ESGCT_The Hague Congress 2014 by Catherine Charnock

European Society of Gene and Cell Therapy Finnish Society of Gene Therapy 17–20 SEPTEMBER SEPTEMBER 2015 2015 • FINLANDIA HALL, HELSINKI

www.fsgt.fi • www.esgct.eu Hague_covers.indd 1

ESGCT and NVGCT Congress 2014 • Programme and Information Book

Collaborative Congress 2015

1944 Avery, MacLeod & McCarty 1953 Watson & Crick 1973 Graham & van der Eb 1989 Capecchi 1991 Culver, Anderson & Blaese 2000 Venter – Collins 2000 Cavazzana-Calvo et al 2012 EC approves first gene therapy

The XXIInd Annual ESGCT Congress in collaboration with the NVGCT 23–26 October 2014 WorldForum, The Hague, The Netherlands

www.esgct.eu • www.nvgct.nl 01/10/2014 10:12

berken boschb lok

du i

pa rk w

van

scheveningseweg

kapelw

pa rk -

nieuwe

we g

b sjac o

hu ver

an sm ple

ett a al

testr. tr. terna

ierstr stra aa att

ierstr s

cht

weg

burn

aat

't ho enstraa t

raam

batjan str.. str

suma trastr

cura

laçaostraat catten van burc h

an 28 coppes

sstr tr..

egra

ban ank kas astra traa at

traat at

delis tr.

riouw

atjeh stra str aa att

ginn

delis tr.

onin

jnla

burg. de monchyplein

balist istrra aa at

ka bu rn rg eb . ee

billit on str. mado astrer . soen strda .

astr.

kkastr.

borneos

esstra at at

mala

oflaa

lombokk str. -

riou rio uw wsstr.

celeb

burg . ppa ati alex an veldder-

na na anlow . u tr pa s

llllw we

kerkh

astr. bank

esstra at at

kkastr.

flaan

bank

celeb

mala

sstr traa t

sstr tra aa att timor

sstr tr..

kerkho

park ken

in se

vin

eg yw pu

prinsevink enp

pr 10

va n

n. nsstr.

cr em erw la eg an

nw eg

sto lk

an speijkstraat

pr. willems

aa t st r do or n

weg

kl n.

j. de graeffln.

de perponcherstraa traatt an eten ete nstr.

boni

an sla der

sta dh

aan

stadhouders l

scheveningse

k-k

val eri u ss tr.

scheveningseweg

staten laan sta atenlaa n

la er la an

et ze bo va n

dtan

gel

erik ik-

slin

van

fred

aan

bev

erin ing

ela erl

van

an bo etz tr. traat

van merlen merle nstraa traatt

conradkade

carnegielaan

l an mea erdevan rvoo rt

riouw

straa traatt

meerder rdervoort

nepweg len

v ary

egie n carn plei

t duc

eg rgw

laan van

kade

obrechtstr. obrechts tr.

ers we g

Bosjes

lin

ra st

er la

eg nrustw

as s

. teld

Scheveningse

weg olweg tol

te bui

la ne

ke . nb str u n ba

schuytstr. schuytst r.

jacob catsl sla aan nbu ruste

lin

plein

kade

bl an

gr oo t

eg lw el

oplaa n

Zorgvliet

badhuisweg

ff.. b.m

a bi to

k 17

va n

laan

tsoen

park

oeko

badhuisweg

ria an g

badhuisweg

ersplan

17 sw ee pyrmont-

https://twitter.com/esgct

tstr a

hts tr.

emma

SPEAKER READY ROOM

Volga Room 1

stadhoud

MEMBERSHIP QUERIES Please go to the registration desk during the opening hours

rec

21 16

pro

frisolaan

in og rt he

ee lin c

For convenience, you can check in coats and luggage near the entrance on the ground floor.

sch

waldeck

hollanders erstraat 2e van blanken burg str.

an joh

n la a itt wit de jan willem

anw plesm

laan

Van Stolkpark 20

churchillchurchill plein

nc ni en elp alex. r.j. schimm gogel gogel ale weg xa nd er g o gel

dankert ertsstra at at

eweg hog

eg van len nep w

straat at

laan

belvedere-

Westbroekpark

straat at

nicolai-

eisenhowerlaan

wer

e uw nie

lübeck-

van hoornbeeks ekstraa traatt van bleiswijks bleiswijkstraa traatt

at aa ra ksttr ijks

ia rtusv ert hu b

plantsoe n

koningin

COATS AND LUGGAGE

kade

ers-

ten hoves hovestraa traatt

Rosarium

g alweg kanaalwe

nslag ag

de an johittlaan w

stadhoud

oginne

preside nt kenned ylaan

pansier- str.

erpsestr. antw

duin

nieu

jolstr stra aa att

inghar

24 wittlaan laan

dee d

elis

corn

g weeg

eg dyw ran

cornelis

straat

lag ijs

straat at

kerklaan

blei

cat sheu euv el

ze l-

franke

n klaa van der heimstra heimstraat at

siusstr. str.

straat at

st r .

55 28

seinpost ststr.

b ger p.s.

antonie hein ant

loo-

oldeenbarneveltlaan johan van old

at traat sstra

at straat

van

tr.. sstr ss

Need a job? Need to hire? Want to share information with the other participants? Post resumes, employment opportunities or information on the designated boards located near the registration desk.

nho

.d..goe a. v.d

JOB OPPORTUNITIES AND OTHER IMPORTANT INFORMATION

n va

viviens enstraa traatt

dijkveld

van

vij

paul ulus us buijsstraa buijsstraatt

laan

kenstraat

ikerik

at straat

n statenlaa

er ijg

Doornpark

ri end

fred

eise frederik frederik hendrikhendri kplein

Please go to the registration desk during the opening hours

a an ikla ndr hen

frankenslag

van

aat str

ningen

fran

t straa

PAYMENT QUERIES

g ag a

sl ken

kkbentinc

tr. sens aers van Statenkwartier 17

n statenlaa

st ra at

aerssenstraatt van aerssenstraa

maurits-

de van wee

van beu

aa t

ijg

fran

de ngka hari f. pr o

at cksstraat duyyck du antonie ant

Located on ground floor

tr de

kent 26 fran t straa traa sens 17 aers van lag kens fran

duplein in -

w duin est

rnstraat doo

z de

Gaëlle Jamar – Event Manager Vanessa Sampson – Payment & Membership

wil

eg inw

at traat schols

aat lstraa scholstr

CONGRESS OFFICE INFORMATION

westdu

g nwe tdui wes

oksstraat lstok koppelst

kinact nin a menstr

ill

Tel: +31-70-3525354 Fax: +31-70-3525353 Email: info@worldhotelbelair.com www.worldhotelbelair.com

lem

THE HAGUE HOSPITALITY INFORMATION DESK

lellykad dr. le e

Worldhotel Bel Air The Hague Johan de Wittlaan 30 2517 JR, The Hague

ven ven 2e Binnenha

aat astr douz

SPEAKER HOTEL INFORMATION:

Thursday 23 October 08.00–20.00 Friday 24 October 08.00–20.00 Saturday 25 October 08.00–20.00 Sunday 26 October 08.30–14.30

de ngka hari

REGISTRATION & INFORMATION DESK

lindoduin

Tel: +31 (0)70 306 63 66 Fax: +31 (0)70 306 64 43 Email: info@worldforum.nl www.worldforum.nl

tr. g

Gaëlle Jamar, Event Manager Tel: +44 7766 475379 email: office@esgct.eu eg kerweg schokkerw

World Forum Churchillplein 10 2517 JW The Hague The Netherlands

CONTACT NUMBER IN CASE OF EMERGENCY

a str een th da

VENUE INFORMATION

gro hovensentraa traat

https://www.facebook.com/esgct 1

FRONT COVER IMAGE: © WORLDFORUM BACK COVER IMAGE: LEFT: © AKSELI GALLEN-KALLELA: THE DEFENCE OF THE SAMPO (DETAIL), 1896. TEMPERA ON CANVAS. 122 X 125cm. TURKU ART MUSEUM. TOP RIGHT: (NÄKYMÄ TÖÖLÖNLAHDELTA): © EERO VENHOLA/FINLANDIA HALL. BOTTOM RIGHT: (FINLANDIA-TALO ITÄINEN JULKISIVU): © KATRI PYYNÖNEN/FINLANDIA HALL PRINTED BY PARK LANE PRESS ON FSC CERTIFIED PAPER, USING FULLY SUSTAINABLE, VEGETABLE OIL-BASED INKS, POWER FROM 100% RENEWABLE RESOURCES AND WATERLESS PRINTING TECHNOLOGY. PRINT PRODUCTION SYSTEMS REGISTERED TO ISO 14001: 2004, ISO 9001: 2008, EMAS STANDARDS AND OVER 97% OF WASTE IS RECYCLED BOOK DESIGN BY CATHERINE CHARNOCK CREATIVE: WWW.CATHERINECHARNOCK.CO.UK.

Hague_covers.indd 2

01/10/2014 10:14

PLATINUM PARTNERS

GOLD PARTNERS

SILVER PARTNERS

CONGRESS SUPPORTERS

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

CONTENTS 1

Partners â&#x20AC;&#x201C; overview

ESGCT Excellence Awards

Welcome address

6 Boards 7

Scientific committees

Programme at a glance

16 Partners 32

Exhibition hall

33 Posters 34 Exhibitors 40 Programme

Visiting The Hague

European Society of Gene and Cell Therapy Achievement Awards

ESGCT evaluation

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

ESGCT EXCELLENCE AWARDS We are delighted to present an exceptional field of award winners in 2014

Outstanding Achievement

Manuel Grez, Institute for Biomedical Research, Georg-Speyer-Haus, Frankfurt

Young Investigators

Volker Busskamp Centre for Regenerative Therapies, Dresden

Vincenzo Cerullo University of Helsinki

Travel Grants olychronis Fatouros, D P ZIF, Heidelberg University Hospital Pietro Genovese, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan Sonia Guedan Carrio, U niversity of Pennsylvania, Philadelphia

Johanna Laakkonen, U niversity of Eastern Finland, Kuopio

ico Lachmann, R EBIRTH Cluster-of Excellence, Hannover Medical School N Kathleen Börner, D ZIF, Heidelberg University Hospital

elia D’Avola, C D linica Universidad de Navarra, Pamplona Alba Rodríguez-García, I nstituto Catalan de Oncología-IDIBELL, L´Hospitalet de Llobregat aria Grazia Biferi, I nstitut de Myologie, INSERM-CNRS-UPMC-UMR 974, Paris M Camille Lévy, Université de Lyon-1; CIRI, Inserm U1111

WELCOME ADDRESS On behalf of the European and the Dutch Societies of Cell and Gene Therapy, we welcome you to the city of The Hague for the joint 2014 Congress. Europe continues to be at the forefront of translational medicine in the area of gene and cell therapy. The ESGCT Annual Congress is the leading arena for reporting latest progress, nurturing scientific exchange among scientists and clinicians, training young researchers and disseminating knowledge to our stakeholders and society at large. We are witnessing remarkable advances in the treatment of several diseases, such as hemophilia, immune-hematological and storage diseases as well as some types of cancer. Long-term safe and stable gene replacement has now been demonstrated in clinical trials with clear therapeutic benefits to the patients. The molecular and cellular mechanisms underlying these therapeutic benefits are increasingly understood by in-depth molecular follow up of human patients conducted with powerful new technologies, which provides unprecedented insights into complex pathophysiological processes, such as stem cell activity, tumor progression and the deployment of an immune response.

Scientists have shown the potential of targeted genome editing with an expanding toolbox of protein- and RNA-based engineered nucleases, vector engineering to modify tissue tropism and have refined the genetic design of integrating vectors to increase safety and improve regulation of transgene expression. T-cell engineering strategies are showing the therapeutic potential of adoptive therapy in cancer patients. These advances are increasing our confidence and making our research goals even more ambitious. We now aim to achieve: • stringently regulated expression of therapeutic transgenes, • correction rather than replacement of malfunctioning genes, • targeted delivery and lower toxicity of vector administration, • reduced immune activation and induction of tolerance to the transgene product, • improved engraftment of transplanted cells and tissue regeneration.

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

We work closely with Regulatory Authorities to improve the framework for the conduct of gene and cell therapy clinical trials. Most importantly, as gene therapy is finally coming of age, we welcome an increasing involvement of the pharmaceutical industry in our field. Only through a close engagement with industry we can orchestrate regulatory adjustments and make our therapies a clinical reality. This yearâ&#x20AC;&#x2122;s Congress will report exciting new findings in all the major areas of cell and gene therapy: cancer, infectious and inherited disease, cell reprogramming, stem cell biology and regenerative medicine, vector targeting, gene editing strategies and nanotechnologies, immune response to vector and cell transplantation, imaging, manufacturing and ethical and regulatory issues of gene and cell therapy. Latest results from several clinical trials will be presented. In total the Congress includes 98 speakers, 93 selected abstracts and 758 delegates. We hope that you will join the social events including the Welcome Reception and poster parties.

Finally, we are indebted to patientsâ&#x20AC;&#x2122; organisations, the EU, academic regional and corporate sponsors who supported us in the preparation, the management and also the costs of the Congress. Many of these organisations will be involved as exhibitors and we hope that you will take the time to visit them. We hope you will find the Congress as stimulating as we expect it to be, and that you will enjoy both the outstanding scientific program and your stay in the charming city of The Hague. Luigi Naldini ESGCT President Hidde Haisma ESGCT and NVGCT Boards Gerard Wagemaker NVGCT President

BOARDS ESGCT Board Luigi Naldini, Italy (President) Nathalie Cartier-Lacave, France (VicePresident) Christof von Kalle, Germany (Treasurer) Len Seymour, UK (General Secretary) Alessandro Aiuti, Italy

Robin Ali, UK Hildegard Büning, Germany Sarah Ferber, Israel Hidde Haisma, The Netherlands Zoltan Ivics, Germany Denis Vallese (co-opted member for young investigators)

NVGCT Board Gerard Wagemaker (President) Piter Bosma (Secretary) Jeroen de Vrij (Treasurer) Victor van Beusechem Winald Gerritsen Hidde Haisma

Mirjam Heemskerk Rob Hoeben Fons van de Loo Enrico Mastrobattista Margriet Vervoordeldonk

Local Organising Committee Gerard Wagemaker (President) Piter Bosma (Secretary) Jeroen de Vrij (Treasurer) Victor van Beusechem Winald Gerritsen Hidde Haisma

Organisers: Wats.on Consultancy: Renée Watson Gaëlle Jamar Vanessa Sampson Emma Clare

Mirjam Heemskerk Rob Hoeben Fons van de Loo Enrico Mastrobattista Margriet Vervoordeldonk Leonie Kaptein

With special thanks to

THE HAGUE CONVENTION BUREAU

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

SCIENTIFIC COMMITTEES Cardiovascular diseases

Non-viral vectors

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

Daniel Scherman (Chair) Hidde Haisma (Chair) Ernst Wagner J.P. Behr George Dickson Zoltan Ivics

Neurological and muscular diseases

Infection, immune and vaccines

Nicole Déglon (Chair) Maurilio Sampaolesi (Chair) Nathalie Cartier (Chair) Nicholas Mazarakis Dominic Wells Anders Björklund

David Klatzmann (Chair) Mary Collins (Chair) Naomi Taylor Ben Berkhout Dorothee Von Laer Zelig Eshhar

Viral vectors

Stem cells and reprogramming

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

Stefan Karlsson (Chair) Christopher Baum (Chair) Juan Bueren Tim O’Brien Catherine Verfaillie Katarina Le Blanc Marina Radrizzani Willem Fibbe

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

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

Please note: speakers and talk titles may change

PROGRAMME AT A GLANCE ESGCT/ ASGCT REGULATORY AND CLINICAL TRIALS WORKSHOP Mississippi Sponsor: ASGCT, Genosafe

09.00

Introduction, A lessandro Aiuti and Robin Ali

09.15

Planning a clinical trial

09.45

Manufacturing of gene and cell products

10.45

Overcoming barriers to gene and cell therapy trials part 1

11.25

Company showcase: five minute presentations by Terumo, Genosafe, Pall Life Sciences, Medgenics and Aldevron

12.00

Lunch and company posters

13.00

Overcoming barriers to gene and cell therapy trials part 2

14.00

Case studies

16.00

Discussions

P. 40

THURSDAY 23 OCTOBER 2014

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

THURSDAY 23 OCTOBER 2014 P. 42

EDUCATION AND PATIENTS’ DAY Sponsors: ZonMw, Dimension Therapeutics 09.30

Opening lecture Amazon 1: Lessons learned for therapy development for Duchenne Muscular Dystrophy Amazon

10.45

Morning break – Amazon foyer

11.15

2a: Technologies: general overviews evolutionary approaches Yangtze 2

12.15

Lunch: round table discussions – Amazon foyer

13.45

3a: Technologies: general overviews Yangtze 2

15.00

Closing Amazon

16.30

Afternoon break – Amazon foyer

2b: Technologies: selected topics Yangtze 1

3b: Technologies: selected topics Yangtze 1

2c: Patient perspectives: hemophilia & liver diseases Amazon 3c: Patient perspectives: acquired diseases Amazon

1: Highlights of clinical progress Auditorium Sponsor: Généthon

18.30

Welcome reception – Poster Session 1 Onyx room Sponsor: Human Gene Therapy

P. 45

MAIN CONGRESS 17.00

PROGRAMME AT A GLANCE FRIDAY 24 OCTOBER 2014 MAIN CONGRESS

2: Highlights of clinical progress II Auditorium Sponsor: SillaJen

10.00

Morning break

10.30

2a: AAV Vectors Auditorium Sponsor: 4D Molecular Therapeutics

12.30

ESGCT General Assembly – Auditorium

12.30

Lunch – Exhibition and posters

14.30

3: Emerging technologies in hematopoietic stem cell gene therapy Auditorium Sponsor: Editas Medicine

16.00

Afternoon break

16.30

3a: Genetic vaccines and Ad vectors Auditorium Sponsor: Omnia Biologics

18.30

Poster session 2 – Onyx room

20.00

Speakers’ Dinner – by invitation only Coaches will depart from the World Forum main entrance at 19.45

3b: Epigenetic regulation and gene editing I Mississippi Sponsor: Supersist

3c: Gene and cell therapy for ocular diseases Amazon Sponsor: Oxford BioMedica

3d: Genetic instability syndromes and vector genotoxicity Yangtze Sponsor: EuroFancoLen

P. 46

2d: Cardiovascular and pulmonary diseases Yangtze

P. 48

2c: Muscle gene and cell therapy Amazon Sponsor: AFM Telethon

P. 49

2b: Innate immunity and stem cell therapy Mississippi Sponsor: Avalanche Biotech

P. 46

08.30

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

SATURDAY 25 OCTOBER 2014 MAIN CONGRESS 4: Gene and cell therapy for cancer Auditorium Sponsor: bluebirdbio

10.00

Morning break

10.30

4a: RNA therapeutics Yangtze Sponsor: InCellArt

12.30

NVGCT General Assembly â&#x20AC;&#x201C; Auditorium

12.30

Lunch â&#x20AC;&#x201C; Exhibition and posters

14.00

5: New approaches to engineering vectors and cells Auditorium Sponsor: Sangamo

15.00

5a: Primary immune deficiencies Auditorium Sponsors: net4CGD, Cellpid

17.00

Afternoon break

17.30

6a: Regenerative medicine Amazon Sponsor: Pall Life Sciences

P. 52 P. 54

4d: Immunotherapy of cancer I Amazon Sponsor: GE Healthcare

5b: Gene and cell therapy for neural diseases Mississippi Sponsor: Oxford BioMedica

5c: RNA vectors Yangtze

5d: Bioprocessing of cell and gene therapy products Amazon Sponsor: Oxford BioMedica

6b: Inflammatory and autoimmune diseases Yangtze Sponsor: Arthrogen

6c: Oncolytic therapies Auditorium Sponsor: SAFC

6d: Immunotherapy of Cancer II Mississippi

P. 55

4c: Blood coagulation diseases Auditorium Sponsor: Dimension Therapeutics

P. 57

4b: Gene and cell therapy for metabolic diseases Mississippi Sponsor: uniQure

P. 52

08.30

PLEASE NOTE: At 2am last night the clocks turned back one hour for the end of Daylight Saving Time

PROGRAMME AT A GLANCE SUNDAY 26 OCTOBER 2014

7a: Exosomes and non-viral delivery Yangtze Sponsor: Arthrogen

7b: Infectious diseases Amazon Sponsor: BD Biosciences

7c: Gene Editing II Auditorium Sponsors: Supersist, bluebirdbio

7d: Hematopoietic stem cell and HSC gene therapy Mississippi Sponsor: bluebirdbio

11.00

Morning break

11.30

Presidential symposium and awards ceremony Auditorium Sponsors: IMI, EBiSC, StemBANCC, Généthon, Human Gene Therapy

P. 63

09.00

P. 60

MAIN CONGRESS

A unique combination of capabilities, premium development and manufacturing services Our broad range of experience and expertise is across every phase of the product development lifecycle â&#x20AC;&#x201C; from discovery, new product development, GMP manufacture & supply, to regulatory affairs and clinical trial management. As an integral part of our LentiVectorÂŽ platform, our in-house know-how and capabilities continue to drive delivery of our pipe-line and those of our key partners. It is this unique value proposition that makes Oxford BioMedica such a partner of choice to other companies working with Advanced Therapy Medicinal Products (ATMPs). oxfordbiomedica.com

Visit us at booth 4 and online at sigma-aldrich.com/safc

Building better solutions together for biopharmaceutical development and manufacturing

PARTNERS PLATINUM PARTNERS Bluebird bio is developing innovative gene therapies for severe genetic disorders. The companyâ&#x20AC;&#x2122;s proprietary platform treats genetic diseases by placing a functional gene into the patientâ&#x20AC;&#x2122;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 Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. The company was launched in 2013 by Fidelity Biosciences and REGENX Biosciences. Dimension has now raised $30M with funding from Fidelity Biosciences and OrbiMed. Our team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases. We are focused on advancing our platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics across a wide array of single-gene rare diseases. www.dimensiontx.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

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

GOLD PARTNERS Avalanche is a clinical-stage biotechnology company focused on discovering and developing novel gene therapies to transform the lives of patients with sight-threatening ophthalmic diseases. Using a next generation gene therapy platform, the Ocular BioFactory™, the Company is developing products designed to provide long-term benefit or a functional cure by inducing a sustained expression of a therapeutic protein with a one-time administration in the eye. www.avalanchebiotech.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 immune-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. www.cell-pid.eu Editas Medicine is a transformative genome editing company founded by world leaders in the fields of genome editing, protein engineering, and molecular and structural biology, with specific expertise in CRISPR/Cas9 and TALENs technologies. The company’s mission is to translate its genome editing technology into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level. The company has generated substantial patent filings and has access to intellectual property covering foundational genome editing technologies, as well as essential advancements and enablements that will uniquely allow the company to translate early findings into viable human therapeutic products. www.editasmedicine.com 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.Généthon.fr

GOLD PARTNERS 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 The Innovative Medicines Initiative (IMI) is working to improve health by speeding up the development of, and patient access to, innovative medicines, particularly in areas where there is an unmet medical or social need. It does this by facilitating collaboration between the key players involved in healthcare research, including universities, the pharmaceutical and other industries, small and medium-sized enterprises (SMEs), patient organisations, and medicines regulators. IMI is a partnership between the European Union and the European pharmaceutical industry, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA). IMI has a budget of €3.3 billion for the period 2014-2024. Half of this comes from the EU. The other half comes from large companies, mostly from the pharmaceutical sector; these do not receive any EU funding, but contribute to the projects ‘in kind’, for example by donating their researchers’ time or providing access to research facilities or resources. www.imi.europa.eu

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

GOLD PARTNERS StemBANCC is a large-scale, 5 year academic-industry partnership in the area of stem cell research (2012 â&#x20AC;&#x201C; 2017) with a budget in excess of 55 million EUR. It is a collaborative project between pharmaceutical companies, research institutions and small and medium enterprises (SMEs) to exploit the rich expertise and enhance knowledge transfer between academia and industry for patient benefit. The aim of the StemBANCC is to generate and characterise 1 500 high-quality human induced pluripotent stem cell lines that can be used by researchers to study a range of diseases, including diabetes and dementia. The project will also investigate the use of human iPS cells for toxicology testing; here the team will use the iPS cells to generate liver, heart, nerve and kidney cells. The cell lines will help to improve and speed up the drug development process, and ensure that patients benefit from more effective and safer drugs. www.stembancc.org EBiSC (European Bank for induced pluripotent Stem Cells) is a large European public private partnership project supported jointly by the Innovative Medicine Initiative and members of the European Federation of Pharmaceutical Industries and Associations (EFPIA). EBiSC is designed to address the increasing demand by iPSC researchers for quality-controlled, disease-relevant research grade iPSC lines, data and cell services. Its goal is to demonstrate an operational banking and distribution service of iPSC lines after 3 years and to establish subsequently for Europe a centralised, not-for-profit bank providing all qualified users with access to scalable, cost-efficient and customised products. Led by Pfizer Ltd and managed by Roslin Cells, the EBiSC Consortium including EFPIA partners boasts the leadership, scientific expertise, facilities, networks and experience to achieve these goals and, being representative of all stakeholders from tissue donors to clinical and academic iPSC researchers and industrial users, to respond appropriately to advances in science and society. www.ebisc.eu The goal of Supersist project is the clinical translation of new gene targeting technologies for correcting inherited mutations and empowering adoptive immunotherapy of cancer. Substantial evidence supports the therapeutic potential of ex vivo gene therapy based on Hematopoietic Stem Cell (HSC) or T lymphocytes to treat inherited diseases or cancer. www.supersist-project.eu

SILVER PARTNERS AFM (French Muscular Dystrophy Association) has a single objective: to defeat neuromuscular diseases, which are devastating muscle-wasting diseases. Created in 1958 by a group of patients and their families, and recognised as being of public utility in 1976, it has set itself two missions: curing neuromuscular diseases and reducing the disabilities they cause. www.afm-france.org BD Biosciences is a world leader in bringing innovative diagnostic and research tools to life science researchers, clinical researchers, laboratory professionals and clinicians who are involved in basic research, drug discovery and development, biopharmaceutical production and disease management. The BD Biosciences segment is focused on continually advancing the science and applications associated with cellular analysis and products that help grow living cells and tissue. Products/Services: 1) Fluorescence-activated cell sorters and analysers; 2) Monoclonal antibodies and kits for cell analysis; 3) Reagent systems for life science research; 4) Cell culture media and supplements for biopharmaceutical manufacturing. www.bdbiosciences.com SAFC is a trusted manufacturer of specialty chemicals and biologics for commercial life science applications. We provide unique and innovative technologies and services for customers requiring a reliable partner throughout the development and manufacturing process. SAFC and BioReliance offer world-class process development, manufacturing and testing capabilities for virus based therapeutic products. www.bioreliance.com www.sigmaaldrich.com/safc

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

SILVER PARTNERS EuroFancoLen is a collaborative project with an innovative approach to developing, for the first time, an efficient and safe gene therapy of FA based on two recent innovations: 1) Discovery of potent HSC mobilisers, such as plerixafor 2) Development of a new lentiviral vector by members of this Consortium, designed as Orphan Drug by the Europan Commission in December 2010. The main objective of this project is, therefore, the development of a multicentric Phase I/II gene therapy trial for FA-A patients, based on the genetic correction of plerixafor+G-CSF mobilised HSCs with the novel lentiviral vector, accompanied by comprehensive and groundbreaking safety and efficacy patient monitoring studies. www.eurofancolen.eu 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 GenoSafe is a CSO specialised in the evaluation of the quality, efficacy and safety of gene and cell therapy products. We support through research stages to final clinical phases: from study design, development/validation of analytical methods, and product testing to control of viral vectors batches (rAAV, rHIV, rMLV), preclinical evaluation, clinical trial and finally, patient follow-up. www.genosafe.org

SILVER PARTNERS In-Cell-Art is a French biopharmaceutical firm specialising in the development of Nanotaxi® for macromolecule (DNA, mRNA). Its founder including a Nobel laureate designed new classes of vectors which enable them to cross the cell barrier safely and efficiently. One of our objectives is to tap into the potential of messenger RNA to produce proteins at reduced time and costs. In-Cell-Art made several proofs of concepts demonstrating Nanotaxi® can deliver mRNA inside cells and overcome its limited efficacy and stability by not only dramatically increasing the level of expression but also modulating it to restore physiological condition. For our partners, In-Cell-Art can bring its highly technical competencies and unique resources in the discipline of nucleic acid delivery which have been developed over 20 years of research, and specific patents for formulations and targeting of nucleic acids to host cells in a controlled manner. www.incellart.com Net4CGD, Gene Therapy for X-linked Chronic Granulomatous Disease (CGD), is a large-scale integrating project in the health research of the European commission 7th Framework Programme (FP7). The Net4CGD project is focused on the clinical development of a new orphan drug of gene therapy for X-linked form of CGD to become a new treatment option for patients. The Net4CGD consortium consists of 11 partners institutions and is coordinated by Genethon, France. www.net4cgd.eu Pall Life Sciences is a global leader in enabling process materials and process technology for semiconductor, display and life science industries. At Pall Life Sciences, process ingenuity unleashes new process possibilities for customers. www.pall.com PlasmidFactory is Europe’s leading contract manufacturer for plasmid DNA. Production ranges from research to industrial scale. We produce plasmids in modern laboratories to the highest quality of standards and according to your individual wishes. www.PlasmidFactory.com

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

SILVER PARTNERS 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 uniQure is the world leader in the development of human gene based therapies. uniQure’s Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure’s product pipeline of gene therapy products in development comprise of hemophilia B, acute intermittent porphyria, Parkinson’s disease and SanfilippoB. Using adenoassociated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design the world’s first stable and scalable AAV manufacturing platform, validated in the approval process of Glybera. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene. www.uniqure.com ZonMw – The Netherlands Organisation for Health Research and Development. Progress requires research and development. ZonMw funds health research and stimulates use of the knowledge developed to help improve health and healthcare in the Netherlands. www.zonmw.nl

CONGRESS SUPPORTERS The 4D vision is to design, develop and commercialise transformative gene therapeutic products from our proprietary “directed evolution” AAV variants platform for serious unmet medical conditions in partnership with complimentary biopharmaceutical companies. www.4dmoleculartherapeutics.com AAV Life’s lead indication is for Friedreich’s Ataxia Cardiomyopathy. Our program is based on the successful use of a gene-therapy approach in a mouse model developed to mimic the gene defect and cardiac symptoms of Friedreich’s Ataxia. The approach made use of an adeno-associated virus (AAV) to introduce a normal gene into cardiac tissue. The therapy restored cardiac function and reversed pathological heart enlargement in mice that had already progressed to heart failure. www.aavlife.com Aldevron provides custom plasmid DNA manufacturing to support many gene and cell therapy applications. With our experience and scientific expertise, we have developed screening strategies to optimize yield and stability of even the most challenging vector plasmids. We also provide consistency at all levels, with a deliberate pathway from research to clinical requirements. Visit with our team to learn about our additional services including, mRNA, protein production, gene synthesis and antibody development. Aldevron’s mission is to advance science, and we have solutions that are designed for you. www.aldevron.com Arthrogen BV is a biotechnology company focused on development of innovative gene therapy approaches for the treatment of rheumatoid arthritis (RA) and other immune mediated diseases. Arthrogen is particularly focusing on disease-regulated expression of therapeutic genes using adeno-associated virus (AAV) as vector for local delivery in the joint. One lead compound is currently in pre-clinical development and the phase Ib clinical trial is planned for 2015. www.arthrogen.nl

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

CONGRESS SUPPORTERS The demographic change poses a challenge for modern societies due to the increased risk of aging-associated diseases. In this context, it is CECAD’s mission to define the molecular mechanisms underlying lifespan regulation as potential novel targets for prevention, diagnosis, and treatment of aging-related damage and pathology. CECAD encompasses six reasearch areas which cover a diverse but closely interlinked range of topics that engage a broad spectrum of model organisms. Furthermore, it unifies over 40 research groups from the University of Cologne, the University Hospital, the MPI for Biology of Aging, the MPI for Metabolism Research and the German Center for Neurodegenerative Diseases. The research areas are supported by three platforms ensuring (1) access to state-of-the-art technology, (2) coordination of education and training activities, gender equality programs, and public outreach and (3) translation of basic to clinical research. CECAD is funded by the DFG within the Excellence Initiative. www.cecad.uni-koeln.de 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. www.genzyme.com Built around independent modules that can interact with each other, LabCollector LIMS can manage a variety of day-to-day useful lab information and samples in any kind of laboratory: biobanks, life sciences R&D, chemistry… Scientists can also expand LabCollector by choosing additional extensions from our catalog such as an Electronic Lab Notebook (ELN), a data/Temperature logger, a Wokflow Manager and many more. www.labcollector.com

CONGRESS SUPPORTERS Omnia offers translational medicine services in the areas of process development, assay development, GMP manufacture, and fill-finish for preclinical and clinical programs for therapeutics that do not have effective or known manufacturing protocols. These include vaccines, autologous therapeutics, new gene therapeutic platforms, and stem cell technologies. The company seeks and has established relationships with both biopharmaceutical companies and academic investigators throughout the world. www.omniabiologics.com Promethera Biosciences develops innovative therapies for the treatment of liver disease. It is currently developing two products based on a recently discovered and patented cell, the Heterologous Human Adult Liver Progenitor Cells (HHALPC): • Promethera® HepaStem is a cell therapy product based on the use of allogeneic stem cells isolated from healthy adult human livers. These cells can be used to treat a wide variety of liver diseases, from rare inborn metabolic diseases, (which can be classified as ‘orphan diseases’ and mainly affecting children), to acquired deficiencies affecting adults. Promethera® HepaStem is currently in clinical development in paediatric patients suffering from orphan diseases such Crigler-Najjar Syndrome and Urea Cycle Disorders. • Promethera® HepaScreen is a different, non-therapeutic product that uses the same adult human liver-derived stem cells as a biotechnology tool designed for evaluation of new chemical entities. This product meets a real need in the market, which currently lacks sufficiently predictive models to assess the hepatic metabolism of chemical compounds in the body. This unique cell model will be made available to the pharmaceutical industry to evaluate the metabolism and toxicity of new drugs in humans in a more reliable way than animal experimentation, thereby reducing the need and number of animals for such experiments. www.promethera.com

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

CONGRESS SUPPORTERS The REBIRTH Cluster of Excellence (From Regenerative Biology to Reconstructive Therapy) is an internationally renowned institution for regenerative medicine. REBIRTH is funded since 2006 by the Excellence Initiative of the German Federal and State Governments. A network of over 30 institutes with 60 different work groups and over 250 researchers are developing gene- and cell-based therapeutic strategies for diseases of the heart, lungs, liver and blood through interdisciplinary collaboration between the various scientific disciplines integrated within REBIRTH. By drawing on the knowledge gained from fundamental research conducted within REBIRTH and its translation into experimental medicine, and by identifying relevant mechanisms involved in regenerative processes in the human body REBIRTH aims to develop new approaches and technologies for medical use. These will be applied in everyday clinical routine for the benefit of patients implemented for example at Hannover Medical School. www.rebirth-hannover.de REGENXBIOâ&#x20AC;&#x2122;s NAVÂŽ Technology has ushered in a new era of gene therapy innovation. We are leveraging our proprietary NAV Technology and collaborating with world-class clinical advisors to advance the development of life-changing gene therapy treatments for lysosomal storage disorders and ocular diseases. Beyond these core areas of focus, we are licensing NAV Technology to enable global strategic partners to advance gene therapy programs for other serious diseases with unmet needs, including in diseases such as hemophilia, Pompe disease and spinal muscular atrophy. www.regenxbio.com SillaJen Inc (Busan and Seoul, S Korea) is the global leader in the development of oncolytic vaccinia immunotherapy with Pexa-Vec (aka JX-594; previously Jennerex Inc) for primary hepatocellular carcinoma (HCC). We reported IV delivery of Pexa-Vec to diverse solid tumors (Nature 2011), and improved overall survival in a randomised dose-finding clinical trial (Nature Med 2013). Initiation of a Phase 3 clinical trial is planned for 2015. Patients with advanced first-line HCC will be randomised to treatment with Pexa-Vec followed by sorafenib versus sorafenib alone, with a primary endpoint of overall survival duration. www.sillajen.com

bluebird bio is proud to support the ESGCT and its members.

TRANSFORMING THE LIVES OF PATIENTS WITH SEVERE GENETIC AND ORPHAN DISEASES

bluebird bio (NASDAQ: BLUE) has two clinical-stage products in development, one for childhood cerebral adrenoleukodystrophy (CCALD) and another for both beta-thalassemia and sickle cell disease. We also have an early-stage chimeric antigen receptor-modified T cell (CAR-T) program for oncology in collaboration with Celgene Corporation, and as a result of our recent acquisition of Pregenen, we now have access to gene editing technologies that have broad potential application in the gene therapy and cancer immunotherapy fields. Please visit us at www.bluebirdbio.com to learn more about participating in clinical studies, to explore avenues for scientific collaboration or to view opportunities to join our growing team. Follow us on Twitter (@bluebirdbio) and YouTube.

RNAdestination™ Art to connect messenger RNA and novel biotherapeutics

In-Cell-Art gives a short presentation at S4a: RNA Therapeutics session on

25th OCT from 10.30–12.30 In-Cell-Art is a French biopharmaceutical company specializing in the development of Nanotaxi® for macromolecular drugs (DNA, mRNA). Its founder and research team including a Nobel laureate designed new classes of vectors organized on a nanometric scale, which enable them to cross the cell barrier safely and efficiently. In-Cell-Art made several proof of concepts demonstrating Nanotaxi® can deliver messenger RNA inside cells and overcome its limited efficacy by not only dramatically increasing the level of expression but also modulating it to restore physiological conditions. This could also help to avoid unwanted adverse effect of uncontrolled protein expression as observe when delivering DNA molecules. Our scientific team accumulates expertise in mRNA formulation, working in RN-ARMORVAX

134x196_livrecongres.indd 1

with Sanofi Pasteur and CureVac. Its objective is to develop a new universal mRNA-based vaccine technology platform with budget of US$ 33.1 million co-financed by the USA Defense Advanced Research Projects Agency. For our partners, In-Cell-Art can bring its highly technical competencies and unique resources in the discipline of nucleic acid delivery which have been developed over 20 years of research, and specific patents for proprietary formulations and targeting of nucleic acids to host cells in a controlled manner.

T +33 (0)2 40 71 67 17 • Fax +33 (0)2 72 88 22 01 info@incellart.com • www.incellart.com

09/09/14 17:33

Expedite Stem Cell analysis with BD Accuri™ sytems As Easy as Stem Cell Analysis Is Going to Get Determine the pluripotency and differentiation of stem cell populations as well as their proliferation and apoptosis with the powerful BD Accuri C6 platform and ready-to-go BD reagent kits. Predefined settings matched to the BD kits shorten the workflow and increase the consistency of results in your stem cell research. BD Biosciences Europe bd_accuri@europe.bd.com bdbiosciences.com/eu Class 1 Laser Product. For Research Use Only. Not for use in diagnostic or therapeutic procedures. BD, BD Logo and all other trademarks are property of Becton, Dickinson and Company. © 2014 BD A608-00

Gene Therapy for Rare Diseases “We are passionate in our quest to serve patients and value our relationship with patient communities and the organizations that serve them.” Amber Salzman, CEO AAVLife - 183 Avenue de Choisy 75013 Paris - France

+33.970.468.222

amber@aavlife.com

Xuri . Believe in better futures. ™

Finding solutions. Advancing breakthrough cell therapies. Xuri products deliver solutions to accelerate access of safe and ground-breaking treatments, supporting the transition from research to clinic with confidence. Making cellular therapies a clinical reality. www.gelifesciences.com/xuri

GE and GE monogram are trademarks of General Electric Company. Xuri is a trademark of General Electric Company or one of its subsidiaries. © 2014 General Electric Company – All rights reserved. First published Sep. 2014. GE Healthcare UK Limited, Amersham Place, Little Chalfont, Buckinghamshire, HP7 9NA, UK 09/2014

EXHIBITION HALL 1

P 1-50 P 51-100 P 101-150 P 151-197

Coffee/Tea Catering

21 32

Gene Therapy Office

HELSINKI TOURISM BOARD

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

POSTERS POSTERS BY CATEGORY

SESSION

P001–P008

Cancer

P009–P025

Cardiovascular, lysosomes and metabolic disorders

P026–P045

Gene and cell targeting

P046–P064

Non-viral vectors

P065–P071

Regenerative medicine and tissue engineering

P072–P077

RNA therapeutics

P078–P103

Viral vectors

P104–P127

Immunotherapy and autoimmunity

P128–P132

Insertional mutagenesis

P133–P145

Manufacturing, bioprocessing, regulation and ethics

P146–P164

Ocular and neural disorders

P165–P187

Single gene disorders

P188–P197

Stem cells and transdifferentiation

EXHIBITORS Booth 1: L onza offers world class technology platforms in the areas of GMP cell culture and viral-based therapeutic manufacturing, custom biotherapeutic culture media, a large selection of primary and stem cells and a full line of custom bioassays. Our extensive experience in cell therapy process optimisation and scale-up innovation helps clients to safely and effectively advance their products through all phases of the commercial pipeline and maximise their return on investment. Our new viral-based therapeutics group provides viral vaccine manufacturing as well as viral vector mediated gene therapies. Our staff can design, develop, and implement a manufacturing process that meets your autologous or allogeneic therapeutic applications. www.lonza.com Booth 2: P all Life Sciences: see page 22 www.pall.com Booth 3: T erumo BCT, a global leader in blood component, therapeutic apheresis and cellular technologies, is the only company with the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction. We believe in the potential of blood to do even more for patients than it does today. This belief inspires our innovation and strengthens our collaboration with customer. www.terumobct.com Booth 4: S AFC: see page 20 www.sigmaaldrich.com/safc Booth 5: C EVEC has developed the human amniocyte based expression platform CAP速 for the transient and stable production of superior Biopharmaceuticals. We offer CAP速 based cell line development as well as cGMP contract manufacturing services. The technology is additionally available for inhouse-use under flexible licensing terms. CAP速 cells are immortalised suspension cells of non-tumorous origin and have been adapted to chemically defined, animal component free culture conditions. Complex proteins and biomolecules derived from CAP can be produced at high yields and exhibit authentic human glycosylation patterns. The platform is also a versatile production system for gene therapy vectors (e.g. Lentivirus and Adenovirus), viruses and vaccines. www.cevec.com

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

EXHIBITORS Booth 6: O xford Genetics specialises in creating DNA plasmids and components that are fully inter-compatible. We provide a unique range of DNA products that make genetic engineering easier and more efficient, whilst also offering both custom cloning and DNA synthesis. Whatever your cloning requirements, we’ve got all bases covered. www.oxfordgenetics.com Booth 7: M iltenyi Biotec ‘From Bench to bedside’ Miltenyi Biotec is Germany‘s largest independent, privately owned Biotech company. Since pioneering MACS magnetic cell separation technology in 1990, we have grown into a vibrant, multinational team of more than 1200 biomedical scientists, physicians, engineers, and support groups. We develop and manufacture a portfolio of outstanding products ranging from unique cell labeling reagents, through sophisticated cell separation and analysis devices, to innovative systems for clinical applications. From research tools to GMP reagents for sophisticated applications, such as cellular therapy, the creativity of our interdisciplinary teams is reflected in the excellence of our products. www.miltenyibiotec.com Booth 8: G énéthon: see page 17 www.genethon.fr Booth 9: G enosafe: see page 21 www.genosafe.org Booth 10: PlasmidFactory: see page 22 www.plasmidfactory.com

Booth 11: E UFETS, 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 35

EXHIBITORS Booth 12: W ith 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 â&#x20AC;&#x201C; and it is cost effective â&#x20AC;&#x201C; to concentrate all analytical expertise on one site. www.quality-assistance.com Booth 13: IMI: see page 18 www.imi.europa.eu

Booth 14: InCellArt: see page 22 www.incellart.com

Booth 15: H elsinki Tourism Board. www.visithelsinki.fi Booth 16: G E Healthcare: see page 21 www.gelifesciences.com

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

EXHIBITORS Booth 17: P eproTech was established in 1988 by a group of scientists who decided to focus their efforts on the development and production of recombinant cytokines for life-science research. Today, PeproTech is a world leader in supplying high quality cytokine products including E. coli, insect, and mammalian cell-derived recombinant proteins, their monoclonal/ polyclonal antibodies, ELI SA development kits, and other cytokine-related reagents. www.peprotechec.com Booth 18: T he 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 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. www.med.unc.edu/genetherapy/vectorcore Booth 19: C ellGenix is an international leading manufacturer and supplier of high quality cytokines and serum-free medium for the ex vivo cell culture of DC, T-cells, NK-cells, hematopoietic stem cells, MSC, chondrocytes, ESC and iPS. CellGenix products are used world wide in clinical trials in academia, commercial trials, production of vaccine and in translation, validation and testing or assay development by biotechnology partners. The manufacturing is in accordance with GMP guidelines and USP. www.cellgenix.com Booth 20: H uman Gene Therapy: see page 18 www.liebertpub.com/hum

EXHIBITORS Booth 21: I zon Science designs and manufactures precision instrumentation for nano- and micro-scale particle analysis. The instrument systems, the qNano, qViro-X and qMicro, are now in use in a wide range of research institutes and universities around the world. The basis of Izon’s instrumentation is a unique, dynamically tunable nanopore technology platform which utilises a technology called TRPS (Tunable Resistive Pulse Sensing). This technology platform is world-leading in offering an affordable, robust and highly flexible technology for real-time particle detection, quantitation and characterisation of individual particles at the nanoscale, across a wide range of applications. TRPS represents a new paradigm in nanoparticle analysis, offering a unique alternative to the existing laserbased technologies available on the market. It offers applications not previously available to researchers and at a fraction of the cost of the incumbent technologies. Izon Science Europe is a Limited Liability Company registered in in the UK, with private shareholding. For further information please contact Hans van der Voorn (Executive Chairman) via info@izon.com www.izon.com

Gene Therapy Office

Booth 22: T he aim of the Gene Therapy Office is to streamline the licensing and permit granting procedures for clinical gene therapy studies in The Netherlands and to provide more insight into the procedures for investigators. The Gene Therapy Office serves as a central contact point between investigators and the different regulatory organisations. It is primarily a service for professionals conducting clinical trials on gene therapy. The Gene Therapy Office comes into play as soon as you want to carry out a clinical trial on gene therapy. Although there are currently various definitions of gene therapy, the Gene Therapy Office defines it as follows: ‘Clinical research in humans either involving activities with genetically modified organisms (GMO) or whereby changes are made to the genetic material of human cells’. www.loketgentherapie.nl Booth 23: B D Biosciences: see page 20 www.bdbiosciences.com

Booth 24: R OKEPIE® is part of Sulfateq BV, a growing company that developed a revolutionairy technology based on hibernation. Our first market ready RUO-product is ROKEPIE®-S01. It enables a hibernation-like state of cells making preservation at 2-8° C possible. Cells and possibly tissues can thus be stored or transported under hypothermic conditions. More information and free sample at booth 24. 38

www.rokepie.com

Targeting unmet global healthcare needs Partnering and licensing opportunities Using our LentiVector速 technology, one of the leading gene delivery systems available, we are focused on developing high quality gene-based medicines with broad-based and high-growth market appeal. Our product candidates target unmet and poorly treated disease areas, such as ocular and central nervous system disorders. These products fit right into the rapidly changing healthcare landscape as payers and policy-makers look towards safer and more effective treatments that will make a significant impact on reducing the socioeconomic pressures resulting from increasingly ageing populations. Oxford BioMedica is committed to advancing the wider development of gene and cell therapies by offering licences under its LentiVector速 platform IP. For more information please contact the business development team at licensing@oxfordbiomedica.co.uk A valued partner With world class expertise and extensive know-how in discovery, new product development, process scale-up, GMP manufacturing & supply to regulatory strategy complemented by a broad range of analytical capabilities, we have emerged as a valued partner of choice for companies working with Advanced Therapy Medicinal Products (ATMPs). oxfordbiomedica.com

PROGRAMME THURSDAY 23 OCTOBER 2014 ESGCT/ ASGCT REGULATORY AND CLINICAL TRIALS WORKSHOP Mississippi Chairs: Robin Ali, Alessandro Aiuti 09.00

Introduction, A lessandro Aiuti and Robin Ali

09.15

Planning a clinical trial INV020 Planning an academic clinical trial Laura Clifton-Hadley, CR UK and UCL Cancer Trials Centre, London

09.45

Manufacturing of gene and cell products INV021 From early-phase clinical trials to market: the product manufacturing challenge Fulvio Mavilio, Généthon, Evry

10.15

INV022 Clinical bio-manufacturing facility Eleanor Berrie, University of Oxford

10.45

Overcoming barriers to gene and cell therapy trials part 1 INV023 Running a trial through a spin out – Cancer Gene Therapy Len Seymour, University of Oxford

11.05

INV024 Running a trial in an academic institution: TIGET Rare Diseases Giuliana Ferrari, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan

11.25

Company showcase: five minute presentations by Terumo, Genosafe, Pall Life Sciences, Medgenics and Aldevron

12.00

Lunch and company posters

13.00

Overcoming barriers to gene and cell therapy trials part 2 INV026 Gene and cell therapy clinical trials: ethical and regulatory view Sol Ruiz, CAT, Spanish Medicines Agency (AEMPS), Madrid

13.30

INV025 Immunological and metabolic correction after lentiviral vector mediated hematopoietic stem cell gene therapy for ADA deficiency Bobby Gaspar, UCL Institute of Child Health, London

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME THURSDAY 23 OCTOBER 2014 ESGCT/ ASGCT REGULATORY AND CLINICAL TRIALS WORKSHOP Mississippi Chairs: Robin Ali, Alessandro Aiuti 14.00

Case studies INV028 and INV029 Case study Chiesi Andrea Chiesi, Chiesi Group and Graziella Pellegrini, Centre for Regenerative Medicine ‘Stefano Ferrari’, Moden

14.40

INV030 Genome editing with zinc finger nucleases Phil Gregory, Sangamo BioSciences, Inc, Richmond, CA

15.20

INV031 Gene therapy with alipogene tiparvovec (Glybera®) reduces pancreatitis incidence in patients with lipoprotein lipase deficiency Deya Corzo, uniQure BV, Amsterdam

16.00

Discussions

PROGRAMME THURSDAY 23 OCTOBER 2014 EDUCATION AND PATIENTS’ DAY

09.30 Amazon

Opening lecture Chairs: Len Seymour, Sam Wadsworth Opening and welcome Sam Wadsworth, Dimension Therapeutics Overview of the current state of the art in gene and cell therapy Len Seymour, University of Oxford

Amazon

Education session 1 INV001 and INV002 Lessons learned for therapy development for Duchenne Muscular Dystrophy Annemieke Aartsma-Rus, Leiden University Medical Centre; Elizabeth Vroom, Duchenne Parent Project Netherlands

10.45

Morning break – Amazon foyer

11.15

Education session 2a: Technologies: general overviews evolutionary approaches Yangtze 2 Chair: Hildegard Büning

Education session 2b: Technologies: selected topics Yangtze 1 Chair: Mirjam Heemskerk

Education session 2c: Patient perspectives: hemophilia & liver diseases Amazon Chair: Piter Bosma

INV003 Viruses with good intentions: developing oncolytic agents for therapy of cancer Rob Hoeben, Leiden University Medical Centre

INV005 Redirecting T-cells by chimeric antigen receptors Hinrich Abken, University of Cologne

INV007 Clinical advances in gene therapy from bench to bedside: a patient’s perspective Adam Jones, University of Sunderland

INV004 Directed evolution approaches – a powerful technology to improve Adenoassociated viral vectors Hildegard Büning, University of Cologne

INV006 Genetic information exchange by vesicles Michiel Pegtel, VU University, Amsterdam

INV008 Gene therapy for hemophilia Frank Leebeek, Erasmus Medical Centre, Rotterdam

12.15

Lunch: round table discussions – Amazon foyer

INV009 Towards a cure for Crigler-Najjar syndrome: a collaborate effort of researchers and patients Piter Bosma, AMC Hospital, Amsterdam

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME THURSDAY 23 OCTOBER 2014 EDUCATION AND PATIENTS’ DAY 13.45

15.00 Amazon

Education session 3a: Technologies: general overviews Yangtze 2 Chair: Angelo Lombardo

Education session 3b: Technologies: selected topics Yangtze 1 Chairs: Axel Schambach, Claudia Waskow

Education session 3c: Patient perspectives: acquired diseases Amazon Chair: Rob Hoeben

INV010 Exploiting artificial nucleases for targeted genome editing in human cells Angelo Lombardo, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan

INV012 Opening-up the stem cell niche for human hematopoietic stem cells in the mouse – an optimal model to study human hematopoiesis in vivo. Claudia Waskow, Technische Universität Dresden

INV014 Oncolytic adenoviral therapy to enhance the cure rate for patients with localised prostate cancer Chris Bangma, Erasmus Medical Centre, Rotterdam

INV011 Gene expression switches Ben Berkhout, University of Amsterdam

INV013 Retro- and lentiviral vectors for gene therapy: from basic biology to clinical application Axel Schambach, Hannover Medical School

INV015 Gene therapy for arthritis Margriet Vervoordeldonk, Arthrogen BV, Amsterdam

Closing Chair: Len Seymour INV016 Cystic Fibrosis patients taking the lead Jacquelien Noordhoek, NCFS Dutch Cystic Fibrosis Foundation, Baarn; VU University, Amsterdam INV017 Organoid cultures for developing Cystic Fibrosis therapies Jeffrey Beekman, University Medical Centre, Utrecht INV018 European Union support to gene and cell therapy research in Horizon 2020 (2014–2020) David Gancberg, European Commission, Brussels INV019 Writing a Horizon 2020 application: do’s and don’ts Ellen Schenk, Erasmus Medical Centre, Rotterdam

16.30

Afternoon break – Amazon foyer 43

PROUD SPONSOR OF

European Society of Gene & Cell Therapy Conference

Dimension Therapeutics is a gene therapy company developing novel treatments for rare diseases. Dimension is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics across a wide array of single-gene rare diseases.

For more information about Dimension, please visit

www.dimensiontx.com

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME THURSDAY 23 OCTOBER 2014 MAIN CONGRESS 17.00

1: Highlights of clinical progress

Auditorium

Chairs: Luigi Naldini, Fulvio Mavilio Opening and welcome Gerard Wagemaker, Erasmus Medical Center, Rotterdam INV032 Hematopoietic stem cell gene therapy for lysosomal storage disorders Alessandra Biffi, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan INV033 Immunological and metabolic correction after lentiviral vector mediated hematopoietic stem cell gene therapy for ADA deficiency Bobby Gaspar, UCL Institute of Child Health, London INV034 Clinical progress update Marina Cavazzana, Hôpital Universitaire Necker – Enfants Malades, Paris INV035 Gene therapy for Wiskott-Aldrich Syndrome Alessandro Aiuti, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan

18.30

Welcome reception – Poster Session 1 Onyx room Posters 1–103. See page 33 for details

PROGRAMME FRIDAY 24 OCTOBER 2014 MAIN CONGRESS 08.30

2: Highlights of clinical progress II

Auditorium

Chairs: David Kirn, Len Seymour INV036 Targeted oncolytic and immunotherapeutic viruses: emerging multi-mechanistic biologics for cancer David Kirn, 4D Molecular Therapeutics; SillaJen, San Francisco, CA INV037 Enadenotucirev, a group B oncolytic adenovirus: assessment of potency, safety and selectivity Kerry Fisher, PsiOxus Therapeutics, Oxford INV038 What T-cells see on human cancer Ton Schumacher, The Netherlands Cancer Institute, Amsterdam

10.00

Morning break

10.30

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

Auditorium

2a: AAV Vectors Chairs: Hildegard Büning, David Schaffer INV039 Directed evolution of new adeno-associated viruses for therapeutic gene delivery David Schaffer, 4D Molecular Therapeutics, San Francisco, CA; University of California, Berkeley INV040 Pre-existing immunity to AAV overcome by in silico ancestral capsid design Luk Vandenberghe, Harvard Medical School, Boston, MA Proffered papers OR084 Off-target-free gene delivery to clinically relevant cell types by receptor-targeted adeno-associated viral vectors Robert Münch, Paul-Ehrlich-Institut, Langen OR085 Capsid-engineering overcomes barriers toward endothelial cell transduction Li-Ang Zhang, University of Cologne OR086 Successful repeated hepatic gene delivery in mice and non-human primates achieved by sequential administration of AAV2/5 and AAV1 vector serotypes Anna Majowicz, uniQure BV, Amsterdam; CIMA, University of Navarra, Pamplona OR087 Exhaustive characterisation of DNA contaminants in rAAV productions by next generation sequencing Benjamin Cogné, University of Nantes; INSERM, UMR 1089, Nantes

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME FRIDAY 24 OCTOBER 2014 10.30 Mississippi

2b: Innate immunity and stem cell therapy Chairs: Federico Mingozzi, Axel Schambach, Thomas Chalberg INV041 The great escape: immune evasion by EpsteinBarr virus Maaike Ressing, Leiden University Medical Centre INV042 Adeno-associated viral (AAV) vectors are the most promising platform for in vivo gene transfer Federico Mingozzi, Généthon, Evry Proffered papers OR005 Stimulated granulocyte differentiation in an induced pluripotent stem cell model of severe congenital neutropenia by vitamin B3 Dirk Hoffmann, Hannover Medical School OR006 Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatous disease Giada Farinelli, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR007 Ectopic gp91phox expression is detrimental to XCGD iPS cell-derived neutrophils Huan-Ting Lin, University of Tokyo OR008 Large-scale hematopoietic differentiation of human induced pluripotent stem cells provides granulocytes or macrophages for cell and gene therapies Thomas Moritz, REBIRTH Cluster of Excellence, Hannover Medical School

10.30 Amazon

2c: Muscle gene and cell therapy Chairs: Hidde Haisma, Johnny Huard INV043 The role of stem cell exhaustion in aging and disease Johnny Huard, University of Pittsburgh, PA INV044 Gene replacement therapy for myotubular myopathy Anna Buj-Bello, Généthon, Evry Proffered papers OR009 Adeno-associated virus vector (AAV) microdystrophin gene therapy for Duchenne muscular dystrophy OR011 Dystrophin deficient rats: generation and characterisation of a new model for Duchenne Muscular Dystrophy Caroline Le Guiner, Généthon, Evry OR010 MMP-9 serum levels increase over time in Duchenne Muscular Dystrophy patients and decrease upon treatment with drisapersen Pietro Spitali, Leiden University Medical Centre 47

PROGRAMME FRIDAY 24 OCTOBER 2014 10.30 Yangtze

2d: Cardiovascular and pulmonary diseases Chairs: Seppo Ylä-Herttuala, Sarah Ferber INV045 Cardiac regeneration: Stem cells and beyond Marie Jose Goumans, Leiden University Medical Centre INV046 Systemic delivery of AAVrh10 expressing frataxin corrects the severe mitochondrial cardiomyopathy of frataxin deficient mice Hélène Puccio, IGBMC, Strasbourg Proffered papers OR012 Molecular mechanisms of vascular hyperpermeability in VEGF therapy Johanna Laakkonen, University of Eastern Finland, Kuopio OR013 ‘First in human’ clinical trial using allogeneic Cardiac Stem/Progenitor Cell (CSCs) for Acute Myocardial Infarction (AMI) treatment. Luis Rodriguez-Borlado, Coretherapix S L, Madrid OR014 A PET based approach for highly sensitive monitoring of teratoma formation from pluripotent stem cells Cajetan Lang, University of Rostock OR015 Pulmonary transplantation of multipotent or pluripotent-stem cell derived macrophage progenitors as a novel treatment option for Pulmonary Alveolar Proteinosis Nico Lachmann, REBIRTH Cluster-of Excellence, Hannover Medical School

12.30

ESGCT General Assembly – Auditorium

12.30

Lunch – Exhibition and posters

14.30

3: Emerging technologies in hematopoietic stem cell gene therapy

Auditorium

Chairs: Luigi Naldini, Keith Joung INV047 Targeted genome and epigenome editing using engineered CRISPR-Cas and TALE technologies Keith Joung, Harvard Medical School, Boston, MA INV048 Development and applications of CRISPR-Cas9 for genome editing Feng Zhang, The Broad Institute, Cambridge, MA INV049 Prosthetic networks – synthetic biology-inspired treatment strategies for metabolic disorders Martin Fussenegger, Swiss Federal Institute of Technology, Zürich

16.00

Afternoon break

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME FRIDAY 24 OCTOBER 2014 16.30

Parallel Sessions 3a, 3b, 3c, 3d

Auditorium

3a: Genetic vaccines and Ad vectors Chairs: Kerry Fisher, Dale Vanderputten INV050 The use of AAV vectors to express antibodies to prevent and/or treat infectious diseases James Wilson, University of Pennsylvania INV051 In vivo barriers and advances in adenovirus vector technology Stefan Kochaneck, University of Ulm Proffered papers OR016 Oncolytic adenovirus loaded with MHC-I restricted peptide as platform for oncolytic vaccine Vincenzo Cerullo, University of Helsinki OR017 Lentiviral-based anti-HIV therapeutic vaccine: design, preclinical studies and phaseI/II clinical trial preliminary results Cécile Bauche, Theravectys, Villejuif OR018 Development of a next generation Semliki Forest virus-based DNA vaccine against cervical cancer Stephanie van de Wall, University of Groningen OR019 ORCA-010, a novel potency enhanced oncolytic adenovirus, exerts strong antitumor activity in preclinical models Wenliang Dong, ORCA Therapeutics BV; VU University, Amsterdam

16.30 Mississippi

3b: Epigenetic regulation and gene editing I Chairs: Didier Trono, Hidde Haisma INV052 Architecture and dynamics of genomenuclear lamina interactions Bas van Steensel, Netherlands Cancer Institute, Amsterdam INV053 Transposable elements and their epigenetic control mechanisms are key regulators of transcriptional networks in pluripotent stem cells Didier Trono, EPFL, Lausanne Proffered papers OR020 Targeted genome editing in human long-term repopulating hematopoietic stem cells for correction of SCID-X1 Pietro Genovese, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR021 Targeted genome editing by lentiviral protein transduction of ZFN and Cas9 proteins Yujia Cai, Aarhus University

PROGRAMME FRIDAY 24 OCTOBER 2014 OR022 CCR5-Uco-TALEN: A novel highly active TAL effector nuclease (TALEN) for the targeted knock-out of the HIV-co-receptor CCR5 Ulrike Mock, University Medical Centre Hamburg-Eppendorf OR023 Towards TALEN-mediated targeting of FANCA in the AAVS1-homolog safe harbor locus in FA-A mice Maria Jose Pino-Barrio, CIEMAT/CIBERER, Madrid 16.30 Amazon

3c: Gene and cell therapy for ocular diseases Chairs: Robin Ali, Kyriacos Mitrophanous INV054 WNT7A and PAX6 define corneal epithelium homeostasis and pathogenesis Kang Zhang, University of California, San Diego INV055 Engineering light responses and outer segments Botond Roska, Friedrich Miescher Institute for Biomedical Research, Basel Proffered papers OR024 Evaluation of an optimised injection system for retinal gene therapy Dominik Fischer, University Hospital, Tübingen; University of Oxford OR025 Preventing visual loss after corneal trauma by targeting miR-145 Christiane Gras, Hannover Medical School OR026 Effective delivery of large genes to the retina by dual AAV vectors Ivana Trapani, TIGEM, Naples OR027 Development of EncorStat®, donor corneal tissue genetically engineered to prevent graft rejection Scott Ellis, Oxford BioMedica Ltd

16.30 Yangtze

3d: Genetic instability syndromes and vector genotoxicity Chairs: Juan Bueren, Christof von Kalle INV056 Mutatis mutandis: why a cell makes mutations and how this can benefit our fitness Niels De Wind, Leiden University Medical Centre INV057 Towards the lentiviral gene therapy of mobilised CD34+ cells from Fanconi anemia patients Juan Bueren, CIEMAT/CIBERER, Madrid Proffered papers OR028 Comparative AAV wild-type and rAAV vector-mediated genomic integration profiles in human diploid fibroblasts analysed by 3rd generation PacBio DNA sequencing Regine Heilbronn, Charité University Medicine, Berlin

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME FRIDAY 24 OCTOBER 2014 OR029 A new bioinformatics tool to improve precision and quality of vector integration site identification after genomic alignment Andrea Calabria, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR030 High throughput, high nucleotide resolution integration site analysis of wtAAV reveals viral instability during the process of integration Karl Petri, National Center for Tumor Diseases (NCT); German Cancer Research Center (DKFZ), Heidelberg OR031 Targeted Sequencing for Detection of Vector Integration Sites Stefan Wilkening, National Center for Tumor Diseases (NCT); German Cancer Research Center (DKFZ), Heidelberg 18.30

Poster session 2 – Onyx room Posters 104–197. See page 33 for details

20.00

Speakers’ Dinner – by invitation only Coaches will depart from the World Forum main entrance at 19.45

PROGRAMME SATURDAY 25 OCTOBER 2014 MAIN CONGRESS 08.30

4: Gene and cell therapy for cancer

Auditorium

Chairs: Rob Hoeben, Victor van Beusechem INV058 TRUCKs: the new generation of CARs Hinrich Abken, University of Cologne INV059 Development of Chimeric Antigens Receptors for the treatment of cancer Richard Morgan, bluebirdbio, Cambridge, MA INV060 Systemic virotherapy for multiple myeloma Stephen Russell, Mayo Clinic, Rochester, MN

10.00

Morning break

10.30

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

Yangtze

4a: RNA therapeutics Chairs: Annemieke Aartsma-Rus, Bruno Pitard INV061 Beyond clinical trials for antisense-mediated exon skipping for Duchenne Muscular Dystrophy Annemieke Aartsma Rus, Leiden University Medical Centre INV062 Pip peptide conjugates of exon skipping PMO for therapy of Duchenne Muscular Dystrophy Michael Gait, Medical Research Council, Cambridge Proffered papers OR032 Synthetic mRNA encoding vaccinia virus PKR inhibitors improves immunisation with Semliki Forest virus replicons Tim Beissert, University Medical Center, Mainz OR033 Messenger RNA in cancer immunotherapy: TriMix delivering an antitumor message Karine Breckpot, Vrije Universiteit, Brussels OR034 Best of both worlds – a novel versatile AAV vector toolbox for combinatorial CRISPR and RNAi expression Dirk Grimm, DZIF, Heidelberg University Hospital OR035 In vivo mRNA introduction using polyplex nanomicelles to treat neurological disorders Keiji Itaka, University of Tokyo

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME SATURDAY 25 OCTOBER 2014 10.30 Mississippi

4b: Gene and cell therapy for metabolic diseases Chairs: Gerard Wagemaker, Harald Petri INV063 Perinatal gene therapy ameliorates a mouse model of neuronopathic Gaucher disease Simon Waddington, UCL Institute for Women’s Health, London INV064 Toward a curative single intervention therapy in Pompe disease Niek van Til, University Medical Centre Utrecht Proffered papers OR036 Phase 1 clinical trial of liver directed gene therapy with rAAV5/2PBGD in acute intermittent porphyria: safety data Gloria Gonzalez-Aseguinolaza, Centro de Investigación Médica Aplicada (CIMA), Pamplona OR037 Full correction of neurologic and somatic lysosomal pathology in Mucopolysaccharidosis type IIIB by AAV9-based gene therapy Virginia Haurigot, Universitat Autònoma de Barcelona OR038 Development of ex vivo gene therapy for familial LCAT deficiency syndrome by self-transplantation of therapeutic-enzyme secreting adipocytes Masayuki Kuroda, Chiba University OR039 Lentiviral vector based hematopoietic stem cell gene therapy mediates sustained expression of functional thymidine phosphorylase in a mouse model for mitochondrial neurogastrointestinal encephalomyopathy Rana Yadak, Erasmus Medical Center, Rotterdam

10.30 Auditorium

4c: Blood coagulation diseases Chairs: Sam Wadsworth, Zoltan Ivics INV065 Long term safety and efficacy of a novel selfcomplementary adeno-associated viral vector encoding human FIX in patients with severe hemophilia B Amit Nathwani, Royal Free NHS Trust; UCL Cancer Institute; NHSBT, London INV066 Platelet-mediated gene therapy of hemophilia Shi Qizen, Medical College of Wisconsin, Milwaukee Proffered papers OR040 Safety and efficacy of AAV5-hFIX in non-human primates Bart Nijmeijer, uniQure BV, Amsterdam OR041 Baboon envelope pseudotyped LVs mediate high level gene transfer in human B cells allowing secretion of FIX at therapeutic levels in humanised NSG mice Els Verhoeyen, Université de Lyon-1; CIRI, INSERM U1111; INSERM U1065, Nice 53

PROGRAMME SATURDAY 25 OCTOBER 2014 OR042 Targeting FVIII expression to specific cell-types to overcome immunological responses for hemophilia A gene therapy Antonia Follenzi, Università del Piemonte Orientale, Novara 10.30 Amazon

4d: Immunotherapy of cancer I Chairs: Mirjam Heemskerk, Attilio Bondanza INV067 PK/PD modeling of CAR-T-cell immunotherapy targeted at the cancer-initiating antigen CD44v6 Attilio Bondanza, San Raffaele University Hospital and Scientific Institute, Milan INV068 High affinity T-cell receptors for targeting B-cell malignancies Mirjam Heemskerk, Leiden University Medical Centre Proffered papers OR043 Redirection of CD4+ T-cells with ICOS-based chimeric antigen receptors Sonia Guedan Carrio, University of Pennsylvania, Philadelphia OR044 NY-ESO-1-specific Single Edited T-cells efficiently eliminate multiple myeloma without inducing xenogeneic GvHD Sara Mastaglio, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR045 A traceless selection system that allows efficient generation of auxiliary vector-free Transposon and CRISPR-modified T-cell products Riccardo Mezzadra, NKI-AvL, Amsterdam OR046 Donor T-cells for hematopoietic-restricted minor histocompatibility antigens are induced in patients with combined Graft-versusLeukemia and Graft-versus-Host disease Margot Pont, Leiden University Medical Centre

12.30

NVGCT General Assembly – Auditorium

12.30

Lunch – Exhibition and posters

14.00

5: New approaches to engineering vectors and cells

Auditorium

Chairs: Luigi Naldini, Phil Gregory INV069 Derivatives of human pluripotent stem cells: the new patient? Christine Mummery, Leiden University Medical Centre INV070 Genome editing with zinc finger nucleases Phil Gregory, Sangamo BioSciences, Inc, Richmond, CA INV071 Novel rAAV vectors for episomal and integration based gene transfer Mark Kay, Stanford University, CA

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME SATURDAY 25 OCTOBER 2014 15.00

Parallel sessions 5a, 5b, 5c, 5d

Auditorium

5a: Primary immune deficiencies Chairs: Alessandro Aiuti, Harry Malech INV072 Progress in gene therapy for PIDs Adrian Thrasher, UCL Institute of Child Health, London INV073 Update on gene therapy trials for severe combined immunodeficiency and WiskottAldrich Syndrome David Williams, Boston Children’s Hospital, Harvard Medical School, Boston, MA Proffered papers OR047 Genetic correction of induced pluripotent stem cells from a WiskottAldrich Syndrome patient normalizes the immune defects Brian Davis, University of Texas, Houston OR048 Ex vivo Gene Therapy in a Mouse Model of Leukocyte Adhesion Deficiency Type I Elena Almarza, CIEMAT/CIBERER, Madrid OR049 Finding levels of RAG1 expression that will correct RAG1 Severe Combined Immunodeficiency (Final) Karin Pike-Overzet, Leiden University Medical Centre OR050 Transplantation of human SCID stem cells in NSG mice gives new insights into human T-cell development and reveals where SCID mutations act Anna-Sophia Wiekmeijer, Leiden University Medical Center

15.00 Mississippi

5b: Gene and cell therapy for neural diseases Chairs: Joost Verhaagen, Nathalie Cartier INV074 Gene therapy strategies for Alzheimers disease Nathalie Cartier, Université Paris Sud; INSERM U986; CEA MIRcen INV075 Efficient lentiviral vector-mediated delivery of hARSA in the brain of juvenile non-human primates Angela Gritti, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan Proffered papers OR051 New model for Ubiquilin2-linked ALS: investigation of pathological mechanisms and therapeutic perspectives Maria-Grazia Biferi, Institut de Myologie, INSERM-CNRS-UPMC-UMR 974, Paris 55

PROGRAMME SATURDAY 25 OCTOBER 2014 OR052 High level expression of human iduronidase throughout the brain in a murine model of mucopolysaccharidosis type I (MPS I) after noninvasive AAV-mediated gene delivery to the CNS Karen Kozarsky, ReGenX Biosciences, Washington DC OR053 OXB-102: an enhanced gene therapy for Parkinson’s disease Kyriacos Mitrophanous, Oxford BioMedica Ltd OR054 AAV-mediated delivery of SMN1 in a mouse model of spinal muscular atrophy Martine Barkats, Institut de Myologie; UPMC-AIM UMR S974, INSERM U 974, CNRS FRE 3617, Paris 6 15.00 Yangtze

5c: RNA vectors Chairs: Sarah Ferber, Manfred Schmitt INV076 HIV-1 capsid controls tropism through evasion of innate sensors Greg Towers, UCL, London INV077 Defining the integration landscape of viral vectors in the era of next generation sequencing Manfred Schmidt, National Center for Tumor Diseases (NCT); German Cancer Research Center (DKFZ), Heidelberg Proffered papers OR055 Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter Aaron Cooper, University of California Los Angeles (UCLA) OR056 Development of F/HN pseudotyped Lentivirus for airway gene transfer Deborah Gill, UK Cystic Fibrosis Gene Therapy Consortium, Oxford, Edinburgh & London OR057 BET-independent MLV-based vectors target away from promoters and regulatory elements Sara El Ashkar, KU Leuven OR058 Measles virus glycoprotein pseudotyped lentiviral vectors transduce HSC SCID repopulation cells at efficiencies reaching up to 100% Camille Lévy, Université de Lyon-1; CIRI, INSERM U1111

15.00 Amazon

5d: Bioprocessing of cell and gene therapy products Chairs: Otto Merten, James Miskin INV078 A new downstream process for large scale manufacturing of AAV9 vectors Matthias Hebben, Généthon, Evry INV079 High titer production of GMP grade SIN gamma-retroviral vectors Klaus Kühlcke, Eufets, Idar-Oberstein

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME SATURDAY 25 OCTOBER 2014 Proffered papers OR059 Development of the manufacturing process for the ex vivo gene therapy for ADA-SCID (GSK2696273): process design, validation and comparability Nina Kotsopoulou, GlaxoSmithKline, Stevenage OR060 GLP preclinical studies for gene therapy medicinal products combine highest regulatory standards with outstanding scientific significance Aisha Sauer, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR061 Automated magnetic isolation and lentiviral vector modification of human CD34+ cells in a functionally closed system Ian Johnston, Miltenyi Biotec GmbH, Bergisch Gladbach OR062 High yield adenovirus production in fixed-bed bioreactor Hanna Lesch, FKD Therapies; FinVector Vision Therapies, Kuopio 17.00

Afternoon break

17.30

Parallel sessions 6a, 6b, 6c, 6d

Amazon

6a: Regenerative medicine Chairs: Joost Verhaagen, Nathalie Cartier INV080 Generation of dopamine neurons for cell therapy in Parkinson’s disease Malin Parmar, Lund University INV081 Gene therapy for neurotrophic factors to promote regeneration of injured peripheral nerves Joost Verhaagen, Netherlands Institute for Neuroscience, Amsterdan INV082 Stem cells based technologies as non-viral transfection approaches Marcelle Machluf, Technion- Israel Institute of Technology, Haifa Proffered papers OR063 Phase 1-2 clinical trial in patients with decompensated liver cirrhosis treated with bone-marrow derived endotelial progenitor cells: preliminary safety and efficacy analysis Delia D’Avola, Clinica Universidad de Navarra, Pamplona OR064 Advanced tuning of Notch signaling to regulate in vivo myogenic repair of murine and human mesoangioblasts Mattia Quattrocelli, KU Leuven

PROGRAMME SATURDAY 25 OCTOBER 2014 17.30 Yangtze

6b: Inflammatory and autoimmune diseases Chairs: Margriet Vervoordeldonk, Rosa Bacchetta INV083 Adult stem cells organoids and regenerative medicine Robert Vries, University Medical Centre, Utrecht INV084 Generation of regulatory T-cells by FOXP3 gene transfer: different approaches towards CD4FOXP3 T-cell-based therapy for autoimmune diseases Rosa Bacchetta, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan Proffered papers OR065 Antigen-specific myeloid-derived suppressor cells ameliorate experimental autoimmune encephalomyelitis Silvia Casacuberta-Serra, Universitat Autònoma de Barcelona OR066 Modulation of the ATP:adenosine balance by AAV-5 mediated gene delivery of CD39-CD73 in fibroblast-like synoviocytes from rheumatoid arthritis patients Susanne Snoek, Arthrogen BV, Amsterdam OR067 Modulation of mouse models of rheumatoid arthritis by systemic delivery of immunoregulatory bovine milk derived exosomes Fons van de Loo, Radboud University Medical Center, Nijmegen

17.30 Auditorium

6c: Oncolytic therapies Chairs: Len Seymour, Victor van Beusechem INV085 Armed oncolytic adenovirus can overcome critical obstacles in adoptive T-cell therapy of solid tumors Akseli Hemminki, University of Helsinki, Finland INV086 Preliminary results of the phase 1 trial with the Delta24RGD oncolytic adenovirus, administered by CED in patients with recurrent Glioblastoma Clemens Dirven, Erasmus Medical Centre, Rotterdam Proffered papers OR068 Immune checkpoint blockade enhances oncolytic Measles virus therapy Christine Engeland, National Center for Tumour Diseases (NCT); German Cancer Research Center (DKFZ), Heidelberg

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME SATURDAY 25 OCTOBER 2014 OR069 Decapitated reoviruses with a little iLOV Diana van den Wollenberg, Leiden University Medical Centre OR070 Insertion of an albumin-binding domain in adenovirus hexon improves the pharmacokinetics and antitumor efficacy of oncolytic adenoviruses Luis Alfonso Rojas, Instituto Catalan de Oncología-IDIBELL, L´Hospitalet de Llobregat OR071 Immunotherapy against pancreatic cancer using sequential administration of antigenically distinct oncolytic viruses expressing oncostatin M Ruben Hernandez-Alcoceba, Centro de Investigación Médica Aplicada (CIMA), Pamplona 17.30 Mississippi

6d: Immunotherapy of Cancer II Chairs: Zoltan Ivics, Laurence Cooper INV087 Human translation of Sleeping Beauty system and next-generation clinical trials Laurence Cooper, MD Anderson Cancer Centre, Houston, TX Proffered papers OR072 An innovative CAR-T-cell spacer allowing selection/tracking and enabling superior antitumor effects in vivo Monica Casucci, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR073 TAP-independent presentation of exogenous epitopes by oncolytic adenoviruses enhances specific immune responses and antitumor activity Alba Rodríguez-García, Instituto Catalan de Oncología-IDIBELL, L´Hospitalet de Llobregat OR074 Alpharetroviral vectors for the transduction of primary human natural killer cells: selective enhancement of tumor cytotoxic activity by chimeric antigen receptors Julia Suerth, Hannover Medical School OR075 Targeted in vivo TCR gene transfer into mouse T-cell subsets for immunotherapy of cancer Wolfgang Uckert, Humboldt University, Berlin OR076 Re-engineering of human CYP4B1 for optimal catalytic processing of 4-ipomeanol and use as a suicide gene in adoptive cell therapy Constanze Wiek, Heinrich Heine University, Düsseldorf OR077 Long-term episomal gene transfer for safe engineering of T-cells for adoptive cell therapy of cancer Di Yu, Uppsala University 59

PLEASE NOTE: At 2am last night the clocks turned back one hour for the end of Daylight Saving Time

PROGRAMME SUNDAY 26 OCTOBER 2014 MAIN CONGRESS 09.00

Parallel sessions 7a, 7b, 7c, 7d

Yangtze

7a: Exosomes and non-viral delivery Chairs: Fons van de Loo, Enrico Mastrobattista INV088 Clinical potential of cell-derived vesicles: challenges to analyse the molecular composition of the extracellular vesicle pool Marca Wauben, Utrecht University INV089 Extracellular vesicles for therapeutic RNA delivery: Promises and pitfalls Pieter Vader, University Medical Centre, Utrecht Proffered papers OR078 Gene therapy based on cytidine deaminase-targeting overcomes pancreatic cancer resistance to chemotherapy Marion Gayral, Paul Sabatier University; INSERM U1037, Toulouse OR079 Results of a phase IIb non-viral gene therapy trial from the UK CF Gene Therapy Consortium Uta Griesenbach, UK Cystic Fibrosis Gene Therapy Consortium, Oxford, Edinburgh & London OR080 Virus-free delivery of microRNA into freshly isolated patient derived CD105+ MSCs using a novel magnet-bead based vector system Paula Müller, University of Rostock OR094 Bio-inspired approaches for siRNA delivery Koen Raemdonck, Ghent University

09.00 Amazon

7b: Infectious diseases Chairs: Ben Berkhout, Luc van der Laan INV090 Preclinical studies on Newcastle disease virus for the treatment of pancreatic adenocarcinoma Ron Fouchier, Erasmus Medical Centre, Rotterdam INV091 RNAi -based gene therapy for HIV-1 Ben Berkhout, University of Amsterdam Proffered papers OR081 A therapeutic anti-Hepatitis C virus shmiRNA integrated into the miR-122 genomic locus mediates a potent anti-viral response Elena Senis, DZIF, Heidelberg University Hospital

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME SUNDAY 26 OCTOBER 2014 OR082 Integration driven HIV-1/STAT5B chimeric transcripts confer a selective advantage to blood cells in patients under anti-retroviral therapy Daniela Cesana, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR083 Cross-Clade inhibition of HIV on primary cells by CXCR4 or CCR5 fused to the C34 peptide from gp41 HR2 Michael Holmes, Sangamo BioSciences, Inc, Richmond, CA 09.00 Auditorium

7c: Gene Editing II Chairs: Toni Cathomen, Christof von Kalle INV092 Gene editing in pluripotent stem cells to model primary immune deficiencies Toni Cathomen, University Medical Centre, Freiburg INV093 Targeted editing of the human (epi)genome using artificial transcriptional repressors Angelo Lombardo, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan Proffered papers OR001 A CRISPR way to eradicate latent HIV: Targeted excision of integrated HIV-1 DNA from human cells through vector-based genome engineering Kathleen Börner, DZIF, Heidelberg University Hospital OR002 An AAV vector toolbox for CRISPR/Cas9-mediated genome engineering Polychronis Fatouros, DZIF, Heidelberg University Hospital OR003 Adenoviral vector DNA is a preferred homologous recombination substrate for accurate genome editing using engineered nucleases Ignazio Maggio, Leiden University Medical Center OR004 Engineering human models of tumor-associated chromosomal translocations with the RNA-guided CRISPR–Cas9 system Raúl Torres, Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid

PROGRAMME SUNDAY 26 OCTOBER 2014 09.00 Mississippi

7d: Hematopoietic stem cell and HSC gene therapy Chairs: Juan Bueren, Nathalie Cartier INV094 Outcomes of gene therapy for b‑Thalassemia Major via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral bA‑T87Q‑Globin vector Salima Hacein-Bey-Abina, Hôpital Universitaire Necker – Enfants Malades, Paris Proffered papers OR088 A non human primate model for autologous transplantation of IPSCderived hematopoietic cells Leila Maouche-Chrétien, University Paris Sud 11; CEA-iMETI, INSERM U962, Fontenay aux Roses OR089 Exploring bone marrow microenvironment in a murine model of ß-thalassemia Annamaria Aprile, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR090 Mesenchymal stromal cells enhance the engraftment of low numbers of hematopoietic stem cells in a mouse model of autologous transplantation Maria Fernandez-Garcia, CIEMAT/CIBERER, Madrid OR091 Retroviral expression of dominant-negative Mpl disrupts THPO/MPL signaling and HSC maintenance Saskia Kohlscheen, Paul-Ehrlich-Institute, Langen OR092 CD34+ cells isolated from different sources: exploring their biology for future clinical perspectives Maria Rosa Lidonnici, HSR TIGET, San Raffaele Telethon Institute for Gene Therapy, Milan OR093 Defining regulatory elements in human embryonic and somatic stem cells by MLV integration profiling Valentina Poletti, Généthon, Evry

11.00

Morning break

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

PROGRAMME SUNDAY 26 OCTOBER 2014 11.30

Presidential symposium and awards ceremony

Auditorium

Chairs: Luigi Naldini, Gerard Wagemaker INV097 Keynote lecture; Nobel Laureate for Medicine Shinya Yamanaka, Centre for iPS Cell Research and Application, Kyoto University INV098 The innovative medicines: a European engine for stem cell research Michel Goldman, Innovative Medicines Initiative (IMI), Brussels INV099 StemBANCC – Patient iPSCs to study a wide range of diseases Martin Graf, Hoffmann-La Roche Ltd, Basel INV100 EBiSC: the European bank for induced pluripotent stem cells Timothy Allsopp, Pfizer Ltd, Ipswich Young Investigator Awards OR101 MiRNAs 182 and 183 are necessary to maintain adult cone photoreceptor outer segments and visual function Volker Busskamp, Centre for Regenerative Therapies, Dresden OR102 Development of oncolytic vaccines for cancer treatment: the past, the present and the future! Vincenzo Cerullo, University of Helsinki Oustanding Achievement Award INV103 Gene therapy of chronic granulomatous disease: lessons learned and future perspectives Manuel Grez, Institute for Biomedical Research, Georg-Speyer-Haus, Frankfurt Presentation of new president and 2015 Congress

VISITING THE HAGUE

The Hague, international city of peace and justice, was named Holland’s Best City 2013-2015. Below is a guide to help you find your way around the city and get the most out of your visit.

See...

SEE

From the recently renovated Mauritshuis, where you can see Vermeer’s ‘Girl with the Pearl Earring’, to the Gemeentemuseum Den Haag, which houses a unique collection of Mondriaan paintings, there is plenty of art and culture at The Hague. Scan this code for the city’s must-see attractions.

denhaag.com/en/must-see

Find...

Scan this code for a selection of maps of The Hague. Or go to http:// www.denhaag.nl/home/den-haagop-kaart.htm for an interactive map in Dutch where you can select what you’re looking for (e.g. parking). It also has a route planner (fill in the complete street address followed by a comma and ‘Den Haag’).

http://en.denhaag.nl/en/residents/news-and-events/to/Maps-of-The-Hague.htm

Eat...

The multicultural nature of The Hague is reflected in the wide variety of cuisine, including some of the best Indonesian restaurants in the country. Scan this code for a list of restaurants and a link to Tripadvisor restaurant reviews.

http://www.denhaag.nl/en/residents/to/Restaurants-nightlife.htm 64

ESGCT/NVGCT COLLABORATIVE CONGRESS 2014

Go...

tripadvisor.co.uk/Travel-g188633-s302/The-Hague:The-Netherlands:Getting.Around.html

Relax...

The Hague boasts of being the greenest city in Europe. Escape the bustle of the city and take a stroll on the beach at Scheveningen, or wander through the Palace Garden, one of the city’s best kept secrets.

http://denhaag.com/en/outdoor

Shop... © DEN HAAG MARKETING/JURJEN DRENTH

It is fairly easy to get around The Hague. Scan this code for information about trams, busses, tickets and parking. Note that public transport does not run all night; there are a few night busses, but they only run once an hour, serving a limited number of areas.

Scan this code for information about where to shop, from indoor shopping, to fashion, art and design or the biggest market in Europe, which opens on Mondays, Wednesdays, Fridays and Saturdays.

holland.com/uk/tourism/cities-in-holland/visit-the-hague/shopping-in-the-hague-2.htm

Help...

There are Tourist Information Points at several locations in The Hague. Scan this code for opening hours. VVV The Hague Telephone: (+31) (0)70 361 88 60 Email: vvv@denhaag.com Twitter: www.twitter.com/the_hague

http://www.denhaag.nl/en/visitors/to/VVVtouristinformationshop.htm 65

EUROPEAN SOCIETY OF GENE AND CELL THERAPY ACHIEVEMENT AWARDS Outstanding Achievement Award: In collaboration with Human Gene Therapy and Mary Ann Liebert Publishers, ESGCT presents one award for an established researcher who has made a long-term, outstanding contribution to the field. €2000 honorarium and 30 minute presentation during the annual congress. Young Investigator Awards: In collaboration with Généthon, €1000 and a 15 minute presentation during the annual congress for up to four researchers who are showing exceptional promise. Travel grants: Supported by the national societies, up to 10 awards of €250 for PhD and first post docs. These will be awarded on the basis of abstract score. Application and nomination details are available at www.esgct.eu/awards Note: Eligibility criteria applies

ESGCT EVALUATION We do hope you have enjoyed the ESGCT/NVGCT Collaborative Congress 2014. We really value your feedback about all aspects of the Congress. We would be very grateful if you could take a few minutes to complete this online questionnaire either during or soon after the Congress. https://www.surveymonkey.com/s/XPBDLXX A link is also available from the ESGCT website. If you enjoyed this meeting, would you consider hosting/organising an ESGCT or NVGCT congress in 2017? If so, please forward your proposal to the ESGCT Board as soon as possible at office@esgct.eu, or the NVGCT Board at p.j.bosma@amc.uva.nl. Thank you in advance for your time. ESGCT and NVGCT Team

The Leader in Gene Therapy Delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product GlyberaÂŽ.

www.uniqure.com

NOW WITH STORAGE TEMPERATURE* TRACKING

LAN Network or Internet Â° 80 C

30 DAYS SMS or Email * You can also track humidity, gaz, pressure and much more...

www.LabCollector.com

AgileBio SARL 75 rue de Lourmel 75015 Paris Tel : 01 41 79 15 85 - Fax: 01 72 70 40 22

PRIX GALIEN 2012

GENE THERAPY FOR RARE DISEASES Advancing Therapies From Research to Patient Treatment

Created in 1990 by AFM-TELETHON and funded through the donations collected annually during the Telethon fundraising event, Genethon, a pioneer in deciphering the human genome and identifying the genes associated with genetic diseases is today fully dedicated to the design, development and GMP production of gene therapy treatments for rare diseases. In 2013, Genethon has opened Genethon BioProd, a 5000 mÂ˛ facility fully dedicated to GMP-production and QC of gene therapy products. Genethon conducts R&D programs from discovery to clinicals trials in the fields of neuromuscular diseases, immunodeficiencies, liver diseases and eye disorders.

FOR MORE INFORMATION and details: www.genethon.fr

ÂŠ C. Bernard - C. Hargoues J.P. Pouteau

Member of

European Union support to gene and cell therapy research in Horizon 2020 (2014â&#x20AC;&#x201C;2020) The European Union, through its Framework Programmes for Research and Technological Development, has financially supported collaborative efforts in gene and cell therapy research to bring the technology closer to the clinic and to the market. Most of the latest projects involve (often multicentre, international) clinical trials and reflect the European leading position in the field. The new European programme for research and innovation, Horizon 2020 (2014â&#x20AC;&#x201C;2020), will continue to support gene and cell therapies as innovative/advanced technologies for tackling health challenges. An overview of some of the funding opportunities will be presented during the Education Day, 23 October 2014 (collaborative research projects, Marie Sklodowska-Curie training actions, European Research Council frontier research grants) as well as a selective overview of some ongoing FP7 projects. References: D Gancberg et al., Mol Ther 2012, 20: 2191 D Gancberg et al., Hum Gene Ther 2014, 25: 1 D Gancberg et al., Hum Gene Ther Clin Dev 2014, 25: 51-71 David Gancberg Directorate Health, Directorate-General for Research and Innovation, European Commission CDMA 0/174, B-1049 Brussels, Belgium Phone: +32 2 2984566 Fax: +32 2 2994693 E-mail: david.gancberg@ec.europa.eu

European Society of Gene and Gell Therapy International Society for Stem Cell Research JOINT CONGRESS 18â&#x20AC;&#x201C;21 October 2016 Florence, Italy The congress will take place at the Firenze Fiera, with plenary sessions and exhibitor halls at the stunning historical Palazzo Dei Congressi. Parallel and poster sessions will take place at the Palazzo Degli Affari.

BSGCT

ANNUAL CONFERENCE 2015 Tuesday 9th June-Thursday 11th June 2015 Technology & Innovation Centre, Glasgow, Scotland Main topics: • • • • • • •

Clinical trials in novel therapeutics Genome/cell engineering technologies RNA-based therapeutics Cancer Cutting edge viral technologies Clinical gene and cell therapy approaches Organ regeneration/tissue engineering approaches

Including lectures from Nathalie Cartier, Inserm, Doris Taylor, Texas Heart Institute, R Jude Samulski, UNC-CH School of Medicine and Adrian Thrasher, University College London. Please see website for details of associated meetings taking place during the same week.

Abstract Submission deadline: Friday 24th April 2015 Website: www.bsgct.org

French Society of Cell and Gene Therapy

Thematic days

19–20 March 2015 Le Kremlin Bicêtre, Paris 19 March 2015: Ethical and regulatory aspects of gene and cell therapy: Societal aspects of stem cells Organised in collaboration with DIM Biothérapies Paris, Ile de France

20 March 2015: Emerging modalities of gene regulation: from basic science to gene therapy

SAVE THE DATE!

The Annual Congress of the SFTCG will take place in March 2016 in Marseille See www.sftcg.fr for details

https://twitter.com/SFTCG

Spanish Society for Gene and Cell Therapy

8th Biennial Congress 4â&#x20AC;&#x201C;6 November 2015 Kursaal Congress Centre San Sebastian-Donostia, Spain The main conference gathering researchers on stem cells, pluripotency, gene modification, cancer, biomaterials, tissue and organ engineering. Come and join gene & cell-based therapy experts in one of the most beautiful cities in Europe! Fellowships from sister European societies available.

www.setgyc.es https://www.facebook.com/setgyc

https://twitter.com/setgyc

German Society for Gene Therapy DG-GT e.V.

XXI Annual Meeting Vienna, 26-28 February 2015

Center of Pharmaceutical Sciences AlthanstraĂ&#x;e 14, UZA II 1090 Wien Austria

From basic concepts to clinical applications The XXI annual meeting of the German Society for Gene Therapy DG-GT will take place in the beautiful city of Vienna, Austria from 26-28 February, 2015. At the Faculty Center of Pharmaceutical Sciences of the University of Vienna, a two and a half day meeting together with an attractive social programme, presenting Viennaâ&#x20AC;&#x2122;s highlights, will be organised. Special attention will be paid to topics like cancer gene therapy including oncolytic viruses, molecular imaging, cutting edge vector technologies and an update on clinical trials. Latest developments in gene and cell therapy will be discussed. During a half day educational session preceding the meeting, state of the art technologies in vector design and production, molecular imaging, disease models and clinical issues will be discussed. Students will have the possibility to apply for a limited number of travel grants. The meeting website is open for abstract submission with abstract deadline scheduled for December 2014. Further information on travel and accommodation is also available there.

http://dg-gt2015.univie.ac.at/

The Premier Journal on Gene Therapy

Methods Editor: Thierry VandenDriessche, PhD

Clinical Development Editor: Barry J. Byrne, MD, PhD

Editor-in-Chief: James M. Wilson, MD, PhD Deputy Editor: George Dickson, BSc, PhD Human Gene Therapy Editor: Mark A. Kay, MD, PhD

The Official Journal of

berken boschb lok

du i

pa rk w

van

scheveningseweg

kapelw

pa rk -

nieuwe

we g

b sjac o

hu ver

an sm ple

ett a al

testr. tr. terna

ierstr stra aa att

ierstr s

cht

weg

burn

aat

't ho enstraa t

raam

batjan str.. str

suma trastr

cura

laçaostraat catten van burc h

an 28 coppes

sstr tr..

egra

ban ank kas astra traa at

traat at

delis tr.

riouw

atjeh stra str aa att

ginn

delis tr.

onin

jnla

burg. de monchyplein

balist istrra aa at

ka bu rn rg eb . ee

billit on str. mado astrer . soen strda .

astr.

kkastr.

borneos

esstra at at

mala

oflaa

lombokk str. -

riou rio uw wsstr.

celeb

burg . ppa ati alex an veldder-

na na anlow . u tr pa s

llllw we

kerkh

astr. bank

esstra at at

kkastr.

flaan

bank

celeb

mala

sstr traa t

sstr tra aa att timor

sstr tr..

kerkho

park ken

in se

vin

eg yw pu

prinsevink enp

pr 10

va n

n. nsstr.

cr em erw la eg an

nw eg

sto lk

an speijkstraat

pr. willems

aa t st r do or n

weg

kl n.

j. de graeffln.

de perponcherstraa traatt an eten ete nstr.

boni

an sla der

sta dh

aan

stadhouders l

scheveningse

k-k

val eri u ss tr.

scheveningseweg

staten laan sta atenlaa n

la er la an

et ze bo va n

dtan

gel

erik ik-

slin

van

fred

aan

bev

erin ing

ela erl

van

an bo etz tr. traat

van merlen merle nstraa traatt

conradkade

carnegielaan

l an mea erdevan rvoo rt

riouw

straa traatt

meerder rdervoort

nepweg len

v ary

egie n carn plei

t duc

eg rgw

laan van

kade

obrechtstr. obrechts tr.

ers we g

Bosjes

lin

ra st

er la

eg nrustw

as s

. teld

Scheveningse

weg olweg tol

te bui

la ne

ke . nb str u n ba

schuytstr. schuytst r.

jacob catsl sla aan nbu ruste

lin

plein

kade

bl an

gr oo t

eg lw el

oplaa n

Zorgvliet

badhuisweg

ff.. b.m

a bi to

k 17

va n

laan

tsoen

park

oeko

badhuisweg

ria an g

badhuisweg

ersplan

17 sw ee pyrmont-

https://twitter.com/esgct

tstr a

hts tr.

emma

SPEAKER READY ROOM

Volga Room 1

stadhoud

MEMBERSHIP QUERIES Please go to the registration desk during the opening hours

rec

21 16

pro

frisolaan

in og rt he

ee lin c

For convenience, you can check in coats and luggage near the entrance on the ground floor.

sch

waldeck

hollanders erstraat 2e van blanken burg str.

an joh

n la a itt wit de jan willem

anw plesm

laan

Van Stolkpark 20

churchillchurchill plein

nc ni en elp alex. r.j. schimm gogel gogel ale weg xa nd er g o gel

dankert ertsstra at at

eweg hog

eg van len nep w

straat at

laan

belvedere-

Westbroekpark

straat at

nicolai-

eisenhowerlaan

wer

e uw nie

lübeck-

van hoornbeeks ekstraa traatt van bleiswijks bleiswijkstraa traatt

at aa ra ksttr ijks

ia rtusv ert hu b

plantsoe n

koningin

COATS AND LUGGAGE

kade

ers-

ten hoves hovestraa traatt

Rosarium

g alweg kanaalwe

nslag ag

de an johittlaan w

stadhoud

oginne

preside nt kenned ylaan

pansier- str.

erpsestr. antw

duin

nieu

jolstr stra aa att

inghar

24 wittlaan laan

dee d

elis

corn

g weeg

eg dyw ran

cornelis

straat

lag ijs

straat at

kerklaan

blei

cat sheu euv el

ze l-

franke

n klaa van der heimstra heimstraat at

siusstr. str.

straat at

st r .

55 28

seinpost ststr.

b ger p.s.

antonie hein ant

loo-

oldeenbarneveltlaan johan van old

at traat sstra

at straat

van

tr.. sstr ss

Need a job? Need to hire? Want to share information with the other participants? Post resumes, employment opportunities or information on the designated boards located near the registration desk.

nho

.d..goe a. v.d

JOB OPPORTUNITIES AND OTHER IMPORTANT INFORMATION

n va

viviens enstraa traatt

dijkveld

van

vij

paul ulus us buijsstraa buijsstraatt

laan

kenstraat

ikerik

at straat

n statenlaa

er ijg

Doornpark

ri end

fred

eise frederik frederik hendrikhendri kplein

Please go to the registration desk during the opening hours

a an ikla ndr hen

frankenslag

van

aat str

ningen

fran

t straa

PAYMENT QUERIES

g ag a

sl ken

kkbentinc

tr. sens aers van Statenkwartier 17

n statenlaa

st ra at

aerssenstraatt van aerssenstraa

maurits-

de van wee

van beu

aa t

ijg

fran

de ngka hari f. pr o

at cksstraat duyyck du antonie ant

Located on ground floor

tr de

kent 26 fran t straa traa sens 17 aers van lag kens fran

duplein in -

w duin est

rnstraat doo

z de

Gaëlle Jamar – Event Manager Vanessa Sampson – Payment & Membership

wil

eg inw

at traat schols

aat lstraa scholstr

CONGRESS OFFICE INFORMATION

westdu

g nwe tdui wes

oksstraat lstok koppelst

kinact nin a menstr

ill

Tel: +31-70-3525354 Fax: +31-70-3525353 Email: info@worldhotelbelair.com www.worldhotelbelair.com

lem

THE HAGUE HOSPITALITY INFORMATION DESK

lellykad dr. le e

Worldhotel Bel Air The Hague Johan de Wittlaan 30 2517 JR, The Hague

ven ven 2e Binnenha

aat astr douz

SPEAKER HOTEL INFORMATION:

Thursday 23 October 08.00–20.00 Friday 24 October 08.00–20.00 Saturday 25 October 08.00–20.00 Sunday 26 October 08.30–14.30

de ngka hari

REGISTRATION & INFORMATION DESK

lindoduin

Tel: +31 (0)70 306 63 66 Fax: +31 (0)70 306 64 43 Email: info@worldforum.nl www.worldforum.nl

tr. g

Gaëlle Jamar, Event Manager Tel: +44 7766 475379 email: office@esgct.eu eg kerweg schokkerw

World Forum Churchillplein 10 2517 JW The Hague The Netherlands

CONTACT NUMBER IN CASE OF EMERGENCY

a str een th da

VENUE INFORMATION

gro hovensentraa traat

https://www.facebook.com/esgct 1

Hague_covers.indd 2

01/10/2014 10:14

European Society of Gene and Cell Therapy Finnish Society of Gene Therapy 17–20 SEPTEMBER SEPTEMBER 2015 2015 • FINLANDIA HALL, HELSINKI

www.fsgt.fi • www.esgct.eu Hague_covers.indd 1

ESGCT and NVGCT Congress 2014 • Programme and Information Book

Collaborative Congress 2015

The XXIInd Annual ESGCT Congress in collaboration with the NVGCT 23–26 October 2014 WorldForum, The Hague, The Netherlands