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 WORKSHOPTuesday 21st February 2017, Copthorne Tara Hotel, London, UK

REGISTER BY 31ST OCTOBER AND SAVE £400REGISTER BY 3OTH NOVEMBER AND SAVE £200REGISTER 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 Offi cer & President USA, Quark Pharmaceuticals Inc

PAST ATTENDEE TESTIMONIALS:

“Overall very good organisation/speakers” GSK “The conference was very well organised, interesting talks.”2016 Delegate

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 fi eld

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 diffi cult 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 specifi c route for cell entry

• Improvements in subcellular targeting to directly improve the therapeutic index and thus the clinical viability of siRNA therapies

• The ineffi ciencies 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 OPPORTUNITIESSMi 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 specifi c to your industry. Should you wish to join the increasing number of companies benefi ting from sponsoring our conferences please call:

Alia Malick on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk

Offi cial Publications Supported by

DRUG FORMULATION

*Subject to fi nal confi rmation

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 OPPORTUNITIESWant 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) fulfi lling 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 Offi ce (EPO) and elsewhere

• Understanding the ‘problem-and-solution’ approach applied by the EPO

• How to draft successful patent specifi cations for RNA therapeutics

Mark Chapman, Partner, Life Sciences, Carpmaels & Ransford

12.20 Networking Lunch

KEYNOTE ADDRESS13.30 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 • Identifi cation of key quality attributes and correlation to

in vitro/in vivo effi cacy • 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 Offi cer & president USA, Quark Pharmaceuticals Inc

CLOSING KEYNOTE16.00 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

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 fi eld 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? • Baffl ed by target product profi les and integrated

development plans?

Programme08.30 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 profi le • 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) benefi ts from a management team experienced in all aspects of drug discovery and development, a global network for research (involving centres of scientifi c 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 WORKSHOPTuesday 21st February 2017

08.30 – 12.30Copthorne Tara Hotel, London, UK

Sponsorship and Exhibition OpportunitiesSMi 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 specifi c to your industry.

Should you wish to join the increasing number of companies benefi ting from sponsoring our conferences please call: Alia Malick on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk

OCTOBER

European Market Access, Pricing & Reimbursement in Pharma10th - 11th October 2016London, UK

Orphan Drugs19th - 20th October 2016London, UK NOVEMBER

Superbugs & Superdrugs USA14th - 15th November 2016New Jersey, USA

Biosimilars USA16th - 17th November 2016New Jersey, USA

DECEMBER

Cold Chain Distribution12th - 13th December 2016London, UK

JANUARY

Pharmaceutical Microbiology18th - 19th January 2017London, UK

Social Media In The Pharma Industry18th - 19th January 2017London, UK

Pre-Filled Syringes Europe18th - 19th January 2017London, UK FEBRUARY

Parallel Trade6th - 7th February 2017London, UK

3D Cell Culture22nd - 23rd February 2017London, UK

RNA Therapeutics22nd - 23rd February 2017London, UK MARCH

Superbugs & Superdrugs - A Focus on Antibacterials20th - 21st March 2017London, UK

Paediatric Clinical Trials20th - 21st March 2017London, UK

Drug Discovery27th - 28th March 2017London, UK

Asthma & COPD29th - 30th March 2017London, UK APRIL

Controlled Release3rd - 4th April 2017London, UK

Adaptive Designs3rd - 4th April 2017London, UK

Pre-Filled Syringes East Coast26th - 27th April 2017Boston, USA MAY

Lyophilisation8th - 9th May 2017London, UK

Orphan Drugs Europe15th - 16th May 2017Berlin, Germany

Pharma Logistics and Supply Chain18th - 19th May 2017London, UK

Pain Therapeutics22nd - 23rd May 2017London, UK

Highly Potent Active Pharmaceutical Ingredients22nd - 23rd May 2017London, UK JUNE

Pre-Filled Syringes West Coast5th - 6th June 2017San Diego, USA

Microbiology USA8th - 9th June 2017San Diego, USA

ADMET12th - 13th June 2017London, UK

Immunogenicity12th - 13th June 2017London, UK

BioBanking14th - 15th June 2017London, UK

Molecular Diagnostics 14th - 15th JuneLondon, UK JULY

Allergies6th - 7th July 2017London, UK

Peptides6th - 7th July 2017London, 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 REGISTERwww.therapeutics-rna.com

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