Discussion Day Tuesday 13th September 2016

08.00 Registration & Coffee Stream A: Autologous vs. Allogeneic: Which Will Succeed in the Race to Market? Leader: Sicco Popma Scientific Director, Gene Modified Cell Therapy Leader, Johnson & Johnson 9.00 Autologous vs. Allogeneic: Which Will Succeed in the Race to Market? 10.30 Morning Refreshments & Networking 11.00 Summary & Discussion: Autologous vs. Allogeneic: Which Will Succeed in the Race to Market? 12.00 Networking Lunch Stream C: Solid Tumor Target Identification and Validation Leader: Edmund Moon, Assistant Professor, University of Pennsylvania

1.00 Solid Tumor Target Identification and Validation 2.30 Afternoon Refreshments & Networking 3.00 Summary & Discussion: Solid Tumor Target Identification and Validation 4.00 Post Discussion Day Debrief 5.00 Close of Discussion Day

Stream B: Overcoming the Inhibitory Tumor Microenvironment Leader:

9.00 Overcoming the Inhibitory Tumor Microenvironment 11.00 Summary & Discussion: Overcoming the Inhibitory Tumor Microenvironment

Stream D: The Transformative Power of Correlative Sciences in Adoptive Immuno Gene Therapies Leader: Jos Melenhorst, Director, Product Development & Correlative Sciences laboratory, University of Pennsylvania 1.00 The Transformative Power of Correlative Sciences in Adoptive Immuno Gene Therapies 3.00 Summary & Discussion: The Transformative Power of Correlative Sciences in Adoptive Immuno Gene Therapies

Conference Day 1 Wednesday September 14th, 2016 08.00 Registration & Breakfast 09.00 Chairman’s Opening Remarks Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, Director, Clinical Cell and Vaccine Production Facility, University of Pennsylvania Updates from Leaders in the Field 09.10 The Race is On: CAR-Ts Where are We Now? David Chang, CMO & EVP R&D, Kite Pharmaceuticals 09.40 Engineered T Cells for Hematologic Malignancies and Solid Tumors: The Juno Program Mark Frohlich, EVP, Portfolio Strategy, Juno Therapeutics 10.10 Natural Killer Receptor T-Cells: A Different Approach to Target and Attack Cancer Steve Buckanavage, VP, Marketing, Celyad 10.40 Novartis CTL019 Case Study David Lebwohl, Senior Vice President & Global Program Head, CAR-T Program, Novartis 11.10 Speed Networking & Morning Refreshments STREAM A Discovery & Genetic T-Cell Engineering Chair: Dolores Schendel, CEO & CSO, Medigene Target Identification & Validation 12.10 – 12.40 Understanding and Improving the Activity of CAR-T cells in Solid Tumors Jennifer Brogdon, Director, Exploratory ImmunoOncology, Novartis 12.42 – 1.12 Targeted Insertion of an Anti-CD19 CAR into Primary Human T cells Using ARCUS Gene Editing Technology Bruce McCreedy, SVP of Cell Therapy, Precision Biosciences 1.14 – 1.44 The ABC’s of Adoptive T Cell Therapy Using Optimal- Affinity Natural TCRs Specific for

STREAM B Translation & Clinical Development Chair: Michael Kalos, CSO, Cancer Immunobiology, Eli Lilly Robust Clinical Research

STREAM C Manufacturing, Supply Chain & Commercialization Chair: Sadik Kassim, Associate Director, Novartis Manufacturing Strategies

12.10 – 12.40 Translation of Preclinical Research into the Clinic Stephan Grupp, Director, Cancer Immunotherapy Frontier Program, Children’s Hospital of Philadelphia 12.42 – 1.12 Improving Analysis of CAR T-Cell Function through High-Throughput MultiParameter Flow Cytometry Tamara Laskowski, Post-Doctoral Fellow, CAR T-Cell Therapy Lab, MD Anderson Cancer Center

12.10 – 12.40 Automating CART cell Manufacturing: Building a Solid Foundation Rodney Rietze, Lead, cGMP Process Automation, Novartis

1.14 – 1.44 Single-Cell Metrics Of The Efficacy Of CAR+ T Cells Navin Varadarajan, Assistant

12.42 – 1.12 Key Considerations when Evaluating CMOs for Late Stage Clinical and Commercial Production of Cell-Based Immunotherapies Claudia Zylberberg, CEO, Akron Biotech 1.14 – 1.44 Large-Scale, Reproducible Biomanufacturing of HighQuality Cells: A Roadmap from

Hematological Malignancies and Solid Tumors Dolores Schendel, CEO & CSO, Medigene

1.45 Networking Lunch 2.45 – 3.15 Gene Editing and Vector Engineering to Support Commercialization of CAR-T Immunotherapies Martha Rook, Head of Gene Editing & Novel Modalities, MilliporeSigma 3.17 – 3.47 Treatment of Solid Cancers by Adoptive Cell Transfer of Chimeric Antigen Receptor Transduced T Cells Steven Feldman, Director, Surgery Branch Vector Production Facility, NCI 3.50 – 4.50 PANEL: Innovations in Enhancing Efficacy and Improving Safety Profiles Moderator: Dolores Schendel, CEO/CSO, Medigene Tim Clay, Senior Director, Research Projects Leader US, Cell & Gene Therapy Discovery Research, GSK Klaus Kühlcke, CEO, EUFETS Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, Director, Clinical Cell and Vaccine Production Facility, University of Pennsylvania

Professor, University of Houston

the National Cell Manufacturing Consortium Krishnendu Roy, Director, Center for Cell Manufacturing & ImmunoEngineering, Georgia Institute of Technology

2.45 – 3.15 Further Development Of CAR-T Cells for the Treatment of Cancer Rick Morgan, VP Immunotherapy, bluebird bio

2.45 – 3.15 Innovative Solutions for Manufacturing of Next Generation Cell Therapies Nina Bauer, Senior Manager, Commercial Development, Autologous Cell Therapy, Lonza

3.17 – 3.47 CAR T Cells Go Back to School Marcela Maus, Director, Cellular Immunotherapy, MGH Cancer Center

3.17 – 3.47 Challenges in CAR-T Cell Manufacturing Isabelle Rivière, Director, Michael G. Harris Cell Therapy & Cell Engineering Facility, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center

3.50 – 4.50 PANEL: Developing and Translating Clinical Trials from Bench to Bedside

3.50 – 4.50 PANEL: From Manufacturing to Market Access: Getting Cellular Therapies to Patients in Need

Moderator: Michael Kalos, CSO, Cancer Immunobiology, Eli Lilly Navin Varadarajan, Assistant Professor, University of Houston Sicco Popma, Scientific Director, Gene Modified Cell Therapy Leader, Johnson & Johnson Stephan Grupp, Cancer Immunotherapy Frontier Program, Children’s Hospital of Philadelphia Louis Masi, Co-Founder & EVP, FloDesign Sonics

Peter Hoang, SVP, Business Development & Strategy, Bellicum Pharmaceuticals 4.50 Afternoon Refreshments & Poster Session Viral Vector Transfection & Demonstrating Efficacy in Solid Genetic Engineering Tumor Indications

Moderator: Sadik Kassim, Associate Director, Novartis Steve Buckanavage, VP, Marketing, Celyad Steven Feldman, Director, Surgery Branch Vector Production Facility, NCI Rodney Rietze, Lead cGMP Process Automation, Novartis Fred Koller, VP, Business Development, Miltenyi Biotec

Transportation, Supply Chain & Logistics

5.10 – 5.40 Reinventing the Lentivirus System to Create High Titer Packaging Cells Ryan Cawood, CEO, Oxford Genetics

5.10 – 5.40 Next-Generation Investigative CAR-T Platforms Towards the Goal of Personalized Immunotherapy Robert Berahovich, Director, CAR-T Discovery, ProMab Biotechnologies

5.42 – 6.12 TALEN® Based Targeted Genome Modifications for Improved CAR-T Cell Adoptive Immunotherapy Laurent Poirot, Head, Early Discovery, Cellectis

5.42 – 6.12 Development of Cancer-Specific anti-EGFR CAR-T cells for the Treatment of Patients with Glioblastoma Zonghai Li, President & CEO, CARsgen

6.14 – 6.44 Novel Cellular Engineering: Controlling Specificity and Activity of Adoptive Cellular Therapies Peter Emtage, SVP Research & Development, Cell Designs Lab

6.14 – 6.44 TCR-Like CARs Targeting Intracellular CancerSpecific Antigens for Treatment of Solid Tumors Cheng Liu, CEO, Eureka Therapeutics

6.45

CAR-TCR Party Hosted by Juno Therapeutics The Ritz-Carlton Boston

7.45

Close of Conference Day 1

5.10 – 5.40 Critical Assessment of the Impact of Packaging and Logistics Decisions on the CAR-T Clinical Pipeline Mark Sawicki, CCO, Cryoport Jim Reiner, Senior Product Manager, UPS 5.42 – 6.12 Analytical Challenges Around Manufacturing Christopher Wiwi, Director, Analytical R&D, Celgene Cellular Therapeutics

6.14 – 6.44 Characterization of CAR-T Therapies: Advancing Towards Precision Sadik Kassim, Associate Director, Novartis

Conference Day 2 Thursday September 15th, 2016 08.00 Breakfast Briefing by Draper Laboratory Invite only 08.00 Coffee & Networking 09.00 Chairman’s Opening Remarks Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, Director, Clinical Cell and Vaccine Production Facility, University of Pennsylvania Updates from Leaders in the Field 09.10 Near Term Targets and Next Generation TCR Engineered T Cell Therapy Gwendolyn Binder- Scholl, CTO, Adaptimmune 09.40 Controlling Potency, Persistence, and Toxicity of T Cell Therapies Using Molecular Switches David Spencer, CSO, Bellicum Pharmaceuticals 10.10 Tailored T Cell Therapies: The Autolus Approach Christian Itin, CEO, Autolus 10.40 Building Automation, Reproducibility, and Transferability into CAR-T Cell Manufacturing Fred Koller, Vice President, Business Development, Miltenyi Biotec 11.10 Speed Networking & Morning Refreshments STREAM A Discovery & Genetic T-Cell Engineering Chair: Tim Clay, Senior Director, Research Projects Leader US, Cell & Gene Therapy Discovery Research, GSK Innovative Methods to Overcoming Safety & Toxicity Challenges 12.10 – 12.40 Next-Generation Bio-Engineering of Immune Cells for Potency and Persistence Laurence Cooper, CEO, ZIOPHARM Oncology

STREAM B Translation & Clinical Development Chair: Michael Kalos, CSO, Cancer Immunobiology, Eli Lilly

12.42 – 1.12 Pfizer Allogeneic CAR-T Therapy Barbra Sasu, Inter-rim CSO, Rinat/Pfizer

12.42 – 1.12 PD1 Inhibition and Autologous T cells Expressing a GD2 Specific CAR with CD28 and OX40 Costimulatory

Combination Therapies & Enhancing the Efficacy of Frontline Cancer Therapies 12.10 – 12.40 The Horse Before the CAR-T? Pretreatment With Oncolytic Herpes Simplex I Viroimmunotherapy Augment CAR-T Cell Therapy Kellie Haworth, Pediatric Hematology, Oncology Fellow, Nationwide Children’s Hospital

STREAM C Manufacturing, Supply Chain & Commercialization Chair: Sadik Kassim, Associate Director, Novartis

Regulatory Expectations & Guidelines 12.10 – 12.40 Clinical Considerations for CAR-T Cell Therapy: A Regulatory Perspective Kristin Baird, Medical Officer, Division of Clinical Evaluation, Pharmacology & Toxicology, Office of Cellular, Tissue & Gene Therapies, CBER, FDA 12.42 – 1.12 EMA Regulatory Perspective: Evolving the Regulatory Conversation to Match Scientific Innovation

1.14 – 1.44 Identifying the Best Killers: Kinetic Assessment of CAR-T Cell Potency Against Liquid and Solid Tumors Yama Abassi, VP, ACEA Biosciences

1.44 Networking Lunch The Future of CAR-T Development: Emerging Innovations & Technologies 2.45 – 3.15 New Models of Engineered T Cells for Cancer Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, Director, Clinical Cell and Vaccine Production Facility, University of Pennsylvania 3.17 – 3.47 Potential of CAR T Cells for Treating Infectious Diseases Thor Wagner, Assistant Professor, Seattle Children’s Hospital

Endodomains in Combination for Children with Neuroblastoma Andras Heczy, Director, Liver Tumor Program, Assistant Professor, Baylor College of Medicine 1.14 – 1.44 Cytokines as Ancillary Reagents: Ways to Reduce Risk and Associated Costs Timothy Manning, Product Manager, Recombinant Proteins R&D Systems, BioTechne Unconventional CAR Approaches: Alternative CAR vehicles & Looking Beyond Cancer 2.45 – 3.15 Novel CARs Introduced into NK Cells Facilitate Potent Tumor-Cell Killing that Results in Tumor Regression Rohit Duggal, Director, Experimental Therapy, Sorrento Therapeutics 3.17 – 3.47 Target Engineered Natural Killer Cells as “Off-theShelf” Cellular Therapeutics Hans Klingemann, VP, R&D, NantKwest

Paula Salmikangas, Chair, Committee for Advanced Therapies, EMA

1.14 – 1.44 Your CAR-T Therapy has the Green Light? How Strong is Your Market Access Strategy? Jan Nielsen, Vice President, Sonexus™ Access & Patient Support, Cardinal Health Specialty Solutions Pricing, Partnering & Novel Business Models

2.45 – 3.15 How Will New Cancer Cell Therapies be Reimbursed? Edmund Pezalla, VP, National Medical Director, Pharmaceutical Policy & Strategy, Aetna 3.17 – 3.47 Delivering Optimized Cell Therapies Through Integrated Technology Modules Axel Hoos, Senior Vice President, TA Head Oncology R&D and Head, ImmunoOncology, GSK

3.47

Afternoon Refreshments & Networking

4.15

A Patient’s Experience with the Initial CART 19 Trial and Perspectives on Patient Access to New Breakthroughs for Cancer Treatment Doug Olson, Patient Advocate, The Leukemia and Lymphoma Society

4.45

Chairman’s Closing Remarks Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, Director, Clinical Cell and Vaccine Production Facility, University of Pennsylvania

5.00

Emily Whitehead Foundation Silent Auction

6.00

Close of Conference Day 2

Focus Day: Downstream Processing Friday September 16th, 2016 08.00 Registration & Coffee Stream E: CAR-T Cell Therapy: Regulatory Challenges and Opportunities Leaders: Paula Salmikangas, Chair, Committee for Advanced Therapies, EMA Kristin Baird, Medical Officer, Division of Clinical Evaluation, Pharmacology and Toxicology, Office of Cellular, Tissue and Gene Therapies CBER, FDA

9.00 CAR-T Cell Therapy: Regulatory Challenges and Opportunities 10.30 Morning Refreshments & Networking 11.00 Summary & Discussion: CAR-T Cell Therapy: Regulatory Challenges and Opportunities 12.00 Networking Lunch Stream G: Pricing and Reimbursement Strategies for Engineered Cell Therapies Leader: Edmund Pezalla, VP, National Medical Director, Pharmaceutical Policy & Strategy, Aetna 1.00 Pricing and Reimbursement Strategies for Engineered Cell Therapies 2.30 Afternoon Refreshments & Networking 3.00 Summary & Discussion: Pricing and Reimbursement Strategies for Engineered Cell Therapies 4.00 Post Focus Day Debrief 5.00 Close of Focus Day

Stream F: Development Across the spectrum: Pre-clinical and Trial Design, Academic Involvement, Industry Scalability, and Clinical Distribution Sarah Nikiforow, Assistant Medical Director, Cell Manipulation Core Facility, Dana Farber Cancer Centre Helen Negre, Technical Director, Novel Cell Therapies, Dana Farber Cancer Centre Marcela Maus, Director, Cellular Immunotherapy, MGH Cancer Center 9.00 Automatizing Single Use Systems and Novel Production Paradigms for Effective Manufacturing 11.00 Summary & Discussion: Automatizing Single Use Systems and Novel Production Paradigms for Effective Manufacturing Stream H: Current Trends and Best Practices in Outsourcing for Long Term Cell Immunotherapy Success Leader: Peter Olagunju, Senior Director, Vendor Management, bluebird bio 1.00 Current Trends and Best Practices in Outsourcing for Long Term Cell Immunotherapy Success 3.00 Summary & Discussion: Current Trends and Best Practices in Outsourcing for Long Term Cell Immunotherapy Success