Accelerating CGT Adoption: Proof of Concept to Standard of Care
Presidential Plenary – Mastering Pluripotent Cells With Therapeutic Intent
John Rasko, AO, BSc(Med), MBBS(Hons), PhD, University of Sydney, AUS
Mario Capecchi, PhD, 2007 Nobel Laureate (Physiology or Medicine), University of Utah, USA
Keynote speaker sponsored by Cytotherapy, The official journal of the International Society for Cell & Gene Therapy
Marc Peschanski, MD, PhD, I-Stem, FRA
Rudolf Jaenisch, MD, Whitehead Institute and Massachusetts Institute of Technology, USA
Plenary Session 2 – Mesenchymal Stromal Cells- Full Circle from Basic Research Insights to Clinical Trial Updates
This session will bring together speakers who will provide a full spectrum overview of latest developments in the field of mesenchymal stromal cell research. This includes insights into newly hypothesized mechanisms of action and single cell characterization of mesenchymal stromal cells, clinical translation challenges using pooled bone marrow-derived mesenchymal stromal cells for treatment of refractory Graft-vs. Host Disease, and updates from a pioneering European trial on the use of mesenchymal stromal cells to treat refractory Sclerodoma.
Sowmya Viswanathan, PhD, University Health Network Cell Therapy, CAN
Jacques Galipeau, MD, University of Wisconsin-Madison, USA
Halvard Bönig, MD, PhD, Johann-Wolfgang-Goethe University, GER
Dominique Farge-Bancel, MD, Paris Diderot University and Hôpital Saint-Louis, FRA
Plenary Session 3 – Advances and Emerging Technologies in Immune-Mediated Cancer Treatments
CAR-T Cells represent the first example of a cellular therapy that has moved from immunological concepts developed in research laboratories to worldwide and large-scale industry-manufacturing of potent therapeutic agents. The bumpy road* for these developments exemplify the many scientific challenges that await developers of this class of therapeutics. While scientific developments continue to occur at a high pace for CAR-T and CAR NK therapies, the race to solve the logistical, medical, financial and societal issues is engaged, with the goal to bring this class of therapeutics to all patients in need of them. CAR-T and CAR NK Cells will likely pave the way for other forms of immune cellular therapies, but also for regenerative medicines.
Bruce Levine, PhD, University of Pennsylvania, USA
Christian Chabannon, MD, PhD, Institut Paoli Calmettes, FRA
Marcela Maus, MD, PhD, Massachusetts General Hospital and Harvard Medical School, USA
Michael Hudecek, MD, University of Würzburg, GER
Plenary Session 4 – Is Big Pharma ready for industrial scale Cell and Gene Therapy?
Recent successes in the market authorization of cell and gene therapy products has generated tremendous excitement. However, to be successful, the industry now needs to move into a phase of industrialization, where like the car industry it needs the “eureka” moment of standardization that allows the efficient flow from product to patient. To realize this, we need to overcome the barriers between all the silos in the system (e.g. hospital, manufacture, testing, etc.) so that patients are efficiently treated.
Anthony Ting, PhD Athersys, USA
Plenary Session 5 – Gene Engineering: The Past, Present and the Future
Gene-engineering is currently ‘in-vogue’ as a potential treatment for multiple monogenetic and rare diseases. This session will highlight the progress in the field of gene-insertion, vector biology and gene-editing for both the ex-vivo and in-vivo approaches of gene manipulation. The current challenges and safety issues will be discussed, with the spotlight on ongoing clinical trials as examples of potential curative therapies.
Sandeep Soni, MD, Standford University, USA
Donald Kohn, MD, University of California Los Angeles, USA
Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), SPA
Plenary Session 6 – Basics and translational potential of extracellular vesicles including exosomes
Most cells release Extracellular Vesicles (EVs) into their environment. In particular, small EVs of 50-200 nm that include exosomes, have been shown to mediate intercellular communication in many physiological and pathophysiological processes. Small EVs from some cell sources, e.g. mesenchymal stromal cells (MSCs), can alleviate pathological processes and are considered as novel therapeutic agents. Within the session basic aspects of EV biology and their therapeutic potential will be discussed.
Bernd Giebel, PhD, University Hospital Essen, Institute for Transfusion, GER
Clotilde Théry, PhD, Institut Curie, INSERM, FRA
Sai-Kiang Lim, PhD, Institute of Medical Biology, A*STAR, SGP
Rebecca Lim, PhD, Monash University, AUS