INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018 - 13-15 SEPTEMBER MADRID, SPAIN

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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018 - 13-15 SEPTEMBER MADRID, SPAIN
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

           ADVANCING IMMUNO-GENE THERAPY
           ACROSS DISEASES

           INTERNATIONAL
           CONFERENCE ON
           LYMPHOCYTE
           ENGINEERING
           2018
           13-15 SEPTEMBER
           MADRID, SPAIN

                                           PROGRAMME
LYMPHOCYTE.KENES.COM
#ICLE2018                                                    #ICLE2018                           1
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018 - 13-15 SEPTEMBER MADRID, SPAIN
Kenes
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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018 - 13-15 SEPTEMBER MADRID, SPAIN
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

TABLE OF CONTENTS

CONFERENCE INFORMATION
Welcome                                                                                               4

Venue Map                                                                                             5

General Information                                                                                   6

Information for Oral Presenters and Chairpersons                                                      8

Information for E-Poster Presenters and Chairpersons                                                  9

Poster Presentations                                                                                10

SCIENTIFIC PROGRAMME
Thursday, 13th September                                                                            12

Friday, 14th September                                                                              14

Saturday, 15th September                                                                            18

RECOGNITION, ACKNOWLEDGEMENTS & INDUSTRY SUPPORT
Acknowledgements                                                                                    21

Company Profiles                                                                                    22

ICLE WELCOME RECEPTION                                                                              24

GETTING AROUND IN MADRID                                                                            25

#ICLE2018                                                                                                     3
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018 - 13-15 SEPTEMBER MADRID, SPAIN
WELCOME
    Dear Colleagues,

    It is our great pleasure to welcome you to the first International Conference on Lymphocyte
    Engineering (ICLE) 2018!

    Great strides have been made in recent years in the engineering of lymphocytes for the
    treatment of cancer, autoimmune diseases, pathogenic diseases and primary immune
    deficiencies. Combining gene therapy, cell therapy and immune therapy, the emerging
    field of lymphocyte engineering employs cutting edge technologies of viral and non-viral
    vectorology, genome editing and protein design as well as advanced clinical protocols for
    cell harvest, expansion and re-infusion. With groundbreaking clinical success leading
    to FDA approved products and with vibrant, exponentially growing research, lymphocyte
    engineering is the wave of the future.

    Still, the challenges are formidable and the stakes are high. Realizing the full promise of
    lymphocyte engineering will require the combined efforts of a dedicated community. ICLE
    2018 is our way to facilitate the sharing of data and ideas, promote collaborations, and
    address common challenges to advance clinical translation. We set the aim to provide an
    effective environment for active learning, discussions of real-world case studies and for
    networking between like-minded peers.

    It is time to catch the wave of what is most pioneering and innovative in lymphocyte
    engineering!

    On behalf of the Advisory Board,

    Adi Barzel
    President of the Israeli Society for Gene and Cell Therapy (ISGCT),
    ICLE conference chair,
    Senior lecturer at Tel-Aviv University

4
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018 - 13-15 SEPTEMBER MADRID, SPAIN
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

VENUE MAP

LEVEL 4

                                 ZARAGOZA II                                 ZARAGOZA I

                                                      PLENARY HALL

                                                             ENTRANCE

                                  REGISTRATION AREA
              Res
                 ear

                                  ZARAGOZA III & IV                   02      06        TT5
                    ch T
                        hea
                           ter

                                                                       e-Poster

Exhibitor               Booth No            Logo

CellGenix                         2

GeneWerk                         TT5

VIVEbiotech                       6

#ICLE2018                                                                                                                5
GENERAL INFORMATION

    HOTEL VENUE
    Meliá Avenida America, Calle de Juan Ignacio Luca de Tena, 36, 28027 Madrid, Spain
    Tel: + (34) 914232400

    REGISTRATION DESK HOURS
    Thursday, 13 September		             08:00 – 18:00
    Friday, 14 September		               07:30 – 18:00
    Saturday, 15 September		             07:30 – 16:00

    EXHIBITION OPENING TIMES
    Thursday, 13 September		             10:00 – 18:00
    Friday, 14 September		               10:00 – 18:00
    Saturday, 15 September		             09:30 – 14:00

    PARTICIPANTS BADGE
    Upon registration, you will receive your name badge. You are kindly requested to wear your
    badge during all sessions and events.

    WI FI
    Free WIFI will be available at the Conference. Please be aware that public WIFI capacity is
    always limited and therefore limited to email and web browsing activity.
    Network:           icle18
    Password:          icle18madrid

    1:1 MEETINGS
    During the conference days you will have an opportunity to arrange 1:1 meetings with
    Faculty members, Exhibitors and other Attendees you want to meet in person and share
    your experience and ideas in the field of lymphocyte engineering. Just install the ICLE
    Meeting Diary interactive mobile app to your smartphone or portable devices and:
    • Access your Personal Meeting Agenda;
    • Request/ Accept or Decline meeting invitations with the people you want
      at the designated meeting time slots available during the coffee and lunch
      breaks in the Exhibition & Networking area.
    • Check the Full Conference Agenda;
    • Keep the Conversation Going in Social Media
    To run the application you may choose one of the options below:
    1 Scan the QR code. Your ID is the email you used to register for the conference; The default
      password is ICLE2018. To change the default pass and set your personal one you may
      click on the forgotten password link and follow the steps;
    2 Click on the link that will be sent to your personal email. You will be automatically logged
      into the app.
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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

REFRESHMENTS AND LUNCH
Refreshments and lunch will be served in the Exhibition & Catering Hall (Zaragoza Hall 3&4,
Level 4) at the times indicated on the timetable.

ABSTRACTS
All accepted abstracts are published in the November issue of Human Gene Therapy online
journal. A link to the journal is also published on the conference website.

PHOTOGRAPHY AND RECORDING PRIVILEGES
No photographs, video recording or audio recording is permitted in the scientific sessions
unless otherwise authorized by the Advisory Board.

MOBILE PHONES
Mobile phones must be muted during all sessions.

GUEST ATTENDANCE POLICY
All event activities (including meal functions, exhibition, etc.) are exclusively reserved for
registered attendees. Non-registered guests (including children, family members, col-
leagues, etc.) are not allowed in any of the event areas. Badges provided at registration are
required for entrance into all functions and will be strictly enforced.

LIABILITY AND INSURANCE
The Conference Secretariat and Organizers cannot accept liability for personal accidents or
loss of or damage to private property of participants. Participants are advised not to leave
their personal belongings unattended in session halls and throughout the Venue hotel.

SMOKING POLICY
Smoking is prohibited throughout the Venue hotel. Please be considerate of the rules of the
venue and your fellow-participants.

SOCIAL MEDIA
Join us on Facebook, Twitter and LinkedIn to start advancing research, clinical translation
and treatment in immuno-gene therapy across related disease specialties and keep the
conversation going after the event.
Use the conference hashtag #ICLE2018 to let people know what is going on during the event!

LOCAL INFORMATION
Please visit the hotel concierge for local information.

CONFERENCE SECRETARIAT
                   www.lymphocyte.kenes.com
                   Rue François- Versonnex 7, 1207 Geneva, Switzerland.
                   www.kenes.com

#ICLE2018                                                                                                           7
INFORMATION FOR ORAL PRESENTERS
    AND CHAIRPERSONS
    Please check the PROGRAMME in case of last minute scheduling changes.

    PRESENTATION FORMAT
    Invited Speakers will have 20 minutes for presentation which include 15 minutes for their
    talk + 5 minutes for questions and discussion.

    Oral Abstract Presenters will have 15 minutes for presentation which include 10 minutes
    for their talk + 5 minutes for questions and discussion.

    ORAL COMMUNICATIONS
    As Oral Communication speakers you are requested to arrive at least 1 hour before your
    session in order to provide the Speaker Ready Desk with your slides. If using a Power Point
    (or any other computer) presentation, please note you need to bring it on a memory stick
    (using the USB port in the computer).
    We strongly recommend not using own computers for presentation – please note that the
    time used for connecting own computer will be deducted from the total presentation time.
    However, if you choose to use your own computer please note it has to have HDMI socket
    for external signal. In such a case please come to update the AV support about it and come
    to test in the session hall during a coffee or lunch break prior to your session, at least 30
    minutes before the start of the session.

    VIDEO FILMS IN YOUR PRESENTATION
    If combining video films with PowerPoint, please make sure to check it in the session hall
    during a coffee or lunch break prior to your session, at least 30 minutes before the start of
    the session.

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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

INFORMATION FOR E-POSTER
PRESENTERS AND CHAIRPERSONS
There will be E-Poster Presentation Sessions in the coffee breaks during all conference
days as well as in the lunch break on 15th September. All participants are invited to
the E-Poster Presentation Station to hear the selected short presentations and to ask
questions.
All E-Posters can also be viewed during the conference days on a separate E-Poster Station,
located in the E-Poster Viewing Area in the same hall.
Location: Zaragoza Hall 3&4, Level 4
ePoster Session                               Date & Time             Chairman
Session 1                                     13th September
                                                                      Sonia Guedan (Spain)
Hematological Malignancies                    16:00 – 16:40

Session 2
                                              14th September
Safety switches & controlled expression/                              Tal Akriv (Israel)
                                              10:20 – 11:00
Hematological Malignancies

Session 3
                                              14th September
Target & Receptor discovery/Non-viral                                 Natalia Gritsenko (Israel)
                                              15:30 – 16:10
vectors

Session 4                                     15th September
                                                                      Yuval Raviv (Israel)
Safety switches and controlled expression     10:00 – 10:30

Session 5                                     15th September
                                                                      Daniel Nataf (Israel)
Solid Tumors/Other                            12:00 – 13:30

#ICLE2018                                                                                                       9
POSTER PRESENTATIONS
     The main opportunity for standard poster viewing and discussion is during the lunch and
     coffee breaks each day and authors are requested to stand by their poster to answer
     questions.
     Location: Zaragoza Hall 3&4, Level 4

     POSTERS
     Topic /Abstract Title                                                                          Board
     GENOME EDITING AND ALLOGENEIC ACT
     TIM-3 TARGETING CAR-T CELLS, AS A POTENTIAL IMMUNOTHERAPY FOR AML.                               1
     S. Lee, Z.Y. Ye, H. Huang, M.K. Soh, Cheng-I WANG (Singapore)

     HEMATOLOGICAL MALIGNANCIES
     KINETICS OF EXPANSION AND PHENOTYPE OF UCART19 CELLS IN THE FIRST CLINICAL                       2
     TRIAL OF A UNIVERSAL ALLOGENEIC CAR-T CELL PRODUCT
     A. Jozwik, C. Graham, A. Dunlop, K. Sanchez, D. Yallop, P. Patten, A. Pagliuca, G. Mufti, L.
     Gauthier, A. Zinai, S. Dupouy, S. Balandraud, C. Poirot, A. Philippe, F. Simon, C. Konto, B.
     Ganguly, T. Pertel, B. Sasu, R. Benjamin (UK)
     CLINICAL MANUFACTURING OF CHIMERIC ANTIGEN RECEPTOR (CAR) T CELLS FOR                            3
     ADOPTIVE IMMUNOTHERAPY
     K. Mølgaard, Ö. Met, I.M. Svane (Denmark)
     DETECTION NPM1 MUTATION AND CLINICAL CHARACTERS IN ADULT PATIENTS WITH                           4
     ACUTE MYELOID LEUKEMIA IN THE MONGOLIA
     S. Oyunbileg, B. Davaabayar, N. Oyunderlger, B. Sevjidma (Mongolia)
     DETECTION FLT3/ITD MUTATION AND CLINICAL CHARACTERS IN ADULT PATIENTS WITH                       5
     ACUTE MYELOID LEUKEMIA IN THE MONGOLIA
     S. Oyunbileg, B. Davaabayar, B. Khishigjargal, N. Oyundelger, B. Sevjidmaa (Mongolia)

     OTHER
     CUMULATIVE CHEMOTHERAPY DEPLETES NAÏVE T CELLS : IMPLICATIONS FOR                                6
     CELLULAR THERAPY
     D. Barrett, R. Das, J. Storm (USA)
     EXPRESSION AND FUNCTIONAL DYNAMICS OF TRPV1 ION CHANNEL IN THE CANINE PBMC                       7
     J. Bujak, P. Bednarczyk, D. Kosmala, I. Szopa, K. Majchrzak (Poland)

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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

CLINICAL-GRADE ASPERGILLUS-SPECIFIC T CELLS: SWITCH FROM MAGNETIC SELECTION                                 8
WITH LYSATE TO SHORT TERM EXPANSION PROCEDURE WITH PEPTIDE POOLS
C. Hoffmann-Freimüller, R. Geyeregger, S. Tischer, N. Frank, J. Stemberger, B. Maecker-
Kolhoff, R. Blasczyk, G. Fritsch, B. Eiz-Vesper (Austria)
COMPREHENSIVE ANALYSIS OF STARTING MATERIALS FOR GENE-MODIFIED CELL-                                        9
BASED IMMUNOTHERAPIES
A. Luostarinen, S. Ylä-Herttuala, J. Nystedt, T. Kaartinen (Finland)
OPTIMISATION OF CYTOKINE-DEPENDENT EXPANSION FOR TCR-TRANSDUCED                                            10
AUTOLOGOUS T-CELLS
R. Petrovic, D. Biziato, A. Toufexi, J. Kerby (UK)
CTSTM DYNABEADSTM CD3/CD28 ENABLES SIMULTANEOUS ONE-STEP NAÏVE AND                                         11
EARLY MEMORY T ISOLATION AND ACTIVATION
T.E. Stav-Noraas, R. Hartberg, T. Looney, L. Miller, G. Lowman, H. Almåsbak (Norway)
IMPACT OF THE SIGNAL STRENGTH AND TEMPERATURE DURING T LYMPHOCYTES                                         12
ACTIVATION AND PROLIFERATION - STUDY ON COMPANION DOG MODEL
I. Szopa, J. Bujak, A. Łabedz, K. Majchrzak (Poland)

SAFETY SWITCHES AND CONTROLLED EXPRESSION
PRECLINICAL OPTIMIZATION OF CHIMERIC ANTIGEN RECEPTOR CD123-EXPESSING                                      13
T-CELLS FOR ACUTE MYELOID LEUKEMIA AND INCLUSION OF AN INDUCIBLE CASPASE
9 SUICIDE GENE TO INCREASE SAFETY
M.L. Baroni (Spain)

SOLID TUMORS
GENETIC ENGINEERING OF TUMOUR INFILTRATING LYMPHOCYTES (TIL) WITH A NOVEL                                  14
GROWTH FACTOR RECEPTOR FOR TREATMENT OF SOLID TUMOURS
M. Le Brocq, G. Owens, J. McCaffrey, A. Patel, R.D. Guest, R.E. Hawkins, J.S. Bridgeman (UK)
A CAR-T CELL THERAPY TARGETING GLYPICAN-3 POSITIVE HEPATOCELLULAR                                          15
CARCINOMA
B. Wang, E. Ngoh, M.K. Soh, Y. Yeap, H. Huang, H.C. Tan, Y. Hu, C.I. Wang (Singapore)

TARGET AND RECEPTOR DISCOVERY
OPTIMISING CHIMERIC ANTIGEN RECEPTOR T-CELL THERAPY FOR HAEMATOLOGICAL                                     16
MALIGNANCIES
A. Bahashwan, M. Kalaitsidou, A. Schutt, D. Gilham, R. Hawkins (UK)

#ICLE2018                                                                                                                11
SCIENTIFIC PROGRAMME
     THURSDAY, 13 SEPTEMBER 2018
     11:00 – 11:15 Zaragoza I & II
                   WELCOME ADDRESS
                   Adi Barzel (Israel)
     11:15 – 13:00 SESSION 1
                   CLINICAL UPDATES IN HEMATOLOGICAL MALIGNANCIES
                   Chair: Michal Besser (Israel)
     11:15         KEYNOTE LECTURE
                   ACCELERATING THE EVOLUTION OF CELL THERAPY
                   Malcolm Brenner (USA)
     12:00         CLINICAL EXPERIENCE WITH LOCALLY-PRODUCED CAR-T AND TIL
                   Michal Besser (Israel)
     12:20         CAR-T THERAPY FOR B CELL MALIGNANCIES
                   Jennifer Brogdon (USA)
     12:40         XCELLERATE - NEXTGEN MANUFACTURING PLATFORM FOR IMPROVED CAR T
                   CELLS
                   Lothar Germeroth (Germany)
     13:00-14:30   Zaragoza III & IV
                   Lunch, Poster Viewing, Networking & Exhibition
     14:30 – 16:00 Zaragoza I & II
                   SESSION 2
                   FIGHTING SOLID TUMORS WITH ENGINEERED LYMPHOCYTES
                   Chair: Marcela Maus (USA)
     14:30         TARGETING OF MULTIPLE TUMORS AND VIRAL-INFECTED CELLS USING T-CELLS
                   ENGINEERED TO EXPRESS NCR-BASED CHIMERIC RECEPTORS
                   Cyrille Cohen (Israel)
     14:50         ENGINEERING T CELLS FOR THE IMMUNOTHERAPY OF SOLID TUMORS
                   Stephen Gottschalk (USA)
     15:10         CAR T CELLS IN GLIOBLASTOMA AND OTHER SOLID TUMORS
                   Marcela Maus (USA)
     15:30         ABSTRACT ORAL PRESENTATION
                   REDIRECTING SWITCHABLE UNICAR T CELLS FOR ELIMINATION OF
                   RADIORESISTANT CANCER CELLS
                   A. Feldmann, C. Arndt, R. Bergmann, N. Berndt, J. Jureczek, S. Albert, D. Lindner,
                   S. Koristka, J. Steinbach, G. Ehninger, M. Krause, I. Kurth, A. Dubrovska, M. Bachmann
                   (Germany)
     15:45         ABSTRACT ORAL PRESENTATION
                   IMPROVING THE EXPANSION AND PERSISTENCE OF CAR T CELLS USING
                   ONCOLYTIC RHABDOVIRAL VACCINE VECTORS IN PAEDIATRIC SARCOMAS
                   F. Zemp, J. Rajwani, K. Potts, S. Van, D. Mahoney (Canada)
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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

16:00-16:40   Zaragoza III & IV
              Coffee Break, ePoster Presentations & Poster Viewing, Networking & Exhibition
              EPOSTER SESSION 1
              HEMATOLOGICAL MALIGNANCIES
              Chair: Sonia Guedan (Spain)
16:10         LYMPHOCYTE-SPECIFIC DELIVERY OF PROTEIN THERAPEUTICS BY GENETICALLY
              ENGINEERED EXOSOMES
              D. Levy, M. Do, B. Lu (USA)
16:20         SUPER-RESOLUTION MICROSCOPY DSTORM REVEALS CD19DIM EXPRESSION ON A
              SUBSET OF MYELOMA CELLS THAT CAN BE TARGETED WITH CD19-CAR T CELLS
              T. Nerreter, S. Letschert, S. Doose, S. Danhof, H. Einsele, M. Sauer, M. Hudecek
              (Germany)
16:30         ENGINEERING CD38LOW NATURAL KILLER CELLS TO TRANSIENTLY EXPRESS
              CD16 (F158V) M-RNA ENHANCES THE CYTOTOXIC POTENTIAL OF DARATUMUMAB
              AGAINST MULTIPLE MYELOMA WITH MINIMAL NK CELL FRATRICIDE
              S. Sarkar, S. Chauhan, A. Natoni, J. Daly, R. Henderson, M. O'Dwyer (Ireland)
16:40 – 17:50 Zaragoza I & II
              SESSION 3
              NON VIRAL VECTORS AND TRANSPOSONS
              Chair: Michael Hudecek (Germany)
16:40         ENGINEERING LYMPHOCYTES WITH RNAI NANOMEDICINES
              Dan Peer (Israel)
17:00         NEW TARGETS AND TECHNOLOGIES FOR CAR-T
              Michael Hudecek (Germany)
17:20         Abstract Oral presentation
              VAPOR NANOBUBBLE PHOTOPORATION AS A NEW, SAFE AND EFFICIENT
              TECHNIQUE FOR THE DELIVERY OF SMALL INTERFERING RNA TO CYTOTOXIC T
              CELLS
              J. Van Hoeck, L. Wayteck, R. Xiong, K. Braeckmans, S.C. De Smedt, K. Raemdonck
              (Belgium)
17:35         CRISPR/CAS9 UNITES WITH SLEEPING BEAUTY TO GENERATE CAR T CELLS WITH
              ENHANCED THERAPEUTIC INDEX FOR FIGHTING AGAINST IMMUNOSUPPRESSIVE
              TUMOR MICROENVIRONMENT
              R. Monjezi, C. Miskey, T. Stüber, E. Grueso, M. Schleef, M. Schmeer, H. Einsele, Z. Ivics,
              M. Hudecek (Germany)
18:30         NETWORKING WELCOME RECEPTION
              More info on p. 24.

#ICLE2018                                                                                                              13
FRIDAY, 14 SEPTEMBER 2018
     08:30 – 10:20 Zaragoza I & II
                   SESSION 4
                   GENOME EDITING AND ALLOGENEIC ACT
                   Chair: Ayal Hendel (Israel)
     08:30         GENE EDITED ALLOGENEIC T CELL THERAPIES
                   Waseem Qasim (UK)
     08:50         CRISPR GENE CORRECTION FOR HEMATOPOIETIC STEM CELLS
                   Ayal Hendel (Israel)
     09:10         GENE EDITED UNIVERSAL OFF-THE-SHELF IMMUNOTHERAPIES: A FUTURE-
                   DEFINING SHIFT IN SIMPLICITY, AVAILABILITY, AND COST-EFFECTIVENESS
                   André Choulika (USA)
     09:30         MEGATAL TARGET GENE DISRUPTION FOR THE ENHANCEMENT OF T CELL
                   THERAPIES
                   Michael Magee (USA)
     09:50         ABSTRACT ORAL PRESENTATION
                   GENERATION OF "OFF-THE SHELF" αCD19-CAR T CELLS FOR THE TREATMENT OF
                   B CELL MALIGNANCIES
                   M. Tristán-Manzano, R. Martín-Guerra, N. Maldonado, S. Sánchez-Hernández,
                   M. Cortijo, C. Marañon, C. Herrera, M.D. Carmona, J.A. Marchal, K. Benabdellah,
                   F. Martín (Spain)
     10:05         ABSTRACT ORAL PRESENTATION
                   GENOME EDITING USING ARCUS HOMING ENDONUCLEASES AND AN OPTIMIZED
                   MANUFACTURING PROCESS FOR PRODUCTION OF OFF-THE-SHELF ALLOGENEIC
                   CAR T THERAPIES
                   B. Mccreedy, A. Martin, D. MacLeod, C. Pham, A. Brown, J.A. Hux, J. Lape, C. Beard,
                   J. Smith, K. Nguyen, V. Senyukov, J. Anthony, K. Sivarajan, L. Som, M. Triggiano,
                   D. Jantz (USA)
     10:20 – 11:00 Zaragoza III & IV
                   Coffee Break, ePoster Presentations & Poster Viewing, Networking & Exhibition
                   EPOSTER SESSION 2
                   SAFETY SWITCHES & CONTROLLED EXPRESSION/
                   HEMATOLOGICAL MALIGNANCIES
                   Chair: Tal Akriv (Israel)
     10:30         HIGHLY EFFICIENT AND SELECTIVE CAR-GENE TRANSFER USING CD4- AND CD8-
                   TARGETED LENTIVIRAL VECTORS
                   A. Jamali, L. Grimm, C.J. Buchholz, J. Hatrmann (Germany, Iran)
     10:40         POST-TRANSPLANT IMMUNOTHERAPY WITH WT1-SPECIFIC CTLS FOR HIGH-RISK
                   ACUTE MYELOGENOUS LEUKEMIA: A PROSPECTIVE CLINICAL PHASE I/II TRIAL
                   H.J. Kim, H.J. Sohn, J.A. Hong, H.J. Lee, D.H. Sohn, C.A. Shin, S.J. Hyun, C.H. Hong,
                   H. Choi, A.R. Shin, H.I. Cho, W.S. Min, T.G. Kim (Republic of Korea)

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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

11:00-12:30   Zaragoza I & II
              SESSION 5
              NEW HORIZONS FOR LYMPHOCYTE AND HSC ENGINEERING
              Chairs: Matthew Porteus (USA)
                      Beatriz Martin-Antonio (Spain)
11:00         HIGHLY EFFICIENT EDITING OF PRIMARY HUMAN BLOOD CELLS BY HOMOLOGOUS
              RECOMBINATION
              Matthew Porteus (USA)
11:20         ENGINEERING REGULATORY T-CELL THERAPEUTICS
              Andrew Scharenberg (USA)
11:40         ENGINEERING T CELLS AND B CELLS EX VIVO AND IN VIVO
              Adi Barzel (Israel)
12:00         ABSTRACT ORAL PRESENTATION
              NK CELLS FROM DIFFERENT SOURCES AS A PROMISING ALTERNATIVE FOR CAR-
              BASED IMMUNOTHERAPY AGAINST HEMATOLOGICAL CANCERS
              L. Herrera, A. Boronat, P. Zuñiga, I. Martin, J. Anguita, B. Marzal, S. Santos,
              M.A. Vesga, F. Borrego, M. Juan, C. Eguizabal (Spain)
12:15         ABSTRACT ORAL PRESENTATION
              HARNESSING CD8 CO-RECEPTOR FUNCTION FOR IMMUNOTHERAPY WITH LOW-
              AVIDITY TCR TRANSGENIC T CELLS
              G. Bajwa, E. Hoyer, D. Brenner, M. Martinez-Paniagua, N. Nouraee, F. Sadeghi,
              I. Gruber, M. Hebeisen, N. Rufer, M.F. Wu, W. Tao, N. Varadarajan, M. Brenner, C. Arber
              (Switzerland)
12:30-14:00   Zaragoza III & IV
              LUNCH, PIPELINE PRESENTATIONS, NETWORKING & EXHIBITION
              PIPELINE PRESENTATIONS
12:40         GENEWERK, TOWARDS SAFER THERAPIES
              Wei Wang (GeneWerk Germany)
12:55         LENTIVIRAL VECTORS: SAFETY AND MANUFACTURING RELATED ASPECTS
              Juan Carlos Ramírez (VIVEbiotech. Spain)

#ICLE2018                                                                                                              15
14:00 – 15:30 Zaragoza I & II
                   SESSION 6
                   ENGINEERING LYMPHOCYTES TO CLEAR INFECTIONS
                   Chair: Paula Cannon (USA)
     14:00         ENGINEERING RESISTANCE AND PERSISTENCE- T CELLS FIGHT BACK AGAINST
                   HIV
                   Bruce Levine (USA)
     14:20         ENGINEERING HSC AND LYMPHOCYTES TO SUPPRESS HIV
                   Paula Cannon (USA)
     14:40         ENGINEERING ANTIVIRAL CAR T CELL IMMUNITY THROUGH HEMATOPOIETIC
                   STEM/PROGENITOR CELLS
                   Scott Kitchen (USA)
     15:00         ABSTRACT ORAL PRESENTATION
                   EVALUATION OF A FULLY HUMAN CAR FOR THE TREATMENT OF HEPATITIS B
                   VIRUS INFECTION IN AN IMMUNOCOMPETENT MOUSE MODEL
                   M. Festag, K. Wisskirchen, J. Hasreiter, S. Schreiber, H. Abken, U. Protzer (Germany)
     15:15         ABSTRACT ORAL PRESENTATION
                   ANTIBODY-SECRETING T CELLS ENGINEERED FOR TRIPARTATE IMMUNE
                   RESPONSE AGAINST THE HIV RESERVOIR
                   A. Powell, Y. Ren, M. Korom, H. Goldstein, D.F. Nixon, C. Bollard, R.M. Lynch,
                   R.B. Jones, C.R. Cruz (USA)
     15:30-16:10   Zaragoza III & IV
                   Coffee Break, ePoster Presentations & Poster Viewing, Networking & Exhibition
                   EPOSTER SESSION 3
                   TARGET & RECEPTOR DISCOVERY/NON-VIRAL VECTORS
                   Chair: Natalia Gritsenko (Israel)
     15:40         DIMERISATION IS ESSENTIAL FOR HIGH LEVEL CYTOKINE SECRETION BY T CELLS
                   BEARING A CD28-BASED 2ND GENERATION CAR: POTENTIAL RELEVANCE FOR
                   CONTROLLING CYTOKINE RELEASE SYNDROME
                   A. Kilgallon, M. Kalaitsidou, G. Kueberuwa, A. Schutt, D.E. Gilham, R.E. Hawkins, (UK)
     15:50         SURFACE MOBILITY AND CLUSTER FORMATION OF VARIOUS MELANOMA
                   ASSOCIATED ANTIGENS MODULATES CHIMERIC ANTIGEN RECEPTOR (CAR)
                   MODIFIED T CELL ACTIVATION
                   A. Szoor, A. Holzinger, A. Pham, H. Abken, G. Vereb (Hungary)
     16:00         GENERATION OF WILMS’ TUMOR 1 TCR-REDIRECTED CD4 AND GAMMA-DELTA T
                   CELLS BY RNA ELECTROPORATION AND CO-TRANSFER OF CD8 MRNA
                   D. Campillo, G. Roex, H.H. Van Acker, Z.N. Berneman, E. Lion, V.F.I. Van Tendeloo
                   (Belgium)

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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

16:10 – 17:40 Zaragoza I & II
              SESSION 7
              OPTIMIZING RECEPTOR AND VECTOR DESIGN
              Chairs: Gideon Gross (Israel)
                      Julio Delgado (Spain)
16:10         FROM A CANCER CAUSING VIRUS TO A SAFER TOOL FOR GENE THERAPY AND
              IMMUNOTHERAPEUTICS
              Axel Schambach (Germany)
16:30         RECEPTOR-TARGETED VIRAL VECTORS IN IMMUNOTHERAPY
              Christian Buchholz (Germany)
16:50         CAR T CELL THERAPY: THE CD19 PARADIGM
              Xiuyan Wang (USA)
17:10         ABSTRACT ORAL PRESENTATION
              OVERCOMING FRATRICIDE TO DELIVER A CLINICAL GRADE CAR T CELL PRODUCT
              S. Bornschein, B. Demoulin, E. Breman, A. Michaux, J. Houssa, F. Huberty, B. Violle,
              C. Jacques-Hespel, C. Marchand, J. Marijsse, T. Nguyen, N. Ramelot, D. Daro,
              V. Steenwinckel, S. Agaugue, D. Gilham (Belgium)
17:25         ABSTRACT ORAL PRESENTATION
              CELL THERAPY WITH ENGINEERED HLA-A2 SPECIFIC CAR-CD8+TREGS TO AVOID
              TRANSPLANT REJECTION
              S. Bezie, N. Vimond, V. Daguin, F. Bell ier-Waast, F. Duteille, M. Levings, B. Charreau,
              I. Anegon, C. Guillonneau (France)

#ICLE2018                                                                                                              17
SATURDAY, 15 SEPTEMBER 2018
     08:30 – 10:00 Zaragoza I & II
                   SESSION 8
                   SAFETY SWITCHES & CONTROLLED EXPRESSION
                   Chairs: Daniel Powell (USA)
                           Francisco Martin Molina (Spain)
     08:30         STRATEGIES FOR QUANTITATIVE CONTROL OF REDIRECTED GENE-ENGINEERED T
                   CELL ACTIVITY
                   Daniel Powell (USA)
     08:50         USING MRNA FOR IMPROVING T CELL PERFORMANCE IN ADOPTIVE CELL
                   THERAPY
                   Gideon Gross (Israel)
     09:10         ISOLATION AND IDENTIFICATION OF OPTIMAL T CELLS AND RECEPTORS FOR
                   ADOPTIVE IMMUNOTHERAPY
                   Dirk Busch (Germany)
     09:30         ABSTRACT ORAL PRESENTATION
                   ARMING HUMAN REGULATORY T CELLS FROM HEALTHY DONORS OR MULTIPLE
                   SCLEROSIS PATIENTS WITH SWITCHABLE CHIMERIC ANTIGEN RECEPTORS FOR
                   THE TREATMENT OF INFLAMMATORY DISEASES
                   A. Kegler, S. Koristka, R. Bergmann, C. Arndt, A. Feldmann, S. Albert, G. Ehninger,
                   M. Bornhäuser, M. Schmitz, K. Akgün, T. Ziemssen, M. Bachmann (Germany)
     09:45         ABSTRACT ORAL PRESENTATION
                   DEVELOPMENT OF CLINICAL-GRADE DOXYCYCLINE-INDUCIBLE CAR T CELLS
                   N. Maldonado, M. Tristán-Manzano, S. Sánchez-Hernández, C. Marañón, C. Herrera,
                   M.D. Carmona, F. Martin, K. Benabdellah (Spain)
     10:00 – 10:30 Zaragoza III & IV
                   Coffee Break, ePoster Presentations & Poster Viewing, Networking & Exhibition
                   EPOSTER SESSION 4
                   SAFETY SWITCHES AND CONTROLLED EXPRESSION
                   Chair: Yuval Raviv (Israel)
     10:10         ELIMINATION OF CAR-ENGINEERED LYMPHOCYTES BY AUTOLOGOUS ANTI-CAR T
                   CELLS
                   S. Koristka, P. Ziller-Walter, A. Feldmann, C. Arndt, A. Kegler, S. Albert, G. Ehninger,
                   M. Bornhäuser, M. Bachmann (Germany)
     10:20         DEVELOPMENT AND CHARACTERIZATION OF NOVEL ANTI-GD2 TARGET MODULES
                   FOR RETARGETING OF UNIVERSAL CAR T CELLS TOWARD GD2 EXPRESSING
                   TUMORS
                   N. Mitwasi, A. Feldmann, R. Bergmann, N. Berndt, C. Rössig, M. Bachmann (Germany)

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INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

10:30-12:00   Zaragoza I & II
              SESSION 9
              NEW TARGETS AND NEW RECEPTORS
              Chairs: Dinorah Morvinski (Israel)
                      Rafael Duarte (Spain)
10:30         DESIGNER T CELLS FOR IMMUNOTHERAPY OF CANCER
              Wolfgang Uckert (Germany)
10:50         P32, A NOVEL TARGET OF CAR T-CELL THERAPY FOR GLIOMAS
              Dinorah Friedmann-Morvinski (Israel)
11:10         DAYS OF THUNDER: CHALLENGES WHEN SEEKING NEW CARS
              Barbara Savoldo (USA)
11:30         ABSTRACT ORAL PRESENTATION
              CHIMERIC ANTIGEN RECEPTORS WITH A MYD88 AND CD40 ENDODOMAIN ENDOW
              T CELLS WITH SUPERIOR ANTITUMOR ACTIVITY
              B. Prinzing, G. Krenciute, S. Gottschalk (USA)
11:45         ABSTRACT ORAL PRESENTATION
              A SEMI-AUTOMATED METHOD FOR THE ISOLATION AND INITIAL
              CHARACTERIZATION OF NEOANTIGEN-SPECIFIC T CELL RECEPTORS
              A. Bracher, D. Sommermeyer (Germany)
12:00-13:30   Zaragoza III & IV
              Lunch, ePoster Presentations & Poster Viewing, Networking & Exhibition
              EPOSTER SESSION 5
              SOLID TUMORS/OTHER
              Chair: Daniel Nataf (Israel)
12:10         TUMOR-ASSOCIATED FIBROBLASTS: GENERIC TARGET OF CANCER
              IMMUNOTHERAPY
              F. Bojin, O. Gavriliuc, V. Paunescu (Romania)
12:20         ACTIVE MODULATION OF THE TUMOR MICROENVIRONMENT BY ENGINEERED T
              CELLS
              E. Ceccarello, T. Tabaglio, D.W.K. Boon, W.K.L. Teo, D.Z.M. Tan, J.J.Y. Aw, S.S.F. Koh,
              A. Pavesi, E. Guccione, A. Bertoletti (Singapore)
12:30         TARGETING MULTIPLE TUMORS AND VIRAL INFECTED CELLS USING T-CELLS
              ENGINEERED TO EXPRESS A NCR-BASED CHIMERIC ANTIGEN RECEPTOR
              V. Eisenberg, S. Hoogi, S. Mayer, K. Shamalov, T. Barliya, A. Pogrador, C.J. Cohen
              (Israel)
12:40         FINE-TUNING OF CD28 SIGNALING MOTIFS INCREASES ANTI-TUMOR ACTIVITY
              AND PERSISTENCE OF CHIMERIC ANTIGEN RECEPTOR-MODIFIED T CELLS
              S. Guedan, A. Posey, A. Wing, V. Casado-Medrano, C. Shaw, R.M. Young, C.H. June
              (Spain, USA)

#ICLE2018                                                                                                                19
12:50         CAR-T CELL IMMUNOTHERAPY IN SOLID TUMORS: COLORECTAL CANCER
                   E. Ponterio, C. Valvo, M. Cappellari, L. Pasquini, A. Boe, P. Romania, E. Petrucci,
                   C. Amoreo, M.R. Sciuto, R. Dattilo, E. Pilozzi, L. Ricci-Vitiani, M. Biffoni, R. De Maria,
                   T.L. Haas (Italy)
     13:00         ADOPTIVE T-CELL IMMUNOTHERAPY FOR THE TREATMENT OF GLIOBLASTOMA
                   L. Rousso Noori (Israel)
     13:10         IMPROVING CEA-TARGETED CAR-T CELL THERAPY FOR SOLID TUMOURS
                   W. Zheng, G. Kueberuwa, E. Cheadle, A. Armstrong, R.E. Hawkins (UK)
     13:20         ROLE PLAY BY GALECTIN-1 IN REGULATORY T-CELL IMMUNOTHERAPY FOR
                   MODULATION OF IMMUNE RESPONSES IN THE TREATMENT OF AUTOIMMUNE
                   DISEASES
                   R. Fernadez-Perez, O. Aberquilla-Fernandez, R. Sanchez-Dominguez, J.A. Bueren,
                   M.I. Garin (Spain)
     13:30 – 15:35 Zaragoza I & II
                   SESSION 10
                   CONCLUDING KEYNOTE LECTURES
                   Chair:   Adi Barzel (Israel)
     13:30         WHAT’S NEXT? FROM PERSONALIZED TO “OFF THE SHELF” CARS
                   Anat Globerson Levin (Israel)
     13:50         KEYNOTE LECTURE
                   T CELL THERAPY FOR CANCER
                   Carl June (USA)
     14:35         PANEL DISCUSSION
                   LYMPHOCYTE ENGINEERING – WHAT’S NEXT?
                   Moderator: Adi Barzel (Israel)
                   Panelists:
                   Carl June (USA)
                   Malcolm Brenner (USA)
                   Jennifer Brogdon (USA)
                   André Choulika (USA)
     15:35         CONCLUSIONS AND CLOSING ADDRESS

20
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

RECOGNITION, ACKNOWLEDGEMENTS
AND INDUSTRY SUPPORT
SUPPORTERS & EXHIBITORS

START-UP INDUSTRY HUB

SUPPORTER OF THE SCIENTIFIC SESSIONS

PROGRAMME PARTNERS

MEDIA PARTNERS

#ICLE2018                                                                                  21
EXHIBITOR & SUPPORTER
     COMPANY PROFILES
     CELLGENIX GMBH
     Am Flughafen 16
     79108 Freiburg
     Germany
     Booth # 2
     www.cellgenix.com

     GENEWERK GMBH                                               
     Im Neuenheimer Feld 582
     D-69120 Heidelberg
     Germany
     Booth # TT5
     www.genewerk.com
     GeneWerk GmbH offers an integral platform for the analysis of gene therapy vectors
     and designer nucleases safety in preclinical and clinical studies. DNA- or RNA-fusion
     sequences are detected via PCR- and target enrichment-based approaches combined
     with customized bioinformatics of next-generation sequencing data, thereby enabling
     the identification of gene therapy vectors integration sites and gene editing on-/off-target
     analysis. GeneWerk GmbH applications for immunotherapy also comprise the use of PCR-
     or target enrichment-based workflows coupled to high-throughput sequencing for T and B
     cell immunerepertoire studies. Dedicated bioinformatics complement our services to make
     the most of our customer’s data and provide customized solutions.

22
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

NOVARTIS INSTITUTES FOR BIOMEDICAL 
RESEARCH, INC.
181 Massachusetts Ave
4A-011-01-A
Cambridge, MA 02139
USA
www.novartis.com/our-science/novartis-institutes-biomedical-research
Our mission is to discover new ways to improve and extend people`s lives. Our vision is to
be a trusted leader in changing the practice of medicine.
We use science-based innovation to address some of society’s most challenging healthcare
issues. We discover and develop breakthrough treatments and find new ways to deliver
them to as many people as possible.

VIVEBIOTECH
Mikeletegi Pasealekua 81,
San Sebastián 20009,
Gipuzkoa
Spain
Booth # 6
www.vivebiotech.com
VIVEbiotech is a company fully specialized in lentiviral vectors with two areas of expertise:
1. GMP CDMO: GMP Contract Development and Manufacturing Organization specialized in
   lentiviral vector bioprocess development (from very early stages) and GMP manufacturing.
   Working with companies based in Europe and USA being flexible in terms of technical
   adaptation and slots availability.
2. Innovation: VIVEbiotech´ strategy is focused on providing a response to the main hurdles
   that currently exist within the gene therapy field:
   a.     The need for cost-effective processes.
   b.     The enhancement of the safety profile of viral vectors: worldwide licensed non-
   integrative episomal stable lentiviral vector (LENTISOMA).

#ICLE2018                                                                                                         23
ICLE WELCOME RECEPTION
     All delegates are welcome to an exciting welcome and networking reception on 13th
     September, hosted at Santiago Bernabéu Stadium.
     Pick Up Point: Melia Avenida America hotel lobby at 18:30
     Departure time: 18:45 – 19:00
     Transport & Location: There will be an organized bus service to Santiago Bernabeu
     Stadium, entrance: Gate 44
     Welcome reception: Emirates VIP Room 19:30 - 21:00
     Returning time: 21:00 - 21:30
     Drop off Points: Plaza de Espana and Melia Avenida America Hotel

24
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

GETTING TO MADRID DOWNTOWN
Melia Avenida America has good connections with the city centre.
BY BUS
The bus stop Ios Molinos is just next to the hotel from where you can take bus #146 to get to
Callao Square (Plaza del Callao).
BY METRO
The closest metro station is Suanzes, within a 15 min walk from the hotel. To get to the
center take line 5 (the green line, going to Casa de Campo) and travel to Gran Via or Callao
(Plaza del Callao). The cost for a single trip is EUR 2.
BY TAXI
If you prefer to take a taxi from Melia Avenida America, getting to the downtown costs about
EUR 20-25.

ATTRACTIONS & SIGHTSEEING
The Plaza Callao is famed as much for its role as the heart of cinematic and theatrical
Madrid as it is for its eternal bustle and art deco style. Architecture, cinema buffs, many
restaurants and trade centers, you will find much to discover here. The square is located
just off the world famous Gran Via - one of the city’s main arteries and most iconic avenue,
today known as the Spanish Broadway.
The symbol of Gran Via is located on Calle Alcalá: the Metropolis Building, designed by
Jules and Raymond Fevrier. In this area you will find luxury shops, boutiques and others.
The second section, up to Plaza Callao, has a more American style, with the Telefónica
building, Palacio de la Prensa and Palacio de la Música. The last branch reaches Plaza de
España, with modern landmark buildings such as Lope de Vega and Carrión Building, also
known as the Capitol. This is the Broadway Madrileña.
The Gran Vía reaches Plaza de España through the central fountain monument of Miguel de
Cervantes, surrounded by the Casa Gallardo, Edificio de la Compañía Asturiana de Minas,
Torre de Madrid (142 meters) and the Building España (117 meters).

GETTING TO ADOLFO SUÁREZ MADRID BARAJAS INTERNATIONAL AIRPORT
There is a free of charge shuttle service for the hotel guests, available upon request. To use
the shuttle, please go to hotel Reception and book your ride.

#ICLE2018                                                                                                        25
NOTES

26
INTERNATIONAL CONFERENCE ON LYMPHOCYTE ENGINEERING 2018

         THANK YOU
           FOR YOUR
      PARTICIPATION!
#ICLE2018                                                              27
SAVE THE DATE
           ADVANCING IMMUNO-GENE THERAPY
           ACROSS DISEASES

          INTERNATIONAL
          CONFERENCE ON
          LYMPHOCYTE
          ENGINEERING
          2019
          12-14 SEPTEMBER
          LONDON, UK

28 LYMPHOCYTE.KENES.COM
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