Deliver the Next Generation of Safe, Effective - CAR

Transcription

In support of the
September 13th-16th, 2016
BOSTON, MA
Deliver the Next Generation of Safe, Effective
& Commercially Viable CAR-T and TCR Cell
Therapies to Market
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
Kristin Baird
Medical Officer, Division of Clinical
Evaluation, Pharmacology & Toxicology,
Office of Cellular, Tissue & Gene Therapies
CBER, FDA
Gwendolyn Binder-Scholl
CTO
Adaptimmune
Expertise Partners:
www.car-tcr-summit.com
Cell Immunotherapy
@CAR_TCell Tel: +1 212 537 5898 | Email: [email protected]
RESEARCHED &
DEVELOPED BY:
CAR-TCR Summit 2016 Boston, MA
September 13th - 16th, 2016
Get Ahead in the CAR-TCR Race
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 endto-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.
Navigate the CAR-TCR Development Timeline
1
Target ID:
6
Clinical Trial Development:
2
CAR Construct and Assembly:
7
Regulatory Compliance:
3
Pre Clinical Modeling:
8
Scalable Manufacture:
4
Translational Research:
9
Commercialization:
5
Safety and Efficacy Profiles:
Steven Feldman, NCI will share the NCI’s
CAR-T experience for the treatment of solid
cancers from pre-clinical development to
clinical outcomes
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
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
Sadik Kassim, Novartis discusses analytical
strategies to characterize CAR-T and
TCRs before administration to advance
towards precision
David Spencer, Bellicum Pharmaceuticals
will share strategies on how to control potency,
persistence, and toxicity of T cell therapies using
molecular switches
Tel: +1 212 537 5898
10
Improve the design of early phase trials to
maximize learning and deep mechanistic
understanding of CAR-T cells with Marcela
Maus, MGH Cancer Center
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
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
Edmund Pezalla, Aetna provides the payer
perspective and aims to answer the question:
How will these cellular immunotherapies
be reimbursed?
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
Email: [email protected]
@CAR_TCell
Cell Immunotherapy
Why You Should Attend the CAR-TCR Summit:
David Meek
President,
Oncology
Baxalta
“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.”
Tim Clay
Senior Director,
Cell & Gene
Therapy
Discovery
Research
GSK
“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!”
Laurent Poirot
Head, Early
Discovery
Cellectis
“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.”
Cheng Liu
CEO
Eureka
Therapeutics
“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.”
Rick Morgan
VP,
Immunotherapy
bluebird bio
“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.”
Tel: +1 212 537 5898
Jennifer Brogdon
Senior Investigator
Novartis
“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.”
@CAR_TCell
Cell Immunotherapy
David Lebwohl
SVP & Executive
Global Program
Head, CAR-T
Global Team, Cell
& Gene Therapies
Unit
Novartis
“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.”
Hans Klingemann
VP, R&D
NantKwest
“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.”
Peter Emtage
SVP Research
& Development
Cell Design Labs
“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.”
Speakers
Industry Drug Developers
Gwendolyn
Binder-Scholl
CTO
Adaptimmune
Peter Hoang
Martin Pule
David Meek
David Spencer
Founder & CSO
Autolus
President, Oncology
Baxalta
CSO
Bellicum
Pharmaceuticals
Peter Olagunju
Rick Morgan
Zonghai Li
VP, Immunotherapy
bluebird bio
President & CEO
CARsgen
Peter Emtage
Laurent Poirot
SVP, Business
Development & Strategy
Bellicum
Pharrmaceuticals
Senior Director, Vendor
Management
bluebird bio
Christopher Wiwi
Vladimir Jankovic
Director, Analytical R&D
Celgene Cellular
Therapeutics
Director, Preclinical
& Translational
Development
Celgene Cellular
Therapeutics
SVP Research &
Development
Cell Design Labs
Head, Early Discovery
Cellectis
Yihong Yao
Christian Homsy
Steve Buckanavage
CSO
CBMG
CEO
Celyad
VP, Marketing
Celyad
Michael Kalos,
CSO, Cancer
Immunobiology,
Eli Lilly
Cheng Liu
Axel Hoos
Senior Vice President,
TA Head Oncology R&D,
and Head, ImmunoOncology
GSK
Tim Clay
Sicco Popma
CEO
Eureka Therapeutics
Mark Frohlich
Arie Belldegrun
Margo Roberts
Dolores Schendel
EVP, Portfolio Strategy
Juno Therapeutics
CEO
CSO
CEO/CSO
Medigene
Hans Klingemann
David Lebwohl
Jennifer Brogdon
Rodney Rietze
VP, R&D
NantKwest
SVP & Executive Global
Program Head, CAR-T
Global Team, Cell &
Gene Therapies Unit
Novartis
Sadik Kassim
Barbra Sasu
Associate Director
Novartis
Senior Director
Rinat/Pfizer
Senior Director, Research
Projects Leader US,
Cell & Gene Therapy
Discovery Research
GSK
Senior Investigator
Novartis
Lead cGMP Process
Automation
Novartis
Rohit Duggal
Laurence Cooper
Director, Experimental
Therapy
Sorrento Therapeutics
David Kirn
CEO & Co-Founder
4D Molecular
Therapeutics
Tel: +1 212 537 5898
@CAR_TCell
Scientific Director,
Gene Modified Cell
Therapy Leader
Johnson & Johnson
Cell Immunotherapy
CEO
ZIOPHARM Oncology
Academics
Andras Heczy
Director, Liver Tumor
Program, Assistant
Professor
Baylor College of
Medicine
Navin Varadarajan
Jason Luke
Larry Corey
President & Director
Emeritus
Fred Hutchinson Cancer
Research Center
Krishnendu Roy
Assistant Professor,
Medicine
University of Chicago
Marcela Maus
Atsushi Natsume
Steven Feldman
Assistant Professor
University of Houston
Director, Cellular
Immunotherapy
Massachusetts General
Hospital Cancer Centre
Kellie Haworth
Bruce Levine
Pediatric Hematology/
Oncology Fellow
Nationwide Children’s
Hospital
Stephan Grupp
Director, Cancer
Immunotherapy Frontier
Program
Children’s Hospital of
Philadelphia
Director, Clinical Cell
& Vaccine Production
Facility, Perelman
School of Medicine
& Abramson Cancer
Center, University of
Pennsylvania
Director, Center for
Cell Manufacturing &
ImmunoEngineering
Georgia Institute of
Technology
Associate Professor
Nagoya University
Director, Surgery Branch
Vector Production
Facility
National Cancer
Institute
Edmund Moon
Jos Melenhorst
Assistant Professor
University of
Pennsylvania
Director, Product
Development &
Correlative Sciences
laboratory
University of
Pennsylvania
Isabelle Rivière
Director, Michael G. Harris Cell
Therapy & Cell Engineering Facility,
Center for Cell Engineering
Memorial Sloan Kettering
Cancer Center
Regulatory Bodies, Patient Advocacy & Healthcare Providers
Edmund Pezalla
VP, National Medical
Director, Pharmacy &
Policy Strategy
Aetna
Kristin Baird
Medical Officer, Division
of Clinical Evaluation,
Pharmacology &
Toxicology, Office of
Cellular, Tissue & Gene
Therapies
CBER, FDA
Paula Salmikangas
Chair, Commitee for
Advanced Therapies,
EMA
Doug Olson
Patient Advocate
The Leukaemia &
Lymphoma Society
Solution and Service Providers
Yama Abassi
Claudia Zylberberg
Jan Nielson
Robert Margolin
Mark Sawicki
Klaus Kühlcke
John Rozembersky
Tom Duensing
VP
ACEA Biosciences
CCO
Cryoport
CEO
EUFETS
Fred Koller
Ryan Cawood
VP, Business
Development
Miltenyi Biotec
Tel: +1 212 537 5898
CEO
Akron Biotech
CEO
Oxford Genetics
Vice President
Sonexus™ Access
& Patient Support
Cardinal Health
VP, BioPharm & Life
Science Applications
FloDesign Sonics
Martyn Lewis
CTO
ProMab
Biotechnologies
@CAR_TCell
Cell Immunotherapy
VP Corporate
Development
Cognate BioServices
CTO
Intellicyt
Bruce McCreedy
SVP, Cell Therapy
Precision Biosciences
Agenda at a Glance
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
Target Identification & Validation
Clinical Development Stream
Robust Preclinical Research
Manufacturing Stream
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
Innovative Methods to Overcoming Safety
& Toxicity Challenges
Clinical Development Stream
Combination Therapies & Enhancing the
Efficacy of Frontline Cancer Therapies
Manufacturing Stream
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
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
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
Conference Day 1: Wednesday September 14th 2016
8.00
Breakfast & Registration
9.10
The Race is On: CAR-Ts Where are We Now?
9.40
Engineered T Cells for Hematologic Malignancies and Solid
Tumors: The Juno Program
Arie Belldegrun, CEO,
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
Mark Frohlich, EVP,
Portfolio Strategy, Juno
Therapeutics
•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
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
Christian Homsy, CEO,
Celyad
•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
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?
11.10
Networking & Refreshments
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
David Lebowhl, SVP &
Executive Global Program
Head, CAR-T Global Team,
Cell & Gene Therapies
Unit, Novartis
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
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
Jennifer Brogdon, Senior Investigator,
Novartis
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
12.40 Advancing the Discovery
of Immunotherapeutics with
Large Scale, Multiplexed
Experiments on Cells and
Proteins of Immune System
12.40 Key Considerations when
Evaluating CMOs for Late
Stage Clinical and Commercial
Production of Cell-Based
Immunotherapies
• Evaluate new tools which are needed
to evaluate the effects of therapies on
cells and proteins in suspension
• Learn to keep cost of goods low so
your CAR-T can soar high: Small
changes, incredible impact?
• The iQue® Screener is the first
platform capable of performing
large scale, multiplexed experiments
on cells and proteins of the
immune system
• The critical attributes needed within
a CMO to support the rapid clinical
development and commercialization
of these platform cell-based
immunotherapies
• Discuss how this system is being
employed to discover new antibodies,
and to understand functional efficacy
of immuno-oncology therapies
• Navigate the main points where
cost can and should be controlled,
considering safety and compliance
Tom Duensing, CTO, Intellicyt
Bruce McCreedy, SVP of Cell Therapy,
Precision Biosciences
• 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
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
1.10 The ABC’s of Adoptive T
Cell Therapy Using OptimalAffinity Natural TCRs Specific for
Hematological Malignancies and
Solid Tumors
It 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 Cells
Using 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,
1.40
Lunch & Networking
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
1.10 Large-Scale, Reproducible
Biomanufacturing of HighQuality 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 closedsystem, 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
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
2.40 CAR T Cells Go Back
to School
2.40 Challenges in CAR-T
Cell Manufacturing
• CAR T cells were initially developed
and translated to clinical trials in the
academic setting but have now moved
toward an industry setting
• 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
• 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
• 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
• 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
• 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
3.10 PANEL: Innovations
in Enhancing Efficacy and
Improving Safety Profiles
3.10 PANEL: Developing and
Translating Clinical Trials from
Bench to Bedside
Tim Clay, Senior Director, Research
Projects Leader US, Cell & Gene
Therapy Discovery Research,
GSK
Sadik Kassim, Associate Director,
Novartis
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
4.10
Navin Varadarajan, Assistant Professor,
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
Tel: +1 212 537 5898
• 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
@CAR_TCell
Cell Immunotherapy
3.10 PANEL: From
Manufacturing to Market Access:
Getting Cellular Therapies to
Patients in Need
Steven 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
Viral Vector Transfection &
Genetic Engineering
Demonstrating Efficacy in Solid
Tumor Indications
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
4.40 Next-Generation
Investigative CAR-T Platforms
Towards the Goal of
Personalized Immunotherapy
4.40 Critical Assessment of
the Impact of Packaging and
Logistics Decisions on the CAR-T
Clinical Pipeline
• 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
• 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
• 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
• Our proprietary strategy of coexpressing genes that alleviate
toxicities has allowed us to express
helper proteins constitutively at high
levels, overcoming one of the major
limitations
• 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
• 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
Martyn Lewis, CTO,
ProMab Biotechnologies
5.10 TALEN® Based Targeted
Genome Modifications for
Improved CAR-T Cell Adoptive
Immunotherapy
5.10 Development of
Cancer-Specific anti-EGFR
CAR-T cells for the Treatment of
Patients with Glioblastoma
• 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
• 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 Y022engineered CAR-T cells could
specifically recognize and eliminate
cancer cells with EGFR amplification
but not primary keratinocytes
• 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
Tel: +1 212 537 5898
• 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
Transportation, Supply
Chain & Logistics
@CAR_TCell
Cell Immunotherapy
• 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
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
5.40 TCR-Like CARs Targeting
Intracellular Cancer-Specific
Antigens for Treatment of
Solid Tumors
• 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
• 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
• 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 selfrenewal
• 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
• 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 CD19targeting 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
• 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
Cheng Liu, CEO, Eureka Therapeutics
Laurence Cooper, CEO, ZIOPHARM
Oncology
6.10
Chairman’s Closing Remarks
6.15
Annual CAR-TCR Party Hosted by Juno Therapeutics
7.15
Close of Day 1
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
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
Conference Day 2: Thursday September 15th 2016
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
Gwendolyn Binder-Scholl,
CTO, Adaptimmune
•Translational approaches for investigating and addressing these
challenges in the context of TCR engineered T cells for solid tumor therapy,
will be discussed
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 tumormediated MHC down regulation, preventing TCR-based therapies from
detecting cancer associated antigens
Aaron Foster,
Senior Director, Product
Discovery, Bellicum
Pharmaceuticals
•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
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.
10.40
Margo Roberts, CSO, Kite
Pharmaceuticals
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
11.10
Networking & Refreshments
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
Fred Koller,
Vice President,
Business Development,
Miltenyi Biotec
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
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 genemodified cells, following a single
rimiducid dose while continuing
some therapy, due to residual, nonalloreactive 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 MCenhanced 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
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
David Spencer, CSO, Bellicum
Pharmaceuticals
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
11.40 Clinical Considerations
for CAR-T Cell Therapy: A
Regulatory Perspective
Adoptive 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
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
12.10 EMA Regulatory
Perspective: Evolving the
Regulatory Conversation to
Match Scientific Innovation
• We conducted a Phase I study with
adaptive trial design of T cells
expressing a GD2 CAR with both CD28
and OX40 costimulatory endodomains
• Will these therapies be accelerated
through development with
breakthrough designation given
resounding early results?
• We tested the safety, persistence and
antitumor efficacy of the CAR T in
11 patients with neuroblastoma, in 3
cohorts
• Understanding requirements for
validating and translating preclinical
research on novel targets
• 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 coadministration of PD1 Ab
• What are the European regulatory
considerations?
• What standards need to be in
place for therapy development and
manufacturing?
Paula Salmikangas, Chair, Committee
for Advanced Therapies, EMA
Andras Heczy, Director, Liver Tumor
Program, Assistant Professor, Baylor
College of Medicine
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
12.40 Pfizer Allogeneic CAR-T
• Heme targeting strategy
• Selection of CAR characteristics
Barbra Sasu, Senior Director,
Rinat/Pfizer
• 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
• The xCELLigence assay will be shown
to provide a simple and efficient
workflow
• Allow sufficient time to get your
product launched
• W
e
will demonstrate how
xCELLigence is being used to study
the fine-tuning of CAR-T cell
killing activity using immune
modulating agents
• 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
Yama Abassi, VP, Acea Biosciences
Lunch & Networking
Tel: +1 212 537 5898
• 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
• We will present data demonstrating
that kinetic assessment of CAR-T cell
potency using xCELLigence provides
superior sensitivity and dramatically
expanded assay
1.40
12.40 Your CAR-T Therapy has
the Green Light? How Strong is
Your Market Access Strategy?
@CAR_TCell
Cell Immunotherapy
The Future of CAR-T
Development: Emerging
Innovations & Technologies
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
Unconventional CAR Approaches:
Alternative CAR vehicles &
Looking Beyond Cancer
2.10 Novel CARs Introduced
into NK Cells Facilitate Potent
Tumor-Cell Killing that Results
in Tumor Regression
Rohit Duggal, Director,
ExperimentalTherapy, Sorrento
Therapeutics
• This technology is now in global
multi-center clinical trials
• 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
2.40 Further Development Of
CAR-T Cells for the Treatment
of Cancer
2.40 Target Engineered Natural
Killer Cells as “Off-the-Shelf”
Cellular Therapeutics
• One anti-BCMA CAR (bb2121)
candidate was selected based on
strong surface expression, superior
biological activity to multiple
BCMA+ cell lines, and low antigenindependent reactivity. Robust
recognition of as little as 220 BCMA
molecules/cell permitted reactivity to
B cell tumors including primary CLL
• NantKwest has developed the NK
cell line NK-92 as an “off-the-shelf”
activated natural killer (aNK) cell
platform
Tel: +1 212 537 5898
• 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
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.
• Calculation of value, cost offset and
budget impact?
Bruce Levine, Director, Clinical
Cell & Vaccine Production Facility,
Perelman School of Medicine &
Abramson Cancer Center, University
of Pennsylvania
• 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.
2.10 How Will New Cancer Cell
Therapies be Reimbursed?
• What this means for development and
launch of oncology products?
• 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
• Based on this and other data, bluebird
bio, in collaboration with Celgene, has
initiated a phase I clinical trial of antiBCMA CAR T cells in patients with
multiple myeloma
Pricing, Partnering & Novel
Business Models
@CAR_TCell
Cell Immunotherapy
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 immunooncology (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 immunooncology 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
• 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 cellmediated cytotoxicity
• bb2121 CAR T cells cultured with
PI3K inhibition expressed markers
associated with T cell memory such
as; CD62L, CD127, and CD197
• 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)
• 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
Hans Klingemann, VP, R&D, NantKwest
• Only mice treated with bb2121 CAR
T cells cultured with PI3K inhibition
were able to control the tumor
re-challenge
• 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 immunooncology is more than any one
company can manage
• 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
• Partnerships and licensing
arrangements give us maximum
flexibility and a much higher likelihood
of success. Baxalta’s immuneoncology portfolio reflects a range
of approaches, derived from internal
innovation as well as synergizing with
external innovation
Rick Morgan, VP Immunotherapy,
bluebird bio
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
4.40
David Meek, President,
Oncology, Baxalta
Axel Hoos, Senior
Vice President, TA
Head Oncology R&D
and Head, ImmunoOncology, GSK
A Patient’s Experience with the Initial CART 19 Trial and Perspectives on
Patient Access to New Breakthroughs for
Cancer Treatment
Doug 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.
5.10
Chairman’s Closing Remarks
5.15
Close of 2nd CAR-TCR Summit 2016
Tel: +1 212 537 5898
Frederic Lehmann,
VP, Head, Oncology
Franchise, Celyad
@CAR_TCell
Doug Olson, Patient
Advocate, The
Leukemia and
Lymphoma Society
Cell Immunotherapy
Pre-Conference Workshops: Tuesday September 13th 2016
A
Autologous vs. Allogeneic:
Which Will Succeed in the Race to Market?
Time: 9.00am - 12.00pm
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
Sicco Popma
Scientific Director, Gene
Modified Cell Therapy Leader
Johnson & Johnson
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.
• 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.
- OR -
B
Overcoming the Inhibitory Tumor Microenvironment
Time: 9.00am - 12.00pm
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.
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
Jason Luke
Assistant Professor, Medicine
University of Chicago
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.
Pre-Conference Workshops: Tuesday September 13th 2016
C
Solid Tumor Target
Identification and Validation
Time: 1.00pm - 4.00pm
Edmund Moon
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.
- OR -
D
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.
The Transformative Power of Correlative Sciences
in Adoptive Immuno-Gene Therapies
Time: 1.00pm - 4.00pm
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.
Tel: +1 212 537 5898
Assistant Professor
University of Pennsylvania
@CAR_TCell
Cell Immunotherapy
Jos Melenhorst
Director, Product Development &
Correlative Sciences laboratory
University 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.
Post-Conference Workshops: Friday September 16th 2016
E
CAR-T Cell Therapy:
Regulatory Challenges and Opportunities
Time: 9.00am - 12.00pm
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
Paula Salmikangas
Chair, Committee for Advanced
Therapies
EMA
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.
• 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.
Kristin Baird
Medical Officer, Division of Clinical
Evaluation, Pharmacology and
Toxicology, Office of Cellular,
Tissue and Gene Therapies
CBER, 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.
- OR -
F
Automatizing Single Use Systems and
Novel Production Paradigms for Effective Manufacturing
Time: 9.00am - 12.00pm
Rodney Rietze
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.
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
Lead cGMP Process Automation
Novartis
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.
Post-Conference Workshops: Friday September 16th 2016
G
Pricing and Reimbursement
Strategies for Engineered Cell Therapies
Time: 1.00pm - 4.00pm
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.
Edmund Pezalla
VP, National Medical Director,
Pharmaceutical Policy & Strategy
Aetna
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.
- OR -
H
Current Trends and Best Practices in
Outsourcing for Long Term Cell Immunotherapy Success
Time: 1.00pm - 4.00pm
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.
Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy
Peter Olagunju
Senior Director, Vendor
Management
bluebird bio
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.
Sponsors
Expertise Partners
Celyad
With 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 Biotec
Miltenyi 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/
Program Partners
Panel Partner
Hosting Partner
Exhibitors
For Sponsorship Opportunities Contact:
Jonathan Kilby-Philips
Commercial Manager
Tel: +1 212 537 5898
Tel: +44 (0)203 141 8700
@CAR_TCell
Cell Immunotherapy
Pricing & Venue
Solution/Service Provider Pricing
Register & Pay before
July 15th 2016
Standard Prices
Conference + 4 Workshops
$7695 (save $1000)
$7895 (save $800)
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$5897 (save $400)
Conference + 1 Workshop
$4698 (save $400)
$4898 (save $200)
Conference Only
$3699 (save $200)
$3899
Workshops (each)
$1199
Pharma & Biotech Drug Developer Pricing
Register & Pay before
July 15th 2016
Standard Prices
$5595 (save $1000)
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Conference Only
$2799 (save $200)
$2999
Workshops (each)
$899
Academics are entitled to 40% off the Pharma & Biotech prices.
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
Register
Team Discounts
Venue
www.car-tcr-summit.com/register
Tel: +44 (0)203 141 8700
• 10% discount – 3 delegates
• 15% discount – 4 delegates
• 20% discount – 5 or more
delegates
Hyatt Regency Boston
One Avenue de Lafayette
Boston, Massachusetts,
USA, 02111
Mail:
Hanson Wade
4th Floor, 52 Grosvenor Gardens,
London, SW1W 0AU
Please note that discounts are only
valid when three or more delegates
from one company book and pay at the
same time. Team discounts cannot be
applied to academic pricing.
www.regencyboston.hyatt.com
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Changes to Conference & Agenda: Hanson Wade reserves the right to postpone or cancel an event, to change the location or alter the advertised speakers. Hanson Wade is not responsible for any loss or damage or costs incurred as a result of substitution, alteration,
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Tel: +1 212 537 5898
@CAR_TCell
Cell Immunotherapy

Deliver the Next Generation of Safe, Effective - CAR

Transcription

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