16 - 17

FEB2015Marriott Regents Park Hotel, London, UK

RNA Therapeutics

@SMIPHARM

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

CHAIRS FOR 2015:• Claude Paul Malvy, Professor,

Universite Paris Sud

• Joerg Kaufmann, Chief Scientific Officer, Silence Therapeutics

KEY SPEAKERS INCLUDE: • Bo Rode Hansen, VP, Drug Discovery &

Alliance, Roche

• Rodger Novak, CEO, CRISPR Therapeutics

• Annemieke Aartsma-Rus, AssociateProfessor, Leiden University Medical Center

• Nagy Habib, Chairman and Co Founder,MiNa Therapeutics

• David Blakey, Chief Scientist, AstraZeneca

From Cell to Sales: Clearing the Path to Market

PLUS AN INTERACTIVE HALF-DAY POST-CONFERENCE WORKSHOPWednesday 18th February, Marriott Regents Park Hotel, London, UK

RNAi therapeutics; appreciating the gap in delivery from preclinical studies to clinical successWorkshop Leaders: Prof Andrew David Miller, Professor of Organic Chemistry & Chemical Biology and CSO, GlobalAcorn Ltd

Terry Wilkins, Yorkshire Forward Professor of Nanomanufacturing Innovation, CEO, NanoManufacturing Institute (NMi), University of Leeds

8.30am – 12.30pm

BUSINESS BENEFITS FOR 2015: • Take away best practice learning on efficient and

targeted efforts to maximise return on investment • Hear key case studies from leading companies and

gain the edge in confronting the challenges ofoligonucleotide R&D

• Discover the latest clinical data in RNAi, ASO and miRNA medicine including the latest RNAactivation approaches

• Review new conjugate/aptamer/lipid/polymer/nanoparticle delivery systems to enhancethe efficacy and bioavailability of compounds

• Update on the state of CRISPR technology fortherapy

• Enhance knowledge on intellectual propertyissues (IP)

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SMi presents the 6th annual conference on…

Sponsored by

Register online at: www.therapeutics-rna.com • Alternatively fax y

RNA Therapeutics Day One | Monday 16th February 2015

8.30 Registration & Coffee

9.00 Chairman's Opening RemarksClaude Paul Malvy, Professor, Universite Paris Sud

RNA THERAPEUTICS

9.10 OPENING KEYNOTERNAi therapeutics: Opportunities, obstacles and solutions• Challenges for developing effective RNAi - based drugs• Research on better delivery systems• Validation in NHPs• Identifying the right targets and indications• Update on clinical status of Atu027Joerg Kaufmann, Chief Scientific Officer, Silence Therapeutics

9.40 RNAi therapeutics for eye conditions• Update on the clinical programme of Bamosiran, an siRNA for

the treatment of glaucoma• New designs and candidates: impact on biodistribution within

the eye• Advantages of RNAi therapeutics in ophthalmologyCovadonga Paneda, R&D Manager, Sylentis

10.10 Morning Coffee

10.40 From Bench to Bedside - The evolution of mirror-imageoligonucleotides as new therapeutic modalities• The benefits of Spiegelmers• Preclinical & clinical data• Overview NOX-E36 (drug for diabetic nephropathy)Sven Klußmann, Co-Founder & Chief Technology Officer,Noxxon Pharma

11.10 RNA activation in therapy• New oligonucleotides as anticancer drugs in several

applications• The use of small activating RNA in the treatment of NASH and

liver failure• A new approach for the treatment of diabetes• RNA aptamers for pancreatic cancerNagy Habib, Chairman and Co-Founder, MiNA Therapeutics

11.40 CRISPR-Cas as a “can opener” for genome editing?First insights into early translational work for human therapeutics • Introduction to CRISPR-Cas - a scientific thriller• What makes CRISPR-Cas special? - Differentiation

regarding other gene editing technologies• Perspective provided on potential for translation into human

medicines - challenges and opportunities• CRISPR-Cas as a role model for personalized medicineRodger Novak, CEO, CRISPR Therapeutics

12.10 Exon skipping in Duchenne Muscular Dystrophy: a tale of twodiseases• Duchenne and Becker Muscular Dystrophy compared• Splice switching using anti-sense oligonucleotides• Learnings from a global clinical programme• Acclerated regulatory paths to approvalGiles Campion, Chief Medical Officer and SVP R&D, ProsensaTherapeutics

12.40 Networking Lunch

1.40 Hepatic targeting and avoidance of oligonucleotide drugs:What is the real story?• Understanding hepatic uptake of large molecule drugs,

including oligonucleotides• Compare uptake activity in different types of liver cells• Insights into uptake receptors and metabolism• A need for new ways of explaining hepatotoxicity caused by

uptake of large molecule drugsBård Smedsrod, Chief Scientific Officer, D'Liver

MODIFICATIONS AND FORMULATIONS

2.10 The use of LNA/UNA-aptamers for anticancer and delivery purposes• Locked nucleic acid (LNA) is a structurally rigid modification that

increases the binding affinity of a modified-oligonucleotide• Compare the synthetic routes to both of these modifications,

contrast the structural features, examine the hybridizationproperties of LNA and UNA modified duplexes

• LNA and UNA are also emerging as versatile modifications foraptamers

Jesper Wengel, Professor, BioNEC Center Director, University ofSouthern Denmark

2.40 Transfection: Viral and synthetic techniques converge • What can be learned from viral delivery?• Convergence between a viral and synthetic system• Development of the Viromer technology• Cell uptake and delivery dataSteffen Panzner, Managing Director, Lipocalyx

3.00 KEYNOTE ADDRESS Delivering macromolecules to the right address - many handsmake light work• Synthesis of drug/polyterpene bioconjugates • Design of the nanoassemblies and their functionalization with

recognition ligands• Cell and tissue imaging• Pharmacological evaluation in vitro (cell culture) and in vivoSteven Hood, Director, Scinovo, GSK

3.30 Afternoon Tea

4.00 Protecting biotech inventions, how much for how little?• Why use a patent to protect a biotech invention?• An underlying principle - The extent of patent

protection should be justified by the technical contribution to the art

• Application of this principle during prosecution of patent applications and post-grant

• Implications for when to file and what to include in your patentapplication

James Legg, Patent Attorney, Boult Wade Tennant

4.30 Biochemically modified messenger RNA encoding nucleases forin vivo gene correction of severe inherited lung diseases• Modified messenger RNA• In vivo treatment• Life-saving efficacy• Limiting off-target effectsMichael Kormann, Group Leader, Pediatric Infectiology andImmunology, Eberhard-Karls-University Tuebingen

5.00 Round Table Discussion - Developing RNA therapeutics for rare diseases• Identifying and facing early stage and

clinical challenges• How to best understand and seizing opportunities

in the market placeGiles Campion, Chief Medical Officer and SVP R&D, ProsensaTherapeutics

5.30 Chairman's Closing Remarks and Close of Day OneClaude Paul Malvy, Professor, Universite Paris Sud

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 andexpand your client base within the context of an independentdiscussion 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

SCIENTIFICSPOTLIGHT

Official Media Partners

PART 1

IP FOCUS

PART 1

your registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711

Day Two | Tuesday 17th February 2015

8.30 Registration & Coffee

9.00 Chairman's Opening RemarksJoerg Kaufmann, Chief Scientific Officer, Silence Therapeutics

RNA THERAPEUTICS

9.10 OPENING ADDRESS: THE STATE OF PLAYRNA Therapeutics: The 3rd major engine of pharmaceutical value creation?• Review of RNA Therapeutics technology• Current state of RNA Therapeutics clinical pipeline• Assessment of RNA Therapeutics pre-clinical pipeline• RNA Therapeutics business and financial market considerationsDirk Haussecker, Consultant, RNA therapeutics

9.40 RNA Therapeutics: Unlocking the potential with locked nucleic acids• What is new after several decades of learnings from developing

RNA therapeutics? • Why has the success rate of RNA therapeutics drug

development been low with very few marketed drugs as result?• What is the most recent thinking on how to increase the

productivity of RNA therapeutics development? • Why has Roche recognized Locked Nucleic Acids as the

preferred chemistry and modality for RNA therapeuticsdevelopment?

Bo Rode Hansen, VP, Drug Discovery & Alliance, Roche

10.10 Networking towards clinical application of antisense mediatedexon skipping• Overview of how exon skipping can be exploited to treat

different diseases with unmet medical need• Discussion of path from proof of concept to clinical trials using

Duchenne muscular dystrophy as a showcase• Discussion of joint efforts to overcome translational challenges

for clinical implementationAnnemieke Aartsma-Rus, Associate Professor, Leiden UniversityMedical Center

10.40 Morning Coffee

11.10 KEYNOTE ADDRESSTackling Intractable targets in oncology - progress towards viabletherapeutic oligonucleotides platforms• Robust activity in tumours has been limited with initial ASO

chemistries• Next generation ASOs demonstrate robust target knock down in

multiple preclinical cancer models including primary tumourexplants and human tumour xenografts

• AZD9150, a constrained ethyl ASO vs STAT3 has completed initialtrials in cancer patients and shown encouraging signs of clinicalactivity

• Preclinical and clinical data will be shared that suggest nextgeneration ASO technology is poised to become a keytherapeutic modality to modulate intractable oncology targets

David Blakey, Chief Scientist, AstraZeneca

11.40 Panel Debate - Collaborative working from academia to big pharma• Preparing to colaborate with big pharma

- do's and dont's• Exploring collaborative models• The role of platform providersJoerg Kaufmann, Chief Scientific Officer, Silence Therapeutics

12.20 Networking Lunch

1.30 In vivo RNAi and functional genomics• The use of siRNAs in vivo to study gene function

in the liver of mouse models• Demonstration of progress in siRNA delivery to

mouse hepatocyte• RNA interference to examine the activities of specific

genes in animal models• Analysis of three different cases of multiple mRNAs

knockdown in studies of endocytosis, hypoxia and HYPPOpathway

Victor Kotelianski, Director of the Centre for Research,Education and Innovation, Skolkovo Institute of Science andTechnology

2.00 KEYNOTE ADDRESSFrom RNA splicing to Eteplirsen trials: a drug for Duchennemuscular dystrophy (DMD)• Invention of RNA splicing modulation • From thalassemia to DMD• Insight in to Eteplirsen induced exon skipping of 51 in

dystrophin pre-mRNA in DMD patients• The results of the continuing trial after 120 weeks of

treatment will be presentedRyszard Kole, Distinguished Scientific, Sarepta Therapeutics

MODIFICATIONS AND FORMULATIONS

2.30 Fusion-oncogene junctions in cancers: Delivery and specific targets for siRNAs• Double targeting for Ewing sarcoma• A new siRNA delivery agent for Ewing cells• Literature review of siRNA and other cancers with fusion

oncogeneClaude Paul Malvy, Professor, Universite Paris Sud

3.00 Afternoon Tea

3.30 Taking siRNA beyond the liver: Multifunctional nano-complexes for targeted siRNA delivery to brain, lung andtumours• Targeted lipid-peptide nanocomplex formulations:

modulation of surface properties for improved targetingspecificity and toxicity profiles

• Self-assembly protocols by microfluidics for improved siRNAnanoparticle homogeneity, stability and storage

• Silencing of therapeutic targets in tumour, lung and brain• Therapeutic efficacy of siRNA nano-complexes by systemic

delivery to tumoursStephen Hart, Professor in Molecular Genetics, UCL

4.00 CLOSING KEYNOTEOligonucleotide therapies for neuromuscular disease• Oligonucleotides and their potential• Review of current data• Explore advance therapies currently availableMatthew Wood, Professor of Neuroscience and AssistantHead of Research, University of Oxford

4.30 Chairman's Closing Remarks and Close of Day TwoJoerg Kaufmann, Chief Scientific Officer, Silence Therapeutics

Interested in promoting your services to this market? Contact Teri Arri, SMi Marketing on +44 (0) 207 827 6162 or email tarri@smi-online.co.uk

SCIENTIFICSPOTLIGHT

PART 2

Supported by

PANEL DEBATE

PART 2

HALF-DAY POST-CONFERENCE WORKSHOPWednesday 18th February, 2015

8.30am – 12.30pmMarriott Regents Park Hotel, London, UK

RNAi therapeutics; appreciating the gap in delivery from preclinical studies to

clinical success

Workshop Leaders: Prof Andrew David Miller, Professor of Organic Chemistry

& Chemical Biology and CSO, GlobalAcorn Ltd Terry Wilkins, Yorkshire Forward Professor of Nanomanufacturing

Innovation, CEO, NanoManufacturing Institute (NMi),University of Leeds

Overview of workshop:The need for functional delivery of RNAi effectors to target cells isa primary problem that is holding back the field of RNAitherapeutics. Nanoparticles, molecular conjugates, and otherdelivery technologies have their place but are they really workingwell enough? If not, why not? And if they do appear to beworking, what will it actually take to realize clinical success? Thisworkshop is designed to help attendees appreciate themagnitude of the gap that must be bridged betweenacademic-preclinial studies and the needs of the pharmaindustrial for success in RNAi therapeutics.

Why should you attend this workshop:This workshop is a must attend for all those puzzled, perplexed orconfused about what it is really going to take to move fromsuccessful RNAi therapy experiments in preclinical models ofhuman disease to effective implementation of RNAi typetherapeutics in clinic.

Programme:

8.30 Registration and coffee

9.00 Opening remarks

9.15 Where are we really with functional RNAi effector delivery?

10.15 Bridging the gap from lab preparation to kg manufacture

10.45 Afternoon tea

11.15 Integrated development plans-nonclinical studies to clinic

11.45 Improving on the status quo

12.15 Closing remarks

12.30 End of workshop

About the workshop leader:Prof Andrew David Miller is a leading chemist expert in theunderstanding and exploitation of molecular mechanisms inbiology. The overall goal of his academic research has been andcontinues to be the design and creation of advancedtherapeutics and diagnostics that address unmet medical needsin the management of chronic diseases. Up until 2010, he was a fulltime academic at Imperial College London, where he was fullProfessor of Organic Chemistry and Chemical Biology since 2002,plus founding Director of the Imperial College Genetic TherapiesCentre from 1998. Since 2010, Prof Miller has been based at King’sCollege London from where he has been developing his career asan academic entrepreneur, founding and running GlobalAcornsince 2011. GlobalAcorn’s Bio-Nano pipeline of products indiscovery and development derives from academic researchcarried out exclusively by Prof Miller and key collaborators over thepast 15 years (research spend at least $30 million).

About GlobalAcorn Ltd:GlobalAcorn Ltd is abiopharmaceutical company,dedicated to CancerNanomedicine, that was founded

to make full use of leading edge nanotechnologies and bio-pharmaceutical agents created by Professor Andrew DavidMiller (King’s College London) with key collaborators.GlobalAcorn Ltd currently discovers and develops advancedtherapeutic, diagnostic, and theranostic products that togetheraddress unmet medical need in the treatment and diagnosis ofcancers. Bio-Nano products in pipeline possess both novelty andwell defined target product profiles. www.globalacorn.com

Sponsored by

Lipocalyx is active in nucleic acid delivery. OurViromers are novel synthetic vectors emulatingviral uptake and endosome escape andprovide a safe and efficient way to deliversiRNA, mRNA or DNA. The company wasfounded in 2011 in Halle(Saale), a Germanbiotech hub. Lipocalyx is offering off-the shelfresearch reagents and development servicesfor the industry. www.lipocalyx.de

Silence Therapeutics plc is a leading RNAinterference (RNAi) or ‘gene silencing’ platformtechnology company which has proprietarydelivery systems. Combined, the RNAi anddelivery platforms enable the development ofmultiple products to multiple drug targetsallowing the development of novel therapeuticsfor diseases with high unmet medical need.

Silence is one of only a handful of listedcompanies globally which has human validatedRNA delivery technology. Its platform is one ofthe safest and most widely tested, with over 400patients dosed and no immune responseobserved so far. Silence’s RNAi platform hasbeen used in three Phase 2 clinical trials, two ofwhich are awaiting results. It also has a robust IPestate protecting its proprietary technology.www.silence-therapeutics.com

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RNA THERAPEUTICS Conference: Monday 16th & Tuesday 17th February 2015, Marriott Hotel Regents Park, London, UK Workshop: Wednesday 18th February 2015, London

4 WAYS TO REGISTERwww.therapeutics-rna.com

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