15-17 May 2019 Business Design Center, London World Advanced … · 2019-05-28 · 1 15-17 May 2019 Business Design Center, London World Advanced Therapies & Regenerative Medicine

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15-17 May 2019

Business Design Center, London

World Advanced Therapies & Regenerative

Medicine Congress 2019 Working Agenda

The World Advanced Therapies & Regenerative Medicine Congress is an exhibition and conference that

has grown to become one of the best and most established cell and gene events in the world today.

The 2019 edition will be in its 14th year and it continues to grow and attract a large audience of biotech,

pharma, start-ups, investors, research and technology companies from around the world. The

conference and exhibition will host 1000+ attendees, 250 speakers and 80 exhibition booths. Over 500

different global companies are represented every year at this event. 

Over the three days we will feature 150 presentations, 25 roundtable debates and several hours of

networking. The new pre-conference day on Wednesday 15th May features several important summits

on everything from Investment to CAR-T, Rare Diseases, Bioprocessing and Logistics. 

The event is an experience designed to help you get the most out of the 2 or 3 days that you are in

attendance. The exhibition will feature two full days of content as part of our Innovation Zone where

exhibition visitors will be able to hear about some of the latest innovations from start-ups, labs and

accelerators. 

In short, this year promises to be the most spectacular event yet.

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Please note that this is a working agenda that is updated monthly. To view the latest live version

please go to: https://www.terrapinn.com/congress/advanced-therapies-regenerative-medicine/

Table of Contents (click to navigate document) Confirmed Speakers .................................................................................................................. 3

Keynotes ................................................................................................................................ 3

Main agenda speakers (including seminars and workshops): ................................................. 3

Investors, VCs and Pitching Companies ................................................................................. 7

Cord Blood Speakers: ............................................................................................................ 7

Seminar theatre presenters: ................................................................................................... 8

Sponsored sessions: .............................................................................................................. 9

ESTEEMED ADVISORY BOARD ..........................................................................................10

AGENDA OVERVIEW ...........................................................................................................11

Pre-Conference Workshops and Summits Day Wednesday 15th May .......................................13

Gene Editing Summit .............................................................................................................13

Process Considerations for Cryogenic and Regenerative Medicine Commercialization .........14

CAR-T manufacture and imaging ..........................................................................................15

Standards development process/clinical trials .......................................................................15

AI in Healthcare Summit ........................................................................................................17

Gene Therapy for Rare Disease Summit (coming soon) ........................................................18

Cord Blood Workshops ..........................................................................................................19

Main Conference Agenda 16th –17th May ..................................................................................20

Keynote Sessions ..................................................................................................................20

Day 1: Thursday 16th May 2019 ............................................................................................20

Day 2: Friday 17th May 2019 .................................................................................................24

ADVANCED THERAPIES TRACKS ......................................................................................25

PRICING AND MARKET ACCESS ....................................................................................25

GENE MODIFIED CELL THERAPY ...................................................................................25

IMMUNOTHERAPY ...........................................................................................................30

IN VIVO GENE THERAPY .................................................................................................32

CELL THERAPY AND REGENERATIVE MEDICINE .........................................................34

CLINICAL DEVELOPMENT AND TRIALS .........................................................................36

MANUFACTURE AND BIOPROCESSING TRACKS .............................................................39

CELL THERAPY MANUFACTURING ................................................................................39

VIRAL VECTOR MANUFACTURING .................................................................................43

PITCH AND PARTNER .........................................................................................................48

CORD BLOOD ......................................................................................................................51

EXHIBITION VISITOR AGENDA: ON – FLOOR SEMINAR THEATRE .................................57

Day 1: Thursday 16th May 2019 .........................................................................................57

Day 2: Friday 17th May 2019 .............................................................................................60

https://www.terrapinn.com/congress/advanced-therapies-regenerative-medicine/

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Confirmed Speakers

Keynotes

Shinya Yamanaka, Director of CiRA (Center for iPS Cell Research and Application), Kyoto University Kathy High, President and Head of R&D, Spark Therapeutics Stephan Grupp, Chief, Cellular Therapy and Transplant Section, Children’s Hospital of Philadelphia Bob Smith, SVP, Global Gene Therapy Business, Rare Disease, Global Innovative Pharma, Pfizer TBC Andrew Obenshain, Head of Europe, bluebird bio Roy Baynes, SVP and Head Global Clinical Development, Chief Medical Officer, Merck, Sharp & Dohme James Miskin, CTO, Oxford Biomedica Andre Choulika, CEO, Cellectis Antonio Regalado, Senior Editor for Biomedicine, MIT Technology Review Derek Jantz, Co-founder and CSO, Precision BioSciences Fred Chereau, President and CEO, LogicBio Therapeutics Lothar Germeroth, SVP, Managing Director, Juno Therapeutics, a Celgene Company Jeffery L. Kutok, Chief Scientific Officer, Infinity Pharmaceuticals TBC Daniel Kraft, Founder & CMO, Exponential Medicine; Faculty Chair for Medicine and Neuroscience, Singularity University (14)

Main agenda speakers (including seminars and workshops):

Adam Gridley, President & CEO, Histogenics Adi Barzel, Senior Lecturer, Tel Aviv University, President, Israeli Society of Gene and Cell Therapy Ajan Reginald, Chief Executive Officer, Celixir Alan K. Smith, Executive Vice President, Technical Operations, Bellicum Pharmaceuticals Alain Vertès, Vice President, Alliance Management, Mesoblast Albert Ribickas, Bone Marrow Transplant Manager, Moffitt Cancer Center Alberto Sanchez Fueyo, Professor in Hepatology, Kings College London Alexander Natz, Secretary General, EUCOPE Andrea Spezzi, Chief Medical Officer and Co-founder, Orchard Therapeutics Angela Scott, Chief Operating Officer, TC Biopharm Annamaria Merico, Head of Technology Transfer, Fondazione Telethon Barry Rosen, Senior Principal Scientist/Director, AstraZenca Benjamin Shepherd, Director, Therapeutics, Organovo Benoît Champluvier, Advisor, Bone Therapeutics Bob Deans, CTO, BlueRock Therapeutics Bodil Willumsen, Supplier Relationship Manager, External Supply Operations, Cell and Gene Therapies, Novartis Brian Bronk, Head of Business Development, Rare Diseases & Rare Blood Disorders, Sanofi Brian Miller, CEO, Sentien Biotechnologies Chaim Lebovits, CEO, Brainstorm Cell Therapeutics Charles-Etienne De Cidrac, Director of Health Insurance, AXA Claudia Mitchell, Chief Operations Officer, Universal Cells, Inc.


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Cornelia Kasper, Full University Professor, BOKU Cristiana Boi, President of the European Membrane Society, Assistant Professor, DICAM, Alma Mater Studiorum, Università di Bologna Danielle Curfs, CEO, Medace Danilo Maddalo, Lab Head, Oncology Pharmacology, Novartis Institutes for BioMedical Research David Courtman, Director, Biotherapeutics Core Facilities, Ottawa Hospital Research Institute; CSO, Northern Therapeutics Davide Danovi, Director, Cell Phenotyping Platform, Centre for Stem Cells & Regenerative Medicine, King's College London David Morrow, Programme Manager, EATRIS David Pauza, CSO, American Gene Technologies Detlev Parow, Head of Department of Medicines, Therapeutic Appliances and Remedies, DAK Devyn Smith, Chief Strategy Officer & Head of Operations, Sigilon, Inc Didier Caizergues, Head of regulatory Affairs Department, Genethon Giorgio Iotti, Head of Program Leadership and Control, Rare Disease Unit, Chiesi Farmaceutici SpA Dominik Egger, Postdoctoral Research Scientist, Biotechnology, BOKU Douglas Losordo, EVP, Global Head of Research and Development & CMO, Caladrius Biosciences Elliot Dunster, Head of External Affairs, Association of the British Pharmaceutical Industry (ABPI) Eric Alton, Professor, Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London; Honorary Consultant Physician, Royal Brompton Hospital Eric Soller, Vice President of Corporate Development & Strategy, BlueRock Therapeutics Evren Alici, Assistant Professor and Group Leader, Haematology, Karolinska Institutet Farzin Farzaneh, Head of Molecular Medicine, Kings College London Felicia Pagliuca, VP Cell Biology R&D and Co-Founder, Semma Therapeutics Francesco Dazzi, Professor of Regenerative and Haematological Medicine, KHP Lead for Cellular Therapies, King’s College London Frank Barry, Professor, Cellular Therapy, The National University of Ireland; Senior Scientist, Toronto Arthritis Programme Frank Hecht, Vice President Marketing and Sales, CellGenix GmbH Fraser Wright, CTO, Axovant Frédéric Triebel, CSO & CMO, Immutep Limited. Giuseppe Mazza, Chief Executive & Scientific Officer, Engitix Graciana Diez-Roux, Chief Scientific Officer, Tigem (Telethon Institute of Genetics and Medicine) Greg Bonfiglio, Managing Director, Proteus Sp Haifeng Chen, CEO, Virovek, Inc. Hans Keirstead, Chief Executive Officer, Aivita Biomedical Hansjörg Hauser, Head of Department Gene Regulation and Differentiation, Helmholtz Centre for Infection Research Ian McKay, Innovation Lead Advanced Therapies, Innovate UK Ioannis Papantoniou, ATMP Bioprocessing Coordinator, KU Leuven Ivan Wall, Professor in Regenerative Medicine, Cell & Gene Therapy Bioprocessing, School of Life & Health Sciences, Aston University Jaap Jan Boelens, Chief of Service; Stem Cell Transplant and Cellular Therapies, MSKCC Jack Price, Head, Division of Advanced Therapies, National Institute for Biological Standards and Control Jacqueline Barry, Chief Clinical Officer, Cell and Gene Therapy Catapult Jean-Phillipe Combial, Co-Founder & CEO, Vivet Jane Andrews, Senior Director, Strategic Marketing, BioBridge Global


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Jane Lebkowski, President, Regenerative Patch Technologies Janet Glassford, Senior Quality Assessor, Medicines and Healthcare Products Regulatory Agency (MHRA) Jean-Pierre Latere, COO, Celyad Jelena Ochs, Manager Business Unit Life Sciences Engineering, Fraunhofer Institute for Production Technology IPT Jim Kovach, Director, Entrepreneurship and Innovation, UC Davis Health Joanna Miller, Science Director, Cell Therapy Sciences UK John Campbell, Associate Director, Tissues, Cells & Advanced Therapeutics, SNBTS John Johnston, Clinical Assessor, Biologicals Unit, MHRA John Maher, Consultant and Senior Lecturer in Immunology, King's College London, CSO, Leucid Jonathan Sheffield, CEO, NIHR Josefina Nilsson, Head of Business Unit, EM Service, Vironova Joseph Rotino, Vice President of Quality assurance and Regulatory Affairs, Cryoport Karen Hodgkin, Chief Operating Officer, Cell Medica Katarina Le Blanc, Professor/Specialist Physician, Division of Clinical Immunology, Karolinska Institute Katja Schmidt, Head Market Access Oncology, Germany, Novartis Lior Raviv, Vice President Development, Pluristem Therapeutics Lise Marie Grav, Postdoc, CHO Cell Line Engineering and Design, DTU Biosustain Michèle De Guise, Director of Health Services and Technology Assessment, INESSS Luc Boileau, Président-Directeur General, INESSS Lucy Foley, Director of Biologics, The Centre for Process Innovation (CPI) Manfred Schmidt, CEO, GeneWerk GmbH Mangala Srinivas, Multiscale Imaging Lab, Dept. of Tumor Immunology, Radboud University Medical Center Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET Marc Turner, Medical Director, Scottish National Blood Transfusion Service Pinar Akcakaya, Senior Research Scientist, AstraZeneca Marcos Valadares, Co-founder and CEO, PluriCell Biotech Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL Mark Sawicki, Chief Commercial Officer, Cryoport Martin Birchall, Professor of Laryngology and a Consultant in ENT Surgery, UCLH Mary Murphy, Senior Lecturer in Regenerative Medicine; Principal Investigator Orthobiology, REMEDI Michael Dombeck, Vice President, Business Development, Precision Biosciences Michael D. West, Chief Executive Officer, AgeX Therapeutics, Inc. Michael Hunt, CEO, ReNeuron Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon Mike Leek, CEO & Founder, TC Biopharm Mike Rice, Principal, Defined Health Miguel Forte, Chief Executive Officer, Zelluna Immunotherapy Miriam Haak, Director Business Development, Miltenyi Biotec GmbH Narayanan, Vice President, Disruptive Biologics, Voisin Consulting Life Sciences Neill Moray Mackenzie, Chairman, Immetacyte & Cellular Therapeutics Nick Medcalf, Innovation Lead, Innovate UK Nneka Onwudiwe, Founder and Chief Executive Officer, Pharmacoeconomics Consultants of America


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Ohad Karnieli, Chair, Process and Product Development Subcommittee, ISCT; CEO & Co-Founder, ATVIO Biotechnology Pascale V Guillot, Head of the Cellular Reprogramming and Perinatal Therapy, University College London Institute for Women’s Health Paul S Carter, Scientific Leader, Downstream Process Development, Cell & Gene Therapy, GlaxoSmithKline Peter Saltonstall, CEO, National Organization of Rare Disease Provisionally CONFIRMED: Masahide Yano, Research Scientist, Center for Drug Evaluation & Research, FDA Qasim Rafiq, Senior Lecturer (Associate Professor), Bioprocessing of Regenerative, Cellular and Gene Therapies, Department of Biochemical Engineering, UCL RA Session, Chief Business Officer & Head of Corporate Strategy, 4DMT Rabia Ozden, Chief Medical Officer, Nightstar Robert Beckman, Managing Partner, The Channel Group; Director of Business Development, Orgenesis Robert J Thomas, Professor in Manufacturing for Cell and Gene Therapies; EPSRC Early Career Fellow, Centre for Biological Engineering (CBE) Rodney L Rietze, Director, Strategic Development and Innovation, Cell and Gene Therapy Development and Manufacturing, Novartis Rogerio Vivaldi, President and Chief Executive Officer, Sigilon Therapeutics Ross A Macdonald, Managing Director & Chief Executive Officer, Cynata Therapeutics Limited Sam Hall, Principle, Apple Tree Partners Sandro Gomes, Investigator, Cell & Gene Therapy Product Development, GSK Sean Ainsworth, CEO, Immusoft Corporation Seppo Ylä-Herttuala, Academy Professor, Molecular Medicine, University of Eastern Finland Sergey A. Piletsky, Professor of Bioanalytical Chemistry, Chemistry Department, College of Science and Engineering, University of Leicester Shirley Bartido, Director, Regulatory Affairs, Cellectis Simone Dimartino, Senior Lecturer, Institute for Bioengineering, University of Edinburgh Stefano Menegatti, Assistant Professor in Chemical and Biomolecular Engineering, North Carolina State University Stefano Zancan, Head of Clinical Development and Operations, Fondazione Telethon Stefanos Theoharis, SVP, Partnering and Corporate Development, CellMedica Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center of Pre-Clinical Studies, Deputy Director, McGowan Institute for Regenerative Medicine (MIRM) Susan L. Solomon, CEO and Founder, The New York Stem Cell Foundation Research Institute Sven Kili, Principal, Sven Kili Consulting Takashi Kei Kishimoto, Chief Science Officer, Selecta Biosciences Inc Thomas Müller, Head of Directorate General “Drugs, Medical Devices, Biotechnology”, Federal Ministry of Health, Germany Tim Bertram, CEO, inRegen Uta Griesenbach, Professor, Molecular Medicine, National Heart and Lung Institute, Imperial College London; President, British Society for Gene and Cell Therapy Victoria English, Co-founder & Editor, Evernow Publishing Ltd, publishers of MedNous Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England Walter Van Dyck, Area Chair Technology & Operations Management, Director, Vlerick Healthcare Management Centre Yen Choo, CEO, Progenitor Therapeutics


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Representative from FUJIFILM Diosynth Biotechnologies Texas Representative from GE Healthcare Representative from Irvine Scientific Representative from Comecer Representative from Pall Biotech (141)

Investors, VCs and Pitching Companies

Agnieszka Janeczek, Interim CEO, Renovos Alan J Muir, Director Life Science & Healthcare Investment, Seven Hills Venture Partners Armando Cuesta Díaz, Founding Partner, Ilana Capital David Mazzo, CEO, Caladrius Biosciences Dmitry Kuzmin, Managing Partner, 4BIO Capital Dominic Schmidt, Partner, Syncona Edward Hodgkin, Partner, Syncona Partners Govind Pindoria, Director of Venture Support Unit, Imperial Innovations Jeff Ross, CEO, Miromatrix Michael Stein, Chairman & CEO, Oxstem Shay Hershcovich, Head of Business Development, Betalin Therapeutics Vijay Barathan, Life Science Partner, Catapult Ventures Group Glenn Pierce, EIR, Third Rock Ventures, CMO, Ambys Medicines (13)

Cord Blood Speakers:

Joanne Kurtzberg, Director, Carolinas Cord Blood Bank, Chief Scientific Officer, Robertson Clinical & Translational Cell Therapy Program, Director, Pediatric Blood and Marrow, Duke Center for Autism Elizabeth Shpall, Professor, Howard and Lee Smith Chair in Cancer Research, Director, Cell Therapy Laboratory and Cord Blood Bank, Deputy-Chair, Stem Cell Transplantation and Cellular Therapy, MD Anderson Cancer Center Omar Aljiwati, Associate Professor, University of Rochester Medical Center Guy Parkes, Head of Stem Cell Donation & Transplant, NHSBT Marcie Finney, Executive Director, Cleveland Cord Blood Center Daniel Gibson, Head of Cord Blood Innovation and commercialisation, Anthony Nolan Joshua Hare, Professor of Medicine, University of Miami Beth Shaz, Chief Medical and Scientific Officer, Executive Vice President, New York Blood Center Maria Craig, Professor of Paediatric Endocrinology, The Children's Hospital at Westmead Aisha Khan, Executive Director Laboratory Operations, Interdisciplinary Stem Cell Institute, University of Miami Miller School of Medicine Machi Scaradavou, Medical Director, New York Blood Center's National Cord Blood Program, Pediatric Hematologic Oncologist, MSKCC Chris Bond, Managing Director, Future Health Biobank Nicolas Pineault, Development Scientist, Canadian Blood Services Xiaokui Zhang, CSO, Celularity Moshe Israeli, Director of Tissue Typing Laboratory, Rabin Medical Centre Amnon Pelz, CEO, Taburit


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Jaap Jan Boelens, Chief of Service; Stem Cell Transplant and Cellular Therapies, MSKCC Mayur Abhaya, CEO, LifeCell Helen Papadaki, Professor of Haematology, Public CBB of Crete Irene Martini, Scientific Director, Smart Bank and Sorgente Maria Schoepe, Deputy Head of Production, Vita34 Sue Mundy, Head of Cord Blood Collection, Anthony Nolan Cord Blood Programme Dmitrii Ivolgin, Medical Director, Pokrovskij Stem Cell Bank Irene Mavroudi, Researcher, Public CBB of Crete Diana Hernandez, Group Leader Immunotherapy, Anthony Nolan Joana Correia, Executive Director and Chief Scientific Officer, Exogenus Zoran Ivanovic, Scientific Director, French Blood Institute Susana Cantero Peral, Associate Consultant I-Research, Mayo Clinic Rachel Manley, Head of Global Outreach, Save the Cord Foundation Warachaya Sutheesophon, Medical Director, Thai StemLife Abhijit Bopardikar, Director, ReeLabs Kavita Raj, Consultant Haematologist, Guy's and St Thomas' Hospital Jeff Drew, CSO, Cells4Life Tomasz Baran, Board Member, PBKM/FamiCord Roger Horton, Cord Blood Bank Operational Manager, Anthony Nolan Greg Bonfiglio, Managing Director, Proteus Sp Chris Leonforte, Processing and Quality Control Manager, Anthony Nolan Irina Evseeva, Head of Specialist Services, Anthony Nolan Roberto Gramignoli, Professor, Karolinska Institutet Glyn Stacey, Executive Director, International Stem Cell Banking Initiative Andy Peniket, Clinical Director of Haematology and Bone Marrow Transplantation, Oxford University Hospitals Eduardo Olavarria, BMT Program Director, Consultant Haematologist, Imperial College Healthcare NHS Trust Mathias Svahn, CEO, NextCell Pharma (43)

Seminar theatre presenters:

Adam Perriman, Associate Professor, Reader in Biomaterials, School of Cellular and Molecular Medicine, University of Bristol Ben Carter, Chief Technical Officer, CytoSeek Dan Grant, CEO, MTPConnect Dominic O'Regan, Director & Investor, New Mosaic Ltd Matthew Wheeler, Associate Director, VectorBuilder Europe Michal Wlodarski, Principal Healthcare & Life Sciences, Cambridge Innovation Consulting Ltd Representative from BioM Sarah Haywood, CEO, MedCity Kathrin Adlkofer, CEO, Cellbox Solutions Rachel Smith, Senior Clinical Project Manager, TCTC Group Julia Schaft, Managing Director, BioRN Fabien Moncaubeig, COO, Treefrog Therapeutics Israeli Society of Gene and Cell Therapy Healthgen Biotechnology


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Albcura Cobra Biologics Scinus Cell Expansion Yiannis Kiachopoulos, CEO, Causaly Inc AMSBIO (19)

TOTAL NUMBER OF SPEAKERS: 229

Sponsored sessions:

1. CellGenix – Roundtable 2. Cryoport – Keynote day 1 (not opening), track presentation (cold chain), panel discussion (& full day pre-conference workshop) 3. Fujifim Diosynth Biotechnology – track presentation (gene therapy manufacture) 4. Hitachi – Track presentation (bioprocessing), pre-event workshop (bioprocessing) 5. Miltenyi Biotec – Track presentation (viral vectors manufacture track presentation) 6. Defined Health – Moderating a panel discussion (pricing, reimbursement market access) 7. CPI – Center for Process Innovation (viral vectors manufacture track presentation) 8. VirOnova – Presentation (characterisation) 9. BioBridge Global – Presentation 10. Irvine Scientific – presentation (cell therapy manufacturing) 11. Virovek – Presentation (cell therapy manufacturing) 12. GE Healthcare – panel discussion, pre-conference workshop (process considerations for

cryogenic and regenerative medicine commercialization) 13. Genewerk – Presentation and roundtable (clinical development of gene therapy) 14. Comecer – Presentation (cell therapy manufacturing) 15. Pall Biotech – Presentation (viral vector manufacture)


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ESTEEMED ADVISORY BOARD

Cell and Gene Advisory Board: Andre Choulika, CEO, Cellectis Annie Hubert, Senior Director, EU Section & Public Policy, Alliance for Regenerative Medicine David Morrow, Programme Manager, EATRIS Dmitry Kuzmin, Managing Partner, 4BIO Capital Eric Soller, Vice President of Corporate Development & Strategy, BlueRock Therapeutics Jaap Jan Boelens, Pediatrician - Oncologist / Immunologist, ISCT Regional Vice-President, Europe 2018 – 2020 Jacqueline Barry, Chief Clinical Officer, Cell and Gene Therapy Catapult Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon Miguel Forte, Chief Executive Officer, Zelluna Immunotherapy Robert Beckman, Managing Partner, The Channel Group; Director of Business Development, Orgenesis Sven Kili, Principal, Sven Kili Consulting Bioprocessing Advisory Board: Bo Kara, Head Process Development, Cell Gene Therapy CMC, GSK James Miskin, CTO, Oxford Biomedica Lucy Foley, Director of Biologics, The Centre for Process Innovation (CPI) Mark Sawicki, Chief Commercial Officer, Cryoport Qasim Rafiq, Senior Lecturer (Associate Professor), Bioprocessing of Regenerative, Cellular and Gene Therapies, Department of Biochemical Engineering, UCL Steven Pincus, Associate Vice President Virology and Analytical Methods Development, Fujifilm Diosynth Biotechnology Ohad Karnieli, Chair, Process and Product Development Subcommittee, ISCT; CEO & Co-Founder, ATVIO Biotechnology Mitchel Sivilotti, Chief Operating Officer, CCRM Stem Cells and Regenerative Medicine Advisory Board: Alain Vertès, Managing Director, NxR Biotechnologies Claudia Mitchell, Chief Operations Officer, Universal Cells Inc. Norio Nakatsuji, Professor Emeritus, Kyoto University & Chief Advisor, Stem Cell & Device Laboratory, Inc Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center of Pre-Clinical Studies, Deputy Director, McGowan Institute for Regenerative Medicine (MIRM) Susan L. Solomon, CEO and Founder, The New York Stem Cell Foundation Research Institute


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AGENDA OVERVIEW

Pre-conference day Wednesday 15TH May 2019:

Conference day 1: Conference Day 1: Thursday 16th May 2019:

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Conference day 2: Day 2: Conference Day 2: Friday 17th May 2019:


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Pre-Conference Workshops and Summits Day

Wednesday 15th May

The summits will begin with a welcoming and networking breakfast at 08:30 at

the Business Design Center. The day will end with the opening networking drinks

reception at 17:30.

Gene Editing Summit

Wednesday 15th May 2019 Gene Editing Summit

09:00 Using CRISPR for preclinical drug discovery

Danilo Maddalo, Lab Head, Oncology Pharmacology, Novartis Institutes for BioMedical Research (CONFIRMED)

09:20 Precise genome engineering to empower drug discovery Barry Rosen, Senior Principal Scientist/Director, AstraZenca (CONFIRMED)

09:40 In vivo genome and base editing treatments of a human PCSK9 knock-in hypercholesterolemic mouse model

• Generation of humanized hypercholesterolemic in vivo model

• Understanding off-target effect of genome and base editing in vivo Pinar Akcakaya, Senior Research Scientist, AstraZeneca (CONFIRMED) 10:00 CRISPR-mediated genome engineering of CHO cell lines for production of therapeutic proteins

• Discuss the latest development in genome editing tools for CHO cell line development

• Recognize the impact that CRISPR-mediated genome editing has on accelerating the development of CHO cell lines

• Understand how the combination of genome editing and systems biology can be applied to improve the production of therapeutic proteins

Lise Marie Grav, Postdoc, CHO Cell Line Engineering and Design, DTU Biosustain (CONFIRMED)

10:20 Morning networking break

Synthetic biology for cell and gene therapies

11:00 Synthetic biology for cell and gene therapies presentations followed by panel discussion

• Moving from autologous to allogenic function

• Where are we really headed? Bob Deans, CTO, BlueRock Therapeutics (CONFIRMED) Jim Collins, Termeer Professor of Bioengineering in the Department of Biological Engineering and Institute for Medical Engineering & Science, MIT (invited) Peter Zandstra, Professor & Director, Michael Smith Laboratories, University of British Columbia / Professor, University of Toronto Synbio Canada (invited)

12:00 Networking Lunch

13:00 Gene Editing workshop Hansjörg Hauser, Head of Department Gene Regulation and

Differentiation, Helmholtz Centre for Infection Research (TBC)


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14:20 Networking refreshment break

15:00 Closing keynote panel: Advances in genome editing – the next frontier in human health Andrea Ballabio, Scientific Director, Telethon Institute (invited)

17:30 End of Pre-conference day followed by opening networking drinks

Process Considerations for Cryogenic and Regenerative Medicine

Commercialization

HOSTED BY

Wednesday 15th May 2019 Process Considerations for Cryogenic and Regenerative Medicine Commercialization

9:00 Welcome remarks:

Mark Sawicki, Chief Commercial Officer, Cryoport (CONFIRMED)

09:20 Panel Discussion: Integration and data continuity throughout the regenerative medicine production process Be the Match BioTherapies Representative (CONFIRMED) Jim Kovach, Director, Entrepreneurship and Innovation, UC Davis Health (CONFIRMED) GE Healthcare Representative (CONFIRMED) Derrell Porter, SVP – Global Commercial Head, Atara (invited) TrakCel Representative Orchard Therapeutics Representative


11:00 Panel discussion: Systems compliance and traceability during product distribution Mark Sawicki, Chief Commercial Officer, Cryoport (CONFIRMED) World Courier Representative (CONFIRMED) Tim Valco, VP Global Supply Chain, Atara (invited)


13:00 Panel discussion: Regenerative medicine globalization; considerations for effective launch activity beyond your initial geographic launch

Be The Match Representative (CONFIRMED) Alain Vertès, VP Alliance Management, Mesoblast (CONFIRMED) Joe Rotino, VP of Quality Assurance and Regulatory Affairs, Cryoport (CONFIRMED) Andy Chan, Chief Legal Officer, Corporate Development & Secretary, CBMG (invited) Bruce Phelan, Consultant, Blue Fin Group (invited) Gilead Representative (invited)

14:20 Networking break

15:00 Scalability (Series of 30 min panel discussion sessions)


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• Systems (GE, Agilent)

• Data (Be The Match, Trakcel)

• Infrastructure (World Courier, Cryoport, Hitatchi) Joe Rotino, VP of Quality Assurance and Regulatory Affairs, Cryoport (CONFIRMED) Be The Match BioTherapies Representative (CONFIRMED) GE Healthcare Representative (CONFIRMED) Bob Preti, CEO, Hitachi (invited) Agilent Representative (invited) TrakCel Representative (invited)


CAR-T manufacture and imaging

Wednesday 15th May 2019

CAR-T manufacture and imaging 09:00 Workshop: Automating CAR-T manufacturing, a step by step guide Rodney L Rietze, Director, Strategic Development and Innovation, Cell and Gene Therapy Development and Manufacturing, Novartis (CONFIRMED)

10:00 Networking break

11:00 Workshop: The future of cell therapy manufacturing technologies: All-in-one platforms or unit operation-specific devices?

Sponsored by Hitachi TBC: Bob Preti, CEO, Hitachi other panellists TBC


13:00 Manufacture and bioprocess of CAR-T

Moderator: Qasim Rafiq, Senior Lecturer (Associate Professor), Bioprocessing of Regenerative, Cellular and Gene Therapies, Department of Biochemical Engineering, UCL (CONFIRMED)

14:20 Afternoon networking break

15:00 Using imaging to improve the success rate of cell therapies on solid tumors

Host: David Morrow, Programme Manager, EATRIS (CONFIRMED) Mangala Srinivas, Multiscale Imaging Lab, Dept. of Tumor Immunology, Radboud University Medical Center (CONFIRMED) Seppo Ylä-Herttuala, Academy Professor, Molecular Medicine, University of Eastern Finland (CONFIRMED) Christopher Mann, Scientific and Regulatory Affairs Manager, Asphalion, S.L (TBC)


Standards development process/clinical trials

Wednesday 15th May 2019 Standards development process/clinical trials


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Chaired by: Sven Kili, Principal, Sven Kili Consulting Ltd. (CONFIRMED)

09:00 Focus group: To advance process, measurement, and analytical techniques of regenerative medicine products and services

Moderator: Sven Kili, Principal, Sven Kili Consulting Ltd. (CONFIRMED)

Jack Price, Head, Division of Advanced Therapies, National Institute for Biological Standards and Control (CONFIRMED)

Allison Getz, Senior Operations Programme Manager, Standards Coordinating Body (SCB) (invited) Richard McFarland, Chief Regulatory Officer, ARMI; Secretary, Standards Coordinating Body (invited) Representative from United States Pharmacopeia (USP) (invited)

10:00 Morning Networking Break

11:00 Focus group (continued): To advance process, measurement, and analytical techniques of regenerative medicine products and services

Moderator: Sven Kili, Principal, Sven Kili Consulting Ltd. (CONFIRMED)

Jack Price, Head, Division of Advanced Therapies, National Institute for Biological Standards and Control (TBC)

Allison Getz, Senior Operations Programme Manager, Standards Coordinating Body (SCB) (invited) Richard McFarland, Chief Regulatory Officer, ARMI; Secretary, Standards Coordinating Body (invited) Representative from United States Pharmacopeia (USP) (invited)


13:00 The EBMR (electronic batch manufacturing record)– product quality in a black box

• Ensuring product delivery, quality control, traceability.

• How can we reduce manufacturing man hours and increase efficiency using software?

• Focus group to develop software to trace product back to patient

Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL (CONFIRMED) Representative from Werum IT Solutions (TBC)

14:20 Afternoon Networking break

15:00 Clinical trial focus workshop: What needs to be done to get these experimental treatments to patients in the clinic?

• Centralized and decentralized models

• Sourcing starting material: apheresis, tumor, bone marrow

• From manufacture to back into local environment

• Design of clinical trial

Chair: Jacqueline Barry, Chief Clinical Officer, Cell and Gene Therapy Catapult (CONFIRMED) Sven Kili, Principal, Sven Kili Consulting Ltd. (CONFIRMED) Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon (CONFIRMED) Karen Hodgkin, Chief Operating Officer, Cell Medica (CONFIRMED) Neil Bell, Senior Vice President & Head of Clinical Operations, Autlous (CONFIRMED)


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Representative from bluebird bio (invited) Finn Willingham, Northern Alliance Advanced Therapies Treatment Centre Operations Manager, Newcastle Hospitals (invited)


AI in Healthcare Summit

David Sloman, Regional Director, NHS London

Jim Fackler, Associate Professor of Anesthesiology and Critical Care Medicine, John Hopkins School of Medicine

Nasir M Rajpoot, Head, AI for Cellular Pathology, Tissue Image Analytics Lab, University of Warwick

Goran Nendic, Director, Health eResearch Centre, Farr Institute

Wednesday 15th May 2019 AI in Healthcare Summit

08:00 Registration Opens

09:00 Opening Remarks from the Chair

09:10 Developing an NHS Example Hospital David Sloman, Regional Director, NHS London

09:20 Panel: AI at the Bedside: Trust me I’m a machine

• Developing systems synergistically to a whole health ecosystem

• How AI can be of assistance in the ICU, Nursing Facilities, Out Patients, Home Patients etc…

• Understanding what the pinpoints are in a clinical system and how they can be alleviated with AI Chair: Jim Fackler, Associate Professor of Anesthesiology and Critical Care Medicine, John Hopkins School of Medicine


11:00 Data analytics and AI for Healthcare Transformation Payam Barnaghi, Professor of Machine Intelligence, University of Surrey (TBC)

11:20 AI and Image analysis in pathology Nasir M Rajpoot, Head, AI for Cellular Pathology, Tissue Image Analytics Lab, University of Warwick

11:40 Automated Clinical Coding Goran Nendic, Director, Health eResearch Centre, Farr Institute


13:00 Presentation available

13:20 Panel discussion Jim Fackler, Associate Professor of Anesthesiology and Critical Care Medicine, John Hopkins School of Medicine David Sloman, Regional Director, NHS London Nasir M Rajpoot, Head, AI for Cellular Pathology, Tissue Image Analytics Lab, University of Warwick Goran Nendic, Director, Health eResearch Centre, Farr Institute Payam Barnaghi, Professor of Machine Intelligence, University of Surrey


15:00 AI IN HEALTHCARE WORKSHOP SLOT AVAILABLE Please contact Erica Baeta (+44 (0)207 092 1152, [email protected]) for further details



mailto:[email protected]

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Gene Therapy for Rare Disease Summit (coming soon)


Gene therapy for rare diseases summit 09:00 Chairs welcome

09:20 TBC: Late preclinical and early clinical updates on Sanofi’s rare disease projects Brian Bronk, Head of Business Development, Rare Diseases & Rare Blood Disorders, Sanofi (CONFIRMED)

09:40 Preclinical and clinical development of tolerogenic nanoparticles to mitigate immunogenicity against enzyme and gene therapies for orphan diseases

• Immunogenicity is a common cause of treatment failure and hypersensitivity reactions with biologic therapies, particularly for enzyme therapies and gene therapies for orphan and rare diseases

• We have developed technology to induce antigen-specific immune tolerance to biologic therapies • I will present case studies from our lead clinical program in severe refractory gout and preclinical

program in gene therapy Takashi Kei Kishimoto, Chief Science Officer, Selecta Biosciences Inc (CONFIRMED)

10:00 Morning Networking Break

11:00 Moving to phase III and beyond Birgitte Volck, President of R&D, Avrobio (TBC)

11:20 PANEL: Multi-stakeholder strategies from government, patient associations and advocates to improve rare disease treatment access

• Drug Development and Challenges to Patient Leaders

Debra Miller, Founder and President, CureDuchenne (invited) Neena Nizar, Founder and Executive Director, Jansen's Foundation (invited)

11:40 Roundtable discussions

ROUNDTABLE 1: Pediatric trials – reducing the

financial, logistical and emotional burden for patients and families

participating in pediatric trials (PPMD)

ROUNDTABLE 2: Compassionate Use Programs – helping patients get access to medication, educating the community, physicians and investors to ensure safe access to experimental treatments while staying true to corporate strategy

ROUNDTABLE 3: Virtual Trials – enabling clinical trials to be more virtual, reducing distance times, overcoming geographic barriers and increasing data collection through the use of use of wearables

ROUNDTABLE 4: Expanded access – incorporating the patient advocacy perspective to develop a review process and a formalized procedure on expanded access

12:00 Networking lunch

13:00 TBC: Implementation of cross-border healthcare: can you guarantee a patient access to therapies in another country? Alexander Natz, Secretary General, EUCOPE (TBC)

13:20 Company presentation 1




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15:00 Panel discussion: Tackling the ongoing challenges of research and development of rare disease medicines

• As an industry how can we position ourselves specifically as small companies?

Alexander Natz, Secretary General, EUCOPE (TBC)


Cord Blood Workshops

Cord Blood Workshops Wednesday 15th May

11:00 A true understanding of the biology of stem cells Irene Martini, Scientific Director, Smart Bank and Sorgente (CONFIRMED)


13:00 Cord blood bank sustainability

• Provoking greater use of cord with high quality impact

• Understanding the hurdles in selecting cord in the clinical transplant setting

• Diversification of products and services

• Creating a sustainable collection model – strategies that have bene adopted Daniel Gibson, Head of Cord Blood Innovation and commercialisation, Anthony Nolan (CONFIRMED) Roger Horton, Cord Blood Bank Operational Manager, Anthony Nolan (CONFIRMED) Diana Hernandez, Group Leader Immunotherapy, Anthony Nolan (CONFIRMED) Chris Leonforte, Processing and Quality Control Manager, Anthony Nolan (CONFIRMED) Irina Evseeva, Head of Specialist Services, Anthony Nolan (CONFIRMED)

14:00 Operational, strategic and economic impact of FDA licensure for HPC, cord blood

• Operational impact of FDA Licensure experience that facilitated process improvement, facility design and manufacturing productivity.

• FDA license economic impact on cost per cord blood unit, price per unit, capital and re-curing expenses

• Strategically leveraging FDA license to enter new markets and optimize manufacturing Marcie Finney, Executive Director, Cleveland Cord Blood Center (CONFIRMED)


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Main Conference Agenda 16th –17th May

Keynote Sessions

Opening Drinks Reception: Wednesday 15th May

Pre-Conference: Wednesday 15th May 2019

Opening networking drinks reception

17:30 Inspirational Keynote: Dean Kamen, Founder, FIRST; President, DEKA Research & Development Corporation (invited)

Day 1: Thursday 16th May 2019

Conference Day 1: Thursday 16th May 2019

World Advanced Therapies and Regenerative Medicine Congress Morning Plenary

08.45 Chair’s opening remarks: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

09:00 Fireside chat: A new era in medicine- introducing gene therapy

• What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.

• Staying true to our roots while preparing the patient, medical and policy maker communities for a new chapter in the retinal disorders textbook

• Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseases Kathy High, President and Head of R&D, Spark Therapeutics (CONFIRMED) Interviewer: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

Keynote panel


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09.30 (ex vivo) gene edited therapeutics and (in vivo) gene therapy: how can gene technology be used now and, in the future, to treat and cure diseases in the clinic

• Current opportunities and limitations in gene therapy

• Moving into larger diseases from rare diseases, what challenges need to be overcome?

• Risk/benefit acceptance of the general population and the medical system

• How do we want to use it in the future and what challenges need to be overcome to do so?

• Tackling market access challenges and reimbursement hurdles

• How do you move towards that goal in a way that engages stakeholders?

• Supply chain and logistics

Chair: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED) James Miskin, CTO, Oxford Biomedica (CONFIRMED) Kathy High, President and Head of R&D, Spark Therapeutics (CONFIRMED) Andrew Obenshain, Head of Europe, bluebird bio (TBC) Lothar Germeroth, SVP, Managing Director, Juno Therapeutics, a Celgene Company (CONFIRMED) Andre Choulika, CEO, Cellectis (CONFIRMED) Bob Smith, SVP, Global Gene Therapy Business, Rare Disease, Global Innovative Pharma, Pfizer (CONFIRMED) Representative from Novartis (invited)

10:30 Morning refreshments 11:30 Keynote presentation: Recent Progress in iPS Cell Research and Application Nobel Prize-winner: Shinya Yamanaka, Director of CiRA (Center for iPS Cell Research and Application), Kyoto University (CONFIRMED) 11:50 State of the industry address: future state regulatory and compliance considerations for cold chain

distribution of regenerative therapies

• Current compliance and regulatory practices are insufficient in managing risk during distribution

• Informatics integration, data continuity, and leveraging real time data collection will be key considerations

• Compliance practices including supply chain traceability are likely to be regulated in the near future

Mark Sawicki, Chief Commercial Officer, Cryoport (CONFIRMED)

12:10 Technology and Strategy Roundtables 25 senior level tables hosted by thought leaders on key challenges and opportunities in next-generation therapeutic development. Participants are invited to join the group discussions on a topic of importance to them.

• At each roundtable, the moderator will talk for 5 minutes to introduce the topic highlighting particular challenges specific to that area

• The table will then move on to discussion for 25 minutes.

• There will then be one rotation, giving participants the opportunity to have join two round tables in total

(2 x 30-minute rotations and max 15 PAX) Table 1. Raw material supply for the commercial manufacturing of cell and gene therapies Frank Hecht, Vice President Marketing and Sales, CellGenix GmbH (CONFIRMED)

Table 2. How to manage cell and gene therapy delivery from ‘end to end’ Mark Sawicki, Chief Commercial Officer, Cryoport (CONFIRMED)

Table 3: Navigating the pathway to clinical success Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon (CONFIRMED)

Table 4. Stem cells for neurological applications Chaim Lebovits, CEO, Brainstorm Cell Therapeutics (CONFIRMED)


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Table 5. MSCs Alain Vertès, Vice President, Alliance Management, Mesoblast (CONFIRMED)

Table 6. A holistic approach to analytical support, for the biotech industry Sergey A. Piletsky, Professor of Bioanalytical Chemistry, Chemistry Department, College of Science and Engineering, University of Leicester (CONFIRMED)

Table 7. Small molecule drug discovery to selectively stimulate regenerative processes Yen Choo, CEO, Progenitor Therapeutics (CONFIRMED)

Table 8. Cell Line development for optimum manufacturing Cornelia Kasper, Full University Professor, BOKU (CONFIRMED) Hansjörg Hauser, Head of Department Gene Regulation and Differentiation, Helmholtz Centre for Infection Research (TBC)

Table 9. Tissue engineering for organ regeneration Benjamin Shepherd, Director, Therapeutics, Organovo (CONFIRMED) Jeff Ross, CEO, Miromatrix (CONFIRMED)

Giuseppe Mazza, Chief Executive & Scientific Officer, Engitix (CONFIRMED)

Table 10. How companies can interact with the regulator and various pitfalls to be aware of (aimed at start-up companies and those from outside the EU (not used to EU or UK ways) John Johnston, Clinical Assessor, Biologicals Unit, MHRA (CONFIRMED)

Table 11. Purification of stem cells Stefano Menegatti, Assistant Professor in Chemical and Biomolecular Engineering, North Carolina State University (TBC)

Table 12. Challenges and opportunities of developing cell therapy in Latin America Marcos Valadares, Co-founder and CEO, PluriCell Biotech (CONFIRMED)

Table 13. New strategies for renal failure Brian Miller, CEO, Sentien Biotechnologies (CONFIRMED)

Table 14. Deal making and finance Michael Hunt, CEO, ReNeuron (CONFIRMED)

Table 15: Making allogeneic cell therapy a reality Rogerio Vivaldi, President and Chief Executive Officer, Sigilon Therapeutics (CONFIRMED) Devyn Smith, Chief Strategy Officer & Head of Operations, Sigilon, Inc (CONFIRMED)

Table 16. Exploring the crossover between human and veterinary applications for stem cells Joanne Miller, Science Director, Cell Therapy Sciences (CONFIRMED)

Table 17. Roundtable discussion available If you are interested in being involved, please contact Erica Baeta at: [email protected] or +44 207 092 1152

Table 18. Building a diversified portfolio of C&GT medicines

Ajan Reginald, Chief Executive Officer, Celixir (CONFIRMED)

Table 19. How to monitor the safety of viral vectors and other genome modifying therapies in clinical trials? Manfred Schmidt, CEO, GeneWerk GmbH

Table 20. Process development and scale-up strategy for a cryo-product in the context of a broad allogeneic cell therapy Benoît Champluvier, Advisor, Bone Therapeutics (CONFIRMED)

Table 21. Automation and continuous manufacture Lior Raviv, Vice President Development, Pluristem Therapeutics (CONFIRMED)

Table 22. Using ATMPs to tackle aging

Brian Culley, CEO, BioTime (invited)

Table 23. Process analytics and control Julian Morris, Technical Director Centre for Process Analytics and Control Technology (CPACT) Visiting Professor, Department Pure and Applied Chemistry, University of Strathclyde (invited)

Table 24. Developing exosome therapies Ivan Wall, Professor in Regenerative Medicine, Cell & Gene Therapy Bioprocessing, School of Life & Health Sciences, Aston University (TBC)


13:10 Lunchtime keynote: Daniel Kraft, Founder & CMO, Exponential Medicine; Faculty Chair for Medicine And Neuroscience, Singularity University (invited)

14:10 Conference streams begin (see below)



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Day 2: Friday 17th May 2019

Conference Day 2: Friday 17th May 2019

World Advanced Therapies and Regenerative Medicine Congress Morning plenary

08:30 Chairs opening remarks: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

08:40 Keynote presentation: Gene editing therapeutics Edward Rebar, CTO, Sangamo Therapeutics (CONFIRMED)

09:00 Keynote panel discussion: Exploring advances in gene editing technology, therapeutics and research

• Best strategies for implementing new methods across the field of clinical research, now and for the future

• Optimisation of the delivery of gene editing technology,

• How to minimise off-target effects and mutations for the safe and effective delivery of gene editing technology

• Discussion of ethical and regulatory challenges, current legislation, how to navigate ethical concerns in light of the growing scale of genome editing. Most recently the CRISPR babies that were born in 2018

Chair: Antonio Regalado, Senior Editor for Biomedicine, MIT Technology Review (CONFIRMED) Derek Jantz, Co-founder and CEO, Precision BioSciences (CONFIRMED) Fred Chereau, President and CEO, LogicBio Therapeutics (CONFIRMED) Edward Refbar, CTO, Sangamo Therapeutics (CONFIRMED) Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

09:40 Keynote presentation: CAR T Immunotherapy – Opportunities and Challenges

• Understand the role of CAR-T cell therapy in relapsed ALL

• Understand the risk factors and therapies for Cytokine Release Syndrome (CRS)

• Understand the importance of T cell proliferation and persistence in CAR-T cell efficacy

Stephan Grupp, Chief, Cellular Therapy and Transplant Section, Children’s Hospital of Philadelphia (CONFIRMED)

10:00 Keynote panel: Innovations in cellular and combination immunotherapy: current learnings, challenges and future applications

• Using the immune system to identify and fight cancer cells: where are we and what do we need to maximise patient benefit in the future?

• Improving cellular immunotherapy success by using genetic biomarkers to create precision medicines and improve success rates

• Where are we with combination immunotherapy, what needs to be done to improve efficiency and effectiveness in the future.

• Moving from oncology to non-oncology applications, how can we make the shift to new therapy indications

• Preventing relapse and resistance

Chair: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED) Roy Baynes, SVP and Head Global Clinical Development, Chief Medical Officer, Merck, Sharp & Dohme (CONFIRMED) Frédéric Triebel, CSO & CMO, Immutep Limited. (CONFIRMED) Stephan Grupp, Chief, Cellular Therapy and Transplant Section, Children’s Hospital of Philadelphia (CONFIRMED) Lothar Germeroth, SVP, Managing Director, Juno Therapeutics, a Celgene Company (CONFIRMED) Jeffery L. Kutok, Chief Scientific Officer, Infinity Pharmaceuticals, Inc. (CONFIRMED)


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10:40-11:00 Networking refreshment break followed by conference streams

ADVANCED THERAPIES TRACKS

PRICING AND MARKET ACCESS

PRICING AND MARKET ACCESS: VALUE, PRICING, AFFORDABILITY


Moderated by:

14:10 Chair’s remarks

14.15 Tackling the issue of pricing reimbursement and market access for ATMPs

Robert Beckman, Managing Partner, The Channel Group; Director of Business Development, Orgenesis

(CONFIRMED)

14.35 Implementing strategic development and innovating disruptive business models for transformative cell and gene therapies

• Prioritizing opportunities within the complex landscape of advanced therapeutics

• Developing innovative healthcare business models for in-vivo and ex-vivo manufactured gene and cell therapies

• Maintaining robust and sustainable growth in rare disease

• Perspectives from investors, biotech and pharma

Moderator: Mike Rice, Principal, Defined Health (CONFIRMED) Panelists: Sam Hall, Principle, Apple Tree Partners (CONFIRMED) RA Session, Chief Business Officer & Head of Corporate Strategy, 4DMT (CONFIRMED) Jean-PHillipe Combial, Co-Founder & CEO, Vivet (CONFIRMED) Neil Johnston, Head Business Development & Licensing, Novartis Pharmaceuticals (invited) Brian Kaspar, CSO, AveXis (invited) Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics (invited)

15.15 Afternoon Refreshment Break

PRICING AND MARKET ACCESS: VALUE, PRICING, AFFORDABILITY 15.45 The Rise of CAR-T: Ensuring Access to Transformative Cancer Therapies

• The rise of immuno-oncology, most recently CAR-T, represents a new generation of transformative cancer therapies offering hope for the patients of today and tomorrow. However, with this revolutionary approach to treating cancer, there are potential hurdles to patient access that must be acknowledged and overcome.

• This session will focus on the transformative potential of CAR T as well as key challenges ahead for

patients, providers, payers, and innovators. Topics will include: value and cost (to the patient and the


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healthcare system), coverage and reimbursement, and the potential for innovative payment

arrangements.

James Noble, CEO, Adaptimmune (invited) Gregg Sando, CEO, CellMedica (invited) Richard Bagger, Executive Vice President, Corporate Affairs and Market Access, Celgene Corporation (invited) Henry Grabowski, Professor Emeritus of Economics, Duke University (invited) Gwen Nichols, Chief Medical Officer, Leukemia and Lymphoma Society(invited) Noopur Raje, Director, Multiple Myeloma Program, Medical Oncology, Massachusetts General Hospital (invited)

16.25 Innovative reimbursement & financing models for rare disease treatments in Europe and the USA Nick Medcalf, Innovation Lead, Innovate UK (CONFIRMED) Carla Deakin, Associate Director – Accelerated Access Collaborative Secretariat & NICE Office for Market Access, NICE (CONFIRMED) Giorgio Iotti, Head of Program Leadership and Control, Rare Disease Unit, Chiesi Farmaceutici SpA (TBC) Alexander Natz, Secretary General, EUCOPE (TBC) Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics (invited) Bhash Parasuraman, Vice President, Patient Health Impact Rare Disease, Pfizer (invited) Peter Saltonstall, CEO, National Organization of Rare Disease (invited)

17.00 Presentation

Reserved for PRMA Consulting

17.25 Panel discussion: Finding the right framework for reimbursement and market access for cell and gene therapies

• Structured reimbursement system for hospital-based treatments in Europe

• What is possible for statutory health insurance framework in the EU?

• Contract possibilities with manufacturers, hospitals and insurance providers

Chair:

Detlev Parow, Head of Department of Medicines, Therapeutic Appliances and Remedies, DAK (CONFIRMED) Katja Schmidt, Head Market Access Oncology, Germany, Novartis (CONFIRMED) Peter Saltonstall, CEO, National Organization of Rare Disease (CONFIRMED) Nneka Onwudiwe, Founder and Chief Executive Officer, Pharmacoeconomics Consultants of America (CONFIRMED) Walter Van Dyck, Area Chair Technology & Operations Management, Director, Vlerick Healthcare Management Centre (CONFIRMED) Charles-Etienne De Cidrac, Director of Health Insurance, AXA (CONFIRMED) Michèle De Guise, Director of Health Services and Technology Assessment, INESSS (CONFIRMED) Thomas Müller, Head of Directorate General “Drugs, Medical Devices, Biotechnology”, Federal Ministry of Health, Germany (CONFIRMED) Carla Deakin, Associate Director – Accelerated Access Collaborative Secretariat & NICE Office for Market Access, NICE (CONFIRMED)

Representative from bluebird bio (invited) Wim Goettsch, Special HTA-advisor, Dutch National Health Care Institute (Zorginstituut Nederland) (invited) Vitaly Omelyanovskiy, General director, Center of Healthcare Quality Assessment and Control, Ministry of Health of the Russian Federation (invited) Saira Jan, Director of Pharmacy Strategy and Clinical Integration, Horizon Blue Cross Blue Shield (invited)

18:25 End of Day One – Off-Site Networking Drinks for all paying delegates


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PRICING AND MARKET ACCESS: APPROVAL AND EVIDENCE

Conference Day 2: Friday 17th May 2019 Moderated by: 11.05 The newest changes in regulation and market access for ATMPs in Germany Thomas Müller, Head of Directorate General “Drugs, Medical Devices, Biotechnology”, Federal Ministry of Health, Germany (CONFIRMED)

11.25 Breaking into the US market and overcoming regulatory hurdles

Reserved for Cardinal Health

12.05 Case study: building a cell therapy business in Japan

Adam Gridley, President & CEO, Histogenics (CONFIRMED)

12.25 Panel focusing on Japanese regulation and commercial ecosystem

Chair: Greg Bonfiglio, Managing Director, Proteus Sp (CONFIRMED) Adam Gridley, President & CEO, Histogenics (CONFIRMED) Yoshiaki Maruyama, Review Director, Office of Celluar and Tissue Based Products, PMDA (invited)

13.05 Networking Lunch

APPROVAL AND EVIDENCE

14.05 Starting materials and standards in relation to advanced therapies

Jack Price, Head, Division of Advanced Therapies, National Institute for Biological Standards and Control (CONFIRMED)

14.25 US Cell & Gene Therapy Regulatory Policy Update

Recent or planned updates to the regulatory framework for cell and gene therapy products and experience with the new RMAT (Regenerative Medicine Therapy Designation) will be reviewed and discussed.

Shirley Bartido, Director, Regulatory Affairs, Cellectis (CONFIRMED)

14.50 Ensuring fair pricing of pharmaceuticals and looking into the Canadian landscape for highly priced medicines

Tanya Potashnik, Director Policy and Economic Analysis, Patented Medicine Prices Review Board, Government of Canada (invited)

15.10 Afternoon Refreshments

MARKET ACCESS

15.40 Common pitfalls and hurdles when reaching registration and market access for a new next generation therapeutic • What are the best ways to approach getting a new therapeutic modality registered and on the market? • Key lessons learned from Strimvelis • How we can overcome key challenges and get these therapies registered? Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon (CONFIRMED)

16:00 What is going on in Quebec about Market Access?

• Examining Quebec life sciences strategy: Integration of medicines and technological innovations

• Achieving cost reduction and improving access to medicines


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• Re-editing the evaluation process for the introduction of innovative medication and technologies Luc Boileau, Président-Directeur General, INESSS (CONFIRMED)

16.20 Panel discussion: ‘Getting it right first time’ – Taking steps early on to ensure success in the later stages

• Preparation for market access • Planning early in the clinical trials process to avoid failure later on in the development process • Improving the awareness of working with the clinical centre • Supply term agreement with hospital and insurance providers • Framework administration to provide expanded access and clinical trials • Being aware & working well in advance on quality agreements • Maintaining chain of custody (both ways) of samples and quality control during transfer of clinical samples • Tackling the reluctance to think about this planning early on

Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon (CONFIRMED)

Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL (CONFIRMED) Albert Ribickas, Bone Marrow Transplant Manager, Moffitt Cancer Center (TBC)

Didier Caizergues, Head of Regulatory Affairs Department, Genethon (invited) Representative from United States Pharmacopeia (USP) (TBC) 17:00 Chair’s closing remarks

17:10 End of conference, thanks for coming!


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GENE MODIFIED CELL THERAPY


GENE MODIFIED CELL THERAPY

14:10 Chair’s opening remarks: Helen Tayton-Martin, Chief Business Officer, Adaptimmune (invited)

14:15 Getting genetic therapies from the bench to the bedside: Meeting the challenges of commercialisation

• What are the biggest challenges to making gene therapy commercially viable

• What system change is needed

• What have we learned as an industry so far

Andrew Obenshain, Head of Europe, bluebird bio (TBC)

14:35 Creation of off-the-shelf TCR-less CAR T cell therapies

Speaking opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, [email protected]) for further details)

14:55 How the allogeneic approach will make cell therapies accessible for more patients through gene editing

Andre Choulika, CEO, Cellectis (CONFIRMED)

15:15 Afternoon refreshment break

GENE MODIFIED CELL THERAPY: Improving clinical success

15:45 Using immunotherapy to target HIV

David Pauza, CSO, American Gene Technologies (CONFIRMED)

16:05 Engineering B cells as a living and evolving drug

• CRISPR/Cas9 B cell engineering

• in vivo activation of adoptively transferred engineered B cells

• Affinity maturation of engineered B cells to counteract HIV evolution Adi Barzel, Senior Lecturer, Tel Aviv University, President, Israeli Society of Gene and Cell Therapy (CONFIRMED)



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16:25 Short (10 minute) company presentations followed by panel discussion: Development strategies for CAR-T and TCR therapies with the late stage in mind

• Allogenic vs autologous

• Understanding different opportunities: selecting the correct product for the appropriate purpose • Improving outcomes tackling solid tumours with CAR-T therapy • Moving towards a more off the shelf approach for better cost of goods

Miguel Forte, Chief Executive Officer, Zelluna Immunotherapy (CONFIRMED)

Neill Moray Mackenzie, Chairman, Immetacyte & Cellular Therapeutics (CONFIRMED)

Evren Alici, Assistant Professor and Group Leader, Haematology, Karolinska Institutet (CONFIRMED) Stefanos Theoharis, SVP Corporate Development & Partnering, Cell Medica (CONFIRMED) Sean Ainsworth, CEO, Immusoft Corporation (CONFIRMED)

Michael Dombeck, Vice President, Business Development, Precision Biosciences (CONFIRMED)

17:45 Panel discussion: Using imaging to improve the success rate of cell therapies

Host: David Morrow, Programme Manager, EATRIS (CONFIRMED)

Panellists:

Mangala Srinivas, Multiscale Imaging Lab, Dept. of Tumor Immunology, Radboud University Medical Center (CONFIRMED)

Seppo Ylä-Herttuala, Academy Professor, Molecular Medicine, University of Eastern Finland (CONFIRMED) Christopher Mann, Scientific and Regulatory Affairs Manager, Asphalion, S.L (TBC)


IMMUNOTHERAPY

Day 2: Conference Day 2: Friday 17th May 2019 IMMUNOTHERAPY

Chaired by: Roy Baynes, SVP and Head Global Clinical Development, Chief Medical Officer, Merck, Sharp and

Dohme (TBC)

11.00 Chair’s remarks

11:05 Opening presentation Jeffery L. Kutok, Chief Scientific Officer, Infinity Pharmaceuticals (CONFIRMED)

11:25 Combining a soluble LAG-3 protein with an anti-PD-1 antibody in phase I-II trials


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Frédéric Triebel, CSO & CMO, Immutep Limited. (CONFIRMED)

11:45 Using Gamma-Delta T-Cells for Immuno-oncology applications

• Using V Delta 2 T-Cells to sense and react to metabolic tumors • Using viral vectors to create a catalytic event via T-Cell receptor and kill like an NK Cell • Broad spectrum for tumor application and sensitive to tumor inhibition


12:05 Allogeneic gamma-delta CAR-T

• Clinical strategy, transition from autologous to allogeneic

• Manufacture of GMP allogeneic banks

• CAR-T modification of allogeneic cells

Mike Leek, CEO & Founder, TC Biopharm (CONFIRMED)

12:25 Panel discussion: Precision medicine for immunotherapy Using biomarkers and diagnostics to start correlating success rates in immunotherapy: how to know when to apply these Which is more important: stratification of patients using biomarkers or identifying the correct cells for a specific application? Gene editing therapeutics Hans Keirstead, Chief Executive Officer, Aivita Biomedical (CONFIRMED) Susan L. Solomon, CEO and Founder, The New York Stem Cell Foundation Research Institute (CONFIRMED) Laurence Cooper, Chief Executive Officer, Ziopharm Oncology (invited) Damien Page, Medical Leader, Personalised Health, Roche (invited) Francesco Dazzi, Professor of Regenerative and Haematological Medicine, KHP Lead for Cellular Therapies, King’s College London (TBC) Masahide Yano, Research Scientist, Center for Drug Evaluation and Research, FDA (TBC) Farzin Farzaneh, Head of Molecular Medicine, Kings College London (TBC)



14:10 Clinical and commercial application of scaled human stem cell derivates in immune-oncology

New clinical data for 3 immune-oncology clinical trials for ovarian, glioblastoma and melanoma treatment Hans Keirstead, Chief Executive Officer, Aivita Biomedical (CONFIRMED)

14:30 Case study of regulatory challenges and achievements for ilixadencel • ATMP Classification as somatic cell therapy medicinal product – December 2015 • Scientific Advice (national and EMA, pre-IND) • IND cleared by FDA – December 2016 • ilixadencel (INN) – from WHO January 2017 • EMA ATMP Certification – March 2018 Margareth Jorvid, Head of Regulatory Affairs and QA, Immunicum AB (CONFIRMED)

14:50 Tools and technologies for immunotherapy development

Speaking opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, [email protected]) for further details

15:10 Afternoon Refreshments

15:40 Targeting solid tumors using tailor made TCR therapies



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Laurence Cooper, Chief Executive Officer, Ziopharm Oncology (invited)

16:00 Personalised cancer immunotherapy, tumour-derived T cell therapy for non-small cell lung cancer

Iraj Ali, CEO, Achilles Therapeutics (invited)

16:20 Tackling challenges when dealing with the tumor microenvironment

Matt Hewitt, Director, Tumour Immunology & Microenvironment, Bellicum Pharmaceuticals (invited)

16:40 Chair’s Closing Remarks

16:45 Close of conference

IN VIVO GENE THERAPY


IN VIVO GENE THERAPY


14:15 Presentation

Bob Smith, SVP, Global Gene Therapy Business, Rare Disease, Global Innovative Pharma, Pfizer (TBC)

14:35 Panel discussion: Delivering on the Promise of Gene Therapy

Making genetic therapies a reality for patients

Attendees will have the opportunity to hear from a panel of experts as they discuss the importance of commercial, scientific and clinical working together, every step of the way, from development to commercialization, to bring a new treatment to patients.

Brian Bronk, Head of Business Development, Rare Diseases & Rare Blood Disorders, Sanofi (CONFIRMED) Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc. (TBC)

Stefano Zancan, Head of Clinical Development and Operations, Fondazione Telethon (CONFIRMED)

Rogerio Vivaldi, President and Chief Executive Officer, Sigilon Therapeutics (CONFIRMED)

Andrew Obenshain, Head of Europe, bluebird bio (CONFIRMED) Birgitte Volck, President of R&D, Avrobio (TBC)

Debra Miller, Founder and President, CureDuchenne (invited) Emmanuel Dulac, Chief Commercial Officer, ALNYLAM PHARMACEUTICALS (invited) Gilles Besin, Sr. Director, Immunology, Moderna Therapeutics (invited)

Chris Ashton, CEO, Achilles Therapeutics (invited) James Wilson, University of Pennsylvania (invited)


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15:15 Afternoon Refreshment Break

GENE THERAPY

15.45 Case study: TIGEM’s gene therapy projects

Graciana Diez-Roux, Chief Scientific Officer, Tigem (Telethon Institute of Genetics and Medicine) (CONFIRMED)

16:05 Translating in vivo genome editing capabilities into therapeutics

• Optimizing the nuclease to ensure in vivo specificity

• Determining the best delivery system

• Evaluating appropriate disease models

Derek Jantz, Co-founder and CSO, Precision BioSciences (CONFIRMED)

16:25 The road to restoring sight in patients with rare inherited retinal diseases

• Phase I & II clinical trials data using gene therapy for retinal diseases

Rabia Ozden, Chief Medical Officer, Nightstar (CONFIRMED)

16:45 Successfully developing and commercialising a gene therapy


17:05 Series of 10-minute updates from gene therapy organisations followed by a panel discussion on strategically improving issues in vivo and ex vivo gene therapy

• In vivo gene therapy – moving away from the bias of AAV

• Why the field has accelerated so rapidly catalyzed CAR-T

• Solving the problem of high manufacturing costs

• AAV vs Lenti platforms pros and cons

How industry, academia and patient organizations can work together to improve outcomes for patients.

Chair: James Miskin, CTO, Oxford Biomedica (CONFIRMED)

Use of lentiviral vectors for the lung: approach, challenges and potential indications

Uta Griesenbach, Professor, Molecular Medicine, National Heart and Lung Institute, Imperial College London; President, British Society for Gene and Cell Therapy; Strategy Group Member, UK Cystic Fibrosis Gene Therapy Consortium (CONFIRMED)

Fraser Wright, CTO, Axovant (CONFRMED)

Bodil Willumsen, Supplier Relationship Manager, External Supply Operations, Cell and Gene Therapies, Novartis (CONFRMED) Andrea Spezzi, Chief Medical Officer and Co-founder, Orchard Therapeutics (TBC) Soraya Bekkali, CEO, Gyroscope Therapeutics (invited)



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Invited companies:

Sanofi (invited)

Novartis (invited)

18.25 Close of Day 1 – Off-site Evening Drinks Reception for all paying delegates

CELL THERAPY AND REGENERATIVE MEDICINE

Day 2: Conference Day 2: Friday 17th May 2019


Chaired by: Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center of Pre-Clinical

Studies, Deputy Director, McGowan Institute for Regenerative Medicine (MIRM) (CONFIRMED)

11:05 The changing paradigm for cell therapy • How we access partnerships through technology, process development and manufacturing

• Shifts challenges and opportunities

Eric Soller, Vice President of Corporate Development & Strategy, BlueRock Therapeutics (CONFIRMED)

11:25 Clinical trial updates new data and strategies


11:45 Moving from autologous towards fully allogenic cell therapy

Claudia Mitchell, COO, Universal Cells, Inc. (CONFIRMED)

12:05 Platform technology for regenerative medicine cell therapy, stem cells, immune-oncology


12:25 Panel Discussion: Harnessing the body's regenerative capabilities – therapeutic development from the clinic to commercialization

This panel will examine and discuss the ways regenerative medicine is being revived in the biotech industry and how some companies are using these new techniques and platforms to create better, faster, safer and potentially curative treatment options for a multitude of disease indications

Key topic: Moving from autologous to fully allogeneic cell therapy

Chair: Antonio Regalado, Senior Editor for Biomedicine, MIT Technology Review (invited) Benjamin Shepherd, Director, Therapeutics, Organovo (CONFIRMED) Bob Deans, CTO, BlueRock Therapeutics (CONFIRMED) Cornelia Kasper, Full University Professor, BOKU (CONFIRMED) Claudia Mitchell, COO, Universal Cells, Inc. (CONFIRMED) Michael D. West, Chief Executive Officer, AgeX Therapeutics, Inc. (CONFIRMED) Felicia Pagliuca, Scientific Co-Founder, Vice President of Cell Biology Research and development, Semma Therapeutics (CONFIRMED)




35






Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center of Pre-Clinical Studies, Deputy

Director, McGowan Institute for Regenerative Medicine (MIRM)

14:10 Immunology of the stem cell: host response and effect of cell therapy

• With graft versus host disease success is related to patient’s response to the materials, what is the innate immune response?

• What is the effect of repetition of treatment?

• Mechanism of action

Katarina Le Blanc, Professor/Specialist Physician, Division of Clinical Immunology, Karolinska Institute (CONFIRMED)

Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL (CONFIRMED) Ross A Macdonald, Managing Director & Chief Executive Officer, Cynata Therapeutics Limited (CONFIRMED)

Francesco Dazzi, Professor of Regenerative and Haematological Medicine, KHP Lead for Cellular Therapies, King’s College London (TBC) Frank Barry, Professor, Cellular Therapy, The National University of Ireland; Senior Scientist, Toronto Arthritis Programme (invited)

Lauren Black, Associate Professor, Biomedical Engineering, Black Lab, Tufts (invited)

Jennifer Elisseeff, Morton Goldberg Professor, Wilmer Eye Institute and Biomedical Engineering, Translational

Tissue Engineering Center, Board of Maryland's Technology Development Corporation (TEDCO), Johns Hopkins

– Baltimore (invited)

14:50 Collaborative phenotyping at King’s: the stem cell hotel

Davide Danovi, Director, Cell Phenotyping Platform, Centre for Stem Cells & Regenerative Medicine, King's

College London (CONFIRMED)



15:40 Overcoming barriers to translation of tissue engineered therapies in the UK and EU: defining better routes to clinic for complex therapies

Martin Birchall, Professor of Laryngology and a Consultant in ENT Surgery, UCLH (CONFIRMED)


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16:00 Regenerative medicine (chaired by Dr. Stephen Badylak): Current carriers and storage solutions for cell therapies

• The ancillary components of cell therapy and its importance on clinical outcomes

• Creating the ideal microenvironment for cells during and after introduction to the patient

• Counteracting viability risk factors: taking out of the frozen environment, suspension, and injection

• What is the best suspension solution to use? How is the cellular treatment dissipated after injection?

• How can we make this process more robust?

Katarina Le Blanc, Professor/Specialist Physician, Division of Clinical Immunology, Karolinska Institute (CONFIRMED)

Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL (CONFIRMED) Francesco Dazzi, Professor of Regenerative and Haematological Medicine, KHP Lead for Cellular Therapies, King’s College London (TBC) Lauren Black, Associate Professor, Biomedical Engineering, Black Lab, Tufts (invited) Jennifer Elisseeff, Morton Goldberg Professor, Wilmer Eye Institute and Biomedical Engineering, Translational Tissue Engineering Center, Board of Maryland's Technology Development Corporation (TEDCO), Johns Hopkins – Baltimore (invited)

David Courtman, Director, Biotherapeutics Core Facilities, Ottawa Hospital Research Institute; CSO, Northern

Therapeutics (CONFIRMED)


16.45 Close of conference

CLINICAL DEVELOPMENT AND TRIALS


CLINICAL TRIALS: CELL THERAPY

Chaired by: Jacqueline Barry, CCO, Cell and Gene Therapy Catapult (CONFIRMED)

14:10 Chair’s remarks Jacqueline Barry, CCO, Cell and Gene Therapy Catapult (CONFIRMED)

14:15 TBC: Case study: IC-T immunotherapy programme, implementing clinical trials and translation to patient care

• Examples of working with various sizes of biotech and pharma

• Logistics, coordination, communication cooperation

• Setting a benchmark of requirements for protocols to streamline operations from both sides Albert Ribickas, Bone Marrow Transplant Manager, Moffitt Cancer Center (CONFIRMED)

14:35 Panel discussion: Clinical development of next generation cellular therapeutics

• Clinical updates and discussion Ajan Reginald, Chief Executive Officer, Celixir (CONFIRMED) Jane Lebkowski, President, Regenerative Patch Technologies (CONFIRMED)


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Chaim Lebovits, CEO, Brainstorm Cell Therapeutics (CONFIRMED)

15:15 Afternoon Refreshment Break

CLINICAL TRIALS: CELL THERAPY & CAR-T

15:45 Conditioning for CAR-T reconstitution: early clinical data and analysis

Jaap Jan Boelens, Chief of Service; Stem Cell Transplant and Cellular Therapies, MSKCC (CONFIRMED)

16:05 Single Administration of CD34 cell Reverses Cardiovascular disease: Evidence from >700 patients in randomized clinical trials • CD34 cells are naturally occurring, pre-programmed vascular repair cells • Pre-clinical models document the ability of locally delivered CD34 cells to induce the formation of new

micro vessels in ischemic tissue, resulting in improved perfusion and function • Clinical studies in multiple cardiovascular diseases provide evidence of durable clinical benefit following a

single administration of autologous CD34 cells

Douglas Losordo, EVP, Global Head of Research and Development & CMO, Caladrius Biosciences (CONFIRMED)

16:25 Panel discussion: Advanced Therapies Treatment Network: facilitating adoption, proof of adoption and how we get there, challenges and adoption - What are your challenges? Chair: Jacqueline Barry, Chief Clinical Officer, Cell and Gene Therapy Catapult (CONFIRMED) Panellists: Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon (CONFIRMED) Marc Turner, Medical Director, Scottish National Blood Transfusion Service (CONFIRMED) Ian McKay, Innovation Lead Advanced Therapies, Innovate UK (CONFIRMED) Representative from bluebird bio (invited) Neil Bell, Senior Vice President & Head of Clinical Operations, Autolus (invited) Representative from Novartis Finn Willingham, Northern Alliance Advanced Therapies Treatment Centre Operations Manager, Newcastle Hospitals (invited)

17:00 New clinical data: Effects of an autologous cell therapy in patients with advanced chronic kidney disease associated with diabetes mellitus • Translational medicine of a cellular therapy for chronic kidney disease • Delivering cellular therapies from-and-to the patient • Defining a dosing regimen Tim Bertram, CEO, inRegen (CONFIRMED)

17:15 Phase III clinical data

Adam Gridley, President & CEO, Histogenics (CONFIRMED)

17:45 Panel discussion: Ensuring safe clinical development of Cell Therapies (CAR-T, DCs, Tregs etc.)

• Safety of new T-Cell Therapies while going through trials

• Safety signals: how can we feed this back to developers?

• What should the regulators be looking for?

• Challenges and problems when developing new T-Cell therapies in the clinic Chair: Victoria English, Co-founder & Editor, Evernow Publishing Ltd, publishers of MedNous (CONFIRMED)


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Douglas Losordo, EVP, Global Head of Research and Development & CMO, Caladrius Biosciences (CONFIRMED)

Jaap Jan Boelens, Chief of Service; Stem Cell Transplant and Cellular Therapies, MSKCC (CONFIRMED)

Sanchez Fueyo, Professor in Hepatology, Kings College London (CONFIRMED)

John Maher, Consultant and Senior Lecturer in Immunology, King's College London, CSO, Leucid (TBC) Anne Flament, Medical Director; Head Clinical Development & Medical Affairs (EU), Celyad (invited)


Conference Day 2: Friday 17th May 2019

CLINICAL DEVELOPMENT: GENE THERAPY

Chaired by:


11:05 TBC Clinical trials case study 1 - Telethon

Stefano Zancan, Head of Clinical Development and Operations, Fondazione Telethon (CONFIRMED)

11:25 Maximising benefits from clinical trials developing safe and efficacious gene therapies as quickly as possible Giorgio Iotti, Head of Program Leadership and Control, Rare Disease Unit, Chiesi Farmaceutici SpA (TBC)

11:45 Gene therapy clinical development case study Andrea Spezzi, Chief Medical Officer and Co-founder, Orchard Therapeutics (CONFIRMED)

12:05 Personalisation of Research Jonathan Sheffield, CEO, NIHR (CONFIRMED)

12:25 New clinical data: UK Cystic Fibrosis gene therapy program and CRM Eric Alton, Professor, Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London; Honorary Consultant Physician, Royal Brompton Hospital (CONFIRMED)

12:45 Vector safety in gene and immune gene therapies • Comprehensive integration profile of your viral vectors • Pharmacovigilance and vector safety in pre- and clinical trials • Determine potential on-/off-target effects of genome editing

Manfred Schmidt, CEO, GeneWerk GmbH (CONFIRMED)

13.05 Networking Lunch

14:10 A multi-stakeholder’s collaborative model to improve the success of translational research: a charity’s perspective

Annamaria Merico, Head of Technology Transfer, Fondazione Telethon (CONFIRMED)

14:30 Developing a viral vector therapeutic for PKU


14:50 Regulatory panel on GCP for ATMP


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Didier Caizergues, Head of regulatory Affairs Department, Genethon (CONFIRMED)


15:40 Cell therapy for neurorepair

Pascale V Guillot, Head of the Cellular Reprogramming and Perinatal Therapy, University College London Institute for Women’s Health (CONFIRMED)

16:00 Panel discussion: Improving patient outcomes in clinical trials and using technology for patient monitoring Jonathan Sheffield, CEO, NIHR (CONFIRMED) Eric Alton, Professor, Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London; Honorary Consultant Physician, Royal Brompton Hospital (TBC)



MANUFACTURE AND BIOPROCESSING TRACKS

CELL THERAPY MANUFACTURING

Day 1 - Conference Day 1: Thursday 16th May 2019

PREPARING FOR LATE STAGE


14:15 In-house manufacture vs. outsourcing – why and when?

When should you think about in-house manufacture instead of outsourcing?

Devyn Smith, Chief Strategy Officer & Head of Operations, Sigilon, Inc (CONFIRMED)

14:35 Case study: Developing an allogenic product - procurement to patient • Platform selection – Why Gamma-delta T cells? • Understanding of the translational challenges to allogeneic treatments • Appreciation of operational challenges Angela Scott, Chief Operating Officer, TC Biopharm (CONFIRMED)

14:55 Are we asking the right questions to achieve deliverable advanced therapies? Lessons from the past and the challenges of the future

• An overview of almost forty years of cell therapeutics development • Establishing the potential of cell-based therapies, enabling technologies and organizations • Developing an applicable regulatory framework and guidance and a better appreciation of drug

development principles • Themes of the present and future as we advance deliverability of cell-based therapies

Robert Preti, Chief Executive Officer and President, Hitachi Chemical Advanced Therapeutics Solutions, LLC (TBC)


40




BIOPROCESSING AND MANUFACTURE FOR CELL THERAPY

15.45 Cell Therapy and Cell – Gene Bio-Manufacturing: A SWOT Analysis of CDMO Market Demand and Future Capacity

• Market analytics will be presented to identify Industry Strengths, Weaknesses, Opportunities and Threats

• Industry weaknesses and opportunities are described

• A comprehensive view of future market trends will be presented

Jane Andrews, Senior Director, Strategic Marketing, BioBridge Global (CONFIRMED)

16:05 Novel closed systems for CAR T-cell production • Closed non-magnetic selection technology (automated T-CATCH) • Soluble reversible activation technology (Expamer) • Short production process

Lothar Germeroth, SVP, Managing Director, Juno Therapeutics, a Celgene Company (CONFIRMED)

16:25 Manufacturing cell therapy

Representative from Irvine Scientific

16.45 A cell culture GMP facility of the future implemented in the present

Reserved for COMECER and CO.DON AG 17:00 Quality systems for regenerative medicine delivery: basic quality requirement for distribution

• Current and future required quality systems standards for the regenerative medicine industry • A comprehensive review of the basic quality systems requirements • Regulatory considerations critical in the successful launch and management of the risks in distribution

Joseph Rotino, Vice President of Quality assurance and Regulatory Affairs, Cryoport (CONFIRMED)


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17:25 Supply chain management, logistics and scale out for T-Cell products

• Case study of a soon to be market authorised product

• Manufacture and logistics of delivery to the patient

• Maintaining quality product at every stage of the delivery process

Alan K. Smith, Executive Vice President, Technical Operations, Bellicum Pharmaceuticals (CONFIRMED)

17:45 Panel discussion: How to manage cell and gene therapy delivery from ‘end to end’ • Sample collection, handling and logistics • Regulation • Product distribution • Cryo-shipping strategies • Manufacturing automation and cell therapy • Reimbursement and integrated care


Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL (CONFIRMED) Lior Raviv, Vice President Development, Pluristem Therapeutics (CONFIRMED) Angela Scott, Chief Operating Officer, TC Biopharm (CONFIRMED) Albert Ribickas, Bone Marrow Transplant Manager, Moffitt Cancer Center (CONFIRMED)

Marc Turner, Medical Director, Scottish National Blood Transfusion Service (TBC) Jean-Pierre Latere, COO, Celyad (TBC)


Day 2 – Day 2: Conference Day 2: Friday 17th May 2019 CELL THERAPY MANUFACTURING


11:05 Enabling commercial scale-out of T-cell manufacturing

• What are the key challenges in scaling an autologous process for commercial?

• The role of closed systems in scalability

• Understanding and controlling Key Quality Attributes Sandro Gomes, Investigator, Cell & Gene Therapy Product Development, GSK (CONFIRMED)

11:25 Opening presentation Reserved for Hamilton Bonaduz AG

11:45 Building a ‘smart city’ for cell and gene

• Concept and design • GMP space building


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• Prefab space • Commercial GMP & vector production • Patient facing/hospital scenario

Jim Kovach, Director, Entrepreneurship and Innovation, UC Davis Health (CONFIRMED)

12:05 Optimizing CGT manufacturing workflows for industrial scale manufacturing”

• Miltenyi Biotec’s generic GMP CAR T Manufacturing protocol as a model for complex CGT manufacturing • Ways towards efficient large-scale processing • TechTransfer concepts for further scale out

Miriam Haak, Director Business Development, Miltenyi Biotec GmbH (CONFIRMED)

12:25 Manufacturing advances and scale-up for simple and complex ATMPs

• Starting with the end in mind – moving from early to later phase trials with a core validated protocol • Incremental improvement in processes to achieve large increases in output • Automation in advanced therapeutics manufacturing John Campbell, Associate Director, Tissues, Cells & Advanced Therapeutics, SNBTS (CONFIRMED)

12:45 Engineering for cell therapy manufacturing

If you are interested in being involved, please contact Erica Baeta at: [email protected] or +44 207 092 1152


SCALE UP & SCALE DOWN

14:10 Moving from artisanal to robust and broad manufacturing models

• Introducing a road map to move the entire industry forward

• Educational work force development projects

• Technical projects such as automated control of microfluidics to measure, control and develop processes better

Richard McFarland/ Becky Robinson, ARMI (invited)

14:30 Plenary with Viral Vectors Manufacturing: Panel discussion: Building your own manufacturing facility Vs. outsourcing the process to partners

Which is the best path to commercial scale? Real life stories from people who have their own facility Vs. people who have outsourced.

Is there a middle ground? What to learn from outsourcing parts of your process, where do you invest and who do you bring in as partners?

Do CMOs have the ability to work at global scale?

Danielle Curfs, CEO, Medace (CONFIRMED)



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Ohad Karnieli, Chair, Process and Product Development Subcommittee, ISCT; CEO & Co-Founder, ATVIO Biotechnology (CONFIRMED) Jim Kovach, Director, Entrepreneurship and Innovation, UC Davis Health (CONFIRMED) Lior Raviv, Vice President Development, Pluristem Therapeutics (CONFIRMED) Farzin Farzaneh, Head of Molecular Medicine, Kings College London (TBC) Leone Patterson, Interim President, CEO, SVP, SVP Chief Financial Officer, Adverum Biotechnologies (invited) Representative from Autolus (invited)

Yossef Levy, VP Cell Production, Brainstorm Cell Therapeutics (invited)


PROCESS DEVELOPMENT

15:40 How much process risk am I carrying forward into clinical stage production?

Robert J Thomas, Professor in Manufacturing for Cell and Gene Therapies; EPSRC Early Career Fellow, Centre for Biological Engineering (CBE) (CONFIRMED)

16:00 Panel discussion: Moving towards full automation and closed systems

• Manufacturing processes • Quality processes • Logistics processes • How do we do improve all these to decrease the cost of manufacture?

Mary Murphy, Senior Lecturer in Regenerative Medicine; Principal Investigator Orthobiology, REMEDI (CONFIRMED)

Ioannis Papantoniou, ATMP Bioprocessing Coordinator, KU Leuven (CONFIRMED)

Lior Raviv, Vice President Development, Pluristem Therapeutics (CONFIRMED) Suzanne S. Farid, Professor of Bioprocess Systems Engineering, UCL (invited)

Jelena Ochs, Manager Business Unit Life Sciences Engineering, Fraunhofer Institute for Production Technology IPT (TBC)



VIRAL VECTOR MANUFACTURING

Day 1 Conference Day 1: Thursday 16th May 2019

VIRAL VECTOR MANUFACTURE: Process development 14:10 Chair’s remarks

Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET (TBC)


44



14:15 Case study: building an in-house baculovirus 200l GMP manufacturing facility

• Volume/application vs safety

• Haemophilia A/B

• How do you tackle the high treatment burden?

Mehdi Gasmi, CSO and CTO, Adverum Therapeutics (invited)

14:35 Manufacturing viral vectors on a commercial level

Paul S Carter, Scientific Leader, Vector Downstream Process Development, Cell & Gene Therapy, GlaxoSmithKline (CONFIRMED)

14:55 TBC: Suspension Vero cell line for production of viral vaccines and viral therapeutics

As the acceptances of viral vectors as a delivery system for therapeutics grows, biomanufacturers are looking for an alternative to the classical adherent cell production models

John Madsen, Head of PD Operations, FUJIFILM Diosynth Biotechnologies Texas (TBC

15:15 Afternoon Networking Break

15:45 Continuous, integrated processes supporting robust and cost-effective viral vector productions

• Real data from constitutive production and from continuous downstream purification of, eg., lentivirus and oncolytic viruses confirms these as key steps to improve consistency, robustness and reduced costs of goods for cell and gene therapy.

Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET (CONFIRMED)

16:05 TBC: Viral vector manufacturing case study

Lucy Foley, Director of Biologics, The Centre for Process Innovation (CPI) (CONFIRMED)

16:25 Accurate purity data in time helps to avoid late surprises when scaling up. Automated TEM analysis data of gene therapy vectors and VLPs


45



Challenges with scale up of gene therapy processes include surprises in purity profiles. Robust analytical solutions are required to to avoid late surprises. Transmission electron microscopy (TEM) provide unmatched insights in characterization of viral gene vectors.

MiniTEM automatically provides:

• Comparative metrics on purity profiles of viral particle samples • Automatically differentiates intact viral particles from process related impurities • Correlation between potency and viral particle morphology

Josefina Nilsson, Head of Business Unit, EM Service Vironova (CONFIRMED)

16:45 BAC-to-AAV: The Technology for Large Scale AAV Manufacturing

• Current AAV production systems and their challenges • First generation system for AAV production in Sf9 cells and its deficiencies • Virovek's serum-free "BAC-to-AAV" technology and its advantages • Novel three-phase partitioning (TPP) as upstream process for large scale AAV purification Haifeng Chen, CEO, Virovek, Inc. (CONFIRMED

17:05 PANEL: progress and challenges in Viral Vector Manufacturing: assays and analysis

How to look at the percentage empty vs filled viral particles

Looking a potency of construct, how can this be measured and what are the tools?

Infection of cell: analysing gene expression, functionality, level and potency

Chair: Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET (CONFIRMED)

Panellists:

Paul S Carter, Scientific Leader, Downstream Process Development, Cell & Gene Therapy, GlaxoSmithKline (CONFIRMED)

Fraser Wright, Translational Research and Development, Gene Therapy, AKOUOS (invited)

17:45 BIOPROCESSING CLOSING PLENARY: Bioprocessing Panel discussion: How to manage cell and gene therapy delivery from ‘end to end’ • Sample collection, handling and logistics • Regulation • Product distribution • Cryo-shipping strategies • Manufacturing automation and cell therapy


46



• Reimbursement and integrated care


Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL (CONFIRMED) Lior Raviv, Vice President Development, Pluristem Therapeutics (CONFIRMED) Marc Turner, Medical Director, Scottish National Blood Transfusion Service (TBC) Jean-Pierre Latere, COO, Celyad (TBC)


Conference Day 2 - Day 2: Conference Day 2: Friday 17th May 2019

VIRAL VECTOR MANUFACTURE 11:00 Chair’s remarks

11:05 Application of the new EU GMP guidelines specific to ATMPs – a quality assessor’s perspective

Janet Glassford, Senior Quality Assessor - Medicines and Healthcare Products Regulatory Agency (MHRA) (CONFIRMED)

11:25 TBC: Getting it right first time, viral vector production from end to end

Presentation from Pall

11:45 TBC: Technology for safe and effective viral vector manufacture

Reserved MolMed

12:05 Improving safety and efficacy of viral vector manufacture


12:25 Panel discussion: Assays - specific challenges, duration, standardization

• Potency assays for comparability, characterization and release

• What type of release assays are adequate for a regulator to approve the product in both cell and gene therapies?

• How do regulators define quality in release assays and how can the industry use this as a guide for regulatory approval?

• How do regulators validate assays? What kind of assays can actually be validated?

• Viral vectors and gene modified cell products: are these release assays really necessary? Do you need one for your starting material and one for your end product?

• Advice on extractables and leachable - what is the regulatory guidance on determining the purity of the final product? Which tests have worked well for products that have made it to market?

• Different regulations for EU and US- can we harmonise on the quality aspect of release testing?

• How do you predict if a batch will be successful at a clinical level?

• Consistency: How do you define your critical quality attributes and assays, so you know each batch is the same?



47



Janet Glassford, Senior Quality Assessor - Medicines and Healthcare Products Regulatory Agency (MHRA) (CONFIRMED)

Sergey A. Piletsky, Professor of Bioanalytical Chemistry, Chemistry Department, College of Science and Engineering, University of Leicester (TBC)

Timothy J. Miller, President & CEO, Abeona Therapeutics (invited)

Joseph Rabinowitz, Senior Director, of AAV Capsid Development, Bamboo Therapeutics, Inc., (invited)



14:10 Designing your viral vector manufacturing facility with the end in mind


14:30 Plenary with Cell Therapy Manufacturing Panel discussion: Building your own manufacturing facility Vs. outsourcing the process to partners

Which is the best path to commercial scale? Real life stories from people who have their own facility Vs. people who have outsourced.

Is there a middle ground? What to learn from outsourcing parts of your process, where do you invest and who do you bring in as partners?

Do CMOs have the ability to work at global scale?


Ohad Karnieli, Chair, Process and Product Development Subcommittee, ISCT; CEO & Co-Founder, ATVIO Biotechnology (CONFIRMED) Farzin Farzaneh, Head of Molecular Medicine, Kings College London (TBC) Representative from Autolus (invited)


15:10 Afternoon Networking Break

VIRAL VECTOR MANUFACTURE: Preparing for late stage

15:40 Highlighting and addressing ‘critical path’ issues in bringing vector manufacturing processes from Phase I/II to commercial scale


16:00 BIOPROCESSING CLOSING PLENARY: Panel discussion: Building your own manufacturing facility Vs. outsourcing the process to partners

• Which is the best path to commercial scale? Real life stories from people who have their own facility Vs. people who have outsourced.




48



• Is there a middle ground? What to learn from outsourcing parts of your process, where do you invest and who do you bring in as partners?

• Do CMOs have the ability to work at global scale? t


Ohad Karnieli, Chair, Process and Product Development Subcommittee, ISCT; CEO & Co-Founder, ATVIO Biotechnology (CONFIRMED) Jim Kovach, Director, Entrepreneurship and Innovation, UC Davis Health (CONFIRMED) Lior Raviv, Vice President Development, Pluristem Therapeutics (CONFIRMED) Farzin Farzaneh, Head of Molecular Medicine, Kings College London (TBC) Representative from Autolus (invited)


16.40 Chair’s Closing Remarks


PITCH AND PARTNER

Day 1 - Conference Day 1: Thursday 16th May 2019

INVESTOR FORUM PANELS

14:10 Chair’s remarks Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

14:15 Investor panel: How to attract investment into cell and gene therapy: hear how different investors decide on who and what they invest in, and why

• Compare and contrast different VC methodologies

• What other types of innovative funding is there?

• Unorthodox ways to get money

• What do pharma look for when in-licensing?

Dmitry Kuzmin, Managing Partner, 4BIO Capital (CONFIRMED)

Alan J Muir, Director Life Science & Healthcare Investment, Seven Hills Venture Partners (CONFIRMED)

Dominic Schmidt, Partner, Syncona (CONFIRMED)

Marc Estigarribia, Head of Cross Border Origination and Engagement, Investment Banking, MSQ Ventures (invited)

14:55 Multi-perspective discussion: How to partner with public/private organizations

• How to approach big pharma/government organisations for support

• How can companies correctly identify a fit and interest?

• Can partnering prevent biotech start-ups from getting stuck before Ph1?


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• Do and don’ts with partnerships

Edward Hodgkin, Partner, Syncona Partners (CONFIRMED)

Gareth King, Investment Manager, Catapult Ventures (CONFIRMED)

Glenn Pierce, EIR, Third Rock Ventures, CMO, Ambys Medicines (invited)


PITCH AND PARTNER

INVESTOR JUDGES: Vijay Barathan, Life Science Partner, Catapult Ventures Group (CONFIRMED)

Armando Cuesta Díaz, Founding Partner, Ilana Capital (CONFIRMED) Glenn Pierce, EIR, Third Rock Ventures, CMO, Ambys Medicines (CONFIRMED)

Ian McKay, Innovation Lead Advanced Therapies, Innovate UK (TBC)

15:45 Pitch 1. Harnessing synthetic nanoclay gels for regenerative medicine

Agnieszka Janeczek, Interim CEO, Renovos (CONFIRMED)

16:00 Pitch 2. Regenerative medicine – a small molecule approach

Michael Stein, Chairman & CEO, Oxstem (CONFIRMED)

16.15 Pitch 4.

David Mazzo, CEO, Caladrius Biosciences (CONFIRMED)

16:30 Pitch 5. A new TCR approach to take solid tumours

Miguel Forte, Chief Executive Officer, Zelluna Immunotherapy (CONFIRMED)

16:45 Pitch 6. Taking the bull by its horns: regenerating human hearts with human heart cells

Marcos Valadares, Co-founder and CEO, PluriCell Biotech (CONFIRMED)

17:00 Pitch 7. Pioneering a new class of medicines: shielded living therapeutics Rogerio Vivaldi, President and Chief Executive Officer, Sigilon Therapeutics (CONFIRMED)


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17:15 Pitch 8. Bioengineering Transplantable Whole Organs

Jeff Ross, CEO, Miromatrix (CONFIRMED)

17:30 Pitch. 9 RESERVED FOR LCT

17:45 Pitch 10. Sponsorship opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, [email protected]) for further details

18:00 Pitch 11.

Shay Hershcovich, Head of Businshiess Development, Betalin Therapeutics (CONFIRMED)

18:15 Judges deliberation, winners will be announced at the networking drinks reception.




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CORD BLOOD

Conference Day 1: Conference Day 1: Thursday 16th May

08:00 Registration opens

08:50 Welcome from Terrapinn

08:55 Chair’s opening remarks Moshe Israeli, Director of Tissue Typing Laboratory, Rabin Medical Centre (CONFIRMED)

Keynotes – Novel Applications and the Future of Cord Blood

9:00 Extending cord blood to regenerative therapies for the brain

• The history and evolution of cord blood banking and transplantation

• Quality measures of cord blood units

• New clinical applications for cord blood therapies Joanne Kurtzberg, MD; Director, Marcus Center for Cellular Cures; Director, Carolinas Cord Blood Bank; Director, Pediatric Blood and Marrow Transplant Program (CONFIRMED)

9:20 Cord blood for prevention of type 1 diabetes

• CD4+CD25+ regulatory T cells (Treg) in umbilical cord blood provide potential as an immunotherapeutic tool

• Considerable evidence indicates that type 1 diabetes is characterised by abnormalities in Treg number and function

Maria Craig, Professor of Paediatric Endocrinology, The Children's Hospital at Westmead (CONFIRMED)

9:40

Keynote Panel Discussion - The future of the cord blood industry – what are the advances that need to be made?

• Cord blood banking

• Research and collaborative efforts

• Regulation

• Public awareness and education Joanne Kurtzberg, Director, Carolinas Cord Blood Bank, Chief Scientific Officer, Robertson Clinical & Translational Cell Therapy Program, Director, Pediatric Blood and Marrow, Duke Center for Autism (CONFIRMED) Elizabeth Shpall, Professor, Howard and Lee Smith Chair in Cancer Research, Director, Cell Therapy Laboratory and Cord Blood Bank, Deputy-Chair, Stem Cell Transplantation and Cellular Therapy, MD Anderson Cancer Center (CONFIRMED) Guy Parkes, Head of Stem Cell Donation & Transplant, NHSBT (CONFIRMED) Colleen Delaney, Founder and Chief Medical Officer, Nohla Therapeutics (TBC) Mayur Abhaya, CEO, LifeCell (CONFIRMED)

10.20 Networking Refreshment Break

Roundtable Discussion Session


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11:20 Cord Blood banking business models

Amnon Pelz, CEO, Taburit

Commercialisation of regenerative medicine

products Greg Bonfiglio, Managing

Director, Proteus Sp

Reserved for sponsor If you are interested in being involved, please

contact Erica Baeta at: [email protected] or

+44 207 092 1152

Title to be announced Chris Bond, Managing Director,

Future Health Biobank

Hospital exemption procedures Mathias Svahn, CEO, NextCell

Pharma

12:20 An overview of World Cord Blood day Rachel Manley, Head of Global Outreach, Save the Cord Foundation (CONFIRMED)


Cord Blood Expansion and Engraftment

Chair: Moshe Israeli, Director of Tissue Typing Laboratory, Rabin Medical Centre (CONFIRMED)

14:10 Cord blood expansion for engraftment and regenerative medicine

• Current state of the art for cord blood expansion in the transplant setting

• MSCs for regenerative medicine and GVHD

• Will discuss the GMP production of cord blood tissue derived MSCs for clinical use Elizabeth Shpall, Professor, Howard and Lee Smith Chair in Cancer Research, Director, Cell Therapy Laboratory and Cord Blood Bank, Deputy-Chair, Stem Cell Transplantation and Cellular Therapy, MD Anderson Cancer Center (CONFIRMED)

14:30 Improvement of functional preservation of cord blood stem and progenitor cells stored at +4°C after collection or after ex vivo expansion: development of a potentially injectable conservation solution.

• After ex vivo expansion, the hematopoietic stem cells (Scid Repopulating Cells) rapidly decline in human-albumin solution (used routinely) and their activity is almost completely lost after 48h. This can be prevented by using an appropriate culture medium instead of human albumin (antioxidants, nutritive elements, microelements).

• The decline of hematopoietic stem cell activity (although to a lesser extent) detected after cord blood collection using the standard routine procedure can be prevented by using the same culture medium.

• This medium is not an optimal choice since it should be removed before injection of the cells into the patient. Thus, we have developed a conservation solution composed of already registered injectable pharmaceutical products, able to reproduce the same beneficial effect using both freshly collected cord blood cells and ex vivo expanded ones

Zoran Ivanovic, Scientific Director, French Blood Institute (CONFIRMED) 14:50

Optimization of a stem cell agonist cocktail for the expansion of cord blood hematopoietic stem cells

• Review advances in HSC expansion technologies

• Optimization of small molecules-based HSC agonist cocktail by statistical design of experiments

• Engraftment properties of cord blood hematopoietic stem cells expanded by small molecules Nicolas Pineault, Development Scientist, Canadian Blood Services (CONFIRMED)

15:10 Unique features of reconstituting immune cells after cord blood transplantation; opportunities for adjuvant immunotherapies

• Immune reconstitution after cord blood transplantation is distinct from reconstitution after other cell sources

• Early immune reconstitution after cord blood transplantation is excellent and has unique features to get better infectious and disease control


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• Immune reconstitution monitoring should be standardized / harmonized across clinical trials Jaap Jan Boelens, Chief of Service; Stem Cell Transplant and Cellular Therapies, MSKCC (CONFIRMED)

15:30 Networking Refreshment Break

Collection, Bioprocessing and Storage

Chair: TBC

16:30 Advances and challenges in umbilical cord blood and tissue bioprocessing

• Umbilical cord tissue and blood is the rapidly advancing field of regenerative medicine for both autologous and allogeneic therapeutic applications

• Optimization of GMP-compliant manufacturing strategies, collection and bioprocessing of cord blood and tissue

• Comparison with other type of cells

• Regulatory challenges and considerations for minimal manipulation and homologous use

• Future direction; iPSCs or exosomes Aisha Khan, Executive Director Laboratory Operations, Interdisciplinary Stem Cell Institute, University of Miami Miller School of Medicine (CONFIRMED)

16:50 Reserved for sponsor If you are interested in being involved, please contact Erica Baeta at: [email protected] or +44 207 092 1152

17:10 Toticyte – a revolution in cord blood processing

• >99.5% haematocrit removal

• At least 3x more viable stem cells at point of use than any other method

• Maximum cell recoveries, even for small volumes

• Primitive cell types retained

• Clinically compatible

• HTA authorised use, CE marked, ISO13485 certified Jeff Drew, CSO, Cells4Life (COFIRMED)

17:30

Panel discussion: the operational challenges of cord blood collection and cryopreservation • Review of collection procedures

• Best practices recommendations: modifying infrastructure and support

• Methods to improve conversion rate

Sue Mundy, Head of Cord Blood Collection, Anthony Nolan Cord Blood Programme (CONFIRMED) Nicolas Pineault, Development Scientist, Canadian Blood Services (CONFIRMED) Warachaya Sutheesophon, Medical Director, Thai StemLife (CONFIRMED) Mike Watts, Director of Cellular Therapy Laboratory, UCLH (invited)

18:10 End of Day 1 Remarks

18:15 Offsite Networking Drinks


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Conference Day 2: Friday 17th May

08:55 Chair’s welcome back

Novel Applications of Cord Blood

Chair: TBC

9:00 Using the whole cord blood to make novel therapies

• Use of umbilical cord blood cells for immunotherapies

• Potential uses of cord blood plasma and platelets

• Uses of cord blood derived mesenchymal stem cells Beth Shaz, Chief Medical and Scientific Officer, Executive Vice President, New York Blood Center (CONFIRMED)

9:20 The application of hyperbaric oxygen therapy to improve umbilical cord blood stem/progenitor cell homing and engraftment

• Erythropoietin signaling mediates umbilical cord blood CD34+ cell differentiation, bone marrow homing, and engraftment

• bloodHyperbaric oxygen (HBO) therapy in the setting of clinical umbilical cord blood transplantation is well-tolerated and is potentially associated with improved long-term outcomes

• HBO reduces erythropoietin serum level and improves blood count recovery in the setting of clinical umbilical cord blood transplantation

Omar Aljiwati, Associate Professor, University of Rochester Medical Center (CONFIRMED)

9:40 Title TBC Machi Scaradavou, Medical Director, New York Blood Center's National Cord Blood Program, Pediatric Hematologic Oncologist, MSKCC (CONFIRMED)

10:00 Therapeutic potential of UCB-derived extracellular vesicles

• Clinical use of Extracellular vesicles: Opportunities and Threats

• How to go from UCB to a GMP-compliant product based in extracellular vesicles for Clinical application

• Therapeutic potential of Exogenus Therapeutics’ proprietary small extracellular vesicles

• Pipeline development and future Roadmap Joana Correia, Executive Director and Chief Scientific Officer, Exogenus (CONFIRMED)

10.20 Networking Refreshment Break

Cord Blood Banking


11:30 Translational medicine in Russia: how it works

• Transitioning from a cord blood bank to a centre of fundamental research and regenerative medicine

• Preliminary research results (morphofunctional properties of mesenchymal stromal cell during long-term expansion and methods of intravital MMSC labeling with superparamagnetic nanoparticles)

• Experience in clinical application of cell technologies (traumatology, liver diseases) as well as particularities of national federal legislation in the field of regenerative medicine will be discussed

Dmitrii Ivolgin, Medical Director, Pokrovskij Stem Cell Bank (CONFIRMED)

11:50 Case study of one of the largest cord blood banks in Europe

• Success story and challenges met along the way

• Research updates


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Tomasz Baran, Board Member, PBKM/FamiCord Group (CONFIRMED)

12:10 Can frequency of therapeutic application of stem cells be influenced?

• The meaning of availability, reimbursement, gaining scientific reputation and clinical evidence Maria Schoepe, Deputy Head of Production, Vita34 (CONFIRMED)

12:30 Strategies of operation of the Public Cord Blood Bank of Crete

• Optimizing donor recruitment

• Enrichment of the national repository Helen Papadaki, Professor of Haematology, Public CBB of Crete (CONFIRMED) Irene Mavroudi, Researcher, Public CBB of Crete (CONFIRMED)


Research and Development

Chair: TBC

13:50 Application of Cord Blood NK cell therapies in the clinic

• Clinical studies suggest that adoptive transfer of allogeneic natural killer (NK) cells represent a promising treatment for patients with hematological malignancies and solid tumors

• Celularity is developing an allogeneic, off-the-shelf NK cell product that is derived from placental hematopoietic stem cells and exhibits substantial cytolytic activity against various cancer cell lines, primary AML and primary MM cells

• Clinical and translational development of Celularity NK cell product will be discussed Xiaokui Zhang, CSO, Celularity (CONFIRMED)

14:10 Intravenous infusion of umbilical cord tissue (UC) derived Mesenchymal Stem Cells (MSCs) versus bone marrow (BM) derived MSCs to evaluate cytokine suppression in Patients with chronic inflammation due to Metabolic Syndrome (CERES)

• A Phase I/II, Randomized, Placebo-controlled Comparative Study to compare Umbilical Cord Tissue (UC)

Derived Mesenchymal Stem Cells (MSCs) Versus Bone Marrow (BM) Derived MSCs

Joshua Hare, Professor of Medicine, University of Miami (CONFIRMED)

14:30 Success from a phase I clinical trial: cord blood derived mononuclear cells for the treatment of hypoplastic left heart syndrome

• What is HLHS?

• Pre-clinical development using UCB-MNCs in small and large animals

• Phase I clinical study and results Susana Cantero Peral, Associate Consultant I-Research, Division of General Internal Medicine, Mayo Clinic (CONFIRMED)

14:50 Driving public banked cord blood inventory utilization through research and development

• Optimizing HPC,Cord Blood inventory for use in stem cell transplant

• Developing clinical pathways and product pipelines through Research

• Leveraging existing infrastructure, experience and resources to diversify cord blood utility with a Cell Therapy Incubator Laboratory

Marcie Finney, Executive Director, Cleveland Cord Blood Center (CONFIRMED)

15:10 Cellular Mechanisms in support of allogenic human Amnion Epithelial Stem Cell Transplantation without Immunosuppression

• Stem cell properties typically expressed by human amnion epithelial (AE) stem cells

• How to isolate and identify placenta-derived AE cells

• AE transplantation in preclinical model of liver diseases

• Route of infusion to enhance liver engraftment


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• How allogenic AE cells may avoid immunorejection without immunosuppressant in support Roberto Gramignoli, Professor, Karolinska Institutet (CONFIRMED)

15:30 Networking Refreshment Break

Haploidentical Transplants: Clinical Updates


16:00 A snapshot of current research into Haploidentical transplants

• Summary of the advancements in Haplo-SCT in recent years and comparison of other transplantation modalities

• Strategies for improving clinical outcomes

• Donor selection and graft-versus-host disease Moshe Israeli, Director of Tissue Typing Laboratory, Rabin Medical Centre (CONFIRMED)

16:20 Haploidentical vs cord blood transplantations

• Pros and cons of both – case study examples

• Is there sufficient data to support one donor type over another?

• Is institutional practice influenced by experience and research interest? Chair: Moshe Israeli, Director of Tissue Typing Laboratory, Rabin Medical Centre (CONFIRMED) Kavita Raj, Consultant Haematologist, Guy's and St Thomas' Hospital (CONFIRMED) Andy Peniket, Clinical Director of Haematology and Bone Marrow Transplantation, Oxford University Hospitals (CONFIRMED) Eduardo Olavarria, BMT Program Director, Consultant Haematologist, Imperial College Healthcare NHS Trust (CONFIRMED) Ronjon Charaverty, Professor of Haematology & Cellular Immunotherapy, UCL (invited) Jakob R. Passweg, Chair of Haematology, University of Basel (invited)

17:00 Chair’s final remarks



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EXHIBITION VISITOR AGENDA: ON – FLOOR SEMINAR THEATRE

Day 1: Thursday 16th May 2019

Exhibition Visitor Agenda: Talks and Panels

Day 1 –Conference Day 1: Thursday 16th May 2019

Location: Seminar Theatre

09:30

09:40

09:50

10:00

10:10

10:20

Chair: Gopalan Narayanan, Vice President, Disruptive Biologics, Voisin Consulting Life Sciences (VCLS) (CONFIRMED)

10:30 Conference breaks for coffee and networking

10:30 Australian Regenerative Medicine Opportunities Dan Grant, CEO, MTPConnect (CONFIRMED)

10:40 Introducing MedCity and the Advanced Therapies Network

Sarah Haywood, CEO, MedCity (CONFIRMED)

10:50 Presentation from BIOM

Representative from BioM

11:00 Cellbox - The Live Cell Shipper Transport at 37°C

• Status Quo for shipping cell cultures - Cold chain technologies. • From Innovation to Product - The Cellbox portable CO2 incubator as a solution for shipping live

cell cultures. • The advantages of live cell shipping - A case study of the Cellbox in action.

Kathrin Adlkofer, CEO, Cellbox Solutions (CONFIRMED)


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11:10 VectorBuilder: A revolutionary platform for vector design, custom cloning and virus packaging

• With over 156,000 delivered to date

• Expansive collection of components reducing cost and turnaround time

• High quality virus packaging: Lentivirus, Adenovirus, AAV and MMLV

Matthew Wheeler, Associate Director, VectorBuilder Europe (CONFIRMED)

11:20 Artificial membrane-binding proteins for cell therapies

• The biodistribution of therapeutic cells

• Artificial membrane-binding protein (AMBP) technology

• Augmentation of therapeutic cells by AMBPs Ben Carter, Chief Technical Officer, CytoSeek (CONFIRMED)

11:30 – Conference sessions resume

11:30 Reserved for AMSBIO

11:40

11:50

12:00

12:10

12:20

12:30

12:40

12:50 Cytoplasmic membrane re-engineering for next-gen cell therapies

• Describes a new methodology for the synthesis of novel artificial cell membrane binding proteins.

• Technology can be readily applied to therapeutic cells to provide tissue homing, adhesion, extracellular matrix formation and hypoxia resistance.

• Platform is extremely versatile and could be utilized to display virtually any protein on any cell. Adam Perriman, Associate Professor, Biomaterials, School of Cellular & Molecular Medicine, University of Bristol (CONFIRMED)

13:00

13:10- Conference Breaks for lunch

Location: Seminar

13:10 KEYNOTE PRESENTATION BY DANIEL KRAFT

13:20

13:30 Bioengineering Transplantable Whole Organs Miromatrix is a biotechnology company engaged in the development of fully biological human organs to solve the chronic shortage of transplantable organs utilizing its patented perfusion decellularization/recellularization technology, as well as the development of the next generation of acellular based products. Jeff Ross, CEO, Miromatrix (CONFIRMED)


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13:40 Challenges with advanced therapies in clinical trials

Rachel Smith, Senior Clinical Project Manager, TCTC Group (CONFIRMED)

13:50 BioRN initiatives in leveraging the unique combination of global pharma and leading academic

institutions in the region

• HAEP – a new instrument for facilitated technology transfer between SMEs, academia and global healthcare companies

• Health Axis Europe critical mass: a large international network of innovators

• The vibrant Rhine/Main/Neckar life science region

Julia Schaft, Managing Director, BioRN (CONFIRMED)

14:00 A new 3D cell culture platform (encapsulation) led to unprecedented population doubling (>

x100/passage) while improving genetic stability and cell differentiation (functional neurons)

• C-Stem™ encapsulation, cultivation and formulation platform enabled:

• Pluripotent Stem Cell amplification factor of >100 x per passage

• Improved iPSC genetic stability after high number of doubling

• Dramatic improvement of neuron functionalization demonstrated on animal model

• Scalable differentiation of iPSC into numerous cell/tissue type (cardiomyocytes, chondrocytes, gut crypt)

Fabien Moncaubeig, COO, Treefrog Therapeutics (CONFIRMED)

14:10- Conference resumes

14:10 Reserved for Healthgen Biotechnology

14:20 Reserved for Albcura

14:30

14:40

14:50

15:00

15:15- Conference breaks for coffee and networking

Location: Seminar

15:20 Reserved for Future Family


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15:40 Reserved for Israeli Society of Gene and Cell Therapy

15:50

16:00 Best practice in successfully commercializing an academic spin-out company

• Guidance for both the investor and University side at this early spin-out and seed investment stage to try and help each side understand the other’s perspectives.

• Best practices from both investor and academic perspectives

• Suggested advice of how to bridge the different starting points

Govind Pindoria, Director of Venture Support Unit, Imperial Innovations (CONFIRMED) Dominic O'Regan, Director & Investor, New Mosaic Ltd (CONFIRMED)

16:10

16:20

16:30

16:40 – main conference resumes


Exhibition Visitor Agenda: Talks and Panels


Location: Seminar Theatre

Chair:

10:00

10:10 Presentation from Cobra Biologics

10:20

10:30

10:40 Presentation from Scinus Cell Expansion

10:50

11:00

11:00 – Conference Sessions resume

11:10 MANUFACTURE AND BIOPROCESSING

11:20 Light distilleries of cells and proteins

• Proteins and cells are bound on the adsorbent in dark and are recovered by applying light at a

specific wavelength

• This is a very innovative and safe manner to purify therapeutics that are so biochemically delicate

that current purification strategies fail. We call this technology "Light distilleries of cells and proteins

Stefano Menegatti, Assistant Professor in Chemical and Biomolecular Engineering, North Carolina State University (CONFIRMED)

11:30

11:40 The AUTOSTEM PROJECT Mary Murphy, Senior Lecturer in Regenerative Medicine; Principal Investigator Orthobiology, REMEDI (CONFIRMED)

11:50

12:00 Composite ceramic monolithic columns for chromatographic separations

• Flow properties • The challenge of functionalization • Impact on biomolecule downstream process Cristiana Boi, President of the European Membrane Society, Assistant Professor, DICAM (CONFIRMED)

12:10

12:20


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12:30 3D-print your own chromatographic column

• Development of new materials compatible with downstream operations and 3D printing technology • Demonstration of protein capture from cell culture supernatant • Opportunities and challenges of 3D printed chromatography columns Simone Dimartino, Senior Lecturer, Institute for Bioengineering, University of Edinburgh (CONFIRMED)

12:40

12:50

13:05 – Conference streams break for lunch

Location: Seminar

13:05 – 13:45

Women in STEM Lunchtime panel discussion

13:50 Next generation technologies: Implantable robotics, a rapidly moving and exciting field that dovetails with advances in tissue engineering and biomaterials Martin Birchall, Professor of Laryngology and a Consultant in ENT Surgery, UCLH (CONFIRMED)

14:05 – Conference sessions resume

14:00-14:40

Inside the ethics committee: Ethics behind CRISPR and gene edited therapeutics Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

14:40

14:50

15:00

15:10 – Conference sessions break for coffee

Location: Seminar

15:10 Brexit: the potential policy, regulatory and public affairs outcomes of Brexit and the possible implications for the life science industry Pharmaceutical companies continue to plan for the UK’s exit from the EU. As they do, the terms on which the UK will leave - and the terms of the future relationship – remain uncertain Elliot Dunster, Head of External Affairs, Association of the British Pharmaceutical Industry (ABPI) (CONFIRMED) Representatives: CRUK Wellcome MHRA &EMA

15:20

15:30

15:40

15:40 Conference sessions resume

15:40 – Exhibition hall is closed, thanks for coming!


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15-17 May 2019 Business Design Center, London World Advanced … · 2019-05-28 · 1 15-17 May 2019 Business Design Center, London World Advanced Therapies & Regenerative Medicine

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