PLEASE NOTE THIS IS AN EARLY DRAFT AGENDA …...PLEASE NOTE THIS IS AN EARLY DRAFT AGENDA AND ALL SPEAKERS ARE IN THE PROCESS OF BEING INVITED UNLESS SPEIFIED AS ‘ONFIRMED’ Johannes

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PLEASE NOTE THIS IS AN EARLY DRAFT AGENDA AND ALL SPEAKERS ARE IN THE PROCESS OF BEING INVITED UNLESS SPECIFIED AS ‘CONFIRMED’

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Table of Contents 2020 Advanced therapies speakers ............................................................................................................................................................................................................................ 2

2020 Sponsored content: ............................................................................................................................................................................................................................................ 7

Advisory Board (2020): ............................................................................................................................................................................................................................................... 9

Supporting partners: ................................................................................................................................................................................................................................................... 9

PRE-CONFERENCE WORKSHOP AND SUMMIT DAY ................................................................................................................................................................................................. 10

CONFERENCE DAY 1 - TUESDAY 31st March 2020 ................................................................................................................................................................................................... 11

CONFERENCE DAY 2 - WEDNESDAY 1st April 2020 .................................................................................................................................................................................................. 16

Networking break content: Seminar Theatre Agenda ............................................................................................................................................................................................. 22

2020 Advanced therapies speakers

Keynotes:

Martin Pule, SVP, Founder & CSO, Autolus; Clinical Senior Lecturer, Haematology, UCL Cancer Institute

Prasad Adusumilli, Deputy Chief and Attending, Thoracic Surgery; Vice Chair, Translational Research, Dept. of Surgery, Head, Solid Tumors Cell Therapy, Cellular Therapeutics

Center (CTC); Director, Mesothelioma Program, Memorial Sloan-Kettering Cancer Center

Emanuele Ostuni, Head of CAR-T Europe, Novartis

Pascal Touchon, President, Chief Executive Officer & Board Member, Atara Biotherapeutics

Federico Mingozzi, CSO, Spark Therapeutics

Steve Kanner, CSO, Caribou Biosciences

Adrian Woolfson, Head of R&D, Sangamo Therapeutics

Fred Chereau, President & CEO, LogicBio Therapeutics

Christopher R. Heery, CMO, Precision BioSciences

Joanne Kurtzberg, Professor of Pediatrics and Pathology; Director, Marcus Center for Cellular Cures, Duke University Medical Center

Anthony Lodge, CMC – Regulatory Lead, Kite Pharma

TBC Bradley Campbell, President and COO, Amicus

Confirmed and provisionally confirmed speakers:

Abdulkader Rahmo, CEO, SMSbiotech

Adrian Woolfson, EVP, Head of R&D, Sangamo Therapeutics

Agnieszka Janeczek, Founder/COO, Renovos

Aisha Khan, Executive Director of Lab Operations at the Interdisciplinary Stem Cell Institute, Miller School of Medicine, University of Miami

Aisleen McColl-Carboni, Senior Scientist, Sanofi

Alain Vertès, Managing Director, NxR Biotechnologies GmbH

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

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

Aline Miller, Chief Executive Officer and Co-Founder, Manchester BIOGEL

Albert Ribickas, Bone Marrow Transplant Manager, Moffitt Cancer Center

Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)

Amandine Breton, Cell Therapy Operations Manager, Orchard Therapeutics

André Gerth, CEO, BioPlanta

Andrew Sewell, Distinguished Research Professor and Wellcome Trust Senior Investigator, Division of Infection and Immunity, Cardiff University School of Medicine

Angela Columbano, Director of Business and Development, Genethon

Annie Hubert, Senior Director, European Public Policy, Alliance for Regenerative Medicine

Antonio Rodríguez Acosta, Deputy Qualified Person. Cellular Manufacturing Unit, Regional University Hospital -Biomedical Research Institute (IBIMA) Málaga

Anthony Lodge, CMC – Regulatory Lead, Kite Pharma

Axel Böhnke, Director Market Access EU North, PTC Therapeutics


Barry Rosen, Senior Principal Scientist/Director, AstraZenca

Bill Tente, Chief Regulatory Officer, Humacyte

Bjkarki Johannesson, Senior Research Investigator, NYSCF

Bo Kara, Vice President, Process Development, Evox Therapeutics

Bob Valamehr, Chief Development Officer, Fate Therapeutics

Bobby Gaspar, CSO, Orchard Therapeutics

Boris Greber, Chief Scientific Officer, Head of Production, RheinCell Therapeutics

C. David Pauza, CSO, American Gene Technologies

Cecilia Götherström, Associate Professor, Department of Clinical Science, Intervention & Technology, Karolinska Institutet

Chaim Lebovits CEO, Brainstorm Cell Therapeutics

Chandler Crews, President, The Chandler Project

Chirag Desai, Professor of Surgery, Director, Chronic Pancreatitis & Autologous-Islet Cell Transplant Program, UNC School of Medicine

Chris Coughlin, CMO, Rubius Therapeutics

Christine Le Bec, Head of CMC Gene Therapy, working on AAV vectors, Sensorion

Christoph Herwig, Professor of Biochemical Engineering, Vienna University of Technology

Christopher R. Heery, CMO, Precision BioSciences

Claire Booth, Senior Clinical Lecturer, Gene Therapy, UCL GOS Institute for Child Health

Colleen Delany, Founder & CSO, Nohla Therapeutics

Cornelia Kasper, Full University Professor, BOKU

Cynthia Bamdad, CEO, Minerva Biotechnologies

Daniel O’Mahony, Partner, Seroba Life Sciences

Danilo Maddalo, Laboratory Head, Novartis Institutes for Biomedical Research

Darren Walsh, Country Manager UK / ROI, (Senior Director – Market Access and Govt. Affairs EMEA), Orchard Therapeutics

Dave Greenwald, Vice President, Business Development, Deerfield Management

Dave Lennon, President, AveXis

Dave Morris, VP Therapeutic Discovery, Precision BioSciences

David Blakey, CSO, MiNA Therapeutics

David Gilham, VP Research & Development, Celyad

David Morrow, Senior Scientific Programme Manager-ATMP/Vaccines, EATRIS

David Pearce, President of Innovation and Research, Sanford Health

David Schaffer, Professor, Chemical and Biomolecular Engineering, University of California, Berkeley, and Co-Founder, 4D Molecular Therapeutics

David Mazzo, President & CEO, Caladrius Biosciences

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

Dawn Henke, Senior Technical Program Manager, Standards Coordinating Body

Dawn Maier, Sr. Director Process Development & Manufacturing, ElevateBio

Debora Esposito, Assistant Professor, Regenerative Medicine, North Carolina State University

Deepak Jain, COO, InRegen

Derek Dashti, Founder, Inventor & CEO, D&P Bioinnovations

Detlev Parow, Head of Department of Medicines, Therapeutic Appliances and Remedies, DAK

Devyn Smith, Chief Operating Officer & Head of Strategy, Sigilon Therapeutics

Didier Caizergues, Head of Regulatory Affairs Department, Genethon

Dmitry Kuzmin, Managing Partner, 4Bio Capital

Dominic Schmidt, Partner, Syncona

Dominic O'Regan, Director & Investor, New Mosaic Ltd

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

Edward Hodgkin, Partner, Syncona Partners

Edward van Wezel, Managing Partner, Biogeneration Ventures


Elena Beltrami, Business Development Manager, Telethon

Elena Costariol, EngD Researcher, UCL

Elena Piletska, Professor of Bioanalytical Chemistry, University of Leicester

Elizabeth Schwarzbach, Chief Business Officer, The New York Stem Cell Foundation

Elsa Abranches, Director, UK Stem Cell Bank, NIBSC-MHRA

Elzbieta Maria Piatkowska, CEO, Remissio

Emanuele Ostuni, Head of CAR-T Europe, Novartis

Emmett Schmidt, Associate Vice President, MSD

Eric Alton, Professor, Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London; Chair, NIHR ATMP Coordination Group

Eric Michael David, CEO, BridgeBio Gene Therapy

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

Erik Richardson, Senior Manager, Gene Therapy R&D, Apellis Pharmaceuticals

Evelina Vågesjö, CEO, Ilya Pharma

Faruque Ghanchi, Consultant Ophthalmologist, Head, Bradford Ophthalmology Research Network (BORN), Specialty Lead- Ophthalmology, Bradford Teaching Hospitals

NHS Foundation Trust

Fatima Ann Sulaiman, Head of Research and Services, Scleroderma and Raynaud’s UK (SRUK)

Federico Mingozzi, CSO, Spark Therapeutics

Florence Allouche, President – CEO, SparingVision; Associate Professor, Faculty of Pharmacy, University of Paris

Francesca Bellintani, Downstream Process Development Manager, MolMed

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

Frank Luyten, Director of the Skeletal Biology and Engineering Research Center & Prometheus, K.U.Leuven, BE; Medical/Scientific Director, REGMEDXB

Fred Chereau, CEO, LogicBio Therapeutics

Friedemann Loos, Innovation Manager, BioRN

Gabriel Brooks, Head of Clinical R&D, 4DMT

Giuseppe Mazza, Co-Founder and CEO, Engitix Ltd

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

Hans Keirstead, Chief Executive Officer, AIVITA Biomedical

Hansjörg Hauser, Head of Department Gene Regulation and Differentiation, Helmholtz Centre for Infection Research

Hans-Juergen Fuelle, VP, Head Global Regulatory Strategy, AveXis

Hilary Hehman, Director, Partnerships and Alliances, Fred Hutch Cancer Research Center

Igor Rudychev, Head of Digital, Data, and Innovations, US Oncology, AstraZeneca

Ioannis Papantoniou, Principal Investigator, FORTH (Foundation Of Research and Technology); Adjunct Professor, KU Leuven

Jaime Shamonki, Chief Medical Officer, Generate Life Sciences

Jan Schrooten, CEO, Antleron, Board Member, flanders.bio

Jane Lebkowski, President, Regenerative Patch Technologies

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

Jarema (Jarko) Kochan, Head of Biomarkers, Diagnostics and Assay Development, Turnstone Biologics

Jean-Philippe Combal, Co-Founder & CEO, Vivet Therapeutics

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

Jim Richardson, Senior Science and Standards Liaison, United States Pharmacopeia

Joachim G Aerts, Head of the Department, Professor of Pulmonary Medicine, Pulmonary Diseases, Erasmus University Medical Center Rotterdam

Joanna Miller, Science Director, Cell Therapy Sciences

Joanne Kurtzberg, Professor of Pediatrics and Pathology; Director, Marcus Center for Cellular Cures, Duke University Medical Center

Joe Dupere, CEO, Rexgenero

Joery De Kock, Professor, Vrije Universiteit Brussel; Scientific Advisory Board Member, RheinCell Therapeutics


Johannes C. M. van der Loo, Director, Clinical Vector Core, The Children’s Hospital of Philadelphia

Johannes P.J.M. de Munter, CEO, Neuroplast

John Johnston, Clinical Assessor, Biologicals Unit, MHRA

John Maher, Consultant and Senior Lecturer in Immunology, King's College London, CSO, Leucid

John Sinden, CEO, Glialign

John Wagner, Professor; Director, Institute for Cell, Gene and Immunotherapies, Department of Pediatrics, Blood and Marrow Transplantation, University of Minnesota

Johnny Huard, Director and Chief Scientific Officer, Center for Regenerative Sports Medicine, Steadman Philippon Research Institute (SPRI)

Jonathan Pollett, Chief Research Officer, Imagine Pharma

Juergen Weisser, Managing Director, RheinCell Therapeutics

Julian Gough, Chief Scientific Officer, Mogrify

Jürgen Kuball, Center of Translational Immunology (CTI), Dept of Hematology, UMC Utrecht

Kate Strayer-Benton, Senior Director of Business Development, Belfer Office For Dana-Farber Innovations, Dana-Farber Cancer Institute

Katja Kleinsteuber, Scientist II, Cellular Analytics, Bluebird Bio

Kristian Elverum, SVP, Business Development & Strategy, Rubius Therapeutics

Lawrence S. Lamb, Executive Vice President/ Chief Scientific Officer, Incysus

Lawrence Thompson, Senior Principal Scientist, Analytical Research & Development, BioTherapeutics Pharm Sciences, Pfizer

Leo Groenewegen, Business Development Manager, cellulREvolution

Leonard Pattenden, Head of Biotherapeutics Development & Drug Supply, Biotherapeutics Development Unit, Cancer Research UK

Lior Raviv, Vice President Development & Operations, Pluristem therapeutics

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

Luc Boileau, President and Chief Executive Officer, National Institute for Clinical Excellence in Health and Social Services (INESSS)

Magdi Elsallab, Doctoral Student, BIH Center For Regenerative Therapies Charite

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

Marco Fadda, ATMP Solutions Manager, COMECER

Margareth Jorvid, Head of Regulatory Affairs and QA, Immunicum

Margarida Serra, Head of Stem Cell Bioengineering Laboratory, Animal Cell Technology Unit, iBET

Maria Mirotsou, Senior Director, Astellas Institute of Regenerative Medicine

Maria Papathanasiou, Research Associate Centre for Process Systems Engineering (CPSE) Dept. of Chemical Engineering, Imperial College London

Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL; CSO, INmuneBio

Mark Sawicki, Chief Commercial Officer, Cryoport

Martin Pule, SVP, Founder & CSO, Autolus; Clinical Senior Lecturer, Haematology, UCL Cancer Institute

Mary Murphy, Professor in Regenerative Medicine, REMEDI

Masahiro Kino-Oka, Professor, Department of Biotechnology, Graduate School of Engineering, Osaka University

Mathias Svahn, CEO, NextCell Pharma

Maximillian Krippl, Data Scientist, Novasign; Research Assistant, BOKU

Michael Hunt, CEO, Reneuron

Michael West, Founder and CEO, AgeX Therapeutics

Michela Gabaldo, Head of Alliance Management, Fondazione Telethon

Michèle de Guise, Director of Health Services and Technology Evaluation, National Institute for Clinical Excellence in Health and Social Services (INESSS)

Miriam Haak, Director Business Development, Miltenyi Biotec

Moshe Israeli, Medical Director, Rabin Medical Center

Nada Alaaeddine, Managing Partner, Cormedicine Clinic of Regenerative Medicine; Associate Professor & Head of Research cCommittee, Faculty of Medicine, The

Lebanese University

Neil McGowan, Head of Manufacturing, Tissues, Cells and Advanced Therapeutics, Scottish National Blood Transfusion Service

Neill Moray Mackenzie, Chairman, Immetacyte

Nina Bauer, Commercial Head of Cell Therapy, MilliporeSigma Merck KGaA

Nneka Onwudiwe, Founder and Chief Executive Officer, Pharmaceutical Economics Consultants of America (PECA) LLC


Owen Smith, Investment Director, 4BIO Capital

Pamela Tranter, Head, Translational Research Group, UCL

Pascal Touchon, President, Chief Executive Officer & Board Member, Atara Biotherapeutics

Patrick J. Hanley, Director, Cellular Therapy Laboratory, Children's National Health System

Paul Gissen, LMCB Group Leader, UCL Professorial Research Associate, UCL

Paul S Carter, Scientific Leader – Vector Downstream Process Development, Cell & Gene Therapy Platform CMC, Medicinal Science and Technology, R&D, GSK

Peter Hamley, CSO, Samsara Therapeutics

Petra Reinke, Director, Berlin Center for Advanced Therapies (BeCAT), BIH Center for Regenerative Therapies (BCRT)

Philip J. Brooks, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health

Pierre Albert Colcomb, Director Business Development, Genethon

Pinar Akcakaya, Senior Research Scientist, AstraZeneca

Prasad Adusumilli, Deputy Chief And Associate Attending, Thoracic Surgery Director, Mesothelioma Program, Memorial Sloan-Kettering Cancer Center

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

RA Session, Chief Business Officer, Gene Therapy, BridgeBio

Raghuram Selvaraju, Managing Director, H.C. wainwright

Randolph Corteling, Head of Research, Reneuron

Ravindra Gupta, Professor of Clinical Microbiology, Department of Medicine, University of Cambridge

Ricardo Baptista, Director Process & Analytical Development, Cellectis

Robert Deans, Chief Innovation Officer, BlueRock Therapeutics

Robert Jones, Vice President, Global Bioservices, Cryoport

Roland Pochet, Professor, Université Libre de Bruxelles; Secretary General, Belgian Brain Council; Patient Representative, CAT EMA

Roy Baynes, SVP and Head Global Clinical Development, Chief Medical Officer, Merck, Sharp & Dohme

Russell Cruz, Assistant Professor, Children's National Medical Center; Co-Founder, Mana Therapeutics

Sakis Mantalaris, Professor, BioMedical Systems Engineering Laboratory, Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology

Sam Hall, Partner, Apple Tree Partners

Sara Mole, LMCB Group Leader, Professor of Molecular Cell Biology, UCL

Sean Palecek, Director for Research, National Science Foundation Center for Cell Manufacturing Technologies (CMaT)

Sebastian Kress, Postdoctoral Scientist, Institute of Cell and Tissue Culture Technologies, BOKU

Seokjoong Kim, Executive Director of R&D and Strategic Alliances, Toolgen

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

Shane Kovacs, Chief Business and Financial Officer, BlueRock Therapeutics

Simon Waddington, Professor of Gene Therapy, University College London

Stephanie Schorge, Professor of Translational Neuroscience, Wellcome Chair in Pharmacology, UCL School of Pharmacy

Stefano Baila, CEO, Anemocyte

Stefano Zancan, Head of Clinical Development and Operations, Fondazione Telethon

Stefanos Theoharis, Previously SVP Corporate Development and Partnering, Cell Medica, Independent Consultant

Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center for Pre-Clinical Studies, Deputy Director, McGowan Institute for Regenerative Medicine

(MIRM)

Steve Kanner, CSO, Caribou Biosciences

Steve Oh, Director Stem Cell Bioprocessing & Institute Professor, Stem Cell Group, Bioprocessing Technology Institute, A*Star

Steven Pincus, Head of Science and Innovation, FUJIFILM Diosynth Biotechnologies Texas

Steven M. Altschuler, Member of the Board, Spark Therapeutics; Managing Director, Healthcare Ventures, ZIFF Capital Partners

Steven P. Lee, Senior Research Fellow, Institute of Immunology and Immunotherapy, University of Birmingham

Stewart Abbot, Chief Operating Officer, Adicet Bio

Stuart Collinson, CEO, Oxstem

Suzanne S. Farid, Professor of Bioprocess Systems Engineering & Co-Director Future Targeted Healthcare Manufacturing Hub, UCL

Sven Kili, Principal, Sven Kili Consulting; CMO, Xintela

Tanner Dockendorf, Senior Manager, Client Relations, Aldevron


Tara Britt, Associate Chair, North Carolina Rare Disease Network

Ted Tuddenham, Emeritus Professor of Haemophilia, UCL

Tim Davies, Director Business Development, A4PBio

Tim Luker, Senior Director External Innovation, Eli Lilly

Todd McDevitt, Senior Investigator, Gladstone Institutes; Professor, Bioengineering & Therapeutic Sciences, UCSF

Troels Jordansen, CEO, Glycostem Therapeutics

Uta Griesenbach, Professor, Molecular Medicine, National Heart and Lung Institute, Imperial College London; President, British Society for Gene and Cell Therapy;

Director (non-exec), Cell and Gene Therapy Catapult

Victoria English, Co-founder & Editor, Evernow Publishing Ltd, publishers of MedNous

Vijay Curthan, Investment Director, Catapult Ventures

Vittorio Colizzi, Professor of Immunology, University of Rome Tor Vergata

TBC Antonio Regalado, Senior Editor for Biomedicine, MIT Technology Review

TBC Cedrik Britten, VP & Head, Oncology Cell Therapies Unit, GSK

TBC Doug Danison, Vice President, (market) Access, Value & Evidence Strategy (AVES), bluebird bio

TBC Gocha Shatirishvili, Medical Director, Geocord, Farmax Ltd

TBC Giorgio Iotti, Head of Program Leadership and Control, Rare Disease Unit, Chiesi Farmaceutici SpA

TBC Bradley Campbell, President and COO, Amicus

TBC Joshua Anderson, Senior Manager, Clinical Supply Operations, Adaptimmune

TBC Krishna Komanduri, Director, Adult Stem Cell Transplant Program, Sylvester Cancer Center

TBC Miguel Forte, CEO, Bone Therapeutics

TBC Nikolai Kunicher, CEO, Betalin Therapeutics

TBC Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics

TBC Stefan Gluck, Previously VP, GMA Early Assets, Celgene

TBC Jak Knowles, VP Venture Investments, Human, Bayer

Representative from AMSBIO

Representative from Advatis

Representative from Lonza

Representative from Anjarium

Representative from Atelerix

Representative from Cenya

Representative from DenovoMATRIX

Representative from EverZom

Representative from EvoBioTech

Representative from HAYA Therapeutics

Representative from NETRI

Representative from REM Analytics

Representative from Wren Healthcare

Representative from Pall

(242/300)

2020 Sponsored content: Cryoport: 20-minute keynote session

20-minute presentation Patient Delivery track

Participation in a panel discussion

FUJIFILM Irvine Scientific – 20-minute Cell Therapy Manufacturing track

CellGenix: 45-minute roundtable discussion

COMECER – 20-minute Cell Therapy Manufacturing track

Miltenyi biotec – 20-minute Cell Therapy Manufacturing track

Lonza – 20-minute Cell Therapy Manufacturing track


MolMed – 20-minute presentation Viral Vector Manufacturing track

Aldevron – 20-minute presentation Gene Therapy track

VivaBioCell – 20-minute presentation Cell Therapy Manufacturing track

Fujifilm Diosynth Biotechnologies – 20-minute presentation Gene Therapy track

Pall – 20-minute presentation (track TBC)

Schott: 45-minute roundtable discussion

Advatis: 10 min presentation in the Innovation track

Caladrius: 10 min presentation in the Pitch and Partner track

Millipore Sigma, Merck KGaA: 10 min presentation in the Innovation track

AMSBIO: 10 min presentation in the Innovation track

Confirmed start-up companies:

• A4P Consulting Ltd (Logistics) • Alia Therapeutics • Anjarium Biosciences • Atelerix • BioMedha • biotx.ai • bit bio • BiVictriX Therapeutics • Cedios • Cell-Easy • cellulaREvolution • CENSO Biotechnologies • Cenya Imaging • COBO Technologies • CODA Biotherapeutics • denovoMATRIX • Etcembly • EverZom • EvoBioTech SRL • GenEvolutioN • GoodCell • HAYA Therapeutics • Ilya Pharma • InnoVitro • Javelin • Kidney Cure • LightOx • Manchester BIOGEL • Mogrify • Netri • REM Analytics • Remissio • RheinCell Therapeutics GmbH • SEED Biosciences • Strand Therapeutics • Tarian Biotechnology • Ultroxa • Virokine Therapeutics • Wren Healthcare


Advisory Board (2020): Alain Vertès, Managing Director, NxR Biotechnologies

Annie Hubert, Senior Director, European Public Policy, Alliance for Regenerative Medicine

David Morrow, Senior Scientific Programme Manager-ATMP/Vaccines, EATRIS

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

Ioannis Papantoniou, Principal Investigator, FORTH (Foundation Of Research and Technology); Adjunct Professor, KU Leuven

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

Miguel Forte, CEO, Bone Therapeutics

Norio Nakatsuji, Professor Emeritus, Kyoto University & Chief Advisor, Stem Cell & Device Laboratory, Inc

Ohad Karnieli, CEO, ATVIO

Robert Beckman, Managing Partner, The Channel Group; Chairman of the Board, QOSINA

Steven Pincus, Associate Vice President Virology and Analytical Methods Development, FUJIFILM Diosynth Biotechnologies Texas LLC

Sven Kili, Principal, Sven Kili Consulting

Bo Yan, Senior Scientist, Analytical Development, Beam Therapeutics

Supporting partners: EATRIS

Alliance for Regenerative Medicine (ARM)

BioRN

The Japanese Society for Regenerative Medicine

STARBIOS2

TBC UK Department of International Trade

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PRE-CONFERENCE WORKSHOP AND SUMMIT DAY PRE-CONFERENCE WORKSHOP DAY - Monday 30th March 2020

PRE-CONFERENCE WORKSHOP DAY

12:00 Networking Lunch

BIOPROCESS STANDARDS DEVELOPMENT RARE DISEASE CORD BLOOD

Towards CellTherapy4.0 self-adaptive and self-managed manufacturing pipelines

Host: Ioannis Papantoniou, Principal Investigator, FORTH (Foundation of Research and Technology); Adjunct Professor, KU Leuven (CONFIRMED)

Chair: Sven Kili, Principal, Sven Kili Consulting (CONFIRMED)

Chair: Moshe Israeli, Medical Director, Rabin Medical Center (CONFIRMED)

13:00 Introduction Ioannis Papantoniou, Principal Investigator, FORTH (Foundation of Research and Technology); Adjunct Professor, KU Leuven (CONFIRMED)

Standards for Advanced Therapies Dawn Henke, Senior Technical Program Manager, Standards Coordinating Body (CONFIRMED)

Increasing efficiency in rare genetic disease therapeutics development: can alternate approaches from cancer research be adopted Philip J. Brooks, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (CONFIRMED)

Keynote: Novel cellular therapies derived from cord blood monocytes Joanne Kurtzberg, Professor of Pediatrics and Pathology; Director, Marcus Center for Cellular Cures, Duke University Medical Center (CONFIRMED)

13:25 Christoph Herwig, Professor of Biochemical Engineering, Vienna University of Technology (CONFIRMED)

Elsa Abranches, Director, UK Stem Cell Bank, NIBSC-MHRA (CONFIRMED)

The meaning of access to therapy for a patient with a rare disease, and how a clinical site should take into consideration this aspect Stefano Zancan, Head of Clinical Development and Operations, Fondazione Telethon (CONFIRMED)

CB expansion breakthrough Colleen Delany, Founder & CSO, Nohla Therapeutics (CONFIRMED)

13:50 Turn cells into therapies by end-to-end innovation strategies Jan Schrooten, CEO, Antleron, Board Member, flanders.bio (CONFIRMED)

USP Standards for Cell and Gene Therapies Jim Richardson, Senior Science and Standards Liaison, United States

Pharmacopeia (CONFIRMED)

The role of patient organisations in precision medicine development Fatima Ann Sulaiman, Head of Research and Services, Scleroderma and Raynaud’s UK (SRUK) (CONFIRMED)

Cord blood as source marked for novel cellular therapeutics John Wagner, Professor; Director, Institute for Cell, Gene and Immunotherapies, Department of Pediatrics, Blood and Marrow Transplantation, University of Minnesota (CONFIRMED)

14:15 Learning from immunotherapy successes with solid tumours Andrew Sewell, Distinguished Research Professor and Wellcome Trust Senior Investigator, Division of Infection and Immunity, Cardiff University School of Medicine (CONFIRMED)

Policy legislation and global reach for rare diseases Tara Britt, Associate Chair, North Carolina Rare Disease Network (CONFIRMED)

Diverse use of cord blood Marcie R. Finney, Executive Director, Cleveland Cord Blood Center (CONFIRMED)

14:45 Afternoon networking break

BIOPROCESS GENE EDITING RARE DISEASE CORD BLOOD

Manufacture & Bioprocess of Gene Modified Cell Therapies Host: Qasim Rafiq, Senior Lecturer (Associate Professor), Bioprocessing of Regenerative, Cellular and Gene Therapies, Department of Biochemical Engineering, UCL (CONFIRMED)

Chair: Hansjörg Hauser, Head of Department Gene Regulation and Differentiation, Helmholtz Centre for Infection Research (CONFIRMED)

Scientific advice and early dialogue to achieve clinical success and data collection later on

Hosted by EUNetHTA

Innovative Utilization of CB for new therapies

15:15 Suzanne S. Farid, Professor of Bioprocess Systems Engineering & Co-Director Future Targeted Healthcare Manufacturing Hub, UCL (CONFIRMED)

Tackling the challenge of cell quality in large scale genome wide CRISPR screening for drug discovery Todd McDevitt, Senior Investigator, Gladstone Institutes; Professor, Bioengineering & Therapeutic Sciences, UCSF (CONFIRMED)

Panel discussion: Creating a new drug development framework for translating research into drug development: Philip J. Brooks, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (CONFIRMED) Chandler Crews, President, The Chandler Project (CONFIRMED)

CartiCure – An ATMP based on mesenchymal stromal cells for orthopaedic treatments and regenerative medicine André Gerth, CEO, BioPlanta (CONFIRMED)

15.40 Big Pharma: GSK, AstraZeneca/Medimmune, Pfizer (TBC) CRISPR-mediated genome engineering of CHO cell lines for production of therapeutic proteins Lise Marie Grav, Postdoc, CHO Cell Line Engineering and Design, DTU Biosustain (CONFIRMED)

Clinical trials with mesenchymal stem cells from cord tissue, used for treatment of diabetes type 1 Mathias Svahn, CEO, NextCell Pharma (CONFIRMED)

16:05 Biotech: Autolus, Freeline, Oxford Biomedica (TBC) TBC Precise genome engineering to empower drug discovery Barry Rosen, Senior Principal Scientist/Director, AstraZenca (CONFIRMED)

Beyond biostorage: How cord blood banks can stay relevant to customers Jaime Shamonki, Chief Medical Officer, Generate Life Sciences (CONFIRMED)

16:30 More technical, Process Development Scientists Gocha Shatirishvili, Medical Director, Geocord, Farmax Ltd (TBC)

16.50 Panel discussion: Clusters and consortia being developed, office for life sciences (government), HMT (Treasury)

17:10 Nic Pineault (invited) Lazaros Fotopoulos (invited) Daniel Gibson (invited)

17:30 Close of Conference for the day - Opening Networking drinks reception at the Business Design Center


CONFERENCE DAY 1 - TUESDAY 31st March 2020 CONFERENCE DAY 1 - TUESDAY 31st March 2020

Opening Keynotes Day 1 – CAR-T & Immunotherapy focus

08.45 Welcome – Jessica Robinson, Project Director, Advanced Therapies 2020, Terrapinn

08.48 Chair’s introduction: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

09:00

Keynote presentation: Autologous Mesothelin-targeting CAR-T to tackle solid tumours in the clinic

• The presentation will highlight results to date of mesothelin-targeted CAR T-cell clinical trial results

• Advances in preclinical research of CAR T-cell therapy for solid tumors will be presented

• Novel strategies for overcoming hurdles to solid tumor CAR T-cell therapy will be discussed

Prasad Adusumilli, Deputy Chief and Attending, Thoracic Surgery; Vice Chair, Translational Research, Dept. of Surgery, Head, Solid Tumors Cell Therapy, Cellular Therapeutics Center (CTC); Director, Mesothelioma Program, Memorial Sloan-Kettering Cancer Center (CONFIRMED)

09.20 Realizing the promise of ATMPs for patients around the world

• Working with regulators to navigate scale up challenges in manufacturing

• Strategies in supply chain and patient delivery

• Tackling post manufacturing commercialization challenges such as pricing and finding highly skilled experts

• Durability Emanuele Ostuni, Head of CAR-T Europe, Novartis (CONFIRMED) Pascal Touchon, President, Chief Executive Officer & Board Member, Atara Biotherapeutics (CONFIRMED) Christopher R. Heery, CMO, Precision BioSciences (CONFIRMED) Anthony Lodge, CMC – Regulatory Lead, Kite Pharma (CONFIRMED) Jeff Walsh, Chief Strategy Officer, bluebird bio (invited)

10:00 Fireside chat: Changing our approach to rare diseases Bradley Campbell, President and COO, Amicus (TBC)

10:20 Morning coffee break

11.20 Keynote presentation: CAR-T Martin Pule, SVP, Founder & CSO, Autolus; Clinical Senior Lecturer, Haematology, UCL Cancer Institute (CONFIRMED)

11:40 Effective Integration Strategies Through a Compliance Unified Ecosystem (CUE) • Systems Integration continues to be a major issue in the production and distribution of Advanced Therapies • Well-defined strategies for the integration of independent informatics platforms will be critical for future standardization and workflow optimization • Cryoport’s Compliance Unified Ecosystem (CUE) is driving standard practices for systems integration in the industry Mark Sawicki, Chief Commercial Officer, Cryoport (CONFIRMED)

12:00 Technology and Strategy Roundtables will run from 12:00-12:45 with 15 minutes for a quick summary from each table at the front of the room. These will take place in the conference rooms: look for your table number to participate.

MANUFACTURING STEM CELLS & REGENERATIVE MEDICINE COMMERCIALISATION STRATEGIES CLINICAL & THERAPEUTICS FOCUS AREAS

1. Raw material supply for the commercial manufacturing of cell and gene therapies Frank Hecht, Vice President Marketing & Sales, CellGenix (CONFIRMED)

2. Physiological conditions and analytical and monitoring tools for 3 D cell cultivations Cornelia Kasper, Full University Professor, BOKU (CONFIRMED)

3. Sebastian Kress, Postdoctoral Scientist, Institute of Cell and Tissue Culture Technologies, BOKU (CONFIRMED) Reserved for Schott

4. Automation and continuous manufacture Lior Raviv, Vice President Development & Operations, Pluristem therapeutics (CONFIRMED)

5. Considerations for manufacture scale-up from academia to commercial manufacturing Johannes C. M. van der Loo, Director, Clinical Vector Core, The Children’s Hospital of Philadelphia (CONFIRMED)

6. Defining standards for gene therapy Jim Richardson, Senior Science and Standards Liaison, United States Pharmacopeia (CONFIRMED)

7. Mechanisms of MSC action and potency assays Mary Murphy, Professor in Regenerative Medicine, REMEDI (CONFIRMED)

8. MSCs Alain Vertès, Managing Director, NxR Biotechnologies GmbH (CONFIRMED)

9. Exploring the crossover between human and veterinary applications for stem cells Joanna Miller, Science Director, Cell Therapy Sciences UK (CONFIRMED)

10. Regenerative Medicine Petra Reinke, Director, Berlin Center for Advanced Therapies (BeCAT), BIH Center for Regenerative Therapies (BCRT) (CONFIRMED)

11. Cord blood as a source of new cells John Wagner, Executive Medical Director, BMT Program, University of Minnesota Masonic Children’s Hospital, Professor, Department of Pediatric, University of Minnesota (CONFIRMED)

12. Tissue engineering for organ regeneration

14. Making allogeneic cell therapy a reality Devyn Smith, Chief Operating Officer & Head of Strategy, Sigilon, Inc (CONFIRMED)

15. Industry-no profit alliances: how to optimize synergies Elena Beltrami, Business Development Manager, Telethon (CONFIRMED)

16. TBC: Industrialisation Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL; CSO, INmuneBio (CONFIRMED)

17. 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)

18. Ensuring ATMP access to patients Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) (CONFIRMED)

19. CAR-T innovation journey Emanuele Ostuni, Head of CAR-T Europe, Novartis (CONFIRMED)

20. Tackling solid tumours in the clinic Prasad Adusumilli, Attending Surgeon, Memorial Sloan Kettering Cancer Center (CONFIRMED)

21. Neurological applications for ATMPs Please contact Daniel Overd (+44 (0)207 092 1087, [email protected] for further details)

22. Development of regenerative medicines for cardiovascular indications David Mazzo, President & CEO, Caladrius Biosciences (CONFIRMED)

23. Renal applications for ATMPs Please contact Daniel Overd (+44 (0)207 092 1087, [email protected] for further details)

24. Tackling diabetes with ATMPs Please contact Daniel Overd (+44 (0)207 092 1087, [email protected] for further details)

mailto:[email protected]




Dawn Henke, Senior Technical Program Manager, Standards Coordinating Body (CONFIRMED)

13. Longevity/new approaches to diseases of Ageing Peter Hamley, CSO, Samsara Therapeutics (CONFIRMED)

13.00 Lunch

EVIDENCE, PRICING &

MARKET ACCESS

GENE MODIFIED CELL THERAPY

GENE THERAPY IMMUNO-THERAPY

RESEARCH & DEVELOPMENT

STEM CELLS & REGEN MED

CELL THERAPY MANUFACTURE

VIRAL VECTOR MANUFACTURE

PATIENT DELIVERY GENE EDITING PITCH AND PARTNER

INNOVATION

14:20 Chair: Alexander Natz, Secretary

General, European Confederation of Pharmaceutical Entrepreneurs

(EUCOPE) (CONFIRMED)

Solid Tumours

Chair: Miguel Forte, CEO, Bone

Therapeutics (CONFIRMED)

New techniques and technologies

Precision medicine for

immunotherapy Raghuram Selvaraju, Managing

Director, H.C. Wainwright

(CONFIRMED)

New clinical data: updates from

across the ATMP space

From clinic to commercialisation

Chair: Stephen Badylak, Professor,

Department of Surgery, Director,

McGowan Center for Pre-Clinical Studies,

Deputy Director, McGowan

Institute for Regenerative

Medicine (MIRM) (CONFIRMED)

Manufacturing strategies

Process development Chair: Manuel

Carrondo, Professor of Chemical & Biochemical

Engineering, FCT-UNL, Vice-President, IBET (CONFIRMED)

Chair: Kris Elverum, SVP, Business

Development & Strategy, Rubius


Latest global developments

Investor Forum & company pitches Chair: Vivienne

Parry, Writer and Broadcaster, Head

of Engagement, Genomics England

(CONFIRMED)

Special Session: RNA Targeted

Therapeutics Chair: David Pearce,

President of Innovation and

Research, Sanford Health

14:20 TBC Dave Lennon, President, AveXis (CONFIRMED)

Exploiting Natural Receptors to broaden the horizon of CAR-T David Gilham, VP Research & Development, Celyad (CONFIRMED)

Haematopoietic stem cell gene therapy for severe monogenic disorders Bobby Gaspar, CSO, Orchard Therapeutics (CONFIRMED)

PD-1 antibodies are transforming cancer therapy both as mono- and in combination Roy Baynes, SVP and Head Global Clinical Development, Chief Medical Officer, Merck, Sharp & Dohme (CONFIRMED)

Where are we with HIV cure using gene editing? Ravindra Gupta, Professor of Clinical Microbiology, Department of Medicine, University of Cambridge (CONFIRMED)

Stem Cells for neurological applications, Clinical and stem cells-based product development Chaim Lebovits, CEO, BrainStorm Cell Therapeutics (CONFIRMED)

Optimizing Antigen-Specific T Cell Manufacturing Patrick J. Hanley,

Director, Cellular

Therapy

Laboratory,

Children's National

Health System

(CONFIRMED)

Developing robust manufacturing processes for novel gene therapies

Introduction: set the context up from the start on the breadth and complexity of the system Kris Elverum, SVP, Business Development & Strategy, Rubius Therapeutics (CONFIRMED)

Directed evolution of novel aden-associated viral vectors for clinical gene therapy David Schaffer, Professor, Chemical and Biomolecular Engineering, University of California, Berkeley, and Co-Founder, 4D Molecular Therapeutics (CONFIRMED)

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 Investor panellists: Owen Smith, Investment Director, 4BIO Capital (CONFIRMED) Dominic Schmidt, Partner, Syncona (CONFIRMED) Edward van Wezel, Managing Partner, Biogeneration Ventures (CONFIRMED) Daniel O’Mahony, Partner, Seroba Life Sciences (CONFIRMED) Alan J Muir, Director Life Science & Healthcare Investment, Seven Hills Venture Partners (CONFIRMED)

Translation Control Therapeutics: discovery of small molecule drugs that control mRNA translation into proteins Yochi Slonim, Co-founder & CEO, Anima Biotech (CONFIRMED)

14:40 Addressing unique commercial challenges with gene therapy: learnings from Strimvelis Darren Walsh, Country Manager UK / ROI, (Senior Director – Market Access and Govt. Affairs EMEA), Orchard Therapeutics (CONFIRMED)

Using CAR T-cells to target the tumour vasculature Steven P. Lee, Senior Research Fellow, Institute of Immunology and Immunotherapy, University of Birmingham (CONFIRMED)

Tanner Dockendorf, Senior Manager, Client Relations, Aldevron (CONFIRMED)

From big patient-level data to actionable insights and answering right business questions in oncology Igor Rudychev, Head of Digital, Data, and Innovations, US Oncology, AstraZeneca (CONFIRMED)

New clinical data: Effects of an autologous cell therapy in patients with advanced chronic kidney disease associated with diabetes mellitus Deepak Jain, COO, InRegen (CONFIRMED)

Industrialized personalized regenerative medicine, promising but very challenging Johannes P.J.M. de Munter, CEO, Neuroplast (CONFIRMED)

T cells/CART and electroporation process using FISI CDM media Stefano Baila, CEO, Anemocyte (CONFIRMED)

Large Scale Production of LV and RV Vectors for T- and CD34+ Cells Transduction Francesca Bellintani, Downstream Process Development Manager, MolMed (CONFIRMED)

A decentralized approach to advanced therapy supply chain Robert Jones, Vice President, Global Bioservices, Cryoport (CONFIRMED)

NIH Common Fund program on genome editing Philip J. Brooks, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (CONFIRMED)

Tim Luker, Senior Director External Innovation, Eli Lilly (CONFIRMED)

15.00 Recent developments on Gene Therapy in Germany Axel Böhnke, Director Market Access EU North,

Taking Tumour infiltrating T cells into pivotal clinical studies Neill Moray Mackenzie,

Novel approaches to building gene therapy programs: Lessons from BridgeBio Eric Michael David, CEO, BridgeBio

1st-in-Human CAR T Targets MUC1 Cleavage Product Expressed on Solid Tumors Cynthia Bamdad,

Clinical stage allogenic dendritic cell-based immunotherapy targeting mesothelioma and pancreatic cancer

Ground-breaking Innovation in Regenerative Medicine; The Development of the World’s First

Potency assay development: lifecycle approach for cell and gene therapy products Amandine Breton, Cell Therapy

Continuous downstream processing, from gene therapy viruses to exosomes Manuel Carrondo, Professor of

Case study: IC-T immunotherapy programme, implementing tech transfer and laboratory prep

The promise of advanced therapies for 21st Century Medicines Hans-Juergen Fuelle, VP, Head Global Regulatory

Multi-perspective discussion: How to partner with public/private organizations Edward Hodgkin, Partner, Syncona

Taking a novel next-generation biologic from idea to clinical proof of concept Evelina Vågesjö, CEO, Co-founder, Ilya


PTC Therapeutics (CONFIRMED)

Chairman, Immetacyte (CONFIRMED)

Gene Therapy (CONFIRMED)

CEO, Minerva Biotechnologies (CONFIRMED)

Joachim G Aerts, Head of the Department, Professor of Pulmonary Medicine, Pulmonary Diseases, Erasmus University Medical Center Rotterdam (CONFIRMED)

Bioengineered Blood Vessel Bill Tente, Chief Regulatory Officer, Humacyte (CONFIRMED)

Operations Manager, Orchard Therapeutics (CONFIRMED)

Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET (CONFIRMED)

Albert Ribickas, Bone Marrow Transplant Manager, Moffitt Cancer Center (CONFIRMED)

Strategy, AveXis (CONFIRMED)

Partners (CONFIRMED) Shane Kovacs, Chief Business and Financial Officer, BlueRock Therapeutics (CONFIRMED) Dave Greenwald, Vice President, Business Development, Deerfield Management (CONFIRMED) Steven M. Altschuler, Managing Director, Healthcare Ventures, ZIFF Capital Partners (CONFIRMED) Katherine Cohen, Venture Partner, Panacea Venture (invited) John Craighead, VP Investor Relations, Atara Biotherapeutics (invited) Sander van Deventer, EVP, Research & Product Development, UniQure (invited) Jak Knowles, VP Venture Investments Human, Bayer (TBC)

Pharma (CONFIRMED)

15.20 Pilar Pinilla-Dominguez, Senior Scientific Adviser, NICE (invited)

TCR-Engineered T Cells for the Treatment of Solid Cancer: Maximising Scale and Benefit for NY-ESO TCR-T Cedrik Britten, VP & Head, Oncology Cell Therapies Unit, GSK (CONFIRMED)

Gene therapy for epilepsy: Clinical Trials in 2021? Stephanie Schorge, Professor of Translational Neuroscience, Wellcome Chair in Pharmacology, UCL School of Pharmacy (CONFIRMED)

Engineered tumor micro-environment using human extracellular matrix for modelling hot and cold tumours Giuseppe Mazza, Co-Founder and CEO, Engitix Ltd, Royal Free Hospital (CONFIRMED)

Retinal gene therapy Professor Faruque Ghanchi, Consultant Ophthalmologist, Head, Bradford Ophthalmology Research Network (BORN), Specialty Lead- Ophthalmology, Bradford Teaching Hospitals NHS Foundation Trust (CONFIRMED)

The use of cell therapies in regenerative medicine Joe Dupere, CEO, Rexgenero (CONFIRMED)

Fully closed vs isolator approach in C&GT: how can they collaborate and complement for a successful and sustainable future Marco Fadda, ATMP Solutions Manager, COMECER (CONFIRMED)

A look at Fujifilm Diosynth development of an advanced gene therapy platform Steven Pincus, Head of Science and Innovation, FUJIFILM Diosynth Biotechnologies Texas (CONFIRMED)

Supply chain management, logistics and scale out for T-Cell products Alan K. Smith, Executive Vice President, Technical Operations, Bellicum Pharmaceuticals (CONFIRMED)

CRISPR off-targets: how to detect and avoid them Pinar Akcakaya, Senior Research Scientist, AstraZeneca (CONFIRMED)

Plant Micro-vesicles containing smallRNAs in delivering and adjuvancy for sustainably cancer therapy Vittorio Colizzi, Professor of Immunology, University of Rome Tor Vergata (CONFIRMED)

15:40 What incentives are available for developing orphan drugs? Is the current orphan drug legislation & incentives enough? Ulrika Vestin, Expert, Swedish Association of Local Authorities and Regions (invited)

Reaching beyond the limits of nature to cure inherited metabolic disorders of the liver Joery De Kock, Professor, Vrije Universiteit Brussel; Scientific Advisory Board Member, RheinCell Therapeutics (CONFIRMED)

Clinical application of scaled human stem cell derivates in immune-oncology Hans Keirstead, Chief Executive Officer, AIVITA Biomedical (CONFIRMED)

Gene Therapy for neurological disorders Mimoun Azzouz, Chair of Translational Neuroscience, University of Sheffield; PI, Chronic Neurological Disorders, NIHR Sheffield BRC (CONFIRMED)

TBC: Using regenerative medicine to stop aging Michael West, Founder and CEO, AgeX Therapeutics (CONFIRMED)

Lessons learned from potency assays for beta-thalassemia and sickle cell disease autologous gene therapy drug products Ilya Shestopalov, Associate Director of Cell Analytics, bluebird bio (CONFIRMED)

Collaborative strategy for AAV Manufacturing for Biotech: is In or Out the only model? Jean-Philippe Combal, Co-Founder and Chief Executive Officer, Vivet Therapeutics (CONFIRMED)

Case study: gene therapy patient delivery and tech transfer Elena Beltrami, Business Development Manager, Telethon (CONFIRMED)

Development of therapeutic genome editing strategies Seokjoong Kim, Executive Director of R&D and Strategic Alliances, Toolgen (CONFIRMED)

Advancing restorative therapies to treat ischemic disease David Mazzo, President & CEO, Caladrius Biosciences (CONFIRMED)

Panel: Leading approaches for drug discovery in the mRNA space Yochi Slonim, CEO, Anima Biotech (CONFIRMED) Tim Luker, Senior Director External Innovation, Eli Lilly (CONFIRMED) David Blakey, CSO, MiNA Therapeutics (CONFIRMED)

Regenerative medicine – a small molecule approach Stuart Collinson, CEO, Oxstem (CONFIRMED)


16:00 Development of gene therapy in orphan diseases: The bbb perspective Representative, bluebird bio (CONFIRMED)

Identify and overcoming barriers to successful delivery of ATMP trials Eric Alton, Professor, Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London; Chair, NIHR ATMP Coordination Group (CONFIRMED)

Exploring the rise of machine learning and AI in next generation bioprocesses: Single Cell RNA sequencing to drive innovation and quality in iPSC therapeutics Robert Deans, CIO, BlueRock Therapeutics (CONFIRMED)

Manufacturing allogeneic and autologous cell therapies at GMP level Steve Oh, Director Stem Cell Bioprocessing & Institute Professor, Stem Cell Group, Bioprocessing Technology Institute, A*Star (CONFIRMED)

Case study: In-house manufacture vs. outsourcing – why and when? Devyn Smith, Chief Operating Officer & Head of Strategy, Sigilon, Inc (CONFIRMED)

Enabling precision healthcare through patient-centric, model-based supply chain optimisation Maria Papathanasiou, Research Associate Centre for Process Systems Engineering (CPSE) Dept. of Chemical Engineering Imperial College London (CONFIRMED)

Targeted Elimination of viral DNA using ARCUS gene editing Dave Morris, VP Therapeutic Discovery, Precision BioSciences (CONFIRMED)

Advanced therapy using a stem cell derived extracellular matrix Abdulkader Rahmo, CEO, SMSbiotech (CONFIRMED)

Vittorio Colizzi, Professor of Immunology, University of Rome Tor Vergata (CONFIRMED)

Hilary Hehman, Director, Partnerships and Alliances, Fred Hutch Cancer Research Center (CONFIRMED)

16.20 Afternoon break

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

Chair: Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) (CONFIRMED) Detlev Parow, Head of Department of Medicines, Therapeutic Appliances and Remedies, DAK (CONFIRMED) Luc Boileau, President and Chief Executive Officer, National Institute for Clinical Excellence in Health and Social Services (INESSS) (CONFIRMED) Nneka Onwudiwe, Founder and Chief Executive Officer, Pharmacoeconomi

Improving the efficiency of lentiviral vectors Simon Waddington, Professor of Gene Therapy University College London (CONFIRMED)

CAR T-cell immunotherapy of solid tumours: parallel learning from the clinic and lab John Maher, Consultant and Senior Lecturer in Immunology, King's College London, CSO, Leucid Bio (CONFIRMED)

Repair of the microcirculation reverses ischemic tissue damage Douglas Losordo, EVP, Global Head of Research and Development & CMO, Caladrius Biosciences (CONFIRMED)

The importance of robust process development and characterization early in clinical development of autologous cell therapies Dawn Maier, Sr. Director Process Development & Manufacturing, ElevateBio (CONFIRMED)

Lentiviral vector production for advanced therapies Leonard Pattenden, Head of Biotherapeutics Development & Drug Supply, Biotherapeutics Development Unit, Cancer Research UK (CONFIRMED)

Delivering allogenic cell therapy to patients in a clinical setting in USA and across Europe Lior Raviv, Vice President Development & Operations, Pluristem therapeutics (CONFIRMED)

Why Specialised Logistics Tim Davies, Business Development Manager, A4P Consulting (CONFIRMED)

Process Development and Manufacture of CAR-T cells in an automated, stirred-tank bioreactor Elena Costariol, EngD Researcher, UCL (CONFIRMED)

Providing a novel off-the-shelf implant to repair damaged organs Derek Dashti, Founder, Inventor & CEO, D&P Bioinnovations (CONFIRMED)

17.20 Panel discussion on tackling solid tumours: Prasad Adusumilli, Attending Surgeon, Memorial Sloan Kettering Cancer Center (CONFIRMED) Miguel Forte, CEO, Bone Therapeutics (CONFIRMED) Steven P. Lee, Senior Research Fellow, Institute of Immunology and Immunotherapy, University of Birmingham (CONFIRMED)

Panel discussion: Immunogenicity for gene therapy Key unresolved clinical issues in gene therapy: immunosuppression, re-dosing, and in-utero dosing Federico Mingozzi, CSO, Spark Therapeutics (CONFIRMED) Bobby Gaspar, CSO, Orchard Therapeutics (CONFIRMED) Eric Michael David, CEO, BridgeBio

Panel discussion: Precision medicine for immunotherapy Roy Baynes, SVP and Head Global Clinical Development, Chief Medical Officer, Merck, Sharp & Dohme (CONFIRMED) Giuseppe Mazza, Co-Founder and CEO, Engitix Ltd, Royal Free Hospital (CONFIRMED)

Alpha 10 Selected MSCs for the treatment of Osteoarthritis Sven Kili, CMO, Xintela (CONFIRMED)

Panel discussion (potency + assays): Predictive clinical functionality - guaranteeing the success of your cell therapy product Sakis Mantalaris, Professor, BioMedical Systems Engineering Laboratory, Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology (CONFIRMED)

Panel discussion: process development for next generation therapeutics Chair: Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET (CONFIRMED) Bo Kara, Vice President, Process Development, Evox Therapeutics (CONFIRMED) Angela Columbano, Director of Business and Development,

Panel discussion on Patient delivery: Alan K. Smith, Executive Vice President, Technical Operations, Bellicum Pharmaceuticals (CONFIRMED) Elena Beltrami, Business Development Manager, Telethon (CONFIRMED) Robert Jones, Vice President, Global Bioservices, Cryoport (CONFIRMED)

Renovite™ - synthetic nanoclay gels as a platform for regenerative medicine Agnieszka Janeczek, Founder/COO, Renovos (CONFIRMED)

Cost effective manufacturing strategies – thinking outside the box Nina Bauer, Commercial Head of Cell Therapy, MilliporeSigma Merck KGaA (CONFIRMED)

Glialign’s Engineered Neural Tissue (EngNT), an advanced therapy solution for peripheral nerve repair John Sinden, CEO, Glialign (CONFIRMED)


17.40 cs Consultants of America (CONFIRMED) Steven M. Altschuler, Managing Director, Healthcare Ventures, ZIFF Capital Partners (CONFIRMED) Doug Danison, Vice President, (market) Access, Value & Evidence Strategy (AVES), bluebird bio (TBC) Pilar Pinilla-Dominguez, Senior Scientific Adviser, NICE (invited) Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics (invited)

Stefanos Theoharis, Previously SVP Corporate Development and Partnering, Cell Medica, Independent Consultant (CONFIRMED) Neill Moray Mackenzie, Chairman, Immetacyte (CONFIRMED)

Gene Therapy (CONFIRMED) Joery De Kock, Professor, Vrije Universiteit Brussel; Scientific Advisory Board Member, RheinCell Therapeutics (CONFIRMED) Stephanie Schorge, Professor of Translational Neuroscience, Wellcome Chair in Pharmacology, UCL School of Pharmacy (TBC) Representative from bluebird bio

Igor Rudychev, Head of Digital, Data, and Innovations, US Oncology, AstraZeneca (CONFIRMED)

Longevity and new approaches to diseases of ageing Peter Hamley, CSO, Samsara Therapeutics (CONFIRMED)

Frank Luyten, Director of the Skeletal Biology and Engineering Research Center & Prometheus, K.U.Leuven, BE; Medical/Scientific Director, REGMEDXB (CONFIRMED) Maria Mirotsou, Senior Director, Resarch, Astellas Institute of Regenerative Medicine (CONFIRMED) Ilya Shestopalov, Associate Director of Cell Analytics, bluebird bio (CONFIRMED) Sergey A. Piletsky, Professor of Bioanalytical Chemistry, Chemistry Department, College of Science and Engineering, University of Leicester (CONFIRMED)

Genethon (CONFIRMED)

The role of AI in development of immunotherapies: opportunities and challenges Elzbieta Maria Piatkowska, CEO, Remissio (CONFIRMED)

Using big data applications to transform any cell type Julian Gough, Chief Scientific Officer, Mogrify (CONFIRMED)

Juergen Weisser, Managing Director, RheinCell Therapeutics (CONFIRMED)

The latest innovations in cell tracking and immunogenicity David Morrow, Senior Scientific Programme Manager-ATMP/Vaccines, EATRIS (TBC)

18.00 Close of Conference for the day - Please join us for an evening drinks reception at the iconic Emirates Stadium


CONFERENCE DAY 2 - WEDNESDAY 1st April 2020 CONFERENCE DAY 2 - WEDNESDAY 1st April 2020

Opening keynotes day 2 – The latest science in gene therapy and gene editing

08:55 Opening remarks: Antonio Regalado, Senior Editor for Biomedicine, MIT Technology Review (TBC)

09:00 Gene therapy 2.0: New clinical data, therapeutic applications and next generation technologies

• Latest updates on LUXTURNA studies to treat blindness, haemophilia and rare disease

• Tackling the challenges of viral gene therapies i.e. immunogenicity

• Developing the next generation of gene therapies, what does the future hold? Federico Mingozzi, CSO, Spark Therapeutics (CONFIRMED)

09.20 Next-generation gene editing technology for allogeneic immune cell therapeutics

• Next-generation CRISPR-Cas9 technology

• Significantly enhanced editing specificity

• Editing in immune cells for generation of functionally tuned therapeutics Steve Kanner, CSO, Caribou Biosciences (CONFIRMED)

09:40 Repairing the human genome with designed zinc finger editing reagents

• Overview of Sangamo’s zinc finger protein gene editing technology, including new innovations that facilitate specificity, precision, and gene editing efficiency

• Will address specific examples of defined therapeutic applications including the generation of reagents suitable for use in tauopathies, Huntington’s disease, and lysosomal storage diseases Adrian Woolfson, Head of R&D, Sangamo Therapeutics (CONFIRMED)

10:00 In vivo gene editing paediatric patients with rare diseases Fred Chereau, CEO, LogicBio Therapeutics (CONFIRMED)

10:20 Networking refreshments

EVIDENCE, PRICING & ACCESS


GENE THERAPY IMMUNO-THERAPY RESEARCH & DEVELOPMENT




PATIENT DELIVERY

GENE EDITING PITCH & PARTNER INNOVATION

Approval and evidence

Chair: Raghuram Selvaraju,

Managing Director, H.C. wainwright

(CONFIRMED)

Off the shelf: Moving towards

allogeneic models

Immuno-oncology Clinical developments and strategies for Immuno-oncology

Chair: Annie Hubert, Senior Director,

European Public Policy. Alliance for

Regenerative Medicine

(CONFIRMED)

Chair: Stephen Badylak, Professor,

Department of Surgery, Director,

McGowan Center for Pre-Clinical Studies,

Deputy Director, McGowan

Institute for Regenerative

Medicine (MIRM) (CONFIRMED)

Control Strategies Chair: Vivienne Parry, Writer and Broadcaster, Head of Engagement, Genomics England (CONFIRMED)

Investor Judges: Dmitry Kuzmin, Managing Partner, 4BIO Capital (CONFIRMED) Edward Hodgkin, Partner, Syncona Partners (CONFIRMED) Edward van Wezel, Managing Partner, Biogeneration Ventures (CONFIRMED) Daniel O’Mahony, Partner, Seroba Life Sciences (CONFIRMED) Dave Greenwald, Vice President, Business Development, Deerfield Management (CONFIRMED) Steven M. Altschuler, Managing Director, Healthcare Ventures, ZIFF Capital Partners (CONFIRMED) Dominic O'Regan, Director & Investor, New Mosaic Ltd (CONFIRMED) Alan J Muir, Director Life Science & Healthcare Investment, Seven Hills Venture Partners (CONFIRMED)

Exosome Therapeutics Chair: David

Pearce, President of Innovation and

Research, Sanford Health


Vijay Curthan, Investment Director, Catapult Ventures (CONFIRMED)

11:20 FDA's Real-world Evidence Framework for Evaluating RWD/RWE for Regulatory Decisions: A Look at Oncology and Rare Diseases Nneka Onwudiwe, Founder and Chief Executive Officer, Pharmacoeconomics Consultants of America (CONFIRMED)

Creation of off-the-shelf TCR-less CAR T cell therapies Bob Valamehr, Chief Development Officer, Fate Therapeutics (CONFIRMED)

Gene therapy for rare respiratory diseases Uta Griesenbach, Professor, Molecular Medicine, National Heart and Lung Institute, Imperial College London; President, British Society for Gene and Cell Therapy; Director (non-exec), Cell and Gene Therapy Catapult (CONFIRMED)

At the intersection of genetic medicine and immunotherapy: Clinical experience with a cell and gene therapy for HIV disease C. David Pauza, CSO, American Gene Technologies (CONFIRMED)

Assessment of clinical activity of PD-1 checkpoint inhibitor com-bination therapies reported in clinical trials Emmett Schmidt, Associate Vice President, MSD (CONFIRMED)

Challenges and Troubleshooting of starting auto-islet cell transplant program Chirag Desai, Professor of Surgery, Director, Chronic Pancreatitis & Autologous-Islet Cell Transplant Program, UNC School of Medicine (CONFIRMED)

Adjusting control strategies using Digital Twins Christoph Herwig, Professor of Biochemical Engineering, Vienna University of Technology (CONFIRMED)

Plasmid DNA Control Strategy & AAV Production Lawrence Thompson, Senior Principal Scientist, Analytical Research & Development, BioTherapeutics Pharm Sciences, Pfizer (CONFIRMED)

TBC The clinician’s perspective on patient access Marc Turner, Medical Director, Scottish National Blood Transfusion Service (invited)

Leo Groenewegen, Business Development Manager, cellulREvolution (CONFIRMED)

Engineered Exosome Therapeutics – Development and Manufacturing Bo Kara, Vice President, Process Development, Evox Therapeutics (CONFIRMED)

Peter Hamley, CSO, Samsara Therapeutics (CONFIRMED)

11:40 Regulatory approval and assessment of ATMPs – a quality assessor’s perspective Janet Glassford, Senior Quality Assessor - Medicines and Healthcare Products Regulatory Agency (CONFIRMED)

Automating and scaling manufacture of “off-the-shelf” engineered allogeneic CAR-T therapies Ricardo Baptista, Director Process & Analytical Development, Cellectis (CONFIRMED)

Latest data from haemophilia gene therapy trials Ted Tuddenham, Emeritus Professor of Haemophilia, UCL (CONFIRMED)

Kate Strayer-Benton, Senior Director of Business Development Belfer Office For Dana-Farber Innovations, Dana-Farber Cancer Institute (CONFIRMED)

Allogenic CAR T in the clinic and prospects for making CAR T more accessible to patients Christopher R. Heery, CMO, Precision BioSciences (CONFIRMED)

Cultivation strategies for stem cell expansion and cell-based therapy products Cornelia Kasper, Full University Professor, BOKU (CONFIRMED)

Miltenyi biotec TBC Manufacturing viral vectors on a commercial level

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

Self-Assembling Peptide Hydrogels for regenerative medicine; from bench to bedside Aline Miller, Chief Executive Officer and Co-Founder, Manchester BIOGEL (CONFIRMED)

An Exosome-based Drug Delivery Platform Derived from a Human Neural Stem Cell (hNSC) Line Randolph Corteling, Head of Research, Reneuron (CONFIRMED)

12:00 Anthony Lodge, CMC – Regulatory Lead, Kite Pharma (CONFIRMED)

TBC Developing the next-suite of CAR-T using non-viral vectors and using CRISPR in the latest technologies Chris Coughlin, CMO, Rubius

Case study: TIGEM’s gene therapy projects

Graciana Diez-Roux, Chief Scientific Officer,

Small activating RNAs as a novel approach for immunotherapy David Blakey, CSO, MiNA Therapeutics (CONFIRMED)

Update on gene therapy for primary immunodeficiencies Claire Booth, Senior Clinical Lecturer, Gene Therapy, UCL GOS Institute for Child Health (CONFIRMED)

BOOSTB4 project and trial: using stem cells to tackle osteogenesis imperfecta Cecilia Götherström, Associate Professor, Department of Clinical Science, Intervention &

Cytof/Mass Cytometry Analytics for CAR T Drug Products – Opportunities and Challenges Katja Kleinsteuber, Scientist II, Cellular

Challenges and concepts of designing a new GMP manufacturing facility at an academic centre Johannes C. M. van

Cell therapy for retina and stroke indications Michael Hunt, CEO, Reneuron (TBC)

Exosome production, isolation, and characterization Cornelia Kasper, Full University Professor, BOKU (CONFIRMED)



TIGEM (CONFIRMED)

Technology, Karolinska Institutet (CONFIRMED)

Analytics, Bluebird Bio (CONFIRMED)

der Loo, Director, Clinical Vector Core, The Children’s Hospital of Philadelphia (CONFIRMED)

Islet cell therapy for diabetes, using pig lungs as a scaffold Nikolai Kunicher, CEO, Betalin Therapeutics (TBC)

Sebastian Kress, Postdoctoral Scientist, Institute of Cell and Tissue Culture Technologies, BOKU (CONFIRMED)

12:20 Panel discussion: Regulatory submissions: gathering the evidence Chair: Victoria English, Co-founder & Editor, Evernow Publishing Ltd, publishers of MedNous (CONFIRMED) Janet Glassford, Senior Quality Assessor, Medicines and Healthcare Products Regulatory Agency (MHRA) (CONFIRMED) Nneka Onwudiwe, Founder and Chief Executive Officer, Pharmacoeconomics Consultants of America (CONFIRMED) Anthony Lodge, CMC – Regulatory Lead, Kite Pharma (CONFIRMED)

Gamma Delta T-Cells

ImmunoTox: Genetic manipulation of solid tumors to activate the natural γδ T cell response – American Gene C. David Pauza, CSO, American Gene Technologies (CONFIRMED)

Update on Genethon’s pipeline with fresh clinical developments Angela Columbano, Director of Business and Development, Genethon (CONFIRMED)

Immuno-oncology for breast cancer Stefan Gluck, Previously VP, GMA Early Assets, Celgene (TBC)

Panel discussion: Ensuring safe clinical development of next generation therapeutics Chair: Annie Hubert, Senior Director, European Public Policy. Alliance for Regenerative Medicine (CONFIRMED) Jane Lebkowski, President, Regenerative Patch Technologies (CONFIRMED) Boris Greber, Chief Scientific Officer, Head of Production, RheinCell Therapeutics (CONFIRMED) Claire Booth, Senior Clinical Lecturer, Gene Therapy, UCL GOS Institute for Child Health (CONFIRMED)

Clinical translation in regenerative medicine Frank Luyten, Director of the Skeletal Biology and Engineering Research Center & Prometheus, K. U. Leuven, BE; Medical/Scientific Director, REGMEDXB (CONFIRMED)

Bioinspired Manufacturing of hPSC-based Therapy Products Margarida Serra, Head of Stem Cell Bioengineering Laboratory, Animal Cell Technology Unit, iBET (CONFIRMED)

CMC and Analytics Assay development for oncolytics Jarema (Jarko) Kochan, Head of Biomarkers, Diagnostics and Assay Development, Turnstone Biologics (CONFIRMED)

Development and GMP Compliant Manufacturing of Mesenchymal Stem Cell (MSC) Derived Exosomes for Regenerative Medicine Aisha Khan, Executive Director of Lab Operations at the Interdisciplinary Stem Cell Institute Miller School of Medicine, University of Miami (CONFIRMED)

12:40 Genetic engineering of drug-resistant gamma delta T cells for eradication of residual glioblastoma Lawrence Lamb, Executive Vice President and Chief Scientific Officer, Incysus (CONFIRMED)

A virtual model for gene therapy R&D to target the complement system Erik Richardson, Senior Manager, Gene Therapy R&D, Apellis Pharmaceuticals (CONFIRMED)

SPONSOR Discovery of metabolic critical quality attributes for pluripotent stem cell-derived cardiomyocytes Sean Palecek, Director for Research, National Science Foundation Center for Cell Manufacturing Technologies (CMaT) (CONFIRMED)

Advanced, automated and cost-effective manufacturing strategies for mesenchymal stromal cell therapy Frank Barry, Professor, Cellular Therapy, The National University of Ireland; Senior Scientist, Toronto Arthritis Programme (CONFIRMED)

Phase-Appropriate Potency Assays for AAV Gene Therapy Products Aisleen McColl-Carboni, Senior Scientist, Sanofi (CONFIRMED)

Regulatory success parameters for translation of advanced therapies Magdi Elsallab, Doctoral Student, BIH Center For Regenerative Therapies Charite (CONFIRMED)

13:00 Networking Lunch

EVIDENCE, PRICING & ACCESS


GENE THERAPY RESEARCH & DEVELOPMENT




PITCH & PARTNER INNOVATION

Business models and

commercialisation

Gamma Delta T-Cells

Clinical & preclinical updates: Cell therapy Chair: Annie Hubert,

Senior Director, European Public Policy.

Alliance for Regenerative

Medicine (CONFIRMED)


14:00 Evaluating pharma partnerships: how & why Eric Soller, Vice President of Corporate Development & Strategy, BlueRock Therapeutics (CONFIRMED)

Gamma Delta T cells engineered with targeting moieties (CARs and TCRs) directed to cell surface and intracellular antigens Stewart Abbot, Chief Operating Officer, Adicet Bio (CONFIRMED)

Solving problems with AAV neutralisation in gene therapy Elena Piletska, Professor of Bioanalytical Chemistry, University of Leicester (CONFIRMED)

HLA-homozygous (HLAh) induced pluripotent stem (iPS) cells as universal source for ATMP development Boris Greber, Chief Scientific Officer, Head of Production, RheinCell Therapeutics (CONFIRMED)

Measurement of potency in cellular therapies – correlation with in vivo outcomes Neil McGowan, Head of Manufacturing, Tissues, Cells and Advanced Therapeutics, Scottish National Blood Transfusion Service (CONFIRMED)

Building your own closed manufacturing system Troels Jordansen,

CEO, Glycostem

Therapeutics

(CONFIRMED)

Industrialization of gene therapy manufacturing Overcoming manufacturing challenges faced by therapy developers- Effectively preparing for later stage trials and commercialization Pierre Albert Colcomb, Director Business Development, Genethon (CONFIRMED)

Developing the First External Quality Assurance (EQA) Program for ATMPs Antonio Rodríguez Acosta, Deputy Qualified Person. Cellular Manufacturing Unit, Regional University Hospital -Biomedical Research Institute (IBIMA) Málaga (CONFIRMED)

14:20 (to be updated)

Implementing

strategic

development and

innovating

disruptive business

models for

transformative cell

and gene therapies

Chair: Raghuram

Selvaraju,

Managing Director,

H.C. wainwright

(CONFIRMED)

RA Session, Chief

Business Officer &

Head of Corporate

Strategy, 4DMT

(CONFIRMED)

Sam Hall, Partner, Apple Tree Partners (CONFIRMED) Eric Soller, Vice President of Corporate Development & Strategy, BlueRock Therapeutics (CONFIRMED)

TEG’s as next generation of CAR-T Jürgen Kuball, Center of Translational Immunology (CTI), Dept of Hematology, UMC Utrecht (CONFIRMED)

Developing Liver directed gene therapy for paediatric indications Paul Gissen, LMCB Group Leader, UCL Professorial Research Associate, UCL Department of Child Health (CONFIRMED)

Jane Lebkowski, President, Regenerative Patch Technologies (CONFIRMED)

Mechanism of action of MSCs after local injection Mary Murphy, Professor in Regenerative Medicine, REMEDI (CONFIRMED)

The role of metabolism in cellular therapeutics biomanufacturing: can metabolomics be used for quality assurance & control? Sakis Mantalaris, Professor, BioMedical Systems Engineering Laboratory, Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology (CONFIRMED)

TBC Strategies and Advances in AAV Vector Characterization: Product-Related Impurities Christine Le Bec, Head of CMC Gene Therapy, working on AAV vectors, Sensorion (CONFIRMED)

ADVATIS

Representative from AMSBIO

14:40 NK Cells Engineering NK Cell Resistance to the Tumor Microenvironment Russell Cruz, Assistant Professor, Children's National Medical Center; Co-Founder, Mana Therapeutics (CONFIRMED)

TBC Preventing and slowing down progression of cones degeneration, first step in retinitis pigmentosa evolution Florence Allouche, President – CEO, SparingVision; Associate Professor, Faculty of Pharmacy, University of Paris (CONFIRMED)

TBC: Key Considerations in clinical development of advanced therapy medicinal products Giorgio Iotti, Head of Program Leadership and Control, Rare Disease Unit, Chiesi Farmaceutici SpA (CONFIRMED)

Cell characterisation through imaging for cell therapy and drug discovery Davide Danovi, Director, Cell Phenotyping Platform, Centre for Stem Cells & Regenerative Medicine, King's College London (CONFIRMED)

Automation Enabling the industrialization of cell and gene therapies through process development and platforms for commercial readiness Representative of LONZA

Advanced Machine Learning in Bioprocess Development - How Hybrid Models can speed up bioprocess development Maximillian Krippl, Data Scientist, Novasign; Research Assistant, BOKU (CONFIRMED)


15:00 Towards affordable and sustainable ATMP costs Frank Luyten, Director of the Skeletal Biology and Engineering Research Center & Prometheus, K.U.Leuven, BE; Medical/Scientific Director, REGMEDXB (CONFIRMED) Suzanne S. Farid, Professor of Bioprocess Systems Engineering & Co-Director Future Targeted Healthcare Manufacturing Hub, UCL (TBC)

Potentiation of the anti-tumour NK cell response in vitro and in vivo Mark Lowdell, European President, ISCT; Professor of Cell and Tissue Therapy, UCL; CSO, INmuneBio (CONFIRMED)

Development of a new viral vector for the treatment of Hyperoxalurea Sven Kili, Principal, Sven Kili Consulting (CONFIRMED)

Therapeutic potentials of activated islet progenitor cells for the treatment of diabetes Jonathan Pollett, Chief Research Officer, Imagine Pharma (CONFIRMED)

TBC Elizabeth Schwarzbach, Chief Business Officer, The New York Stem Cell Foundation (CONFIRMED)

Fully automated cell production for the therapies of the future Jelena Ochs, Manager Business Unit Life Sciences Engineering, Fraunhofer Institute for Production Technology IPT (CONFIRMED)

Development of novel assays and sensors to support manufacturing and therapy Sergey A. Piletsky, Professor of Bioanalytical Chemistry Chemistry Department College of Science and Engineering, University of Leicester (CONFIRMED)

15:20 World’s first off-the-shelf NK cellular immunotherapy making cancer a chronic disease Troels Jordansen, CEO, Glycostem Therapeutics (CONFIRMED)

TBC Capsid evolution for gene therapy Gabriel Brooks, Head of Clinical R&D, 4DMT (CONFIRMED)

Batten disease – finding the translational path Sara Mole, LMCB Group Leader, Professor of Molecular Cell Biology, UCL (CONFIRMED)

Johnny Huard, Director and Chief Scientific Officer, Center for Regenerative Sports Medicine, Steadman Philippon Research Institute (SPRI) (CONFIRMED)tor, Chronic Pancreatitis & Autologous-Islet Cell Transplant Program Associate Professor of Surgery

Novel concept of “cell manufacturability” and its application to automated cell manufacturing of retina epithelial pigment cells Masahiro Kino-Oka, BioProcess Systems Engineering Lab, Department of Biotechnology, Graduate School of Engineering, Osaka University (CONFIRMED)

Stem cells secretome and their efficiency in the treatment of soft tissue injury and skin rejuvenation Nada Alaaeddine, Managing Partner, Cormedicine Clinic of Regenerative Medicine; Associate Professor & Head of Research Committee, Faculty of Medicine, The Lebanese University (CONFIRMED)

15:40 TBC The difficulties encountered for the development and registration of a product of GT and supports proposed by the European Commission and the European Medicines Agency Didier Caizergues, Head of Regulatory Affairs Department, Genethon (CONFIRMED)

Different approaches to allogeneic therapies Stefanos Theoharis, Previously SVP Corporate Development and Partnering, Cell Medica, Independent Consultant (CONFIRMED)

Preclinical AAV for inflammatory disease and arthritic applications Janneke Meulenberg, COO, Arthrogen (invited)

3D scalable suspension culture of stem cells for tissue modelling with increased yield and reduced cost Todd McDevitt, Senior Investigator, Gladstone Institutes; Professor, Bioengineering & Therapeutic Sciences, UCSF (CONFIRMED)

Panel discussion: moving towards ‘smart’ and ‘flexible’ integrated process pipelines Jelena Ochs, Manager Business Unit Life Sciences Engineering, Fraunhofer Institute for Production Technology IPT (CONFIRMED Mary Murphy, Professor in Regenerative Medicine, REMEDI (CONFIRMED) Margarida Serra, Head of Stem Cell Bioengineering Laboratory, Animal Cell Technology Unit, iBET (CONFIRMED) Masahiro Kino-Oka, BioProcess Systems Engineering Lab, Department of Biotechnology, Graduate School of Engineering, Osaka University (CONFIRMED)

Innovation in the regenerative medicine space David Morrow, Senior Scientific Programme Manager-ATMP/Vaccines, EATRIS (TBC)

16:00 Networking refreshments


EVIDENCE, PRICING & ACCESS STEM CELLS & REGEN MED

Chair: Raghuram Selvaraju, Managing Director, H.C. wainwright (CONFIRMED)

16:30 The importance of interactions with Regulatory Authorities during ATMP development Margareth Jorvid, Head of Regulatory Affairs and QA, Immunicum AB (CONFIRMED)

The influence of immunomodulatory biomaterials upon stem cell fate Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center for Pre-Clinical Studies, Deputy Director, McGowan Institute for Regenerative Medicine (MIRM) (CONFIRMED)

16:50 Looking at value for advanced therapies in Quebec Luc Boileau, President and Chief Executive Officer, INESSS (CONFIRMED) Michèle de Guise, Director of Health Services and Technology Evaluation, INESSS (CONFIRMED)

17:10 TBC Common pitfalls and hurdles when reaching registration and market access for a new next generation therapeutic Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon (CONFIRMED)

Panel discussion: stem cells and regenerative medicine Frank Barry, Professor, Cellular Therapy, The National University of Ireland; Senior Scientist, Toronto Arthritis Programme (CONFIRMED) Debora Esposito, Regenerative Medicine, Assistant Professor, Animal Science Department, Plants for Human Health Institute, NC State University Francesco Dazzi, Professor of Regenerative and Haematological Medicine, KHP Lead for Cellular Therapies, King’s College London (CONFIRMED)

17:30 Patient perspective: advanced therapies and the shortage of medication in Europe Roland Pochet, Professor, Université Libre de Bruxelles; Secretary General, Belgian Brain Council; Patient Representative, CAT EMA (CONFIRMED)

18:00 End of Conference

22


Networking break content: Seminar Theatre Agenda

eminar Theatre Agenda Day 2 - Wednesday 1st April 2020

Location: Seminar Theatre1 (Innovation) Chair:

10:20 Conference breaks for coffee and networking

10:20

10:30

10:40

11:00

11:10

12:00 Conference Sessions resume

13:00 Conference breaks for lunch

13:00

13:10

13:20

13:30

13:40

13:50

14:00

14:10

16:00 Conference sessions resume

16:00

16:10

16:20

16:30

16:30 – Main conference resumes

16:30 Close of exhibition – see you next year!

Seminar Theatre Agenda Day 1 - Tuesday 31st March 2020

Location: Seminar Theatre1 (Innovation) Chair:

10:20 Conference breaks for coffee and networking

10:20 HAEP – A Novel Format to Foster Innovation through Partnering

• technology and innovation scouting service

• sourcing from a vast network

• successful track record

• custom-fit search, care-free match, enabling innovation Friedemann Loos, Innovation Manager, BioRN (CONFIRMED)

10:30 TINs Pamela Tranter, Head, Translational Research Group, UCL (CONFIRMED)

10:40

11:00

11:10

12:00 Roundtables in Session

13:00 Conference breaks for lunch

13:00

13:10

13:20

13:30

13:40

13:50

14:00

14:10

16:20 Conference sessions resume

16:20

16:30

16:40

16:50

17:00 – Main conference resumes

18:00 Close of exhibition – see you tomorrow!

23


Documents

PLEASE NOTE THIS IS AN EARLY DRAFT AGENDA …...PLEASE NOTE THIS IS AN EARLY DRAFT AGENDA AND ALL SPEAKERS ARE IN THE PROCESS OF BEING INVITED UNLESS SPEIFIED AS ‘ONFIRMED’ Johannes