deliver the next generation of safe, effective & commercially...

Expert Speakers Include:Arie Belldegrun CEO Kite Pharmaceuticals

David Lebwohl SVP & Executive Global Program Head, CAR-T Global Team, Cell & Gene Therapies Unit Novartis

Mark Frohlich

EVP, Portfolio Strategy Juno Therapeutics

Gwendolyn Binder-Scholl CTO Adaptimmune

Kristin Baird

Medical Officer, Division of Clinical Evaluation, Pharmacology & Toxicology, Office of Cellular, Tissue & Gene TherapiesCBER, FDA

Deliver the Next Generation of Safe, Effective & Commercially Viable CAR-T and TCR Cell Therapies to Market

In support of theSeptember 13th-16th, 2016BOSTON, MA

Expertise Partners:

Tel: +1 212 537 5898 | Email: [email protected]

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Cell Immunotherapy @CAR_TCell

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Tel: +1 212 537 5898 Email: [email protected]

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September 13th - 16th, 2016CAR-TCR Summit 2016 Boston, MA

The industry-defining meeting dedicated to help accelerate translation, clinical development and commercialization of the next generation of CAR-T & TCR cellular immunotherapies, for liquid and solid tumor indications.

The 2nd Annual CAR-TCR Summit 2016 remains the end-to-end meeting for the crème de la crème of CAR-T and TCR developers.

The CAR-TCR Summit brings together 50+ world-class speakers and over 400 cell therapy experts to drill down into the very latest case studies that capitalize on the promise of transformative CAR-T and TCR therapies.

Learn to create novel next generation CAR constructs to demonstrate significantly effective clinical outcomes and safely scale up manufacturing and development to meet patient demand.

Access never seen before clinical data and exclusively network with scientific and business leaders from large pharma, biotech, academia and the service community to optimize the efficacy of T cell therapies, without ever compromising on safety.

The 2nd CAR-TCR Summit will provide you with the tool kit you need to successfully accelerate your research from the bench to market safely and effectively whilst ensuring commercial viability.

Join all the leading organizations that are pioneering within this collaborative ecosystem to advance the development of CAR-T & TCR cell therapies.

1 Target ID: Steven Feldman, NCI will share the NCI’s CAR-T experience for the treatment of solid cancers from pre-clinical development to clinical outcomes

2 CAR Construct and Assembly: Improve the activity of CAR-T cells in solid tumors through insights into structure-activity relationships which influence CAR design and potency with Jennifer Brogdon, Novartis

3 Pre Clinical Modeling: Using a novel single cell multiparametric assay, Timelapse Imaging Microscopy In Nanowell Grids (TIMING) Navin Varadarajan, University of Houston will demonstrate single-cell metrics indicating the efficacy of CAR-T cells

4 Translational Research: Sadik Kassim, Novartis discusses analytical strategies to characterize CAR-T and TCRs before administration to advance towards precision

5 Safety and Efficacy Profiles: David Spencer, Bellicum Pharmaceuticals will share strategies on how to control potency, persistence, and toxicity of T cell therapies using molecular switches

10 Patient Access: Share the patient’s experience with Doug Olson, The Leukemia and Lymphoma Society the second patient to receive CART 19. Hear his perspectives on patient access to new breakthroughs for cancer treatment

9 Commercialization: Edmund Pezalla, Aetna provides the payer perspective and aims to answer the question: How will these cellular immunotherapies be reimbursed?

8 Scalable Manufacture: Overcome manufacturing standardization, logistics and product characterization challenges to achieve broad usage and eventual commercialization of CAR-T and TCR therapies with Isabelle Rivière, Memorial Sloan Kettering Cancer Center

7 Regulatory Compliance: Understand the key regulatory challenges and opportunities faced as pertains to the development of CAR-T and TCR therapies with Paula Salmikangas, EMA and Kristin Baird, CBER, FDA

6 Clinical Trial Development: Improve the design of early phase trials to maximize learning and deep mechanistic understanding of CAR-T cells with Marcela Maus, MGH Cancer Center

Navigate the CAR-TCR Development Timeline




Why You Should Attend the CAR-TCR Summit:

David Meek President, Oncology Baxalta

Laurent Poirot Head, Early Discovery Cellectis

Rick Morgan VP, Immunotherapy bluebird bio

Tim Clay Senior Director, Cell & Gene Therapy Discovery

Research GSK

Cheng Liu CEO Eureka Therapeutics

Jennifer Brogdon Senior Investigator Novartis

Peter Emtage SVP Research & Development Cell Design Labs

David Lebwohl SVP & Executive Global Program Head, CAR-T Global Team, Cell & Gene Therapies

Unit Novartis

Hans Klingemann VP, R&D NantKwest

“By gathering the industry together at this summit, we will be able to innovate in ways that are not possible if we remain in our individual institutions, universities, and companies. The cross-pollination of ideas will spur the development of these next generations therapeutics.”

“The most exciting aspect is to be able to feed each other with different ways to tackle scientific/technological barriers and by doing so, foster new ideas for the next generation of CAR-T cell treatments.”

“I am most looking forward to hearing about advances in CAR-T applications that go beyond the initial applications of anti-CD19 CARs. It’s great to be part of the interchange of ideas, particularly what is and is not working in the early clinical trials.”

“I am looking forward to talks by all the leading players in the field from academia and industry, and a getting a clear picture of the current status of the field. The size of the meeting and its focus affords great opportunities to meet and network which is invaluable!”

“This summit brings together leaders in the field of CAR-TCR therapy who share a lot of their experience and progress, with the goal of advancing the field as a whole. The collaborative environment and willingness to share information is the highlight of this summit for me.”

“This is a unique opportunity to bring a focused group together to discuss the success and challenges that lie ahead in this field which is quite exciting. Leveraging each other’s learnings is critical to bring this therapy to patients as quickly as possible.”

“I can’t wait to see the emerging data at this summit, especially clinical and manufacturing, coming from all the CAR-T programs. The chance to share ideas with other CAR-T cell therapy leaders is really exciting.”

“I’m most exited about getting a “state of the art” update for this rapidly growing technology/treatment. I can’t wait to see how the various groups address the main issues with any kind of cellular immunotherapy.”

“The CAR-TCR summit brings together key stakeholders in the adoptive cell therapy arena. Considering the infancy of this space, this summit allows for small intimate connections to be forged and this is what is needed to advance these highly specific and exciting treatment modalities. I am looking forward to great science and discussing novel ways to move the field forward.”




Industry Drug Developers

Gwendolyn Binder-Scholl CTO Adaptimmune

David Spencer CSO Bellicum Pharmaceuticals

Zonghai Li President & CEO CARsgen

Laurent Poirot Head, Early Discovery Cellectis

Steve Buckanavage VP, Marketing Celyad

Sicco Popma Scientific Director, Gene Modified Cell Therapy Leader Johnson & Johnson

Dolores Schendel CEO/CSO Medigene

Rodney Rietze Lead cGMP Process Automation Novartis

Laurence Cooper CEO ZIOPHARM Oncology

Martin Pule Founder & CSO Autolus

Peter Hoang SVP, Business Development & Strategy Bellicum Pharrmaceuticals

Christopher Wiwi Director, Analytical R&D Celgene Cellular Therapeutics

Yihong Yao CSO CBMG

Cheng Liu CEO Eureka Therapeutics

Mark Frohlich EVP, Portfolio Strategy Juno Therapeutics

Hans Klingemann VP, R&D NantKwest

Sadik Kassim Associate Director Novartis

David Kirn CEO & Co-Founder 4D Molecular Therapeutics

David Meek President, Oncology Baxalta

Peter Olagunju Senior Director, Vendor Management bluebird bio

Vladimir Jankovic Director, Preclinical & Translational Development Celgene Cellular Therapeutics

Christian Homsy CEO Celyad

Axel Hoos Senior Vice President, TA Head Oncology R&D, and Head, Immuno-Oncology GSK

Arie Belldegrun CEO Kite Pharmaceuticals

David Lebwohl SVP & Executive Global Program Head, CAR-T Global Team, Cell & Gene Therapies Unit Novartis

Barbra Sasu Senior Director Rinat/Pfizer

Michael Kalos, CSO, Cancer Immunobiology, Eli Lilly

Rick Morgan VP, Immunotherapy bluebird bio

Peter Emtage SVP Research & Development Cell Design Labs

Tim Clay Senior Director, Research Projects Leader US, Cell & Gene Therapy Discovery Research GSK

Margo Roberts CSO Kite Pharmaceuticals

Jennifer Brogdon Senior Investigator Novartis

Rohit Duggal Director, Experimental Therapy Sorrento Therapeutics

Speakers




Academics

Regulatory Bodies, Patient Advocacy & Healthcare Providers

Solution and Service Providers

Andras HeczyDirector, Liver Tumor Program, Assistant Professor Baylor College of Medicine

Navin VaradarajanAssistant Professor University of Houston

Kellie HaworthPediatric Hematology/Oncology Fellow Nationwide Children’s Hospital

Paula SalmikangasChair, Commitee for Advanced Therapies, EMA

Yama Abassi VP ACEA Biosciences

Mark Sawicki CCO Cryoport

Fred Koller VP, Business Development Miltenyi Biotec

Stephan GruppDirector, Cancer Immunotherapy Frontier Program Children’s Hospital of Philadelphia

Jason LukeAssistant Professor, Medicine University of Chicago

Marcela MausDirector, Cellular Immunotherapy Massachusetts General Hospital Cancer Centre

Bruce LevineDirector, Clinical Cell & Vaccine Production Facility, Perelman School of Medicine & Abramson Cancer Center, University of Pennsylvania

Edmund Pezalla VP, National Medical Director, Pharmacy & Policy Strategy Aetna

Claudia Zylberberg CEO Akron Biotech

Klaus Kühlcke CEO EUFETS

Ryan Cawood CEO Oxford Genetics

Isabelle RivièreDirector, Michael G. Harris Cell Therapy & Cell Engineering Facility, Center for Cell Engineering Memorial Sloan Kettering Cancer Center

Larry CoreyPresident & Director Emeritus Fred Hutchinson Cancer Research Center

Atsushi NatsumeAssociate Professor Nagoya University

Edmund MoonAssistant Professor University of Pennsylvania

Kristin BairdMedical Officer, Division of Clinical Evaluation, Pharmacology & Toxicology, Office of Cellular, Tissue & Gene TherapiesCBER, FDA

Jan Nielson Vice President Sonexus™ Access & Patient Support Cardinal Health

John Rozembersky VP, BioPharm & Life Science Applications FloDesign Sonics

Martyn Lewis CTO ProMab Biotechnologies

Krishnendu RoyDirector, Center for Cell Manufacturing & ImmunoEngineering Georgia Institute of Technology

Steven FeldmanDirector, Surgery Branch Vector Production Facility National Cancer Institute

Jos MelenhorstDirector, Product Development & Correlative Sciences laboratory University of Pennsylvania

Doug OlsonPatient Advocate The Leukaemia & Lymphoma Society

Robert Margolin VP Corporate Development Cognate BioServices

Tom Duensing CTO Intellicyt

Bruce McCreedy SVP, Cell Therapy Precision Biosciences




Tuesday September 13th – Pre Conference Workshops

Workshop A: Autologous vs. Allogeneic: Which Will Succeed in the Race to Market? - Hosted by Johnson & Johnson

Workshop B: Overcoming the Inhibitory Tumor Microenvironment - Hosted by Univeristy of Chicago

Lunch

Workshop C: Solid Tumor Target Identification and Validation - Hosted by University of Pennsylvania

Workshop D: Biomarkers of Response to Measure Patient Outcomes - Hosted by University of Pennsylvania

Wednesday September 14th – Conference Day One

Plenary: State of the Industry: CAR-Ts Where are We Now?

Plenary: Case Studies from Juno, Novartis and Celyad

Preclinical Discovery Stream Clinical Development Stream Manufacturing Stream

Target Identification & Validation Robust Preclinical Research Manufacturing Strategies

Lunch

Panel: Innovations in Enhancing Efficacy and Improving Safety Profiles

Panel: Developing and Translating Clinical Trials from Bench to Bedside

Panel: From Manufacturing to Market Access: Getting Cellular Therapies to Patients in Need

Afternoon Refreshments

Viral Vector Transfection & Genetic Engineering

Demonstrating Efficacy in Solid Tumor Indications

Analytical Testing & Cryopreservation

Thursday September 15th – Conference Day Two

Plenary: Case Studies from Kite, Bellicum, Adaptimmune and Miltenyi Biotec

Preclinical Discovery Stream Clinical Development Stream Manufacturing Stream

Innovative Methods to Overcoming Safety & Toxicity Challenges

Combination Therapies & Enhancing the Efficacy of Frontline Cancer Therapies

Regulatory Expectations & Guidelines

Lunch

The Future of CAR-T Development: Emerging Innovations & Technologies

Unconventional CAR Approaches: Alternative CAR vehicles & Looking Beyond Cancer

Pricing, Partnering & Novel Business Models

Afternoon Refreshments

Panel: Business Aspects of Engineered T Cell Therapies

Plenary: A Patient’s Experience and Perspectives on Patient Access to New Breakthroughs in Cancer

Friday September 16th - Post Conference Workshops

Workshop E: T Cell Therapy: Regulatory Challenges and Opportunities - Hosted by the FDA & EMA

Workshop F: Automatizing Single Use Systems and Novel Production Paradigms for Effective Manufacturing - Hosted by Novartis

Lunch

Workshop G: Pricing and Reimbursement Strategies for Engineered Cell Therapies - Hosted by Aetna

Workshop H: Current Trends and Best Practices in Outsourcing for Long Term Cell Immunotherapy Success - Hosted by bluebird bio

Agenda at a Glance

8.00 Breakfast & Registration

9.10 The Race is On: CAR-Ts Where are We Now? Arie Belldegrun, CEO, Kite Pharmaceuticals

9.40 Engineered T Cells for Hematologic Malignancies and Solid Tumors: The Juno Program

Juno is currently developing engineered CAR T cells for hematologic malignancies and solid tumors in this presentation we will discuss:

• The CD19 CAR-T program in acute lymphoblastic leukemia, non-Hodgkin lymphoma and chronic lymphocytic leukemia, as well as the CD22 CAR-T program in acute lymphoblastic leukemia

• Biologic insights gained through these studies

• The pipeline of CAR-T and engineered T cell receptor programs in solid tumors

• Strategies to overcome the tumor microenvironment including combinations

Mark Frohlich, EVP, Portfolio Strategy, Juno Therapeutics

10.10 Natural Killer Receptor T-Cells: A Different Approach to Target and Attack Cancer Celyad’s corporate mission is to develop therapies that restore the body’s ability to repair itself. In immuno-oncology, Celyad is taking a very different approach to engineering T-Cells to target and attack cancer cells by using a naturally occurring activating receptor found on Natural Killer cells, called NKG2D. This presentation will review:

• The science underpinning the different approach producing a T-Cell using an activating receptor as the targeting mechanism

• How one NKR-2 construct can target a wide range of both solid and hematological tumors based on the NKG2D receptor binding to multiple ligands expressed on tumor cells

• The pre-clinical evidence will be presented to demonstrate how the weaponized NKR T-Cell established proof of principle in animal models and then how this knowledge is being practically applied in the clinic in the ongoing Phase I feasibility and safety trial

• A look forward to the future pathway for NKR-2 in both hematological and solid tumors

Christian Homsy, CEO, Celyad

10.40 Novartis CTL019 Case Study• The Novartis approach to cell and gene therapy

• CTL019 update in pediatric ALL and non-Hodgkin’s lymphoma

• What’s next for Novartis?

David Lebowhl, SVP & Executive Global Program Head, CAR-T Global Team, Cell & Gene Therapies Unit, Novartis

11.10 Networking & Refreshments

Conference Day 1: Wednesday September 14th 2016




Discovery & Genetic T-Cell Engineering

Translation & Clinical Development

Manufacturing, Supply Chain & Commercialization

Target Identification & Validation Robust Preclinical Research Manufacturing Strategies

12.10 Understanding and Improving the Activity of CAR-T cells in Solid Tumors• Solid tumors pose greater challenges

for this therapeutic approach, both in terms of target selection and demonstrating meaningful clinical benefit

• This presentation will highlight our efforts to design a more potent, fully human CAR targeting mesothelin for pancreatic and ovarian cancer

• In depth characterization of a panel of CARs has revealed insights into the structure-activity relationships which influence CAR design and potency

• Efforts are also ongoing to elucidate the mechanisms that are impeding the effectiveness of CAR T cells within solid tumors

Jennifer Brogdon, Senior Investigator, Novartis

12.10 Translation of Preclinical Research into the Clinic• Treating cancers at earlier stages to

increase therapy efficacy

• Proof of concept in solid tumors

• Creating precise enrolment criteria

• Clinical Trial Design

• Persistence vs. efficacy vs. safety

• Non-pivotal trial design to influence pivotal trials

• Establishing Appropriate Primary Endpoints for Engineered T Cell Trials

• Ensuring biological and clinical relevance of end points

Stephan Grupp, Director, Cancer Immunotherapy Frontier Program, Children’s Hospital of Philadelphia

12.10 Automating CAR-T cell Manufacturing: Building a Solid Foundation• “The process is the product”, cells

are living entities that continue to respond to their environment in ways too often undetected (or predicted) by the operator.

• Manual-handling bias is a significant and ongoing concern, as is preserving the safety, potency and identity of the drug product through both the initial transfer of the process and the multiple rounds of process improvements that typically follow

• The Novartis rationale and approach to achieve the goal of automation is to minimize risk (and product loss) by maximizing insight (and control) of the manufacturing process so as to consistently deliver a safe, efficacious and cost-effective drug product at an appropriate scale to meet world-wide need

Rodney Rietze, Lead, cGMP Process Automation, Novartis

12.40 Precision Biosciences• We will explore generating allogeneic

CAR-T therapies using healthy donor PBMCs and a one-step genetic engineering process in which the CAR-encoding transgene is inserted into the native TCRα locus using an engineered ARCUS homing endonuclease

• This process simultaneously introduces the CAR gene stably into the genome while knocking-out the native TCR to prevent GvHD

• When injected into NSG mice bearing CD19+ tumors the CAR-T cells killed tumor cells and significantly increased survival of CAR-T vs. control treated animals with linear, dose proportional kinetics

Bruce McCreedy, SVP of Cell Therapy, Precision Biosciences

12.40 Advancing the Discovery of Immunotherapeutics with Large Scale, Multiplexed Experiments on Cells and Proteins of Immune System• Evaluate new tools which are needed

to evaluate the effects of therapies on cells and proteins in suspension

• The iQue® Screener is the first platform capable of performing large scale, multiplexed experiments on cells and proteins of the immune system

• Discuss how this system is being employed to discover new antibodies, and to understand functional efficacy of immuno-oncology therapies

Tom Duensing, CTO, Intellicyt

12.40 Key Considerations when Evaluating CMOs for Late Stage Clinical and Commercial Production of Cell-Based Immunotherapies• Learn to keep cost of goods low so

your CAR-T can soar high: Small changes, incredible impact?

• The critical attributes needed within a CMO to support the rapid clinical development and commercialization of these platform cell-based immunotherapies

• Navigate the main points where cost can and should be controlled, considering safety and compliance

• New concepts and ideas that can bring opportunities to in-process improvements and address issues relevant to the manufacturing combined with business models and commercialization steps

Claudia Zylberberg, CEO, Akron Biotech Robert Margolin, VP, Corporate Development, Cognate BioServices




1.10 The ABC’s of Adoptive T Cell Therapy Using Optimal-Affinity Natural TCRs Specific for Hematological Malignancies and Solid TumorsIt is critical to consider three ABC’s in the selection of TCR therapy candidates based on the TCR itself and its target ligand.

• A: Feasibility of TCR gene therapy for many patients with different types of malignancy depends on having a library of TCRs that can recognize appropriate MHC-restricted peptide ligands matched to the needs of an individual patient

• B: Safety is coupled to TCR fine specificity and comprehensive evaluation is needed to preclude potential on- and off-target toxicities as far as possible before first-in-man use

• C: Efficacy of TCR gene therapy is coupled to functional avidity and persistence of therapeutic T cells in vivo. TCR selection and choice of recipient T cell populations can be combined to provide therapeutics of potential better efficacy

Examples of approaches applying these ABC’s of TCR gene therapy will be presented and discussed.

Dolores Schendel, CEO/CSO, Medigene

1.10 Single-Cell Metrics Of The Efficacy Of CAR+ T CellsUsing our novel single cell multiparametric assay, Timelapse Imaging Microscopy In Nanowell Grids (TIMING), we compared the efficacy of two second generation CD19-specific CAR constructs – bearing CD8a and IgG4 hinge respectively - by tracking their interaction with NALM-6 tumor cells in vitro. We demonstrate using TIMING that:

• Significantly more CAR+ T cells bearing the CD8a hinge displayed enhanced basal motility and participated in superior serial killing

• To identify biomarkers those that distinguish between killer CAR+ T cells and non-killers, we performed single-cell multiplexed gene expression profiling

• Killer CART cells consistently expressed higher transcript levels of the cytotoxic molecule Granzyme B, the costimulatory receptor CD137 and the regulatory receptor TIM-3

• We confirmed that CD137 was overexpressed in cytotoxic CAR+ T cells and that its stimulation was associated with higher cytotoxicity and lower expression level of the immunoregulatory receptors CTLA4 and PD1and 2B4

• CAR+ T cells endowed with the ligand for CD137, demonstrated superior ability to control tumors in vivo, thus establishing CD137 as the link between function and fate at the single-cell level

• These results demonstrate the utility of our TIMING single-cell methodology in uncovering not only the dynamic profile of T-cell behavior but in also uncovering the phenotypic biomarkers of CAR+ T cells with superior functional efficacy

Navin Varadarajan, Assistant Professor, University of Houston

1.10 Large-Scale, Reproducible Biomanufacturing of High-Quality Cells: A Roadmap from the National Cell Manufacturing Consortium• Large scale manufacturing of

therapeutic immune-cells faces significant challenges including maintaining cell quality throughout the manufacturing process; limitations with scale-up and scale-out; lack of critical quality attributes; lack of scalable closed-system, automated manufacturing

• The National Institute for Standards and Technology (NIST) through its Advanced Manufacturing Technology (AMTech) program helped establish the US National Cell Manufacturing Consortium (NCMC) and tasked it with developing an industry-driven 10 year technology roadmap for Cell Therapy Manufacturing

• In this presentation I will outline the barriers and challenges identified by the cell manufacturing industry and clinical GMP centers and discuss the proposed technology solutions that we must address as a community to make cell-based immunotherapies a viable pharmaceutical product for broad clinical use

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

1.40 Lunch & Networking




2.40 Treatment of Solid Cancers by Adoptive Cell Transfer of Chimeric Antigen Receptor Transduced T Cells• The treatment of hematologic

malignancies by adoptive cell transfer (ACT) using T cells expressing chimeric antigen receptors such as the anti-CD19 CAR has proven to be a highly successful therapeutic modality in several clinical trials

• Treatment of solid cancers using CAR technology has proven more difficult and relates primarily to the difficulty in the identification of suitable cell surface molecules to target

• The Surgery Branch, NCI has initiated several clinical trials for the treatment of solid cancers using CARs targeting tumor antigens such as mesothelin and EGFRvIII as well as VEGFR2 expressed on the tumor vasculature

• A summary of our CAR-T experience for the treatment of solid cancers from pre-clinical development to clinical outcomes will be presented

Steven Feldman, Director, Surgery Branch Vector Production Facility, NCI

2.40 CAR T Cells Go Back to School • CAR T cells were initially developed

and translated to clinical trials in the academic setting but have now moved toward an industry setting

• We will review pre-clinical engineering strategies in detail to enhance CAR T cell safety and efficacy for new targets

• Discover how to improve design of early phase trials to maximize learning and deep mechanistic understanding of CAR-T cells

Marcela Maus, Director, Cellular Immunotherapy, MGH Cancer Center

2.40 Challenges in CAR-T Cell Manufacturing• We have developed platforms

to manufacture T lymphocytes expressing specific CARs that presently support multiple phase I clinical trials at MSKCC for the treatment of leukemia and lymphoma, as well as mesothelioma and ovarian cancer

• T cells genetically modified with replication-defective gammaretroviral vectors encoding CARs can be expanded upon selection and or activation with magnetic or biodegradable beads coated with agonistic anti-CD3 and anti-CD28 antibodies

• These bioprocesses allow the generation of clinical doses in less than two weeks using the Wave™ Bioreactor

Isabelle Rivière, Director, Michael G. Harris Cell Therapy & Cell Engineering Facility, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center

3.10 PANEL: Innovations in Enhancing Efficacy and Improving Safety ProfilesTim Clay, Senior Director, Research Projects Leader US, Cell & Gene Therapy Discovery Research, GSK

Klaus Kühlcke, CEO, EUFETS

Bruce Levine, Director, Clinical Cell & Vaccine Production Facility, Perelman School of Medicine & Abramson Cancer Center, University of Pennsylvania

Dolores Schendel, CEO/CSO, Medigene

Peter Hoang, SVP, Business Development & Strategy, Bellicum Pharmaceuticals

3.10 PANEL: Developing and Translating Clinical Trials from Bench to BedsideSadik Kassim, Associate Director, Novartis

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

John Rozembersky, VP, BioPharm & Life Science Applications, FloDesign Sonics

Michael Kalos, CSO, Cancer Immunobiology, Eli Lilly

3.10 PANEL: From Manufacturing to Market Access: Getting Cellular Therapies to Patients in NeedSteven Feldman, Director, Surgery Branch Vector Production Facility, NCI

Rodney Rietze, Lead cGMP Process Automation, Novartis

Steve Buckanavage, VP, Marketing, Celyad

Fred Koller, VP, Business Development, Miltenyi Biotec

4.10 Afternoon Refreshments




4.40 Reinventing the Lentivirus System to Create High Titer Packaging Cells• We have improved on the technology

to develop packaging cells in several ways, and have produced much more efficient systems for lentivirus production

• One innovation has been to optimize the levels of expression of helper genes in transient systems, by careful choice of promoters and plasmid ratios, allowing us to obtain virus yields that out-perform our competitors up to 4-fold

• Our proprietary strategy of co-expressing genes that alleviate toxicities has allowed us to express helper proteins constitutively at high levels, overcoming one of the major limitations

• These producer cells are robust and easily transfected with plasmids expressing the remaining helper proteins, and can achieve yields up to 10-times those of our competitors

Ryan Cawood, CEO, Oxford Genetics

4.40 Next-Generation Investigative CAR-T Platforms Towards the Goal of Personalized Immunotherapy• We developed our CAR-T platform

based on our 1500-3000 monoclonal antibody clones, and generation platforms that include mouse and rabbit monoclonals, and a human antibody library

• Strategically characterize custom CAR-T approaches with Promab beads that can target cancer cells overexpressing tumor antigens, a range of different effector CAR-T and NK cells targeting a panel of more than 10 distinct tumor antigens, and T cell expansion kits utilizing CD28/CD3

• Using real-time impedance-based cytotoxicity assays to examine the optimization of CAR’s using a bioinformatics approach for the generation of novel, modified antibodies in terms of functional improvements to binding, persistence and cytokine production

Martyn Lewis, CTO, ProMab Biotechnologies

4.40 Critical Assessment of the Impact of Packaging and Logistics Decisions on the CAR-T Clinical Pipeline• Scant peer reviewed research exists

on the impact of logistics strategies including fulfillment, packaging, and transport on clinical sample, product, and data integrity, in particular in the management of regenerative therapies

• In response, in conjunction with a number of our partners and clients, we have conducted a systematic study of the impact of logistics decisions on the integrity of critical biomarker and active clinical product and data generation

• This data will be discussed in relation to its impact on clinical progression in the CAR-T space

Mark Sawicki, CCO, Cryoport

Viral Vector Transfection & Genetic Engineering

Demonstrating Efficacy in Solid Tumor Indications

Transportation, Supply Chain & Logistics

5.10 TALEN® Based Targeted Genome Modifications for Improved CAR-T Cell Adoptive Immunotherapy• Cellectis’ leading gene-editing

platform TALEN® already crossed the doors of research laboratories and possesses all the key features: precision, efficiency and specificity, to design molecular scissors for therapeutic gene editing applications

• Description of how the TALEN® gene-editing technology allows creating allogeneic CAR T-cells but also empowering cells with additional safety and efficacy attributes

• New features include, among other possibilities, control properties designed to prevent engineered cells from attacking healthy tissues, to prevent auto-destruction, and to enable these cells to tolerate standard oncology treatments

Laurent Poirot, Head, Early Discovery, Cellectis

5.10 Development of Cancer-Specific anti-EGFR CAR-T cells for the Treatment of Patients with Glioblastoma• We developed a humanized single

chain antibody Y022 that could specifically recognize both EGFR and EGFRvIII (variant III mutant of EGFR) overexpressed in cancer cells but not EGFR in primary keratinocytes

• Further study revealed that Y022-engineered CAR-T cells could specifically recognize and eliminate cancer cells with EGFR amplification but not primary keratinocytes

• Moreover, the growth of established EGFR- or EGFRvIII-overexpressing glioma xenografts could be efficiently inhibited by the CAR-T cells

• Importantly, partial response was observed in a patient with glioblastoma while no EGFR-related toxicities were observed in phase I clinical studies

Zonghai Li, President & CEO, CARsgen

5.10 Analytical Challenges Around Manufacturing• Cross functional analytical

development to drive the advancement of cellular therapy candidates from research to preclinical, clinical and commercial manufacturing

• Quality Control operations to ensure the quality and conformance of raw materials and products in pre-clinical, IND and clinical/commercial stage production, and in compliance with all applicable regulations and industry standards

• Analytical challenges for commercializing cell therapy products

Christopher Wiwi, Director, Analytical R&D, Celgene Cellular Therapeutics




5.40 Next-Generation Bio-Engineering of Immune Cells for Potency and Persistence• Identify safe CAR and TCR targets

(especially to target solid tumors) and the use of suicide genes for conditional in vivo ablation using NGS technologies

• Reduce burden on manufacturing to produce T cells in real time and improvements in bio-processing to bias the infusion product towards “young” T cells with capacity for self-renewal

• Discover combinations such as co-infusing T cells with multiple specificities; combining the introduced immunoreceptor with cytokine to improve potency; combining genetic elements for the conditional and induced expression of introduced transgenes; combining genetic insertion with genetic editing and combining T-cell therapy with other immunotherapies to recycle effector functions within the tumor microenvironment

• Learn to reliably and efficiently execute all manner of tumor cells based on the targeting of neoantigens which will need to account for the dual pressures of the time to generate the T-cell products and cost

• Address these issues through efficient processing and the use of non-viral approaches to gene transfer, such as using the DNA plasmids from the Sleeping Beauty system

Laurence Cooper, CEO, ZIOPHARM Oncology

5.40 TCR-Like CARs Targeting Intracellular Cancer-Specific Antigens for Treatment of Solid Tumors• Tumor-specific intracellular targets

have been identified as highly specific tumor markers, but are considered “undruggable” by conventional CARs. These tumor-specific proteins are processed and presented as peptide products by class I MHCs on the surface of tumor cells, we developed an antibody discovery platform designed to identify highly specific, fully human scFvs against peptide-MHC complexes. These TCR-like antibodies are then engineered into CARs for treatment of solid tumors

• AFP-targeting CAR-T cells selectively and potently kill HLA-A*02:01+/AFP+ HCC cells in vitro, and cause rapid and profound tumor regression in multiple xenograft models of HCC in vivo. In comparison with CD19-targeting CARs, AFP-targeting CARs have shown a much lower risk of cytokine release syndrome

• The success of TCR-like CARs will dramatically increase the number of tumor-specific targets available for CAR-T therapy, particularly for treatment of solid tumors. This enhanced tumor-specificity combined with a reduced risk of cytokine release syndrome provides an opportunity to develop safer, more effective CAR-T therapies against a wide range of cancers

Cheng Liu, CEO, Eureka Therapeutics

5.40 Characterization of CAR-T Therapies: Advancing Towards Precision• In general, there are three principle

parameters by which investigators assess CAR-Ts in the clinical setting: clinical outcome, CAR-T cell persistence, and patient safety

• From a process development and manufacturing perspective, these parameters are not actionable as they are functions of in vivo CAR-T behavior

• CAR-Ts are “living drugs”; short of clinical trials, it is currently not possible to assess CAR-T safety and efficacy based on in vitro taxonomies of cell phenotype and function

• The field of CART immunotherapy is now at a critical juncture; numerous CAR-Ts are advancing to pivotal trials

• However, little is known about how to characterize these cellular products before administration, and more needs to be understood about how different manufacturing strategies can affect safety and efficacy

• This talk will discuss analytical strategies that may enable for the development of safer and more effective CART therapies for patients

Sadik Kassim, Associate Director, Novartis

6.10 Chairman’s Closing Remarks

6.15 Annual CAR-TCR Party Hosted by Juno Therapeutics

7.15 Close of Day 1




8.00 Breakfast & Networking

9.10 Near Term Targets and Next Generation TCR Engineered T Cell Therapy

• To date, engineered T cell therapy has achieved objective responses in only a small number of haematologic indications, primarily due to limitations on tumor – specific antigen targeting by chimeric antigen receptors

• TCRs overcome this target limitation, but it is complex to generate TCRs of the appropriate specificity and affinity for clinical use

• Furthermore, effective deployment of engineered T cell therapy in solid tumors may require combination approaches or next generation enhancements to the T cells in order to overcome immune suppression or evasion mechanisms present at the tumor site

• Translational approaches for investigating and addressing these challenges in the context of TCR engineered T cells for solid tumor therapy, will be discussed

Gwendolyn Binder-Scholl, CTO, Adaptimmune

9.40 Second Generation CAR-T and TCR Approaches for Enhancing Efficacy and Durability: The Bellicum Approach

• CARs that exhibit enhanced potency, persistence and proliferation to overcome the challenges of solid tumor indications, but also manage the risks associated with therapy-induced toxicity

• TCRs with enhanced co-stimulatory signaling that solve the issue of tumor-mediated MHC down regulation, preventing TCR-based therapies from detecting cancer associated antigens

• Cell therapies that can be modulated within the patient, allowing a clinician to prevent potential toxicity or drive efficacy against difficult tumor types

• In addition we will also discuss the following work that Bellicum Pharmaceuticals has been conducting with a platform technology that has the potential to address the above challenges

Aaron Foster, Senior Director, Product Discovery, Bellicum Pharmaceuticals

10.10 Kite Case Study This session will focus on the Kite Program, clinical updates, future pipeline and challenges in the CAR-TCR space. Full details of this session cannot be revealed at this stage.

Margo Roberts, CSO, Kite Pharmaceuticals

10.40 Building Automation, Reproducibility, and Transferability into CAR-T Cell Manufacturing

• Optimization of research processes often requires months of effort as various reagents and protocols are evaluated and developed to maximize yields as well as time and cost effectiveness

• For clinical translation, GMP suitability and scalability should also be considered early in the process to avoid future incompatibilities and additional re-development

• Recently, Miltenyi has launched the T cell transduction protocol on the Prodigy system for automated production of genetically-modified T cells to enable efficient and cost-effective clinical trials and commercialization

• Examples in chimeric antigen receptor (CAR) T cell applications will be presented, demonstrating efficient integration of key steps including cell selection, activation, transduction, expansion and processing for both research as well as GMP-compatible reagents and automated instrumentation

Fred Koller, Vice President, Business Development, Miltenyi Biotec

11.10 Networking & Refreshments

Conference Day 2: Thursday September 15th 2016




11.40 Controlling Potency, Persistence, and Toxicity of T Cell Therapies Using Molecular Switches• We expanded the use of our core

rimiducid-based, switch technology, known as “Chemically Induced Dimerization (CID)”, to develop molecular “activation” switches to complement our clinically validated inducible Caspase-9-based “safety switch” (CaspaCIDe®)

• CaspaCIDe, has consistently demonstrated prompt termination of cellular toxicity from the gene-modified cells, following a single rimiducid dose while continuing some therapy, due to residual, non-alloreactive cells

• The novel, antigen-independent activation switch, “MC”, based on signaling elements from MyD88 and CD40, can be modified for either constitutive costimulation or for “costimulation on demand” in the rimiducid-inducible MC variant, “iMC”

• When used with tumor-targeted CARs in animal models, intravenous administration of iMC-enhanced CAR-T therapy showed remarkable efficacy against both hematologic and solid tumors

• Engineering T cells to co-express MC-enhanced CARs along with CaspaCIDe led to potent anti-tumor efficacy in vivo with rapidly controlled toxicity despite functional T cell persistence

• By combining iMC with several classes of tumor-specific TCRs, we have demonstrated broad applicability to multiple antigen classes

• Antigen-independent iMC activation within a repressive tumor microenvironment can lead to reversal of commonly observed MHC class I downregulation. All three new platforms provide enhanced control over unpredictable cellular therapies, greatly expanding their utility into broader indications

David Spencer, CSO, Bellicum Pharmaceuticals

11.40 The Horse Before the CAR-T? Pretreatment With Oncolytic Herpes Simplex I Viroimmunotherapy Augment CAR-T Cell Therapy• While CD19-directed CARs

demonstrate significant anti-leukemic efficacy, CARs directed against solid tumors have been less successful. Some potential explanations include limited CAR tumor-homing and infiltration, insufficient proliferation, and poor persistence

• These findings are likely attributable to the immunosuppressive microenvironment, oncolytic viroimmunotherapy is an attractive adjunct to cellular therapies, as these attenuated viruses not only cause direct tumor-specific cell death, but also induce pro-inflammatory signals

• We sought to determine whether the use of oHSV might enhance third-generation GD2-directed human CAR-T cell efficacy in GD2-expressing tumors. Using flow cytometry, we confirmed our CARs expressed CXCR-3 and CCR-5, allowing chemotactic signaling through CXCL-10 and CCL-5, respectively. In transwell migration assays, we found increased CAR migration toward oHSV-infected over non-infected tumor cells

• Survival studies using athymic nude mice and cyclophosphamide lymphodepletion prior to CAR therapy demonstrated delayed tumor growth and prolonged survival of mice treated with combination therapy compared to CAR monotherapy

• These results indicate that Seprehvir is a valuable adjunct to CAR T-cell therapy and the combination should be further explored

Kellie Haworth, Pediatric Hematology, Oncology Fellow, Nationwide Children’s Hospital

11.40 Clinical Considerations for CAR-T Cell Therapy: A Regulatory PerspectiveAdoptive CAR-T cell therapies are an innovative and promising class of products in cancer therapy. However, there are many challenges involved in the development of these complex products.

This presentation will focus on the clinical considerations for CAR-T Cell therapy from a regulatory perspective. In order to further develop these products, careful consideration must be given to the following:

• Selection of ideal antigen targets

• Identification of appropriate patient populations

• Selection of trial design and endpoints

• Management of toxicities

• Additionally, manufacturing and quality control issues need to be taken into account

• The basis for US regulatory approvals will be reviewed, followed by regulatory considerations for efficacy evaluation and risk assessment and mitigation strategies, specifically focused on CAR-T cell therapy development

Kristin Baird, Medical Officer, Division of Clinical Evaluation, Pharmacology & Toxicology, Office of Cellular, Tissue & Gene Therapies, CBER, FDA

Discovery & Genetic T-Cell Engineering

Translation & Clinical Development

Manufacturing, Supply Chain & Commercialization

Innovative Methods to Overcoming Safety & Toxicity

Challenges

Combination Therapies & Enhancing the Efficacy of

Frontline Cancer Therapies

Regulatory Expectations & Guidelines




12.10 Novel Cellular Engineering: Controlling Specificity and Activity of Adoptive Cellular Therapies• At CDL we have developed a suite

of technologies that will provide novel cellular control mechanisms to reduce toxicities while maximizing efficacy

• CDL’s ON-switch and SynNotch technologies allow, for the first time, the ability to control the intrinsic activity of the CAR-T with a small molecule definable mechanisms in the case of ON-Switch and a logic gated decision paradigm in the case of SynNotch

• The application of these systems in the clinic will provide the physicians with the ability to modulate safety issues like CRS and on/off target toxicities while maximizing efficacy

Peter Emtage, CEO & Founder, Cell Designs

12.10 PD1 Inhibition and Autologous T cells Expressing a GD2 Specific CAR with CD28 and OX40 Costimulatory Endodomains in Combination for Children with Neuroblastoma• We conducted a Phase I study with

adaptive trial design of T cells expressing a GD2 CAR with both CD28 and OX40 costimulatory endodomains

• We tested the safety, persistence and antitumor efficacy of the CAR T in 11 patients with neuroblastoma, in 3 cohorts

• The treatments were tolerated well and no dose limiting toxicities were observed

• Antitumor responses were modest. Thus, T cells expressing a GD2 CAR with CD28 and OX40 costimulatory endodomains are safe, expand and are detectable for >4 weeks in vivo but early antitumor responses were disappointing even after lymphodepletion and co-administration of PD1 Ab

Andras Heczy, Director, Liver Tumor Program, Assistant Professor, Baylor College of Medicine

12.10 EMA Regulatory Perspective: Evolving the Regulatory Conversation to Match Scientific Innovation• What are the European regulatory

considerations?

• Will these therapies be accelerated through development with breakthrough designation given resounding early results?

• Understanding requirements for validating and translating preclinical research on novel targets

• What standards need to be in place for therapy development and manufacturing?

Paula Salmikangas, Chair, Committee for Advanced Therapies, EMA

12.40 Identifying the Best Killers: Kinetic Assessment of CAR-T Cell Potency Against Liquid and Solid Tumors • We have developed a cell-based

screening instrument (xCELLigence) that enables quantitative assessment of CAR-T cell cytotoxicity towards both solid and liquid target tumor cells

• We will present data demonstrating that kinetic assessment of CAR-T cell potency using xCELLigence provides superior sensitivity and dramatically expanded assay

• The xCELLigence assay will be shown to provide a simple and efficient workflow

• We will demonstrate how xCELLigence is being used to study the fine-tuning of CAR-T cell killing activity using immune modulating agents

Yama Abassi, VP, Acea Biosciences

12.40 Pfizer Allogeneic CAR-T • Heme targeting strategy

• Selection of CAR characteristics

Barbra Sasu, Senior Director, Rinat/Pfizer

12.40 Your CAR-T Therapy has the Green Light? How Strong is Your Market Access Strategy?• Be clear on your patient population:

demographics, psycho-social needs that may complicate medical, stage in disease process

• Understand payer reimbursement models for your CAR-T therapy

• Know your barriers to entry: competition, provider expectations, access/payers

• Be clear on your program design

• Define and design how you will distribute and manage your product

• Allow sufficient time to get your product launched

• How will you get data back to measure activity and outcomes? What reporting expectations do your C Suite and/or investors have?

Jan Nielsen, Vice President, Sonexus™ Access & Patient Support, Cardinal Health Specialty Solutions

1.40 Lunch & Networking




2.10 New Models of Engineered T Cells for Cancer• Since the 1990’s, we have conducted

clinical trials of gene modified T cells

• Chimeric antigen receptor (CAR) T cells targeting CD19 on B cells leukemias and lymphomas have induced durable complete responses in patients who are relapsed or refractory to all other available treatments

• This technology is now in global multi-center clinical trials

• CAR T cells targeting new targets in hematologic malignancies and in solid tumors are underway and provide demonstration that it is possible to design immunity at will for therapeutic application

Bruce Levine, Director, Clinical Cell & Vaccine Production Facility, Perelman School of Medicine & Abramson Cancer Center, University of Pennsylvania

2.10 Novel CARs Introduced into NK Cells Facilitate Potent Tumor-Cell Killing that Results in Tumor RegressionRohit Duggal, Director, ExperimentalTherapy, Sorrento Therapeutics

2.10 How Will New Cancer Cell Therapies be Reimbursed? In the US multiple value frameworks have been proposed and implemented in an attempt to address payors’ concerns on costs. We will explore the current reimbursement ecology for new products, the implications of new payment methods, and the movement towards outcomes based reimbursement.

• What this means for development and launch of oncology products?

• Calculation of value, cost offset and budget impact?

• How are current therapies viewed by payors and what are the implications for new therapies?

• What are cures and what are they worth?

• What is outcomes based pricing, will it happen?

Edmund Pezalla, VP, National Medical Director, Pharmaceutical Policy & Strategy, Aetna

The Future of CAR-T Development: Emerging

Innovations & Technologies

Unconventional CAR Approaches: Alternative CAR vehicles &

Looking Beyond Cancer

Pricing, Partnering & Novel Business Models

2.40 Further Development Of CAR-T Cells for the Treatment of Cancer• One anti-BCMA CAR (bb2121)

candidate was selected based on strong surface expression, superior biological activity to multiple BCMA+ cell lines, and low antigen-independent reactivity. Robust recognition of as little as 220 BCMA molecules/cell permitted reactivity to B cell tumors including primary CLL

• Based on this and other data, bluebird bio, in collaboration with Celgene, has initiated a phase I clinical trial of anti-BCMA CAR T cells in patients with multiple myeloma

• Prior investigators have shown improved therapeutic efficacy by enriching for memory CAR T cells, yet current selection methods based on antibody-selection are cumbersome, expensive, and are difficult to scale.

2.40 Target Engineered Natural Killer Cells as “Off-the-Shelf” Cellular Therapeutics• NantKwest has developed the NK

cell line NK-92 as an “off-the-shelf” activated natural killer (aNK) cell platform

• These cells have the capacity to destroy infected cells and cancer cells independent of major histocompatibility complex matching, as they lack inhibitory killer-cell immunoglobulin-like receptors

• The safety of aNK cells as well as their activity against a broad range of cancers has been confirmed in several phase I clinical trials

• aNK cells can be administered in the outpatient setting and serve as a universal cell-based therapy without the need for individualized patient matching

2.40 Partnering and External Innovation: A Key Asset for Flexibility in Building a Cancer Portfolio of Combination Therapies• Part of our strategy is to continue

to develop a balanced portfolio within immuno-oncology. From significantly de-risked targets with established clinical proof-of-concept to targets that have a strong biologic rationale and emerging clinical data to exploratory areas of immuno-oncology (such as CAR-T.)

• We actively seek a wide range of collaborations and different relationship structures that expand our capabilities and create synergies for innovation in immuno-oncology drug development. Further, collaboration takes on added importance in the context of immuno-oncology with its diverse technologies, IPs and approaches that are often complementary in unforeseen ways




• We found that addition of a PI3-kinase inhibitor during bb2121 manufacture represented a facile method to enrich for memory-like CAR T cells without a complicated cell sorting procedure

• bb2121 CAR T cells cultured with PI3K inhibition expressed markers associated with T cell memory such as; CD62L, CD127, and CD197

• A defining property of memory T cells is durability despite multiple antigen encounters. In a xenograft “stress test”, we evaluated the persistence of bb2121 CAR T cells, grown under different conditions, for their ability to survive post-clearance of multiple myeloma

• Only mice treated with bb2121 CAR T cells cultured with PI3K inhibition were able to control the tumor re-challenge

• These data demonstrate that a potent, antigen-dependent, memory-like BCMA CAR T cell produced with a manufacturing process that is scalable for industrialization has promise for robust tumor regressions in clinical application

Rick Morgan, VP Immunotherapy, bluebird bio

• The aNK cell platform has been bioengineered to incorporate a high-affinity antibody-binding CD16 receptor (haNK).These cells augment monoclonal antibody (mAb) activity by providing antibody-dependent cell-mediated cytotoxicity

• Both aNK and haNK cells can be bioengineered to express chimeric antigen receptors (CARs) to further optimize targeting and potency. These taNK and t-haNK variants can be generated using mRNA transfection. The t-haNK platform is especially attractive as it allows for dual targeting (i.e. CD19-CAR with a CD20 mAb)

Hans Klingemann, VP, R&D, NantKwest

• Recently, we have established new collaborations in immuno-oncology through deals in checkpoint inhibitors and in gene editing of CAR-T cell therapies

• Partnering to obtain innovative assets from external collaborators is part of our overarching strategy. Access to assets for innovative cancer drug development can come through sole ownership and through partnering

• We actively partner/collaborate for external assets and innovative technologies to complement our efforts and further drive assets forward

• Innovation in such a complex and rapidly evolving space as immuno-oncology is more than any one company can manage

• Partnerships and licensing arrangements give us maximum flexibility and a much higher likelihood of success. Baxalta’s immune-oncology portfolio reflects a range of approaches, derived from internal innovation as well as synergizing with external innovation

David Meek, President, Oncology, Baxalta

3.10 Afternoon Refreshments & Networking

3.40 PANEL: The Business of Engineered T Cell Therapies• Ensuring the commercial viability of products• Mergers and acquisitions • Partnering and licensing • Encouraging collaboration in the space• Overcoming IP challenges to enhance forward movement in the field

David Meek, President, Oncology, BaxaltaAxel Hoos, Senior Vice President, TA Head Oncology R&D and Head, Immuno-Oncology, GSK

4.40 A Patient’s Experience with the Initial CART 19 Trial and Perspectives on Patient Access to New Breakthroughs for Cancer TreatmentDoug Olson was diagnosed with CLL in 1996. By 2010 his cancer had become refractory to standard treatments and he was rapidly running out of options. Doug was patient number 2 in the initial 2010 CART 19 safety trial.• The treatment used his own reprogramed T-cells to recognize and kill CD 19

containing CLL cells. Virtually all detectable cancer cells were eliminated in less than 4 weeks after the initial infusion of the reprogramed T-cells.

• He has been in complete remission for over 5 ½ years.The speed of advances is breathtaking in immunotherapy and other promising new treatments for cancer, which are saving lives and providing hope to all cancer patients. However, these breakthrough treatments are extremely expensive and making these treatments accessible to all patients has become a major challenge. Issues and challenges to patient access to cancer therapy will be discussed.

Doug Olson, Patient Advocate, The Leukemia and Lymphoma Society

5.10 Chairman’s Closing RemarksFrederic Lehmann, VP, Head, Oncology Franchise, Celyad

5.15 Close of 2nd CAR-TCR Summit 2016




Pre-Conference Workshops: Tuesday September 13th 2016




Autologous vs. Allogeneic: Which Will Succeed in the Race to Market?

Overcoming the Inhibitory Tumor Microenvironment

Autologous vs. allogeneic is a time long debate that has been apparent in the cell therapy space for many years. With the emergence of cell immunotherapies and their current success, this debate is reignited. Join this workshop to weigh the risks and benefits of each cell type and discuss:

• Current successes and positives seen with allogeneic and autologous approaches

• The different production processes and how they vary in complexity and expense

• Overcoming rejection and immunogenicity

• Ensuring long term efficacy of cells

• Is allogeneic the way forward for pharma from a cost and value perspective?

• Will we still need autologous once allogeneic cells have shown proof of concept and success in trials?

• Can we create a controlled defined product?

• Is there possibility for standardization?

Leave this workshop having explored the different possibilities for cell therapies and come to a decision about which you think will succeed the race to market.

The solid tumor microenvironment provides a safe haven for tumor cells amongst other attributes providing immune escape. This tumor microenvironment is key to tumor survival attend this workshop to identify and discuss:

• Mechanisms to overcome the inhibitory environment

• Utilizing combination drugs to provide optimum conditions for engineered T cell therapies

• Genetic engineering mechanisms to prime T cells for attack

• Enhancing the potency of engineered T cells to overcome the tumor microenvironment

• CAR/TCR cells that are resistant to immunosuppressive agents

• Complimentary immunomodulating agents

This workshop provides the platform to fuel discussion and debate, enable knowledge sharing and allow you to make the connections you need.

Time: 9.00am - 12.00pm

Time: 9.00am - 12.00pm

Sicco PopmaScientific Director, Gene Modified Cell Therapy LeaderJohnson & Johnson

Jason LukeAssistant Professor, MedicineUniversity of Chicago

Experienced, innovative and strategic Scientific Director with proven history of successful project leadership in a high-performance, multimillion dollar, venture environment investigating transplantation, immune mediated inflammatory diseases and stem cell therapy.

Jason J. Luke, M.D., F.A.C.P. is an Assistant Professor of Medicine at the University of Chicago where he specializes in the management of patients with melanoma and early phase drug development. Dr. Dr. Luke has served as the chair of the education committee and as a member of the scientific committee for the melanoma track of the ASCO annual meeting. Dr. Luke has received several awards including a Paul Calabresi Career Development in Clinical Oncology Award (K12), an ASCO Merit Award as well as Young Investigator Awards from the Melanoma Research Alliance, the Cancer Research Foundation and the Conquer Cancer Foundation of ASCO. Dr. Luke’s research has been supported by ASCO, the National Comprehensive Cancer Network and the National Cancer Institute.

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Pre-Conference Workshops: Tuesday September 13th 2016




Solid Tumor Target Identification and Validation

The Transformative Power of Correlative Sciences in Adoptive Immuno-Gene Therapies

Understand how we can effectively target solid tumors will ensure the continued success of genetically engineered cell immunotherapies. We will aim to tackle and pose the main challenges and questions in this space to learn and debate the following:

• Tumor microenvironment and how we can overcome the immunosuppressive environment

• Delivery methodologies: Local vs. systemic to ensure appropriate tumor accessibility

• How can we ensure that cell therapies retain potency in the tumor microenvironment?

• Current solid trial data: Where are we seeing success if any?

• Enhancing proliferation for improved efficacy of cell activity

• Improved target ID and validation for increased T cell activation

• Antigenic landscape of solid tumors: Ensuring target specificity

Leave this workshop with the knowledge you need to tackle solid tumor cancers and translate the early success seen with liquid tumors.

Adoptive cell therapy using gene-engineered T cells to boost the in vivo anti-tumor effect has made significant progress over the past decade, and has evolved into a highly effective treatment modality for patients with various forms of leukemia.

In this workshop I will highlight:

• Our most recent progress in the treatment over 200 patients with chimeric antigen receptor-expressing T (CART) cells

• Share data from our treatment of patients with acute and chronic lymphoid leukemias and multiple myeloma

• Discuss biomarkers of product potency and also toxicity, including our work on predictive modeling of cytokine release syndrome in acute lymphocytic leukemia

• Our experience with identifying predictive and targetable biomarkers of cytokine release syndrome, and predictive biomarkers of response.

• Our experience with CART cell therapies in this patient group, emphasizing the significance of correlative studies in the evolution of this form of curative cancer therapies

At this workshop share knowledge on the current biomarkers under investigation and begin to understand and develop your own biomarker strategies for your trials.

Time: 1.00pm - 4.00pm

Time: 1.00pm - 4.00pm

Edmund MoonAssistant ProfessorUniversity of Pennsylvania

Jos MelenhorstDirector, Product Development & Correlative Sciences laboratoryUniversity of Pennsylvania

Dr. Melenhorst obtained his degree of associate professor in continuing education at the Moller institute, Tilburg, Netherlands, and his Masters degree in Medical Biology from the Nijmegen University, Netherlands. He did his PhD at the Leiden University, Netherlands, on the pathogenesis of Aplastic Anemia.

In 1998 Dr. Melenhorst moved to Bethesda, Maryland, where he did his research - first as a post-doc, later as a staff scientist - at the National Heart, Lung, and Blood Institute, National Institutes of Health, on the immunobiology of marrow failure and leukemic disorders, and allogeneic stem cell transplantation. In 2012 he was recruited by Dr. Bruce Levine as Deputy Director of the clinical Cell and Vaccine Production Facility at the University of Pennsylvania. In that position he was responsible for, a.o., building and overseeing activities in the Quality Control and Process Development groups.

Edmund K. Moon is an Assistant Professor of Medicine in the Division of Pulmonary, Allergy, and Critical Care at the University of Pennsylvania. He trained in both pulmonary and critical care fellowship and interventional fellowship at UPenn. He underwent research fellowship training under the co-mentorship of Dr. Steven M. Albelda and Dr. Carl H. June. His overall research interest is understanding the phenotype of hypofunction that adoptively transferred engineered T cells undergo upon infiltration into solid tumors. He has a particular interest in using this understanding to further modify engineered T cells to increase their resilience to the immunosuppressive microenvironment of lung cancer and malignant pleural mesothelioma tumors. - OR -

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Automatizing Single Use Systems and Novel Production Paradigms for Effective Manufacturing

Manufacturing of engineered cellular therapies is complex and expensive, riddled with manual steps there is a large room for error and quality control is paramount.

This workshop aims to discuss:

• Strategies to simplify manufacturing processes

• How to decrease the cost of goods

• Innovative technologies to optimize the manufacturing approach

• How scalable are T cell engineered technologies?

• How do we ensure scale up and scale out occurs effectively?

• Adopting single use systems

• What does the ideal strategy need to look like?

• Reliability of the product

• What lessons can be learnt from previous attempts at commercial-scale cell therapy transfer?

Explore the possibilities and take a hands on practical approach to creating the best strategy possible with a room full of experts in engineered T cell transfer.

Time: 9.00am - 12.00pm

Post-Conference Workshops: Friday September 16th 2016

CAR-T Cell Therapy: Regulatory Challenges and Opportunities

As engineered T cell therapies are quickly becoming a clinical reality, we are seeing more candidates move through the development phases towards approval. However, as a novel therapy there is still much to be understood from the regulatory perspective for both the developers and the regulatory bodies. This workshop is an opportunity to engage with representatives from two of the biggest regulatory bodies to understand the key challenges and opportunities faced as pertains to the development of CAR-T and TCR therapies. We will be discussing:

• An overview of the regulatory process from start to finish

• The regulatory perspectives on the unique challenges facing adoptive cellular therapies

• Approaches to addressing safety and toxicity concerns

• Approaches to standardization of the manufacturing process across manufacturing sites and demonstration of comparability

• Discussion of quality control and assurance aspects

Leave this workshop with practical knowledge to help approach regulatory requirements for your engineered cell therapy.

Time: 9.00am - 12.00pmPaula SalmikangasChair, Committee for Advanced TherapiesEMA

Kristin BairdMedical Officer, Division of Clinical Evaluation, Pharmacology and Toxicology, Office of Cellular, Tissue and Gene TherapiesCBER, FDA

Kristin Baird, MD, is a Medical Officer in the Oncology Branch of the Office of Cellular, Tissue and Gene Therapies, CBER/FDA. Dr. Baird first joined the FDA in 2013 and serves on the CAR T-cell and the CAR T-cell Cytokine Release Syndrome Working Groups.

Dr. Salmikangas is a biochemist by original training, with a Ph.D. in muscle cell biology. Dr. Salmikangas has worked as a senior researcher at Finnish Medicines Agency, Finland since 2003. Her main areas of expertise are biological medicinal products, especially cell-based medicinal products and combination products.

Rodney RietzeLead cGMP Process AutomationNovartis

Dr. Rietze is a cell biologist with 15+ experience in basic research, drug discovery and developing cell-based therapies for neural, immune and cardiovascular indications in academic, biotech and pharmaceutical settings.

Dr. Rietze began his research career in the laboratory of Professor Samuel Weiss, who is credited with the discovery of adult neural stem cells (NCSs), studying the role of these cells in adult neurogenesis.

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Current Trends and Best Practices in Outsourcing for Long Term Cell Immunotherapy Success

Discover how various types of organizations identify outsourcing partners throughout the drug development process and cultivate partnerships that best fit their strategic objectives.

Attend this workshop to discuss:

• Key things to consider and look for in a CMO relationship

• What are some key considerations in an internal vs. outsource build decision?

• How can organizations best position itself in the cost/time/quality triangle?

• How do organizations ensure that they select vendors that are the best fit?

• What regulatory considerations are most critical to consider in a partnership context?

• How do organizations successfully manage the vendor selection process?

• How do organizations successfully manage the CMO relationship?

• How do organizations structure a relationship for optionality and scalability as you approach commercial?

Leave this workshop with the knowledge you need to implement a robust strategy to select, manage and maintain vendor relationships.

Time: 1.00pm - 4.00pmPeter OlagunjuSenior Director, Vendor Managementbluebird bio

Pricing and Reimbursement Strategies for Engineered Cell Therapies

The question of how adaptive T cell therapies should be priced is on everybody’s mind. From driving down the cost of goods to ensuring a truly effective product. Setting pricing still remains a hot topic within the engineered T cell therapy space.

This workshop will discuss:• Complexity of pricing a “living drug”

- Variability of effect- Product loss and failed attempts- Changes in supply/capacity and setting of care

• Reimbursement models considering cost of care in pricing strategies• Comparing engineered T cell therapy with current front line therapies• Is the benefit to patients worth the cost? Outcomes based pricing

structures• Dosage considerations• Cure vs. long term repeated treatments?

- How do we define a cure?- What if the cure does not materialize: the role for outcome - based concepts

We will use role-playing and other interactive tools to engage with each other and with expert stakeholders across the full CAR-TCR development chain to understand the varying perspectives on pricing and reimbursement of these therapies. Leave this workshop with more clarity on potential pricing and reimbursement structures.

Time: 1.00pm - 4.00pmEdmund PezallaVP, National Medical Director, Pharmaceutical Policy & StrategyAetna

Edmund Pezalla, M.D., MPH, is Aetna’s National Medical Director for Pharmaceutical Policy and Strategy. He is responsible for the integration of pharmacy policy and activities into Aetna’s overall strategy and operations. Dr. Pezalla also serves as the lead clinical spokesperson for Aetna in pharmacy related issues and represents Aetna on industry work groups and conferences.

Dr. Pezalla is Aetna’s leading executive on pharmaceutical development, reimbursement strategy and drug evaluation. He is active on projects with the IOM, CDC and FDA as well as MIT’s Center for Biomedical Innovation.

Peter is currently the Senior Director of Vendor Management with bluebird bio. In this role, Peter has responsibility for managing the global contract manufacturing and testing network. Prior to bluebird bio, Peter most recently served as Senior Director, Global Technical Operations at Valeant. He was at Dendreon for roughly 5 years where he managed the US and EU manufacturing operations and supply chain, including contract manufacturers, external testing sites, and the apheresis network for those regions.

Post-Conference Workshops: Friday September 16th 2016

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CelyadWith product candidates in oncology and cardiology, Celyad seeks to address diseases with high unmet medical needs such as heart failure and cancer. Celyad leverages unique know-how in taking cell based therapies from bench to Phase III, as well as the manufacturing and logistical infrastructure for such complex products. Celyad builds it business model on partnering with prominent research institutions and develops those programs from bench to commercial applications.www.celyad.com

Miltenyi BiotecMiltenyi Biotec’s mission is to improve scientific understanding and medical progress. We provide products and services that advance biomedical research and cellular therapy. Honoring this mission drives our commitment to support the translation of basic research into therapy in the areas of immunology, cancer, neuroscience and stem cell biology. We innovate products that address sample preparation, separation of cells and their analysis, and that advance the concept of cellular therapy.www.miltenyibiotec.com/en/

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For Sponsorship Opportunities Contact:

Jonathan Kilby-Philips Commercial Manager

Tel: +44 (0)203 141 8700 Email: [email protected]




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It’s rare to attend a conference and find every talk of interest. The inaugural CAR-T Summit 2015 assembled experts and front line companies to create an engaging, informative conference with just the right amount of networking opportunities.

Jeff Till, Director, External Innovation, EMD Serono

VenueHyatt Regency BostonOne Avenue de LafayetteBoston, Massachusetts, USA, 02111 www.regencyboston.hyatt.com

Academics are entitled to 40% off the Pharma & Biotech prices.

deliver the next generation of safe, effective & commercially...

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