ESGCT Conference Book

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

Versailles_2012_book.indb 1 01/10/2012 15:16

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


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


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


5


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


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


LES ESPACES / MEETING SPACE

Office TraiteurCaterers’ service areaTRAITEUR


BOILEAU50 m²

MONTESQUIEU50 m²

Galerie LE NÔTRE280 m²

COLBERT130 m²

Balcon amphithéâtreAuditorium balcony

RICHELIEU900 places

Ba

300 places


SERVICES

Salles / Rooms

Entrées / Entrance

ACCES / ACCESS

Ascenseur / Lift

Escalier / Stair

2Niveau Level


VAUBAN50 m²

PASCAL50 m²

FOYER CONDÉ100 m²

SCÈNEstage

AMPHITHÉÂTREauditoriumRICHELIEU

1 200 places

SCÈNE


SERVICES

Salles / Rooms

Entrées / Entrance

ACCES / ACCESS

Ascenseur / Lift

Escalier / Stair

1Niveau Level



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


15.40 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


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


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


16.30 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 ?


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


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


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

• Bulk Harvests

• Virus Banks

• Replication Competent Viral Vectors

Pre-clinical and Clinical Trials Support

• Biodistribution Studies

• Biopotency Assays

www.biooutsource.com

Your partner for release testing Gene Therapy Products

• GLP, GMP and GXP compliant testing

• EMA, FDA, CBER, ICH Guidelines

• Batch Release

• Stability

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.

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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.

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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


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


17


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.


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


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


19


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


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


21


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

22


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


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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


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


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


27


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


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


29


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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3_sponsors_exhibitors.indd 33 01/10/2012 15:37

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


C’est bien le temps qu’il vous faudra maintenant pour la réalisation de vos tests LAL avec la sécurité d’une méthode conforme pharmacopée et validée FDA

Nous sommes tellement convaincus des bénéfices que le PTS et le MCS apporteront à votre laboratoire que nous vous invitons à en tester un gratuitement.

www.stopwastingtimeandmoney.net

Dosage d’endotoxines en 15 minutes

[email protected]

edit any gene at any position in any genomeCustom TALEN™ and exclusive support for all your gene editing projects only available at Cellectis bioresearch

With over twelve years expertise in genome customization Cellectis bioresearch is the dedicated service provider in Custom TALEN™ design, production, validation and comprehensive project support.

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for more information, please visit cellectis-bioresearch.com*


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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

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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

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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

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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

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PrOgraMMefriDaY26OCtOber2012

Or005 high-levelclusteringofintegrationsitesinproto-oncogenesandleukemogenesisingamma-retroviralWasgenetherapyAnna Paruzynski, German Cancer Research Center, Heidelberg


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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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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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


Chair: Marc PeschanskiinV031 humanembryonicstemcellsandiPsforregenerativemedicine:

tworealprospectsMarc Peschanski, I-Stem, Evry

inV032 Mesenchymalstromalcells:anewparadigmforcellularimmunemodulationWillem Fibbe, LUMC, Leiden

inV033 skeletalmusclestemcellsinregenerativebiologyShahragim Tajbakhsh, Institut Pasteur, Paris


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


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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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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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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08.30 3: Current status of cell and gene therapy applications in man


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



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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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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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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13.00 lunch– Mazarin Room; Postersession2(P149-P298)– Gallerie Le Nôtre

14.30 4: immunology


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



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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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


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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08.30 5: Vision for the future


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


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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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


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


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




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


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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


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Programmemonday 29 october 2012

08.30 7: Vision for a healthy world


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


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

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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

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Congress soCial aCtivities

Guided tour of the historic city of Versailles, passinG by the palace Duration 2h30. Times to be arranged for each group: meet with your guide at the entrance of the Palais des Congrès.

the city of versailles was laid out as a setting for the Palace and is rich in buildings of great interest to visitors.

the saint-louis quarter, built on the site of louis Xiii’s former deer park, is today one of the city’s historic focal points. laid out somewhat in the form of a chessboard around its magnificent cathedral, it is also remarkable for its “carrés”, small houses with typically Xviiith century mansard roofs, and for the royal Kitchen garden created by the garden designer de la Quintinie between 1678 and 1683.

the notre-Dame quarter was a new town created at the instigation of louis Xiv in his desire to embellish the approaches to the Palace. visitors enjoy leisurely strolls here in the heart of the city, the streets lined with interesting shops and buildings such as the théâtre Montansier, the Church of notre-Dame, the statue in the emblematic Place Hoche, the Hôtel du Baillage in the antiques area and the market hall in the Place du Marché.

Guided tour of the palace of Versailles: the KinG’s and Queen’s apartments and the hall of mirrors Duration 90 minutes every lunch time, starting at 12h30.

Meet with your guide at the entrance of the Palais des Congrès. enter the private residence of the sun King and discover the King’s apartments dedicated to the theme of the planets.

enjoy sumptuous surroundings in which gold and marble form a harmonious decor. Walk the length of the Hall of Mirrors, now more dazzling than ever before. the recently restored glittering mirrors and candelabras reinforce the magical and prestigious spectacle of the long line of scintillating giltwork. Continue through the elegant Queen’s apartments and the bedchamber of Marie-antoinette.

For more information on both tours please visit the Tourism Office Booth in the entrance hall of the Palais des Congrès.

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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

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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).

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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


85


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

86

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


© C

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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


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


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


including DNA, RNA, and Cell Therapies


Human Gene Therapy Methods

www.liebertpub.com/hgtb


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


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


Human Gene Therapy Methods

www.liebertpub.com/hgtb

European Society of Gene and Cell Therapy

The Official Journal of:


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


• Viral Clearance

• Customized Cell Culture

• Central Lab

• Biomarkers

• Immunogenicity

• Potency / Impurities

• Protein expression


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


• 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

UsefUlnUMberslocalpolice:vehicleremoval______________________________________ 0139502548

Poisoncontrolcentre____________________________________________ 0140054848

Policestation __________________________________________________ 0139247000

Carpound_____________________________________________________ 0139530899

gendarmerie___________________________________________________ 0139675000

Dutypharmacies=>policestation_________________________________ 0139247000

Phébus–urbantransport ________________________________________ 0139201620

fireservice ______________________________________________________________ 18

emergencyservices_______________________________________________________ 17

emergencymedicalservice__________________________________ 15or0130849600

snCf(nationalrailoperator)______________________________________ 0892353535

Yvelinesemergencydoctors’service________________________________ 0139585858


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

ESGCT Conference Book

Documents

Transcript of ESGCT Conference Book