Pre-Meeting Workshops

Now that CAR T cells have been in clinical use for over 5 years, this session will focus on the clinical results and longer-term follow up of CAR T cell studies in hematologic malignancies, on the consensus grading systems that have emerged as CAR T cells enter general practice and there are more and more clinical studies, and on how clinical centers manage the administration and collection of long-term follow-up data required by regulatory bodies.

Cameron Turtle, MBBS, PhD—Adult NHL

Shannon Maude, MD, PhD—Pediatric ALL

Jim Kochenderfer, MD—MM/BCMA

Sattva Neelapu, MD—ASBMT CRS grading and ASBMT neurotox grading

Sarah Nikiforow, MD, PhD—Long term follow-up program development

Matt Frigault, MD—Requirements to establish a phase 1 program

This session will focus on the scientific innovation as well as the safety and regulatory aspects of next-generation genetic engineering strategies that are in development that will enable complex strategies to target novel antigens not amenable to standard autologous CAR T cell therapies or to enable allogeneic cellular products.

Wendell Lim, PhD—Syn–notc

Justin Eyquem, PhD—TRAC locus targeting

Saar Gill, MD, PhD—CD33 edit and CD33 CAR-T cells

J. Keith Joung, MD, PhD—Measuring off-target effects of editing – Regulatory aspects of QC/release in gene edited products

CAR T cells are in wide clinical use in B cell cancers as autologous cellular products that have been transduced to express a single transgene. This session will focus on innovations with alternative cellular products and indications, such as allogeneic NK cells, stem cells, and potential uses of engineered cell therapies on non-cancer indications.

Katy Rezvani, MD, PhD—NK-CARs

Michel Sadelain, MD, PhD—iPSC-derived IEC

Marcela Maus, MD, PhD—Treg CARs

Mike Milone, MD, PhD—Non-cancer – pemphigus

Cyril Konto MD (Allogene Therapeutics)—Allogeneic CAR T cells

Introduction to Gene Editing led by Morgan Maeder, PhD.

The National Institutes of Health recently awarded its first grants through the Somatic Cell Genome Editing program. In this session we will hear from some of these initial awardees.

Betty Poon, PhD—Introduction to NIH Genome Editing Consortium Grants

Shengdar Tsai, PhD—A Novel Human T-cell Platform to Define Biological Adverse Effects of Genome Editing

Guangping Gao, PhD—CRISPR-based in vivo gene editing in multiple target organs

Aravind Asokan, PhD—Evolving AAV for Efficient Neuromuscular Genome Editing

A series of presentations from some of the leading companies in gene editing, describing their pre-clinical and clinical programs.

Shawdee Eshghi, PhD, Caribou Biosciences—Identification of high efficiency and high specificity guides for CRISPR-Cas9 engineering of allogeneic CAR-T cells

Leonela Amoasii, PhD, Exonics Therapeutics—CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular Dystrophy

Derek Jantz, PhD, Precision BioSciences—Engineered Meganucleases for Gene and Cell Therapy

Charlie Albright, PhD, Editas Medicine—Gene editing therapies for inherited retinal diseases

Giuseppe Ciaramella, PhD, Beam Therapeutics

Tony Ho, MD, CRISPR Therapeutics—Bringing CRISPR to the Clinic

The National Institutes of Health recently awarded its first grants through the Somatic Cell Genome Editing program. In this session we will hear from some of these initial awardees.

Erik Sontheimer, PhD—Enhancing Genome Editing with Chemically Engineered Guides and New Cas9 Orthologs

Shaoqin Sarah Gong, PhD—Multifunctional CRISPR/Cas9 Delivery Nanoplatforms for Targeted Genome Editing

Zheng-Yi Chen, DPhil—Development of bioreducible lipid nanoparticles for the delivery of editing agents in the mammalian inner ear

Gene editing is a dynamic area of research, attracting many new investigators. This session highlights the work of some of the outstanding junior investigators in the field.

Pietro Genovese, PhD—Advanced Genetic Engineering of Hematopoiesis for the Treatment of Inherited Diseases

Ben Kleinstiver, PhD—Enhancing Genome Editing with Engineered CRISPR Proteins

Thomas Gaj, PhD—Application of Genome Editing Technologies for the Treatment of Neurodegenerative Diseases

Prashant Mali, PhD—Therapeutic strategies via genome and transcriptome engineering

Pinar Akcakaya, PhD—Evaluating in vivo off-targets of CRISPR-Cas gene editing

Jennifer Adair, PhD—Precision gene editing without electroporation in blood repopulating cells using a novel CRISPR nanoformulation

A series of presentations from some of the leading companies in gene editing, describing their pre-clinical and clinical programs.

Edward Rebar, PhD, Sangamo Therapeutics—Therapeutic Genome Editing and Gene Regulation Using Designed Zinc Finger Proteins

Julianne Smith, PhD, Cellectis—TALEN Gene Editing for “Off-the-Shelf” Allogeneic CAR T-Cell Products

Philip Gregory, DPhil, bluebird bio—MegaTAL driven genome editing for enhanced CAR-T cell anti-tumor activity

Andy Scharenberg, MD, Casebia Theapeutics—Casebia therapeutics: driving gene editing innovation

Fred Chereau, MBA, LogicBio—Innovative promoterless nuclease-free genome editing

Sean Burns, Intellia Therapeutics—Delivering on the therapeutic promise of CRISPR/Cas9

Patient Perspective Keynote

Mark Skinner, JD

Yoko Momonoi, MS, Director RegCMC, Celgene—Process Development Challenges

Jason Hamilton, PhD—Analytical Development Challenges

Katy Spink, PhD

Michael Boychyn, PhD

Panel Discussion Moderated by John Tomtishen

Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research

Martina Schüssler-Lenz, MD, Chair, EMA Committee for Advanced Therapies

Manuela Corti, PhD

Debra Segal, PhD

Panel Discussion Moderated by Adora Ndu, PharmD, JD

Chair: Eduard Ayuso, DVM, PhD

Victor (Xiaobin) Lu, PhD

Richard Snyder, PhD

Kim Warren, PhD

Pratik Jaluria, PhD

Chair: H. Trent Spencer, PhD

Harrison Brown, PhD

Fraser Wright, PhD

Hank Fuchs, MD

Jessica Tucker, PhD—Modernizing Gene Therapy Oversight and the Role of the RAC

Celia Witten, MD PhD—Patient Focused Drug Development

Ronald Bartek

Sue Kahn, MBA

Maria Kefalas, PhD

Kristen Harrington-Smith, MBA

Ramjay Vatsan, PhD

Anne-Virginie Eggimann, MSc

Joann Parker, MS

Tim Hunt, JD—Overview of Pricing, Reimbursement, and Payment Models: ASGCT View

William Smith, PhD—Limitations of QALYs in Cost-Effectiveness Reviews

Jeremy Allen, MPA—Enabling Legislation for Novel Payment Models

Jugna Shah, MPH—Medicare Coverage & Reimbursement Issues

Rachel Pryor, MSW—Issues in Medicaid Reimbursement of Gene Therapies