This document provides information about the Cell & Gene Therapy conference taking place on October 10-11, 2018 in London. It includes details about the agenda, speakers, workshops, and registration information. The two-day conference will focus on topics related to manufacturing, regulation, and translational research of cell and gene therapies. There will be presentations from industry experts on developing stem cell and gene therapies, as well as workshops on practical development issues and competitive intelligence in the cell and gene therapy field.

1. www.cellandgeneconference.com
register online or fax your registration to +44 (0) 870 9090 712
or call +44 (0) 870 9090 711
a: practicalities in the development of
stem cell therapies
Workshop leader:
paul stroemer, Director,
advanced Therapies consultancy
08.30 - 12.30
B: strategic competitive intelligence in the
cell and gene therapy space
Workshop leaders:
Timos papagatsias, CEO,
lucidQuest
13.30 - 17.00
plus TWo inTeracTiVe half-DaY pre-conference WorKshops | TuesDaY 9Th ocToBer 2018, copThorne Tara hoTel, lonDon, uK
sMi pharma
@sMipharm
#sMicgt
chairs for 2018:
• Pamela tranter, Head of Translational Research Group, ucl
• giuseppe ronzitti, Cell and Gene Therapy Specialist, genethon
KeY speaKers incluDe:
• Janet glassford, Senior Quality Assessor (biologicals), Mhra
• Kei Kishimoto, Chief Scientifi c Offi cer, selecta Biosciences
• shailesh gupta, Senior Scientist, astraZeneca
• Peggy sotiropoulou, R&D Manager, Research and Development,
celyad
• Julie Kerby, Head of Manufacturing Development, cell and gene
Therapy catapult
• lior raviv, Vice President, Development, pluristem
• diego aridgo, R&D Rare Disease Unit Head, chiesi
• Mehdi gasmi, Chief Science and Technology Offi cer, adverum
Biotech
• emanuela cuomo, Associate Director, Discovery Sciences, Cellular
Biology Team, astraZeneca
• Yen choo, Founder and Executive Chairman, plasticell
• ian McKay, Innovation Lead Advanced Therapies, innovate uK
2018 featured highlights:
• Discuss the challenges of accessing patients with commercial
ATMPs
• understand the advantages of a risk-based approach to cell and
gene therapy manufacturing
• hear the MHRA’s perspective of UK and EU regulatory affairs
regarding ATMPs
• explore the unique applications of the CRISPR/Cas9 system for drug
development at AstraZeneca
• gain insight into GMP Manufacture of plasmid DNA at the NHSBT
WORKSHOPS: 9TH
CONFERENCE: 10TH-11TH
OCT
2018COPTHORNE TARA HOTEL, LONDON, UK
SMi Presents the Launch of…
Cell & Gene
Therapy
from manufacturing to patient access: discover the world
of advanced therapy medicinal products
register BY 31st MaY and saVe £400
register BY 29th June and saVe £300
register BY 31st august and saVe £200

2. Manufacturing, regulation and translational research
Therapeutic applications of ATMPs
MAY 2018
Injectable Drug Delivery
16th - 17th May 2018,
London, UK
Pain Therapeutics
21st - 22nd May 2018,
London, UK
Highly Potent
Active Pharmaceutical
Ingredients
21st - 22nd May 2018,
London, UK
Pre-Filled Syringes
West Coast
4th - 5th June 2018,
San Diego, CA
Pharmaceutical
Microbiology West Coast
7th - 8th June 2018,
San Diego, CA
Drug Safety
11th - 12th June 2018,
London, UK
Pharmaceutical Freeze
Drying Technology
13th - 14th June 2018,
London, UK
BioBanking
13th - 14th June 2018,
London, UK
JULY 2018
ADMET
9th - 10th July 2018,
London, UK
Molecular Diagnostics
9th - 10th July 2018,
London, UK
SEPTEMBER 2018
Immuno-Oncology
26th – 27th September
2018, London, UK
Biosimilars And Biobetters
26th - 27th September
2018, London, UK
OCTOBER 2018
Cell & Gene Therapy
10th - 11th October 2018,
London, UK
Orphan Drugs
17th - 18th October 2018,
London, UK
NOVEMBER 2018
Superbugs & Superdrugs
USA
12th - 13th November
2018, New Jersey, USA
Biosimilars North America
14th - 15th November
2018, New Jersey, USA
Lyophilization USA
15th - 16th November
2018, New Jersey, USA
Ophthalmic Drugs
26th - 27th November
2018, London, UK
DECEMBER 2018
Cold Chain
10th - 11th December
2018, London, UK
JUNE 2018
SMi PHARMACEUTICAL EVENT PLANNER 2018
Cell & Gene Therapy
Day One | Wednesday 10th October 2018 www.cellandgeneconference.com
8.30 Registration & Coffee
9.00 Chairman’s Opening Remarks
Pamela Tranter, Head of Translational Research Group, UCL
OPENING ADDRESS
9.10 Mitigating the immunogenicity of AAV gene therapy vectors with
tolerogenic nanoparticles
• Cellular and humoral immunity against AAV can affect the
safety and efficacy of gene therapy and the ability to re-treat
patients later in life
• We have developed synthetic vaccine particles encapsulating
rapamycin (SVP-Rapamycin) that have been shown to mitigate
immunogenicity against a variety of biologic drugs by inducing
tolerogenic dendritic cells and antigen-specific regulatory T cells
• We will present pre-clinical data demonstrating the ability of
SVP-Rapamycin to inhibit the formation of anti-AAV antibodies
and share data from an ongoing clinical trial of SVP-Rapamycin
combined with a therapeutic enzyme
Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences
9.50 Becoming cell therapy makers - Opportunities and challenges in
“in house” manufacturing of cell therapy products
• Reproducible manufacturing techniques - the basics of process
development
• The importance of closed and automated manufacturing
technologies
• Data management for improvement of process understanding
and control
• The importance of raw materials management
Lior Raviv, Vice President, Development, Pluristem
10.30 Morning Coffee
11.00 Spotlight: UK and EU regulatory affairs: MHRA’s perspective
• What support is available for companies from early in the
development process?
• Designing clinical trials in a way that comes up with the
evidence regulators need to accelerate ATMPs
• Ensuring safety while reducing cost of development: working
together with regulators to make the approval process more
efficient
• Impact of Brexit and further initiatives
Janet Glassford, Senior Quality Assessor (biologicals), MHRA
11.40 Opportunities and challenges of translating cell and gene
therapies in academia
• Review of the exciting and diverse pipeline of cell and gene
therapy projects within UCL with example case studies
• Opportunities for project funding: internal, public funds, industry
partners and University spin outs
• Understanding the de-risking and evidence required to support
the commercial route forward
Pamela Tranter, Head of Translational Research Group, UCL
12.20 Networking Lunch
1.20 Intravitreal rAAV gene therapy approaches for the treatment of
retinal disorders
• Indications that are currently pursued at Adverum
Biotechnologies
• Development of novel AAV capsid targeting the eye
• Advantages of gene therapy approaches to retinal disorders
Mehdi Gasmi, Chief Science and Technology Officer,
Adverum Biotech
2.00 Targeting solid and liquid malignancies with the same CAR
• What is an ideal target for CAR-T cell therapy?
• Challenges in targeting solid tumours using CAR T cells
• Autologous versus allogeneic CAR-T cells
Peggy Sotiropoulou, RD Manager, Research and Development,
Celyad
2.40 Afternoon Tea
3.10 Risk-based approaches to cell and gene therapy manufacturing
• Why manufacturing remains a significant challenge for cell and
gene therapies
• How Quality Target Product Profiles and process mapping
techniques can be used to generate a process risk assessment
and help you understand and accelerate your process
development
Julie Kerby, Head of Manufacturing Development,
Cell and Gene Therapy Catapult
3.50 Overcoming adeno-associated virus gene therapy limitations in
genetic neuromuscular diseases
• Rare, genetic neuromuscular diseases constitute a bigger
challenge given the absence of knowledge on the
physiopathological mechanisms and of therapeutic options
• Adeno-associated virus (AAV) vector-based gene therapy
became a principal actor in the development of therapies for
monogenic diseases
• Successful human trials of gene transfer in the liver for
hemophilia A and B, in the eye for congenital blindness and in
the nervous system for spinal muscular atrophy have unveiled
the therapeutic potential of this viral vector platform
• Development of technological tools to overcome the current
limitations of AAV gene therapy applied to neuromuscular
diseases
Giuseppe Ronzitti, Cell and Gene Therapy Specialist, Genethon
4.30 Chairman’s Closing Remarks and Close of Day One
Register online at www.cellandgeneconference.com

3. uPdates on cell theraPY deVeloPMent strategies
sponsorship anD eXhiBiTion opporTuniTies
sMi offer sponsorship, exhibition, advertising and branding packages, uniquely tailored
to complement your company’s marketing strategy. prime networking opportunities exist
to entertain, enhance and expand your client base within the context of an independent
discussion specific to your industry.
should you wish to join the increasing number of companies benefiting
from sponsoring our conferences please call: alia Malick, Director,
on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk
MarKeting PartnershiP oPPortunities
sMi group is offering companies the opportunity to partner on our
dedicated events in order to help raise your company profile,add value,
create awareness of your products/services to our key audience within
the pharmaceutical industry.
interested in partnering? contact pav solanki, sMi Marketing on
+44 (0) 20 7827 6048 oremail: psolanki@smi-online.co.uk
cell gene therapy
www.cellandgeneconference.com day two | thursday 11th october 2018
8.30 registration coffee
9.00 chairman’s opening remarks
giuseppe ronzitti, Cell and Gene Therapy Specialist, genethon
oPening address
9.10 advances in cell therapy process development and optimisation
at plasticell
• Combinatorial screening of parameter space for process
development and optimisation in cell and gene therapy
• Expansion of hematopoietic stem cells from cord blood for
allogeneic transplantation
• Enhancement of viral transduction effi ciency for autologous
gene therapy to treat rare diseases
Yen choo, Founder and Executive Chairman, plasticell
9.50 generation of endogenous reporter iPs cell lines for optimizing
differentiation protocols, understanding disease biology driving
drug discovery projects
• CRISPR/Cas9 in engineering iPS cells to generate cellular
reagents for drug discovery
• Clone screening capabilities/methodologies
• Characterization, validation potential application of PGE
modifi ed iPS cell lines
shailesh gupta, Senior Scientist, astraZeneca
10.30 Morning coffee
11.00 supporting advanced therapies – the innovate uK perspective
• Support for advanced therapies – from Innovate UK and across
Government
• Innovate UK support for the commercialisation of advanced
therapies
• How the Advanced Therapy Treatment Centres will enable
adoption of advanced therapies in the NHS
ian McKay, Innovation Lead, Advanced Therapies, innovate uK
11.40 spotlight: harnessing crispr/cas9 for drug development
• Genome editing for target identifi cation
• Genome editing for cell model generation and target validation
• Genome editing to understand drug resistance
• Genome editing as a therapeutic
emanuela cuomo, Associate Director, Discovery Sciences, Cellular
Biology Team, astraZeneca
12.20 networking lunch
inTeracTiVe KeYnoTe aDDress
1.20 Towards operational excellence in cell and gene therapy
manufacturing and delivery
• First generation processes in cell and gene therapy industry
• ’Needle to Needle’ challenges
• Lean Six Sigma for advanced therapy medicinal products
fabio d’agostino, Partner, alira health
2.00 accessing patients with commercial advanced therapies
• Addressing market access and reimbursement of advanced
therapies (ATMPs)
• Logistics and restricted access programs constraints in bringing
ATMPs to patients in a commercial setting
• Rare disease patient access with ATMPs
Diego aridgo, RD Rare Disease Unit Head, chiesi
2.40 afternoon Tea
3.10 gMP Manufacture of Plasmid dna
• Good plasmid DNA manufacturing practice begins with an
appropriate vector design. The eukaryotic promoter, gene
sequence, and poly-A site primarily affect the therapeutic
effi cacy, remaining part of the vector is important for
manufacturing
• Choice of the plasmid’s origin of replication (ori) is critical for
the plasmid copy number and for the cultivation titer. ColE1-
derived, high copy-number pUC ori is widely established due to
high copy number. Introduction of random or defi ned mutations
into the pUC ori can further increase the plasmid yield
• Selecting appropriate host strain, maximizing titer by upstream
optimization, and achieving superior performance in small and
large-scale cGMP manufacturing
paul lloyd-evans, Head of Clinical Biotechnology Centre, nhs
Blood and Transplant
3.50 scalable production of adult progenitor cell populations for
skeletal regeneration – bridging the gap
• Comparison across bioreactor systems - visualization tools for
bioprocess effi ciency
• Evaluation of functionality of expanded cell populations in small
animal models – setting the target quality profi les
• Development of real-time decision-making tools for optimising
bioprocess management for high-risk autologous production
• Adding a third dimension - controlling complexity in tissue
engineered ATMPs
ioannis papantoniou, ATMP Bioprocessing Coordinator, Skeletal
Biology and Engineering Research Centre, Ku leuven
4.30 chairman’s closing remarks and close of Day Two
alternatively fax your registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
MeDia parTners:

4. HALF DAY PRE-CONFERENCE WORKSHOP A
Tuesday 9th October 2018
Copthorne Tara Hotel, London, UK
8.30 - 12.30
Practicalities in the Development
of Stem Cell Therapies
Workshop Leader:
Paul Stroemer, Director,
Advanced Therapies Consultancy
Workshop Overview:
All cell therapies have unique challenges in their
development pathway. The route from initial innovation
to commencing clinical trials is rarely straight forward.
This workshop will focus on issues encountered in the
translational arc of cell therapies to help companies
minimize early setbacks. Topics for the day will cover the
span of product development from initial proof of concept
to clinical studies. Active participation in the workshop will
be highly encouraged.
Key Benefits of Attending:
• Acquire insight into the development process for cell
therapies
• Learn to identify potential issues and solutions to
maintain program timelines
• Participate in a meeting simulating interaction with a
competent authority
Agenda
8.30 Registration and coffee
9.00 Opening remarks and introductions
9.10 Overview of cell therapies
• Current products
• Prospective indications
• Regulatory outlook
9.40 Bridging the “Valley of Death”
• Developing the Target Product Profile
• Design of testing strategy
• Translation to the clinic
10.30 Morning Coffee
11.00 Manufacturing issues
• Technology transfer
• Product characterization
• Assay development
• Delivery challenges
11.30 Interactive Session: Preparation for a Competent
Authority meeting
• Candidate ATMP scenario
• Preparation of questions
• Simulated meeting
12.20 Closing remarks
12.30 End of workshop
About the Workshop Leader:
Paul is the Director of Advanced Therapies Consultancy.
He has a wealth of experience in the Pre-clinical, Safety,
Regulatory and Clinical study aspects of stem cell therapies.
Paul worked with ReNeuron in the development of its lead
cell line. He earned a PhD in Neuroscience at the University
of Texas Medical Branch at Galveston with
About the Organisation:
Advanced Therapies Consultancy was started in early 2018
to help emerging (and mature) biotechnology companies in
developing novel products. Mistakes are commonly made
early in developing advanced therapies. The Advanced
Therapies Consultancy helps clients focus on clinical use of
their product. This approach identifies potential issues that
can be rectified in the development pathway saving time
and money.

5. HALF DAY PRE-CONFERENCE WORKSHOP B
Tuesday 9th October 2018
Copthorne Tara Hotel, London, UK
13.30 - 17.00
Strategic Competitive Intelligence in
the Cell and Gene Therapy space
Workshop Leader:
Timos Papagatsias, CEO,
LuciQuest
Workshop Overview:
This workshop will focus on how stakeholders can combine
the latest advancements in Strategic Competitive
Intelligence in order to uncover opportunities and threats
for their companies and brands in the Cell and Gene
Therapy space. Workshop participants will also be provided
with a short step-by-step guide on how to use competitive
intelligence to generate actionable insights which will help
them gain a competitive advantage over others.
Benefits and Attending:
• Learn about Strategic Competitive Intelligence and how
to generate actionable insights
• Understand how to most effectively conduct CI in the Cell
and Gene Therapy space and remain competitive
Agenda
13.30 Registration and coffee
14.00 Introduction to Strategic Competitive Intelligence
• What CI is, what it is not and how to do it
• What you can and what you cannot do with CI
• Evolution of CI and its tools
• Importance of CI in the Cell and Gene Therapy
space
14.30 Afternoon Tea
15.00 How to gain or maintain a competitive advantage
in the Cell and Gene Therapy space, whether you
are a newcomer or established player
• What can you learn from your competitors and
the marketplace using CI in the Cell and Gene
Therapy field?
• Using CI to improve strategic and tactical
decisions on vector platform choice, trial design,
regulatory approach, payor interactions etc.
• Minimizing uncertainty in a rapidly moving space
• Combining database use and targeted primary
competitive intelligence
• Leveraging social media and social networks
to monitor competitor activity and identify
emerging threats and opportunities
15.30 “Future-proofing” your efforts by identifying
early warning signals and opportunities for your
company in the Cell and Gene Therapy space
• Short step-by-step guide / case study
16.00 QA
17.00 Closing remarks
About the Workshop Leader:
Timos has over 10 years’ experience working as a strategy
and intelligence consultant across the Pharma / Biotech and
the Life Sciences Investment industries. He has advised 18
out of 20 top Pharma and Biotechnology companies and
has also worked with a large number of medium and small
size firms on multiple topics (market entry and market access
optimization, patient centricity, digital healthcare and social
media, clinical product development and trial design, brand
positioning, portfolio analysis etc.) across therapy areas
and geographies. He is currently the CEO of LucidQuest,
a boutique strategic intelligence consultancy that focuses
both on traditional therapy areas (oncology, haematology,
immunology, CV/Met, CNS etc.) as well as on developing
insights into patient centricity, digital healthcare/IoE and
personalized medicine (gene and cell therapies).
About the Organisation:
LucidQuest provides strategic intelligence and evidence-
based investment insights for the Life Sciences and other
industries. We partner with leading Pharma, Biotech and
Healthcare companies and consultancy firms, providing
support across multiple therapy areas (respiratory, CV
Met, onco / haem, CNS, vaccines, gene and cell therapy
etc.) and also in the rapidly evolving Digital Healthcare /
IoE space. We further provide investment insights to hedge
funds, PE, VC, angel investors etc., leveraging our in-house
and our network’s scientific and commercial expertise.

6. Cell GeNE THERAPY
Conference: 10th -11th October 2018, Copthorne Tara Hotel, London, UK
Workshops: 9th October 2018, Copthorne Tara Hotel, London, UK
4 WAYS TO REGISTER
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PHONE on +44 (0) 870 9090 711
Online at www.cellandgeneconference.com
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