SMi Group announces the return of its 8th annual RNA Therapeutics conference to London on the 22nd - 23rd of February 2017. Technology in RNA interference, oligonucleotides and mRNA has revolutionised the way infectious disease, cancer and neurological disorders are treated. It is now possible to target previously ‘un-druggable’ sites, therefore expanding the range of therapeutics that selectively silence genes before the disease develops.

AstraZeneca has added another $140 million to its $1.2 billion investment into Moderna Therapeutics’ mRNA technology*. Consequently continued collaboration between pharmaceutical giants and innovative biotechnology companies, has created considerable funding and buzz around RNA research.

The main challenge in RNA therapeutics continues to be the delivery of RNA based drugs to target sites outside the liver. Join us in exploring the different delivery systems in use and in development such as transportation with nanoparticles and the ability to enhance drug stability.

We will be reviewing clinical trial updates in RNA- based therapeutics and discussing the current regulations involved in getting drug approval. Learn about the main developments currently shaping the industry and how best to tackle challenges facing RNA therapeutics.


  • Overcome challenges in regulation and maximise therapeutics manufacturing by seeing BioNTech up-scale personalized RNA Nanomedicines in cancer Vaccines.
  • Gain practical skills from MiNA Therapeutics on RNA activation and the results of their clinical trials. They will be discussing the impact saRNA is having on patients suffering from liver disease- discover how you too can strengthen your approach to clinical trials.
  • AstraZeneca will be Exploring emerging antisense oligonucleotide drugs and ways in you can improve drug delivery and optimise clinical results.
  • Hear from GSK on the progress of their 5 year IMI COMPACT project and the results of their collaboration with other pharmaceutical companies into the optimisation of oligonucleotide drug delivery.
  • Find out how to strategically plan and execute a successful clinical study with an exclusive update from Exicure on their unique nucleic acid based topical treatment for psoriasis.

Advanced Cell Diagnostics SRL; Affymetrix; Aldevron; Alexion GmbH; Amgen Gmb H; Aptamer group; Avencia Group; Bayer Pharma AG; BioNTech AG; Boehringer Ingelheim; Boehringer Ingelheim Pharma GmbH & Co. KG; Boult Wade Tennant; Covington & Burling Llp; Cristal Therapeutics; CureVac GmbH; Drug Target Review; Erdmann Technologies GmbH; Institute for Research in Biomedicine (IRB Barcelona); K.U.Leuven; Karolinska Institutet; MiNA Therapeutics; ProQR Therapeutics; RCSI Royal College of Surgeons in Ireland,; Rigontec GmbH; Sarepta Therapeutics; Silence Therapeutics GmbH; Silenseed; THERMO FISHER SCIENTIFIC; ThermoFisher; Translational Oncology (TRON); Universita Magna Graecia - Catanzaro; Universite Paris Sud; University of Amsterdam; University Of Twente / 20Med; Vascular and Genomic Center Changhua Christian Hospital;

Conference programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Nagy Habib

Nagy Habib, Professor Department of Surgery & Cancer and Co-founder MiRNA Therapeutics, Imperial College London

9:10 RNA activation and its clinical application

Nagy Habib

Nagy Habib, Professor Department of Surgery & Cancer and Co-founder MiRNA Therapeutics, Imperial College London

  • 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
  • 9:50 Collaborating to deliver macromolecules across biological barriers – a review of IMI COMPACT

    Steve Hood

    Steve Hood, Director, Scinovo, GSK

  • What are the challenges of Oligo Delivery?
  • How do we optimise delivery
  • Who are COMPACT and what have we achieved?
  • 10:30 Morning Coffee

    11:00 Delivery of mRNA using the Viromer® system

    Steffen Panzner

    Steffen Panzner, Managing Director, Lipocalyx

  • A lesson learned from a virus: the Viromer delivery system
  • Formulation aspects of Viromer nanoparticles
  • Viromer: mRNA delivery on multiple routes
  • 11:40 Therapeutic antisense oligonucleotides in oncology

    Mark Edbrooke

    Mark Edbrooke, Senior Principal Scientist, AstraZeneca

  • 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
  • 12:20 Networking Lunch

    13:30 RNA Therapeutics Harnessing the Potential of Locked

    Bo Rode Hansen

    Bo Rode Hansen, Director, Technology and Partnered Discovery, Santaris Pharma A/S

  • 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
  • 14:10 Sites of RNA silencing: Cell entry of exosomes as a novel paradigm for therapeutic RNA delivery

    Nicole  meisner-kober

    Nicole meisner-kober, Senior Investigator, Novartis Institutes for Biomedical Research

  • 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
  • 14:50 Afternoon Tea

    15:20 RNAi and tumor microenvironment: pre-clinical and clinical evidence

    Amotz Shemi

    Amotz Shemi, CEO, Silenseed

  • The case of silencing mutated KRAS
  • Prolonged drug release continuously affects the tumor microenvironment
  • RNAi and immunotherapy
  • 16:00 Individualized mRNA-based cancer immunotherapy

    Andreas Kuhn

    Andreas Kuhn, Vice President RNA Biochemistry & Manufacturing, BioNTech

  • 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
  • 16:40 Chairman’s Closing Remarks and Close of Day One

    Nagy Habib

    Nagy Habib, Professor Department of Surgery & Cancer and Co-founder MiRNA Therapeutics, Imperial College London

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Heinrich Haas

    Heinrich Haas, VP RNA Formulation and Drug Developement , BioNTech AG

    9:10 Antisense in the fourth decade: Third generation antisense (3GA) fulfi lling the promise

    Sudhir Agrawal

    Sudhir Agrawal, President & C S O, Idera Pharmaceuticals

  • 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 that are in clinical development
  • The design of 3GA is optimized to realize the potential of antisense
  • 9:50 Interacting with the medicines regulator on RNAi therapies

    John Johnston

    John Johnston, Clinical Assessor, Biologicals & Biotechnology Unit, MHRA

  • A brief description of the regulatory environment
  • How to interact with the regulator
  • Some thoughts on reporting clinical data
  • 10:30 Morning Coffee

    11:00 Nano-therapeutics; the best of all possible worlds?

    Andrew Miller

    Andrew Miller, CSO, KP Therapeutics Ltd

  • 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
  • 11:40 Protecting your innovative products and methods: How to patent RNA therapeutics

    Mark  Chapman

    Mark Chapman, Partner, Carpmaels & Ransford

  • 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 specifi cations for RNA therapeutics
  • 12:20 Networking Lunch

    13:30 Intravenously injectable RNA Nanomedicines as cancer vaccines: case study with RNA-Lipoplexes

    Heinrich Haas

    Heinrich Haas, VP RNA Formulation and Drug Developement , BioNTech AG

  • 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
  • 14:10 The challenges and lessons learnt about the delivery of small and large nucleic acids to cancers

    Sanyogitta Puri

    Sanyogitta Puri, Associate Prinicipal Scientist, AstraZeneca

  • Formulation of siRNA vs plasmid
  • mRNA delivery to tumours
  • Identification of key quality attributes and correlation to in vitro/in vivo effi cacy
  • Key challenges for development and manufacturing therapeutics
  • 14:50 Afternoon Tea

    15:20 Protecting the kidney: using siRNA targeting p53 to prevent and ameliorate acute kidney injury and delayed graft functio

    Shai Erlich

    Shai Erlich, VP, Pharmaceutical Development, Quark Biotech Inc

  • 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
  • 16:00 Spherical Nucleic Acids:Structures for topical gene regulation

    David Giljohann

    David Giljohann, CEO, Exicure Inc.

  • 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
  • 17:00 Chairman’s Closing Remarks and Close of Day Two


    Official Publication



    Copthorne Tara Hotel

    Scarsdale Place, Kensington, London, United Kingdom

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.



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    Copthorne Tara Hotel

    Scarsdale Place
    London W8 5SR
    United Kingdom

    Copthorne Tara Hotel

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.




    speaker image






    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.


    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.


    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SMI Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.


    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@smi-online.co.uk

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