Plenary & Concurrent Sessions

Stay up to date on session topics, themes, and confirmed speakers.  This page will be continually updated as the program is finalized.

When there was no beaten path, these pioneers created the path from discovery and technology development, to clinical trial translation, regulatory approval, and commercialization for gene modified immune cell therapies.  Now, we look at what will be needed to take our ability to design immunity from generation 1.0 to 2.0.

  • Address the advantages, disadvantages, and unknowns of various gene editing approaches
  • Bring new technologies for gene modification and immune cell engineering to the clinic
  • Move from concept to scalable product that can achieve regulatory approval and benefit patients

This session will discuss the application of computational modeling and other engineering approaches to cell and gene therapies and to tissue engineering.  Key goals and objectives are to:

  • Understand advances in utilizing in silico and computational modeling to cell and gene therapies 
  • Illustrate advances in tissue engineering approaches to CGT
  • Understand immune challenges to CGT tissue engineering
1. MSC Plasticity and Effect of In-Vivo Environments on MSC Actions. What Actually Happens to the MSCs After Administration?
  • Chair: Daniel J. Weiss, MD, PhD, University of Vermont, United States
  • Speakers: 
    • Tracy Heng, PhD, Monash University, Australia
    • Sara Rolandsson Enes, PhD, Lund University, Sweden
2. Prospects and Challenges for iPSC-based Cell Therapy
  • Chair: Shin Kawamata, MD, PhD, Foundation for Biomedical Research and Innovation, Japan
  • Speakers:
    • Guojun Bu, PhD, Mayo Clinic, United States
    • Shin Kaneko, MD, PhD, Kyoto University, Japan
    • Maxime Feyeux, PhD, TreeFrog Therapeutics, France
    3. Non-Malignant Applications of CAR T
    • Chair: Ken Micklethwaite, MBBS, PhD, FRACP, FRCPA, The University of Sydney, Westmead Hospital, Australia
    • Speakers:
      • Qizhi Tang, PhD, University of California, San Francisco, United States
      • Boro Dropulic, PhD, MBA, Caring Cross, United States
      • Michael Milone, MD, PhD, University of Pennsylvania, United States
    4. State of the Art in EV (Exosome) Research: Rigor and Function
    • Chair: Bernd Giebel, PhD, University of Duisburg-Essen, Germany
    • Speaker:
      • Joshua Welsh, PhD, National Institutes of Health, United States
      • Randolph Corteling, PhD, ReNeuron, United Kingdom
    5. In-Vivo Gene Editing
    • Chair: Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Spain 
    • Speakers:
      • Sean Burns, MD, Intellia Therapeutics, United States
      • Nikoletta Psatha, PhD, Aristotle University of Thessaloniki, Greece
      • John Sheridan, PhD, Cystic Fibrosis Foundation, United States

      The transition from pre-clinical to clinical studies is a well-trodden, yet unique journey that is impacted by your drug product, the facility/resources you have available, and the complexity of your manufacturing process. 

      • What are the risks of new gene modification systems and gene therapy products and how can we ameliorate those risks?
      • How to develop potency assays for an early phase product to ensure a smooth transition not only into later phase studies but also into the market
      • Navigating Technology Transfer to ensure feasibility of successful products and avoiding costly delays due to supply chain, raw materials, and the like. etc.


      1. Enough Bait Cutting, Time to Fish- Rethinking first in Human MSC Studies
      • Chair: Jacques Galipeau, MD, University of Wisconsin-Madison, United States
      • Speakers: 
        • Dominique Farge, MD, PhD, Paris University and McGill, France
        • Karin Tarte, PhD, Rennes University, France
        • Fermín Sánchez‐Guijo, MD, PhD, IBSAL-Hospital Universitario De Salamanca, Spain
      2. Weaponized Immune Cells
      • Chair: Chrystal Louis, MD, CRISPR, United States
      • Speakers:
        • Catherine Bollard, MD, MBChB, Children's National Research Institute, United States
        • Carlos Ramos, MD, Baylor College of Medicine, United States
        • Kevin Tosh, PhD, Carisma Therapeutics Inc., United States
        3. Ethical Considerations and Consent in Novel Therapies
        • Chair: Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Baylor College of Medicine, United States
        • Speakers:
          • Akshay Sharma, MBBS, St. Jude Children's Research Hospital, United States
          • Patricia Zettler, JD, The Ohio State University Moritz College of Law, United States
        4. First in Human Study Design
        • Chair: Catherine Bollard, MBChB, MD, Children's National, United States
        • Speakers:
          • Kristin Baird, MD, IQVIA, United States
          • Joycelynne Palmer, PhD, City of Hope, United States
          5. Will CMC Trump Your Data?
          • Chair and speaker: Thomas Heathman, PhD, Ori Biotech, United States
          • Speaker:
            • Anthony Davies, PhD, Dark Horse Consulting, United States
            This session will highlight the human applications of allogeneic cell-gene therapies, provide clinical updates and discuss some of the challenges in the field.  
            • Describe various applications of allogeneic CGT
            • Update on clinical trials and patient outcomes
            • Identify current challenges in the field
            1. Regulatory T Cells: Clinical Applications
            • Chair: Alexey Bersenev, MD, PhD, Yale University, United States
            • Speakers: 
              • Maria Grazia Roncarolo, MD, Stanford, United States
              • Megan Levings, PhD, University of British Columbia, Canada
            2. Hematopoietic Stem Cell Engineering for Gene Therapy
            • Chair: Sandeep Soni, MD, CRISPR Therapeutics, United States
            • Speakers:
              • Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Spain
              • Mort Cowan, MD, University of California San Francisco, United States
              3. MSC Trials in COVID-19
              • Chair: Jan Nolta, PhD, UC Davis, United States
              • Speakers:
                • Carolyn Hendrickson, MD, MPH, University of California San Francisco, United States
                • Giacomo Lanzoni, PhD, University of Miami, United States
              4. CAR T Cell Manufacturing 2.0 - Accelerating the Time to Patient Treatment
              • Chair: Bruce Levine, PhD, University of Pennsylvania, United States
              • Speakers:
                • David Steensma, MD FACP, Novartis, United States
                • Charles Calderaro, MBA, Kite Pharma, United States

                  5. Pitfalls in Late Stage CMC: What's the Solution
                  • Chairs:
                    • David DiGiusto, PhD, Resilience, United States
                    • Miguel Forte, MD, PhD, Bone Therapeutics, Belgium
                  • Speaker:
                    • Peter Marks, MD, PhD, FDA, United States
                  This session will discuss the challenges and pitfalls of preparing for and executing a flawless CGT product launch. It will include input from all the major stakeholders, including clinicians, patients, payers, developers and manufacturers.

                  • What are the critical success factors to be considered in planning a CGT launch?
                  • In thinking about a successful commercial CGT product – what are the key value drivers for each group of stakeholders?
                  • What lessons have been learned by the speakers in planning and carrying out a launch?
                    1. Make it, don’t break it! – Assuring global commercial success through a strong CGT manufacturing strategy
                    • Chair: Phil Vanek, PhD, GammaBio, United States
                    • Speakers: 
                      • Jacqueline Barry, PhD, CGT Catapult, United Kingdom
                      • Nicholas Ostrout, PhD, Charles River, United States
                      • Derrell Porter, MD, Cellevolve Bio, United States

                    2. Shining a Light on Unlicensed CGTs: Where is the Line Between the Right to Care, Ethics, and Patient Safety?

                    • Chair:  Michela Gabaldo, MSc, Fondazione Telethon, Italy
                    • Speakers: 
                      • Ilona Reischl, PhD, Austrian Agency for Health and Food Safety, Austria
                      • Laertis Ikonomou, PhD, University at Buffalo, United States
                      • Simone Boselli, EURORDIS, Belgium
                      • Michael Lehmicke, MSc, Alliance for Regenerative Medicine, United States
                      3. Are Cell & Gene Therapies a First World only Luxury?
                      • Chair: Sven Kili, MBChB, MRCS (Eng), Antion Biosciences, United Kingdom
                      • Speakers: 
                        • Michael Pepper, MBChB, MD, PhD, Institute for Cellular and Molecular Medicine, South Africa
                        • Boro Dropulić, PhD, MBA, Caring Cross, United States
                        • Maroun Khoury, PhD, Universidad de los Andes, Chile
                        • Tay Salimullah, BSc Mgmt, Novartis, United States
                      4. Do Accelerated Pathways Live up to Their Promise?
                      • Chair: Sharon Rooney-Mayer, MBA, Novartis, United States
                      • Speakers: 
                        • Colin Lee Novick, BA, Innovacell K.K., Japan
                        • Wendy L. Corbett, PhD, MBA, Bristol Myers Squibb, United States

                        5. Raiders of the Lost Art: Investing In The Next CGT Gem
                        • Co-Chairs:
                          • Reni Benjamin, PhD, JMP Securities, United States
                          • Asthika Goonewardene, MBA, Truist Securities, United States
                        • Speaker: 
                          • Konstantina Katcheves, MSc, Bristol Myers Squibb, United States
                          • Bin Lu, PhD, Temasek, Singapore
                          • Cariad Chester, BA, TCG X, United States