Research Xchange Forum 2019
Insights | Translation | Solutions
Transformative Technologies: CAR-T Cell Therapy
The 3rd annual Research Xchange Forum, held on April 25, 2019 at Science/AAAS in Washington DC, provided the rare opportunity to meet with academic scientists and industry leaders in CAR-T therapy research. The event fostered the exchange of ideas and enable potential collaborations between academia and industry. By this, we hope to inspire a new generation of scientists in this rapidly growing and very promising area of research. A series of fascinating lectures were held by experts working in basic and applied research and/or development on the latest on CAR-T therapies.
In addition to the lectures, live demonstrations were held of groundbreaking technologies to enable and accelerate CAR-T workflows (the Intellicyt® iQue Screener PLUS and IncuCyte® S3 Live-Cell Analysis System) to provide both sectors a platform for learning, dialogue and discussion. These activities were further enhanced by an expert panel discussion about the present and future of CAR-T therapies. In addition to the forum, the winner and three finalists of the 2018 "Sartorius & Science Prize for Regenerative Medicine & Cell Therapy" presented their research and received their awards during an evening ceremony.
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Learn about events at #RXF2019, including lectures by CAR-T scientists and industry experts, live demos of groundbreaking technologies to enable and accelerate CAR-T workflows, and more!
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- Expert insights from leading international scientists working in the academia or industrial biopharma R&D
- Research results from the winners of the "Sartorius & Science Prize for Regenerative Medicine & Cell Therapy"*
- Panel discussion about the present and the future of CAR-T cell therapies
Live demonstrations of the latest groundbreaking technologies being utilized to propel CAR-T therapy research forward including:
- IncuCyte® S3 Live-Cell Analysis System — Enables real-time, automated imaging and analysis of immune cell activation and function, directly from the incubator over days, weeks or months.
- Intellicyt® iQue Screener PLUS — high throughput, suspension cell and bead analysis platform for rapidly profiling cell phenotype and function in cell therapy workflows.
Awards Ceremony for the Winners of the Sartorius & Science Prize
The winner and finalists competing for the "Sartorius & Science Prize for Regenerative Medicine & Cell Therapy" received their awards at a ceremony held in their honor.
*Invited guests only
- Talks from leading scientists and industry experts in the CAR-T space
- Panel discussion about the present and future of CAR-T
- Live demo of the Intellicyt® iQue Screener PLUS and IncuCyte® S3 Live-Cell Analysis System
- Award Ceremony (evening event) — Science Prize Winners* (invitation only)
Barbara and Edward Netter Prof. in Cancer Gene Therapy
Univ. of Pennsylvania
Sr. VP Immunogenetics Editas
Other speakers from:
- MD Anderson Cancer Center
- Boston University
- St. Jude Childrens Hospital
- Colorado State University
- Horizon Discovery
- See all below...
Dr. Bruce Levine
Philadelphia, Pennsylvania, USA
Barbara and Edward Netter Professor in Cancer Gene Therapy at University of Pennsylvania
“From Boutique to Global: Chimeric Antigen Receptor T Cells as a Model for Clinical Development and Commercialization”
Dr. Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, is the Founding Director of the Clinical Call and Vaccine Production Facility (CVPF) in the Department of Pathology and Laboratory Medicine and the Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania. He received a B.A. (Biology) from Penn and a Ph.D. in Immunology and Infectious Diseases from Johns Hopkins. First-in-human adoptive immunotherapy trials include the first use of a lentiviral vector, the first infusions of gene edited cells, and the first use of lentivirally-modified cells to treat cancer. Dr. Levine has overseen the production, testing and release of 3,000 cellular products administered to > 1,200 patients in clinical trials since 1996. He is co-inventor of the first FDA approved gene therapy (Kymriah), chimeric antigen receptor T cells for leukemia and lymphoma, licensed to Novartis. Dr. Levine is co-inventor on 25 issued US patents and co-author of > 160 manuscripts and book chapters with Google Scholar citation h-index of 78. He is co-founder of Tmunity Therapeutics, a spinout of the University of Pennsylvania. Dr. Levine is President Elect of the International Society for Cell and Gene Therapy and serves on the Board of Directors of the Alliance for Regenerative Medicine. He has been interviewed by the NY Times, Wall Street Journal, Washington Post, NPR, Time Magazine, National Geographic, Forbes, BBC, and other international media outlets.
Dr. Tamara J. Laskowski
MD Anderson Cancer Center, USA | Senior Research Scientist
“High-throughput flow cytometry-based platform for interrogation of genetically-modified lymphocytes: a rapid and efficient method for assessing anti-tumor response”
Dr. Tamara J. Laskowski received her doctorate degree in Human Molecular Genetics and Immunology from University of Texas Health Science Center where her work focused on genome editing of patient-derived stem cells to correct genetic mutations causative of Wiskott-Aldrich Syndrome, an immunodeficiency disorder that results in severe impairments to the immune system. Subsequently, Tamara joined Dr. Laurence J.N. Cooper’s laboratory at MD Anderson Cancer Center as a fellow. Her work focused on engineering stem cells with the goal of generating off-the-shelf NK and T-cell immunotherapies for targeting solid tumor malignancies. Tamara’s work has led her to transition to Dr. James Allison’s Immunotherapy Platform in the Immunology department of MD Anderson. In her new role as Senior Research Scientist, Dr. Laskowski’s work primarily involves immune-monitoring of patients undergoing clinical trials I Immunotherapy and development of novel assays for interrogating tumor immune infiltrate. Dr. Laskowski also shares an interest in technology innovation, and has developed novel multi-plex assays for testing therapies against solid tumors. In 2017, this work led to an invitation to participate in the National Science Foundation (NSF) Innovation Corps, an exclusive program through which she was trained to develop strategies for expanding the economic and societal benefits of innovative ideas which have commercialization potential. She was the sole recipient of an award for outstanding performance upon completion of the program.
Dr. Richard Morgan
Cambridge, Massachusetts , USA
Editas Medicine, USA | Senior Vice President of Immunogenetics
Topic: “Pioneering the Possible”
Currently I am Senior Vice President Immunogenetics, Editas Medicine, Cambridge, MA and serve on the Board of Directors of the American Society of Gene and Cell Therapy. The majority of my career was spent in various role at the National Institutes of Health, Bethesda MD. As a member of the team that published the first approved human gene transfer experiment in 1990 (N. Engl. J. Med. 323:570, 1990), I have seen the gene therapy field grow and have grown with it. For the last 15 years, my research concentrated on cancer immunotherapy. I have extensively published on T-cell receptor (TCR) gene therapy and was the first author on the initial report where this technology was shown to lead to cancer regression in patients (Science 314:126, 2006). In 2013, I made the switch from academia to industry to lead the immunotherapy efforts at bluebird bio and recently moved to Editas Medicine to focus on gene editing applications in immunotherapy. As Vice President of Immunotherapy at bluebird bio, I oversaw research programs on various aspect of immunotherapy, including the development of bluebird’s lead oncology product, an anti-BCMA CAR T cell. At Editas Medicine, my goal is to develop cellular treatments that go beyond standard TCR/CAR-based antigen targeting, to produce gene edited effector cells designed to function within the hostile tumor microenvironment.
Dr. Verena Brucklacher-Waldert
Cambridge, United Kingdom
Principal Scientist at Horizon Discovery
“Gene-modulation technologies in the development of cell-based therapies”
Verena is a Principal Scientist at Horizon Discovery Ltd, based in Cambridge, UK. Horizon Discovery is a gene editing company that designs and engineers genetically modified cells and then applies them to aid clients engaged at every stage of the healthcare continuum, from sequence to treatment. Verena received her doctorate in Neuroimmunology from the University of Tuebingen, Germany, and gained a postdoctoral research fellowship from the German Research Foundation that enabled her to intensify her studies on T cell biology in Marc Veldhoen's lab in Cambridge, UK. Before Verena joined Horizon Discovery she developed assays for hit identification and lead characterization of multifunctional, oncology-relevant antibody-fragments for the biopharmaceutical company Crescendo Biologics. At Horizon Discovery she is providing high level scientific expertise and technical leadership for new and ongoing activities within the Immunology services team.
Dr. Nicolas Preyat
MaSTherCell, Belgium | Project and Development Manager
“Industrialization of the Manufacturing of a Gene Therapy Medicinal Product”
Nicolas Preyat has more than 10 years of experience in biotechnologies and management. He holds a master degree in biochemistry and molecular biology from the Université Libre de Bruxelles in Belgium. In the same university he completed his Ph.D. with a focus on the molecular regulation of cell death and the control of innate immune responses. Nicolas Preyat then further strengthens his scientific skills with post-doctoral studies where he investigated new therapeutic strategies to fight cancer. He also developed tools to characterize biomarkers for inflammatory diseases. Nicolas Preyat is now Project and Development Manager for Masthercell, a global contract development and manufacturing organization with a strong expertise in cell and gene therapy. Through the various positions he assumed, Nicolas Preyat has acquired a deep expertise in cell culture, analytical assays, technology transfer, good manufacturing practices and project management.
Dr. Yvonne Chen
Los Angeles, CA, USA
University of California | Assistant Professor
“Engineering Next-Generation Chimeric Antigen Receptors”
Dr. Yvonne Chen is an Assistant Professor in the Department of Chemical and Biomolecular Engineering at the University of California, Los Angeles. The Chen Laboratory focuses on applying synthetic biology and biomolecular engineering techniques to the development of novel mammalian-cell systems, particularly cell-based immunotherapy for cancer. Prior to joining the Department of Chemical and Biomolecular Engineering at the University of California, Los Angeles in 2013, Yvonne was a Junior Fellow in the Harvard Society of Fellows. Yvonne has been a recipient of the NIH Director’s Early Independence Award, the Hellman Fellowship, the ACGT Young Investigator Award in Cell and Gene Therapy for Cancer, and the NSF CAREER Award. She is also a Member Researcher in the Parker Institute for Cancer Immunotherapy. Yvonne earned her Ph.D. in Chemical Engineering from the California Institute of Technology, and completed postdoctoral training at the Center for Childhood Cancer Research within the Seattle Children’s Research Institute, and at the Department of Systems Biology at Harvard Medical School. She received her B.S. in Chemical Engineering from Stanford University.
Dr. Stephen Gottschalk
Memphis, Tennessee, USA
Chair, Department of Bone Transplant and Cellular Therapy at St. Jude Children's Research Hospital
“Engineering T cells for the immunotherapy of solid tumors”
Dr. Gottschalk is the Chair of the Department of Bone Marrow Transplant and Cellular Therapy at St Jude Children’s Research Hospital. He is a physician scientist, who is interested in hematopoietic stem cell transplantation and cell-based immunotherapies. He is leading a team of MD and PhD researchers, who are actively conducting clinical studies with antigen-specific T cells for patients with hematological malignancies, brain and solid tumors. In the laboratory, his team is focused on improving T-cell Therapy for cancer using genetic approaches. For his contributions to the fields of hematopoietic stem cell transplantation and cell-based immunotherapies he was elected into the American Society of Clinical Investigation.
Dr. Jean Peccoud
Fort Collins, Colorado, USA
Professor of Chemical and Biological Engineering
Abell Endowed Chair in Synthetic Biology
“Synthetic Biology: fostering the cyber-biological revolution”
Dr. Peccoud’s research program focuses on synthetic biology informatics. His group combines computational and experimental efforts to develop predictive models of behaviors encoded in synthetic DNA sequences. He is particularly interested in using methods from synthetic biology to optimize biomanufacturing processes used to produce biologic drugs, antibodies, and other proteins of commercial interest. Peccoud is also actively engaged in efforts to understand the security implications of synthetic biology.
Shortly after completing a graduate research project in molecular immunology, Jean Peccoud’s scientific interests shifted to computational biology. In 1989, he published one of the first articles describing a mathematical model of molecular noise in gene regulatory networks. In 1993, he recognized the importance of real-time PCR and developed new statistical techniques suitable to analyze this new type of data. In 2002, he observed with excitement the very early developments of synthetic biology. After exploring the potential applications of this new technology to plant biotechnology, he blazed a trail in synthetic biology informatics.
Jean Peccoud came to Colorado State University from the Virginia Bioinformatics Institute at Virginia Tech. He brought with him a diverse experience that includes working for Fortune 500 and start-up companies. He is the founding Editor-in-Chief of the journal Synthetic Biology published by Oxford University Press.
Dr. Wilson Wong
Cambridge, Massachusetts, USA
Assistant Professor at Boston University
“Mammalian Cell Design Using Synthetic Biology”
Wilson Wong is an Assistant Professor in the Biomedical Engineering Department at Boston University, and a core member of the BU Biological Design Center. The Wong is a design-driven lab is focused on developing synthetic biology tools in mammalian systems for cell-based immunotherapy. He received his B.S. degree in Chemical Engineering from UC Berkeley and a Ph.D. degree in Chemical Engineering from UCLA. He did his postdoctoral work at UCSF. He is the recipient of the NIH Director’s New Innovator, NSF CAREER, ACS Synthetic Biology Young Investigator, BU Ignition, and BU College of Engineering Early Career Research Excellence Award.
Yaron Fuchs, PhD
Technion Israel Institute of Technology, Israel
Sartorius & Science Grand Prize Winner
Born in Haifa, Israel, Dr. Fuchs is an Assistant Professor in the Faculty of Biology, the Lokey Interdisciplinary Center for Life Sciences & Engineering and the Technion Integrated Cancer Center. Dr. Fuchs is also a Deloro Career Advancement Chair and an Alon Fellow.
He began his academic career at Haifa University where he received a B.Sc., followed by a direct Ph.D. track for outstanding students, which was conducted at the Technion Israel Institute of Technology. Upon completion of his doctorate degree, he performed his post-doctoral research at The Rockefeller University and Howard Hughes Medical Institute. Recently he returned to the Technion where he heads the Laboratory of Stem Cell Biology and Regenerative Medicine.
Dr. Fuchs has had a long-term interest in different modes of cell death and how they regulate diverse aspects of stem cell biology and stem cell-dependent processes. His research has taken advantage of state of the art mouse models where he manipulates and traces different stem cell populations.
Throughout his career, Dr. Fuchs has received more than 20 awards for his scientific excellence and his unique teaching style. He has published in leading scientific journals and has spoken nationally and internationally about his own research focusing on adult stem cells in regeneration and cancer.
Adult stem cells are characterized by their ability to self-renew and differentiate into distinct cell types, positioning them as critical drivers of tissue replenishment and repair. While great strides have been made in our understanding of the various pathways that control the fate and function of stem cells, very little is known regarding the specific mechanisms that govern their elimination. By investigating the hair follicle and intestine, two systems which rely heavily on stem cells, I discovered key proteins that regulate stem cell apoptosis (programmed cell death). Importantly, I found that we can harness the incredible potential of stem cell apoptosis to drive tissue repair and regeneration. In this talk, I also discuss the effect dying cells have on their cellular environment and the critical non-apoptotic roles of proteins that have been classically considered to drive cell death.
It is my hope that these findings may be translated into novel stem cell and apoptosis-based approaches for regenerative medicine and tumor therapy.
C. Florian Bentziger, PhD
Assistant Professor in the Department of Pharmacology-Physiology at Université de Sherbrooke
Sartorius & Science Prize Finalist
Florian Bentzinger is an Assistant Professor in the Department of Pharmacology-Physiology of the Université de Sherbrooke in Canada. His lab studies the skeletal muscle stem cell niche in health and disease. Florian received his master's and PhD degree in molecular biology with a minor in pharmaceutical biology under the supervision of Prof. Markus Rüegg from the University of Basel in Switzerland. During his early studies he focused on the molecular mechanisms underlying different forms of muscular dystrophy. He then became a postdoctoral researcher under the direction of Prof. Michael Rudnicki at the Ottawa Hospital Research Institute (OHRI), Sprott Center for Stem Cell Research in Canada, and began to focus on the microenvironmental regulation of skeletal muscle stem cells. Before his appointment at the Université de Sherbrooke, Prof. Bentzinger held a permanent position in the Skeletal Muscle Aging Department of the Nestlé Institute of Health Sciences in Lausanne, Switzerland.
Stem cells in adult tissues are controlled by intrinsic programming and extrinsic signals provided by the surrounding microenvironment, often referred to as the niche. Particularly, stem cells that maintain and repair skeletal muscle tissue are strongly dependent on extrinsic regulation. Fundamental properties of these cells, such as quiescence, self-renewal and the ability to differentiate, are largely determined by the composition of the niche. Diseases of the musculature lead to changes in the niche that negatively affect stem cell function. Importantly, alterations in the stem cell microenvironment also underlie the impaired regenerative capacity of muscle tissue that accompanies aging and certain multisystemic conditions. In spite of the importance of the muscle stem cell niche, its architecture and the regulatory signals it generates remain poorly understood. We recently started to systematically analyze the composition of the muscle stem cell niche and provide examples of how these insights can be exploited for therapeutic applications.
Ritu Raman, PhD
Cambridge, Massachusetts, USA
Postdoctoral Fellow at Massachusetts Institute of Technology
Sartorius & Science Prize Finalist
Ritu Raman is a AAAS L’Oréal USA For Women in Science postdoctoral fellow in the lab of Prof. Robert Langer at MIT. She is passionate about understanding and utilizing the dynamic adaptive nature of biological systems, and aims to establish a lab designing responsive biohybrid implantable devices that improve human health and quality of life. Ritu graduated magna cum laude with a degree in Mechanical Engineering from Cornell University in 2012, and earned her M.S. (2013) and Ph.D. (2016) as an NSF Graduate Fellow from the University of Illinois at Urbana-Champaign. She has received several awards for her commitment to scientific innovation, including being named to the Forbes 30 Under 30 list for Science, nominated as an Innovation and Technology Delegate to the International Achievement Summit, shortlisted for the Nature Research + Estée Lauder Inspiring Science Award, and awarded the Illinois Innovation $15k Prize. Ritu grew up in India, Kenya, and the United States, and learned to appreciate and thrive in diverse and dynamic environments. Her experiences have taught her that technical innovation drives positive social change, and this inspires her to democratize and diversify STEM education around the world. She is deeply invested in science communication, policy, and outreach, and has been recognized with several honors for her commitment to advancing underrepresented minorities in STEM, including receiving the highest collegiate honor from the Society of Women Engineers and being named the Cambridge Science Festival’s Curious Scientist of the Year.
Disease or damage that limits the functionality of skeletal muscle severely impacts human health, mobility, and quality-of-life. There is thus a critical need to uncover the underlying structure and cell-cell communication that drives the formation, maturation, and responsive behaviors of muscle. We have developed a mesoscale in vitro skeletal muscle model that enables ready visualization of cell-cell communication and tissue-wide coordinative function. The model is composed of engineered tissue coupled to a flexible 3D printed skeleton. Contraction of the tissue deforms the skeleton, providing a straightforward visual and quantitative measure of tissue functionality. This enables testing the effect of various biochemical and mechanical stimuli and optimizing tissue viability, maturation timeline, and force production. Our model offered the first proof that light stimulation “exercise” of optogenetic muscle could enhance force production, and the first demonstration of bidirectional locomotion and rotation in an engineered skeletal muscle-powered system. It also served as a test platform to design and optimize a protocol to guide and accelerate muscle healing after induced damage. This system can be used to develop a deeper understanding of muscle development and adaptation, and we are optimistic these insights will aid efforts to replace and recover muscle loss-of-function in vivo.
Dr. Daniele VF Tauriello
Institute for Research in Biomedicine, Spain
Sartorius & Science Prize Finalist
Daniele received his degrees (Chemistry and Biomolecular Sciences) and his doctorate (Cell Biology) from Utrecht University and the University Medical Centre Utrecht, the Netherlands. During his doctoral research, he used and developed biochemical tools to study molecular mechanisms in receptor-proximal Wnt signaling. While a postdoctoral fellow at the Institute for Research in Biomedicine (IRB) Barcelona, Spain, he developed an immunocompetent, metastatic and transplantable mouse model for intestinal cancer, and leveraged this new platform to uncover the immuno-evasive role of TGFβ in the tumour immune microenvironment of colorectal cancer metastasis. His work may contribute to an expanded efficacy of checkpoint inhibition therapy across patient cohorts and cancer types. In 2019, Daniele will set up his lab at the Radboud Institute for Molecular Life Sciences (RIMLS), Radboudumc in Nijmegen, the Netherlands. There he will continue his immuno-oncological work on overcoming stromal immune evasion mechanisms to improve immunotherapy in cancer.
Metastatic cancer cells must reprogram the tumour stroma, or tumour microenvironment, before they can metastasize. Tumours must also contend with the immune system before they can spread. If the road to metastasis is full of obstacles, how is it so common? The answer probably lies in the fact that as cancer cells reorganize their environment, they thoroughly manipulate the stroma to not just shut down any recruited immune cells, but to suppress the mobilization of an effective immune response in the first place.
I generated a mouse model of metastatic colorectal cancer that recapitulates relevant characteristics of human metastatic disease, and that can be transplanted by the use of tumour organoids. Leveraging this immunocompetent platform, we demonstrated the role of TGFβ signaling in suppressing the immune response. Blockade of this pathway led to T-cell-mediated immunity, preventing metastatic initiation in the liver.
Moreover, TGFβ blockade rendered late-stage metastases susceptible to immune checkpoint inhibition treatment. Whereas this form of therapy failed by itself—as it has in the clinic for most patients with metastatic colorectal cancer—the combined therapy synergized to cure the majority of mice with advanced liver metastases. Thus, our results indicate that immunotherapies can be made more efficient for a wider range of patients by understanding and neutralizing immune evasion mechanisms in the tumour microenvironment.
Awards Ceremony for the Winners of the Sartorius & Science Prize
The winner and three finalists competing for the Sartorius & Science Prize for Regenerative Medicine & Cell Therapy received their awards at the ceremony held in their honor.
Congratulations to Grand Prize winner Yaron Fuchs and to the three finalists C. Florian Bentzinger, Ritu Raman and Daniele Tauriello. A big thank you goes out to all participants for making this awards ceremony such a special event.
Panel Discussion: Present and future of CAR-T
What are novel technologies that are coming up that are interesting, that are exciting in terms of, helping scientists discover CAR-T therapies or cell therapies? These were among the questions asked during the panel discussion: Present and Future of CAR-T therapies.
The panelists were Dr. Bruce Levine, Ph.D.; Dr. Rick Morgan, PhD; Dr. Yvonne Chen, PhD and Dr. John O’Rourke, PhD. The discussions were moderated by Dr. Tom Duensing, Head of BioAnalytics Product Development at Sartorius.