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North American June 28-30, 2016 • Boston, MA Pre-Conference Workshops Day: June 28, 2016 Main Conference: June 29-30 Seaport Hotel & Seaport World Trade Center BOOK THE ALL ACCESS PASS AND GET 2 FULL WORKSHOPS ON JUNE 28TH! See Pricing for Details. Presents… 2016 Media Partners Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. Over the past 3 years, the industry has been a hive of activity, with a 6 companies forging deals valued at over half a billion dollars in total. Meet and Network with Representatives from: CAR T, TCR’s and Beyond A focus on the issues that matter: Commercial Deliverability & Academic Collaboration. Proof of Principle – Case Studies on Solid Tumor Malignancies; Safety – Open discussions on Toxicity; T-Cell Quality - What T-Cell Subsets to use? Finding Targets - Overcoming the micro environment and immunoescape. Hear from the most influential names in Adoptive T-Cell Therapies with executive speakers from the top pharmaceutical companies globally as well as the world’s leading clinical investigators. Benefit from over 12 hours of networking across the 3 day congress, allowing participants to build collaborative relationships. Join us as we deliver a comprehensive assessment of the scalability, manufacturing and commercialization questions around T-Cell Immunotherapy Meet with the technology providers currently pioneering developments for T-Cell engineering and distribution. We aim to foster further consensus with a focus on academic collaboration, networking and business partnering within the fields of CAR-T, TCR’s and TIL’s. Key topics we’ll address include: Robert Hawkins Professor of Medical Oncology, University of Manchester and CEO Cellular Therapeutics Ltd Nabil Ahmed Associate Professor, Baylor College of Medicine, USA Jan Joseph (Jos) Melenhorst Director, Product Development & Correlative Sciences laboratory, University of Pennsylvania Hans Schreiber Professor, Department of Pathology/MPMM, Cancer Research Center, University of Chicago Andrew Gerry Director of Preclinical Research, Adaptimmune Andrew Sewell Professor, Cardiff Institute of Infection & Immunity, Cardiff University Gain insight from speakers including: Dr. Yangbing Zhao Adjunct Associate Professor in the Department of Pathology & Laboratory Medicine at the University of Pennsylvania

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Page 1: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

North American

June 28-30, 2016 • Boston, MA Pre-Conference Workshops Day: June 28, 2016

Main Conference: June 29-30Seaport Hotel & Seaport World Trade Center

BOOK THE ALL ACCESS PASS AND GET 2 FULL WORKSHOPS ON JUNE 28TH! See Pricing for Details.

Presents…

2016 Media Partners

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected].

Over the past 3 years, the industry has been a hive of activity, with a 6 companies forging deals valued at over half a billion dollars in total.

Meet and Network with Representatives from:

CAR T, TCR’s and Beyond

A focus on the issues that matter: Commercial Deliverability & Academic Collaboration. Proof of Principle – Case Studies on Solid Tumor Malignancies; Safety – Open discussions on Toxicity; T-Cell Quality - What T-Cell Subsets to use? Finding Targets - Overcoming the micro environment and immunoescape.

Hear from the most influential names in Adoptive T-Cell Therapies with executive speakers from the top pharmaceutical companies globally as well as the world’s leading clinical investigators.

Benefit from over 12 hours of networking across the 3 day congress, allowing participants to build collaborative relationships.

Join us as we deliver a comprehensive assessment of the scalability, manufacturing and commercialization questions around T-Cell Immunotherapy

Meet with the technology providers currently pioneering developments for T-Cell engineering and distribution.

North American

We aim to foster further consensus with a focus on academic collaboration, networking and business partnering within the fields of CAR-T, TCR’s and TIL’s.

Key topics we’ll address include:

Robert Hawkins Professor of Medical Oncology, University of Manchester and CEO Cellular Therapeutics Ltd

Nabil Ahmed Associate Professor, Baylor College of Medicine, USA

Jan Joseph (Jos) Melenhorst Director, Product Development & Correlative Sciences laboratory, University of Pennsylvania

Hans Schreiber Professor, Department of Pathology/MPMM, Cancer Research Center, University of Chicago

Andrew Gerry Director of Preclinical Research, Adaptimmune

Andrew Sewell Professor, Cardiff Institute of Infection & Immunity, Cardiff University

Gain insight from speakers including:

Dr. Yangbing Zhao Adjunct Associate Professor in the Department of Pathology & Laboratory Medicine at the University of Pennsylvania

Page 2: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected].

The advances in T-Cell therapy have garnered a deserved global interest with even President Obama calling for a ‘moon shot’ to cure cancer and hailing immunotherapy as a revolutionary step forward. CD19 CAR T therapy has shown the world how effective t-cell therapy can be, but many significant academic and commercial challenges remain. Key topics of discussion at the congress will include:

• Safety is of primary concern with controlling levels of toxicity being a key topic of discussion

• How to create a universal ‘off the shelf’ product

• Managing effective translation in the clinic

• Solid Tumor Targets - Finding out what makes an antigen a good antigen? And for what tumor?

• Commercial deliverability: Scalability, manufacturing and commercialization.

• Immunoescape and altering the tumor micro environment

The Adoptive T-Cell Therapy Congress is a platform committed to furthering consensus on developing state of the art T-cell therapies by focusing on academic collaboration, business partnering and commercialization. The congress will focus on the fields of CAR-T, TCR’s, TIL’s, NK immune checkpoints and biospecifics with sessions including: advancements in T-cell engineering; efficacy in different cancer models and results from human clinical trials. Attending the congress brings you together with thought leaders from biotech, big pharma, and academia as well as the wider service community and gives you access to strategies that are critical to furthering the development of viable T-cell therapies.

Join us and network with thought leaders such as:

• Robert Hawkins, Professor of Medical Oncology, University of Manchester and CEO, Cellular Therapeutics Ltd

• Nabil Ahmed, Baylor College of Medicine, USA

• Andrew Sewell, Cardiff University, UK

• Sicco Popma, Scientific Director, Gene Modified Cell Therapy Leader at Janssen R&D

• Hans Schreiber, University of Chicago, USA

• Andrew Gerry, Director of Preclinical Research, Adaptimmune

Now is the time to discover more about the exciting new developments within adoptive T-cell therapies. Join us in Boston this coming June 28-30, 2016 for an exciting networking, learning and benchmarking forum.

We offer special early bird prices off industry rates as well as reduced academic rates, so book now online at www.tcellcongress.com.

I look forward to meeting you in Boston!

Best Regards

Calan Smith Event Director, Kisaco Research

Welcome to the Adoptive T-Cell Therapy Congress

2

Science Magazine declares Cancer Immunotherapy ‘Breakthrough of the Year’ with T-cell’s on the attack front page article.

• Preclinical and clinical

scientists

• Clinical investigators

• Postdoctoral fellows

involved in cancer

research

• Regulatory scientists

• Healthcare

professionals

Pharmaceutical industry

• Reagents and

equipment suppliers

• Distributors

• Cold Chain logistics

• Biopharma

• Research Institutes

• Associations

• Conferences in the Human Microbiome, Adoptive T-Cell Therapy, Plant Improvement Technologies and more

• Over 70+ academia, scientists and industry executives onsite

• Global industry partners including BaseClear, Libragen, List Laboratories, Synthetic Biologics, BioMed Central, Gen Eng News, All Seq and more

• CPD Accreditation-Approved by the Royal College of Physicians

Kisaco Research works with the early adopters and leaders of growth markets in driving their respective industries forward and in providing the right knowledge, learning and social opportunities to stimulate business growth quickly and effectively.

Kisaco Research produces, designs and hosts B2B industry conferences and exhibitions. Our platforms are neutral, so that our attendees get the right information from the most relevant people. Our level of research ensures the topics and products we offer are of utmost relevance and timeliness; our 30+ years of combined experience in the event industry means we have an unmatched level of strategic social engineering onsite.

Join our conferences to ensure you benefit from the high-quality knowledge, learning and networking opportunities. Find out more about our upcoming events by emailing [email protected].

About Kisaco Research

Who Will You Meet?

Kisaco Research’s Global ‘Next in Pharma’ Conference Portfolio

PS. Complement your main conference with a

selection of workshops on June 28th - Save when

you book the All Access Pass!

Page 3: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

2016 Speaker FacultyNorth American

Jan Joseph (Jos) Melenhorst

Director, Product Development & Correlative

Sciences laboratory, University of Pennsylvania

Sicco Popma Scientific Director, Gene Modified Cell Therapy Leader at

Janssen R&D

Claudia Zylberberg CEO

Akron Biotech

Chiara Bonini Experimental Hematology

San Raffaele Scientific Institute

John R. Desjarlais Chief Scientific Officer

Xencor, Inc

Andrew Gerry Director of Preclinical Research,

Adaptimmune

Dr. Yangbing Zhao Adjunct Associate

Professor in the Department of Pathology

& Laboratory Medicine at the University of

Pennsylvania

Robert Hawkins Professor of Medical Oncology, University

of Manchester and CEO Cellular Therapeutics Ltd

Hans Schreiber Professor,

Department of Pathology/MPMM, Cancer Research CenterUniversity

of Chicago

Hinrich Abken Professor

University of Cologne

Nabil Ahmed Associate

Professor, Baylor College of

Medicine, USA

Arnaud Foussat Chief Scientific

Officer TxCell SA

Gary C. du Moulin Associate Professor of

Drug Regulatory AffairsMassachusetts College

of Pharmacy and Health Sciences University

Julianne Smith Group Leader at

Cellectis Therapeutics

Andrew Sewell Professor,

Cardiff Institute of Infection &

ImmunityCardiff University

Peter Hoang SVP, Business

Development and Strategy Bellicum

Pharmaceuticals

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. 3

Page 4: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

Workshop Day

North American

08:30 Registration Opens for Workshop A

9:00 WORKSHOP A: T-Cell based treatment of inflammation: antigen-specific Treg cells, TCR and CAR engineered Treg cells in transplantation, autoimmunity and chronic inflammatory diseasesAn Emerging Field with Potential for industrialisation

Regulatory T cells “Treg” naturally control inflammation and induce immune tolerance. As such, these cells can be used as efficient pharmaceutical agents through adoptive cell therapy protocol to treat patients with inflammatory diseases, including autoimmune diseases, chronic inflammation and transplantation. Several subtypes of Treg cells and multiple approaches are being currently tested, not only in models on disease in animals but also in patients in early stage clinical trials. Those approaches include the use of polyclonal Tregs, TCR engineered Treg and CAR-Tregs. For therapeutic purposes, Treg cells can be either isolated directly from peripheral blood using a cell sorting device or differentiated in vitro with specific differentiation cocktails. Taking into account the extremely low frequency/numbers of Treg cells obtained with the two methods, an ex-vivo expansion is considered mandatory for obtaining doses allowing therapeutic intervention. Being able to produce therapeutic Tregs on an industrial scale will be a first step towards meeting the healthcare community’s transplantation needs and its need for treatments for autoimmune and chronic inflammatory diseases. This is especially the case for patients who are refractory to available medications. With such a market potential, a future Treg treatment could very well represent the next breakthrough in the medical landscape.

Zelig Eshhar, Weizmann Institute of Science, REHOVOT, ISRAEL

Kate Mc Donald, University of British Columbia, VANCOUVER, CANADA

Qizhi Tang, University of California, SAN FRANCISCO

James Riley, University of Pennsylvania, PHILADELPHIA

Ignacio Anegon, Inserm UMR1064, Center for research in transplantation and Immunologie, FRANCE

Arnaud Foussat, TxCell SA Valbonne Sophia, ANTIPOLIS FRANCE

12:00 Networking Lunch for All Workshop Attendees

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. 4

TUESDAY, JUNE 28, 2016

12:30 Registration Opens for Workshops C and D

Choose Workshop C or D

13:00 WORKSHOP C: CAR T-Cell Workshop Personalized or off-the shelf CAR T-Cells: Will there be a winner or is there room for both?In this workshop we will compare and discuss personalized and off-the shelf approaches for gene modified cell therapy approaches across a range of product development stages.

· Brief overview of gene modified cell therapies ( Including CAR T-Cells)

· Compare (pro/con): Discovery, Preclinical, and Manufacturing/Supply Chain

· Break

· Compare (pro/con): Regulatory, Clinical and Commercial

In the end participants will walk away with a completed “work sheet” detailing whether Personalized treatments or Off-The-Shelf solutions are better in particular categories.. A pros & cons sheet will also be generated for each topic for attendees to take notes on. Topics include Discovery, Preclinical, Manufacturing, Supply Chain, Regulatory, Clinical and Commercial.

Sicco Popma, Scientific Director and leader of Gene-Modified Cell Therapies at JBIO (JANSSEN R&D)

WORKSHOP D: Approaches to Re-Imbursement of Adoptive Cell Therapy – with Professor Robert Hawkins, Chief Executive Officer and Co-founder of Cellular Therapeutics Ltd, CRUK Chair of Medical Oncology at The University of Manchester & Consultant in Medical Oncology at The Christie NHS Foundation Trust Robert’s research interests are in gene and immunotherapy. In addition to clinical training at the Royal Marsden Hospital and Addenbrookes Hospital in Cambridge he was an MRC Research Fellow with Dr Greg Winter and Dr Cesar Milstein at the MRC laboratory of Molecular Biology in Cambridge. His work there was important for the development of commercially successful antibodies.

His PhD was in antibody engineering and as a Cancer Research UK Senior Clinical Fellow he developed translational research interests in antibody based gene therapy. He was first appointed as a consultant in Medical Oncology at Addenbrooke’s Hospital in Cambridge in 1995 and then became Professor of Oncology at the University of Bristol in 1996. In 1998 he moved to the Christie Hospital to become Professor and Director of Medical Oncology. Clinically, Professor Hawkins focuses on treating renal cancer and on developing immunotherapy, particularly cellular immunotherapy. He heads a clinical research group undertaking trials and also a range of early phase clinical trials of biological agents including vaccines, antibody based molecules and cell therapies. He has successfully led the clinical development of biological agents from Phase I to Phase III studies. He has received grant funding from Cancer Research UK, the Kay Kendall Leukaemia Fund and the European Union (he is Coordinator of the ATTACK project and was coordinator of the ATTRACT integrated training network) and has published widely in scientific and clinical journals as well as an inventor/co-inventor on several antibody related patents.

Professor Hawkins led the development of the GMP authorized facility which has lead to a commercial venture - Cellular Therapeutics Ltd (CTL).

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08:30 Registration for All Attendees

08:50 Chairperson’s Opening of Conference

09:00 CAR T-Cell Targeting Dr Melenhorst has used advanced culture techniques,

molecular biology, and high definition flow cytometry to interrogate human T cell immunology and leukemia biology directly ex vivo and in vitro, and is still actively involved in research to understand how leukemias subvert immune surveillance, and the immune biology of acute myeloid leukemias.

In August of 2012, Dr. Melenhorst started working as deputy director of the clinical Cell and Vaccine Production Facility (CVPF), Hospital of the University of Pennsylvania, Department of Pathology and Laboratory Medicine, University of Pennsylvania. In January, 2014, Dr. Melenhorst assumed responsibility of the Product Development and Correlative Sciences laboratory which investigates and enhances the in vivo potency of gene therapy products and designs improved gene therapy manufacturing processes.

Jan Joseph (Jos) Melenhorst, Director, Product Development & Correlative Sciences laboratory, UNIVERSITY OF PENNSYLVANIA

09:30 TCRs in Immunotherapy The Cardiff T-cell modulation group consists of a number of

world-class researchers with a diverse skill and knowledge base that covers all areas of T-cell biology including T-cell genetics, molecular biology, protein chemistry, crystallography, cell biology and clinical investigations. The overall goal of the Cardiff T-cell modulation group is to understand the genetic, biochemical and cellular mechanisms that govern T-cell responses to human disease.

Our research outputs are extremely wide ranging and include basic studies which are aimed at understanding how the T-cell immune response is regulated, through to translational studies which are aimed at developing tools, diagnostics and treatments for human diseases such as cancer, HIV, EBV, tuberculosis and many more.

Andrew Sewell, Cardiff Institute of Infection & Immunity, UK

10:00 Cancer Immunotherapy and Gene Therapy

Hematologic malignancies, such as leukemia and lymphoma are among the most frequent human cancers. Pre-clinical and clinical studies, including bone marrow transplantation studies, demonstrated that the immune system plays a crucial role in controlling these diseases. The main goals of the laboratory are to investigate, potentiate and exploit the mechanisms underlying the immune response to hematologic malignancies through the genetic manipulation of effector cells and antigen presenting cells.

Chiara Bonini, Experimental Hematology, SAN RAFFAELE SCIENTIFIC INSTITUTE

10:30 Morning Networking Break: Coffee, Tea & Refreshments

11:00 Controlling CAR-T-Cell safety and efficacy using molecular switches

Regulating CAR-T survival, persistence and expansion in vivo following adoptive transfer is critical for maximizing therapeutic efficacy while also managing CAR-T-related toxicity. We present two molecular switches, inducible Caspase-9 (iCasp9) and inducible MyD88/CD40 (iMC) that can be used to control T-Cell levels and function in vivo by systemic administration of the small molecule dimerizing ligand, rimiducid. Here, we first demonstrate that CAR-T-Cells containing iCasp9 can be partially or completely eliminated in a rimiducid dose-dependent manner, which corresponds to a rapid reduction of cytokine levels and toxicity. Conversely, activation of iMC in CAR-T-Cells by rimiducid provides a potent costimulatory signal which, together with CAR activation, drives T-Cell proliferation, survival and anti-tumor efficacy in multiple tumor models. Thus, iCasp9 and iMC can be used as “off” and “on” molecular switches, respectively, to control T-Cell behavior in vivo to maximize therapeutic efficacy and mitigate potential toxicities.

Peter Hoang, SVP, Business Development and Strategy, BELLICUM PHARMACEUTICALS

11:30 Panel Discussion: Safety Concerns in T-cell Therapy

Despite the growing number and length of remissions using T-cell therapy to treat leukemia and lymphomas, key challenges remain with the first and foremost being, safety.

What new methods are being considered to overcome high levels of toxicity and other adverse effects?

Moderator: Peter Hoang, SVP, Business Development and Strategy, BELLICUM PHARMACEUTICALS

Panelists:

Jan Joseph (Jos) Melenhorst, Director, Product Development & Correlative Sciences laboratory, UNIVERSITY OF PENNSYLVANIA

Chiara Bonini, Experimental Hematology, SAN RAFFAELE SCIENTIFIC INSTITUTE

Andrew Sewell, Cardiff University, UK

12:30 Networking Lunch

14:00 Adaptimmune – Transforming T-Cell Therapy

Adaptimmune is a clinical-stage biopharmaceutical company developing engineered TCRs for adoptive T-Cell therapy. Our lead therapeutic is autologous T-Cells expressing an enhanced affinity TCR specific for NY-ESO (NY-ESOc259-T), and is employed in multiple oncology indications. NY-ESOc259-T has generally been well tolerated in Phase 1/2 trials in solid tumours and in hematologic cancer types and we have seen responses and preliminary evidence of tumor reduction in patients with highly refractory cancers. A second program targeting the MAGE-A10 tumor antigen is now open in initial studies in patients with advanced stage NSCLC.

Conference Day OneNorth American

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. 5

WEDNESDAY JUNE 29, 2016

Page 6: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

Target identification and validation, together with a broad and robust preclinical safety testing strategy are critical in the development of safe and efficacious affinity-enhanced TCRs. Engineering TCRs specific for tumor antigens which are self-antigens, requires balancing the need for increasing affinity to the target peptide to allow recognition of immune-selected tumours, with the risk of cross-reactivity, which increases at higher affinities. Safety screening includes both the identification of both on-target and off-tumor recognition (antigen expression in normal tissues in addition to tumor) and off-target toxicity (recognition of non-target antigens). Avoidance of these toxicities requires that the expression profile of the target antigen be shown to be specific to tumor tissues, and that the TCR is shown to be specific. Furthermore, understanding of the frequency, level and distribution of expression of target antigen in tumor tissues is key to selecting a TCR therapeutic with potential for broadly applicable therapeutic efficacy.

Andrew Gerry, Director of Preclinical Research, ADAPTIMMUNE

14:30 Tumor Infiltrating Lymphocytes for Metastatic Cutaneous and Non-Cutaneous Melanoma

We have established the UK’s only GMP-compliant and MHRA (Medicines and Healthcare Products Regulatory Agency) licensed unit capable of producing multiple T-Cell product types (CAR or TCR-modified and natural T-Cells (TIL)) using ‘clean room free technology’. This unit has produced melanoma-derived TIL products which have been successfully returned to patients. This study supports the success of melanoma TIL therapy seen in other centers worldwide and suggests that this is a viable means of treating a disease which has few effective options.

Robert Hawkins, Professor of Medical Oncology, UNIVERSITY OF MANCHESTER and CEO, CELLULAR THERAPEUTICS LTD

15:00 Engineering Best in Class T-Cells to Treat Cancers

Despite impressive clinical efficacy of T-Cells engineered to express chimeric antigen receptors (CAR), the current applications of CAR T-Cell therapy, especially for treating solid tumors, are limited by some major challenges, such as the lack of cancer specific targets, treatment associated toxicities, interference of tumor microenvironment (TME), cancer immune evasion and so forth. Our work is focused on generating best in class T-Cells with the aims to solve these challenges.

Using different tumor mouse models, we tested CAR T-Cells generated by different manufacture protocols, such as CD3/CD28 Dynal beads, OKT3/IL2, K562 based artificial APC and rapid T-Cell expansion protocol, compared with a novel T-Cell expansion protocol that uses RNA electroporation.

The results of the studies show that the ways of generating T-Cells influence greatly for their anti-tumor activities, both in vitro and in vivo.

Target-mediated toxicity is a major limitation in the development of CAR for adoptive cell therapy of solid tumors. We developed a strategy to adjust the affinities of the scFv component of CAR to discriminate tumors overexpressing the target from normal tissues that express it at physiologic levels. Our studies show that the use of affinity-tuned scFvs offers a strategy to empower wider use of CAR T-Cells against validated targets widely overexpressed on solid tumors, including those considered undruggable by this approach.

In addition to express CAR to re-direct T specificity to the tumors, the T-Cells can be further modified to avoid TEM suppression by co-introducing PD1-CD28 switch receptors or knocking out PD1 in the T-Cells using CRISPR. Furthermore,

using universal CAR T to treat cancer patients holds great promise. Clinical scale universal CAR T-Cell manufacture was developed by utilizing high efficient CRIPR technology. Pre-clinical animal study showed that the anti-tumor activity of the PCRISPR gene edited universal CAR T-Cells was as potent as the CAR T-Cells without CRISPR gene editing.

Yangbing Zhao, University of Pennsylvania, USA

15:30 Afternoon Networking Break: Coffee, Tea & Refreshments

16:00 Allogeneic Models in Adoptive T-Cell Therapy

Dr. Sicco Popma is currently Scientific Director and leader of Gene-Modified Cell Therapies at JBIO (Janssen R&D) leading the development of CAR T-Cell therapies. He was a founding member of a cell therapy venture within Janssen R&D in 2005 and led cell therapy related immunology research. He became Scientific Leader within the cell therapy product team and was responsible for preclinical and clinical immunology, biodistribution, biomarkers, and surgical drug delivery development. Prior to joining Janssen R&D in 2003, he was a member of the Clinical Cell and Vaccine Production Facility at the University of Pennsylvania developing and producing clinical cell therapies. In addition, Dr Popma has extensive experience in xenotransplantation and holds a degree in Medical Sciences and Microbiology.

Sicco Popma, Scientific Director and leader of Gene-Modified Cell Therapies at JBIO (JANSSEN R&D)

16:30 PANEL DISCUSSION: Pre-Clinical Trial Decision Making

What do we need in the pre-clinical setting? What background data is needed to appropriately move forward to a clinical trial? What are the triggers involved?

We explore the level of information and confidence required when going into clinical trials and what those implications are for business development.

Moderator: Robert Hawkins, CEO, CELLULAR THERAPEUTICS LTD.

Panelists:

Sicco Popma, Scientific Director and leader of Gene-Modified Cell Therapies at JBIO (JANSSEN R&D)

Andrew Gerry, Director of Preclinical Research, ADAPTIMMUNE

Yangbing Zhao, University of Pennsylvania, USA

CONFERENCE DAY ONE: WEDNESDAY, JUNE 29, 2016

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. 6

Page 7: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

08:00 Registration Opens

08:30 Chairperson’s Opening of Conference

09:00 Development of CAR T-Cells for Solid Tumors: adopting realistic pragmatism!

Dr. Ahmed is a physician scientist engaged in translational research focusing on adoptive immunotherapy with gene-modified effector cells, to improve therapy for brain tumors. Dr Ahmed’s initial studies demonstrated that antigen-specific cytotoxic T-Cells could eradicate established brain tumors in medulloblastoma and Glioblastoma models. Subsequent studies have demonstrated that the tumor-specific T-Cells, unlike conventional therapies, can effectively target the stem cell compartment in the tumor eradicating experimental tumors in animal models. Aligning with his primary interest is studying the role of T-Cells in creating a tumor niche and studying T-Cell migration to distant tumor sites in the brain. Dr. Ahmed therefore has experience in developing and translating cell and gene therapy studies to the clinic. Currently, Dr. Ahmed is the principal investigator on 3 clinical trials targeting Glioblastoma and Osteosarcoma by administering tumor-specific T-Cells to. All studies are FDA, IRB, RAC and IBC approved.

Nabil Ahmed, Baylor College of Medicine, Texas, USA

09:30 Tumor Immunology; Tumor Progression, Tumor-Specific T-cell Clones; Characterization of Tumor Variants; Molecular Genetics of Tumor Antigens1. Mechanism of tumor escape from host immunity.

Development of new strategies to prevent this escape by genetic engineering and immune manipulations.

2. Mechanisms leading to paracrine stimulation of tumor growth. Novel approaches of using tumor stroma as therapeutic target.

3. Use of tumor-specific mutant proteins or viral oncoproteins (E6/E7 of HPV) as target for immune prevention of primary cancer development.

4. Identification of the molecular basis and genetic origins of tumor-specific target antigens and target molecules that cannot be lost by cancer cells.

Hans Schreiber, University of Chicago, USA

10:00 The Fourth Generation of CAR T-Cells: TRUCKs

Adoptive therapy with engineered T-Cells with an antigen-specific chimeric antigen receptor (CAR) is achieving impressive efficacy in early phase trials, in particular in hematologic malignancies, strongly supporting the notion that the immune system can control cancer. Such CAR T-Cells can substantially reduce the tumor burden as long as the targeted antigen is present on the cancer cells, however, loss of target antigen make them invisible to CAR T-Cells and may contribute to deadly tumor relapses. We discuss the fourth generation of CAR T-Cells, so called TRUCKs, which release inducible IL-12 or any other cytokine upon CAR engagement in the targeted tumor lesion. Locally accumulating IL-12 in turn attracts an innate immune cell response towards those cancer cells that are invisible to CAR T-Cells.

Elimination of antigen-loss tumor cells was accompanied by the accumulation of activated macrophages through a TNF-alpha mediated process. The IL-12 supplement by CAR T-Cells has the advantage to target otherwise not accessible tumor lesions, to achieve therapeutic levels in a localized fashion, to reduce systemic toxicity and to recruit and activate innate immune cells. The strategy combines antigen-redirected immunotherapy with an antigen-independent anti-tumor response. We discuss major consequences for future strategies the immune therapy of malignant diseases and other applications of TRUCKs as targeted living factories.

Hinrich Abken, Genetics & Immunology, University of Cologne, GERMANY

10:30 Morning Networking Break: Coffee, Tea & Refreshments

11:30 PANEL DISCUSSION: Targets in Adoptive T-cell Therapy

Current targets for adoptive T-Cell therapies are limited and challenging. As a result, increased discovery efforts have been launched to continue advancing the field. In this discussion leading professors and clinicians will address the need to discover new, novel classes of targets expressed on the cell membrane and inside the cell, mainly with tumor-specific and mutated antigens.

What new targets are being considered for t-cell immunotherapies?

Moderator: Nabil Ahmed, Baylor College of Medicine, Texas, USA

Panelists:

Hans Schreiber, University of Chicago, USA

Hinrich Abken, Genetics & Immunology, University of Cologne, GERMANY

Arnaud Foussat, Vice President for Research & New Products, TXCELL SA

12:00 Assessing Regulatory T-Cells as Therapy

The use of T lymphocytes for treating diseases has been a much discussed topic of translational medicine for decades. The last five to 10 years have seen a combination of a considerable evolution of research and development tools, new promises of regenerative medicine that can be considered as breakthrough treatment modalities for the care of patients, and a strong will of regulatory bodies to structure a framework for cell based medicinal products. These combined elements have allowed the initiation of a new era in T-Cell based inununotherapy with a faster development of therapeutic approaches using T-Cells.

Most of the ‘F cell based products in development, are aimed at fighting inflammation and cancer through the use of effector T-Cells isolated from a patient’s blood. These autologous T-Cells are either educated or engineered using molecular biology tools to specifically recognize and kill a specific tumor or a cellular target. infected by a specific pathogen. decent clinical trials in oncology with effector.

T-Cells have shown compelling results that have led to top pharmaceutical companies, as well as worldwide investors, entering the field. Effector T-Cells represent one side of the immune system. On this side, T-Cell immunity can be viewed as an entity aimed at protecting the organism against pathogens such as virus, bacteria and against tumor development.

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. 7

Conference Day TwoNorth American

THURSDAY JUNE 30, 2016

Page 8: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

This property is the basis of the development of T-Cell based therapy in oncology and infectious diseases. However, a second side of T-Cell immunity co-exists and is directed at the establishment of tolerance. This immune mediated tolerance is part. of the need to control the response of the body to our environment as we are constantly surrounded by external antigens that the organism has to classify as non-harmful. a decrease in the frequency of allergen-specific Treg cells has been observed in allergic patients in multiple studies. Inversely, induction of remission in autoimmune patients or response to treatments such as anti-TNF alpha blocking agents, steroid compounds or other immunomodulatory molecules often come with the restoration of a normal Treg cell compartment.

Arnaud Foussat, Vice President for Research & New Products, of TXCELL SA

12:30 Networking Lunch

14:00 Bispecific antibodies for T cell redirection and dual checkpoint blockade

We have optimized a plug-and-play, Fc-containing bispecific antibody platform with high stability, efficient production, and antibody-like pharmacokinetics. This optimized bispecific format resembles a standard monoclonal antibody, with one of the Fab arms replaced by a stability-optimized single-chain Fv (scFv) (scFv-Fab-Fc). We will present application of the platform to create a pipeline of CD3 bispecifics for T cell redirection, and dual checkpoint blockade bispecifics for T cell activation.

John R. Desjarlais, Chief Scientific Officer at XENCOR, INC

14:30 Panel Discussion: Beyond CAR-T Therapy

Challenging Adoptive T-cell Therapy

• Are there interests in combining adoptive t-cell therapies with other types of targeted agents?

• Are CAR-T-Cell therapies struggling to prove concept?

• How do we provide a new generation of t-cell therapies that provide enhanced efficacy?

• The solid tumor, how are you going to the T-cells in, how are they going to find, target, persuade the tumor, check-point inhibitors blockers?

• Using tumor check-point inhibitors blockers to get the micro environment to go from cold to hot.

• Adaptive memory. Can you get the immune system of the patient to actually create its own memory?

• Creating a micro environment that becomes immunogenic and more receptive to T-cells and you then make those cells killer t-cells.

• Immunoescape, altering the tumor micro environment.

Hosted by Andrew Sewell, CARDIFF INSTITUTE OF INFECTION & IMMUNITY, UK,

Claudia Zylberberg, CEO at AKRON BIOTECH

John R. Desjarlais, Chief Scientific Officer at XENCOR, INC

15:00 Afternoon Networking Break: Coffee, Tea & Refreshments

15:30 Targeted Genome Modifications for improved CAR T-cell immunotherapy

Cellectis is a biopharmaceutical company with a single goal: to develop adoptive immunotherapies for cancer.

In order to address unmet medical needs, Cellectis is developing immunotherapies based on CAR T-cells. The specificity of those allogeneic therapies is that T-cells from healthy donors are genetically edited with our proprietary technology TALEN®, to seek and destroy cancer cells. This approach could lead to a drug that would be cost-effective, easily distributed across all geographies and available to patients who don’t have enough T-cells to undergo an autologous CAR-T therapy (based on the patient’s own T-cells).

Gene editing T-cells, which are cells from the immune system, can be engineered to express a Chimeric Antigen Receptor or CAR, a molecule that enables them to recognize specific antigens that are present on the surface of cancer cells. By targeting a protein expressed by tumoral cells, the CAR leads the immune system towards cancer, which is recognized as harmful. The immune system is then able to attack cancer cells

Julianne Smith, Group Leader at CELLECTIS THERAPEUTICS

16:00 Elements of Quality Assurance and Quality Control for Adoptive T-Cell Therapies: Points to Consider

Ensuring safety and efficacy of T-cell immunotherapies will largely depend upon robust systems of quality assurance and quality control. A 26 year experience in developing quality systems for the cell therapies including two product approvals will be summarized in this presentation. The advent of ISO based global quality standards through ICH agreements and guidance from FDA and international regulators have resulted in a robust quality framework. Moreover, the U.S. Pharmacopeia and other standard setting organizations have translated a quarter century of experiential observations and data into quality best practices. Taken together developers of T-cell therapies can optimize their development programs to lower regulatory hurdles and address the unmet needs of our patients.

Gary C. du Moulin, Ph. D, M.P.H., RAC , Associate Professor of Drug Regulatory Affairs, MASSACHUSETTS COLLEGE OF PHARMACY AND HEALTH SCIENCES UNIVERSITY, BOSTON, MA (Formerly V.P., Quality Systems, Genzyme Biosurgery)

17:00 Close of Conference

CONFERENCE DAY TWO: THURSDAY, JUNE 30, 2016

Register online at www.tcellcongressusa.com. Call us directly at +44 (0)20 3696 2920 or email [email protected]. 8

Far from the typical ‘meet-and-greet’ exhibition experience, you – as a sponsor or exhibitor – will be positioned as a partner of the event

with a focus on the benefits of your product and brand, rather than just a name on an exhibition list.

With our extensive marketing experience and strategy, your partnership with the conference will grant you a sponsorship package

that is an extension and enhancement of your current marketing and branding efforts.

Ask us about the different ways you can get involved. Contact Stephen Swarray to find out more about each package by emailing

at [email protected] or by calling +44 (0)20 3696 2920.

Interested in Brand Solution Opportunities?

Page 9: North American - Home | Kisaco Research · • Commercial deliverability: Scalability, manufacturing and commercialization. • Immunoescape and altering the tumor micro environment

North American

Venue & AccommodationSeaport Hotel & Seaport World Trade Center200 Seaport Blvd,Boston, MA 02210, United StatesT: +1 877-732-7678

Kisaco Research is pleased to announce that all attendees of the Adoptive T-Cell Therapy Congress USA will be able to access an exclusive group rate at the Seaport Hotel & Seaport World Trade Center . Rates start from $359 for a standard single room, excluding tax and breakfast.

Book by calling +1 877-732-7678, through their online booking system, or by emailing: [email protected]. More information is available online at www.4cityhotels.com/kisacoadoptivetcellboston.html.

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