RNA therapeutics is arguably at the forefront of the treatment for a number of complex medical disorders. From Oncological implications to cardiovascular disease, the investigation into the mechanisms which inhibit specific gene transcription could provide the key to the eradication of a number of diseases. SMi’s 2013 RNA Therapeutics conference will look to discuss and share some of the latest findings and research in the field of RNAi technologies and the delivery mechanisms by which these therapies are administered to their target tissues.

A selection of distinguished speakers will look to provide the latest insights into some of the most promising technologies in RNAi development and delivery as well as addressing some of the current IP and regulatory challenges faced in the development and protection of these technologies.

Developing an understanding in how Epigenetic mechanisms can be used to alter gene expression is undoubtedly a prime target for the development of compounds which offer the ability to alter the processes of translation of a peptide. SMi’s 2013 RNA Therapeutics conference offers the chance to meet the industry leaders in this field and provides the perfect opportunity to network and identify new strategies for RNAi development and application.

The field of genetic medicine and the process of manipulating gene expression is still relatively new and as such evolves continuously at an almost alarming rate. SMi’s 2013 event will look to address the timeliest, ground breaking research that has been carried out throughout 2012 and into 2013 and address the future implications of these findings. Testimonials from our highly successful event in 2012 include:

“Enjoyable conference” – Thermo Fisher Scientific
“Great Overview of Novel Approaches” – GlaxoSmithKline
“Very exciting Data” – Novartis Pharma

  • Identify new delivery technologies which enhance the efficacy and bioavailability of your compounds for the appropriate tissue
  • Discover new approaches in the field of RNAi technologies
  • Evaluate the current landscape with respect to the current technologies and how these are being applied
  • Network with some of the industry leaders within the field of RNA therapeutics
  • Overcome the challenges of developing RNAi technologies and the mechanisms by which they alter gene expression

SMi's RNA Therapeutics event is a must attend for the following:

Formulation Scientist

Research assistants

Research Scientists

Clincal scientist

Biomedical scientists



Post Doctoral Fellows

Clinical Investigator


Medicinal Chemists


AstraZeneca; AuraSense Therapeutics LLC; Avidity IP; Centro Hospitalar de Tras-os-Montes e Alto Douro; Chemconsilium; Cliniques Universitaires UCL de Mont-Godinne; Covington & Burling Llp; Faculty Of Pharmacy University Lisbon; GlaxoSmithKline; GSK; Imperial College London; Institute of Cancer Research; InteRNA Technologies; Kilburn & Strode; King Saud University; Kings College London; Kings College University; Merck & Co Inc; Octo Plus; Pierre Fabre Medicaments; Queen Mary And Westfield College; Sanofi-Aventis Deutschland GmbH; Silence Therapeutics AG; Sylentis; UCL Institute of Child Health; UMC Utrecht; Universite Paris Sud; University College London; University Of Southampton; University Paris Sud; Weill Cornell Medical College in Qatar; Yale University;

Conference programme

8:30 Registration and Coffee

9:00 Chairmans opening Remarks

David Thurston

David Thurston, Professor of Drug Discovery, Kings College University

9:40 Identification and validation of anti-angiogenic miRNAs with antitumor efficacy after systemic delivery

Raymond Schiffelers

Raymond Schiffelers, Associate Professor Clinical Chemistry, UMC Utrecht

  • We have screened a lentiviral miRNA with> 1100 sequences for antiangiogenic activity in vitro. The effects of the strongest inhibitors were validated in a panel of angiogenesis assays, including proliferation, migration, tube formulation and chorioallantoic membrane assay.
  • The therapeutic efficacy of the most robust miRNA was tested ina  murine model of cancer by local injection and electroporation, showing significant inhibition of tumour growth and reduced vascularization.
  • By formulating the miRNA into an endothelial cell-targeted nanoparticle, we also achieved therapeutic efficacy after systemic administration.
  • Analysis of the transcriptome of angiogenic endothelial cells after transfection of the miRNA showed reduced expression of 7 molecular targets for angiogenesis inhibition for which drug molecules are currently used in clinical trials.


  • 10:20 Morning coffee

    10:50 Targeting transcription factors with DNA interactive small molecules

    Khondaker Miraz Rahman

    Khondaker Miraz Rahman, Lecturer in Medicinal Chemistry, Kings College London

  • Transcription factors are emerging new targets for drug discovery
  • Targeting transcription factors directly can have many advantages compared to targeting upstream kinases
  • Various mechanisms can be used including the inhibition of protein-protein interactions (PPIs) and protein-DNA interaction (PDIs)
  • A number of duplex-DNA and promoter G- Quadruplex-binding agents are being developed to target the PDI interaction, and some examples will be presented
  • 11:30 Quantitation of physiological and cellular barriers to siRNA delivery: applications to PK/PD

    Jerome Hochman

    Jerome Hochman, Senior investigator, Merck & Co Inc

  • Quantitative evaluation of biochemical, physical and physiological barriers which restrict distribution, tissue penetration, cytosolic delivery and RISC loading siRNA in vivo
  • For LNP's compositional changes were reflected in differences in siRNA attrition during different stages of siRNA delivery
  • The ability to identify key barriers to siRNA delivery permits improved mechanistic understanding and focussed optimization of siRNA delivery vehicles
  • 12:10 Lunch

    13:20 Exosomes are natural nanovehicles that can target antigen specifically and deliver dsRNA of choice for physiologic and exquisitely specific small RNA therapy

    Philip Askenase

    Philip Askenase, Professor, Yale University

  • We describe antigen-specific exosomes for the first time that can be coated with light chain antibodies of choice
  • We describe for the first timepassive transfection of exosomes with selected miRNA to deliver specific genetic therapy
  • Together, this enables construction of natural physiological long circulating exosomes that antigen-specifically target cells to deliver specific genetic therapy relevant to many diseases
  • 14:00 Polyalkylcyanoacrylate and nanodiamond nanoparticles for the delivery of oligonucleotides targeting the EWS/Fli-1 oncogene associated to Ewing sarcoma

    Claude Paul Malvy

    Claude Paul Malvy, Director IFSBM Dean of the Medicine Facility, Universite Paris Sud

  • The prevalence of EWS/Fli-1 Oncogene in Ewing Sarcoma patients
  • The need to protect antisense oligonucleotides and siRNA until they reach their EWS/Fli-1 mRNA target
  • Biodegradable and biocompatible polyalkylcyanoacrylate coated with chitozan
  • Developing cationic nanodiamond vectors and evaluating their efficiency for anionic siRNA delivery
  • 14:40 Afternoon tea

    15:00 Update on Unitary Patent and Unified Patent Court reform in Europe

    Nick Bassil

    Nick Bassil, Partner, Kilburn & Strode

  • Status of legislation
  • Relationship to current system
  • Details of main features
  • Strategies for working with new system
  • 15:40 Regulatory Challenges in commercialising Nanotechnology

    Brian Kelly

    Brian Kelly, Associate, Covington & Burling Llp

  • Regulatory Framework
  • Challenges: Classification, testing and risk management
  • Avenues to patient use
  • Reimbursement
  • 16:20 Chairmans closing remarks and end of Day 1

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    George Okafo

    George Okafo, Scientific Director, GSK

    9:10 RNA therapy that improves liver failure and controls tumour burden simultaneously

    Nagy Habib

    Nagy Habib, Professor Department of Surgery & Cancer, Imperial College London

  • We have used a targeted approach for synthesising clinical grade saRNA and applied this to a number of important areas of translational medicine.
  • We have been able to transform isolated autologous adult hematopoetic stem (CD34+ cells) into glucose sensitive insulin secreting cells using MafA-saRNA
  • Using the same approach, we have also been able to show improvement in liver function using rodent studies of hepatocellular carcinoma injected with C/ EBPa saran
  • Surprisingly, we have demonstrated a reduction in tumour burden in vivo
  • 9:50 CD99 antibody bound nanoparticles for the delivery of siRNA targetting the EWS/Fli-1 oncogene

    Jean Remi  Bertrand

    Jean Remi Bertrand, Researcher, University Paris Sud

  • Tumour growth was found to be inhibited by delivering siRNA associated nanoparticles in vivo  in a double targeted approach
  • siRNA targetting EWS/Fli-1 mRNA junction was therefore the first target
  • Nanoparticles targeted towards the highly overexpressed CD99 glycoprotein on Ewing Sarcoma cells to aid delivery of siRNA to tumour cells was the second target approach
  • Gene expression responsible for Ewing Sarcoma was inhibited by 78 + 6% by associating siRNA with CD99 nanoparticles compared with an inhibition of only 41 + 9% achieved with non targeted nanoparticles.
  • 10:30 Morning Coffee

    11:00 Overview of CMC analytical control strategies for Synthetic Oligonucleotide Therapeutics

    George Okafo

    George Okafo, Scientific Director, GSK

  • Regulatory expectations
  • overview of oligonucleotide impurities
  • Analytical methodology- different approaches and how they are used
  • setting specifications at different stages for different types of oligonucleotides
  • 11:40 Development of an RNAi therapeutic on Oncology

    Ansgar  Santel

    Ansgar Santel, Senior Scientist, Silence Therapeutics AG

  • Proprietary delivery technology (AtuPLEX) enables delivery of therapeutic doses of chemically stabilised siRNA into the vascular endothelium as target organ after systemic (intravenous) injection
  • The novel liposomal siRNA formula became implemented in the new investigational drug Atu027 for the development of a novel first-in-class cancer drug
  • Atu027 has shown to enable RNAi mediated knockdown after systemic administration in vivo and demonstrated anticancer and anti-metastatic activity in a preclinical setting and has then been assessed in Phase-1 clinical trials in human subjects with advanced solid tumours


  • 12:20 Networking Lunch

    13:30 siRNA therapeutics for ocular conditions

    Covadonga Paneda

    Covadonga Paneda, R&D Manager, Sylentis

  • SYL040012 a siRNA for the treatment of glaucoma targeting ADRB2
  • Biodistribution and efficacy of SYL040012
  • Preclinical development of SYL040012
  • Clinical status of SYL040012 and other compounds
  • 14:10 Oligonucleotide synthesis for RNAi

    Brian Sproat

    Brian Sproat, Managing Director, Chemconsilium

  • Modern Solid-phase synthesis of oligonucleotides- the pros and cons
  • Purification and analytical methods-making the best choices
  • problems and precautions during annealing
  • Prospects for the future
  • 14:50 AZD9150, a Next Generation Antisense Oligonucleotide targeting STAT3 - preclinical and early clinical experience

    David Blakey

    David Blakey, Chief Scientist, AstraZeneca

  • High affinity next generation  (constrained ethyl) ASOs lead to a robust activity in extra-hepatic tissues including tumours
  • The human-specific STAT3 next generation ASO (AZD9150) demonstrates potent and selective inhibition of target RNA and protein levels in tumours in a broad range of cancer models including human tumour xenografts and several primary patient-derived xenograft models
  • AZD9150 has completed a Phase 1 dose escalation trial incancer patients, has shown encouraging signs of clinical activity and is now in expanded clinical studies including patients with refractory lymphomas
  • 15:30 Afternoon Tea and Close of Conference


    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.




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    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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