullierMarcPeschanskynaomitaylor
Organisers:Wats.onConsultancy:renéeWatson,gaëlleJamar,Vanessasampson
With special thanks toalberttasteyre,DiMstempolebiotherapieMaryDean,asgCtfreddybecher,localexpertadvisorstéphanieYeunès,inserMPatrickMinari,inserM
bOarDs
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4
WelCOMeaDDress
OnbehalfoftheeuropeansocietyofgeneandCelltherapyandthelocalorganisingcommittee,itismypleasuretowelcomeyoutoVersaillesfortheannualCongress2012,jointlyorganisedwiththefrenchsocietyforCellandgenetherapy.this2012editionisofparticularimportance,aswecelebratethe20thanniversaryoftheeuropeansociety.
Withincreasingnumbersofclinicalsuccessesandpromisingnewresearchleads,geneandcelltherapyisarapidlygrowingfieldineuropeandworldwide.forthisexceptional2012edition,wehaveworkedtoofferyouanexcitingprogramme,inviting120prestigiousspeakersandinternationallyacclaimedspecialistsinthefieldsofcellandgenetherapy,immunotherapy,stemcellsandregenerativemedicine.87Oralpresentationsandalmost300posterswillbepresentedtoofferyouthelatestdatafromaroundtheworldandtoenableinternationalscientificinteractions.byholdingtheCongressinasmall,focussedarea,wehopetocreateafriendlyandinteractivecommunity,allowingallmemberstodiscussadvancesincellandgenetherapy,majorconcepts,andourvisionforthefutureofahealthyworld.
Versaillesistheidealsettingtocometogetherandshareinanimportantmomentinfrance’srichhistory.thePalaisdesCongrèsdeVersaillesisresplendantinmarbleandoak,withfrescoesbyChapelain-Midy,andbas-reliefsbyCollarmarini.theChâteaudeVersailles,aUnesCOworldheritagesite,isamongthemostbeautifulachievementsof18thcenturyfrenchart.YouwilldiscoverVersaillesthroughpedestrianwalkways,visitstothepalaceanditsgardens,andthefabulousbartabasequestrianartsacademy.
iwishtowarmlythankyouforyourparticipationandtothankourpartners,sponsorsandexhibitorsfortheirmajorcontributions.thanksalsotothewholeteamthathasassistedwiththeCongresspreparationandmanagement,withoutwhosecommitment,energy,enthusiasmandgenerositythiscongresswouldneverhavebeenpossible.
nathalieCartier
PresidentofsftCgPresidentoftheesgCt2012OrganisingCommittee
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5
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
Cardiovascular diseasesandybaker(Chair)seppoYlä-herttuala(Chair)MaurogiaccaMosheflugelmanKeithChannonPatrickMost
neurological and muscular diseasesnicoleDéglon(Chair)Mauriliosampaolesi(Chair)nathalieCartier(Chair)nicholasMazarakisDominicWellsandersbjörklund
Viral vectorshildegardbüning(Chair)luiginaldini(Chair)robhoebenakselihemminkiannasalvettialbertoepsteinelsVerhoeyen
Genetic and metabolic diseasesedvardsmith(Chair)thierryVandenDriessche(Chair)fatimaboschManuelgrezrobinalibeatthony
non-viral vectorsDanielscherman(Chair)hiddehaisma(Chair)ernstWagnerJ.P.behrgeorgeDicksonZoltanivics
infection, immune and vaccinesDavidKlatzmann(Chair)MaryCollins(Chair)naomitaylorbenberkhoutDorotheeVonlaerZeligeshhar
Stem cells and reprogrammingstefanKarlsson(Chair)Christopherbaum(Chair)JuanbuerentimO’brienCatherineVerfaillieKatarinaleblancMarinaradrizzaniWillemfibbe
Ethics and regulatory affairsOdileCohen-haguenauer(Chair)KlausCichutek(Chair)richardashcroftalastairKentgöstagahrtonsergebraunOttoMertenMartinschleefKlausKühlckeMichaelfuchs
sCientifiCCOMMittees
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Bureaux organisateurs
HALL D'ACCUEILlobby
MAZARIN
RACINE MOLIÈRE
Sanitaires / Restrooms
SERVICES
Salles / Rooms
Entrées / Entrance
ACCES / ACCESS
Ascenseur / Lift
Escalier / Stair
0Niveau Level
Monte-charge / Goods lift
TRAITEUR
entréeentrance
Parkings / Car parks
Bus
RER
Vestiaires / Cloakroom
Privé
Niveau
-1
LES ESPACES /MEETING SPACE
300 m²
720 m²
Office TraiteurCaterers’ service areaTRAITEUR
LULLI350 m²
BAR SALON72 m²
privéprivate
SERVICES
ACCES / ACCESS
Ascenseur / Lift
Escalier / Stair
Salles / Rooms
Entrées / Entrance
-1Niveau Level
Monte-charge / Goods lift
TRAITEUR
Sanitaires / Restrooms
LES ESPACES / MEETING SPACE
Office TraiteurCaterers’ service areaTRAITEUR
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BOILEAU50 m²
MONTESQUIEU50 m²
Galerie LE NÔTRE280 m²
COLBERT130 m²
Balcon amphithéâtreAuditorium balcony
RICHELIEU900 places
Ba
300 places
Sanitaires / Restrooms
SERVICES
Salles / Rooms
Entrées / Entrance
ACCES / ACCESS
Ascenseur / Lift
Escalier / Stair
2Niveau Level
LES ESPACES / MEETING SPACE
VAUBAN50 m²
PASCAL50 m²
FOYER CONDÉ100 m²
SCÈNEstage
AMPHITHÉÂTREauditoriumRICHELIEU
1 200 places
SCÈNE
Sanitaires / Restrooms
SERVICES
Salles / Rooms
Entrées / Entrance
ACCES / ACCESS
Ascenseur / Lift
Escalier / Stair
1Niveau Level
LES ESPACES / MEETING SPACE
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8 8
Education day – Auditorium RichelieuSponsor: Université Paris Descartes
P. 38
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
ProgrAMMe At A gLAnce
Please note: speakers and talk titles may change
thursdAy 25 october
ESGct and SftcG conGRESS opEninG cEREmony
P. 40
18.30 opening symposium – Auditorium Richelieu
20.00 Welcome drinks – Mazarin Room
aSGct/ESGct EaRly phaSE clinical tRialS tRaininG couRSE Colbert/MontesquieuSponsors: ASGCT, ESGCT, Généthon, Genosafe, AFM, Florida Biologix, Jennerex, Molmed, ReGenX Pa
ge 3
6
08.30 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
2_Prog_glance.indd 8 02/10/2012 11:17
9
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
9
main COnFErEnCE08.00 registrationopen
08.30 1: major concepts for cell and gene therapyAuditorium RichelieuSponsor: Cellectis Pa
ge4
2
10.00 Morningbreak
10.30 1a: Gene and cell therapy for PidConde/PascalSponsor: CELL-PID
1b: CancerAuditorium Richelieu
1c: neuro-muscularColbert/MontesquieuSponsor: AFM
1d: regional talentsLulliSponsor: IDF, Stempole
Page
42
12.30 sftCggeneralassembly–Auditorium Richelieu
12.30 lunchandpostersession1*(P001-P148) – Gallerie Le Nôtre
14.30 2: Stem cells and regenerative medicineAuditorium Richelieu P.
48
16.00 afternoonbreak
16.30 2a: Overcoming genotoxicityAuditorium RichelieuSponsor: PERSIST
2b: immune response, immuno-regulations, genetic vaccinesConde/Pascal
2c: adult cell reprogrammingLulliSponsor: Orgenesis
2d: late breaking abstracts and diversified platforms of cell and gene therapyColbert/MontesquieuSponsor: Lysogene
Page
48
18.30 Posterparty1(P001-P148) – Gallerie Le Nôtre
19.00 speakers’dinner– by invitation only
*Please note posters P001-P148 will only be displayed on Friday
friDaY26OCtOber
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10 10
main conference
08.30 3: current status of cell and gene therapy applications in manAuditorium RichelieuSponsor: Généthon Pa
ge 5
4
10.30 Morning break
11.00 3a: cardiovascularAuditorium RichelieuSponsor: ATMI
3b: Gene targetingConde/PascalSponsor: PERSIST
3c: Brain gene therapyLulliSponsors: Braincav, ELA
3d: Joint symposium Japanese and european Societies; cancer and oncolytic virusesColbert/MontesquieuSponsors: Jennerex, JSGT
Page
54
13.00 ESGCT General Assembly – Auditorium Richelieu
13.00 Lunch and poster session 2* (P149-P298) – Gallerie Le Nôtre
14.30 4: immunologyAuditorium RichelieuSponsor: Sigma-Aldrich Pa
ge 6
0
16.00 Afternoon break
16.30 4a: epigeneticsConde/Pascal
4b: GmP and bioprocess development for GT vectorsLulliSponsor: Généthon
4c: Joint Symposium Japanese and european Societies 2; Genetic diseasesColbert/MontesquieuSponsor: JSGT
4d: Targeting vector entry and expressionAuditorium RichelieuSponsor: SPP1230 Pa
ge 6
0
18.45 Poster party 2 (P149-P298) – Gallerie Le Nôtre
20.45 Projection film (Gattaca)
*Please note posters P149-P298 will only be displayed on Saturday
SATurdAy 27 oCTobEr 2012
PLEASE NoTE: At 2am tonight the clocks will turn back one hour for the end of daylight Saving Time
ProGrAMME AT A GLANCE
> Come and watch "GATTACA" by Andrew Niccol with Ethan Hawke,
Uma Thurman and Jude Law
Saturday 27th Octoberat 20h45 (after the poster party)
In the main auditorium, Palais des Congrès de Versailles
* Free access to all congress attendees
© S
ony
Pic
ture
s/In
serm
-P.M
inar
y
Will Genetics createthe perfect world ?
2_Prog_glance.indd 10 02/10/2012 12:55
> Come and watch "GATTACA" by Andrew Niccol with Ethan Hawke,
Uma Thurman and Jude Law
Saturday 27th Octoberat 20h45 (after the poster party)
In the main auditorium, Palais des Congrès de Versailles
* Free access to all congress attendees
© S
ony
Pic
ture
s/In
serm
-P.M
inar
y
Will Genetics createthe perfect world ?
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12 12
main conference08.30 5: Vision for the future
Auditorium RichelieuSponsor: AFM Pa
ge 6
6
10.30 Morning break
11.00 5a: ethical and regulatory issues in cell and gene therapyConde/PascalSponsor: PERSIST
5b: Biotherapies instituteLulliSponsors: Biotherapies Institute, AFM
5c: Liver and metabolismColbert/MontesquieuSponsor: ReGenX
5d: Haematology: to insulate or not to insulateAuditorium RichelieuSponsor: Généthon
Page
66
13.00 Lunch
13.30 career Session: european research councilLulli
14.30 6: ageingAuditorium Richelieu P.
70
16.30 Afternoon break
17.00 6a: eyeAuditorium RichelieuSponsor: Genzyme
6b: Bioprocess of cell therapy productsLulliSponsor: bluebirdbio
6c: imaging and in vivo StudiesConde/Pascal
6d: non-viral gene deliveryColbert/Montesquieu
Page
70
19.00 End of Programme
19.30 Gala dinner – see page 83
ProGrAMME At A GLAncEsundAy 28 octobEr 2012
PLEAsE notE: At 2am last night the clocks turned back one hour for the end of daylight saving time
2_Prog_glance.indd 12 02/10/2012 12:57
13
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
13
main conference08.30 7: Vision for a healthy world
Auditorium RichelieuSponsor: Sangamo Pa
ge 7
8
10.30 Morning break
11.00 Presidential symposiumAuditorium RichelieuSponsor: Genzyme
outstanding achievement awardSponsor: Human Gene Therapy
Young investigator awardsSponsors: Human Gene Therapy, SFTCG, Molmed
Page
78
13.00 End of the Congress
Monday 29 oCtobEr
2_Prog_glance.indd 13 01/10/2012 15:33
BioOutsource Ltd • 1 Technology Terrace • Todd Campus • West of Scotland Science Park • Glasgow • G20 0XAtel: +44 (0)141 946 4222 • email: [email protected] • web: www.biooutsource.com
Biosafety Testing and Characterisation
• Cell Banks
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• Replication Competent Viral Vectors
Pre-clinical and Clinical Trials Support
• Biodistribution Studies
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www.biooutsource.com
Your partner for release testing Gene Therapy Products
• GLP, GMP and GXP compliant testing
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Knockout any human, mouse or rat gene.Accelerate your research and create permanent gene knockouts with CompoZr® Knockout Zinc Finger Nucleases from Sigma® Life Science.
Bioedit.Save time on developing genome editing tools and generate the knockout you need with CompoZr Knockout ZFNs, a proven and functionally validated technology.
Create knockout cell lines or animal models with confidencesigma.com/knockout
©2012 Sigma-Aldrich Co. LLC. All rights reserved. SIGMA and SIGMA-ALDRICH are trademarks of Sigma-Aldrich Co. LLC, registered in the US and other countries. CompoZr is a registered trademark of Sigma-Aldrich Co. LLC. Where bio begins is a trademark of Sigma-Aldrich Co. LLC. iPad is a trademark of Apple, Inc. registered in the US and other countries.
Visit us at Booth #3 for a chance
to win an Ipad.
sh5419_BioEdit_A5.indd 1 9/17/2012 8:55:20 AMVersailles_2012_book.indb 14 01/10/2012 15:16
Knockout any human, mouse or rat gene.Accelerate your research and create permanent gene knockouts with CompoZr® Knockout Zinc Finger Nucleases from Sigma® Life Science.
Bioedit.Save time on developing genome editing tools and generate the knockout you need with CompoZr Knockout ZFNs, a proven and functionally validated technology.
Create knockout cell lines or animal models with confidencesigma.com/knockout
©2012 Sigma-Aldrich Co. LLC. All rights reserved. SIGMA and SIGMA-ALDRICH are trademarks of Sigma-Aldrich Co. LLC, registered in the US and other countries. CompoZr is a registered trademark of Sigma-Aldrich Co. LLC. Where bio begins is a trademark of Sigma-Aldrich Co. LLC. iPad is a trademark of Apple, Inc. registered in the US and other countries.
Visit us at Booth #3 for a chance
to win an Ipad.
sh5419_BioEdit_A5.indd 1 9/17/2012 8:55:20 AMVersailles_2012_book.indb 15 01/10/2012 15:16
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
Ourresearchprogramme’sfocus: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
PartnersPlaTinum ParTnErS
16
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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
GOld ParTnErS
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
17
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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 ([email protected]) 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
SilVEr ParTnErS
18
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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
SilVEr ParTnErS
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
19
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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
SilVEr ParTnErS
20
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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
SilVEr ParTnErS
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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, [email protected].
www.celogos.fr
new to nPG: Molecular Therapy-Nucleic Acids online. open access. High- Quality 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
COnGrESS SuPPOrTErS
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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
COnGrESS SuPPOrTErS
ESGCT/SFTCG CollaboraTivE ConGrESS 2012
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The biggest innovation in cell cultureproduction 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 anchorage-dependent cell processes. To arrange an iCELLis trial with bench-top iCELLis nano, please contact us via [email protected].
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.
European Leukodystrophies Association (ELA)Founded in France in 1992, the European Leukodystrophies Association (ELA) is an association of motivated and well-informed 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 - France00 33 3 83 33 48 59 - [email protected]/en
CYTOKINES • ANTIBODIES • ANIMAL-FREE PRODUCTS • ELISA KITS • CHEMOKINES • GROWTH FACTORS • TNF SUPERFAMILY • STEM CELL PRODUCTS
Versailles_2012_book.indb 24 01/10/2012 15:17
The biggest innovation in cell cultureproduction 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 anchorage-dependent cell processes. To arrange an iCELLis trial with bench-top iCELLis nano, please contact us via [email protected].
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.
CYTOKINES • ANTIBODIES • ANIMAL-FREE PRODUCTS • ELISA KITS • CHEMOKINES • GROWTH FACTORS • TNF SUPERFAMILY • STEM CELL PRODUCTS
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exhibitOrs
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booth1:GenoSafe: see page 19
www.genosafe.org
booth2: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
booth3:Sigma-aldrich: see page 21
www.sigma-aldrich.com
booth3:SaFC: see page 21
www.safcglobal.com
booth4: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
booth5:PeproTech: see page 20
www.peprotechec.com
exhibitOrs
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booth6: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
booth7: 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
booth8:biooutsource: see page 18
www.biooutsource.com
exhibitOrs
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booth9: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
booth10: aTMi: see page 18
www.atmi-lifesciences.com
booth11: 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
exhibitOrs
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booth12: 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
booth13: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
booth14: 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
exhibitOrs
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booth15: 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
booth16: 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
booth17: 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
booth18: PlasmidFactory: see page 20
www.PlasmidFactory.com
exhibitOrs
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exhibitOrs
booth19: 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
booth20:Charles river: see page 19
www.criver.com
booth21: 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
booth22: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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exhibitorsbooth 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
ESGCT/SFTCG COLLABORATIVE COnGRESS 2012
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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
ReseaRch PRioRity PRogRam 1230 (sPP1230)MECHanISMS oF GEnE VECToR EnTRy anD PERSISTEnCE
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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.
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Versailles_2012_book.indb 35 01/10/2012 15:17
36 36
Programmethursday 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
4_programme.indd 36 02/10/2012 11:06
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nOtes
I never said it was possible. I only said it was true. — Charles Richet
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Programmethursday 25 october 2012
Education dayAuditorium Richelieu
09.00 Gene transfer strategiesChair: 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 integrationChair: Christof von Kalle
INV005 controlling vector tropis�m by trans�ductional targetingEls Verhoeyen, Université de Lyon
INV006 exploiting microrNa regulation for genetic engineeringBernhard Gentner, San Raffaele Telethon Institute for Gene Therapy, Milan
INV007 retroviral and lentiviral integration s�ites� s�tudyChristof von Kalle, German Cancer Research Center, Heidelberg
INV008 clonality s�tudies� in gene therapyChristopher Baum, Hannover Medical School
12.15 Lunch – Mazarin Room
13.30 Gene therapy and immune responsesChair: 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
4_programme.indd 38 02/10/2012 09:15
39
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nOtes
I never said it was possible. I only said it was true. — Charles Richet
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40 40
Programmethursday 25 october 2012
Education dayAuditorium Richelieu
15.00 cell therapyChair: Christian Jorgensen
inv009 methods for iPs cell generation and quality control assessmentOlivier Féraud, Ingestem Consortium, Villejuif
inv010 application of induced Pluripotent stem cells in hematological genetic diseasesJosé C. Segovia, CIEMAT-CIBERER, Madrid
inv011 adiPoa project: adipose derived stroma cells for osteoarthritisChristian Jorgensen, University of Montpellier
inv012 treg based cell therapyDavid Klatzmann, Pitié Salpêtrière Hospital, Paris
16.15 afternoon break
16.45 Regulatory aspects of clinical trials in cell or gene therapyChair: Nicolas Ferry
inv015 safety testing of high titre adenovirusDaniel Galbraith, BioOutsource, Glasgow
inv016 dealing with regulatory authoritiesNicolas 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, 2011Jean Claude Ameisen, Université Paris Diderot
20.00 Welcome drinks – Mazarin Room
4_programme.indd 40 02/10/2012 11:20
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nOtes
I never said it was possible. I only said it was true. — Charles Richet
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Programmefriday 26 october 2012
main conference08.00 registration open
08.30 1: major concepts for cell and gene therapy
Auditorium Richelieu
Chair: Luigi NaldiniiNV017 the clonal repertoire of gene modified cells in
humansChristof von Kalle, German Cancer Research Center, Heidelberg
iNV018 In vivo genome editing as an approach to the treatment of genetic diseaseKatherine A. High, University of Pennsylvania, Philadelphia
iNV019 targeted gene correction in a mouse disease modelToni 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 PiDChairs: Alessandro Aiuti, Donald KohniNV020 gene therapy for primary immunodeficiencies
Alessandro Aiuti, HSR-TIGET, Milan
iNV021 gene therapy for ada-deficient severe combined immune deficiencyDonald Kohn, University of California, Los Angeles
iNV022 development of gene therapy for HLH due to perforin deficiency and for XLP1Bobby Gaspar, University College London
Proffered papersor001 Lentiviral vector mediated gene therapy for the treatment of
Wiskott-aldrich SyndromeSamantha 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 modelsKerstin 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 integrationJanine Reichenbach, University Children’s Hospital, Zürich
or004 efficient generation of gene-corrected and reprogramming factor-free iPS cells from Scid-X1 patientsAngelo Lombardo, San Raffaele Telethon Institute for Gene Therapy, Milan
4_programme.indd 42 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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PrOgraMMefriDaY26OCtOber2012
Or005 high-levelclusteringofintegrationsitesinproto-oncogenesandleukemogenesisingamma-retroviralWasgenetherapyAnna Paruzynski, German Cancer Research Center, Heidelberg
Auditorium Richelieu
1b: CancerChairs: Pierre Cordelier, Ian McNeishinV023 Phase1genetherapytrialforpancreaticcancer:frombenchto
bedsideLouis Buscail, Hôpital de Rangueil, Toulouse
inV024 OncolyticVacciniainducesprogrammednecroticcelldeathinovariancancerIain McNeish, Queen Mary University of London
inV025 anovelrnai-basedtreatmentforpancreaticcancer(PC):pre-clinical&clinicalresultsEithan Galun, Hadassah Hebrew University Hospital, Jerusalem
Proffered papersOr006 safetyandefficacyofrepeatedinfusionsofCelYVirinchildrenwith
metastaticneuroblastomaJavier Garcia-Castro, Instituto de Salud “Carlos III”, Madrid
Or007 lentiviralvector-basedinsertionalmutagenesisidentifiesnewlivercancergenesandmolecularnetworksthathaveapivotalroleinhumanhepatocarcinogenesisMarco Ranzani, San Raffaele Telethon Institute for Gene Therapy, Milan sPP1230award“Mechanismsofvector-hostinteractions”
Or008 redirectionofth17cellswithaniCOs-basedCarenhancesfunction,antitumoractivityandpersistenceofth17cellsSonia Guedan, University of Pennsylvania, Philadelphia
Or009 lymfactintM(lx-1101):anadenovirally-deliveredVegf-CgenetherapyincombinationwithlymphnodetransferforthetreatmentofpatientswithsecondarylymphoedemaassociatedwithbreastcancerAlan Boyd, Laurantis Pharma, Turku, Finland
Colbert/Montesquieu
1c: neuro-muscularChairs: Thomas Voit, George DicksoninV026 genetherapyforDuchenneMuscular
Dystrophy:microdystrophins,oligonucleotidesandendonucleasesGeorge 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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PrOgraMMefriDaY26OCtOber2012
inV027 rna-modulatingtherapeuticsforDuchenneMuscularDystrophyJudith van Deutekom, Prosensa Therapeutics, Leiden
inV028 genetherapyforspinalmuscularatrophy:perspectivesandproblemsThomas Voit, Institut de Myologie, Paris
Proffered papersOr010 Monitoringbyserummirnaofagenetransfertreatmentina
g-sarcoglycanopathymousemodelDavid Israeli, Généthon, Evry
Or011 raaV9-mediatedgenetransferinthespinalcordofafelinemodelofmotorneurondegenerationThomas Bucher, Université de Nantes
Or012 humanmesoangioblastscanelicitalloreactivet-cellimmuneresponses:implicationsforallogeneiccelltherapyofDMDMaddalena Noviello, San Raffaele Scientific Institute, Milan
Or013 aaV-mediatedgenereplacementtherapyforx-linkedmyotubularmyopathyAnna Buj-Bello, Généthon, Evry
Lulli 1d: regional talentsChair: Nathalie Cartier introductionbytheChairinV029 Majorconceptsforcellandgenetherapy
Jude Samulski, University of North Carolina, Chapel Hill
inV030 MeganucleasesforgenomeengineeringPhilippe Duchateau, Cellectis Therapeutics, Paris
Proffered papersOr014 sustainedstimulation/expansionofregulatorytcellstreat
autoimmunediseasewithoutimpairingeffectorimmuneresponsestoinfection,vaccinationandcancerGuillaume Churlaud, Pitié Salpêtrière Hospital, Paris
Or015 aaVgenetherapyforalzheimerdisease:consequencesofaaV-mediatedcholesterol24-hydroxylaseoverexpressioninthY-taU22mousemodelMarie-Anne Burlot, Université Paris Descartes
Or016 exonskippinggenetherapyforDystrophicepidermolysisbullosaSandrina Turczynski, Université Paris Descartes
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I never said it was possible. I only said it was true. — Charles Richet
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Or017 sMnrescuebyusingoligonucleotidesoftricyclo-Dnatoinduceexon7inclusioninsMn2mrnaValérie Robin, Institut de Myologie, Paris
12.30 sftCggeneralassembly–Auditorium Richelieu
12.30 lunch– Mazarin Room; Postersession1(P001-P148) – Gallerie Le Nôtre
14.30 2: Stem cells and regenerative medicine
Auditorium Richelieu
Chair: Marc PeschanskiinV031 humanembryonicstemcellsandiPsforregenerativemedicine:
tworealprospectsMarc Peschanski, I-Stem, Evry
inV032 Mesenchymalstromalcells:anewparadigmforcellularimmunemodulationWillem Fibbe, LUMC, Leiden
inV033 skeletalmusclestemcellsinregenerativebiologyShahragim Tajbakhsh, Institut Pasteur, Paris
16.00 afternoonbreak
16.30 Parallel Sessions 2a, 2b, 2c, 2d
Auditorium Richelieu
2a: Overcoming genotoxicityChairs: Christopher Baum, Eugenio MontiniinV034 engineeringmammaliancellsfortherapeuticapplications
Martin Fussenegger, Swiss Federal Institute of Technology, Basel
inV035 thefutureoftheretrosChristopher Baum, Hannover Medical School
inV036 integrationsiteanalysisinaclinicaltrialoflentiviralvectorbasedhematopoieticstemcellgenetherapyforMetachromaticleukodystrophyEugenio Montini, San Raffaele Telethon Institute for Gene Therapy, Milan
Proffered papersOr018 lentiviralvectorsdesignedforliver-directedgenetherapydonot
displaydetectablegenotoxicityinsensitivein vivoassaysMarco Ranzani, San Raffaele Telethon Institute for Gene Therapy, Milan
Or019 high-throughputmonitoringofbonemarrowclonalityinpre-clinicalandclinicalgenetherapystudiesStephanie Laufs, National Center for Tumor Diseases, Heidelberg
Or020 trackingt-memorystemcellsinhumansbyretroviraltaggingSerena Scala, HSR-TIGET, Milan
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I never said it was possible. I only said it was true. — Charles Richet
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Or021 identificationofadomainresponsibleforthedose-dependentantiproliferativeandapoptoticeffectsofSleeping BeautytransposaseMelanie Galla, Hannover Medical School sPP1230award“Mechanismsofvector-hostinteractions”
Conde/Pascal 2b: immune response, immuno-regulations, genetic vaccinesChairs: Olivier Boyer, Gilles Marodon
inV037 regulatingdendriticcellsignallingtoimprovevaccinationMary Collins, University College London
inV038 nKcellsandinnateimmunityEric Vivier, Centre d’Immunologie de Marseille-Luminy (CIML)
inV039 ChimpanzeeadvectortechnologyplatformforgeneticvaccineapplicationsStefano Colloca, Okairos, Pomezia
Proffered papersOr022 engineeringlentiviralvectorstotargetdendriticcells:thenanobody
displaytechnologyCleo Goyvaerts, Vrije Universiteit Brussels
Or023 genetransferinnaive-derivedmemorystemt-cells:anovelpromisingplatformforcancerimmunotherapyNicoletta Cieri, San Raffaele Scientific Institute, Milan
Or024 In vitroandin vivomodulationofaaVcapsidCD8+tcellresponseswithigg-derivedMhCclassiiepitopesFederico Mingozzi, Children’s Hospital of Philadelphia, Pennsylvania
Or025 incorporationofantigensintoviralcapsidsaugmentsefficacyofadeno-associatedViral(aaV)vector-basedvaccinesAngela Nowag, University Hospital Cologne
Lulli 2c: adult cell reprogrammingChairs: Sarah Ferber, Hidde Haisma
inV040 transdifferentiation:autologouscellreplacementtherapySarah Ferber, Sheba Medical Centre, Tel Hashomer
inV041 reprogrammingthroughthepluripotentestateasanewparadigmforhumancellrejuvenationJean Marc Lemaitre, Institute of Fuctional Genomics, Montpellier
inV042 DirectgenerationoffunctionaldopaminergicneuronsfromhumanfibroblastsVania Broccoli, San Raffaele Scientific Institute, Milan
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I never said it was possible. I only said it was true. — Charles Richet
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Proffered papersOr026 iPsC-basedgenetherapyofcongenitalerythropoieticporphyria
Aurélie Bedel, Université Bordeaux Segalen
Or027 impairedepithelialdifferentiationofinducedpluripotentstemcellsfromectodermalDysplasiaeeCpatientsisrescuedbyaPr-246/PriMa-1MetDaniel Aberdam, Université Paris Descartes
Or028 generationofdisease-freeiPscellsfromfanconianemiamicewithahypomorphicmutationinBrca2.Victoria Moleiro, CIEMAT/CIBERER, Madrid
Or029 Mitoticcorrectionoftrisomy21bydisomiccellsegregationoccurredinhumanpluripotentstemcellsfromDownsyndromeLucie Tosca, Université Paris-Sud, Villejuif
Colbert/Montesquieu
2d: late breaking abstracts and diversified platforms of cell and gene therapyChairs: Amina Zinaï, Sophie Gomez
Proffered papersOr030 intracerebralgenetherapyforsanfilipposyndrome
Amina Zinaï, Lysogene, Paris
Or031 Cancerregressionandneurologictoxicityfollowinganti-Mage-a3tCrgenetherapyRichard A. Morgan, National Cancer Institute, Bethesda, MD
Or032 self-inactivatinglentiviralvectorsforcorrectionofrag1severeCombinedimmunodeficiencyKarin Pike-Overzet, Leiden University
Or033 aprb-responsive,rgD-modified,andhyaluronidase-armedcanineoncolyticadenovirusasatherapeuticveterinaryagentandamodelforhumanvirotherapyEduardo Laborda, Institut d’Investigacions Biomèdiques de Barcelona
Or034 lMO2perturbshumantcelldevelopmentin vivobytwodifferentmechanismsAnna-Sophia Wiekmeijer, Leiden University
Or035 analpharetroviralvectorplatformsuitableforpermanentandtransientcellmodificationsJulia D. Suerth, Hannover Medical School
18.30 Posterparty1(P001-P148)– Gallerie Le Nôtre
19.00 speakers’dinner– by invitation only
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I never said it was possible. I only said it was true. — Charles Richet
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PleasenOte:at2amtonighttheclockswillturnbackonehourfortheendofDaylightsavingtime
08.30 3: Current status of cell and gene therapy applications in man
Auditorium Richelieu
Chair: Fulvio MavilioinV043 13yearsofexperiencewithgenetherapyof
primaryimmunodeficienciesAlain Fischer, Hôpital Necker, Paris
inV044 DevelopmentofagenetherapypipelineforinheritedretinaldystrophiesRobin Ali, University College London
inV045 Cart19inducessustainedfunctionalCartcellpersistenceandbcellaplasiaCarl June, University of Pennsylvania, Philadelphia
inV046 stablefactorixactivityfollowingaaV-mediatedgenetransferinpatientswithseverehemophiliab.Amit Nathwani, University College London
10.30 Morningbreak
11.00 Parallel sessions 3a, 3b, 3c, 3d
Auditorium Richelieu
3a: CardiovascularChairs: Michel Puceat, Christian BuchholzinV047 Celltypespecificgenedeliveryinthevascular
systemChristian Buchholz, Paul-Ehrlich-Institut, Langen
inV048 genetically-targetedtherapiesforheartfailure:stepstoclinicalpracticePatrick Most, University Hospital Heidelberg
inV049 regenerativemedicine:transforminghealthcaresolutionsAndré Terzic, Mayo Clinic, Rochester, MN
Proffered papersOr036 aaVbasedgenetherapyrescuesthemurinecardiacphenotype
associatedwithfriedreichataxiaMorgane Perdomini, Université de Strasbourg, Illkirch
Or037 stimulationoftransmuralcapillaryendothelializationofsmall-diametersyntheticvasculargraftsthroughlocaloverexpressionofanovelrecombinantVegfr2-ligandVegf-a109Jarkko Hytonen, University of Eastern Finland, Kuopio
Or038 Pluripotentstemcellscarryingamutatedlamina/C:acellmodeloflaminopathiesforthesearchfornewtherapeutictargets.Anne-Claire Guénantin, Université Paris Descartes
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I never said it was possible. I only said it was true. — Charles Richet
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Conde/Pascal 3b: Gene targetingChairs: Chiara Bonini, Diana SchenkweininV050 t-cellengineeringforadoptive
immunotherapyusingtal-effectornucleases(talens)Andrew Scharenberg, Cellectis Therapeutics, Paris
inV051 tCrgeneeditingtotreatmalignanciesChiara Bonini, San Raffaele Scientific Institute, Milan
inV052 ModifyingthehiV-1integrationmachinerytoyieldsafervectorintegrationDiana Schenkwein, University of Eastern Finland, Kuopio
Proffered papersOr039 retargetingoflentivectorintegrationbyleDgf/p75chimera
functionallyrescuesacellculturemodelforx-linkedCgDRik Gijsbers, KU Leuven
Or040 Developingengineeredzincfingertranscriptionalrepressorsofhuntingtinasapotentialtherapyforhuntington’sdiseasePhilip Gregory, Sangamo BioSciences, Richmond, CA
Or041 CorrectionofhemophiliabphenotypefollowingZfnmediatedgenomeeditinginadultmiceMichael Holmes, Children’s Hospital of Philadelphia, Penns
Or042 De novoandin silicodesignofregulatoryelementsfortargetedandrobusttissue-specificexpressioningenetherapyMarinee Chuah, Free University of Brussels
Lulli 3c: Brain gene therapyChairs: Nathalie Cartier, Alessandra BiffiinV053 ClinicaltrialofhsCgene
therapyformetachromaticleukodystrophyAlessandra Biffi, San Raffaele Scientic Institute, Milan
inV054 hematopoiticstemcellgenetherapyforx-linkedadrenoleukodystrophyNathalie Cartier, Université Paris Descartes
inV055 genetherapyforCnsdisordersRon Crystal, Weill Medical College of Cornell University, New York
inV056 therapyforschwannomasusingaaV-P0-caspaseandexosome-mediateddeliveryoftherapeuticrna/proteinXandra 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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Proffered papersOr043 Mir-155silencingdecreasesneuroinflammationinaβ-stressed
astrocytesandmicrogliacellsJoana Guedes, University of Coimbra
Or044 lentiviraldeliveryofhumanfull-lengthwild-typetauproteinmediatesprogressivespatio-temporalandcortico-corticalpropagatedneurofibrillarydegenerationinratbrainMorvane Colin, Université de Lille
Or045 Widespreadvectordistributionfollowingintra-CsfdeliveryofaaV9vectorsinalargeanimalmodelVirginia Haurigot, CBATEG, Barcelona
Colbert/Montesquieu
3d: Joint symposium Japanese and European Societies; Cancer and oncolytic virusesChairs: Len Seymour, Yasufumi KanedainV057 anon-replicatingoncolyticviralparticleasanovel
therapeutictoolagainstcancersYasufumi Kaneda, Osaka University, Suita
inV058 Coload1agroupboncolyticadenovirus:pre-clinicalassessmentofpotency,safetyandselectivityKerry Fisher, University of Oxford
inV059 ProstatecancergenetherapyinJapanYasutomo Nasu, Okayama University Hospital
inV060 targetedoncolyticandimmunotherapeuticvacciniaJx-594:clinicalproof-of-conceptforsystemicefficacyDavid Kirn, Jennerex, San Francisco, California
inV061 bioKnifetM,auPaactivity-dependentfusogenicsendaivirus,asanewclassofoncolyticbio-devicetotreatmalignanciesYoshikazu Yonemitsu, Kyushu University, Fukuoka
Proffered papersOr046 Combiningoncolysiswithgeneticimmunornasedeliveryas
innovativestrategyforcancertherapyInes Fernández-Ulibarri, DKFZ, Heidelberg
Or047 Oncolyticadenoviruswithtemozolomideinducesautophagy,immunogeniccelldeathandantitumorimmuneresponsespreclinicallyandincancerpatientsIlkka Liikanen, University of Helsinki
13.00 esgCtgeneralassembly–Auditorium Richelieu
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I never said it was possible. I only said it was true. — Charles Richet
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13.00 lunch– Mazarin Room; Postersession2(P149-P298)– Gallerie Le Nôtre
14.30 4: immunology
Auditorium Richelieu
Chair: Naomi TaylorinV062 high-throughputidentificationof
antigen-specifictCrsbytCrgenecaptureTon Schumacher, The Netherlands Cancer Institute, Amsterdam
inV063 tbCJohn Gribben, Barts Cancer Centre, London
inV064 advancinghsCandtlymphocytetherapiesbymanipulatingcellmetabolismNaomi Taylor, IGMM, Montpellier
16.00 afternoonbreak
16.30 Parallel sessions 4a, 4b, 4c, 4d
Conde/Pascal 4a: EpigeneticsChairs: Seppo Ylä-Herttuala, Anna SalvettiinV065 Cancerepigenetics:newparadigmsanddrugs
Frank Lyko, German Cancer Research Center, Heidelberg
inV066 Polycombcomplexesco-associatewithaspecificrnapolymeraseiivariantinescellsAna Pombo, Imperial College School of Medicine, London
inV067 epigeneticregulationofVegfexpressionSeppo Ylä-Herttuala, University of Eastern Finland, Kuopio
Proffered papersOr048 aubiquitouschromatinopeningelement(UCOe)prevents
transgenesilencinginmurinepluripotentcellsandtheirdifferentiatedprogenyMania Ackermann, Hannover Medical School
Or049 DecipheringthemolecularmechanismsofreprogrammingusingmirnascreensNils Pfaff, Hannover Medical School
Or050 lentiviraltransductionofCD34+cellsinducesgenome-wideepigeneticmodificationsDaniel 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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Lulli 4b: GmP and bioprocess development for GT vectorsChairs: Frédéric Revah, Manuel CarrondoinV068 Processdevelopmentinsingleusetofacilitate
cgMPingenetherapyManuel Carrondo, IBET, Oeiras
inV069 largescaleaaVproductionforclinicaltrialsforthetreatmentofneuromusculardiseasesOtto Merten, Généthon, Evry
inV070 lentiviralvectorsforin vivoclinicalapplications:currentandfutureproductionstrategiesJames Miskin, Oxford BioMedica
Proffered papersOr051 PlasmidDnaforindirectclinicalapplications–howmuchgMPis
necessary?�Marco Schmeer, PlasmidFactory, Bielefeld
Or052 DevelopmentofamanufacturingprocessforanoncolyticvaccineMaria Fe C. Medina, McMaster University, Hamilton, Ontario
Or053 invertedterminalrepeatread-throughandreversepackaging:mechanismsofoccurenceandstrategiestominimiseDnaimpuritiesinrecombinantaaV.John Fraser Wright, Children’s Hospital of Philadelphia, Penns
Colbert/Montesquieu
4c: Joint Symposium Japanese and European Societies 2; Genetic diseasesChairs: Yoshikatsu Eto, Nicole DégloninV071 applicationsofinducedPluripotentstem(iPs)
cellsforthepathogenesisoflyosomalstorageDiseases(lsD)Yoshikatsu Eto, Tokyo Jikei University
inV072 generationofdisease-freefanconianemiaiPsC-derivedhematopoieticprogenitorswithZfn-mediatedtargetedadditionofFANCAtotheAAVS1locusPaula Rio, CIEMAT/CIBERER, Madrid
inV073 WhatwelearntfromthegenetherapyforaDadeficiencyMasafumi Onodera, National Center for Child Health and Development, Tokyo
inV074 towardthetreatmentofhuntington’sdiseasewithrnaiNicole Déglon, CEA, Institute of Biomedical Imaging (I2BM) and Molecular Imaging Research Center (MIRCen), Fontenay-aux-Roses
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Programmesaturday 27 october 2012
Proffered papersor054 Lentiviral sgsH expression from the human cd11b promoter in
transplanted haematopoietic stem cells fully corrects behaviour and neuropathology of mucopolysaccharidosis IIIa miceAlexander Langford-Smith, University of Manchester
or055 aaV9 mediated gene therapy of mLd model miceNoriko Miyake, Nippon Medical School, Tokyo
or056 Neonatal intracerebral gene delivery to correct cNs pathology in a mouse model of globoid cell LeukodystrophyAnnalisa Lattanzi, San Raffaele Scientic Institute, Milan
Auditorium Richelieu
4d: Targeting vector entry and expressionChairs: Hildegard Büning, Thierry VandenDriesscheINV075 re-directing vector fate by capsid engineering
Hildegard Büning, University of Cologne
INV076 modulation of adenovirus vector - host interactionsFlorian Kreppel, Ulm University
INV077 solving the t-cell problem: generation of immune stealth aaVThierry VandenDriessche, Vrije Universiteit Brussels
Proffered papersor057 a lentiviral vector pseudotyped with a baboon retrovirus envelope
glycoprotein outperforms VsV-g-LVs for gene transfer into hematopoietic stem cells and lymphocytesAnaïs Girard Gagnepain, Université de Lyon
or058 microrNa-150-regulated vectors for cell type-specific transgene expression in hematopoietic gene therapySebastian Brennig, Hannover Medical School
or059 displaying high affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transferRobert Münch, Paul-Ehrlich-Institut, Langen
or060 regulated lentiviral vector gene transfer for X-cgdValentina 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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I never said it was possible. I only said it was true. — Charles Richet
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08.30 5: Vision for the future
Auditorium Richelieu
Chair: David KlatzmanninV078 geneticengineeringofhumanhematopoiesis
fortreatinginheriteddiseasesandcancerLuigi Naldini, San Raffaele Scientific Institute, Milan
inV079 neuralplasticityandneuronaldiversityintheadultmammalianbrainFred H. Gage, The Salk Institute for Biological Studies, La Jolla, CA
inV080 PulpfictionsDavid Klatzmann, Pitié Salpêtrière Hospital, Paris
10.30 Morningbreak
11.00 Parallel sessions 5a, 5b, 5c, 5d
Conde/Pascal 5a: Ethical and regulatory issues in cell and gene therapyChairs: Odile Cohen-Haguenauer, Jude SamulskiinV081 Ultra-rareorphandiseasesandneedforplatformgenedelivery
Jude Samulski, University of North Carolina, Chapel Hill
inV082 Developmentofgeneandcelltherapyproducts:regulatoryexpectationsJean Hugues Trouvin, Université Paris Descartes
inV083 OverlappingethicalissuesincellandgenetherapiesNancy M. King, Wake Forest School of Medicine, Winston-Salem, NC
Panel discussion
Lulli 5b: Biotherapies instituteChair: Marc PeschanskiinV084 Multicenterinternational
genetherapyclinicaltrialsforWiskott-aldrichsyndromeAnne Galy, Généthon, Evry
inV085 anexon-skippingstrategyusingaaV8-U7snrnavectorsforthetreatmentofDuchenneMuscularDystrophy–resultsingrMDdogsandclinicalperspectivesCaroline Le Guiner, Université de Nantes
inV086 humanpluripotentstemcellsforhDcelltherapy:hopeorhype?�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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Programmesunday 28 october 2012
PLease note: at 2am last night the clocks turned back one hour for the end of daylight saving time
inv087 restoration of vision in the Pde6ß-deficient dog, a large animal model of rod-cone dystrophyLolita Petit, Université de Nantes
Colbert/Montesquieu
5c: Liver and metabolismChairs: Karen Kozarsky, Anne Dubart Kupperschmittinv088 tbc
Fatima Bosch, Universida Autònoma de Barcelona
inv089 Pancreatic beta cell development: experimental models to transfer data from rodent to humanRaphael Scharfmann, Faculté Necker, Paris
inv090 Helper-dependent adenoviral vectors for inborn errors of liver metabolismNicola Brunetti-Pierri, Telethon Institute of Genetics and Medicine, Naples
Proffered papersor061 redifferentiation of expanded human pancreatic β-cell-derived cells
by inhibition of the notcH pathwayYael Bar, Tel Aviv University
or062 Hepatic gene transfer of tFeb, a master autophagy regulator, results in clearance of mutant alpha-1-antitrypsinNunzia Pastore, Telethon Institute of Genetics and Medicine, Naples
or063 optimisation of liver gene transfer for hemophilia bXavier M. Anguela, The Children’s Hospital of Philadelphia, Penns
or064 integration-defective lentiviral vectors expressing engineered hyperactive Factor ix improve vector performance following hepatic transductionNisha Nair, Free University of Brussels
Auditorium Richelieu
5d: Haematology: to insulate or not to insulateChairs: Salima Hacein-Bey, Karim Benihoudinv092 When insulation is not a solution: post-
transcriptional deregulation of gene expression induced by vector integrationFulvio Mavilio, Généthon, Evry
inv093 new chromatin insulators for gene therapyGeorge Stamatoyannopoulos, University of Washington, Seattle
inv091 tbcPhilippe Leboulch, Brigham and Women’s Hospital, Harvard University, MA
BIOSCIENCES
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Selected abstractsOr065 hla-universalplateletsinfusionspreventplateletrefractorinessina
mousemodelChristiane Gras, Hannover Medical School
Or066 CD34+cellsmobilisedbyPlerixafortreatmentinthalassemicpatientsareenrichedinstem/progenitorcellsendowedwithhighreconstitutionpotentialandgenetransfersusceptibilityMaria Rosa Lidonnici, San Raffaele Telethon Institute for Gene Therapy, Milan
Or067 targetingCD133+cellstoimprovelong-termgenemarkinginhematopoieticstemcellbasedgenetherapiesChristian Brendel, Georg-Speyer-Haus, Frankfurt
Or068 Protectionofhematopoieticstemcellsfromstress-inducedfunctionalimpairmentbyverylow-doseinterleukin-1stimulationMakoto Otsu, University of Tokyo
13.00 lunch– Mazarin Room
13.30Lulli
Career Session: European research Council fundingOpportunitiesin2013Dr. Fiona Kernan, Scientific Department, ERC Executive Agency
14.30 6: ageing
Auditorium Richelieu
Chair: Nicolas LevyinV094 glialcellregulationofhematopoieticstemcellhibernationinthe
bonemarrownicheHiromitsu Nakauchi, University of Tokyo
inV095 antisensebasedtherapeuticapproachesinhutchinson-gilfordProgeriaNicolas Levy, Children’s Hospital La Timone, Marseille
inV096 effectofageingandmuscleoriginonhumansatellitecellsVincent Mouly, Institut de Myologie, Paris
16.30 afternoonbreak
17.00 Parallel sessions 6a, 6b, 6c, 6d
Auditorium Richelieu
6a: EyeChairs: Robin Ali, Sam WadsworthinV097 successfulaaV-mediatedgenetherapyof
rod-coneandcone-roddystrophiesindogs:greatpromiseforthetreatmentofrapiddegenerativephotoreceptordefectsFabienne Rolling, Université de Nantes
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Programmesunday 28 october 2012
inv098 optogenetic vision restoration strategy Jens Duebel, Institut de la Vision, Paris
inv099 transplantation of es cell-derived photoreceptorsEmma West, University College London
Proffered papersor069 silencing HLa class i expression in human corneas to decrease the
risk of graft rejection after keratoplastyConstanca 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 mouseYvan Arsenijevic, Jules-Gonin Eye Hospital, Unil, Lausanne
or071 intravitreal delivery of aav-ndi1 for the treatment of Leber Hereditary optic neuropathyNaomi Chadderton, Trinity College Dublin
or072 alterations of epithelial stem cell marker patterns in human diabetic corneas and effects of c-met gene therapyGraziella Pellegrini, University of Modena and Reggio Emilia, Modena
Lulli 6b: Bioprocess of cell therapy productsChairs: Mitch Finer, Gabor Veres, Luc Douayinv100 In vitro generation of red blood cells:
perspectives for transfusion medicineLuc 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 applicationsGabor Veres, bluebirdbio, Cambridge, MA
inv102 Progress towards the cgmP production of hesc derived rbcsJoanne Mountford, University of Glasgow/SNBTS
Proffered papersor073 cd70 increases survival and avidity of cytotoxic t lymphocytes
generated for adoptive cell immunotherapy strategiesMohamad 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 eradicationMonica Casucci, San Raffaele Hospital, Milan
or075 clinical-grade human myoblasts for cell therapy: a chromosomal stability analysisOlivier Boyer, University of Rouen
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Or076 integratedbioprocessesforscalableproduction,purificationandcryopreservationofiPsC-derivedcardiomyocytesMargarida Serra, Universidade Nova de Lisboa, Oeiras
Conde/Pascal 6c: imaging and in vivo studiesChairs: Anne Galy, Serge CharpakinV103 thesodiumiodidesymporter(nis)anditsroleasatheranosticgene
Christine Spitzweg, University of Munich
inV104 imagingbrainactivitybymonitoringPo2transientsincapillariesSerge Charpak, Université Paris Descartes
inV105 MultiparametricspinalMriindegenerativemotorneurondiseasesPierre-François Pradat, Pitié Salpêtrière Hospital, Paris
Proffered papersOr077 Positivemolecularimagingofendogenousmicrornaregulationin
pathophysiologicalconditionsinmicePatrick Baril, Université d’Orléans
Or078 imagingofmesenchymalstemcellrecruitmentintothestromaofhepaticcoloncancermetastasesusingthesodiumiodidesymporter(nis)Kerstin Knoop, University Hospital of Munich
Or079 QuantitativenMr1himagingand31PspectroscopyevaluationoflocoregionalhighvenouspressureraaV8-U7-ese6-ese8exon-skippingtherapyinthegrMDJean-Laurent Thibaud, Ecole Nationale Vétérinaire d’Alfort, Paris
Or080 ChangesintheprogressionofamyloiddepositioninaPP/PsmiceafteroverexpressionofdifferentaPOeisoformsthroughintraventricularinjectionofanadeno-associatedvirusserotype4Eloise Hudry, Harvard Medical School, Mass
Colbert/Montesquieu
6d: non-viral gene deliveryChairs: Daniel Scherman, Patrick MidouxinV106 newavenuesinnonviralgenedeliveryandtherapy
Daniel Scherman, Université Paris Descartes
inV107 UpdateontheUKCfgenetherapyConsortiummulti-dose,non-viral,genetherapytrialEric Alton, Imperial College, London
Proffered papersOr081 nonviralgenetransferviaSleeping beautytransposonforCollagen
ViideliveryinhumanprimarykeratinocytesMaria Carmela Latella, University of Modena and Reggio Emilia, Modena
Versailles_2012_book.indb 74 01/10/2012 15:17
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I never said it was possible. I only said it was true. — Charles Richet
Versailles_2012_book.indb 75 01/10/2012 15:17
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PrOgraMMesUnDaY28OCtOber2012
Or082 In vivomrnaintroductiontocentralnervoussystemusingpolyplexnanomicellesKeiji Itaka, University of Tokyo
Or083 enhancementofZorro-lnaefficiencyinhuntingtingenesilencingviacombinationwithbislnaEman M. Zaghloul, Karolinska Institutet, Stockholm
Or084 non-invasiveimagingofpulmonarygenetransfer:fromprettyimagestoproductdevelopmentPeggy Richard-Fiardo, Laboratoire TIRO, CEA, Nice
Or085 In vivoexpressionofnucleartriggeredPegylatedpolyacridinepeptidepolyplexesKevin Rice, University of Iowa
19.00 endofProgramme
19.30 galadinner– 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
Versailles_2012_book.indb 76 01/10/2012 15:17
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I never said it was possible. I only said it was true. — Charles Richet
Versailles_2012_book.indb 77 01/10/2012 15:17
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Programmemonday 29 october 2012
08.30 7: Vision for a healthy world
Auditorium Richelieu
Chair: Philip Gregoryinv108 a holistic approach towards restoring vision
in retinitis pigmentosa, between prosthetics, optogenetics and rehabilitationJosé-Alain Sahel, Institut de la Vision, Paris
inv109 ZFn-edited cd4+ t cells for Hiv/aidS therapy: phase 1 trials of Sb-728-t in Hiv-infected subjectsPhilip Gregory, Sangamo BioSciences, Richmond
inv110 the role of partnerships in regenerative medicine and gene therapy – a pharma perspectiveJason Gardner, GlaxoSmithKline, Philadelphia, Penn
Proffered papersor087 ProSavin® a gene therapy approach for the treatment of Parkinson’s
disease: a phase i clinical trial updateStéphane Palfi, CEA, Fontenay aux Roses
or086 transcription activator-Like effector nucleases (taLens) for targeted inactivation of Hepatitis b virus cccdnaKristie Bloom, University of the Witwatersrand, Johannesburg
10.30 morning break
11.00 Presidential symposium
Auditorium Richelieu
Chairs: Seppo Ylä-Herttuala, Luigi Naldininew 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
4_programme.indd 78 01/10/2012 15:51
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I never said it was possible. I only said it was true. — Charles Richet
Versailles_2012_book.indb 79 01/10/2012 15:17
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Programmemonday 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-adrenoleukodystrophyDr 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 diseasesDr Randy Chandler, National Institutes of Health Bethesda Maryland
13.00 end of the congress
4_programme.indd 80 02/10/2012 08:50
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I never said it was possible. I only said it was true. — Charles Richet
Versailles_2012_book.indb 81 01/10/2012 15:17
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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.
5_back_matter.indd 82 02/10/2012 09:20
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
La Voie de l’écuyerGala dinner—Sunday 28 October 2012—19.30
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
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 sitewww.acadequestre.fr
A
© G
illE
S Ta
rGaT
5_back_matter.indd 83 02/10/2012 11:08
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COnFErEnCE VEnuE:PalaisdesCongrèsdeVersailles10ruedelaChancellerie,78000,Versaillestel:01.40.68.22.22
hOW TO GET ThErE...
By PlanE (WWW.adP.Fr )• FrOm Orly: fromOrlythebestwaytogettoVersaillesisbytaxi,itisa20minutetaxirideandshouldcostaround40to50euros.
• FrOm CharlES dE GaullE (rOiSSy) On ThE rEr (taxiwouldbeverylongandveryexpensive):theairporttrainstationisalongwalkfromtheterminals(couldbeasmuchas15minutes).
• buyatickettoyourfinaldestination,Versaillesrivegauche.(therearethreedifferentstationsinVersailles,butVersaillesrivegaucheisaroundthecornerfromthePalaisdesCongres).
• Catchrerb(directionsaintremylesChevreuse).
• ChangeatsaintMichelnotreDame(afteraboutonehour),takererligneC(directionVersaillesrivegauche).
• fromVersaillesrivegaucheitisafiveminutewalk.
By Train www.transilien.com, www.ratp.fr
•fromParis montparnassetrainstation:gotoVersailles Chantiertrainstation,thenitisabouta10minutewalktothePalaisdesCongrès.
•fromParis Saint lazareorParis la défensetrainstation:gotoVersailles rive droitetrainstation,thenitisabouta15minutewalktothePalaisdesCongrès.
•fromCentral Paris, rEr C line:gotoVersailles rive Gauchetrainstation,thenitisabouta5to7minutewalktothePalaisdesCongrès.(becareful,makesureyoubuy“Paris-Versaillesrivegauche”ticket.t+ticketisnotvalidforthisjourney).
By BuS•theratPno.171busrunsbetweenthePontdesèvresmetrostationandtheChâteaudeVersailles(PalaisdesCongrèsjustnexttotheChâteau).
By Taxi•richardtaxi:+33666651039http://www.taxi-versailles.com
•listofalltaxisinVersailles:http://www.taxis-paris.fr/versailles.htm
By Car•fromParis,autoroutea13towardsVersailles/saintQuentin-en-Yvelines/rouentoexitno.5Versaillescentre/VersaillesChâteau.headtowardVersailles,thentakeavenuedesetats-Unis,and,finally,avenuedesaintCloud.Versaillescanalsobereachedby:a86,andn10.
•Parking:therearemanyparkinglotsavailableinallkeyareasofthecity:nexttothePalaisdesCongrès,avenuedesceaux,andonthePlaced’armes
traVelinfOrMatiOn
Versailles_2012_book.indb 84 01/10/2012 15:17
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ESGCT/SFTCG CollaboraTivE ConGrESS 2012
Car parking near the palais des CongresParking Place d’Armesplace du Château – F – 78000 Versaillesdistance from palais des Congrès: 200m tel: +33 (0)1 39 53 74 60 number of spaces: 640 open seven days per week
Parking Europe66 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 Saint-Cloud 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 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
5_back_matter.indd 85 02/10/2012 10:43
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restaUrantsnearthePalaisDesCOngres
restaurant Telephone
alaCoiffebretonne–10avenuedugénéraldegaulle 01-30-21-78-22
auChapeaugris–7ruehoche 01-39-50-10-81
auChienquifume–72ruedelaParoisse 01-39-53-14-56
aufouduroy–6ruedelachancellerie 01-30-21-80-81
auPetitMarquis–6rueandréChenier 01-39-50-47-40
brasserieduthéâtre–15ruedesréservoirs 01-39-50-03-21
buffalogrill–7avenuedesceaux 01-30-21-32-03
CafédelaPlaced’armes–1avenuedesaintCloud 01-39-50-03-60
Cheztiouiche-aubonaccueil–4ruestJulien 01-30-21-56-66
laflottille–parcduchâteau 01-39-51-41-58
laMaréedeVersailles–22rueauPain 01-30-21-73-73
latavernedeMaîtreKanter–5rueColbert 01-39-50-02-50
lelimousin–1ruedesatory 01-39-50-21-50
lelondres–7rueColbert 01-39-50-05-79
lerestoduroy–1avenuedestCloud 01-39-50-42-26
leValmont–20rueauPain 01-39-51-39-00
lebanon–11ruedesatory 01-30-21-23-13
lesManèges–2bisavenuedeParis 01-39-07-46-46
lestroisMarches–1boulevarddelareine 01-30-84-52-00
Pizzaevi–15ruedesetatsgénéraux 01-39-50-16-53
restaurantl’entrecote–18bisrueneuvenotreDame 01-39-53-09-53
sister’sCafé–15ruedesréservoirs 01-30-21-21-22
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GENE THERAPY FOR RARE DISEASESAdvancing 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
PRIX GALIEN 2012
Member of
Versailles_2012_book.indb 86 01/10/2012 15:18
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GENE THERAPY FOR RARE DISEASESAdvancing 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
PRIX GALIEN 2012
Member of
Versailles_2012_book.indb 87 01/10/2012 15:18
EDITED BY ODILE COHEN-HAGUENAUER, MD-PHD, ON BEHALF OF THE CLINIGENE NETWORK OF EXCELLENCEFOR THE ADVANCEMENT OF CLINICAL GENE TRANSFER AND THERAPY, EC-FP6, LSHB-CT-2006-018933
Clinical Gene transfer: state-of-the-art The CliniBook
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 - - - - - - - - - - - - - - - - - - - - - - - - - - Country - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -
Quantity - - - - - - - - - - - - - - x 80 € = - - - - - - - - - - - - - - - - - - - + Postage 5 € per book = Total price - - - - - - - - - - - - - - - - - - - - - € Payment by:
Quantity - - - - - - - - - - - - - - x 105 $ = - - - - - - - - - - - - - - + Postage 20 $ per book for the USA = Total price - - - - - - - - - - - - $ Payment by:
n Credit card and Fax: please return this form completed to: +33 (0)1 69 86 06 78 with:
Cardholder’s name - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - Credit card N° |—|—|—|—| |—|—|—|—| |—|—|—|—| |—|—|—|—|Expiration date |—|—| / |—|—| Security code on the back of the credit card |—|—|—|n Cheque (on order: EDK) and send it to: Editions EDK – Groupe EDP Sciences
17, avenue du Hoggar, P.A. de Courtabœuf, 91944 les Ulis cedex A, France.
To receive an invoice, please check this box: n Date - - - - - - - - - - - - - - - - - - - - - - - - - - - Signature - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -
EC-FP6
Foreword L. Luzzatto & I. Verma
y Introduction including prospect for a ➤ O. Cohen-Haguenauerpan-european infrastructure for GT vectorsy Highlights on AAV mediated ➤ F. BoschwithR. Ali, A. Auricchio, E. Ayuso, V. Blouin, G.Dickson, gene transfer M. Giacca, O. Merten, P. Moullier
y Retrovirus mediated gene transfer ➤ P. Cruz & M. CarrondowithC. Baum, C. Buchholz, K. Cichutek, state-of-the-art H. Hauser, A. Schambach
y Highlights on lentivirus ➤ K. Cichutek & M. SchweizerwithP. Alves, O. Cohen-Haguenauer, mediated gene transfer F.L. Cosset, N. Déglon, E. Verhoeyen, D. Wirth
y Highlights on gene-modified ➤ G. GahrtonwithE. Alici, O. Cohen-Haguenauer, P. Cruz, D.Klatzmann, cell therapy W. Lindermaier
y Adenovirus mediated gene transfer: ➤ S. KochanekwithP. Alves, M. Anton, M. Carrondo, B. Gänsbacher, current developments S. Ylä-Herttuala
y Non-viral based gene transfer: ➤ G. DicksonwithC. Baum, J. Bode, Z. Itzvak, Z. Ivics, C. Marie, a new era M. Schleef, M. Schmeer, D. Sherman
y Highlights on iPS induction, genetic ➤ O. Cohen-HaguenauerwithE. Bayart, T. Cathomen, M. Chuah, stability and emerging technologies F. Pâques, J. Smith, T. Vandendriessche
y Preclinical assessment tools ➤ A. Panet & A. JacobswithC. von Kalle, C. Richard, M. Schmidt
y Immune responses and Immunotoxicity ➤ D. KlatzmannwithB. Bellier, A. Galy, F. Mingozzi
y Biosafety platform state-of-the-art ➤ C. von KallewithA. Aiuti, C. Baum, E. Montini, M. Schmidt
y Clinical trials ➤ B. GänsbacherwithR. Ali, A. Aiuti, N. Cartier, M. Eriksdotter-Jönhagen, A. Nathwani, S. Palfi, S. Ylä-Herttuala
y Ethical and regulatory issues ➤ O. Cohen-Haguenauer, A. Kent, N. King
y Integration and dissemination issues ➤ R. Draghia-AkliwithG. Dickson, B. Gänsbacher, A. Kent, D. Klatzmann, L. Naldini, T. Vandendriessche and the CliniGene Coordinator
FlyerOrderA5_Mise en page 1 24/09/12 19:20 Page1
eUrOPeansOCietYfOrgeneanDCelltheraPYaChieVeMentaWarDs
Outstanding achievement award:incollaborationwithhumangenetherapyandMaryannliebertPublishers,esgCtpresentsoneawardforanestablishedresearcherwhohasmadealong-term,outstandingcontributiontothefield.€2000honorariumand30minutepresentationduringtheannualcongress.
young investigator awards:€1000anda15minutepresentationduringtheannualcongressforuptofourresearcherswhoareshowingexceptionalpromise.
Travel grants:upto10awardsof€250forPhDandfirstpostdocs.thesewillbeawardedonthebasisofabstractscore.
applicationandnominationdetailsareavailableatwww.esgct.eu/awards
note:eligibilitycriteriaapplies
esgCteValUatiOnWedohopeyouhaveenjoyedtheesgCt/sftCgCollaborativeCongress2012.WereallyvalueyourfeedbackaboutallaspectsoftheCongress.WewouldbeverygratefulifyoucouldtakeafewminutestocompletethisonlinequestionnaireeitherduringorsoonaftertheCongress.
https://www.surveymonkey.com/s/ESGCT2012
alinkisalsoavailablefromtheesgCtwebsite.
ifyouenjoyedthismeeting,wouldyouconsiderhosting/organisinganesgCtorsftCgcongressin2015?�ifso,pleaseforwardyourproposaltotheesgCtboardassoonaspossibleatoffice@esgct.eu,[email protected]
thankyouinadvanceforyourtime.
ESGCT and SFTCG Team
Versailles_2012_book.indb 88 01/10/2012 15:18
EDITED BY ODILE COHEN-HAGUENAUER, MD-PHD, ON BEHALF OF THE CLINIGENE NETWORK OF EXCELLENCEFOR THE ADVANCEMENT OF CLINICAL GENE TRANSFER AND THERAPY, EC-FP6, LSHB-CT-2006-018933
Clinical Gene transfer: state-of-the-art The CliniBook
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 - - - - - - - - - - - - - - - - - - - - - - - - - - Country - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -
Quantity - - - - - - - - - - - - - - x 80 € = - - - - - - - - - - - - - - - - - - - + Postage 5 € per book = Total price - - - - - - - - - - - - - - - - - - - - - € Payment by:
Quantity - - - - - - - - - - - - - - x 105 $ = - - - - - - - - - - - - - - + Postage 20 $ per book for the USA = Total price - - - - - - - - - - - - $ Payment by:
n Credit card and Fax: please return this form completed to: +33 (0)1 69 86 06 78 with:
Cardholder’s name - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - Credit card N° |—|—|—|—| |—|—|—|—| |—|—|—|—| |—|—|—|—|Expiration date |—|—| / |—|—| Security code on the back of the credit card |—|—|—|n Cheque (on order: EDK) and send it to: Editions EDK – Groupe EDP Sciences
17, avenue du Hoggar, P.A. de Courtabœuf, 91944 les Ulis cedex A, France.
To receive an invoice, please check this box: n Date - - - - - - - - - - - - - - - - - - - - - - - - - - - Signature - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -
EC-FP6
Foreword L. Luzzatto & I. Verma
y Introduction including prospect for a ➤ O. Cohen-Haguenauerpan-european infrastructure for GT vectorsy Highlights on AAV mediated ➤ F. BoschwithR. Ali, A. Auricchio, E. Ayuso, V. Blouin, G.Dickson, gene transfer M. Giacca, O. Merten, P. Moullier
y Retrovirus mediated gene transfer ➤ P. Cruz & M. CarrondowithC. Baum, C. Buchholz, K. Cichutek, state-of-the-art H. Hauser, A. Schambach
y Highlights on lentivirus ➤ K. Cichutek & M. SchweizerwithP. Alves, O. Cohen-Haguenauer, mediated gene transfer F.L. Cosset, N. Déglon, E. Verhoeyen, D. Wirth
y Highlights on gene-modified ➤ G. GahrtonwithE. Alici, O. Cohen-Haguenauer, P. Cruz, D.Klatzmann, cell therapy W. Lindermaier
y Adenovirus mediated gene transfer: ➤ S. KochanekwithP. Alves, M. Anton, M. Carrondo, B. Gänsbacher, current developments S. Ylä-Herttuala
y Non-viral based gene transfer: ➤ G. DicksonwithC. Baum, J. Bode, Z. Itzvak, Z. Ivics, C. Marie, a new era M. Schleef, M. Schmeer, D. Sherman
y Highlights on iPS induction, genetic ➤ O. Cohen-HaguenauerwithE. Bayart, T. Cathomen, M. Chuah, stability and emerging technologies F. Pâques, J. Smith, T. Vandendriessche
y Preclinical assessment tools ➤ A. Panet & A. JacobswithC. von Kalle, C. Richard, M. Schmidt
y Immune responses and Immunotoxicity ➤ D. KlatzmannwithB. Bellier, A. Galy, F. Mingozzi
y Biosafety platform state-of-the-art ➤ C. von KallewithA. Aiuti, C. Baum, E. Montini, M. Schmidt
y Clinical trials ➤ B. GänsbacherwithR. Ali, A. Aiuti, N. Cartier, M. Eriksdotter-Jönhagen, A. Nathwani, S. Palfi, S. Ylä-Herttuala
y Ethical and regulatory issues ➤ O. Cohen-Haguenauer, A. Kent, N. King
y Integration and dissemination issues ➤ R. Draghia-AkliwithG. Dickson, B. Gänsbacher, A. Kent, D. Klatzmann, L. Naldini, T. Vandendriessche and the CliniGene Coordinator
FlyerOrderA5_Mise en page 1 24/09/12 19:20 Page1
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including DNA, RNA, and Cell Therapies
www.liebertpub.com/hum
Human Gene Therapy Methods
www.liebertpub.com/hgtb
www.liebertpub.com/hum
European Society of Gene and Cell Therapy
The Official Journal of:
The Premier Journal on Gene Therapy with Exclusive Coverage of Methods
Editor-in-Chief:James M. Wilson, MD, PhD
Methods Editor:Thierry VandenDriessche, PhD
Versailles_2012_book.indb 90 01/10/2012 15:18
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
Characterization of:• Inflammatory responses• Humoral responses• Cell-mediated responses
Cell lines
• Customized cell line engineering• Stem cell characterization
Clinical trials• Patient Follow-up
Safety and efficacy of biotherapeutic productS
addreSS GenoSafe
1 rue de l’internationaleBP 40064
91002 Evry CedexFrance
www.genosafe.com
ShareholderS
Genethon (60%)AFM (40%)
Founded 2003
contact
Vincent ZulianiPhone: +33(0)1.69.47.11.57
Fax: +33(0)[email protected]
including DNA, RNA, and Cell Therapies
www.liebertpub.com/hum
Human Gene Therapy Methods
www.liebertpub.com/hgtb
European Society of Gene and Cell Therapy
The Official Journal of:
Versailles_2012_book.indb 91 01/10/2012 15:18
w w w . t e x c e l l . c o m
VIRAL SAFETY & IMMUNOLOGYGMP / GLP Services
To get further information
S t a n d n ° 1 1
• Characterization of cells
• Safety Testing
• Biodistribution Studies
• Viral Clearance
• Customized Cell Culture
• Central Lab
• Biomarkers
• Immunogenicity
• Potency / Impurities
• Protein expression
Versailles_2012_book.indb 92 01/10/2012 15:18
w w w . t e x c e l l . c o m
VIRAL SAFETY & IMMUNOLOGYGMP / GLP Services
To get further information
S t a n d n ° 1 1
• Characterization of cells
• Safety Testing
• Biodistribution Studies
• Viral Clearance
• Customized Cell Culture
• Central Lab
• Biomarkers
• Immunogenicity
• Potency / Impurities
• Protein expression
2013www.sftcg.fr
22 March 2013
SFTCG AnnuAl ConGreSS 2013: PAriS
Faculté de médecine de l’université Paris-Sud, Hôpital du Kremlin-Bicêtre
2013Savedate!the
unique opportunities for young scientists to:• shape the programme
• chair sessions• present data
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Cover image © Mairie de Versailles Gala dinner images © 2012 BartabasOther images © Mairie de Versailles, unless otherwise indicated
Design: www.catherinecharnock.co.uk
to landfill
COnGress OffiCe infOrMatiOn
Contact namesGaëlle Jamar – event Manager Vanessa sampson – Payment & Membership
Registration desk opening hoursthursday 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
Payment queriesPlease go to the registration desk during the above opening hours
Membership queriesPlease go to the registration desk during the above opening hours
Contact number in case of emergencyGaëlle Jamar, event Manager +44 7766 475379
Wifi is aVailaBle in the Palais Des COnGres
Wifi naMe: sePCVlOGin: esGCt2012PassWOrD: esGCt2012
covers_imposed2.indd 2 04/10/2012 12:54
ESGCT 21st Anniversary Congressin collaboration with the SETGYC
www.esgct.eu • www.setgyc.es
24-28 October 2013
European Society of Gene and Cell Therapy and
Spanish Society of Cell and Gene Therapy
will hold a joint meeting in Madrid
ESGC
T and SFTCG Congress 2012 • Program
me and Inform
ation Book
25-29 October 2012 | Palais des Congrès de Versailles
www.esgct.eu • www.sftcg.fr
Collaborative Congress 2012
European Society of Gene and Cell TherapyFrench Society for Cell and Gene Therapy