RNA Therapeutics 2017 returns to London next February

1. www.therapeutics-rna.com
Register online or fax your registration to +44 (0) 870 9090 712 or call +44 (0) 870 9090 711
ACADEMIC & GROUP DISCOUNTS AVAILABLE
PLUS INTERACTIVE HALF-DAY PRE-CONFERENCE WORKSHOP
Tuesday 21st February 2017, Copthorne Tara Hotel, London, UK
REGISTER BY 31ST OCTOBER AND SAVE £400
REGISTER BY 3OTH NOVEMBER AND SAVE £200
REGISTER BY 16TH DECEMBER AND SAVE £100
@SMIPHARM
REASONS TO ATTEND:
• Discover the exciting developments of mRNA and
antisense oligonucleotide based therapeutics
• Find out how pharmaceutical and biotech companies
are strategically improving drug delivery systems
• Discuss clinical developments and take away key lessons
for future developments
• Understand the pharmacokinetic considerations of
oligonucleotides and RNA therapies
• Hear how the experts navigate the regulatory landscape
Precision Nano-therapeutics: Minding the gap in delivery
from preclinical studies to clinical success
08.30am – 12.30pm
Leader: Professor Andrew David Miller, Professor of Organic Chemistry & Chemical Biology, CSO,
King’s College London, KP Therapeutics Ltd
SMi Presents the 8th annual Conference on…
RNA Therapeutics
Pushing the boundaries of 21st Century
pharmaceuticals through targeted delivery systems
Copthorne Tara Hotel, London, UK
WORKSHOP: 21ST
CONFERENCE: 22ND-23RD
FEB 2017
CHAIRS FOR 2017:
Nagy Habib, Head of Surgery, Co-Founder, Imperial
College Healthcare NHS Trust, MiNA Therapeutics
Heinrich Haas, Vice President RNA Formulation &
Drug Delivery, BioNTech RNA Pharmaceuticals
KEYNOTE SPEAKERS INCLUDE:
Bo Rode Hansen, Global Head of RNA Therapeutics,
Roche
John Johnston, Clinical Assessor Biologicals and
Biotechnology Unit, MHRA
Nicole Meisner-Kober, Senior Investigator, RNA
Biology, Novartis Institutes for Biomedical Research
Steve Hood, Director, Bioimaging,
GSK
David Giljohann, CEO,
Exicure
Shai Erlich, Chief Medical Officer & President USA,
Quark Pharmaceuticals Inc
PAST ATTENDEE TESTIMONIALS:
“Overall very good
organisation/speakers”
GSK
“The conference was very well
organised, interesting talks.”
2016 Delegate

2. RNA Therapeutics
Day One | Wednesday 22nd February 2017
08.30 Registration & Coffee
09.00 Chairman’s Opening Remarks
Professor Nagy Habib, Head of Surgery,
Co-Founder, Imperial College Healthcare NHS Trust,
MiNA Therapeutics
OPENING ADDRESS
09.10 RNA activation and its clinical application
• Small activating RNA (saRNA) can activate most of the
coding and non-coding genome
• CEBPa is a transcriptional activation of the hepatocyte
that can be upregulated with saRNA
• Clinical application: SARNA CEBPa offers a therapeutic
opportunity for patients with NASH and liver failure
Nagy Habib, Head of Surgery, Co-Founder, Imperial
College Healthcare NHS Trust, MiNA Therapeutics
09.50 Collaborating to deliver macromolecules across
biological barriers – a review of IMI COMPACT
• What are the challenges of Oligo Delivery?
• How do we optimise delivery
• Who are COMPACT and what have we achieved?
Steve Hood, Director, Bioimaging and D@T, GSK
10.30 Morning Coffee
11.00 Delivery of mRNA using the Viromer® system
• A lesson learned from a virus: the Viromer delivery system
• Formulation aspects of Viromer nanoparticles
• Viromer: mRNA delivery on multiple routes
Steffen Panzner, Managing Director, Lipocalyx GmbH
11.40 Therapeutic antisense oligonucleotides in oncology
• The progress of emerging antisense oligonucleotide drugs
and the targeting of un-druggable sites in oncology
• Methods in drug delivery
• Update on clinical trials in the field
Mark Edbrooke, Senior Principal Scientist, AstraZeneca*
12.20 Networking lunch
13.30 RNA Therapeutics Harnessing the Potential of Locked
Nucleic Acid
• Exciting new insights into RNA Therapeutics drug
discovery
• Locked Nucleic acids: an update on how they are drug
candidates for diseases with difficult targets
• Next generation drugs and how they are transforming
the treatment of diseases
• New concepts to conducting drug discovery and
optimising the process
Bo Rode Hansen, Global Head of RNA Therapeutics and
General Manager, Roche Innovation Center Copenhagen,
Roche
14.10 Sites of RNA silencing: Cell entry of exosomes as a novel
paradigm for therapeutic RNA delivery
• Recent data on how exosomes have evolved a specific
route for cell entry
• Improvements in subcellular targeting to directly improve
the therapeutic index and thus the clinical viability of
siRNA therapies
• The inefficiencies of some liposomal delivery vehicles in
delivering siRNA to the endoplasmic reticulum
• Next generation RNA delivery vehicles- models to
enhance the targeting of translation and RNA silencing
sites
Nicole Meisner-Kober, Senior Investigator, RNA Biology,
Novartis Institutes for Biomedical Research
14.50 Afternoon Tea
15.20 RNAi and tumor microenvironment:
pre-clinical and clinical evidence
• The case of silencing mutated KRAS
• Prolonged drug release continuously affects the
tumor microenvironment
• RNAi and immunotherapy
Amotz Shemi, CEO, Silenseed
16.00 Individualized mRNA-based cancer immunotherapy
• Regulatory framework for mRNA-based therapeutics
• Optimisation of mRNA for therapeutic application
• GMP manufacturing of mRNA
• The IVAC MUTANOME concept
• First clinical experience with mRNAs in individualised
cancer immunotherapy
Andreas Kuhn, Vice President RNA Biochemistry &
Manufacturing, BioNTech
16.40 Chairman’s Closing Remarks and Close of Day One
ENHANCING THE DELIVERY OF RNA THERAPEUTICS
www.therapeutics-rna.com
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 on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk
Official Publications Supported by
DRUG FORMULATION
*Subject to final confirmation

3. RNA Therapeutics
Day Two | Thursday 23rd February 2017
Alternatively fax your registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
MARKETING OPPORTUNITIES
Want to know how you can get involved? Interested in promoting your services to this market?
Contact – Teri Arri SMi Marketing on +44 (0) 20 7827 6162 or email: tarri@smi-online.co.uk
CHALLENGES FACED IN GAINING APPROVAL
08.30 Registration & Coffee
09.00 Chairman’s Opening Remarks
Heinrich Haas, Vice President RNA Formulation & Drug
Delivery, BioNTech RNA Pharmaceuticals
OPENING ADDRESS:
09.10 Antisense in the fourth decade:
Third generation antisense (3GA) fulfilling the promise
• First report of antisense in 1978 and the journey so far
• Various mechanisms of action of oligonucleotides have
been reported
• Agonists and Antagonists of TLRs are in clinical
development
• The design of 3GA is optimized to realize the potential
of antisense
Sudhir Agrawal, President of Research, Idera
Pharmaceuticals
09.50 Interacting with the medicines regulator on RNAi therapies
• A brief description of the regulatory environment
• How to interact with the regulator
• Some thoughts on reporting clinical data
John Johnston, Clinical Assessor, Biologicals and
Biotechnology Unit, MHRA
10.30 Morning Coffee
11.00 Nano-therapeutics; the best of all possible worlds?
• New delivery systems for RNAi effectors usually result from
horizontal not vertical developments
• The need for vertical developments in nanoparticle
mediated RNAi effector delivery
• A rational framework for vertical developments
• The potential power of Image guidance in nanoparticle
mediated RNAi effector delivery
Andrew David Miller, Professor of Organic Chemistry &
Chemical Biology, CSO, King’s College London,
KP Therapeutics Ltd
11.40 Protecting your innovative products and methods:
How to patent RNA therapeutics
• Update on relevant developments at the European
Patent Office (EPO) and elsewhere
• Understanding the ‘problem-and-solution’ approach
applied by the EPO
• How to draft successful patent specifications for RNA
therapeutics
Mark Chapman, Partner, Life Sciences,
Carpmaels & Ransford
12.20 Networking Lunch
KEYNOTE ADDRESS
13.30 Intravenously injectable RNA Nanomedicines as Cancer
Vaccines: Case Study with RNA-Lipoplexes
• Proceedings in development of nucleic acid based
nanomedicines
• RNA delivery technologies: challenges and opportunities
• mRNA for cancer vaccination
• Formulation development and preclinical proof-of-
concept for RNA-lipoplex formulations
• GMP manufacturing and clinical testing of RNA-lipoplex
products
Heinrich Haas, Vice President RNA Formulation & Drug
Delivery, BioNTech RNA Pharmaceuticals
14.10 The challenges and lessons learnt about the delivery of
small and large nucleic acids to cancers
• Formulation of siRNA vs plasmid
• mRNA delivery to tumours
• Identification of key quality attributes and correlation to
in vitro/in vivo efficacy
• Key challenges for development and manufacturing
therapeutics
Sanyogitta Puri, Associate Principal Scientist, AstraZeneca
14.50 Afternoon Tea
15.20 Protecting the kidney: using siRNA targeting p53 to prevent
and ameliorate acute kidney injury and delayed graft
function
• Taking advantage of the natural distribution of siRNA to
renal tissue
• The therapeutic approach of temporary p53 inhibition to
prevent acute tissue damage
• Challenges encountered along the journey to phase III
clinical trials
• Detailed results from a large Phase II clinical trial aimed
at prevention of Delayed Graft Function in patient
receiving kidney transplant from deceased donors
Shai Erlich, Chief Medical Officer & president USA,
Quark Pharmaceuticals Inc
CLOSING KEYNOTE
16.00 Spherical Nucleic Acids:
Structures for topical gene regulation
• Spherical Nucleic Acids (SNAs) are Three-dimensional
arrangements of nucleic acids
• SNAs effectively enter cells and tissues through
Scavenger Receptors
• Local, topical application of SNAs have allowed for early
clinical trials
• A Phase I microplaque study in patients with psoriasis was
completed successfully and will be discussed
David Giljohann, CEO, Exicure
16.40 Chairman’s Closing Remarks and Close of Day Two
ADVANCEMENTS IN CLINICAL TRIALS

4. Precision Nano-therapeutics: Minding the
gap in delivery from preclinical studies to
clinical success
The need for functional delivery of RNAi effectors to target
cells is a primary problem that is holding back the field of
RNAi therapeutics. Nanoparticles, molecular conjugates,
and other delivery technologies have their place but are
they really working well enough? If not why not? And if they
do appear to be working, what will it actually take to realise
clinical success? This workshop is designed to help attendees
appreciate the magnitude of the gap that must be bridged
between academic-preclinical studies and the needs of the
pharma industrial for success in RNAi therapeutics.
Reasons to attend:
• Confused about RNAi delivery technologies?
• Confused how to move from bench to clinic?
• Intimidated by regulations and regulators?
• Baffled by target product profiles and integrated
development plans?
Programme
08.30 Registration and coffee
09.00 Session 1: Where are we really with functional
RNAi effector delivery?
• Do RNAi effectors need a delivery system in vivo?
• What delivery systems are most biocompatible?
• How effective are delivery systems today?
• Where should the state of the art be
developed?
09.50 Session 2: Bridging the gap from lab preparation
to kg manufacture
• The importance of cGMP manufacture
• What are key problems in scale-up
manufacture?
10.30 Morning coffee
11.00 Session 3: Integrated development
plans-nonclinical studies to clinic
• Developing a target product profile
• Mapping out an integrated development plan
• Addressing regulatory issues at all stages of
development
11.45 Session 4: Improving on the status quo
• How close are we to routine RNAi therapeutics?
• What are critical problems to address going
forward?
12.20 Closing remarks
12.30 End of workshop
About the workshop leader:
Prof Miller is a leading chemist expert in the understanding
and exploitation of molecular mechanisms in biology.
The overall goal of his academic research has been and
continues to be the design and creation of advanced
therapeutics and diagnostics that address unmet medical
need in the management of chronic diseases. From 1990-
2010, Prof Miller was a member of academic staff in the
Chemistry Department of Imperial College London (UK)
where he became full Professor of Organic Chemistry &
Chemical Biology in 2002, plus founding Director of the
Imperial College Genetic Therapies Centre from 1998.
Since 2010, Prof Miller has been based at King’s College
London from where he has been developing his career
as an academic entrepreneur, this year founding KP
Therapeutics Ltd. The KP Therapeutics Ltd Bio-Nano
pipeline of potential products in discovery & development
all derives from the best of Prof Miller’s personal and
collaborative academic research work over the past two
decades, involving a research spend of at least £20 million.
About the organisation:
KP Therapeutics Ltd is an innovative biopharma company
that addresses areas of unmet medical need in chronic
diseases (such as cancer) by means of its Precision
Therapeutics approaches (PTAs) for diagnosis and
treatment. The company (founded April 2016) benefits
from a management team experienced in all aspects
of drug discovery and development, a global network
for research (involving centres of scientific excellence in
emerging and developed countries), and its pioneering
Bio-Nano discovery & development pipeline.
Leader: Professor Andrew David Miller, Professor of
Organic Chemistry & Chemical Biology, CSO,
King’s College London, KP Therapeutics Ltd
HALF-DAY PRE-CONFERENCE WORKSHOP
Tuesday 21st February 2017
08.30 – 12.30
Copthorne Tara Hotel, London, UK

5. 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 on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk
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VENUE Copthorne Tara Hotel, Scarsdale Place, Kensington, London W8 5SY London
RNA THERAPEUTICS
Conference: Wednesday 22nd & Thursday 23rd February 2017, Copthorne Tara Hotel, London, UK
Workshop: Tuesday 21st February 2017, London, UK
4 WAYS TO REGISTER
www.therapeutics-rna.com
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