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Tuesday, May 1
04:00 PM - 07:00 PM

Registration Open

04:00 PM - 07:00 PM

Speaker Services Open

Wednesday, May 2
07:00 AM - 06:00 PM

Speaker Services Open

08:00 AM - 10:00 AM

GRP Workshop Session 1 - Locally Developed Complex Immunotherapies for Global Supply

Moderator:
Dominic Wall, PhD, FFSc(RCPA), Peter MacCallum Cancer Centre, Australia
Speaker:
Francesco Cicirello, PharmD (GPhC, FOPH), MSc, Inspector, Manufacturing Quality Branch, Medical Devices and Product Quality Division, Therapeutics Goods Administration, Australia (audio conference)

Steven S. Oh, PhD, Chief, Cell Therapies Branch, Office of Cellular, Tissue, and Gene Therapies, Center for Biologics, Evaluation and Research, U.S. FDA, United States

John Ng, B.Eng, Chief Operations Officer and Executive Director, Tessa Therapeutics, Singapore

Patient specific cell-based immunotherapies have evolved into potent therapeutic products that are being considered for multiple markets on the basis of small-scale clinical studies. What does this mean for global supply where the clinical data will predominantly be developed in one market? How is this affected by complex overlays of local donor management and centralized single site manufacturing elsewhere, and what does this mean from the perspective of the inspectorates?
Moderator: Dominic Wall, PhD, FFSc(RCPA), Peter MacCallum Cancer Centre, Australia Speaker: Francesco Cicirello, PharmD (GPhC, FOPH), MSc, Inspector, Manufacturing Quality Branch, Medical Devices and ...
08:30 AM - 10:00 AM

ISCT-CBA Cord Blood Series Session 1 - Current Status of Cord Blood Transplantation

Speakers:
Lessons Learned from Cord Blood Banking and Transplantation
Joanne Kurtzberg, MD Duke University Medical Center, United States

Expansion and Homing
Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States

Manufacturing Novel Cell Therapy Products from Cord and Blood Tissue
Mitchell Horwitz, MD, Duke University School of Medicine, United States
Speakers: Lessons Learned from Cord Blood Banking and Transplantation Joanne Kurtzberg, MD Duke University Medical Center, United States Expansion and Homing Elizabeth J. Shpall, MD, MD Anderson C...
08:30 AM - 10:00 AM

MSC Workshop Session 1 - Development of Potency Assays to Predict MSC Clinical Efficacy

Chair:
Don Phinney, PhD, The Scripps Research Institute, United States
Speakers:
Don Phinney, PhD, The Scripps Research Institute, United States
Massimo Dominici, MD, University of Modena and Reggio Emilia, Italy
Steven R. Bauer, PhD, U.S. Food and Drug Administration (FDA), United States
Chair: Don Phinney, PhD, The Scripps Research Institute, United States Speakers: Don Phinney, PhD, The Scripps Research Institute, United States Massimo Dominici, MD, University of Modena and Reggio ...
08:30 AM - 10:00 AM

Canadian Cell & Gene Therapy Strategy Workshop Session 1 - Cell & Gene Therapy Manufacturing Infrastructure

Chair:
Armand Keating, MD , Director, Division of Hematology, Epstein Chair in Cell Therapy and Transplantation, University of Toronto, Canada

Speakers:
Current State of GMP Manufacturing in Canada
Denis-Claude Roy, MD, PhD, CEO, CellCAN, Montreal, Canada

Current State of GMP Manufacturing in France - Bioregate Perspective
Oumeya Adjali, MD, PhD, Directrice du laboratoire de thérapie génique de Nantes, Unité de thérapie cellulaire et génique du CHU de Nantes, Nantes, France

Current State of GMP Manufacturing in Australia
John Rasko, MD, PhD, ISCT President-Elect, Professor of Medicine, Central Clinical School, Centenary Institute of Cancer Medicine& Cell Biology, Sydney, Australia

Manufacturing Requirements for CAR-T Cell Therapies and Engineered Cells
Isabelle Rivière, PhD, Director, Cell Therapy and Cell Engineering Facility, Memorial Sloan Kettering Cancer Centre, New York, United States

Coordinating clinical needs with manufacturing capabilities requires a vast knowledge of existing infrastructure and a clear vision of the direction for future expansion. In this session, the current cell and gene therapy manufacturing facilities landscape will be reviewed, contrasting academic vs. private centers, production capacity for multicenter clinical trials, centralized vs. decentralized manufacturing, sustainability of current infrastructure and maximizing output will be discussed. The landscape in Canada, France, Australia and the United States will be compared to identify strengths and opportunities.
Chair: Armand Keating, MD , Director, Division of Hematology, Epstein Chair in Cell Therapy and Transplantation, University of Toronto, Canada Speakers: Current State of GMP Manufacturing in Canada D...
08:30 AM - 10:00 AM

ISCT-FACT Cell Therapy Quality Boot Camp Session 1 - Quality Management Program

Adapting a Quality Management Program to Fit Your Needs
Natasha Lapteva, PhD, Houston Methodist Hospital Bone Marrow Transplant (affiliated with Baylor College of Medicine Cell and Gene Therapy, United States

Quality Management Roundtable
Adapting a Quality Management Program to Fit Your Needs Natasha Lapteva, PhD, Houston Methodist Hospital Bone Marrow Transplant (affiliated with Baylor College of Medicine Cell and Gene Therapy, Unit...
08:30 AM - 10:00 AM

Cell Processing 1 - Novel Cell Processing Technologies

Chair:
Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States
Speakers:
Locally Produced Dual Targeted anti-CD19/anti-CD20 CAR-T cell Therapy for Patients with Relapsed, Refractory NHL
Nirav Shah, MD, MS, Medical College of Wisconsin, United States

Improved Cell Recovery and Response by the use of Deterministic Lateral Displacement in Blood Product Processing
Tony Ward, MBA, GPB Scientific, United States

Approaches to iPSC Generation
Vittorio Sebastiano, PhD, Stanford School of Medicine, United State
Chair: Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States Speakers: Locally Produced Dual Targeted anti-CD19/anti-CD20 CAR-T cell Therapy for Patients with Relapsed, Refractory NHL ...
10:00 AM - 10:15 AM

Coffee Break

10:15 AM - 11:45 AM

Cell Processing Track Session 2 - Growth Areas in Novel Cell Therapy Quality Control and Release Testing

Chair:
David DiGiusto, PhD, Stanford Healthcare and Stanford School of Medicine, United States
Speakers:
Approaches to Assessing Cell Function
Adrian Gee, PhD Baylor College of Medicine, United States

Industry Perspective on Quality Control and Release Testing
Jean-Pierre Latere, PhD, Celyad, Belgium

VCN and RCL Testing for Lentiviral Vectors
Lindsey Skrdlant, PhD, Stanford University School of Medicine, United States
Chair: David DiGiusto, PhD, Stanford Healthcare and Stanford School of Medicine, United States Speakers: Approaches to Assessing Cell Function Adrian Gee, PhD Baylor College of Medicine, United Sta...
10:15 AM - 11:45 AM

GRP Workshop Session 2 - CMC Considerations When Expanding the Market into Other Regions

Moderator:
Christopher Bravery, PhD, Consulting on Advanced Biologicals Limited, United Kingdom
Speakers:
I-Ning Tang, MD, MSc, , Senior Medical Reviewer, Center for Drug Evaluation (CDE), Taiwan

Nadine K. Kolas, PhD, Senior Policy Analyst, Health Canada, Canada

Victor Lietao Li, MD, Founder and CEO, Lion TCR Pte Ltd, Singapore

Unlike other biological medicinal products, autologous cell-based products in particular, pose unique challenges because the donor starting material needs to be procured at the hospital and shipped to the manufacturing site within a short period of time (typically less than 48h). Consequently, when expanding operations into other markets, local rules for donor collection need to be addressed.

Some new markets will be too far from the initial manufacturing site to allow timely shipment, in which case manufacturing needs to be located closer to or in the new market. Even simply replicating an existing process at another site can lead to small changes in procedures, equipment and raw materials, but also local regulatory expectations may differ.
Moderator: Christopher Bravery, PhD, Consulting on Advanced Biologicals Limited, United Kingdom Speakers: I-Ning Tang, MD, MSc, , Senior Medical Reviewer, Center for Drug Evaluation (CDE), Taiwan Nad...
10:15 AM - 11:45 AM

ISCT-CBA Cord Blood Series Session 2 - Cord Blood Immunotherapy

Speakers:
Cord Blood NK Cells
Katy Rezvani, MD, PhD, MD Anderson Cancer Center, United States

Cord Blood T Cells (Including HIV)
Catherine Bollard, MBChB, MD, Children's National Medical Center and The George Washington University, United States

New CBT Trials Investigating Third Party Progenitors and EC Based Expansion
Juliet Barker, MBBS, FRACP, Memorial Sloan Kettering Cancer Center, United States
Speakers: Cord Blood NK Cells Katy Rezvani, MD, PhD, MD Anderson Cancer Center, United States Cord Blood T Cells (Including HIV) Catherine Bollard, MBChB, MD, Children's National Medical Center and...
10:15 AM - 11:45 AM

MSC Workshop Session 2 - Methods to Improve MSC Engraftment and Potency In Vivo

Chair:
Jan Nolta, PhD, University of California Davis, United States
Speakers:
Jan Nolta, PhD, University of California Davis, United States
David Courtman, PhD, Ottawa Hospital Research Institute, Canada
Armand Keating, MD, Princess Margaret Hospital, Canada
Chair: Jan Nolta, PhD, University of California Davis, United States Speakers: Jan Nolta, PhD, University of California Davis, United States David Courtman, PhD, Ottawa Hospital Research Institute, ...
10:15 AM - 11:45 AM

Canadian Cell & Gene Therapy Strategy Workshop Session 2 - Investment in Canada: The Global Advantage

Chair:
Michael May, PhD, President & Chief Scientific Officer, Centre for Commercialization of Regenerative Medicine

Speakers:
Leveraging Funding for GMP Manufacturing Infrastructure - Federal Support of Industry Investment: What More Bang For Your Buck Gets You
Aaron Dulgar-Tulloch, PhD, Director, BridGE, GE Healthcare, Toronto, Canada

Doing Business in Canada - Developing Best in Class iPSC Therapies as an Industry Leader
Robert Deans, PhD, Chief Technology Officer, BlueRock Therapeutics, Toronto, Canada

Biomanufacturing for Cell and Gene Therapy Production in Canada
Frank Van Lier, PhD, Director R&D, Bioprocess Engineering, Life Sciences, Human Health Therapeutics, National Research Council Canada, Montreal, Canada

In this session, strategic opportunities that exist for industry to invest in cell & gene therapy infrastructure in Canada will be discussed. How industry investment in Canadian GMP manufacturing facilities strengthens marketing and visibility, the impact of industry-driven later-phase clinical trial and commercialization will be reviewed.
Chair: Michael May, PhD, President & Chief Scientific Officer, Centre for Commercialization of Regenerative Medicine Speakers: Leveraging Funding for GMP Manufacturing Infrastructure - Federal Suppor...
10:15 AM - 11:45 AM

ISCT-FACT Cell Therapy Quality Boot Camp Session 2 - Validation, Verification and Qualification

Using Validation, Verification, and Qualification to Ensure Proper Equipment Operation

Nancy Collins, PhD, FACT Consultant and Clinical Assistant Professor at the University of Toledo
Deborah Griffin, MSc, ASQ CPGP, FACT Consultant

Validation, Verification, and Qualification Roundtable
Using Validation, Verification, and Qualification to Ensure Proper Equipment Operation Nancy Collins, PhD, FACT Consultant and Clinical Assistant Professor at the University of Toledo Deborah Griffin...
11:45 AM - 01:00 PM

Lunch

11:45 AM - 01:00 PM

MSC Workshop Industry Luncheon

12:00 PM - 12:30 PM

GRP Lunch Seminar Presented by HESI-CT-TRACS - Safety Assessment of Cell Therapy Products: Challenges and Current Advances. Perspectives From a New International Multi-Stakeholder Collaborative Platform

Speakers:
Brooke M. Helfer, PhD, Director of Research and Development, Celsense Inc, United States
Yoji Sato, PhD, Head, Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan
Speakers: Brooke M. Helfer, PhD, Director of Research and Development, Celsense Inc, United States Yoji Sato, PhD, Head, Division of Cell-Based Therapeutic Products, National Institute of Health Sci...
12:45 PM - 02:30 PM

GRP Workshop Session 3 - Trial Design and Patient Cohorts

Moderator:
Karen Nichols, Esq, Vice President Regulatory and Quality, Magenta Therapeutics, United States
Speakers:
Marc Turner, MBChB, PhD, MBA, Medical Director, Scottish National Blood Transfusion Service, Scotland

Shari Targum, MD, MPH, Branch Chief, Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, United States (audio conference)

Michael Pepper, MBChB, PhD, MD, Director, Institute for Cellular and Molecular Medicine and SAMRC Extramural Unit for Stem Cell Research and Therapy, University of Pretoria, South Africa

Sponsor wants to adapt their clinical study design to a pediatric population in Europe. They want to use as much of the same design as possible although clinical assessments from the adult study suggest that apheresis yields patient-specific cell populations at collection which may contribute to more variability in manufacturing products. How should the sponsor manage this variability? What, if any, additional product characterization should be applied to the starting T-cell material as well as the final product? Collections will occur at various locations, even if processing is centralized, introducing another element of variability. To what extent should the harvest be considered part of the trial design, and to what extent are failed cell harvests and insufficient recovered cell doses relevant to the regulatory strategy? What is the role of a pediatric investigation plan when administering a personalized medicine?

Given that this is a small (in numbers) patient population, patients will present and perhaps not meet the inclusion criteria – will the sponsor opt for expanded access for these patients? What is the regulatory view of doing such for a CAR-T product?
Moderator: Karen Nichols, Esq, Vice President Regulatory and Quality, Magenta Therapeutics, United States Speakers: Marc Turner, MBChB, PhD, MBA, Medical Director, Scottish National Blood Transfusion...
01:00 PM - 02:30 PM

Cell Processing Track Session 3 - Cell Product Characterization I

Chair:
Ruud Hulspaas, PhD , Cellular Technologies Bioconsulting, United States
Speakers:
Flow Cytometry Standardization and Robust Cell Antigen Quantification: A Road Toward Predicting Cell Biological Characteristics
Heba Degheidy, MD, PhD , FDA, United States

Quantitative Flow Cytometry in Cell Therapy - Challenges and Risk Mitigation
Ines Mende, PhD, Hitachi Chemical Advanced Therapeutic Solutions, LLC

When Stem Cells Meet Immunotherapy: The Critical Role of Multi-Parameter Flow Cytometry In The Development of Off-The-Shelf Cell Therapies
Tamara Laskowski, PhD, MD Anderson Cancer Center, United States
Chair: Ruud Hulspaas, PhD , Cellular Technologies Bioconsulting, United States Speakers: Flow Cytometry Standardization and Robust Cell Antigen Quantification: A Road Toward Predicting Cell Biologica...
01:00 PM - 02:30 PM

ISCT-CBA Cord Blood Series Session 3 - Manufacturing Novel Cell Therapy Products from Cord and Blood Tissue

Speakers:
Characterization of MSCs Derived from Various Donor Sources
Carolyn Yeago, PhD, Georgia Institute of Technology, United States

Manufacturing of Cord Tissue MSCs
Joanne Kurtzberg, MD, Duke University Medical Center, United States

Treatment of Patients with Frailty Syndrome with MSCs
Ivonne Hernandez Schulman, MD, University of Miami Miller School of Medicine, United States

Cord Tissue MSC-Derived Exosomes
Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States
MSC Therapy for Steroid Refractory GVHD in Pediatric Patients
Joanne Kurtzberg, MD, Duke University Medical Center, United States
Speakers: Characterization of MSCs Derived from Various Donor Sources Carolyn Yeago, PhD, Georgia Institute of Technology, United States Manufacturing of Cord Tissue MSCs Joanne Kurtzberg, MD, Duke...
01:00 PM - 02:30 PM

MSC Workshop Session 3 - “Responders” to MSC Therapy – Predicting Clinical Outcomes

Chair:
Katarina LeBlanc, MD, PhD, Karolinska University Institutet, Sweden
Speakers:
Katarina LeBlanc, MD, PhD, Karolinska University Institutet, Sweden
Eleuterio Lombardo, PhD, TiGenix SAU, Spain
Michael Matthay, MD, University of California San Francisco, United States
Anna Krasnodembskaya, PhD , Queen's University Belfast, United Kingdom
Chair: Katarina LeBlanc, MD, PhD, Karolinska University Institutet, Sweden Speakers: Katarina LeBlanc, MD, PhD, Karolinska University Institutet, Sweden Eleuterio Lombardo, PhD, TiGenix SAU, Spain M...
01:00 PM - 02:30 PM

Canadian Cell & Gene Therapy Strategy Workshop Session 3 - Making Frontline Life-Saving Therapies Economically Feasible

Chair:
Lambert Busque, MD, Chief Medical Officer, Center for Commercialisation of Cancer Immunotherapy, Montreal

Speakers:
Health Canada's NOC/C: Requirements for Accessing Novel Frontline Lifesaving Cell & Cell-Based Therapies
Kelly Robinson, MSc, Director, Centre for Evaluation of Radiopharmaceuticals & Biotherapeutics, Biologics and Genetic Therapies Directorate, Health Canada, Ottawa, Canada

The Challenges to HTA Agencies of Expedited Regulatory Approvals and Approaches to Managing the Consequent Risk
Nick Crabb, PhD, Programme Director, Scientific Affairs, National Institute for Health and Care Excellence, London, United Kingdom

Risk Sharing Models for Reimbursement
Richard Maziarz, MD, Professor of Medicine, Oregon Health & Science University, Chair, Interest Group for ASBMT on Value and Health Economics, Portland, United States

Innovative cellular therapies are disruptive technologies to healthcare and require adequate foresight in drafting novel reimbursement policies. In this session, cell & gene therapies, such as viral vector-based gene therapies involving cells, hematopoietic stem cell transplantation, CAR-T cell therapy, and cancer immunotherapy will be discussed in the context of regulatory policy, patient access and novel reimbursement strategies. Should private or public insurance pay?
Chair: Lambert Busque, MD, Chief Medical Officer, Center for Commercialisation of Cancer Immunotherapy, Montreal Speakers: Health Canada's NOC/C: Requirements for Accessing Novel Frontline Lifesav...
01:00 PM - 02:30 PM

ISCT-FACT Cell Therapy Quality Boot Camp Session 3 - Auditing

Closing the Audit Process Through Follow-Up

Nicole Prokopishyn, PhD, Alberta Blood and Marrow Transplant Program: Foothills Medical Centre, Alberta Children's Hospital, Tom Baker Cancer Centre, and Calgary Laboratory Services, Canada

Audit Roundtable
Closing the Audit Process Through Follow-Up Nicole Prokopishyn, PhD, Alberta Blood and Marrow Transplant Program: Foothills Medical Centre, Alberta Children's Hospital, Tom Baker Cancer Centre, and ...
02:30 PM - 02:45 PM

Coffee Break

02:45 PM - 04:00 PM

ISCT-FACT Quality Bootcamp Session 4 - Occurrence Management

Going Beyond Occurrence Management to Mitigate Risks

Mary Ann Kelley, MT(ASCP)BB, Dana-Farber Cancer Institute / Cell Manipulation Core Facility

Occurrence Management Roundtable
Going Beyond Occurrence Management to Mitigate Risks Mary Ann Kelley, MT(ASCP)BB, Dana-Farber Cancer Institute / Cell Manipulation Core Facility Occurrence Management Roundtable
02:45 PM - 04:15 PM

Cell Processing Track Session 4 - Cell Product Characterization II

Chair:
Ruud Hulspaas, PhD, Cellular Technologies Bioconsulting, United States
Speakers:
Case Studies in Multi-Parameter Flow Cytometric Characterization of TCR, TIL, and CAR-T Cell Therapy Products
Kathryn Holderness, MS, Lonza, United States

Mechanisms of Response and Resistance to CD19-targeting Chimeric Antigen Receptors in Leukemia
J. Joseph Melenhorst, PhD, University of Pennsylvania, United States

Case Studies in Multi-Parameter Flow Cytometric Characterization of TCR, TIL, and CAR-T Cell Therapy Products
Sadik Kassim, PhD, Mustang Bio, United States
Chair: Ruud Hulspaas, PhD, Cellular Technologies Bioconsulting, United States Speakers: Case Studies in Multi-Parameter Flow Cytometric Characterization of TCR, TIL, and CAR-T Cell Therapy Products...
02:45 PM - 04:15 PM

ISCT-CBA Cord Blood Series Session 4 - Cord Blood for Regenerative Medicine

Speakers:
Outcomes of Cord Blood Therapies in Pediatric Neurologic Diseases
Joanne Kurtzberg, MD, Duke University Medical Center, United States

MSC Treatment of Chronic Pulmonary Diseases in Adults
Marilyn Glassberg, MD, University of Miami Miller School of Medicine, United States

MSCs - Novel Therapy for Patients with Cystic Fibrosis
Arnold Caplan, PhD, Case Western Reserve University, United States
Speakers: Outcomes of Cord Blood Therapies in Pediatric Neurologic Diseases Joanne Kurtzberg, MD, Duke University Medical Center, United States MSC Treatment of Chronic Pulmonary Diseases in Adults ...
02:45 PM - 04:15 PM

MSC Workshop Session 4 - Summary and Discussion

Chair:
Jacques Galipeau, MD, FRCP(C), University of Wisconsin-Madison, United States
Speakers:
Jacques Galipeau, MD, FRCP(C), University of Wisconsin-Madison, United States
Chair: Jacques Galipeau, MD, FRCP(C), University of Wisconsin-Madison, United States Speakers: Jacques Galipeau, MD, FRCP(C), University of Wisconsin-Madison, United States
02:45 PM - 04:15 PM

Canadian Cell & Gene Therapy Strategy Workshop Session 4 - Strengthening the Canadian RMCT Ecosystem

Speakers:
The Regenerative Medicine Alliance of Canada
Michael Rudnicki, PhD, Chair, Regenerative Medicine Alliance of Canada, Scientific Director, Stem Cell Network, Ottawa, Canada

Examining Canada's Clinical Trials Environment
Erika Kleiderman, BSc, LLB, Academic Associate, Centre of Genomics and Policy, McGill University, Montreal, Canada

Successful Canadian Therapeutics Manufacturing: Challenges of Scale-Up and Expansion
Dina Iezzi, Director, Marketing & Special Projects Therapure Biopharma Inc, Toronto, Canada

Building Successful International Collaborations
Duncan Stewart, MD, President and Scientific Director, Ontario Institute for Regenerative Medicine, Ottawa, Canada

Stem Cell research was pioneered in Canada in the early 60s and ever since Canada’s contribution to regenerative medicine has been unwavering. Today, this emerging Canadian sector has taken hold. In partnership with government, industry and others, Canada is well positioned to realize a robust and globally competitive sector. This session will look at Canada’s regenerative medicine ecosystem in an effort to better understand where the opportunities lie.
Speakers: The Regenerative Medicine Alliance of Canada Michael Rudnicki, PhD, Chair, Regenerative Medicine Alliance of Canada, Scientific Director, Stem Cell Network, Ottawa, Canada Examining Canada...
02:45 PM - 04:45 PM

GRP Workshop Session 4 - Biological Variation and Cell Product Efficacy

Moderator:
Janet Macpherson, PhD, Development Manager, Royal Prince Alfred Hospital, Australia

Speakers:
Christiane Niederlaender, PhD, Senior Quality Assessor and CAT Member, MHRA, United Kingdom

Ramjay S. Vatsan, PhD, Team Lead, Division of Cellular & Gene Therapies, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, United States

Erik Rutjens, PhD, Global Head, New and Enabling Technologies, Cell and Gene Therapy Development and Manufacturing, Novartis Pharmaceuticals Corporation, United States

Inherent variability between products is a given for autologous cellular immunotherapy products due to their patient-derived nature. What strategies can be employed to help predict the likelihood of clinical success, and do regulators consider this important? What should be done to determine the host factors that predict biological response to a product? What technology is available to assist in determining treatment effectiveness? How much data is required? At what stage of product development should these evaluations be undertaken?
Moderator: Janet Macpherson, PhD, Development Manager, Royal Prince Alfred Hospital, Australia Speakers: Christiane Niederlaender, PhD, Senior Quality Assessor and CAT Member, MHRA, United Kingdom ...
04:30 PM - 06:30 PM

Corporate Symposium Hosted by Miltenyi Biotec - Emerging Technologies and Clinical Advancements in Cell Therapy

Chair:
Neehar Bhatia, PhD, Stanford School of Medicine, United States

Speakers:
First Clinical Trials Utilizing the Automated CliniMACS Prodigy®TCT Process
Claire Roddie, PhD, UCL Cancer Institute, United Kingdom
CAR T Cell Production at the Conconi Family Immunotherapy Lab Using the CliniMACS Prodigy platform
John Webb, PhD, British Columbia Cancer Agency, Canada
Adoptive T cell therapy: Enabling technologies for clinical trials
Cassian Yee, MD, University of Texas MD Anderson Cancer Center, United States
CAR T cells: progress and challenges
Crystal Mackall, MD, Stanford University, United States

This symposium will focus on clinical application and development of cell therapies.
Chair: Neehar Bhatia, PhD, Stanford School of Medicine, United States Speakers: First Clinical Trials Utilizing the Automated CliniMACS Prodigy®TCT Process Claire Roddie, PhD, UCL Cancer Institute,...
07:00 PM - 07:30 PM

ISCT 2018 President's Welcome Address

07:30 PM - 09:30 PM

Thursday, May 3
07:00 AM - 06:00 PM

Speaker Services Open

07:00 AM - 07:00 PM

Registration Open

07:30 AM - 08:30 AM

Hot Topic Session 1 - Tregs and Other Cell Subtypes/Immunotherapy in Autoimmune Disease

Chair:
David DiGiusto, PhD, Stanford Healthcare and Stanford School of Medicine, United States
Speakers:
Everett Meyer, MD, PhD, Stanford Healthcare and Stanford University, United States

The Emerging Role of Cell Therapies in Gastrointestinal Disorders
Alan Burns, PhD Takeda Pharmaceuticals, United States

The use of regulatory T-cells to combat autoimmunity and GVHD and other (mesenchymal) forms of stem cells for immune-based disease indications is an area of significant interest to ISCT investigators. In this session the presenters will discuss the identification, pre-clinical evaluation and clinical implementation of various forms of regulatory T-cells in hematopoietic stem cell transplantation and eASC in Crohn's disease.
Chair: David DiGiusto, PhD, Stanford Healthcare and Stanford School of Medicine, United States Speakers: Everett Meyer, MD, PhD, Stanford Healthcare and Stanford University, United States The Emerg...
07:30 AM - 08:30 AM

Hot Topic Session 2 - Approaches to Overcoming Limitations of Cell and Gene Therapies

Chair:
Sandeep Soni, MD, Stanford University, United States
Speakers:
Transduction Efficience is a Critical Component Defining the Clinical Outcome in Gene Therapy
Gabor Veres, PhD bluebird bio, United States

Antibody-based Conditioning for Hematopoeitic Stem Cell Therapies
Judith Shizuru, MD, PhD, Stanford University Medical Center, United States

Programmed Cellular Immunotherapies for Fighting Cancer
Daniel Shoemaker, PhD , Fate Therapeutics, United States
Chair: Sandeep Soni, MD, Stanford University, United States Speakers: Transduction Efficience is a Critical Component Defining the Clinical Outcome in Gene Therapy Gabor Veres, PhD bluebird bio, U...
07:30 AM - 08:30 AM

Corporate Breakfast Tutorial Hosted by GE Healthcare - Learning by doing: Learn from clinical and manufacturing leaders’ through their journey from development to delivery of cellular therapies

Speakers:
Allan Dietz, PhD, Mayo Clinic, United States
Marcela Maus, MD, PhD, Massachusetts General Hospital, United States
Bruce Levine, PhD, University of Pennsylvania, United States

Academic clinical institutions are fueling the remarkable advances in research, development and commercialization of cellular therapies. This progress is driving the need to adopt cGMP production standards during early development phases. Alongside industry, academic institutions must grapple with ways to assure product consistency, safety, and availability of the product. In this session panelists will discuss the evolution and challenges faced by clinical centers in their pursuit of developing and delivering new cellular therapies to the patients they serve.
Speakers: Allan Dietz, PhD, Mayo Clinic, United States Marcela Maus, MD, PhD, Massachusetts General Hospital, United States Bruce Levine, PhD, University of Pennsylvania, United States Academic clin...
07:30 AM - 08:30 AM

Corporate Breakfast Tutorial Hosted by Roche CustomBiotech - Rapid microbiological methods in meeting unique QC and product-specific validation needs of advanced therapy manufacturing

Chair:
Evonne R. Fearnot, MSBME, Roche CustomBiotech, United States

Speaker:
Alexey Bersenev, MD, PhD, Yale University, United States
Sowmya Viswanathan, PhD, University of Toronto, Canada

Rapid microbiological methods (RMMs) are essential for state-of-the-art manufacturing efficiency of advanced therapies because traditional testing methods do not provide shorter testing timelines and lower sample volume utilization. Some advanced therapies involve a manufacturing process and release timeline of just a few days making third-party testing impractical. There are ways to incorporate RMMs for QC testing, such as using a nucleic acid amplification technique as an alternative mycoplasma testing system after appropriate product-specific validation in-house. Leveraging available validation data facilitates a regulatory abbreviated qualification plan for RMMs. Several myths impede advanced therapy manufacturers from adopting these RMMs for QC testing. This session is intended to dispel these myths with accurate information and real-world experience.
Chair: Evonne R. Fearnot, MSBME, Roche CustomBiotech, United States Speaker: Alexey Bersenev, MD, PhD, Yale University, United States Sowmya Viswanathan, PhD, University of Toronto, Canada Rapid mic...
09:00 AM - 10:30 AM

Presidential Plenary on Cancer Immunotherapy

Chair:
Catherine Bollard, MBChB, MD, Children's National Medical Center and The George Washington University, United States
Speakers:
Strategies for Improving Efficacy and Safety of CAR T Cells for Cancer Therapy
Stanley Riddell, MD, Fred Hutchinson Cancer Research Center, United States

CAR T cells for Cancer: Progress and Challenges
Crystal Mackall, MD, Stanford University School of Medicine, United States

Allogeneic Cells with Limited GVHD Potential for Cancer Immunotherapy
Robert Negrin, MD, Stanford University, United States

With Cellular ImmunoTherapy in the spotlight with recent FDA approvals, the Presidential Plenary will focus on Immunotherapy where we are fortunate to have attracted leaders in the field to speak including Stanley Riddell, Crystall Mackall and one of ISCT’s Past Presidents, Robert Negrin. These speakers will focus on the development of novel cell therapeutics for cancer. Dr Riddell will speak on "Beyond CD19-CARs for Hematologic Malignancies", Dr Mackall will speak on "Cell Therapies for Solid Tumors" and Dr Negrin will speak on "Targeting the Microenvironment”.
Chair: Catherine Bollard, MBChB, MD, Children's National Medical Center and The George Washington University, United States Speakers: Strategies for Improving Efficacy and Safety of CAR T Cells for ...
09:00 AM - 07:30 PM

Exhibit Hall Open

10:30 AM - 10:45 AM

Corporate Product Theatre Hosted by GE Healthcare - Simplify Upstream and Downstream Cell Therapy Processing with GE’s Sefia™ S-2000 system

Speaker:
Betrand Foucaut, GE Healthcare - Cell Therapy, Switzerland

Cell processing takes place in a complex environment that poses many challenges. GE Healthcare Life Sciences provides solutions that simplify your everyday operations while maintaining flexibility and performance.

Our newest solution allows you to combine multiple workflow steps. The Sefia S-2000 system is suitable for both upstream and downstream processing. Use the same instrument for both – just select the appropriate combination of protocol software and disposable kits. With one software and kit combination you can prepare apheresis products. Set up the software to run volume reduction, platelet removal, and density gradient-based separation alone or automatically in sequence. With another software and kit combination you can perform downstream operations. Customize the sequence of harvesting, washing, and final formulation operations to fit your workflow needs.
Speaker: Betrand Foucaut, GE Healthcare - Cell Therapy, Switzerland Cell processing takes place in a complex environment that poses many challenges. GE Healthcare Life Sciences provides solutions th...
10:30 AM - 11:00 AM

Coffee Break with Exhibits

10:45 AM - 11:00 AM

Corporate Product Theatre Hosted by MilliporeSigma - Expansion of Human Induced Pluripotent Stem Cells in Suspension Culture

Speaker:
Aletta Schnitzler, PhD, MilliporeSigma, United States

The therapeutic potential of human induced pluripotent stem cells (hiPSCs) is explored in a large array of indications, ranging from acute myocardial infarction to diabetes. The inefficiencies in some current differentiation protocols combined with the large numbers of cells recommended for clinical scale tissue engineering warrant the use of systems that are capable of generating large batches of hiPSCs in a controlled manner. In this study, a stirred-tank 3L bioreactor was used for the final expansion of hiPSCs as aggregates in a system providing pH, dissolved oxygen (DO), temperature and agitation control. Overall, a 125-fold expansion was achieved after a 14 day two-step process. The bioreactor-expanded hiPSCs retained expression of the pluripotency markers and formed tissues of each of the three germ layers. Moreover, hiPSCs cultured as aggregates were also directly differentiated to therapeutically-relevant cell types including cardiomyocytes. The results demonstrate the potential of hiPSC production in controlled stirred suspension systems that can support the production of large batches of cells for research and clinical applications.
Speaker: Aletta Schnitzler, PhD, MilliporeSigma, United States The therapeutic potential of human induced pluripotent stem cells (hiPSCs) is explored in a large array of indications, ranging from ac...
11:00 AM - 12:15 PM

Plenary Breakout - Cancer Immunotherapy I

Chair:
Crystal Mackall, MD, Stanford University School of Medicine, United States

Oral Abstract Presentations:

Novel CAR T that Targets MUC1* Not Full-Length MUC1 for Treatment of Solid Tumor Cancers
Cynthia Bamdad, PhDMinerva Biotechnologies, United States

Engineering the Tgfβ Receptor to Enhance the Therapeutic Potential Of Natural Killer Cells As An Immunotherapy For Neuroblastoma
Rachel Burga, BSc, George Washington University, United States

Factors Promoting CD19-Negative Relapses Following CAR19T Cell Therapy
Alla Dolnikov, PhD Sydney Children's Hospital, Australia

T Cells Engineered With T-Cell Antigen Coupler (TAC) Receptors Display Robust Efficacy Against Solid and Liquid Tumor Xenografts In The Absence Of Any Toxicity
Christopher Helsen, PhD,Triumvira, Canada

Simultaneous Dual CAR Expression to Prevent Relapse in Pre-B Acute Lymphoblastic Leukemia
William Lemieux, MSc, CHU Sainte-Justine, Canada
Chair: Crystal Mackall, MD, Stanford University School of Medicine, United States Oral Abstract Presentations: Novel CAR T that Targets MUC1* Not Full-Length MUC1 for Treatment of Solid Tumor Canc...
11:00 AM - 12:15 PM

Plenary Breakout - Cancer Immunotherapy II

Chair:
Robert Negrin, MD, Stanford University School of Medicine, United States

Oral Abstract Presentations:

BCMA-Specific TAC Receptor-Engineered T Cells For Multiple Myeloma
Ksenia Bezverbnaya, BSc, McMaster University, Canada

shRNA-Mediated TCR Knockdown as a Foundation For Allogeneic CAR19 T-Cells Generated By Single-Step Genetic Modification with the piggyBac Transposase
David Bishop, MBBS, Westmead Institute for Medical Research, Australia

Modulation Of Co-Signaling For Improved Ex Vivo Human Antigen-Specific T Cells Generation For Immunotherapy
Shirin Lak PhD, Hospital Maisonneuve Rosemont, Canada

HIV-Specific T Cells Can Be Generated Against Conserved Non-Escaped HIV Epitopes For Use in a Phase I Clinical Trial: Pre-Clinical Validations and Implications for a Cure Strategy For HIV
Shabnum Patel, PhD , The George Washington University, United States

Phase II Study of Haploidentical Stem Cell Transplantation Using Ex Vivo Photodepletion of Donor Lymphocyte Infusions to Eliminate Anti-Host Reactivity Results in Low Relapse Rates and High Survival Rates: Final 2 Year Follow-Up
Denis-Claude Roy, MD,FRCPC, CellCAN, Hopital Maisonneuve-Rosemont, Canada
Chair: Robert Negrin, MD, Stanford University School of Medicine, United States Oral Abstract Presentations: BCMA-Specific TAC Receptor-Engineered T Cells For Multiple Myeloma Ksenia Bezverbnaya, B...
11:00 AM - 12:15 PM

Strategies for Commercialization Track Session 1 - Future Proofing Your Supply Chain; Using the Lessons of the Past to Create Commercially Viable Logistics Platforms for the Future

Chair:
Simon Ellison, MBA, World Courier, United Kingdom
Speakers:

Lessons Learned from Currently Marketed Cell and Gene Therapies - Personal Reflections
Sven Kili, MBChB, GSK Gene Therapy, United Kingdom

The Race Against Time - the Pioneering Supply Chain Behind the Delivery of PROVENGE, A Commercial Immunotherapy
Christina Yi, Dendreon, United States

The Current and Future Landscape for Cell Therapy Deliverability: a CDMO Perspective
Robert Preti, PhD, Hitachi Chemical Advanced Therapeutics Solutions, United States

This session will use MACI, Strimvelis and Provenge as applied examples, then overlay the broader view of a CMO to identify lessons learned and suggest what supply chains of the future should look like.
Highlighting topics such as how to manage:
- Cost by reducing complexity
- Vein to vein journey as a single inter-related system
- Scale up/out by utilizing a logistics platform early in the development pathway
Chair: Simon Ellison, MBA, World Courier, United Kingdom Speakers: Lessons Learned from Currently Marketed Cell and Gene Therapies - Personal Reflections Sven Kili, MBChB, GSK Gene Therapy, United ...
11:00 AM - 12:15 PM

Strategies for Commercialization Track Session 2 - How do Clinical Trial Design and Subsequent Learnings Impact Commercialization?

Chair:
Uri Herzberg, DVM, PhD, MBA, Celularity, United States
Speakers:

Applying Lessons from Early Stage clinical Trials to the Pivotal Phase III MASTERS-2 Ischemic Stroke Study
Robert Willie Mays, PhD, Athersys, United States

Clinical Trial Data and Learnings Supporting Product Commercialization
Steven Fischkoff, MD, WindMIL Therapeutics, United States

Clinical Trial Design Incorporation of Commercial Considerations
Monica Luchi, MD, FACR, MBA, Celularity, United States

As cell therapy is coming of age, it is evident that along the clinical opportunity, successful commercialization could be a major challenge. Hence, the design of clinical trials, in addition to including the endpoints required for registration, must address elements needed for commercialization. The session objectives will be to share the learnings and experiences of seasoned clinical leaders in the field and highlight the challenges that must still be addressed.
Specific objectives will include the following:
- Adequate Clinical design is key to getting to the appropriate patient profile for best response to the product. Specifically as reimbursement is easier for the patient that shows greater clinical benefit. Examples include patients sub-population in stroke, disease stage in diabetes, and disease severity in Crohn’s
- Similarly, side effects, dosing and treatment expectations for physician and patients are important when formulating care givers’ education around efficacy and safety / side effects, prior to commercialization
- Supply chain issues, formulations, efficiencies in every stage of the product life cycle training of health care providers as well as elements yet unknown (what we “do not know that we don’t know”)
Chair: Uri Herzberg, DVM, PhD, MBA, Celularity, United States Speakers: Applying Lessons from Early Stage clinical Trials to the Pivotal Phase III MASTERS-2 Ischemic Stroke Study Robert Willie Mays, ...
11:00 AM - 12:15 PM

Quality and Operations Track Session 1 - Progress in the World of Standards for Cell and Gene Therapies

Chair:
Sowmya Viswanathan, PhD, University Health Network, University of Toronto, Canada
Speakers:
Standards Coordinating Body (SCB): Advancing Standards for Regenerative Medicines
Jiwen Zhang, PhD, The Standards Coordinating Body, United States

Filling Information Gaps to Accelerate Commercialization: Two Case Studies in the Development of ISO Standards for Regenerative Medicine
Claudia Zylberberg, PhD, Akron Biotech, United States

Standards Activities For Manufacturing Characterisation and Control of Cell-based Products
Jonathan Campbell, PhD, LGC, United Kingdom

Standardization for Regenerative Medicine – Japan’s Contribution
Yutaka Yanagita, PhD,Astellas Pharma Inc., Japan
Chair: Sowmya Viswanathan, PhD, University Health Network, University of Toronto, Canada Speakers: Standards Coordinating Body (SCB): Advancing Standards for Regenerative Medicines Jiwen Zhang, PhD,...
11:00 AM - 12:15 PM

Advanced Practice Professionals (APP) Session 1 - Pharmacy Roles in Cellular Therapy & Hematopoietic Stem Cell Transplant

Speakers:
Old Drug-New Tricks: Post-transplant Cyclophosphamide in HSCT Setting: Key Opportunities for Clinical Pharmacists
Rebecca Gonzalez, PharmD, BCOP, Moffitt Cancer Center, United States

Racing Towards the Finish Line with CAR-T cell Therapy: The Pharmacist's Role in the Pit Crew
Christina Bachmeier, PharmD, BCOP, Moffitt Cancer Center, United States
Speakers: Old Drug-New Tricks: Post-transplant Cyclophosphamide in HSCT Setting: Key Opportunities for Clinical Pharmacists Rebecca Gonzalez, PharmD, BCOP, Moffitt Cancer Center, United States Racin...
12:15 PM - 01:45 PM

Lunch with Exhibits

12:30 PM - 12:45 PM

Corporate Product Theatre Hosted by Beckman Coulter Life Science - Get Lean: Streamlining Single Cell Analysis Workflow

Speaker:
Marilyne Levadoux-Martin, PhD, Beckman Coulter Life Science, Canada

While Flow Cytometry has been a core technology for advancing scientific research in cellular therapy, constant innovation is necessary to eliminate inefficient workflows, sources of variability and user introduced errors. Beckman Coulter’s disruptive technologies are uniquely positioned to reshape your workflow without compromising data quality or compliance.
With Beckman Coulter’s Complete Workflow Solution you can have:
• Consistent assay results by using our proven dried down antibody panels with DURAClone Reagents
• Outstanding sensitivity and electronic record management with the CytoFLEX Flow Cytometer Platform
• Traceable analysis and reporting through user management and LIS integration with Kaluza Analysis Software
• Automated staining protocol, from sample preparation through analysis, by directly integrating a CytoFLEX analyzer with a Biomek i-Series Liquid Handler
Speaker: Marilyne Levadoux-Martin, PhD, Beckman Coulter Life Science, Canada While Flow Cytometry has been a core technology for advancing scientific research in cellular therapy, constant innovatio...
12:30 PM - 01:30 PM

Corporate Tutorial Hosted by BD Biosciences - Improving Cell Therapy Production with Standardized and Consistent Flow Cytometry Assays and Sterile Fluorescence Activated Cell Sorting

Speaker:
Scott Bornheimer, PhD, BD Biosciences, United States

Cells are frequently characterized during cell therapy production to track important subsets for research, process monitoring and QC for final release. Standardized flow cytometry assays that perform consistently across time, operators, instruments and sites are critical in this regard. BD will present technology and methods to meet these needs, based on IVD systems adapted for use in self-validated GMP cell processing. Consistency in cell starting materials, including isolation of target cells and removal of non-beneficial cells, is also important for consistent cell therapy production. Fluorescence activated cell sorting (FACS) can isolate cell subsets based on quantitative expression of multiple markers with excellent purity, viability and yield. We will highlight the current use of FACS in GMP cell processing applications and present technologies for sterile, closed and easy-to-use FACS to facilitate future use.
Speaker: Scott Bornheimer, PhD, BD Biosciences, United States Cells are frequently characterized during cell therapy production to track important subsets for research, process monitoring and QC for ...
12:45 PM - 01:00 PM

Corporate Product Theatre Hosted by CCRM - Developing Closed and Integrated Processes for Cell Therapy Manufacturing

Speaker:
Aaron Dulgar-Tulloch, PhD, BridGE, GE Healthcare, Canada

As cell and gene therapies advance towards market approvals, there is a need for robust manufacturing solutions that are suitable for commercial scale production. Many cell manufacturing processes are currently being carried out with manual handling steps in an open manner, putting them at high risk for process variability. Our team is developing processes suitable for commercial scale manufacturing for both scale-up and scale-out cell production. As an example, we have demonstrated closure and integration of a T-cell production process by: automating the processing of input fresh and frozen apheresis units; closing both small- and large-scale T-cell culture using gas permeable bags and the Xuri™ W25 Cell Expansion System; and integrating cell culture and downstream harvest, wash, and formulation using the Sefia™ Cell Processing System. We have demonstrated a similar conversion from manual tissue culture to a closed and integrated bioreactor-based manufacturing of scaled-up pluripotent stem cells in a 10 L stirred tank reactor. With these strategies we are providing cell manufacturing solutions that can be readily adapted to a variety of customer-specific processes.
Speaker: Aaron Dulgar-Tulloch, PhD, BridGE, GE Healthcare, Canada As cell and gene therapies advance towards market approvals, there is a need for robust manufacturing solutions that are suitable fo...
01:00 PM - 01:15 PM

Corporate Product Theatre Hosted by Miltenyi Biotec - Automated clinical-scale manufacturing of gene-engineered T cells using the CliniMACS Prodigy

Speaker:
Michael Papadimitrious, Miltenyi Biotec, Germany

For over 25 years, Miltenyi Biotec has been providing technologies spanning the entire workflow of cell therapy, with solutions applicable from basic research to GMP manufacturing. The CliniMACS Prodigy is a cell manufacturing platform – one instrument that automates the handling steps in a single-use disposable tubing set. The example of a chimeric antigen receptor (CAR) T cell manufacturing process will be presented, demonstrating an integrated solution for cell selection, activation, transduction, and expansion.
Speaker: Michael Papadimitrious, Miltenyi Biotec, Germany For over 25 years, Miltenyi Biotec has been providing technologies spanning the entire workflow of cell therapy, with solutions applicable f...
01:15 PM - 01:30 PM

Corporate Product Theatre Hosted by Be The Match BioTherapies - Auditing, Onboarding, and Managing Apheresis and Marrow Collection Networks to Support Late-Stage Clinical Study and Commercial Launch of Cell and Gene Therapies

Speaker:
Chris McClain, Be The Match BioTherapies, United States

One of the major obstacles faced by developers of emerging cell and gene therapies is providing reliable, nationwide access to apheresis and marrow collection centers. Over the course of its 30 year operating history, the National Marrow Donor Program (NMDP)/Be The Match has built and managed a national network of more than 80 apheresis centers and 70 collection centers. Through that network, NMDP/Be The Match has managed more than 74,000 stem cell harvests. Be The Match BioTherapies, a subsidiary of NMDP/Be The Match, will describe solutions that it provides to developers of cell and gene therapies to support late-stage clinical development and commercialization of therapies that rely on stem cell harvest for therapeutic starting material.
Speaker: Chris McClain, Be The Match BioTherapies, United States One of the major obstacles faced by developers of emerging cell and gene therapies is providing reliable, nationwide access to apheres...
01:45 PM - 03:15 PM

Plenary Session 2 - Mesenchyme Biology and Translational Use

Chair:
Jacques Galipeau, MD, University of Wisconsin-Madison, United States
Speakers:
What Do We Really Know About MSC-Mediated Immune Regulation?
Martin Hoogduijn, PhD, Erasmus MC, University Medical Center Rotterdam, Netherlands

Mesenchymal Stem Cells: Native Identity, Diversity, and Culture Induced Alterations
Bruno Péault, PhD, University of Edinburgh, Scotland, and University of California at Los Angeles, United States

Gene-enhanced Angiogenic Cell Therapy for Cardiovascular Diseases: an Update on the ENACT-AMI and SAPPHIRE Clinical Trials
Duncan Stewart, MD, The Ottawa Hospital Research Institute, Canada

Mesenchymal stromal cells and related culture adapted cell pharmaceuticals have met important regulatory milestones in the past year with both EMA and likely FDA approvals for clinical use in the making. As the filed strives to develop MSC v2.0, understanding of ontogeny, biology and mechanism of action will inform on how best to improve potency and use of these cell products in expanded clinical applications. The remarkable observation that killed MSCs effect a response from treated mice suggests important unrecognized cell-function autonomous effects of MSC therapy. A point to be addressed by Dr Hoogduijn. On a more basic biology perspective, the deciphering of pericyte form and function in vivo provides important insights on recruitment of endogenous progenitors to affect an injury response. Dr Péault will highlight the biology of vascular fraction-derived MSC-like pericytes and how this knowledge can inform development of this tissue source for translational use. In closing, the translational use of gene engineered endothelial cells foreshadows the development of MSC engineered counterparts, and the clinical lessons learnt and shared by Dr Stewart will provide guidance to the field as well.
Chair: Jacques Galipeau, MD, University of Wisconsin-Madison, United States Speakers: What Do We Really Know About MSC-Mediated Immune Regulation? Martin Hoogduijn, PhD, Erasmus MC, University Medi...
03:15 PM - 03:45 PM

Corporate Product Theatre Hosted by Irvine Scientific - Chemically-Defined Culture Media for Advancing Cell-Based Immunotherapy Technology

Speaker:
Jessie Ni, PhD, Irvine Scientific, United States

Cell-based immunotherapy applications are widely captivating today due to their clinical potential to become life-saving therapies for cancer patients. Generation of sufficient, desired cell populations is an essential task for the successful development of cell-based immunotherapies, which require an effective, scalable, and consistent ex vivo process. A suitable chemically-defined (CD), animal-component-free (ACF) cells basal media for major immune cells, such as T-cells and natural killer (NK) cells, would significantly foster the establishment of such a process. By applying spent media analysis and the quality by design (QbD) approach, we examined the effects of various key media compositions such as amino acids, vitamins, minerals, and lipids on activated human peripheral blood-derived T-cell or NK cell expansion. The results from our studies were used to develop CD, ACF basal expansion media for desired T-cell and NK cell populations that are comparable to media containing serum, and further indicate the need to develop cells- and application-specific basal media to establish an optimal production process for a desired/targeted immune cell-based therapy under CD conditions.
Speaker: Jessie Ni, PhD, Irvine Scientific, United States Cell-based immunotherapy applications are widely captivating today due to their clinical potential to become life-saving therapies for cancer...
03:15 PM - 03:45 PM

Coffee Break with Exhibits

03:45 PM - 05:00 PM

Plenary Breakout 1 - Endothelial Progenitor Cells

Chair:
Duncan Stewart, MD, The Ottawa Hospital Research Institute, Canada
Speaker:
EPC-based Therapies for Neonatal Pulmonary Hypertension
Bernard Thébaud, MD, PhD, The Ottawa Hospital Research Institute, Canada

Oral Abstract Presentations:
A Phase I Trial Of Mesenchymal Stem Cells Transfected With A Plasmid Secreting Interferon Beta In Advanced Ovarian Cancer
Amanda Olson, MD, The University of Texas MD Anderson Cancer Center, United States

Cellular Therapy for Open Angle Glaucoma With Emphasis On Mesenchymal Stem Cells Secretome Induced Trabecular Meshwork Regeneration
Christian Tebid, MSc, crHMR-UdeM, Canada

Potential Anti-Inflammatory Mechanism of Action of Mesenchymal Stromal Cells In Osteoarthritis Patients Results In Overall Improvement In Pain and Symptoms
Sowmya Viswanathan, PhD, University Health Network, University of Toronto, Canada
Chair: Duncan Stewart, MD, The Ottawa Hospital Research Institute, Canada Speaker: EPC-based Therapies for Neonatal Pulmonary Hypertension Bernard Thébaud, MD, PhD, The Ottawa Hospital Research Ins...
03:45 PM - 05:00 PM

Plenary Breakout 2 - Translational Use of Mesenchymal Stem Cells

Chair:
Bruno Péault, PhD, University of Edinburgh, Scotland, and University of California at Los Angeles, United States
Speakers:
Mesenchymal Stromal Cells – Addressing the Dissonance of Pre-Clinical Science and Clinical Development
Jacques Galipeau, MD, University of Wisconsin-Madison, United States

Mesenchymal Stromal Cells: Mechanisms and Safety for ARDS
Michael Matthay, MD , University of California San Francisco, United States

A Phase 3 Single-Arm, Prospective Study of Remestemcel-L, Ex-Vivo, Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Steroid Refractory Acute GVHD in Pediatric Patients
Joanne Kurtzberg, MD, Duke University Medical Center, United States

Oral Abstract Presentation:
Warming Events and MSC Cryopreservation: Observations That Could Lead to the Development of Surrogate Tests for Immunosuppressive Activity
Renée Bazin, PhD,Héma-Québec, Canada
Chair: Bruno Péault, PhD, University of Edinburgh, Scotland, and University of California at Los Angeles, United States Speakers: Mesenchymal Stromal Cells – Addressing the Dissonance of Pre-Clinical...
03:45 PM - 05:00 PM

Quality and Operations Track Session 2 - Applying Change Within Controlled Systems and Processes

Chair:
Rosemarie Bell, BAppSc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia
Speakers:
Defining the Change
Steven Keizer, BSc, MBA, CCRM, Canada

Regulatory Expectations on Managing Change during R&D through Clinical Product Manufacturing
Bangon Longsomboon, MA, Interdisciplinary Stem Cell Institute, United States

Management of a Cell Expansion Platform Change During Clinical Trial Manufacturing
Gerry McKiernan, BSc, Cell Therapies PTY Ltd, Australia

As technologies evolve and new methods of improved manufacturing are developed, we need to be able to implement these new process changes into our clinical manufacturing to deliver the best possible outcome for patients and not be confined to the processes that were deployed in early phase development if they become outdated. Leading on from the what and the why, this presentation will discuss how you manage a major change to a new cell expansion platform during a phase III clinical trial, the wide range of impacts you need to consider and how you manage implementation to maintain a state of control.
Chair: Rosemarie Bell, BAppSc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia Speakers: Defining the Change Steven Keizer, BSc, MBA, CCRM, Canada Regulatory Expectations o...
03:45 PM - 05:00 PM

Advanced Practice Professionals (APP) Session 2 - CAR T Immunotherapy: Science and Logistics

Speakers:
Supporting Investigator Initiated Trials
Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Baylor College of Medicine, United States

Understanding and Managing Toxicities of CAR T Cells
Stanley Riddell, MD, Fred Hutchinson Cancer Research Center, United States
Speakers: Supporting Investigator Initiated Trials Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Baylor College of Medicine, United States Understanding and Managing Toxicities of CAR T Cells Stanley R...
03:45 PM - 05:00 PM

Scientific Area of Focus Session - Stem Cell Biology and Expansion

Chair:
John Rasko, AO, BSc(Med), MBBS(Hons), PhD, MAICD, FFSc(RCPA), FRCPA, FRACP, FAHMS, Royal Prince Alfred Hospital, Australia
Speakers:
Universal Donor Cord Blood Cell Therapies: Expanding Clinical Access and Improving Clinical Feasibility
Colleen Delaney, MD, MSc, Nohla Therapeutics, United States

Cellular engineering with UM171: From Bedside to Benchtop
Guy Savageau, MD, PhD , University of Montreal, Canada

Vascular Niche Signals for Expansion of Engraftable Stem Cells
Shahin Rafii, MD, Weill Cornell Medical College, United States
Chair: John Rasko, AO, BSc(Med), MBBS(Hons), PhD, MAICD, FFSc(RCPA), FRCPA, FRACP, FAHMS, Royal Prince Alfred Hospital, Australia Speakers: Universal Donor Cord Blood Cell Therapies: Expanding Clinica...
03:45 PM - 05:00 PM

ISCT Chief Scientific Officer Abstract Showcase

Chair: Daniel J. Weiss, MD, PhD, University of Vermont School of Medicine, United States

Oral Abstract Presentations:

Identification Of Differential Expression Phenotypes Of CD133+ Stem Cells In Acute and Chronic Myocardial Infarct Patients And Specific Expression Pathways Underpinning Therapeutic Responsiveness In Regenerative Therapy
Shant Der Sarkissian, PhD, MBA , Université de Montréal / CRCHUM, Canada

Developing Of Tissue Engineered Auricular Pavilion Able To Induce Host Perichondrium And Subdermal Tissue Integration. Preclinical Feasibility And Safety Study
Gustavo Moviglia, MD, Maimonides University, Argentina

Large-Scale, Single Cell RNA Sequencing Defines Novel Cellular Subsets Required For Cardiac Repair
Robert Nordon , MBBs, PhD, University of New South Wales, Australia

Designing Microtissue Bioassemblies For Skeletal Regeneration: Healing Critical Size Long Bone Defects
Ioannis Papantoniou, PhD, KU Leven, Belgium

Combining Electrospinning And 3D Printing For A New Generation Of Nerve Guides
Joel Simpson-Edin, MSC, Uppsala University, Sweden
Chair: Daniel J. Weiss, MD, PhD, University of Vermont School of Medicine, United States Oral Abstract Presentations: Identification Of Differential Expression Phenotypes Of CD133+ Stem Cells In Ac...
05:00 PM - 06:30 PM

Advanced Practice Professionals (APP) Session 3 - What's New in Cord Blood Transplantation for Hematologic Malignancies

Speakers:
Intermediate Intensity Double Unit CBT as a Platform for Cellular Therapy
Juliet Barker, MBBS, FRACP, Memorial Sloan Kettering Cancer Center, United States

CBT: Day to day management of CBT recipients: Graft infusion and specialized pharmacology
Valkal Bhatt, PharmD , Memorial Sloan Kettering Cancer Center, United States
Speakers: Intermediate Intensity Double Unit CBT as a Platform for Cellular Therapy Juliet Barker, MBBS, FRACP, Memorial Sloan Kettering Cancer Center, United States CBT: Day to day management of C...
05:15 PM - 07:15 PM

Corporate Symposium Hosted by Novartis Oncology - What Makes CARs Work? — A Look Under the Hood

Reception begins at 17:15. Catering provided courtesy of Novartis.

Chair:
Marcela Maus, MD, PhD, Massachusetts General Hospital, United States
Speakers:
John Bell, PhD, The Ottawa Hospital, University of Ottawa, Canada
Abhijit Chakraborty, PhD Novartis Pharmaceuticals, United States
Marcela Maus, MD, PhD, Massachusetts General Hospital, United States
Bijal Shah, MD, Moffitt Cancer Center, United States

This satellite symposium will serve as a forum in which the panelists will discuss key attributes of emerging chimeric antigen receptor (CAR) T-cell therapies, including
•Contribution of various CAR domains to the efficacy and safety of CAR T-cell therapies
•Current understanding of CAR T-cell cellular kinetics in hematologic malignancies
•Role of lymphodepleting chemotherapy, mechanisms of resistance, and predictors of response, relapse, and safety
Reception begins at 17:15. Catering provided courtesy of Novartis. Chair: Marcela Maus, MD, PhD, Massachusetts General Hospital, United States Speakers: John Bell, PhD, The Ottawa Hospital, Univer...
06:00 PM - 07:30 PM

Poster Session 1

07:00 PM - 09:00 PM

ISCT Early Stage Professionals Networking Reception

By Invitation Only
By Invitation Only
07:30 PM - 09:30 PM

Friday, May 4
06:30 AM - 07:30 AM

ISCT 2018 5K Run the River Event - Registration Required

Meet at the Embassy Suites hotel at 6:15 prior to run start at 6:30.
Meet at the Embassy Suites hotel at 6:15 prior to run start at 6:30.
07:00 AM - 05:00 PM

Speaker Services Open

07:00 AM - 06:00 PM

Registration Open

07:30 AM - 08:30 AM

Hot Topic Session 3 - Immunologic Sculpting of T Cell and Stem Cell Grafts

Chair:
Denis-Claude Roy, MD, FRCPC, University of Montreal, Canada
Speakers:
Haploidentical Transplant in the Era of Graft Engineering: What's New in Pediatrics
Alice Bertaina, MD, PhD, Stanford School of Medicine, United States

Anti-infection and Anti-leukemia Donor T Cells Without GVHD: "Mission Possible"
Denis-Claude Roy, MD, FRCPC, University of Montreal, Canada

Advances in CAR T Cell Therapy: the CD19 CAR Paradigm
Isabelle Rivière, PhD, Memorial Sloan Kettering Cancer Center, United States
Chair: Denis-Claude Roy, MD, FRCPC, University of Montreal, Canada Speakers: Haploidentical Transplant in the Era of Graft Engineering: What's New in Pediatrics Alice Bertaina, MD, PhD, Stanford Sch...
07:30 AM - 08:30 AM

Hot Topic Session 4 - Oncolytic Viruses as Cancer Therapeutics

Co-Chairs:
John Bell, PhD, The Ottawa Hospital, University of Ottawa, Canada
Khalid Shah, MS, PhD, Harvard Medical School, United States

Speakers:
John Bell, PhD, The Ottawa Hospital, University of Ottawa, Canada

Oncolytic Virus Engineered Cell Based Therapeutics for Cancer
Khalid Shah, MS, PhD, Harvard Medical School, United States

Oral Abstract Presentation:
Functional Annotation And Identification Of Putative Drug Target In Strain Ankara
Yashbir Singh
Co-Chairs: John Bell, PhD, The Ottawa Hospital, University of Ottawa, Canada Khalid Shah, MS, PhD, Harvard Medical School, United States Speakers: John Bell, PhD, The Ottawa Hospital, University of ...
07:30 AM - 08:30 AM

Corporate Breakfast Tutorial Hosted by the American Society of Mechanical Engineers (ASME) - Training the Engineering Workforce for Cellular Therapy Manufacturing

Speakers:
Brian Behnke , ASME Learning and Development, United States

Leveraging the talents of skilled engineers working in the cell therapy space remains a challenge for many biopharmaceutical companies. Recognizing that foundational learning and standardization are critical success factors, the American Society of Mechanical Engineers (ASME) has developed a tool to begin filling this need: an introductory cellular therapy manufacturing eLearning course for engineers. Preview this solution that you can use to help educate your current engineering team or onboard your future workforce. Using real world examples and case studies, the course will cover end-to-end cell therapy manufacturing, emphasizing the engineering problems and processes and the critical roles engineers play.
Speakers: Brian Behnke , ASME Learning and Development, United States Leveraging the talents of skilled engineers working in the cell therapy space remains a challenge for many biopharmaceutical comp...
08:45 AM - 10:15 AM

Plenary Session 3 - Exosomes

Chair:
Daniel J. Weiss, MD, PhD, University of Vermont School of Medicine, United States
Speakers:
Manufacturing and Characterization of MSC-EVs for Clinical Testing
Eva Rohde, MD, Paracelsus Medical University, Austria

Exsomes- CAP-2003
Linda Marban, PhD, Capricor, United States

MSC Exosomes and Small EVs: a Comparative Review
Sai-Kiang Lim, PhD, A*STAR Institute of Medical Biology, Singapore
Chair: Daniel J. Weiss, MD, PhD, University of Vermont School of Medicine, United States Speakers: Manufacturing and Characterization of MSC-EVs for Clinical Testing Eva Rohde, MD, Paracelsus Medica...
09:00 AM - 06:30 PM

Exhibit Hall Open

10:15 AM - 10:45 AM

Corporate Product Theatre Hosted by BioLamina - CTG BioLamininTM 521 – a Biologically Relevant Culture Matrix, Enabling Pre-clinical Research Protocols to be Translated and Used for Clinical Trials

Speaker:
Therese Kallur, PhD, BioLamina, Sweden

As an extension of our portfolio of hr laminin cell culture substrates, we now offer a cell therapy grade (CTG) laminin-521 matrix (BioLamininTM 521) for use in research and manufacturing of Cell, Gene, or Tissue-Based Products.
CTG BioLaminin 521 is xeno-free and provides a defined surface for feeder-free culture of human pluripotent stem cells (ESC and iPSC), MSCs, most anchorage-dependent progenitors and differentiated cells such as RPE, hepatocytes, cardiomyocytes and neurons.
CTG BioLaminin 521 recreates a biologically relevant milieu in vitro, promoting high survival and robust single-cell or colony expansion of human pluripotent stem cells, and subsequent cell lineage specification. The cells grow with maintained pluripotency, genetic integrity in a homogeneous monolayer, easy to monitor and maintain.
The substrate is robust, flexible and compliant with any culture medium and protocol. It allows an operator-independent culture maintenance and reliable, standardized protocols which can easily be adapted to automation platforms.
CTG BioLaminin 521 is produced according to FDA and EMEA guidelines (USP <1043>) and is aimed to be an ancillary material in the manufacturing cell products for therapeutic use. It is designed to aid users in the qualification process of raw material.
Speaker: Therese Kallur, PhD, BioLamina, Sweden As an extension of our portfolio of hr laminin cell culture substrates, we now offer a cell therapy grade (CTG) laminin-521 matrix (BioLamininTM 521)...
10:15 AM - 10:45 AM

Coffee Break with Exhibits

10:45 AM - 12:15 PM

Plenary Breakout 1 - Exosome Technology

Chair:
Eva Rohde, MD, Paracelsus Medical University, Austria
Speakers:
Exosome Engineering for Delivery of Therapeutic Proteins: Principles and Applications
Chulhee Choi, MD, PhD, KAIST, Korea

Production of Functionally Bioactive EVs from an Immortalized Human Neural Stem Cell Line and their Therapeutic Applications
Randolph Corteling, PhD, ReNeuron, United Kingdom

Oral Abstract Presentations:
Licensing Increases The Quantity And Immunomodulatory Cargo Of Mesenchymal Stromal Cell Exosomes
Anastasia Cheng, MSc, McGill University, Canada

NK Cell Expansion With Ex21-Exosomes
Alicja Copik, PhD, UCF College of Medicine, United States
Chair: Eva Rohde, MD, Paracelsus Medical University, Austria Speakers: Exosome Engineering for Delivery of Therapeutic Proteins: Principles and Applications Chulhee Choi, MD, PhD, KAIST, Korea Prod...
10:45 AM - 12:15 PM

Plenary Breakout 2 - Lessons Learned During CAR T Commercial Roll Out at Clinical Sites

Chair:
Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States
Speakers:
Preparing Sites to Enter the Commercial CAR T Setting: Focus on Clinical Readiness
Solveig Ericson, MD, PhD, Novartis, United States

Best Practices - Supporting Logistical Accounts Transitions for Commercial CAR T Distribution
Diane Parks, MBA Kite Pharma Inc, United States

Growing Pains in an Academic CAR T Program, Including Transition from Clinical Trials to Commercial Products
Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States
Chair: Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States Speakers: Preparing Sites to Enter the Commercial CAR T Setting: Focus on Clinical Readiness Solveig Ericson, MD, PhD, No...
10:45 AM - 12:15 PM

Strategies for Commercialization Track Session 3 - Tools and Automation Solutions for Accelerating Cell and Gene Therapy Development

Chair:
Julie Murrell, PhD, MilliporeSigma, United States
Speakers:
Qasim Rafiq, PhD, University College London, United Kingdom

Technologies for Radically Reducing Development Timelines of hMSC-based Therapeutic Products
Jon Rowley, PhD, RoosterBio, United States

Strategies and Methodologies for Selecting and Implementing New Technologies for Cell Therapy Processes
Greg Russotti, PhD, Celgene, United States

With recent approvals, the urgency for improvements in Cell and Gene Therapy Development are even more immediate than in the past. Here, we have perspectives from academia, biotech and suppliers:
- What are the drivers for change?
- What sort of technologies and innovations do we use to develop new products and reagents?
- How can we implement these tools into the development cycle?
A panel discussion will follow the speaker presentations and will be open to audience questions.
Chair: Julie Murrell, PhD, MilliporeSigma, United States Speakers: Qasim Rafiq, PhD, University College London, United Kingdom Technologies for Radically Reducing Development Timelines of hMSC-based...
10:45 AM - 12:15 PM

Quality and Operations Track Session 3 - Regulation of MTMMS and ATMPS: Manufacturing, Non-Clinical, and Clinical Perspectives

Chair:
Shirley Bartido, PhD, MBA, Cellectis, United States

Speakers:
GMP Manufacturing Considerations for Advanced Therapeutic Cellular Products
Shirley Bartido, PhD, MBA, Cellectis, United States

Non-clinical Considerations in the Development of Cell and Gene Therapies
Julianne Smith, PhD, Cellectis, France

Regulatory Challenges and Opportunities for Clinical Development of Cell and Gene Therapies
Elena Spanjaard, PhD, Pfizer, United States

Session aims to describe the regulated elements required by authorities in the preclinical assessment and manufacture of a reproducible, safe and efficacious product for its intended clinical use and method of clinical delivery.
Chair: Shirley Bartido, PhD, MBA, Cellectis, United States Speakers: GMP Manufacturing Considerations for Advanced Therapeutic Cellular Products Shirley Bartido, PhD, MBA, Cellectis, United States ...
10:45 AM - 12:15 PM

Scientific Area of Focus - NK Cell Therapy: a New Frontier Revisited

Chair:
Armand Keating, MD, University of Toronto, Canada
Speakers:
Novel Strategies to Activate NK Cells to Treat Cancer
Sarah Cooley, MD, University of Minnesota Department of Medicine, United States

Armored CAR NK Cells for the Treatment of Lymphoid Malignancies
Katy Rezvani, MD, PhD, , MD Anderson Cancer Center, United States

Cell Therapy with Permanent NK Cell Lines
Armand Keating, MD, University of Toronto, Canada
Chair: Armand Keating, MD, University of Toronto, Canada Speakers: Novel Strategies to Activate NK Cells to Treat Cancer Sarah Cooley, MD, University of Minnesota Department of Medicine, United States...
10:45 AM - 12:15 PM

ISCT Presidential Task Force Session

Chair:
Massimo Dominici, MD, University of Medina and Reggio Emilia, Italy
Speakers:
Massimo Dominici, MD, University of Medina and Reggio Emilia, Italy

FDA Perspectives on Stem Cell Therapies
Peter Marks, MD, PhD, US Food and Drug Administration, United States

Regulation of Autologous Cell Therapies in Canada
Nadine Kolas, PhD , Health Canada, Canada

A Physician's Perspective on Unproven Cell Therapies: How Hope Has Become a Commodity
Harold Atkins, MD, Ottawa Hospital Research Institute, Canada

Non Myeloblative HSCT: A Patient's Perspective
Keith Moore, , Canada

Ethical Considerations in Communication Surrounding Cellular Therapies
Aaron Levine, PhD, Georgia Tech, United States

The ISCT Presidential Task Force (PTF) on the Use of Unproven Cellular Therapies (UCT) is a prominent group of academic, industry, bioethical and regulatory experts in the cell therapy field. This session is focused on understanding patient-focused communications surrounding unproven cellular therapies and the role of scientific/medical societies, health-care stakeholders, and regulators to promote rigorous research and development of safe and effective cell therapies. The PTF-UCT will share the results of a global collaborative survey that involved many scientific societies and organizations on how UCT are impacting several fields of biomedical research and clinical practice. Representatives from FDA and Health Canada – two key regulatory bodies -- will then deliver presentations on current regulatory activities also related to unapproved, and potentially unethical, cellular therapies. This session aims to integrate considerations of regulatory issues with patients’ needs. For this reason it will also feature a physician perspective and a patient success story from a regulated clinical trial in a neurological condition, such as multiple sclerosis. Finally, relevant bioethical considerations, focusing on communication practices among various cell therapy stakeholders, will be discussed to highlight how ethical considerations can be integrated into the development of cell-based therapeutics as part of a patient-centric unified process.
Chair: Massimo Dominici, MD, University of Medina and Reggio Emilia, Italy Speakers: Massimo Dominici, MD, University of Medina and Reggio Emilia, Italy FDA Perspectives on Stem Cell Therapies Pe...
12:15 PM - 01:45 PM

Lunch with Exhibits

12:30 PM - 01:00 PM

Corporate Product Theatre Hosted by Cook Regentec - Different Makes The Difference: The CellSeal® System

Speaker:
Sean Werner, PhD, Cook Regentec, United States

The CellSeal® System aims to fill the industry’s need for a cryogenic storage and recovery process. For storing, it is a solution for leakage, breakage, and contamination in cryogenic storage. For packaging, it is a solution for consistent small-batch sealing and automated large-batch filling and sealing. For thawing, it is a solution for standardizing the thawing process through customizable profiles. Through these solutions, we will demonstrate how the CellSeal platform leads to significant gains in processing scale-up, quality, and standardization for cell and gene therapies.
Speaker: Sean Werner, PhD, Cook Regentec, United States The CellSeal® System aims to fill the industry’s need for a cryogenic storage and recovery process. For storing, it is a solution for leakage,...
12:30 PM - 01:30 PM

Corporate Tutorial Hosted by Macopharma - The First Clinical Grade Virally Inactivated Human Platelet Lysate, MultiPL’i: a Powerful, Safe and Standardized Alternative to FBS to Expand Cell Therapy Products

Chair:
Bruno Delorme, PhD, Macopharma Biotherapy, France

Speakers:
Bruno Delorme, PhD, Macopharma Biotherapy, France
Andrew Finnerty, Centre for Manufacturing (CCMI), Ireland
Sabrina Viau, PhD, Macopharma Biotherapy, France
Jorge Burns, PhD, University Politehnica of Bucharest

Human platelet lysate (hPL) represents a powerful xeno-free alternative to fetal bovine serum (FBS) for human mesenchymal stem cell (hMSC) expansion. However, the characterization and the batch-to-batch standardization of such products still remain a challenge. In addition, the general chapter 5.2.12 of the European Pharmacopeia requires the addition of a step of viral inactivation during the production process of such raw material of biological origin used for cell-based and gene therapy medicinal products.
We will report the extensive characterization and document the robust standardization of our different clinical grade hPL products (MultiPL’), including growth factors (GF) contents, multiplex assay and biochemical and proteomic analysis. Data of comparison of hMSCs cultured with either FBS or MultiPL’ will be presented (expansion, morphology, membrane marker expression, potential of differentiation and immunosuppressive properties). We will highlight some key characteristics of hMSC cultured in hPL. Importantly, we will also show that the use of standardized hPL improves the standardization of biological features of hMSCs. Finally, the efficacy of the gamma irradiation to inactivate a broad range of viruses in MultiPL’ will be documented. The impact of the gamma irradiation on MultiPL’ and the biological features of hMSCs cultured in MultiPL’i will be described.
Chair: Bruno Delorme, PhD, Macopharma Biotherapy, France Speakers: Bruno Delorme, PhD, Macopharma Biotherapy, France Andrew Finnerty, Centre for Manufacturing (CCMI), Ireland Sabrina Viau, PhD, Macop...
01:00 PM - 01:15 PM

Corporate Product Theatre Hosted by CellCAN Regenerative Medicine and Cell Therapy Network - More than 4800 days of a Wonderful Second Life! William Brock’s Inspiring Story!

Speakers:
William Brock, Davies Ward Phillips & Vineberg LLP and CellCAN Board of Directors, Canada
Craig Hasilo, PhD Candidate, CellCAN, Canada

Diagnosed in September 2004 with acute myelogenous leukemia, the Montreal lawyer William Brock thought he had just received a death sentence with very little time left to live. Within his own city of Montreal, he discovered a true gem in advanced healthcare: a centre where stem cell transplantation and cell therapy were provided at the highest standards. Shocked to learn that such cutting edge technology existed in Canada, Mr. Brock sought a referral and immediate treatment that would forever change his life and that of everyone around him.
Speakers: William Brock, Davies Ward Phillips & Vineberg LLP and CellCAN Board of Directors, Canada Craig Hasilo, PhD Candidate, CellCAN, Canada Diagnosed in September 2004 with acute myelogenous le...
01:15 PM - 01:30 PM

Corporate Product Theatre Hosted by CellCAN Regenerative Medicine and Cell Therapy Network - How Well Do You Know Canada's Cell & Gene Manufacturing Landscape?

Speaker:
Craig Hasilo, PhD Candidate, CellCAN, Canada

Canada is known for its excellence in research, outstanding publication record on cell & gene therapies and our collaborative culture. Our thriving RMCT ecosystem is on the brink of a major expansion. At the core of our strengths lie our expertise in cell & gene therapy manufacturing for early and later-phase multicenter clinical trials or commercialization efforts. But how well do you know what Canada has to offer in terms of manufacturing capacity? To what extent do Canadian cell & gene therapy manufacturing centres collaborate?

In this interactive session you will learn more about CellCAN’s cell & gene manufacturing facilities and transversal cores, and how they share best practices through a collaborative online platform to improve the quality, safety and feasibility of cell & gene therapies. This unique collaboration will ultimately increase the capacity to rapidly and effectively migrate innovative treatment concepts into standard clinical practice.
Speaker: Craig Hasilo, PhD Candidate, CellCAN, Canada Canada is known for its excellence in research, outstanding publication record on cell & gene therapies and our collaborative culture. Our thrivi...
01:45 PM - 03:15 PM

Plenary Session 4 - Reimagining Cancer Care and Delivering on the Promise of CAR-T Therapies

Chair:
Miguel Forte, MD, PhD, Zelluna Immunotherapy, Belgium
Speakers:
CAR T: Current Status and Perspectives from the Academic Lens
Carl June, MD, University of Pennsylvania, United States

Reimagining Cancer Care and Delivering Kymriah to Patients
Pascal Touchon, DVM, MBA Novartis, United States

Making History - Commercial Launch of CAR-T Therapy
Diane Parks, MBA, Kite Pharma Inc, United States

The session will cover the recent developments and success with CAR-T therapies.
The vision of translation from academia to industrial development and eventual market launch will be discussed focussed on the recent market launches. This will represent an opportunity to give the audience, both the academics as well as the industry, to hear and discuss the different angles and models that lead to the CAR-T success stories. These examples will be looked with an aim to the future as lessons learned and the impact on the field of cell and gene therapy and the commitment of different stakeholders to this emerging value adding immunotherapy approach to unmet medical needs.
Chair: Miguel Forte, MD, PhD, Zelluna Immunotherapy, Belgium Speakers: CAR T: Current Status and Perspectives from the Academic Lens Carl June, MD, University of Pennsylvania, United States Reimag...
03:15 PM - 03:30 PM

Corporate Product Theatre Hosted By AventaCell BioMedical Corp. Ltd - What's New with Human Platelet Lysate?

Speaker:
William Milligan, AventaCell BioMedical Corp., Ltd, Canada, United States, Taiwan

Human platelet lysates are now broadly used to replace FBS and early serum-free supplements for the isolation, expansion and production of multiple cells including; AD MSC, UC MSC, BM MSC, CIK, NK, dendritic cells, and primary tissue cell lines. AventaCell’s UltraGROTM line offers hPL and fibrinogen-depleted hPL (FD hPL) options in both RUO and GMP grade supplements. UltraGROTM GMP products are used in cell therapy research and clinical development. Pathogen reduction will soon be available in GMP UltraGROTM products to better meet regulatory requirements and expand clinical development applications. UltraGROTM supplements have also been shown to effectively replace FBS and serum-free supplements for MSC expansion in bioreactors including; micro-carrier spinner flask bioreactors and the Quantum bioreactor (2016 & 2017 publications). We’re also introducing UltraKURE – NK this year, our first hPL-based kit for NK cell expansion. For more product information, samples, and new developments, please come visit us in Booth #320.
Speaker: William Milligan, AventaCell BioMedical Corp., Ltd, Canada, United States, Taiwan Human platelet lysates are now broadly used to replace FBS and early serum-free supplements for the isolatio...
03:15 PM - 03:30 PM

Coffee Break with Exhibits

03:30 PM - 05:00 PM

Plenary Breakout 1 - Pre-Clinical Application of Exosomes

Chair:
Linda Marban, PhD,Capricor, United States
Speakers:
Heterogeneity of MSCs and Clinical Potential of their Extracellular Vesicles
Lambros Kordelas, PhD, University Hospital Essen, Germany
Apoptopic Exosomes: New Players in the Regulation of the Immune Response
Marie-Josée Hébert, MD, FRCPC, University of Montreal, Canada

Oral Abstract Presentations:
The X-Factor Involved In Apoptosis And Proliferation - Reciprocal Exosome Cross-Talk Between Lymphangioleiomyomatosis-Derived Smooth Muscle Cells (LAM-SMCs) And Endothelial Cells (ECs) Mediates EC Network Disruption/Apoptosis and Promotes LAM-SMC Growth
Mirabelle Ho, PhD, Ottawa Hospital Research Institute, Canada

MSC Exosomes Alleviate Pain and Degeneration In A Rat Model of Temporomandibular Joint Osteoarthritis
Wei Seong Toh , National University of Singapore, Singapore
Chair: Linda Marban, PhD,Capricor, United States Speakers: Heterogeneity of MSCs and Clinical Potential of their Extracellular Vesicles Lambros Kordelas, PhD, University Hospital Essen, Germany Apo...
03:30 PM - 05:00 PM

Plenary Breakout 2 - Market Access & Reimbursement of CAR T Therapies

Chair:
Dawn Driscoll, PhD, MBA, Cell Therapies PTY, Australia
Speakers:
Market Access Learning from the First CAR-T in the Adult Population
Rocio Manghani, Kite Pharma, United States

CAR T Therapy, A Patient's Perspective
Douglas Olson, PhD, Tekjo Consulting, United States

Valuing Cellular, Regenerative, and Gene Therapies: The Intersection of Promise, Uncertainty, and Cost
Daniel Ollendorf, PhD, Institute for Clinical and Economic Review (ICER), United States

Implementing Gene Therapies in the Canadian Health System: What Does the Future Look Like?
Tammy Clifford, PhD , Canadian Agency for Drugs and Technologies in Health (CADTH), Canada
Chair: Dawn Driscoll, PhD, MBA, Cell Therapies PTY, Australia Speakers: Market Access Learning from the First CAR-T in the Adult Population Rocio Manghani, Kite Pharma, United States CAR T Therapy, ...
03:30 PM - 05:00 PM

Strategies for Commercialization Track Session 4 - 21st Century Global Regulatory Impact with Divergent Markets on Commercialization

Chair:
Julie Allickson, PhD, Wake Forest Institute for Regenerative Medicine, United States
Speakers:
DiscGenics Overview. Regulatory Pathways in the US and Japan
Flagg Flanagan, DiscGenics, United States

21st Century Global Regulatory Impact with Divergent Markets on Commercialization
Charlotte Morgan, PhD, Regeneus, Australia

European ATMP Regulation: Milestone or Hindrance?
Rainer Marksteiner, PhD, Innovacell, Austria

Colin Novick, CJ Partners, Japan

Panelists:
Michael Mendicino, PhD, Hybrid Concepts International, United States

To provide listeners with a better understanding of the global regulatory environment and how different jurisdictions compare as described from companies commercializing their technology, we will focus on 3 areas of comparison and contrast for regulatory considerations in different countries/regions:
1. Comparing and Contrasting US and JPN
2. Comparing and Contrasting EU and JPN
3. Australian Clinical Trials leading into JPN (and possible US/EU) clinical trials and/or strategic considerations on why Japan is an enticing location for clinical development of cellular therapies.
Chair: Julie Allickson, PhD, Wake Forest Institute for Regenerative Medicine, United States Speakers: DiscGenics Overview. Regulatory Pathways in the US and Japan Flagg Flanagan, DiscGenics, United S...
03:30 PM - 05:00 PM

Quality and Operations Track Session 4 - Day to Day Operations of a Stem Cell Lab

Chair:
Heather Garrity, MHA, Dana Farber Cancer Institute, United States
Speakers:
Making Novel Normal: Incorporating Commercial CAR T into the Daily Operations of a Lab
Karl Stasko, MPH, Dana Farber Cancer Institute, United States

Ljilijana Vasovic, MD, Weill Cornell Medicine, United States

Christine Rosati, Boston's Children's Hospital, United States

This session is focused mainly on day to day operations of a cell therapy program. It aims to highlight strategies proven effective in managing a high volume lab in addition to accommodating new trials as well as introduce challenges faced when balancing standard of care procedures, including HSCT, with the commercialization of CAR T and other novel cell therapies. The goal of this session is to give insight to members on how to develop a framework that encourages growth in a cell therapy lab while maintaining daily operations.
Chair: Heather Garrity, MHA, Dana Farber Cancer Institute, United States Speakers: Making Novel Normal: Incorporating Commercial CAR T into the Daily Operations of a Lab Karl Stasko, MPH, Dana Farber...
03:30 PM - 05:00 PM

Early Stage Professionals Session 1 - Translating Cell and Gene Therapy Products from Bench to Bedside

Co-Chairs:
Patrick Hanley, PhD, Children's National Medical Center, United States
Fernanda Masri, PhD, Sartorius-Stedim Biotech, United Kingdom
Speakers:
Cell Therapy Translation - Pathways and Pitfalls
Kenneth Micklethwaite, MBBS, PhD , Westmead Hospital, Australia

Paul Eldridge, PhD, UNC Lineberger Advanced Cellular Therapeutics Facility, United States

Developing and Implementing Investigator Initiated Trials: Practical Considerations
Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Baylor College of Medicine, United States

The Path from Lab Bench Discovery to Startup Company
Sadik Kassim, PhD, Mustang Bio, United States

The objective of this session will be to take a practical look at how key stakeholders play a role in translating a cell or gene therapy product from the bench to the bedside. This includes perspectives from a basic scientist, a GMP facility director, a regulatory director, and an investigator involved in translating the therapy to a biotech company.
Co-Chairs: Patrick Hanley, PhD, Children's National Medical Center, United States Fernanda Masri, PhD, Sartorius-Stedim Biotech, United Kingdom Speakers: Cell Therapy Translation - Pathways and Pitfal...
03:30 PM - 05:00 PM

ISCT 2018 Organizing Committee Abstract Showcase

Co-Chairs:
Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States
Rachele Ciccocioppo, MD, University of Verona, Italy

Oral Abstract Presentations:
First Rapid Final Release Test Of ATMPs Prior To Treatment – Validation Of A Robust And Highly Sensitive qPCR Test For Total Bacteria
Kai Nesemann, PhD, Sartorius Lab Instruments GmbH, Germany

Cardiosphere-Derived Cells Show Strong Immunomodulatory Activity And Improve Muscle Physiology When Systemically Delivered In A DMD Mouse Model
Jennifer Moseley, PhD, Capricor Therapeutics, United States

5-Year Performance Data And Robust Flexibility Of A Computerized Physician Ordering System For Collection, Processing, And Administration Of Cellular Therapy Products
Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States

Direct Comparison of Adult Human and Rat Spinal Cord Stem Cell Behavior
Ahmad Galuta , Ottawa Hospital Research Institute, Canada

Towards Gene-Editing Treatment for Alzheimer's Disease: ApoE4 Allele-Specific Knockout Using a CRISPR Cas9 Variant
Dani Offen, PhD, Tel Aviv University, Israel

Expanding Cellular Therapies Through Provision Of A Cord Blood - Derived iPSC Haplobank
Ngaire Elwood, PhD, BMDI Cord Blood Bank, Australia
Co-Chairs: Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States Rachele Ciccocioppo, MD, University of Verona, Italy Oral Abstract Presentations: First Rapid Final Release Test Of A...
05:00 PM - 06:30 PM

Poster Session 2

05:15 PM - 05:45 PM

Corporate Master Class Hosted by Thermo Fisher Scientific - Identifying a lentiviral production platform to achieve cost-effective scalable manufacturing

Speaker:
Calley Hirsch, CCRM, Canada

Lentiviral vectors are gaining increasing momentum as a preferred genetic delivery tool for cell and gene therapies. Manufacturing lentivirus for advanced therapeutics has long centered around transient transfection of adherent cultures for vector production, however, these methods invariably suffer from scale-up, labor, cost and consistency challenges. As an approach to circumvent these problems, transient transfection of suspension cells has been proposed. In this presentation, a lentiviral production screen will be discussed where two GMP accessible suspension cell lines were paired with different media and transfection reagents to identify a lentiviral vector platform that consistently yields the highest titer and lowest production cost. Based on these finding, internal scale-up efforts are ongoing to develop a readily adaptable cGMP lentiviral manufacturing process.
Speaker: Calley Hirsch, CCRM, Canada Lentiviral vectors are gaining increasing momentum as a preferred genetic delivery tool for cell and gene therapies. Manufacturing lentivirus for advanced therape...
07:00 PM - 11:00 PM

ISCT 2018 Gala - Tickets Required

Buses departing from the Westin Hotel starting at 6:30 pm.
Buses departing from the Westin Hotel starting at 6:30 pm.
Saturday, May 5
07:30 AM - 03:00 PM

Registration Open

07:30 AM - 03:00 PM

Speaker Services Open

08:00 AM - 09:00 AM

Hot Topic Session 5 - Solid Organ Transplantation: Tolerance Induction and Tregs Immunotherapy

Chair:
Stephan Busque, MD, MSc, Stanford University, United States
Speakers:
Tolerance Induction after Kidney transplantation: Overview and Stanford's Experience
Stephan Busque, MD, MSc, Stanford University, United States

Therapeutic Cell Transfer to Achieve Transplantation Tolerance: from Facilitating Cells to Regulatory T cells
Joseph Leventhal, MD, PhD, Northwestern Medicine, United States

This session will explore the intersection between solid organ transplantation and cell therapy that is at the basis of tolerance induction after solid organ transplantation. The experience from 2 leading programs in this field (Stanford and Northwestern) will be presented. The potential benefits of using Tregs as immunotherapy after kidney transplant to favor tolerance will also be discussed.
Chair: Stephan Busque, MD, MSc, Stanford University, United States Speakers: Tolerance Induction after Kidney transplantation: Overview and Stanford's Experience Stephan Busque, MD, MSc, Stanford U...
08:00 AM - 09:00 AM

Hot Topic Session 6 - Biomaterials and Clinical Uses

Chair:
May Griffith, PhD, Maisonneuve-Rosemont Hospital Research Centre, University of Montreal, Canada
Speakers:
Biomimetic Materials and Cornea Regeneration: from Bench to Bedside and Back
May Griffith, PhD, Maisonneuve-Rosemont Hospital Research Centre, University of Montreal, Canada

Tissue Engineered Corneal Epithelium for Clinical Application
Lucie Germain, PhD, Quebec University Laval, Canada

Biomaterials for Engineering of Bone
Jöns Hilborn, PhD , Uppsala University, Sweden

Biomaterials to Translate Promising Therapies for Neural Regeneration to Humans
Eve Tsai, MD, PhD , The Ottawa Hospital, Canada

Biomaterials are now being used to enhance cellular therapeutics. These are most widely used as nanoparticles for delivery systems for drugs and bioactives, and also in diagnostics. More recently biomaterials are being developed as scaffolds for exogenous and endogenous stem cells to affect regeneration. This aim of this session is to highlight a few examples of biomaterials designed to enhanced cellular therapeutics in regenerative medicine.
Chair: May Griffith, PhD, Maisonneuve-Rosemont Hospital Research Centre, University of Montreal, Canada Speakers: Biomimetic Materials and Cornea Regeneration: from Bench to Bedside and Back May Gr...
08:00 AM - 09:00 AM

Strategies for Commercialization Track Session 5 - Managing Patient Expectations and Reimbursement/Patient Advocacy/Market Access

Chair:
Gerhard Bauer, PhD, UC Davis, United States
Speakers:
Making Cellular Therapy Products Available in the North American Market
Gerhard Bauer, PhD, UC Davis, United States

Patient Access to CAR-T Before Approval
Dawn Driscoll, PhD, MBA, Cell Therapies PTY, Australia

CAR-T Therapy, A Patient's Perspective
Douglas Olson, PhD, Tekjo Consulting, United States

This session will cover the patient and patient advocacy perspective in regards to reimbursement and market access for novel cellular therapies. Expectations and outcomes from these novel therapies for patients and providers will be examined and equitable access to such therapies for patients, particularly from different countries, will be discussed.
Chair: Gerhard Bauer, PhD, UC Davis, United States Speakers: Making Cellular Therapy Products Available in the North American Market Gerhard Bauer, PhD, UC Davis, United States Patient Access to CAR...
08:00 AM - 09:00 AM

Quality and Operations Track Session 5 - Data Integrity - Requirements, Opportunities, Management, Reconciliation

Chair:
Gabrielle O'Sullivan, PhD, MPH, Royal Prince Alfred Hospital, Australia

Speakers:
The Future of Data Integrity for Cellular Therapies- Why You Need to Think About It Now
James Blackwell, PhD, MBA, The Windshire Group, United States

Using Software to Manage Multicentre Advanced Therapy Supply Cycles
Matthew Lakelin, PhD, TrakCel, United Kingdom

Data Integrity Challenges and Solutions in a Public Hospital Based Cell Therapy Manufacturing Facility
Zlatibor Velickovic, PhD, Royal Prince Alfred Hospital, Australia

Reconciling Data Integrity Requirements and the GDPR
Alex Denoon, BSc, LLB Marriott Harrison UK, United Kingdom

Data integrity is integral to every part of the cell therapy manufacturing and supply chain. All regulatory authorities focus on data integrity issues during cGMP inspections and data integrity problems are increasingly the basis for Warning Letters issued by the FDA. There is also burgeoning interest in developing flexible systems for managing data in multi-centre cell therapy supply cycles that are compliant and validated. Additionally, data integrity and clinical trials are affected by changes to data protection requirements and planning is essential to avoid risks in these areas. This session brings together experts that cover all of these areas. It will be useful to those involved in cell therapy manufacture, supply, clinical trials and regulation.
The aim of the session is to develop an understanding about what is required and what the possibilities are in various cell therapy manufacturing and supply settings.
Chair: Gabrielle O'Sullivan, PhD, MPH, Royal Prince Alfred Hospital, Australia Speakers: The Future of Data Integrity for Cellular Therapies- Why You Need to Think About It Now James Blackwell, Ph...
09:15 AM - 10:45 AM

Plenary Session 5 - ISCT-ASGCT Joint Session: Genome Editing for Benign and Malignant Diseases

Co-Chairs:
Donald Kohn, MD, University of California, Los Angeles, United States
Helen Heslop, MD, Baylor College of Medicine, United States
Speakers:

Gene Editing of Hematopoietic Stem and Progenitor Cells
Matthew Porteus, MD, PhD, Stanford University School of Medicine, United States

Genome Editing with ZFN using Blood Progenitor Cells in HIV/AIDS
John Zaia, MD, City of Hope, United States

Highly Efficient and Specific Multiplexed Gene Editing in T Cells Using Enhanced Zinc-finger Nucleases (ZFNs) Enables Strategic Engineering of Allogeneic T Cell Immunotherapies
Michael Holmes, PhD, Sangamo Therapeutics, United States

In this joint session hosted by ISCT and ASGCT on Genome Editing for Benign & Malignant Diseases, recent advances in gene editing will be reviewed.
Presentations will include: Ex Vivo Gene Modification of HSC by Matthew Porteus (Stanford University); ZFN Targeting of CCR5 in HSC for HIV by John Zaia (City of Hope Medical Center); and In Vivo Targeting of Liver by Michael Homes (Sangamo Biotherapeutics). The goal of the session is to provide attendees with the latest information on the approaches and status of genome editing.
Co-Chairs: Donald Kohn, MD, University of California, Los Angeles, United States Helen Heslop, MD, Baylor College of Medicine, United States Speakers: Gene Editing of Hematopoietic Stem and Progenito...
10:45 AM - 11:00 AM

Coffee Break

11:00 AM - 12:00 PM

Plenary Breakout 2 - Ex Vivo Editing - HSC and iPSC

Chair:
Matthew Porteus, MD, PhD, Stanford University School of Medicine, United States
Speaker:
Improving Homology-Directed Repair in Hematopoietic Stem Cells
Donald Kohn, MD, University of California, Los Angeles, United States

Oral Abstract Presentations:
How Genetically Modified Organism (GMO) Regulations Impact On The Development Of Genetically Modified Cell-Based Therapies In Europe, The U.S. And Japan
Houria Bachtarzi, PhD ERA Consulting (UK) Ltd., United Kingdom
Chair: Matthew Porteus, MD, PhD, Stanford University School of Medicine, United States Speaker: Improving Homology-Directed Repair in Hematopoietic Stem Cells Donald Kohn, MD, University of Californ...
11:00 AM - 12:00 PM

Plenary Breakout 1 - Editing to Enhance Cell Therapy - Universal T Cells

Chair:
Helen Heslop, MD , Baylor College of Medicine, United States
Speaker:
Engineering Regulatory T Cells for Inflamttory Disorders
Andrew Scharenberg, MD, University of Washington, United States

Oral Abstract Presentations:
Single Cell Analysis Of Lentiviral Integration To Support Ex-Vivo Gene Modified Cell Therapy Development
Vincenzo Di Cerbo, PhDCell and Gene Therapy Catapult, United Kingdom

CRISPR-Driven Modeling of Clinically Relevant Genetic Variants in Hematopoietic Stem and Progenitor-Derived Erythroid Cells
Yelena Boccacci, BSc, Université Laval, Héma-Québec, Canada
Chair: Helen Heslop, MD , Baylor College of Medicine, United States Speaker: Engineering Regulatory T Cells for Inflamttory Disorders Andrew Scharenberg, MD, University of Washington, United States O...
11:00 AM - 12:00 PM

Strategies for Commercialization Track Session 6 - Growing the Pie: Has Investor Interest Increased for CGT post-approval of Kymriah, Yescarta and Luxturna?

Chair:
Bill Milligan, Steminent Biotherapeutics, Canada
Speakers:

A Whale Swims into a Pond... Cell and Gene Therapy Hit the Market - Now What?
Patrick Rivers, MBA, Aquilo Capital, United States

Venture Capital Views on Investments in CGTs
Didier Leconte, MBA, FSTQ, Canada

The European Perspective
Matthew Durdy, MBA, Cell and Gene Therapy Catapult, United Kingdom

How have the recent approvals of these two CD19 CAR T therapies and gene therapy influenced investor behaviour? In this session, we'll look at how investors perceive and value CGT investments versus other more traditional treatment modalities and explore what their biggest remaining concerns might be. In addition, we’ll explore how to identify and engage the right investors and how to optimally position your company to raise capital. A must attend session in understanding what has changed for investors and how to leverage it going forward.
Chair: Bill Milligan, Steminent Biotherapeutics, Canada Speakers: A Whale Swims into a Pond... Cell and Gene Therapy Hit the Market - Now What? Patrick Rivers, MBA, Aquilo Capital, United States Ve...
11:00 AM - 12:00 PM

Quality and Operations Track Session 6 - Challenges of cGMP Clean Room Facility for Cell Therapy Manufacturing in an Academic Setting

Chair:
Nadim Mahmud, MD, PhD, University of Illinois Hospital and Health Sciences System, United States
Speakers:
Challenges to Build and Validate a cGMP Facility Without Interruption of Ongoing Graft Processing Services
Nadim Mahmud, MD, PhD, University of Illinois Hospital and Health Sciences System, United States

Risk Management for cGMP Academic Facility
Christopher Garbe, MBA, Gates Biomanufacturing Facility - University of Colorado, United States

How to Set up a GMP Compliant Clean Room for an Academic Vector Production
Aisha Khan, MBA, University of Miami, United States
Chair: Nadim Mahmud, MD, PhD, University of Illinois Hospital and Health Sciences System, United States Speakers: Challenges to Build and Validate a cGMP Facility Without Interruption of Ongoing Graf...
11:00 AM - 12:30 PM

Early Stage Professionals Session 2 - Young Investigator Abstract Showcase

Co-Chairs:
Alireza Abazari, PhD, BioLife Solutions, United States
George Hucks, MD, University of North Carolina Hospitals, United States

Oral Abstract Presentations:
Safety Of Stem Cell-Derived Encapsulated Liver Tissue To Treat Liver Failure: Immune-Isolation And Absence Of Foreign Body Reaction Or Tumor Formation Upon Transplantation Without Immunosuppression
Claudia Raggi, MD, CHU Sainte-Justine, Canada

Combined Cell And Gene Therapy Towards The Treatment Of Age-Related Macular Degeneration And Diabetic Retinopathy
Sabiha Hacibekiroglu, PhD , Lunenfeld-Tanenbaum Research Institute/Mt Sinai Health System, Canada

First Trimester Human Umbilical Cord Perivascular Cells (FTM HUCPVCs) Expanded With cGMP Compliant Human Platelet Outperform Conventional And Older MSC Sources As Regenerative Therapy In A Rat Myocardial Infarction (MI) Model
Alexander Johnston, PhD,Create Fertility Centre, Canada

How to Be Successful at Publishing your Work
John Barrett, MD, George Washington University Cancer Center, United States
Co-Chairs: Alireza Abazari, PhD, BioLife Solutions, United States George Hucks, MD, University of North Carolina Hospitals, United States Oral Abstract Presentations: Safety Of Stem Cell-Derived Enca...
12:00 PM - 01:30 PM

Lunch

12:15 PM - 01:15 PM

01:30 PM - 03:00 PM

Plenary Session 6 - iPSC Regenerative Medicine

Chair:
Andras Nagy, PhD, Lunenfeld-Tanenbaum Research Institute, Sinai Health System, Canada
Speakers:
Translating Stem Cell Biology into Regenerative Medicine
Maria Mirotsou, PhD , Astellas Institute of Regenerative Medicine, United States

Making iPSC Cell Derived Products
Mahendra Rao, MD, PhD, New York Stem Cell Foundation, United States

A Solution for Cell Therapy Safety and Engineered Allotolerance
Andras Nagy, PhD, Lunenfeld-Tanenbaum Research Institute, Sinai Health System, Canada
Chair: Andras Nagy, PhD, Lunenfeld-Tanenbaum Research Institute, Sinai Health System, Canada Speakers: Translating Stem Cell Biology into Regenerative Medicine Maria Mirotsou, PhD , Astellas Instit...
03:00 PM - 03:15 PM

Coffee Break

03:15 PM - 04:30 PM

Plenary Breakout 1 - iPSC and Organogenesis

Chair:
Maria Mirotsou, PhD, Astellas Institute of Regenerative Medicine, United States
Speakers:
Making Functional Islet Cells from Human Pluripotent Stem Cells
Timothy J. Kieffer, PhD University of British Columbia, Canada

Modeling Human Retinal Degenerative Diseases Using iPSC-derived 3D Photoreceptor Sheets
Gilbert Bernier, PhD , Hôpital Maisonneuve-Rosemont, University of Montreal, Canada

Oral Abstract Presentations:
Rapid And Robust Cd4+ And Cd8+ T-, Nk-, B- And Monocyte Cell Reconstitution After Nicotinamide-expanded Cord Blood (nicord) Transplantation
Jaap Jan Boelens
Chair: Maria Mirotsou, PhD, Astellas Institute of Regenerative Medicine, United States Speakers: Making Functional Islet Cells from Human Pluripotent Stem Cells Timothy J. Kieffer, PhD University of...
03:15 PM - 04:30 PM

Plenary Breakout 2 - iPS Generation and Skin and Lung Applications

Chair:
Mahendra Rao, MD, PhD, New York Stem Cell Foundation, United States

Speaker:
Collagen VII Skin Grafts for Chronic RDEB Wounds
Jean Tang, MD, PhD, Stanford University School of Medicine, United States

Oral Abstract Presentations:
Safety Considerations in the Generation of Clinical Grade Autologous iPS Cell Lines
Holly Anderson

Close Neighbors in the Niche: Paracrine Signals from Endothelial Cells Promote Alveolar Epithelial Differentiation of Induced Pluripotent Stem Cell-derived Lung Progenitors
Miriel Ho

Allogeneic Amniotic Epithelial Cells for Established Bronchopulmonary Dysplasia in Premature, Low Birthweight Infants: A first-in-human Safety Trial
Rebecca Lim
Chair: Mahendra Rao, MD, PhD, New York Stem Cell Foundation, United States Speaker: Collagen VII Skin Grafts for Chronic RDEB Wounds Jean Tang, MD, PhD, Stanford University School of Medicine, Unit...
03:15 PM - 04:30 PM

Strategies for Commercialization Track Session 7 - ISCT-BPSA Joint Session: Next Generation Manufacturing for Cell and Gene Therapies

Co-Chairs:
Ohad Karnieli, PhD, MBA, Atvio Biotech, Israel
Eric Isberg, Entegris, United States
Speakers:
Defining Single-Use Systems for Cell & Gene Therapy Manufacturing
Dominic Clarke, PhD, Charter Medical, United States

Bioreactor Process Correlations and Sample-free Cell Expansion
Gary Pigeau, PhD, GE Healthcare, Canada

Supervisory Control Automation for Next Generation Cell Therapy Manufacturing
Randy Schweickart, MS, Juno Therapeutics, United States
Co-Chairs: Ohad Karnieli, PhD, MBA, Atvio Biotech, Israel Eric Isberg, Entegris, United States Speakers: Defining Single-Use Systems for Cell & Gene Therapy Manufacturing Dominic Clarke, PhD, Charte...
03:15 PM - 04:30 PM

Quality and Operations Track Session 7 - Regulatory Expectations for the Manufacturing Control Strategy

Co-Chairs:
Rosemarie Bell, BAppSc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia
Sowmya Viswanathan, PhD, University Health Network, University of Toronto, Canada
Speakers:
CMC Challenges in Cell and Gene Therapies: A Health Canada Perspective
Christopher Storbeck, PhD, BGTD, Health Canada, Canada

Introduction to Process Control Strategy
Scott Burger, MD Advanced Cell and Gene Therapy LLC, United States

Design and Optimisation of Process & Product Controls for Cell-based Products
Paula Salmikangas, PhD, NDA Advisory Services Limited, United Kingdom
Co-Chairs: Rosemarie Bell, BAppSc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia Sowmya Viswanathan, PhD, University Health Network, University of Toronto, Canada Speakers: ...