Phacilitate Cell & Gene Therapy Main Announcement

FORUM 2015
26-28 January, Grand Hyatt Washington, DC
Co-located with
11th Annual
FORUM 2015
26-28 January 2015, Grand Hyatt Washington, DC
Learn from – and meet – an unparalleled assembly of the global Cell & Gene
Therapy community’s premier thought-leaders and decision-makers, including:
www.bioleaders-forum.com
Book by 14Nov for $200discount!
…see inside for the full
panel and agenda!
Perry Karsen,
Chief Executive
Officer,
Celgene Cellular
Therapies
Dr Kenneth LeClair,
Executive Director,
Novartis Cell &
Gene Therapy Unit
Dr James Trager,
Vice President,
Research & Product
Development,
Dendreon
Dr Mahendra Rao,
Consultant,
NYSCF & Vice
President Strategic
Affairs,
Q Therapeutics
Jeb Keiper,
Vice President,
Oncology Business
Development,
GlaxoSmithKline
Worldwide Business
Development
Albert Seymour,
Global Head of
Research and
Nonclinical
Development,
Shire
Pharmaceuticals
Dr C. Randal Mills.
President & Chief
Executive Officer,
California Institute
for Regenerative
Medicine (CIRM)
Dr Keith Wonnacott,
Chief of Cellular
Therapies Unit,
OCTGT, CBER,
US Food and Drug
Administration
Dr Devyn Smith,
Head of Strategy,
Pharmatherapeutics
Research &
Development,
Pfizer
John McDonald,
Vice President,
Business
Development,
Biogen Idec
Dr Hansjoerg Duerr,
Global Head
Haematology,
Bayer Healthcare
Gold Sponsors: Silver Sponsors:
Bronze Sponsors: Roundtable Sponsor: Main Endorsing organisations:
The world’s biggest
and fastest growing
cell & gene therapy
business meeting
The first and still the
leading event specifically
driving product
commercialization
Unparalleled learning and
networking opportunities
with the decision-makers
who matter
14886 C & GT brochure V2_Layout 1 15/10/2014 12:44 Page 1

The world’s biggest and
fastest growing cell & gene
therapy business meeting
n Overall attendance expected
building on last year’s
record – 550+
n Biggest ever panel –
150+ speakers
n Profile of attendees – 76% at
Director level and above
The first and still the leading
event specifically driving
product commercialization
n Unmatched big pharma
participation – the only event that
all of the major pharma and big
biotech players attend
n Investors, regulators, KOLs and
other key stakeholders
n A who’s who of the world’s leading
Cell & Gene Therapy biotechs
Number 1 for thought
leadership, lead generation
and brand promotion
n Expanded agenda, new
interactive conference formats
(roundtables, drop in clinics, tech
room demonstrations, etc)
n New Biotech Showcase
n New partnering tool plus provision
of 1-2-1 meeting area within the
expo
Key organizations already confirmed for the Cell & Gene Therapy Forum 2015:
The single best cell and gene
therapy conference
David Ichikawa, Vice President, Business Development,
Sangamo BioSciences, Inc
FORUM 2015
Amgen, AstraZeneca/MedImmune, Bayer
HealthCare, Biogen Idec, Bristol-Myers
Squibb, Celgene, EMD Serono,
GlaxoSmithKline, Johnson &
Johnson/Janssen R&D, Merck, Novartis,
Pfizer, Roche/Genentech, Sanofi, Shire
California Institute for Regenerative
Medicine (CIRM), Centre for
Commercialisation of Regenerative
Medicine (CCRM), City of Hope, EU
Committee for Advanced Therapeutics
(CAT), Harvard Stem Cell Institute, Landmark
Capital, Leerink Partners, Memorial Sloan
Kettering Cancer Centre, NIH, Oregon
Health Science University Hospital, Paul-
Ehrlich Institute, The University of Texas MD
Anderson Cancer Center, University of
Pennsylvania, US FDA
Adaptimmune, Argos Therapeutics, Asterias
Biotherapeutics, Athersys, bluebird bio,
Capricor, Cell Medica, Celladon
Corporation, CRISPR Therapeutics,
Dendreon, Mesoblast, Northwest
Biotherapeutics, Organogenesis, Oxford
Biomedica, Pluristem Therapeutics,
Promethera Biosciences, Q Therapeutics,
Replicel, Sangamo BioSciences, Tigenix,
Tocagen, TxCell, UniQure
Why Cell & Gene Therapy Forum 2015 is the only event
you absolutely must attend next year:
Brand new, highly sophisticated
and effective 1-2-1 Partnering Tool
Investors, regulators, KOLs:Pharma/Big Biotech: Leading biotechs:
2015 sees the launch of our brand new app – meaning that
you can start benefiting from your attendance at the
Phacilitate Cell & Gene Therapy Forum before the event
even begins!
n Book meetings with fellow attendees
n Find new contacts based on your profile and interests
n Build your conference agenda
n Download exclusive content
n Build your own library to access content easily before, during
and after the event
n Take part in industry polls
14886 C & GT brochure_Layout 1 13/10/2014 13:28 Page 2

The Phacilitate meeting is one of those rare meetings where participants are very open in their
communication and at the same time have their focus on business. It is rare in that over 80% of
conversations were relevant to our business objectives. Definitely coming back in 2015!
Kurt Gielen, Acquisition & Business Development Manager, Chemelot Campus B.V.
The speaker panel for the Phacilitate Cell & Gene Therapy Forum 2015 is unparalleled in terms of its seniority and influence,
delivering all the strategic insight and contacts you need to drive your own business model forward.
We will continue to add speakers to the panel over the coming weeks. You can stay abreast of the latest developments at
any point by visiting the event website http://www.bioleaders-forum.com
Perry Karsen,
Chief Executive Officer,
Celgene Cellular Therapies
Dr Kenneth LeClair,
Executive Director,
Novartis Cell & Gene Therapy Unit
Dr James Trager,
Vice President, Research & Product
Development,
Dendreon
Dr C. Randal Mills.,
President & Chief Executive Officer,
Medicine (CIRM)
Jan Thirkettle,
Head of Advanced Therapy
Delivery,
GlaxoSmithKline
Dr Mahendra Rao,
Consultant, NYSCF & Vice President
Strategic Affairs,
Q Therapeutics
Dr Keith Wonnacott,
Chief of Cellular Therapies Unit,
Office of Cellular Tissue and Gene
Therapies (OCTGT),
US Food and Drug Administration
(FDA)
Jeb Keiper,
Vice President, Oncology Business
Development,
GlaxoSmithKline Worldwide
Business Development
Albert Seymour,
Global Head of Research and
Nonclinical Development,
Shire Pharmaceuticals
Dr Martina Schusler-Lenz,
Vice-Chair, Committee for
Advanced Therapies (CAT) EMA &
Clinical Assessor, Division of
Medical Biotechnology, Paul-
Ehrlich Institute
Dr Devyn Smith,
Head of Strategy,
Pharmatherapeutics Research &
Development,
Pfizer
John McDonald,
Vice President, Business
Development,
Biogen Idec
Dr Jim Faulkner,
Vice President, CMC & Supply,
GlaxoSmithKline Rare Diseases
Dr Knut Niss,
Senior Technical Project Leader,
Novartis
Dr Hansjoerg Duerr,
Global Head Haematology,
Bayer Healthcare
Dr Jane Lebkowski,
Vice President Research &
Development,
Asterias Biotherapeutics
James Noble,
Adaptimmune
Dr Donna Skerrett,
Chief Medical Officer,
Mesoblast
Edward Lanphier,
President & Chief Executive Officer,
Sangamo BioSciences Inc
Dr Greg Russotti,
Vice President Technical
Operations,
Celgene Cellular Therapeutics
Dr John Harrington,
Executive Vice President & Chief
Scientific Officer,
Athersys Inc
Frederick Miesowicz, PhD,
Chief Operating Officer & Vice
President Manufacturing,
Argos Therapeutics Inc
Mitchell H.Finer,
Chief Scientific Officer,
Bluebird Bio
Dr Sven Kili,
Senior Director, Global Medical
Affairs, Biosurgery,
Sanofi Biosurgery
Boris Peaker,
Managing Director, Biotechnology
Stocks,
Cowen and Company
Howard Liang, PhD,
Managing Director Biotechnology
Equity Research,
Leerink Partners LLC
Dr Hans Preusting,
Chief Business Officer,
UniQure
Dr Nessan Bermingham,
Venture Partner, Atlas Ventures,
Founder and Chief Executive
Officer,
Intellia Therapeutics
Prof John Rasko,
Director, Department of Cell and
Molecular Therapies,
Royal Prince Albert Hospital
Dr Richard Maziarz,
Researcher,
Oregon Health Science University
Hospital
Neil Palmer
President and Principal Consultant,
PDCI Market Access
Brock Reeve,
Executive Director,
Harvard Stem Cell Institute
Dr Ian Harris,
Senior Director, Cell Therapy,
Janssen Research & Development
LLC
Dr James Wilson,
Professor of Internal Medicine &
Pathology and Laboratory
Medicine, Director of the Gene
Program, University of
Pennsylvania, Research Director,
Center for Orphan Disease
Research and Therapy
Katherine Tsokas, JD,
Senior Director, Global Regulatory
Affairs, Janssen Research &
Development,
LLC
Dr Samuel Barone,
Avalanche Biotechnologies
Dr Wilfred Dalemans,
Chief Technical Officer,
Tigenix
Dr Theresa Chen,
Pharmacology/Toxicology
Reviewer, Office of Cellular Tissue
and Gene Therapies (OCTGT),
Center for Biologics Evaluation and
Research (CBER),
(FDA)
Dr Denise Gavin,
Team Lead, Gene Therapy Branch,
Office of Cellular Tissue and Gene
Therapies (OCTGT),
(FDA)
Dr Isabelle Riviére,
Director Cell Therapy & Cell
Engineering Facility,
Memorial Sloan Kettering Cancer
Centre
Donald Powers,
Principal Scientist,
Janssen Cell Therapy
Dr Anton Simeonov,
Acting Deputy Scientific Director,
National Centre for Advancing
Translational Sciences (NCATS)
Neil Littman,
Business Development Officer,
Medicine (CIRM)
Dr Ricardo Macarron,
Vice President of Target and
Pathway Validation,
GlaxoSmithKline
Dr Huseyin Mehmet,
Vice President, Head of Cell &
Molecular Biology,
Proteostasis Therapeutics Inc
Colonel Daniel Wattendorf,
Program Manager, Defence
Science Office,
DARPA
Dr Carl G. Simon, Jr,
Biologist and Project Leader of 3D
Tissue Scaffolds,
National Institute of Standards and
Technology (NIST)
Dr Michele Cleary,
Executive Director, Target and
Pathway Biology,
Merck
Dr Artur Isaev,
Human Stem Cells Institute
Professor Asterios Tsiftsoglou,
AUTH Lab of Pharmacology,
School of Pharmaceutical Sciences
& EU CAT Member
Dr Eileen Dolan,
Professor of Medicine, Chair,
Committee on Clinical
Pharmacology and
Pharmacogenomics, University of
Chicago, Associate Director for
Education,
Comprehensive Cancer Center
Carter Cliff,
Business Development,
Cellular Dynamics International Inc
Nick Timmons,
Director, Product and Process
Development,
Centre for Commercialisation of
Regenerative Medicine
Dr Traci Heath Mondoro,
Chief, Translational Blood Sciences
and Resources,
NHLBI
David Hall,
Replicel
Dr Christopher Bravery,
Principal Consultant,
Advbiols
Dr Larry Couture,
Senior Vice President and Founding
Director, Centre for Applied
Technology Development,
Beckman Research Institute of the
City of Hope
Dr Thomas Vogt,
Vice President Discovery & Systems
Biology,
CHDI Management/ CHDI
Foundation
Dr Kim Warren,
Head of Custom Development
Services for Cell Therapy,
Lonza Walkersville Inc
Richard Grant,
Director of Cell Therapy,
Invetech Pty Ltd
Dr Rodger Novak,
CRISPR Therapeutics
Dr John Maslowski,
Vice President Scientific Affairs,
Fibrocell
Dave Backer,
Senior Director & General
Manager,
SAFC Carlsbad
Faraz Ali,
Vice President, Commercial
Planning & External Affairs,
Bluebird Bio
William Podd,
Founder, President & Chief
Executive Officer,
Landmark Capital/Landmark
Angels
Dr Ohad Karnielli,
Vice President, Technology &
Manufacturing,
Pluristem Therapeutics Inc
Eric Halioua,
Co-Founder & Chief Executive
Officer,
Promethera Biosciences
Dr Andrew Hamer,
Vice President, Medical Affairs,
Capricor
Dr Joyce Frey-Vasconcells,
Regulatory Expert,
Frey-Vasconcells Consulting,
LLC
Peter Nolan,
Senior Vice President, Commercial
Development,
Oxford Biomedica
Dr Geoffrey Nichol,
Executive Vice President, Research
& Development,
Sangamo BioSciences
Dr Zorina Pitkin,
Vice President, Quality Systems,
Organogenesis
Dr Aby Mathew,
Senior Vice President & Scientific
Officer,
Biolife Solutions Inc
Dr Manish Singh,
Chairman & CEO,
Lion Biotechnologies, Inc
Lee Buckler,
Managing Director,
Cell Therapy Group, a Division of
CTG Consulting, Co
Professor Miguel Forte,
Senior Vice President, Clinical &
Regulatory,
TxCell
Dr Helen Tayton-Martin,
COO,
Adaptimmune
Simon Ellison,
Senior Business Development
Manager,
Cell Therapy Catapult
Dr Kai Pinkernell,
Global Head of Clinical Business,
Miltenyi Biotec GmbH
Dr Gabor Rubanyi,
Taxus Cardium Pharmaceuticals
Group
Anne-Virginie Eggimann,
Vice President, Regulatory Science,
bluebird bio, Inc
Ed Field,
Treasurer,
ARM
Dr Barbara Thorne,
Senior Director, Process
Development,
Celladon Corporation
Dr Philip D. Gregory,
Senior Vice President, Research &
Sangamo BioSciences
Dr Douglas Jolly,
Co-Founder & Executive Vice
President Research &
Pharmaceutical Development,
Tocagen
Kurt Gielen,
Senior Business Development
Manager, BioMedical Materials,
Brightlands Chemelot Campus
Dr Stefanos Theoharis,
Apceth GmbH & Co. KG
Dr Robert W. Mays,
Head of Neuroscience,
Athersys, Inc
Dr Eytan Abraham,
Head of Cell Therapy Research &
Technology,
Lonza Walkersville Inc
Margarita Gutova, MD,
Associate Research Professor,
Department of Neurosciences,
City of Hope National Medical
Center & Beckman Research
Institute
Who’s speaking?

DAY 1 - Monday, January 26th 2015
Followed by your choice of 4 highly interactive breakout sessions:
Focus session 1 Focus session 2 Focus session 3 Focus session 4
9.00 Chair’s introduction
Dr John Maslowski, Vice President Scientific Affairs, Fibrocell
9.05 Top 10 events in cell & gene therapy of 2014 - celebrating recent progress in the field
whilst putting it into context
• Revealing the results of the 2014 ‘top 10 events ‘ survey: A countdown of the year’s
most significant events in cell therapy/regenerative medicine and in gene therapy,
as nominated and voted for by the global C&GT community
• 2013 top 10 revisited – how have they advanced in 2014?
Ed Field, Treasurer, ARM
Lee Buckler, Managing Director, Cell Therapy Group, a Division of CTG
Consulting, Co
9.20 Delivering a snapshot of recently approved C&GT products and progress
with Phase III clinical candidates on a global basis
Lee Buckler, Managing Director, Cell Therapy Group, a Division of CTG Consulting, Co
9.30 Reviewing FDA Breakthrough Therapy Designations awarded to cell or
gene therapy products in 2014
Dr Joyce Frey-Vasconcells, Regulatory Expert, Frey-Vasconcells Consulting, LLC
9.40 What are the next steps for the cell & gene therapy world’s hottest
technology areas and indications?
• The ongoing emergence of CAR-T cells and cellular immunotherapy in general
• Where next for the adult stem cell therapy field? What lies beyond MSCs?
• Gene editing – the future of gene therapy?
• Pluripotent stem cell based therapeutics: next steps for clinical development and
commercialization
Dr Philip D Gregory, Senior Vice President, Research & Chief Scientific Officer,
Sangamo BioSciences
Dr Jane Lebkowski, Vice President Research & Development, Asterias
Biotherapeutics
Dr Kenneth LeClair, Executive Director, Novartis Cell & Gene Therapy Unit
10.10 Thought leader roundtable: What is holding cell & gene therapy back
(if anything)?
• Identifying, ranking and removing remaining major bottlenecks and obstacles to
progress (Money? Lack of clinical success? Scale-up and Cost of Goods issues?
Potency after stability? Regulatory disharmony? Other?)
10.35 Your choice of highly interactive optional sessions,
each for limited numbers of participants
Workshop (for a maximum of 100 participants)
Manufacturing and process development: Regulatory experts at hand
• During this interactive session, a panel from regulatory agencies and industry will
be addressing the regulatory questions you are facing in your process
development and
• GMP production.
• Access to expert advice
• One-to-one time with agency and industry experts
• Discuss your specific topic of interest
Jiwen Zhang, PhD, Regulatory Affairs Director, GE Healthcare
OR
Roundtable discussion
(Very informal discussion-based session for maximum of 12
participants)
Global expansion into other regions. Top 5 challenges and how to
address them
Kurt Gielen, Senior Business Development Manager, BioMedical Materials,
Brightlands Chemelot Campus
FURTHER SESSIONS WILL BE ADDED OVER THE COMING WEEKS – CHECK
BACK SOON FOR THE LATEST NEWS!
11.30 Morning coffee in the Exhibition/1-2-1 Meeting Area
The Top 10 ‘events’ can relate to anything - a financing round or a clinical trial; a
particular deal or a scientific breakthrough. The only criterion is that the ‘event’ has
had (or will have) a significant impact on the cell & gene therapy sector.
YOU will get the chance to nominate your most significant events of 2014, and to then
vote to help decide the final order of the Top 10. Look out for notification when the
initial nominations survey goes live online towards the end of the year.
Summarizing key trends affecting the C&GT industry over the
past 12 months - lessons learned in the past year
Morning plenary
Cell & Gene Therapy ‘State of the Union’ - analyzing 2014’s big stories;
setting strategic priorities for 2015 and beyond
7.30 Registration & buffet breakfast in the Exhibition/1-2-1 Meeting Area
OR
7.45 An Interactive Workshop
Marken and Global Care Clinical Trials: Bringing the Clinical
Trial to the Patient
Presented by
Cell therapy manufacture 1: Are
we ready to deliver commercially
feasible cellular immunotherapy
products at scale?
(Shared with the Immunotherapy Forum)
12.05 US FDA perspective
Dr Keith Wonnacott, Chief of
Cellular Therapies Unit, Office of
Cellular Tissue and Gene
Therapies (OCTGT), US Food and
Drug Administration (FDA)
12.20 Presentation reserved
12.40 Industry perspective
Dr James Trager, Vice President,
Research & Product
Development, Dendreon
1.00 Automate sooner than later:
The need for flexible, fully
automated processing
platforms
Dr Kai Pinkernell, Global Head of
Clinical Business, Miltenyi Biotec
GmbH
Examining the evolution and
future role of automated cell
therapy bioprocessing
technologies
• Addressing issues of cost and
timing (how/when to automate?)
Logistics 1: Placing logistics at the
heart of the cell & gene therapy
business model
• Maximizing quality control, minimizing
‘Cost of Logistics’ for cell and gene
therapy products
Dr Aby Mathew, Senior Vice
President & Scientific Officer,
Biolife Solutions Inc
12.25 Setting the scene: Why ‘Cost
of Logistics’ is a greater
obstacle than Cost of Goods
to the viable
commercialization of cell
therapy products
Simon Ellison, Senior Business
Development Manager, Cell
Therapy Catapult
Eric Halioua, Co-Founder & Chief
Executive Officer, Promethera
Biosciences
1.05 Questions for the speakers &
panel discussion
• Exploring the impact of short vs.
long product shelf life on Costs
of Logistics
Early-stage R&D strategy:
Optimizing preclinical-Phase I
translation for C&GTs
Katherine Tsokas, JD, Senior
Director, Global Regulatory Affairs,
Janssen Research & Development,
LLC
12.10 Preclinical considerations for
cell and gene therapy
products: US FDA perspective
• Considerations for preclinical
studies to enable initiation of
early-phase clinical trials
• Communications with
CBER/OCTGT
• Regulatory Resources
Dr Theresa Chen,
Pharmacology/Toxicology
Reviewer, Office of Cellular Tissue
and Gene Therapies (OCTGT),
Center for Biologics Evaluation and
Research (CBER), US Food and
Regulator and industry early
stage R&D insights…
Clarification of which
preclinical studies are
requirements – and which are
optimal - for translation to
First-in-Man studies
• Defining adequate controls
Gene therapy clinical update:
Assessing the prospects of leading
product candidates across the key
therapeutic areas
Dr James Wilson, Professor of
Internal Medicine & Pathology and
Laboratory Medicine, Director of
the Gene Program, University of
Pennsylvania, Research Director,
Center for Orphan Disease
Research and Therapy
12.15 Commercialisation of gene
therapy products – a fully
integrated lentiviral vector
strategy
Peter Nolan, Senior Vice President,
Commercial Development,
Oxford Biomedica
12.35 Selective cancer
immunotherapeutics on a
novel replicating retroviral
vector (RRV) platform
• Tumor specific infection and
replication of RRV in preclinical
models and clinical trial patients
• Dual action of direct tumor killing
and anticancer immunity
Case studies: The latest safety
and efficacy data for leading
gene therapy product
candidates in the clinic for
a range of therapeutic
areas/indications – where
are different serotypes
demonstrating the
greatest utility?
Sponsored by:
Sponsored by:

1.20 Buffet lunch in the
Exhibition/1-2-1 Meeting
Area
OR
Lunch Briefing
Optimal Session for a maximum of
50 attendees)
Neural stem cell-mediated
therapy for brain tumors
Margarita Gutova, MD, Associate
Research Professor, Department
of Neurosciences, City of Hope
National Medical Center &
Beckman Research Institute
2.30 Case studies & multiple
stakeholder panel discussion
• How are the leading industrial
and academic players
progressing in developing
economically feasible,
commercial scale
manufacturing models for CAR
T-cell immunotherapy and other
ex vivo genetically modified cell
technologies?
• Ensuring sufficiently robust supply
of starting materials (eg.
lentiviral vectors)
• Clarification of evolving
classification of raw versus
starting materials and the
associated regulatory
repercussions
• Cell procurement
• Cell characterization
• Cost of Goods
• Testing of cells – how much can
you actually test after they have
been modified?
• Traceability
• Point of Care bioprocessing or
regional manufacturing centers
– which model is
optimal/commercially realistic?
Examining the challenges and
benefits in either approach
Speakers/panellists to include:
Dr Larry Couture, Senior Vice
President and Founding Director,
Centre for Applied Technology
Development, Beckman Research
Institute of the City of Hope
Dr Kenneth LeClair, Executive
Director, Novartis Cell & Gene
Therapy Unit
Dr Isabelle Riviére, Director Cell
Therapy & Cell Engineering
Facility, Memorial Sloan Kettering
Cancer Centre
Mitchell H. Finer, Chief Scientific
Officer, Bluebird Bio
J. Joseph Melenhorst, PhD,
Director, Product Development &
Correlative Sciences Laboratories,
Translational Research Program,
Department of Pathology and
Laboratory Medicine, University of
Pennsylvania
3.40 Close of session, followed by
afternoon tea in the Exhibition/1-2-
1 Meeting Area
Sponsored by:
Exhibition/1-2-1 Meeting
Area
OR
Lunch Briefing
50 attendees)
Research Professor, Department
of Neurosciences, City of Hope
2.30 Case study 1
Frederick Miesowicz, PhD, Chief
Operating Officer & Vice
President Manufacturing, Argos
Therapeutics Inc
2.50 Questions & discussion
3.20 Case study 2
Dr Wilfred Dalemans, Chief
Technical Officer, Tigenix
(Provisionally confirmed)
afternoon tea in the Exhibition/1-
2-1 Meeting Area
Sponsored by:
Dr Geoffrey Nichol, Executive Vice
President, Research &
Development, Sangamo
BioSciences
12.40 Early R&D for stem cell based
therapies: Preparing for the
Phase 1 clinical trial and
beyond
Dr Jane Lebkowski, Vice President
Research & Development, Asterias
Biotherapeutics
1.00 …followed by questions for the
speakers & panel discussion
What can be done in practice to
alleviate the lack of insight current
animal models offer for dosing?
Exhibition/1-2-1 Meeting Area
OR
Lunch Briefing
50 attendees)
Research Professor, Department of
Neurosciences, City of Hope
2.30 Presentations & roundtable
discussion
Retrospectives: Lessons learned by
companies and organizations that
have successfully negotiated the
translation processes
• How did they meet the various
expectations and requirements
of investor, regulator and
clinician in order to advance
their product candidates to
human trials?
Speakers & panellists to include:
Anne-Virginie Eggimann, Vice
President, Regulatory Science,
bluebird bio, Inc:
Professor Miguel Forte, Senior Vice
President, Clinical & Regulatory,
TxCell
Katherine Tsokas, JD, Senior
Director, Global Regulatory Affairs,
Janssen Research & Development,
LLC
Dr Stefanos Theoharis,
Apceth GmbH & Co. KG.
1 Meeting Area
Sponsored by:
• Over 70 patients treated in 3 trials
for High Grade Glioma
• Excellent safety profile, tumor
shrinkage, and improved survival
versus relevant historical controls
• Planning registration-directed
trial in HGG and expansion into
other tumor types
Dr Douglas Jolly, Co-Founder &
Executive Vice President Research
& Pharmaceutical Development,
Tocagen
12.50 Progress in gene therapy for
heart disease
• Coronary artery disease and
congestive heart failure are
responsible for the majority of
cardiovascular morbidity and
mortality
• Several gene therapy
approaches have been
introduced in the clinic for these
two indications, using naked
plasmid, adenovirus and AAV
vectors via direct
intramyocardial injections and
intracoronary delivery methods
• Progress in the most advanced
clinical trials will be reviewed
Dr Gabor Rubanyi, Chief Scientific
Officer, Taxus Cardium
Pharmaceuticals Group
1.05 AVA-101 and challenges in
the clinical development of a
gene therapy for ocular
disease
• Ability to translate pre-clinical
animal models
• Selection of appropriate
endpoints
• Need to assess repeat dosing or
dosing in fellow eye
• Varying international regulatory
requirements
Dr Samuel B. Barone, Chief
Medical Officer,
Avalanche Biotechnologies
Exhibition/1-2-1 Meeting Area
OR
Lunch Briefing
50 attendees)
Research Professor, Department of
Neurosciences, City of Hope
2.45 Case study - Haemophilia
Dr Hansjoerg Duerr, Global Head
Haematology, Bayer Healthcare
panel discussion
1 Meeting Area
Sponsored by:
Afternoon plenary
Global regulatory update: Analyzing opportunities and challenges for industry
presented by evolving regulations worldwide
• What are the most significant areas of increasing harmony/disharmony?
Jiwen Zhang, PhD, Regulatory Affairs Director, GE Healthcare
4.25 European (CAT) perspective
• What’s new in Europe which can potentially impact C&GTs? (Eg. Adaptive
Licensing pathway)
Dr Martina Schusler-Lenz, Vice-Chair, Committee for Advanced Therapies (CAT) EMA
& Clinical Assessor, Division of Medical Biotechnology, Paul-Ehrlich Institute
4.45 Japanese perspective
Perspectives on novel Japanese regulatory pathways for cell & gene therapy
products
• What are the challenges in the new legislation for C&GTs from the industry
perspective? And where are the pitfalls?
• Can therapeutic candidates developed and manufactured outside of Japan now
qualify to utilize the new regulatory framework?
• Does a manufacturer need to have fully market-ready bioprocess in order to
receive conditional approval in Japan, or is it possible to validate your
manufacturing process at a later point?
David Hall, Chief Executive Officer, Replicel
5.05 Australasian perspective
Regulation of clinical cell and gene therapy trials Down Under
Prof John Rasko, Director, Department of Cell and Molecular Therapies, Royal Prince
Albert Hospital
5.25 Questions for the speakers, followed by panel discussion with industry
• International harmonization: From basic terminology to aspects such as potency
and dose specification - what are the key points of regulatory convergence and
divergence between the US, Europe and RoW?
• How does this change the strategic picture for cell & gene therapy companies
seeking to play in two or more continents?
Panellist:
Dr Keith Wonnacott, Chief of Cellular Therapies Unit, Office of Cellular Tissue and
Gene Therapies (OCTGT), US Food and Drug Administration (FDA)
6.00 Close of Day 1, followed by a Cocktail Reception in the Exhibition/1-2-1 Meeting Area
Keynote regulators’ perspectives: What are the latest and
forthcoming key developments for cell & gene therapy/ATMP
regulations, particularly with regard to pathways to market
and post-licensure requirements?
Would you like to sponsor this exclusive cocktail reception,
or perhaps a subsequent Speakers’ Dinner celebrating our stellar
panel? Contact Michael Adeniya now! (michael@phacilitate.co.uk)

Informative gathering of
elite bioleaders
Jim Zacka, VP, Worthington Biochemical Corporation
FORUM 2015
DAY 2 - Tuesday, January 27th 2015
Edward Lanphier, President & Chief Executive Officer, Sangamo BioSciences Inc
9.05 VC perspective
Dr Nessan Bermingham, Venture Partner, Atlas Ventures, Founder and Chief
Executive Officer, Intellia Therapeutics
9.25 US State stem cell funding organization
Dr C. Randal Mills. President & Chief Executive Officer, California Institute for
Regenerative Medicine (CIRM)
9.45 Philanthropists/family funds
William Podd, Founder, President & Chief Executive Officer, Landmark
Capital/Landmark Angels
10.05 Cell & gene therapy funding/business opportunities in emerging regions
Presentation reserved
10.25 Wall Street analyst’s Roundtable
Taking the pulse of the cell & gene therapy space from the institutional investors’
perspective – what are their expectations for the short-, mid- and long-terms?
Boris Peaker, Managing Director, Biotechnology Stocks, Cowen and Company
Howard Liang, PhD, Managing Director Biotechnology Equity Research, Leerink
Partners LLC
10.50 What’s coming next? Showcasing the next wave of cell & gene therapy
technologies
What is coming over the horizon? What will follow MSCs and the latest batch of
cellular immunotherapies? What’s will drive further progress in the gene therapy
space? A timely look at the emerging technologies and modalities which will
maintain momentum in the cell & gene therapy sector
11.55 Presentation 1
James Noble, Chief Executive Officer, Adaptimmune
Jeb Keiper, Vice President, Oncology Business Development, GlaxoSmithKline
Worldwide Business Development
12.0 Presentation 2
Edward Lanphier, President & Chief Executive Officer, Sangamo BioSciences Inc
John McDonald, Vice President, Business Development, Biogen Idec
12.05 Big Pharma and Big Biotech presentations & roundtable discussion
• Many in the C&GT sector see 2014 as the year Big Pharma really came to the party
– what does 2015 hold in store?
Panellists:
Perry Karsen, Chief Executive Officer, Celgene Cellular Therapies
Jan Thirkettle, Head of Advanced Therapy Delivery, GlaxoSmithKline
Albert Seymour, Global Head of Research and Nonclinical Development, Shire
Pharmaceuticals
John McDonald, Vice President, Business Development, Biogen Idec
Dr Devyn Smith, Head of Strategy, Pharmatherapeutics Research & Development, Pfizer
12.45 Presentations & panel discussion
How will industry-academia-public sector consortia and other collaborative models
support growth of regenerative medicine through 2015 and beyond?
• Updates on the status and goals of key initiatives in North America and Europe
Panellists:
Dr Mahendra Rao, Consultant, NYSCF & Vice President Strategic Affairs,
Q Therapeutics
Brock Reeve, Executive Director, Harvard Stem Cell Institute
1.20 Buffet lunch in the Exhibition/1-2-1 Meeting Area
Morning plenary
Part 1: Investment trends and keys to funding success for cell & gene
therapy biotechs
Part 2: Licensing and partnering
Updates from traditional and alternative sources of cell & gene therapy
R&D funding
• Is the biotech IPO back to stay?
• What has driven the significant growth in private investment in the space?
• Reviewing the Q2 2014 biotech downturn; what is the status as of January 2015?
Was it a flash in the pan, or something more significant?
• What specific technologies or areas are interesting them currently and why? Where
do they see the greatest potential for significant ROI and/or improvements to
human health?
• What are the latest trends in terms of the nature and structure of C&GT R&D funding
models?
Review and celebration of some of the major deals of 2014
• Brief analysis of key drivers and deal structures from both sides
• How and where was the necessary value created in the eyes of the pharma
stakeholders?
7.30 Buffet breakfast in the exhibition area
OR
7.45 Breakfast Briefing
(Optional session for a maximum of 50 attendees)
Creating a cell therapy manufacturing system for
commercial production
Workshop leader:
Richard Grant, Director of Cell Therapy, Invetech Pty Ltd
Panellists:
Dr Fred Miesowicz, Chief Operating Officer, Argos Therapeutics
Donald Powers, Principal Scientist, Janssen Cell Therapy
Nick Timmons, Director, Product and Process Development, Centre for
Commercialisation of Regenerative Medicine
Sponsored by
Do you represent a biotech with a platform technology which may be of interest
to our audience? Contact Michael Adeniya today (michael@phacilitate.co.uk)
to register your interest in presenting during this dedicated Cell & Gene Therapy
Forum Biotech Showcase morning plenary session.
Focus session 1 Focus session 2 Focus session 3 Focus session 4
Adoptive T cell immunotherapy
clinical updates:
• promise and limitations of CAR-T
• TCRs and TILs waiting in the wings
Simon F. Lacey PhD, Director,
Translational and Correlative
Studies Laboratory, Translational
Research Program, Perelman
School of Medicine, University of
Pennsylvania
2.35 Short presentations and
panel discussion
Drawing on clinical case studies
speakers will address issues
including:
• Safety prediction and
monitoring
• Correlates of on- and off-target
effects
• Efficacy prediction and
monitoring
Gene therapy manufacture: How
are the next wave of products in
late-phase development preparing
for commercial scale?
Product supply for large
market indications:
MYDICAR® (AAV1/SERCA2a)
for heart failure
• Update on the path towards
2000L commercial scale
manufacturing
Dr Barbara Thorne, Senior Director,
Process Development, Celladon
Corporation
Multiple stakeholder presentations…
What are the remaining necessary
technological and strategic
developments to enable
commercially feasible manufacturing
of gene therapy products?
R&D strategy: Defining next steps for
genome editing technologies
•How will the various approaches
address technological and regulatory
challenges?
Dr Thomas Vogt, Vice President
Discovery & Systems Biology, CHDI
Management/ CHDI Foundation
2.30 Case study 1
Dr Philip D Gregory, Senior Vice
President, Research & Chief
Scientific Officer, Sangamo
BioSciences
Case studies: Updates on the
leading technologies approaching
the clinic as well as those already
in trials – how do they compare?
• What will the development
pathway to market for these
products look like?
Clinical/commercial strategy
updates: Preparing MSCs and other
stem cell therapeutics for market
Dr Kim Warren, Head of Custom
Development Services for Cell
Therapy, Lonza Walkersville Inc
2.30 Case study – CV
Dr Andrew Hamer, Vice President,
Medical Affairs, Capricor
2.45 Case study – Musculoskeletal
diseases
Dr Donna Skerrett, Chief Medical
Officer, Mesoblast
Case studies: Examining the latest
clinical, bioprocessing and
commercial business models for
leading adult stem cell therapies in
key indications/therapeutic areas

Excellent presentations and discussions with lively and
productive debate. A very good overall summary of the
potential issues.
Keren Winmill, Non-Executive Director, Biotec Services International
• Specificity – is one protein
enough?
• How broadly do the
technologies scale?
• Application beyond malignant
patients
• How do TCRs and TILs compare
with CAR-T in terms of
commercial promise and
limitations, particularly for solid
tumors?
• Combination strategies – with
checkpoint inhibitors,
vaccines…
• When/how to move
combinations up to earlier-stage
cancer patients
• What has driven the interest of
big pharma to these
technologies?
Speakers include:
Dr Helen Tayton-Martin, COO,
Adaptimmune
David D. Chang, MD, PhD,
Executive Vice President R&D &
Kite Pharma, Inc
Mitchell H. Finer, Chief Scientific
Officer, bluebird bio
Dr Manish Singh, Chairman &
CEO, Lion Biotechnologies, Inc
afternoon tea in the Exhibition/
1-2-1 Meeting Area
2.40 Preparing for file and launch.
The challenges in delivering
and commercialising a gene
therapy medicine.
• What technical and regulatory
challenges need to be solved
for an approvable file?
• What is ‘commercialisation’ of a
gene therapy product and
what will it look like when done
successfully?
Dr Jim Faulkner, Vice President,
CMC & Supply, GlaxoSmithKline
Rare Diseases
2.55 How are the tools and
services sectors preparing to
support large scale gene
therapy manufacture?
• Making commercial scale
manufacturing a reality,
depending on therapeutic
indication
• How tools and service
companies are reacting to the
current high interest in gene
therapy
• The economics behind
potentially curative therapies
Dave Backer, Senior Director &
General Manager, SAFC Carlsbad
3.15 FDA perspective
• Update on guidelines and
thinking around reference
materials for AAV gene therapy
products
Dr Denise Gavin, Team Lead,
Gene Therapy Branch, Office of
Cellular Tissue and Gene
Therapies (OCTGT), US Food and
3.30 …and panel discussion:
• How to ensure both quality and
scalability of gene therapy
manufacturing process to
support the delivery of gene
therapy products to large
patient populations?
• What will be the keys to
adequately controlling Cost of
Goods at commercial scale?
1-2-1 Meeting Area
2.50 Case study 2
Dr Nessan Bermingham, Venture
Partner, Atlas Ventures, Founder
and Chief Executive Officer,
Intellia Therapeutics
3.10 Case study 3
Dr Rodger Novak, Chief Executive
Officer, CRISPR Therapeutics
panel discussion
• Defining and addressing the key
regulatory questions
• How to define efficacy,
potency, quality controls?
1-2-1 Meeting Area
3.00 Case study –
Immunomodulatory activity
in autoimmune disease and
ischemic injury
Dr John Harrington, Executive Vice
President & Chief Scientific Officer,
Athersys Inc
3.15 Case study
Presentation reserved
panel discussion
• Can we reach a consensus on
what the definition of an MSC
should be?
Panellists – speakers of the session,
plus:
Dr Christopher Bravery, Principal
Consultant, Advbiols
Dr Ohad Karnielli, Vice President,
Technology & Manufacturing,
Pluristem Therapeutics Inc
1-2-1 Meeting Area
4.40 Chair's introduction
4.45 How is the commercial manufacturing/product delivery model for
Glybera continuing to evolve?
• How are we addressing/have we addressed quality, stability and purity
challenges?
• How are we meeting the challenges of fulfilling long term follow-up requirements
for gene therapy-treated subjects, as well as more conventional regulatory post-
approval commitments?
Dr Hans Preusting, Chief Business Officer, UniQure
5.05 Short presentations & panel discussion
Updates from leading C&GT product candidates for rare disease indications
• What Phase II/Phase III trial designs are they adopting and why?
• What does the commercial business case look like?
• How do they plan to address the commercial scale up challenge?
• Exploring challenges in technology licensing for rare disease therapeutic
candidates – how can the sector work together to help de-risk R&D?
Panellists:
Faraz Ali, Vice President, Commercial Planning & External Affairs, Bluebird Bio
Dr Jim Faulkner, Vice President, CMC & Supply, GlaxoSmithKline Rare Diseases
Dr James Wilson, Professor of Internal Medicine & Pathology and Laboratory
Medicine, Director of the Gene Program, University of Pennsylvania
6.00 Close of day 2
Would you like to sponsor an exclusive cocktail reception
on the evening of day 2 of the event?
Contact Michael Adeniya now! (michael@phacilitate.co.uk)
Afternoon plenary
Ensuring the next wave of cell & gene therapies for rare disease
indications achieve commercial success
• Robust clinical trial design and commercial scale up – recent lessons learned?

Good talks and networking
opportunity
Mike Adams, Director of Marketing, Client Services and
Proposal Management, Fisher BioServices
FORUM 2015
DAY 3 - Wednesday, January 28th 2015
Faraz Ali, Vice President, Commercial Planning & External Affairs, Bluebird Bio
9.05 Pricing and reimbursement perspective
Neil Palmer, President and Principal Consultant, PDCI Market Access
9.30 Physician’s perspective
Dr Richard Maziarz, Researcher, Oregon Health Science University Hospital
9.55 Industry response – case study
How can a cell therapy company address these challenges?
• How can the industry become more efficient at meeting these challenges?
• Ways for the cell therapy community to learn from Pharma mistakes
• The importance of value
Dr Sven Kili, Senior Director, Global Medical Affairs, Biosurgery Sanofi Biosurgery
10.45 Multiple stakeholder roundtable discussion
• How to ensure the cell & gene therapy industry obtains fair value for what it brings
to the table? (How do you price a therapy that is curative of a chronic disease, for
instance?)
• How can the industry capitalize on EHRs and other emerging data sources to
improve the R&D and commercial prospects of their products?
• Unlocking the potential of cutting-edge informatics and analytics solutions to
transform the cell & gene therapy development and commercial business models
Panellists - speakers of the session, plus:
Dr John Maslowski, Vice President Scientific Affairs, Fibrocell
Morning plenary
Is your cell or gene therapy product candidate aligned with the needs
and capabilities of the marketplace?
• What is and isn’t viable in the eyes of today’s payers, physicians and patients?
Healthcare sector perspectives
• Defining evolving trends and challenges for each stakeholder group which carry
repercussions for the cell & gene therapy industry
• Creative thinking around P&R models for premium-priced cell & gene therapies to
ease the burden of financial risk for practice managers and independent physicians
• What changes can the cell & gene therapy industry initiate to help make both
autologous and allogeneic products viable and affordable for all stakeholders?
OR
Strategy and innovation for cell therapy
manufacturing – creating a roadmap to a commercial future
Dr Knut Niss, Senior Technical Project Leader, Novartis
Dr Jurjen Velthuis, Vice President CMC, Kiadis Pharma
Sponsored by
Focus session 1 Focus session 2 Focus session 3
Cell therapy manufacture 2: Refining process
science to control Cost of Goods:
Dr Ian Harris, Senior Director, Cell Therapy,
Janssen Research & Development LLC
12.05 Allogeneic cell therapy manufacturing:
What are the remaining technology
gaps?
Dr Greg Russotti, Vice President Technical
Operations, Celgene Cellular Therapeutics
Dr Knut Niss, Senior Technical Project Leader,
Novartis
12.45 Reducing cell manufacturing CoGs while
improving product quality - can one
platform do it all?
Dr Eytan Abraham, Head of Cell Therapy
Research & Technology, Lonza Walkersville Inc
1.05 Questions for the speakers & panel
discussion
• Exploring the latest guidelines and thinking
around reference materials for cell therapy
products – repercussions for the industry
Panellists – speakers of the session, plus:
Dr John Harrington, Executive Vice President &
Chief Scientific Officer, Athersys Inc
1.30 Close of the Phacilitate Cell & Gene Therapy
Forum 2015 – buffet lunch in the Exhibition/
1-2-1 Meeting Area
Case studies: How to build CoGs into your
planning from the start of bioprocess
development – lessons learned
Logistics 2: Can next-generation storage
technologies unlock regenerative medicine
markets?
12.25 Controlling cell delivery, from
manufacturing to the patient via
shipment, storage, thawing and routs of
administration
Dr Ohad Karnielli, Vice President, Technology &
Manufacturing, Pluristem Therapeutics Inc
12.45 Industry perspectives…
Identifying best practices to keep regenerative
medicine products stable all the way to the
clinical site
Dr Zorina Pitkin, Vice President, Quality Systems,
Organogenesis
1.15 …followed by questions for the speakers
& roundtable discussion
How can the stem cell therapy industry best
prepare to meet labelling and other supply
chain requirements at commercial scale?
Forum 2015 – buffet lunch in the Exhibition/1-2-1
Meeting Area
Case studies: Comparing and contrasting
different emerging technologies/approaches
to alleviating the challenges in stem cell
freezing and thawing
• How does each example impact cost and
quality?
Clinical strategy: What learnings do recent
Phase II trials provide to help improve
understanding of cell therapy MoA?
Dr Andrew Hamer, Vice President, Medical
Affairs, Capricor
12.05 Case study 1
Dr Robert W. Mays, Head of Neuroscience,
Athersys, Inc.
12.45 Back and forth between bench and
bedside: NHLBI and hematologic cell
therapies
• Two case studies of NHLBI projects that have
produced phase II trials and ancillary studies
• The role of NHLBI in facilitating translational
studies and early phase clinical trials
• Working between sister agencies—how to
navigate the FDA and the NHLBI
Dr Traci Heath Mondoro, Chief, Translational
Blood Sciences and Resources, NHLBI
1.05 Questions for the speakers, followed by
roundtable discussion
• How does pharma assess the positive and
negative in 2014’s Phase II results?
• What is their view on best steps forward for
robust Phase II trial designs - and for R&D as a
whole - for cell & gene therapy products in the
indications in question? (Eg. What more can
we do at an earlier stage, such as in animal
studies?)
• Where are we seeing advancements in
understanding of MoA to improve future trial
designs? (To include a specific focus on the
CNS/ophthalmology areas)
Forum 2015 – buffet lunch in the Exhibition/1-2-1
Meeting Area
Case studies: In depth analysis of 2014’s
major cell therapy Phase II trials - what
can we say we do know – and what don’t
we know – about dosing, potency,
delivery and Mechanism of Action?

Dr Devyn Smith, Head of Strategy, Pharmatherapeutics Research & Development, Pfizer
9.10 Stem cells in drug discovery: a reality check
• Preclinical models for efficacy and safety
• Adoption of new technologies in exciting and stressful times
• Lessons from the high throughput revolution of the 90s
Dr Ricardo Macarron, Vice President of Target and Pathway Validation,
GlaxoSmithKline
9.35 Presentation 2
Dr Huseyin Mehmet, Vice President, Head of Cell & Molecular Biology, Proteostasis
Therapeutics Inc
Updates on leading cell banking initiatives and consortia
• Current status: How are these cell banks being used and how are limitations being
addressed?
- How are they expanding cell lines?
• How are we seeking to coordinate efforts on a multinational basis?
Speakers/panellists include:
Dr Christopher Bravery, Principal Consultant, Advbiols
Dr Artur Isaev, Chief Executive Officer, Human Stem Cells Institute
11.55 DARPA perspective
Colonel Daniel Wattendorf, Program Manager, Defence Science Office, DARPA
12.15 NIH perspective
• What does the future hold for the CRM, particularly relating to stem cell tool
development?
Dr Anton Simeonov, Acting Deputy Scientific Director, National Centre for Advancing
Translational Sciences (NCATS), NIH
12.35 Review of CIRM’s latest stem cell tool funding round – future plans
Neil Littman, Business Development Officer, California Institute for Regenerative
Medicine (CIRM)
12.55 Questions for the speakers & panel discussion
1.20 Buffet lunch in the Exhibition/1-2-1 Meeting Area
Dr Christopher Bravery, Principal Consultant, Advbiols
2.30 US perspective
Dr Joyce Frey-Vasconcells, Regulatory Expert, Frey-Vasconcells Consulting, LLC
2.50 European perspective
Insight in safety and efficacy of stem cell based advanced therapy
medicinal products
Prof Asterios Tsiftsoglou, AUTH Lab of Pharmacology, School of Pharmaceutical
Sciences & CAT Member
3.10 Japanese perspective
David Hall, Chief Executive Officer, Replicell
3.30 Questions for the speakers & regulator/industry roundtable discussion
• How do recent regulatory changes in Japan translate to opportunity for the global
stem cell tool sector?
• What is next for cardio tox standards? How will regulators and industry move this field
forward?
4.00 Close of session, followed by afternoon tea in the Exhibition/1-2-1 Meeting Area
4.40 Chair's introduction
Dr Mahendra Rao, Consultant, NYSCF & Vice President Strategic Affairs, Q Therapeutics
• Fine tuning economies of scale
- What role can cell/tissue banks play in assisting?
• How are standardization initiatives progressing?
• Update on cell differentiation initiatives
• Developing tools that enhance the physiologic relevance of hiPSC models in relation
to drug discovery
• Determining/measuring cell pluripotency
- To what extent are cutting edge epigenetic mapping and deep sequencing
tools and techniques improving assessment of pluripotency?
• Addressing licensing issues and challenges for iPSC-related technology
Neil Littman, Business Development Officer, California Institute for Regenerative
Medicine (CIRM)
Professor John Rasko, Director, Department of Cell and Molecular Therapies, Royal
Prince Albert Hospital
Dr Carl G. Simon, Jr, Biologist and Project Leader of 3D Tissue Scaffolds, National
Institute of Standards and Technology (NIST)
Dr Eileen Dolan, Professor of Medicine, Chair, Committee on Clinical Pharmacology
and Pharmacogenomics, University of Chicago, Associate Director for Education,
6.00 Close of day 2
Dr Michele Cleary, Executive Director, Target and Pathway Biology, Merck
9.10 Establishing iPSC technology for Pharmacogenomic Studies
Dr Eileen Dolan, Professor of Medicine, Chair, Committee on Clinical Pharmacology
and Pharmacogenomics, University of Chicago, Associate Director for Education,
9.40 Case study 2
Reserved for Technology Spotlight Sponsor
10.05 Advent of Industry Scale hiPSC Models & Adoption in Drug Development
• Off the shelf access to pure iPS human tissues at large scale and from desired
genotypes is a significant advance.
• Data thus far present a strong case for use of human iPS models for late stage
discovery and predictive toxicology.
• Cost and concerns about functionality appear to limit application of the models at
earlier stages.
• How do we accelerate use of hiPSC models from ‘cradle-to-grave’ in the discovery
process?
Carter Cliff, Business Development, Cellular Dynamics International Inc
10.30 Case study 4
10.55 Case study 5
12.00 Case study 6
12.25 Case study 7
12.50 Case study 8
1.15 Further questions for the speakers & panel discussion
1.30 Close of Phacilitate Stem Cells as Discovery & Research Tools 2015 – buffet lunch in
the Exhibition/1-2-1 Meeting Area
Plenary
Thought leader analysis of macro trends in the stem cell tools arena
Keynote Presentations: Assessing big pharma and biotech strategies for
stem cell tool adoption and implementation – where do we see the
greatest value moving forward for drug discovery and development?
• Does pharma still see tools as the ‘low hanging fruit’ in the stem cell space? Why then
has uptake been comparatively slow to date across the sector, and what will it take
to change this trend?
• Are we learning lessons in terms of our adoption of potentially disruptive discovery
technologies?
• How do we expect 3D screening and automation to impact discovery research,
particularly in the targeted medicine and rare disease spaces?
• Licensing and partnering trends and deal analysis – what sort of collaborative models
are pharma companies looking to adopt with stem cell tool providers today?
Plenary
Regulatory updates from the US, Europe & Japan
Regulators’ perspectives from 3 continents
• Driving standardization of iPS cells
• What’s the latest view on the acceptability of stem cell-derived insights as predictors
of therapeutic safety and efficacy?
• Progressing standardization of raw/starting materials
Plenary
Driving progress in the iPSC space
DAY 2 - Tuesday, January 27th 2015
DAY 3 - Wednesday, January 28th 2015
Plenary
Next generation technologies showcase
• Reviewing cutting edge applications for screening, toxicology and diagnostics
Case studies: Defining the utility/pros and cons of technologies in
emerging technology areas, including:
• Novel predictive models for phenotypic screening
• Organ/tissue creation for toxicology screening (including 3D organ and tissue printing)
• Robotics and automation
OR
Optional session for a maximum of 50 attendees)
Creating a Cell Therapy Manufacturing System
for Commercial Production
Workshop leader:
Richard Grant, Director of Cell Therapy, Invetech Pty Ltd
Panellists:
Dr Fred Miesowicz, Chief Operating Officer, Argos Therapeutics
Donald Powers, Principle Scientist, Janssen Cell Therapy
Nick Timmons, Director, Product and Process Development, Centre for
Commercialisation of Regenerative Medicine
Sponsored by
OR
Strategy and innovation for cell therapy
manufacturing – creating a roadmap to a commercial future
Dr Knut Niss, Senior Technical Project Leader, Novartis
Dr Jurjen Velthuis, Vice President CMC, Kiadis Pharma
Sponsored by
SCADART attendees are automatically registered to also attend day 1 of the
Cell & Gene Therapy and Immunotherapy Forums on Monday, January 26th

Definitely the best meeting we
have attending in the last year
where we could find potential
pharma/biotech clients.
John R. Jaskowiak, Vice President, Angiocrine Bioscience, Inc.
FORUM 2015
Have you joined our Phacilitate LinkedIn group yet?
This LinkedIn group is an international online community focused on providing pharma and biotech decision makers involved in
the Cell & Gene Therapy field with a platform for senior level knowledge exchange, benchmarking and networking, much as
the Phacilitate Washington’ Forum itself does. Search groups for ‘’Phacilitate’’
It was an excellent meeting,
one of the best strategic/state
of the field meetings I’ve
attended.
Gary Mansfield, Director of Custom Biological
Toxicity Programs, WuXi AppTec
Contact Michael Adeniya now to book your
own private meeting area for the event!
michael@phacilitate.co.uk

Cell Therapy Catapult
The Cell Therapy Catapult was established as a centre of
excellence in 2012 to create a world-leading cell therapy industry
in the UK through innovation and collaboration.
Supported by the UK Government, our mission is to drive the
growth of the industry by helping cell therapy organisations across
the world translate early stage research into commercially viable
and investable therapies.
With one of the largest dedicated cell therapy teams anywhere,
our scientists, business development, manufacturing and
regulatory experts are collaborating with cell therapy
organisations and other interested parties from across the globe.
Together we are also finding solutions to industry-wide challenges
including business models, logistics and reimbursement.
GE Healthcare
GE Healthcare Life Sciences delivers breakthroughs in drug
discovery, biopharmaceutical manufacturing and the latest in
cellular technologies, so scientists and specialists around the world
discover new ways to predict, diagnose and treat disease. For
more information about GE Healthcare Life Sciences, visit our
Website at www.gelifesciences.com.
Lonza
Lonza offers world class technology platforms in the areas of GMP
cell culture and viral-based therapeutic manufacturing, custom
biotherapeutic culture media, a large selection of primary and
stem cells and a full line of custom bioassays. Our extensive
experience in Cell and Gene Therapy process optimization and
scale-up innovation helps clients to safely and effectively
advance their products through all phases of the commercial
pipeline and maximize their return on investment.
Oxford Biomedica logo
Oxford BioMedica is a leading gene-based biopharmaceutical
company developing innovative medicines to improve the lives of
patients with high unmet medical needs. We have established
platform technologies in gene delivery and immunotherapy,
protected by an extensive intellectual property estate. The
Company’s technology platform includes a highly efficient gene
delivery system (LentiVector®), which has specific advantages for
targeting diseases of the central nervous system and the eye; and
is also widely used in the ex-vivo cell therapy arena.
Oxford BioMedica’s product portfolio is focused on high value,
growing markets. These include gene therapy products
engineered to treat Parkinson’s disease (preclinical, Phase I/II),
Motor Neurone Disease (preclinical) and ocular diseases
(preclinical), especially retinal diseases (Phase I and Phase I/II).
Progenitor Cell Therapy
PCT is an industry leader in development and manufacturing of
cell therapy products, and is the only contract development and
manufacturing organization (CDMO) to see Client's cell therapy
product receive marketing approval from the FDA. With over 12
years of exclusive cell therapy-focused experience, PCT has
helped over 100 Clients bridge the gap between discovery and
patient care through unparalleled strategic insight and efficient
transfer of cell-based therapies from laboratory into clinical
practice.
Sangamo Biosciences
Sangamo BioSciences, Inc. is developing novel zinc finger DNA-
binding proteins (ZFPs), for therapeutic gene regulation and
genome editing and has ongoing Phase 2 clinical trials to
evaluate safety and efficacy of a ZFP Therapeutic® for the
treatment of HIV/AIDS. Other therapeutic programs are focused
on monogenic diseases. Sangamo engineers sequence-specific
ZFP Nucleases (ZFNs) for gene modification and ZFP transcription
factors (ZFP TFs) for gene regulation. The company has an
agreement with Shire to develop therapeutics for hemophilia,
Huntingtons’ disease and other monogenic diseases and, in non-
therapeutic applications of its technology, has strategic
partnerships with Dow AgroSciences and Sigma-Aldrich
Corporation.
Fisher Bioservices
Fisher BioServices, part of Thermo Fisher Scientific, is a professional
stem cell and cell therapy services provider. With locations around
the world, Fisher BioServices is committed to providing
biorepository storage, logistics infrastructure and clinical trial
services to the cell therapy industry. Our facilities and capabilities
enable us to provide integrated solutions from collection site or
CMO to repository or clinical site. Services include GTP
biorepository, laboratory services, cell and specimen collection
kits, product distribution, on-site inventory management and ultra
cold chain management. For more information please visit:
www.fisherbioservices.com.
Life Technologies
Life Technologies is a global biotechnology tools company
dedicated to improving the human condition. With more than
50,000 products used by more than 75,000 customers around the
globe, Life Technologies is advancing scientific research in areas
such as next-generation sequencing, drug discovery,
bioproduction and cellular medicine.
Marken
Marken is the leading global clinical supply chain service provider
dedicated 100% to the pharmaceutical and life sciences
industries, supporting over 49,000 investigator sites in more than 150
countries. With decades of experience in the logistics, transport
and distribution of temperature sensitive life saving
pharmaceuticals, clinical trial supplies and specimen collection;
Marken integrates depot and logistics services into solutions that
extend the reach of clinical trials to even the most remote
treatment naïve geographies. Our team members and network of
facilities bridge the distance between patients and the essential
resources of life science companies.
Pall Life Sciences
Pall Life Sciences provides process, pilot and laboratory filtration,
separation, purification and fluid handling devices, systems and
services, with single-use systems available for all unit operations
from cell culture – including cell therapy applications - through
final formulation and filling. Based on Pall’s long history of providing
quality equipment for the biopharmaceutical, vaccine and cell
therapy industries, all products – whether standard or customized
to match users exact process needs – are backed up with
extensive documentation and experience in extractables,
leachables and particulate validation. Fully automated single-use
systems allow process control and data acquisition to meet or
exceed the standards expected from traditional fixed equipment.
New product highlights include microcarriers, pyrogen-free vials
and a range of pharmaceutical packaging.
SAFC
SAFC is the custom manufacturing and services business unit of
Sigma-Aldrich Corporation. As a trusted manufacturer for the life
science and high technology industries, SAFC works closely with
customers to resolve development challenges and accelerate the
product pipeline. Its rich portfolio includes critical raw materials,
contract manufacturing of viral vaccines and gene therapy drug
products, and extensive biologics safety testing services.
apceth
apceth is a pioneering clinical-stage biopharmaceutical
company expanding its pipeline of next-generation cell-based
therapeutics.
Our modular platform technology is based on genetically-
modified MSCs and the lead program, Agenmestencel, is a first-in-
man genetically-modified MSC, for the treatment for cancer. In
addition, we are developing drug candidates for the treatment of
lung diseases and inflammation.
Based in Munich, we provide our know-how, expertise and GMP-
certified facilities to industry and academic partners around the
world.
BioLife Solutions
BioLife Solutions develops, manufactures and markets
biopreservation media and high performance thermal packaging
products for cells, tissues, and organs. The Company's proprietary
HypoThermosol® and CryoStor® platform of solutions are highly
valued in the biobanking, drug discovery, and regenerative
medicine markets. BioLife's products are serum-free and protein-
free, fully defined, and are formulated to reduce preservation-
induced cell damage and death. BioLife's enabling technology
provides academic and clinical researchers significant
improvement in shelf life and post-preservation viability and
function of cells, tissues, and organs. For more information please
visit www.biolifesolutions.com.
Invetech Pty
Invetech is an innovative contract development company
working with cell therapy and bioprocess companies to bring new
products to market by combining our system definition tools,
technology and automation expertise with our client's process
knowledge. We develop appropriate scale-up strategies and
innovative platforms, including functionally closed systems, in
collaboration with processors to meet their quality and
therapeutic goals.
Miltenyi Biotec
Miltenyi Biotec provides innovative products and services that
advance biomedical research and cellular therapy. Our cutting-
edge tools support research at every level, from basic research to
translational research to clinical application. Used by scientists and
clinicians around the world, our integrated technologies cover
techniques of sample preparation, cell isolation, cell sorting, flow
cytometry, cell culture, molecular analysis, and preclinical
imaging. Our more than 25 years of expertise spans research areas
including immunology, stem cell biology, neuroscience, and
cancer. Today, Miltenyi Biotec has more than 1,400 employees in
25 countries – all dedicated to empowering discovery and
impacting lives.
Terumo BCT
Terumo BCT, a global leader in blood component, therapeutic
apheresis and cellular technologies, is the only company with the
unique combination of apheresis collections, manual and
automated whole blood processing, and pathogen reduction
coupled with leading technologies in therapeutic apheresis and
cell processing. We believe in the potential of blood to do even
more for patients than it does today. This belief inspires our
innovation and strengthens our collaboration with customers.
Terumo BCT—Unlocking the Potential of Blood.
Brightlands
A unique location for R&D, scale up and manufacturing in
Biomaterials, Tissue Engineering and Regenerative Medicine.
Brightlands Chemelot Campus is a world-leading innovation
location and home to a vibrant and fast-growing open
community of groundbreaking companies and knowledge
institutes.
It offers state of the art R&D and manufacturing infrastructures, on-
campus education and science-oriented business support,
venture capital, and business development services.
With its location, ecosystem and easily available expertise and
knowledge, Brightlands Chemelot Campus is a unique location for
innovative start-ups and corporations, forward-thinking knowledge
institutes, daring entrepreneurs, brilliant researchers, talented
students, and visionary investors.
GOLD SPONSORS
SILVER SPONSORS
BRONZE SPONSORS
ROUNDTABLE SPONSOR

I found this year's Cell & Gene Therapy
conference was probably the best conference I
have ever been to on all levels – the
presentations, the networking and the venue.
Dr Peter French, Chief Executive Officer, Benitec Biopharma Limited
FORUM 2015
Register now at www.bioleaders-forum.com
Book your conference pass before 14th November to save $200!
Group Discounts
Register 3 conference passes and receive the 4th FREE!
Small Companies
Discounts are available for smaller companies contact team@phacilitate.co.uk now to
find out if you qualify.
Registration
Registration Type Early Bird | Till 14.11.14 Standard Rate
Pharma/Biotech $2,695 $2,895
Academic/Not-for-Profit $1,595
Tool/Service Provider
Book by 14th
November for
our Early Birddiscount!
$3,795
Brand awareness,
key contacts,
thought leadership!
Establish real business leads
with a qualified audience of
prospective clients from
global pharma and biotech
by taking a stand in our
buzzing exhibition hall
Build your brand awareness
and lead generation with pre-
, onsite and post event
marketing and branding to
our network of industry leaders
Give a presentation on your
technology and engage
the attention of our audience
of potential clients and
partners
Sponsor a breakfast or lunch
briefing and be seen as the
industry thought leader
amongst your peers and
competitors
Sponsor a dinner or drinks
reception to remind them of
your market leading values in
an informal setting
Increase your standing within
the industry by taking part in a
panel discussion – a unique
opportunity to publicly voice
your opinion on pressing issues
and be aligned side-by-side
with your sector’s leading
playersEVENT PARTNERS
ADDITIONAL SPONSORS & EXHIBITORS
To find out more about sponsorship and
exhibition opportunities at Phacilitate
Washington 2015 contact Michael
Adeniya on +44 (0)207 384 7951 or on
michael@phacilitate.co.uk

Phacilitate Cell & Gene Therapy Main Announcement

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