emerging cellular therapies: t cells and beyond

Scientific Organizers: Carl H. June, University of Pennsylvania, USA Marcela V. Maus, Harvard Medical School, USABruce R. Blazar, University of Minnesota, USA

Joint with the meeting on Lymphocytes and their Roles in Cancer

A new community of researchers is emerging that focuses on studying and developing cellular therapies for cancer and chronic infections. The field is advancing rapidly due to our deepening understanding of cell biology. New technologies permit efficient cell culture, gene transfer and genetic editing, enabling autologous and allogeneic cell therapy. With rapid development of CAR T cells, breakthrough FDA approval status and fast-tracking clinical trials, competition is fierce in the biotechnology and pharmaceutical industries. Cell therapies with stem cells and T cells are in advanced clinical development that will enable widespread use for cancer and chronic infections such as HIV. Infusions of regulatory T cells (Tregs) have the potential to benefit patients with autoimmunity and transplantation. A goal of this conference is to bring together basic and translational scientists from these fields to identify current opportunities and challenges in cell therapies. Another goal is to bridge the fields of effector T cell and Treg cell therapies. The interdisciplinary features of this meeting should facilitate new collaborations and interactions between investigators who would not normally meet.

Session Topics:• CAR/TCR Preclinical • Workshop 1: Perspectives from Industry and FDA: Roundtable• Synthetic Biology • Engineered T Cells for HIV • CAR/TCR in the Clinic for Cancer • Tregs as Cellular Therapeutics • Workshop 2: Late-Breaking Talks• Beyond T Cells: HSC, T Progenitor and IPSCs I & II• Intersection of Genetic Editing and Cellular Therapy

Scholarship Application & Discounted Abstract Deadline: October 11, 2017Abstract Deadline: November 9, 2017Discounted Registration Deadline: December 13, 2017

February 11–15, 2018 | Keystone Resort | Keystone, Colorado | USA

Emerging Cellular Therapies: T Cells and Beyond

www.keystonesymposia.org/meetings | 1.800.253.0685 | 1.970.262.1230

a 501(c)(3) nonprofit educational organization

Note: Scholarships are available for graduate students and postdoctoral fellows and are awarded based on the abstract submitted. Submitting an abstract is an excellent opportunity to gain exposure for your work. Abstracts submitted by the abstract deadline will also be considered for short talks on the program.Upper image of vaccine-based immunotherapy from novel nanoparticle systems courtesy of Victor Segura Ibarra and Rita Serda, PhD, National Cancer Institute, NIH

Meeting Hashtag: #KScelltherapy www.keystonesymposia.org/18B6

SUNDAY, FEBRUARY 11Arrival and Registration

MONDAY, FEBRUARY 12Welcome and Keynote Address (B6)*Carl H. June, University of Pennsylvania, USA*Bruce R. Blazar, University of Minnesota, USA*Marcela V. Maus, Harvard Medical School, USATon N. Schumacher, Netherlands Cancer Institute, NetherlandsT Cell Recognition in Human Cancer

Welcome and Keynote Address (R1)*Iannis Aifantis, New York University School of Medicine, USAAlexander Y. Rudensky, HHMI/Memorial Sloan Kettering CancerCenter, USARegulatory T Cells and their Functions in the Tumor Microenvironment

CAR/TCR Preclinical (B6)*Carl H. June, University of Pennsylvania, USAPhilip D. Greenberg, University of Washington, USAMaking TCR-Modified T Cells Effective for Leukemia and Solid TumorsCrystal L. Mackall, Stanford University, USAWhat's Next? Preclinical Studies Paving the Way for Future CAR TCell TrialsStanley R. Riddell, Fred Hutchinson Cancer Research Center,University of Washington, USAStrategies to Improve the Efficacy of CAR T Cells in Solid Tumors Reno Debets, Erasmus MC Cancer Institute, NetherlandsShort Talk: T Cell Receptors Equipped with ICOS Enhance T CellPersistence and Mediate Sustainable Anti-Tumor Responses uponAdoptive T Cell TherapyJames Scott-Browne, La Jolla Institute, USAShort Talk: Transcriptional Control of CAR-T Cell Function in SolidTumors

Understanding Lymphocytic Malignancy: Leukemia andLymphoma (R1)*Adolfo Ferrando, Columbia University, USALouis M. Staudt, NCI, National Institutes of Health, USAThe MYD88-TLR9-B Cell Receptor Complex as a Target for Therapyin Aggressive LymphomasCharles G. Mullighan, St. Jude Children's Research Hospital, USAThe Role of CREBBP Mutations in Drug Resistance in AcuteLymphoblastic LeukemiaKatia Georgopoulos, Massachusetts General Hospital, USASuper-Enhancer Circuits in B Cell Development and Transformation

Anastasia N. Tikhonova, New York University Medical School, USAShort Talk: Single-Cell Dynamics of Bone Marrow Microenvironment

Workshop 1: Perspectives from Industry (B6)*Charles Sentman, Geisel School of Medicine at Dartmouth, USAAmy Gilbert, Cell Design Labs, USADevelopment of a SynNotch Combinatorial “and” Gate CAR T forSolid TumorsIan R. Hardy, TCR2 Therapeutics, USAImproving CAR-T Cell Function with Small Molecule Treatment duringProductionKelan Hlavaty, SQZ Biotech, USAEngineering T Cells Using a Microfluidic Intracellular Delivery Methodfor Cell TherapyRobert J. Hofmeister, TCR2 Therapeutics, USAA Novel T Cell Therapy Engaging the Complete T Cell ReceptorLenka V. Hurton, ZIOPHARM Oncology, USARapid Production of T Cells Co-expressing CAR andMembrane-bound IL-15 Potentiates Antitumor Activity and Promotesin vivo MemorySumiti Jain, Sangamo Therapeutics, USAPrecise T Cell Engineering using Enhanced Zinc-finger Nucleases(ZFNs) for Highly Specific and Efficient Dual Knock-Out ofEndogenous T-Cell Receptor and HLA-Class I with Site-SpecificInsertion of a CD19-Inez Johanna, University Medical Center Utrecht, NetherlandsTowards the Next Generation CAR T Cells with TEGs: In vivo Efficacy– Toxicity Profile in Xenografts of Primary Human AML Disease andHealthy Bone MarrowLevi Rupp, Cell Design Labs, USATHROTTLETM CARs: A Novel Platform for Enhanced Safety andEfficacy via Titratable Control of CAR-T Activity using anFDA-Approved Small Molecule

Workshop 1: Understanding and Targeting Lymphocytes inCancer (R1)*Iannis Aifantis, New York University School of Medicine, USAEirini Giannakopoulou, Radiumhospitalet, NorwayT Cell Receptor-Mediated Targeting of Recurrent Neoantigens inAcute Myeloid LeukemiaWenting Zheng, St. Jude Children Research Hospital, USAPI3K Orchestration of the in vivo Persistence of Chimeric AntigenReceptor-Modified T CellsElena Montauti, Northwestern University, USAUSP22 Maintains Regulatory T Cells by Stabilizing Foxp3 ProteinLevel and Impairs Anti-Tumor Immune ResponseJeffrey Ward, Washington University School of Medicine, USARemodeling of Myeloid as well as Lymphoid Compartments followingCheckpoint Blockade Immunotherapy

* Session Chair † Invited but not yet accepted Program current as of May 30, 2022. Meal formats are based on meeting venue. For the most up-to-date details, visit https://www.keystonesymposia.org.

KEYSTONE SYMPOSIAon Molecular and Cellular Biology

Emerging Cellular Therapies: T Cells and Beyond (B6)Scientific Organizers: Carl H. June, Marcela V. Maus and Bruce R. Blazar

Sponsored by BioLegend, Inc., Editas Medicine, Gilead Sciences, Inc., Juno Therapeutics, Regeneron Pharmaceuticals, Inc.,Sangamo Therapeutics, Inc. and Takeda Pharmaceutical Company Limited. This activity was supported by an educational grant

from Celgene Corporation.Lymphocytes and their Roles in Cancer (R1)

Scientific Organizers: Iannis Aifantis, Ugur Sahin and Mikala EgebladFebruary 11-15, 2018 • Keystone Resort • Keystone, CO, USA

Sponsored by Amgen Inc., BioLegend, Inc. and Incyte CorporationDiscounted Abstract & Scholarship Deadline: October 11, 2017 / Abstract Deadline: November 9, 2017 / Discounted Registration Deadline: December 13, 2017

Soki Kashima, Kyoto University, JapanWT1-Specific Cytotoxic T Lymphocytes Regenerated from TCell-Derived iPS Cells Exert Therapeutic Effect in Xenograft Model ofRenal Cell CarcinomaChris Nicolai, University of California, Berkeley, USAMechanisms of MHC-Deficient Tumor Clearance using STINGAgonistsJulie Rytlewski, Bristol-Myers Squibb, USAHistory of Prior Immunotherapy Changes Relationship betweenTumor Mutations and TIL RepertoireAngela Vasaturo, INSERM UMRS 1138, FranceUnraveling the Immune Contexture of Pre-Invasive Lesions of theLung by Multispectral Imaging

Synthetic Biology (B6)*Crystal L. Mackall, Stanford University, USADarrell J. Irvine, Massachusetts Institute of Technology, USAChemically Engineering T Cell TherapiesWendell A. Lim, University of California, San Francisco, USAOptimizing Synthetic Biology Tools for T Cell TherapiesTravis Young, California Institute for Biomedical Research, USAControllable CAR-T Cell TherapyPatrick Ho, University of California, Los Angeles, USAShort Talk: COVERT T Cells: Engineering T Cells to InterrogateIntracellular Antigens

Therapeutic Targeting of Lymphocytic Malignancy (R1)*Charles G. Mullighan, St. Jude Children's Research Hospital, USAAdolfo Ferrando, Columbia University, USAEpigenetic Mechanisms in T-Cell Acute Lymphoblastic LeukemiaIannis Aifantis, New York University School of Medicine, USATargeting Stress Responses in Acute Lymphocytic LeukemiaAndrei Thomas-Tikhonenko, Children's Hospital of Philadelphia andUniversity of Pennsylvania, USAEscape Hatches for Immunotherapies Targeting B-Cell DifferentiationAntigensJacques G. M. Ghysdael, Institut Curie, FranceShort Talk: Triggering the Tcr Developmental Checkpoint Activates aTherapeutically Targetable Anti-Leukemic Pathway in T-Cell AcuteLymphoblastic Leukemia

Poster Session 1

TUESDAY, FEBRUARY 13Engineered T Cells for Viruses (B6)*Philip D. Greenberg, University of Washington, USACatherine M. Bollard, Children’s National Health System, USADeveloping HIV-Specific T Cell Therapies: Lessons from EBV

Paula M. Cannon, University of Southern California, Keck School ofMedicine, USAShutting the Door on HIV by Engineering Hematopoietic Stem Cells James L. Riley, University of Pennsylvania, USAEngineering T Cells to Functionally Cure HIV-1 InfectionScott G. Kitchen, University of California, Los Angeles AIDS Institute,USAEngineering Antiviral CAR T Cell Immunity through HematopoieticStem/Progenitor CellsAlok Vishnu Joglekar, California Institute of Technology, USAShort Talk: Hematopoietic Stem/Progenitor Cells Engineered with TCell Receptors for Immunotherapy for HIV Infection

Cellular Therapies in Blood Malignancies and Beyond (R1)*Ugur Sahin, BioNTech AG, GermanyCarl H. June, University of Pennsylvania, USACAR T Cells and their Therapeutic Promise in the Treatment ofLeukemia Crystal L. Mackall, Stanford University, USACAR Vulnerabilities: Emerging Insights from Clinical TrialsChiara Bonini, Vita-Salute San Raffaele University, ItalyGene Editing of Memory Stem T Cells for Cancer Immunotherapy Justin Eyquem, University of California, San Francisco, USAPrecision T Cell Engineering to Advance Adoptive T Cell Therapies Charlotte Mousset, Radboudumc, NetherlandsShort Talk: Superior Multi-Functionality and Glycolytic Function ofTumor-Reactive CD8+ TSCM-Like T Cells by ex vivo Akt-Inhibition

CAR/TCR in the Clinic for Cancer (B6)*Jeffrey S. Miller, University of Minnesota, USAStephen J. Forman, City of Hope, USACAR T Cells for Treatment of Central Nervous System (CNS)MalignancyMarcela V. Maus, Harvard Medical School, USACAR T Cells Multiple Myeloma and GlioblastomaSaar I. Gill, University of Pennsylvania, USACAR T Cells for Acute Myelogenous LeukemiaJenessa Barbara Smith, Poseida Therapeutics, USAShort Talk: PSMA-Specific CAR T-Stem Cell Memory TherapyEliminates Solid Tumor in Prostate Cancer Model








Lymphocytes as Elements of the Tumor Microenvironment (R1)*Katia Georgopoulos, Massachusetts General Hospital, USADavid H. Raulet, University of California, Berkeley, USANatural Killer Cell Activation and Inhibition: Potential for CancerTherapyMichael Khodadoust, Stanford University, USAT Cell Recognition of Lymphoma ImmunoglobulinMikala Egeblad, Cold Spring Harbor Laboratory, USAVisualization of the Switch from Immune Control to Immune EscapeAnnelise Snyder, University of Washington, USAShort Talk: Anti-tumor Effects of Inflammatory Necroptosis Within theTumor Microenvironment

Poster Session 2

WEDNESDAY, FEBRUARY 14Tregs as Cellular Therapeutics (B6)*James L. Riley, University of Pennsylvania, USABruce R. Blazar, University of Minnesota, USAVimentin Restrains Regulatory T-cell Suppression ofGraft-versus-Host Disease Jeffrey A. Bluestone, University of California, San Francisco, USAManipulating Tregs to Control Tolerance in Autoimmunity and CancerMegan K. Levings, University of British Columbia, CanadaHLA-A2 Directed CAR TregsDavid Klatzmann, Sorbonne University Medical School, FranceIL-2 for Treg-Based Immunotherapies: In (and Out)Angela C. Boroughs, ArsenalBio, USAShort Talk: 4-1BB Co-Stimulation Inhibits CAR Treg SuppressiveFunctionElmar Jaeckel, Hannover Medical School, GermanyShort Talk: Operational Tolerance in Allotransplantation by Use ofRegulatory T Cells with a MHC-Specific Chimeric Antigen Receptor

Emerging Technologies: Visualization and Dissection of theTumor Microenvironment (R1)*Mikala Egeblad, Cold Spring Harbor Laboratory, USADana Pe'er, Memorial Sloan Kettering Cancer Center, USAA Single Cell Approach to Understanding the Tumor MicroenvironmentMatthew F. Krummel, University of California, San Francisco, USAImaging a World beyond Checkpoints: Innate Partners forLymphocytes in Cancer

Jerome Galon, INSERM UMRS1138, Cordeliers Research Center,FranceImmunoscore as a Stronger Predictor of Patient Survival in ColorectalCancer Michael Goldberg, Dana-Farber Cancer Institute, USAEnhancing Cancer Immunotherapy through Immuno-engineering

Workshop 2: Cell Engineering (B6)*Martin Pule, University College London, UKZinaida Good, Stanford University, USAGuiding T-Lymphocyte Differentiation in Cancer ImmunotherapyApplicationsRachel C. Lynn, Lyell Immunopharma, USADisrupting the Balance: Engineering AP1 to Overcome CAR T CellExhaustionShannon K. Oda, Fred Hutchinson Cancer Research Center, USACheating Death: A Fas-4-1BB Immunomodulatory Fusion ProteinObviates a Death Signal to Enhance T Cell Function and AdoptiveTherapy Targeting Leukemia and Solid TumorsWilliam Lemieux, University of Montréal, CanadaEfficient Transduction of Nk Cells Method Resulting in a Robust andSustained Transgene Expression for ImmunotherapyMasataka Suzuki, Baylor College of Medicine, USAAdenovirotherapy Delivering Cytokine and Checkpoint InhibitorAugments Chimeric Antigen Receptor T-Cell against Metastatic Headand Neck CancerTori Yamamoto, NCI, University of Pennsylvania, USACell Intrinsic Fas Signaling Inhibition to Improve Therapeutic Efficacyof T CellsNicholas F. Kuhn, Memorial Sloan Kettering Cancer Center, USADesign of a Systemic Antitumor Response with CD40L-modified CART CellsJoy (Yushu) Xie, Massachusetts Institute of Technology, USAEngineering Single Domain Antibody-Based Chimeric AntigenReceptor T Cells for Treatment of Solid Tumors

Beyond T Cells: HSC, T Progenitor and IPSCs I (B6)*Juan Carlos Zúñiga-Pflücker, Sunnybrook Research Institute,CanadaMartin Pule, University College London, UKNew Designs in Synthetic T CellsJeffrey S. Miller, University of Minnesota, USANovel Ways to Activate and Target NK Cells to Treat CancerLinda T. Vo, University of California, San Francisco, USAT Cell Engineering from Human Pluripotent Stem Cells








Emerging Concepts in the Development of Tumor Vaccines (R1)*Dimitris Skokos, Regeneron Pharmaceuticals, USAUgur Sahin, BioNTech AG, GermanySystemic RNA Delivery, Dendritic Cell Vaccines and Implications forCancer ImmunotherapyCornelis J. M. Melief, Leiden University Medical Center & ISAPharmaceuticals BV, NetherlandsCombination Immunotherapy of Cancers Caused by High-Risk HPV16Johanna Olweus, University of Oslo, NorwaySelecting Immunogenic Targets for Cancer ImmunotherapySamarth Hegde, Icahn School of Medicine at Mount Sinai, USAShort Talk: Tumor Antigenicity Accelerates Early PancreaticAdenocarcinoma Progression

Poster Session 3

THURSDAY, FEBRUARY 15Beyond T Cells: HSC, T Progenitor and IPSCs II (B6)*Bruce R. Blazar, University of Minnesota, USAJuan Carlos Zúñiga-Pflücker, Sunnybrook Research Institute,CanadaGeneration of Human Progenitor T Cells from Stem CellsSjoukje J.C. van der Stegen, Fate Therapeutics, USADevelopment of CD8alphabeta+ CAR T Cells from Human PluripotentStem CellsJakub Tolar, University of Minnesota, USAGene-Edited Stem Cells: From Cloning to ClinicSaar I. Gill, University of Pennsylvania, USAChimeric Antigen Receptor Macrophages for the Treatment of SolidTumorsKatelyn Masiuk, University of California, Los Angeles, USAShort Talk: Hematopoietic Stem Cell Gene Therapy for IPEXSyndrome

Combinatorial Treatments and their Effects on LymphocyteFunction in Cancer (R1)*Ashley M. Laughney, Weill Cornell Medicine, USADavid G. DeNardo, Washington University Medical School, USAThe Pancreatic Tumor Microenvironment; Modulator of TumorProgression and Response to TherapyDimitris Skokos, Regeneron Pharmaceuticals, USAT Cell Dysfunction and Combination ImmunotherapyMichael A. Curran, University of Texas MD Anderson Cancer Center,USAOptimizing T Cell Frequency and Function via Checkpoint Antibody /Vaccine Combinations

Sjoerd H. van der Burg, Leiden University Medical Center,NetherlandsCombination Treatments to Modulate the Microenvironment and BoostTumor-Specific T Cells Yuliya Pylayeva-Gupta, University of North Carolina at Chapel Hill,USAShort Talk: IL-35+ B Cells Establish Immunosuppressive Network inPancreatic Ductal Adenocarcinoma

Intersection of Genetic Editing and Cellular Therapy (B6)*Marcela V. Maus, Harvard Medical School, USAAlex Marson, University of California, San Francisco, USAReprogramming Human T Cell CircuitryAndrew M. Scharenberg, Umoja Biopharma, USAEngineering Regulatory T Cells for Inflammatory DisordersCarl H. June, University of Pennsylvania, USAAdoptive Transfer with Genetically Edited T Cells

Future Directions for Next-Generation Sequencing: Genomics,Epigenetics and Immunogenomics (R1)*Iannis Aifantis, New York University School of Medicine, USAAshley M. Laughney, Weill Cornell Medicine, USADissecting Tumor Cell Plasticity and Population InteractionsSupporting Metastasis using Single Cell GenomicsMickey Atwal, Cold Spring Harbor Laboratory, USAHigh-Throughput Profiling of T Cell Receptor Sequences in CancerImmunotherapyRahul Satija, New York Genome Center, USAIntegrated Analysis of Single Cell Data across Modalities,Technologies and Species

Meeting Wrap-Up: Outcomes and Future Directions (Organizers)(B6)Meeting Wrap-Up: Outcomes and Future Directions (Organizers)(R1)

FRIDAY, FEBRUARY 16Departure








emerging cellular therapies: t cells and beyond

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