Keystone Symposia

Fairmont Banff Springs Floorplan

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Engineering the Genome (Q2)


Organizer(s) Vic Myer and Erik Sontheimer
February 8—12, 2020
Fairmont Banff Springs • Banff, AB Canada
Discounted Abstract Deadline: Oct 29, 2019
Abstract Deadline: Nov 6, 2019
Scholarship Deadline: Oct 29, 2019
Discounted Registration Deadline: Dec 10, 2019

Sponsored by Editas Medicine and Novo Nordisk A/S


Summary of Meeting:
Genome editing is already transforming biological science and promises to do the same for human medicine. Technological capabilities are advancing rapidly and there are expected to be more than 30 genome editing therapies in clinical development by 2020. It is therefore important that practitioners in both academia and industry have the opportunity to learn about the latest improvements and applications. This conference will bridge that gap and provide an ideal environment for scientific exchange. In addition, participants will be educated on the growing roster and utility of genome engineering platforms. There will be sessions on genome editing’s clinical advancements and the evolution of thinking in the field regarding translational sciences. This meeting is being paired with Emerging Cellular Therapies so that participants can come together with researchers who focus on cell-based therapies. Therefore, we anticipate attendees will have a greater awareness of the full range of genome engineering tools, both established and emerging and knowledge of best practices and pitfalls in clinical advancement.

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No registration fees are used to fund entertainment or alcohol at this conference

Conference Program    Print  |   View meeting in 12 hr (am/pm) time


The meeting will begin on Saturday, February 8 with registration from 16:00 to 20:00 and a welcome mixer from 18:00 to 20:00. Conference events conclude on Wednesday, February 12 with a closing plenary session from 17:00 to 19:00, followed by a social hour and entertainment. We recommend return travel on Thursday, February 13 in order to fully experience the meeting.

SATURDAY, FEBRUARY 8

16:00—20:00
Arrival and Registration

Van Horne Foyer
18:00—20:00
Welcome Mixer
No registration fees are used to fund alcohol served at this function.

Van Horne Foyer

SUNDAY, FEBRUARY 9

07:00—08:00
Breakfast

Mezzanine 2
08:00—09:30
Welcome and Keynote Session (Joint)

Van Horne A/B
* Vic E. Myer, Atlas Ventures, USA

* Crystal L. Mackall, Stanford University, USA

Jennifer A. Doudna, HHMI/University of California, Berkeley, USA
Genome Editing with CRISPR-Cas Systems: Challenges and Opportunities in a New Era of Biology

Carl H. June, University of Pennsylvania, USA
Updates with Engineered T Cells

Coffee Break

09:50—12:00
Biochemistry, Biophysics and New Enzymes

Van Horne C
Kira S. Makarova, National Center for Biotechnology Information, USA
In silico Discovery of New Defense Systems

* Erik Sontheimer, University of Massachusetts Medical School, USA
RNA-Guided RNA/DNA Cleavage by CRISPR-Cas Systems

Ilya J. Finkelstein, University of Texas at Austin, USA
Massively-Parallel Profiling of Natural and Engineered High-Fidelity CRISPR Nucleases

Ervin Welker, Research Center of Natural Sciences, Hungary
Short Talk: Ranking between Increased Fidelity SpCas9 Nucleases and Target Sites

Garrett R. Rettig, Integrated DNA Technologies, USA
Short Talk: Engineered A.s. and L.b. Cas12a (Cpf1) Variants with Enhanced Activity

09:50—12:00
Genetically Engineered T Cell Therapies for Cancer: Results from the Clinic

Van Horne A/B
* Chiara Bonini, Ospedale San Raffaele, Italy

Stanley R. Riddell, Fred Hutchinson Cancer Research Center, University of Washington, USA
CAR T Cells for Solid Tumors

Yvonne Y. Chen, University of California, Los Angeles, USA
Synthetic Biology to Engineer More Potent CAR T Cells

Kristen Hege, Celgene, USA
Clinical Development of BCMA Targeted Immunotherapies in Multiple Myeloma

Brooke Prinzing, St. Jude Children's Research Hospital, USA
Short Talk: Chimeric Antigen Receptors with a MyD88 and CD40 Costimulatory Endodomain Endow T Cells with Superior Antitumor Activity

Evan William Weber, Stanford University, USA
Short Talk: The Src Kinase Inhibitor, Dasatinib, Enhances CAR-T Cell Potency by Mitigating T Cell Exhaustion

12:00—17:00
On Own for Lunch

12:00—13:00
Poster Setup

Mezzanine 2
13:00—22:00
Poster Viewing

Mezzanine 2
14:30—16:30
Panel: Ethics of Genome Editing

Van Horne C
* Tim Hunt, Editas Medicine, USA
Ethics of Genome Editing: Framing the Discussion

Fyodor D. Urnov, University of California, Berkeley, USA

Kathy K. Niakan, Francis Crick Institute, UK

Francoise Baylis, Dalhousie University, Canada

Krishanu Saha, University of Wisconsin-Madison, USA

14:30—16:30
Workshop 1: Platforms for Immune Cell Engineering

Van Horne A/B
* Manfred Lehner, Children's Cancer Research Institute, Austria

Carla Alicia Jaeger-Ruckstuhl, Fred Hutchinson Cancer Research Center, USA
The Role of CD27 Co-Stimulation during CAR-T Cell Generation and Immunotherapy

Benjamin Salzer, Children's Cancer Research Institute, Austria
AvidCARs – A Novel Platform for Inducible and Combinatorial CAR Control

Alec Wilkens, Fred Hutchinson Cancer Research Center, USA
NOTCH1 Agonism Alters Phenotype and Function of Gene Engineered CD4+ T Cells

Michela Consonni, Fondazione Centro San Raffaele, Italy
Donor-Unrestricted Adoptive T Cell Therapy of Acute Leukemia by CD1c-Restricted TCR Transfer

Amy N. Courtney, Baylor College of Medicine, USA
NKT Cells Suppress Metastatic Spread of Neuroblastoma by Controlling Tumor-Associated Macrophages

Hyun Cheol Jung, Korea Advanced Institute of Science and Technology, South Korea
Elimination of Amyloid-beta Plaques by Chimeric Phagocytic Receptors

Charlotte U. Brey, Children's Cancer Research Institute, Austria
A Conformation-Specific ON-Switch for Controlling CAR T Cells with an Orally Available Drug

16:30—17:00
Coffee Available

Van Horne Foyer
17:00—19:00
In vivo Genome Editing

Van Horne C
* Fyodor D. Urnov, University of California, Berkeley, USA

James M. Wilson, University of Pennsylvania, USA
In vivo Editing to Primate Liver

Edward J. Rebar, Sangamo Therapeutics, Inc., USA
Liver Editing in vivo for Therapeutic Gene Expression

Jessica Seitzer, Intellia Therapeutics Inc., USA
Short Talk: In vivo Delivery of CRISPR/Cas9 to the Liver using Lipid Nanoparticles Enables Gene Knockout across Multiple Targets in Rodent and Non-Human Primates

David V. Schaffer, University of California, Berkeley, USA
Engineering and Evolving New Viruses for Gene Therapy and Genome Editing

Jessie R. Davis, Harvard University, USA
Short Talk: Cytosine and Adenine Base Editing of Brain, Retina, Heart, Muscle, and Liver Mediated by AAV

17:00—19:00
Engineered Hematopoietic Stem Cells for Treatment of Genetic Diseases

Van Horne A/B
* Gloria Delfanti, Fondazione Centro San Raffaele, Italy

Marina Cavazzana, Paris Descartes University, France
Gene Therapy for Immunodeficiencies

Alessandra Biffi, Dana-Farber Cancer Institute, Boston Children's Hospital, USA
Engineered HSCs for Correction of CNS Disorders

Sebastian Ricardo Palacios, Massachusetts Institute of Technology, USA
Short Talk: Engineered RNAi-based Logic Circuits in Human Induced Pluripotent Stem Cells and their Differentiated Derivatives

Esmond Lee, Stanford, USA
Short Talk: CRISPR- Based Gene Editing Enables FOXP3 Gene Repair in IPEX Patient Cells and HSPCs

19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Mezzanine 2
19:30—22:00
Poster Session 1

Mezzanine 2

MONDAY, FEBRUARY 10

07:00—08:00
Breakfast

Mezzanine 2
08:00—11:15
Genome Editing as a Biology Discovery Tool

Van Horne C
* David R. Liu, Broad Institute, HHMI, and Harvard University, USA

Kathy K. Niakan, Francis Crick Institute, UK
Towards an Understanding of Human Development Using Gene Ablation

Hannah N. De Jong, Stanford University, USA
Short Talk: Deep Mutational Scanning to Identify Cardiac Disease Gene Variants

Philipp Junker, Max Delbrück Center for Molecular Medicine, Germany
Massively Parallel Lineage Tracing Using CRISPR/Cas9

Coffee Break

Alan Huang, Tango Therapeutics, USA
Novel Therapeutic Opportunities Identified by Synthetic Lethal Screening

Andrew Ravanelli, MilliporeSigma, USA
Short Talk: Direct Capture of CRISPR Guides Enables Scalable, Multiplexed, and Multi-Omic Genetic Screens

Birgit C. Schultes, IntelliaTherapeutics Inc., USA
Developing Next-Generation Engineered TCR-T Cells with CRISPR

Barbara Mair, University of Toronto, Canada
Short Talk: High-Throughput Genome-Wide Phenotypic Screening via Immunomagnetic Cell Sorting Identifies QPCTL as Modulator of CD47

08:00—11:15
Next Generation Immune Cell Engineering

Van Horne A/B
* Stanley R. Riddell, Fred Hutchinson Cancer Research Center, University of Washington, USA

Crystal L. Mackall, Stanford University, USA
Engineering Enhanced Potency T Cells

Chiara Bonini, Ospedale San Raffaele, Italy
Multiple Genome Editing of Early Differentiated T Cells for Cancer Immunotherapy

Coffee Break

Christopher A. Klebanoff, Memorial Sloan Kettering Cancer Center, USA
Targeting Solid Malignancies with "Public" Neoantigen TCRs

Aude G. Chapuis, Fred Hutchinson Cancer Research Center, USA
Optimizing T Cell Receptor Gene Therapy Against Solid and Liquid Tumors

Christie Ciarlo, Altius Institute for Biomedical Sciences, USA
Short Talk: Engineering Superior T Cell Therapies without Genetic Modification

Giedre Krenciute, St Jude Children's Research Hospital, USA
Short Talk: DNMT3a-Mediated Epigenetic Programming Limits CAR T Cell Survival and Effector Function

Debattama Sen, Harvard Medical School, USA
Short Talk: Disrupting Enhancers within the Core Epigenetic Program of Exhaustion Improves CD8+ T Cell Responses and Enhances Tumor Control

11:15—17:00
On Own for Lunch

11:15—13:00
Poster Setup

Mezzanine 2
13:00—22:00
Poster Viewing

Mezzanine 2
15:00—16:30
Workshop: Bacterial Adaptive Immunity Anti-CRISPR

Van Horne C
* Yan Zhang, University of Michigan, USA

Joseph Bondy-Denomy, University of California, San Francisco, USA
Inhibition of CRISPR-Cas9 and Cas12 with Bacteriophage Proteins

Jan Mathony, Heidelberg University, Germany
Computational Design of Anti-CRISPR Proteins with Improved Inhibition Potency

Jooyoung Lee, University of Massachusetts Medical School, USA
Tissue-Restricted Genome Editing in vivo Specified by MicroRNA-Repressible Anti-CRISPR Proteins

Dominik Niopek, Heidelberg University Hospital, Germany
Switchable Anti-CRISPR Proteins for Precision Control of CRISPR-Cas9 and -12

Jennifer R. Hamilton, University of California, Berkeley, USA
Delivery of Anti-CRISPR Proteins by Engineered Virus-Like Particles Therapeutically Protect Cells from CRISPR-Cas9 Gene Editing

15:30—16:30
Workshop 2: HSC and iPSC Engineering and Therapy

Van Horne A/B
* Katy Rezvani, University of Texas MD Anderson Cancer Center, USA

Linda T. Vo, University of California, San Francisco, USA
A Stem Cell Platform for Engineering Distinct T Cell Populations de novo for Immunotherapy

Brian Koss, University of Arkansas for Medical Sciences, USA
Epigenetic Control of Tumor-Infiltrating Lymphocyte Metabolic-Exhaustion

Bernhard Lehnertz, University of Montreal, Canada
Engineering of a Genetically Encoded Human Hematopoietic Stem Cell Reporter

Jose A. Vargas Asencio, Massachusetts Institute of Technology, USA
Single Cell Sequencing of Developing Liver-Like Organoids Reveals Expression Programs Associated to Germ Line Trajectories Defined by Synthetic Circuits

16:30—17:00
Coffee Available

Van Horne Foyer
17:00—19:00
Translational Science of Genome Editing: Assessing Specificity, Immunogenicity and Safety

Van Horne C
* Samantha Maragh, National institute of Standards and Technology, USA

Julian Grünewald, Massachusetts General Hospital, USA
Advances in Identifying and Modulating the Specificities of Gene Editing Technologies

Richard A. Morgan, Editas Medicine, USA
Application of Cpf1-Based Genome Editing for ex vivo Cell Therapy

Krishanu Saha, University of Wisconsin-Madison, USA
Short Talk: Patient-Derived, Induced Pluripotent Stem Cells as Translational Tools to Assess Specificity, Immunogenicity, and Safety of Somatic Cell Genome Editors

Bruce R. Conklin, University of California, San Francisco, USA
Short Talk: Modeling Therapeutic Editing in Patient-Derived iPSC Tissues

Zuben E. Sauna, Food and Drug Administration, USA
Understanding and Navigating the Immune Responses to CRISPR-Associated Nuclease Cas9

17:00—19:00
Cell Therapy for Infection and Autoimmune Diseases

Van Horne A/B
* Marina Cavazzana, Paris Descartes University, France

Qizhi Tang, University of California, San Francisco, USA
Manipulating Tregs to Control Tolerance in Autoimmunity and Organ Transplantation

Bruce R. Blazar, University of Minnesota, USA
Treg Therapies in the Setting of Allogeneic HSCT

Hans-Peter Kiem, Fred Hutchinson Cancer Research Center, USA
Engineering Resistance to HIV Infection

Megan K. Levings, University of British Columbia, Canada
CAR-Treg-Based Therapeutics

19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Mezzanine 2
19:30—22:00
Poster Session 2

Mezzanine 2

TUESDAY, FEBRUARY 11

07:00—08:00
Breakfast

Mezzanine 2
08:00—11:00
CRISPR-Based Editing of Non-Immune Cells (Joint)

Van Horne A/B
* Jennifer Gori, Obsidian Therapeutics, USA

Jacob E. Corn, ETH Zürich, Switzerland
How Human Cells Deal with Cas-Based Tools

Scot A. Wolfe, University of Massachusetts Medical School, USA
Precise Therapeutic Gene Correction by a Simple Nuclease-Induced Double-Strand Break

Coffee Break

Matthew Porteus, Stanford University School of Medicine, USA
CRISPR Editing of Human HSCs

Shengdar Q. Tsai, St. Jude Children's Research Hospital, USA
Short Talk: Highly Efficient Genome Editing of Human Hematopoietic Stem Cells and Fetal Hemoglobin Induction for Sickle Cell Disease Therapy

Alex Marson, University of California, San Francisco, USA
Reprogramming Human Immune Cell Circuitry

Connor A. Tsuchida, University of California, Berkeley; University of California, San Francisco, USA
Short Talk: Engineering HIV-1 Virus-Like Particles for CRISPR-Cas9 Genome Engineering in Primary Human T Cells

11:00—17:00
On Own for Lunch

11:00—13:00
Poster Setup

Mezzanine 2
13:00—22:00
Poster Viewing

Mezzanine 2
14:30—16:30
Career Roundtable
Interested participants must sign up at the registration desk on a first-come, first-serve basis. Maximum attendance: 60.

Conservatory
Samuel Haile, Kite Pharma, USA

Irene Jarchum, Nature, USA

Zuben E. Sauna, Food and Drug Administration, USA

Yvonne Y. Chen, University of California, Los Angeles, USA

Kole T. Roybal, University of California, San Francisco, USA

Eric J. Perkins, Addgene, USA

16:30—17:00
Coffee Available

Van Horne Foyer
17:00—19:00
'Off the Shelf' Allogeneic Cell Therapies (Joint)

Van Horne A/B
* Crystal L. Mackall, Stanford University, USA

* Richard A. Morgan, Editas Medicine, USA

Daniel Shoemaker, Fate Therapeutics, USA
Next Generation iPSC Derived Cellular Immunotherapies for Cancer

Hanspeter Waldner, CRISPR Therapeutics, USA
Development of an Allogeneic T Cell Therapy for Oncology

Sonja Schrepfer, University of California, San Francisco, USA
Hypoimmunogenic Derivatives of Induced Pluripotent Stem Cells Evade Immune Rejection in Fully Immunocompetent Allogeneic Recipients

Theodore Roth, University of California, San Francisco, USA
Short Talk: Highly Parallel Knock-In Targeting for Genome Engineering of Cellular Immunotherapies

19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Mezzanine 2
19:30—22:00
Poster Session 3

Mezzanine 2

WEDNESDAY, FEBRUARY 12

07:00—08:00
Breakfast

Mezzanine 2
08:00—11:00
DNA Repair and Genome Editing

Van Horne C
* Cecilia Cotta-Ramusino, Tessera Therapeutics, USA

James E. Haber, Brandeis University, USA
Understanding Cas9-Mediated Gene Targeting by Single-Strand Template Repair

Pavel Zrazhevskiy, Altius Institute for Biomedical Sciences, USA
Short Talk: Single Molecule Imaging of Nuclease-Induced Double Strand Break Repair in Human Cells

Gaëlle Legube, Center for Integrative Biology, France
Transcription Coupled DNA Double Strand Break Repair: Dealing with Breaks on Transcribed Loci

Coffee Break

Jonathan K. Watts, University of Massachusetts Medical School, USA
Chemically Modified DNA Donors for Precision Genome Editing

Albert B. Seymour, Homology Medicines, USA
AAVHSC-Mediated Genome Editing through Homologous Recombination: Applications in Liver-Mediated Diseases

Manda Arbab, Harvard University and Broad Institute, USA
Short Talk: Predictable and Precise Genome Editing Enabled by Systematic Characterization of Editing Outcomes

08:00—11:15
Using Synthetic Biology to Enhance Safety and Efficacy of Cellular Therapeutics

Van Horne A/B
* Alex Marson, University of California, San Francisco, USA

Christine E. Brown, Beckman Research Institute, City of Hope, USA
Advancing CAR T Cell Therapy for Treatment of Brain Tumors

Kole T. Roybal, University of California, San Francisco, USA
Engineering Next-Generation Immune Cell Therapies for Solid Tumors

Coffee Break

Marius Wernig, Stanford University, USA
How to Make a Neuron

H. Kay Chung, The Salk Institute for Biological Studies, USA
Short Talk: A Compact Synthetic Pathway Rewires Cancer Signaling to Therapeutic Effector Release

Andrew Ng, University of California, San Francisco, USA
Short Talk: Modular and Tunable Biological Feedback Control Using a De Novo Protein Switch

Nika Shakiba, Massachusetts Institute of Technology, USA
Short Talk: Synthetic Genetic Feedback Control Circuits to Reprogram Cell Fate

Noreen Wauford, Massachusetts Institute of Technology, USA
Short Talk: Development of a Toggle Switch Based on Post-Transcriptional Regulation that Resists Epigenetic Silencing

Louai Labanieh, Stanford University, USA
Short Talk: Genetically-Encoded Switches for Improving CAR-T Immunotherapy

11:00—17:00
On Own for Lunch

16:30—17:00
Coffee Available

Van Horne Foyer
17:00—18:45
New Genome Editing Tools

Van Horne C
* Scot A. Wolfe, University of Massachusetts Medical School, USA

Erik Sontheimer, University of Massachusetts Medical School, USA
Editing Genomes with Engineered Guide RNAs and Compact, Accurate Cas9 Homologs

Yan Zhang, University of Michigan, USA
Long-Range Genome Engineering in Human Cells with CRISPR-Cas3

David R. Liu, Broad Institute, HHMI, and Harvard University, USA
Base Editing and Prime Editing: Genome Editing Without Double-Strand Breaks

Peter J. Chen, Harvard University, USA
Short Talk: Programmable m6A Modification of RNA with a Cas13-Directed Methyltransferase

17:00—18:45
Non-T Immune Cell Therapies

Van Horne A/B
* Crystal L. Mackall, Stanford University, USA

Katy Rezvani, University of Texas MD Anderson Cancer Center, USA
Off-The-Shelf CAR-Engineered Cord Blood-Derived NK Cells for the Treatment of Cancer

Jeffrey S. Miller, University of Minnesota, USA
Novel Strategies to Activate and Target NK Cells as Off-The-Shelf Therapy to Treat Cancer

Leonid S. Metelitsa, Baylor College of Medicine, USA
NKT Cells as a Platform for Adoptive Cancer Immunotherapy

Eric Lagasse, University of Pittsburgh, USA
Short Talk: The Lymph Node as an Ectopic Transplantation Site for Hepatocytes

18:45—19:00
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)

Van Horne C
18:45—19:00
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)

Van Horne A/B
19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Alberta/New Brunswick
20:00—23:00
Entertainment
Entertainment is not subsidized by conference registration fees nor any U.S. federal government grants. Funding for this expense is provided by other revenue sources.

Alberta/New Brunswick

THURSDAY, FEBRUARY 13

 
Departure


*Session Chair †Invited, not yet responded.


Keystone Symposia thanks our Sponsors(s) for generously supporting this meeting:

Editas Medicine Novo Nordisk A/S

We gratefully acknowledge additional support from these exhibitors at this conference:

10x Genomics BEX CO., LTD.
Cellecta, Inc. Integrated DNA Technologies
MilliporeSigma Synthego
Please stop by to meet these exhibitors during the conference.

We gratefully acknowledge the generous grant for this conference provided by:


National Institutes of Health

Grant No. 1R13TR003025-01

Funding for this conference was made possible (in part) by 1R13TR003025-01 from the National Institutes of Health. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention of trade names, commercial practices, or organizations imply endorsement by the U.S. Government. This award reflects support from the NIH Common Fund Somatic Cell Genome Editing (SCGE) program in the amount of $22,200 total costs.

We gratefully acknowledge additional support for this conference from:

Agilent Aldevron, LLC
American Society of Gene & Cell Therapy (ASGCT) Illumina
New England Biolabs, Inc. Intellia Therapeutics

We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:



Illumina


Sangamo Therapeutics, Inc.


Intellia Therapeutics

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Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:


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Phone:+1 970-262-2690

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Phone:+1 970-262-1179