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John Rasko, AO, BSc(Med), MBBS(Hons), PhD, University of Sydney, AUS
Mario R. 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
Recent advances in genome editing technologies have substantially improved our ability to make changes in the genomes of eukaryotic cells. Viral vectors and programmable nucleases are already revolutionizing our ability to interrogate the function of the genome. This session provides an overview of current progress in qualifying and regulating targeted genome editing technologies as they are being used clinically to correct or introduce genetic mutations to treat diseases that are refractory to traditional therapies. Preclinical assessment and GMP manufacturing of these technologies as well as the regulatory requirements for FIH clinical trials utilizing these technologies will be discussed.
Shirley Bartido, PhD, MBA, Cellectis, United States
Christopher Ballas, PhD, Rocket Pharma, United States
David Jones, FRSB, FBTS, Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom
Stephan Reynier, MSc, Cellectis, France
Optimizing a product through process development is a natural step in translating the therapeutic to the clinic. While this is a critical milestone, many of the processes have not been optimized for larger-scale industrialization. This session will evaluate how industry innovators are implementing new processing platforms and strategies to position future products for sustainable industrialization
Dominic Clarke, PhD, HemaCare Corporation, United States
Denis Bedoret, PhD, MaSTherCell Global, Belgium
Stuart Curbishley, PhD, University of Birmingham Medical School, United Kingdom
Ricardo Batista, PhD, Cellectis, France
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
Today the first two autologous CAR T therapies are commercialized in multiple developed countries, through dozens of health care facilities, and many hundreds of patients are benefiting from treatment! Now “industrializing” CAR T therapy to gain world-wide, broad patient access to this new standard of care, means not only extensive scale-up of manufacturing capacity but also transforming hundreds of health care centers into CAR T competent treatment sites using either autologous or “off-the-shelf” products in treating patients. What are some key considerations for how academia, industry and healthcare centers can meet this challenge? Today we’ll explore autologous, allogeneic, and stakeholder partnering perspectives from three experts active in pursuing this CAR T industrialization vision.
Bill Milligan, Steminent Biotherapies, Canada/Taiwan
Gunther Busam, RPh, PhD, Celgene, Switzerland
Elizabeth Hexner, MD, Penn Medicine, United States
Wen Bo Wang, PhD, Fate Therapeutics, United States
Process Validation – it has to happen! Join us to hear regulatory perspectives and real-world examples of considerations specific to autologous products, including both cells and tissues. Are normal donor cells ok to use? What risks are associated with validating your process using cells or tissues from healthy donors?
Emily Hopewell, PhD, Indiana University School of Medicine, United States
Antonio Ruiz-Garcia, MPharm, Hospital Universitario Virgen de las Nieves, Spain
Corey Smith, PhD, QIMR Berghofer, Australia
Aisha Khan, MSc, MBA, University of Miami, United States
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
Katy Rezvani, MD, PhD, MD Anderson Cancer Center, USA
The Q&O track regulatory session will address the hot CMC topics of 2020. For many ATMP products identification of critical quality attributes (CQAs) that can predict clinical outcome is not easy, especially due to inherent variability of the starting materials. Product characterization and selection of most useful assays will be discussed in this session, as well as the GMP constraints across the US and the EU. In the panel discussion regulatory experts from US, EU and Japan will address the CMC readiness of companies and regulatory authorities in the context of the expedited regulatory programs of ATMPs.
Paula Salmikangas, PhD, NDA Advisory Services Ltd., Finland
Christopher Bravery, PhD, Advbiols, United Kingdom
Shinichi Noda, PhD, Pharmaceuticals and Medical Devices Agency(PMDA), Japan
Rocio Salvador-Roldan, European Commission, Belgium
In this session we will discuss the current landscape of cell and gene therapies from the investor’s perspective. As these therapies reach more advanced stages of development and eventually commercialization the capital requirements will continue to increase – and along with this the need to expand the scope of the investor base. We aim to address the following questions:
- How do investors characterize opportunities for CGT companies relative to more traditional drug modalities?
- What role does pharma need to play to support the CGT ecosystem?
- Will investor appetite for CGT continue to increase, or have we hit a local maximum?
Patrick Rivers, MBA, Aquilo Capital, United States
Jak Knowles, MD, Leaps by Bayer, United States
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, United States
Angela Krackhardt, MD, Technical University of Munich, Germany
Alberto Santagostino, MBA, Lonza, Switzerland
Emanuele Ostuni, PhD, Novartis Oncology, Switzerland
The supply chain is working now but how will it cope when there are tens of therapies and thousands of patients, globally? The “hidden challenge” is the last 100m within the clinical setting. Here, we will describe ways the infrastructure, equipment, staffing, training, and systems need to be designed to operate efficiently to treat the patients.
Julie Murrell, PhD, MilliporeSigma, United States
Christopher Herbert, PhD, Leeds Teaching Hospitals NHS Trust, United Kingdom
Anne Black, The Newcastle Upon Tyne Hospitals NHS Foundation Trust, United Kingdom
Christoph Demmerle, Novartis, Germany
The path to taking a great platform or therapeutic program that has been incubated at an academic center and turning it into a biotech company is fraught with peril and cautionary tales. We want to explore the lessons learned by academic founders of successfully funded academic spin-outs and provide a better blueprint for others who are wondering how to pursue the creation of a company around their novel science.
Patrick Rivers, MBA, Aquilo Capital, United States
Mike Milone, MD, PhD, Cabaletta Bio, United States
Colleen Delaney, MD, MSc, Nohla Therapeutics, United States
Starting and raw material selection is an essential aspect that need to be considered for ATMP manufacturing in order to guarantee quality of medicinal products to be used in patients. During this session the most important aspects regarding starting and raw material selection for ATMP manufacturing are going to be reviewed such as:
- Differences in donor eligibility requirements, regulatory aspects to comply during donor selection and ethical considerations
- Critical aspects to consider when using biological raw materials for ATMP manufacturing
- Challenges to consider during vector production and control
- Appropriate conditions to establish and test when using seed lots and cell banks
Gloria Carmona, MPharm, Andalusian Network for Design and Translation of Advanced Therapies, Spain
Craig Spalding, MBA, Scottish National Blood Transfusion Service, United Kingdom
Rosaria Giordano, MD, Center of Cellular Therapy – Cell Factory Fondazione Ca’ Granda Ospedale Maggiore Policlinico, Italy
Daniela Bischof, PhD, Indiana University School of Medicine, United States
Gerhard Bauer, PhD, University of California Davis Institute for Regenerative Cures, United States
Pavillons des Bercy
Rosemarie Bell, B.App.Sc Micro/Biochem MASM, Q-Gen Cell Therapeutics QIMR Berghofer Medical Research Institute, Australia
Mehrshid Alai-Safer, PhD, Kite Pharmaceuticals, United States
Mohamed Heidaran, PhD, Parexel International, United States
Krishnendu Roy, PhD, Georgia Institute of Technology, United States
Commercialization of cellular and gene therapies is irrelevant if the patients do not receive the therapy they require. As we industrialize the manufacturing of such products, assuming equivalent efficacy, the therapy that is the easiest to adopt, depending on process and cost, and is the least difficult for the patient to tolerate will be the most widely adopted. To arrive at this endpoint, we need to understand the patient’s needs and expectations when they consider cell and gene therapy options. In this context, it is important to discriminate between legitimate, safe and effective therapies versus alternative treatments that are offered to patients who are desperate. Cell and gene therapies, and particularly stem cell therapies need be placed into the category of highly advanced and often life saving therapies, if chosen and administered in a responsible and ethical way.
Gerhard Bauer, PhD, University of California, Davis Institute for Regenerative Cures, United States
Kathie Rodden, NHS University Hospitals of Birmingham, United Kingdom
Mohamed Abou-El-Enein, MD, PhD, Charité – Universitätsmedizin Berlin, Germany
Mark Yarborough, PhD, University of California, Davis, United States
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
Carlos Fonck, PhD, DABT, BioMarin Pharmaceuticals Inc., USA
Julie Allickson, PhD, Wake Forest Institute for Regenerative Medicine, United States
Petter Björquist, PhD, VERIGRAFT AB, Sweden
Martin Birchall, MD, University College London, United Kingdom
Heather Prichard, PhD, Humacyte, United States
Gerry McKiernan, Cell Therapies Pty Ltd, Australia
Sophie Paczensy, MD, PhD, Indiana University School of Medicine, United States
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
Shiraz Ziya, PhD, Sartorius Stedim UK Ltd, United Kingdom
Angela Osborne, PhD, eXmoor pharma concepts ltd, United Kingdom
Linda Kelley, PhD, Moffitt Cancer Center, United States
Recent years have seen an increase in the number of fast track regulatory pathways available to developers, many of which are intended to accelerate commercialisation of promising cell and gene therapies. As these pathways begin to be tested and products are commercialised through them, this session will focus on both the opportunities and challenges that they present. Key differences between geographies will be discussed and how these novel pathways affect global product development plans.
Siofradh McMahon, MSc, CCRM, Canada
Katy Spink, PhD, Dark Horse Consulting, United States