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SMi Group’s announces the return of its 7th annual RNA Therapeutics conference on the 15th & 16th February 2016.

With the recent collaboration between AstraZeneca,  biologics research and development arm, MedImmune, and Moderna Therapeutics and their hope to discover, co-develop and co-commercialise messenger RNA (mRNA) therapeutic candidates for the treatment of a range of cancers, the field of RNA is rapidly changing.
 

The 2016 RNA Therapeutics conference will hone in on some key developments currently shaping the industry, and putting the spotlight on messenger RNA-based Therapeutics.

We will review clinical trial updates in oncology, cardiovascular injury, and ophthalmology, and open the floor for discussion to review existing and emerging concepts in delivery systems, oligonucleotides, aptamer-conjugates as well as messenger RNA therapeutics.

The biggest challenge in enabling RNAi therapy is transporting RNA to target sites outside the liver, and so having targeted delivery systems, which can optimise bioavailability and controllable kinetics remains an issue. However, with exciting concepts such as polymeric nanoparticles and viral vectors as a new class of carriers existing on the horizon, the conference will look at the future of these technologies.
 

Moreover, messenger RNA (mRNA) is attracting considerable interest for its ability to restore gene expression and correct protein production. Thus, we will anticipate what new avenues will open up in gene- based therapies.

https://www.cambridgenetwork.co.uk/news/astrazeneca-and-moderna-therapeutics-announce-new-collaboration/

New for 2016: 

  • Applications and delivery strategies for mRNA therapeutics
  • Building your aptamer: Modifiable building blocks to enhance cell uptake and delivery
  • Assessing pharmacokinetics and translation
  • Upregulation of gene expression miRNA

 

 

Discover exciting developments of mRNA-based therapeutics and delivery systems
Discuss clinical developments and take away key lessons for future developments
Understand the pharmacokinetic considerations of oligonucleotides and RNA therapies
Learn and confront challenges affecting targeted delivery and overcoming biological barriers

 


 

   

Affymetrix; Alios Biopharma; AstraZeneca; Avencia Group; Biopharm Insight; BioPharmaLogic Ltd.; Boehringer Ingelheim Pharma GmbH & Co. KG; Boult Wade Tennant; CRISPR Therapeutics; Eberhard-Karls-University Tuebingen; Galderma France; GlaxoSmithKline; GlobalAcorn Ltd; GSK; Gustave Roussy (CNRS UMR 8203); Institut Scientifique De Sante Publique Louis; InteRNA Technologies; Isarna Therapeutics GmbH; Janssen Pharmaceutica NV; Leeds University; Leiden University Medical Center; Lipocalyx; MHRA; MiNA Therapeutics; Mirna Therapeutics, Inc.; Noxxon Pharma AG; RCSI Royal College of Surgeons in Ireland,; ReNeuron Group Plc; RNA therapeutics; Royal College Of Surgeons In Ireland; Santaris Pharma A/S; Sarepta Therapeutics; Sheffield Hallam University; Silence Therapeutics; Silence Therapeutics AG; Silence-Therapeutics; Skoltech; Sofinnova Partners; Sylentis; Technology Networks; The University of Southampton; THERALDIA CONSULTING LTD.; Tromso University; UCL; Universite Paris Sud; University College Cork; University of Southern Denmark; University Of Twente; University Paris Sud;

Conference programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Ryszard Kole

Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

9:10 Pre-clinical and clinical experience with oligonucleotide drugs

Ryszard Kole

Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

  • Oligonucleotides and siRNAs went through ups and downs during their development as RNA therapeutics.
  • Since the marketing of Fomivirsen for CMV retinitis, Kynamro (mipomersen) as a treatment for hypercholesterolemia adds only a second oligonucleotide to the existing pharmacopeia.
  • It is anticipated that two new oligonucleotide drugs for the treatment of Duchenne muscular dystrophy will soon be evaluated by FDA.
  • 9:50 A new class of RNA therapeutics – RIG-I

    Christine Schuberth-Wagner

    Christine Schuberth-Wagner, SVP Research, Rigontec GmbH

    10:30 Morning Coffee

    11:00 Sponsored session: Single Copy Detection of Specific Transcripts in the Tissue

    Kai Wilkens

    Kai Wilkens, Senior Director Europe, Advanced Cell Diagnostics SRL

    RNAi related RNAscope applications:

  • Tissue specific assessment of efficacy and validation of RNAi approaches
  • Rapid Biomarker development and validation all the way to CDx
  • Drug safety aspects and off-target effects in tissue samples
  • Transfer from cell culture to animal models to clinical
  • 11:40 RNA vaccines development and optimising mRNA therapeutics and delivery

    Mustafa Diken

    Mustafa Diken, Deputy Vice President Immunotherapies and Preclinical Research , BioNTech AG

    12:20 Networking Lunch

    13:30 CRISPR-Cas9 can inhibit HIV-1 replication but also facilitate virus escape

    Ben Berkhout

    Ben Berkhout, Head of the Laboratory of Experimental Virology , University of Amsterdam

  • Describing the emergence of viral escape mutants following CRISPR-Cas9 treatment
  • The effect of NHEJ-associated indels as a result of CRISPR-Cas9-medited gene editing of HIV-1 genes
  • Study of multiple gRNA targets and the correlation between the degree of target sequence conservation
  • 14:10 Keynote Address: Rational design including functionalization of nanoparticles for improved cell delivery

    Christianne Rijcken

    Christianne Rijcken, CSO, Cristal Therapeutics

    14:50 Biotech 2.0 – Medical revolution through natural mRNA

    Nigel Horscroft

    Nigel Horscroft, Director, Alliance Management, CureVac

  • mRNA as  a “data carrier” for a healthy message
  • mRNA technology platform – vaccines (RNActive®) & therapeutics (RNArt®)
  • Pre-clinical and clinical results
  • 15:30 Afternoon Tea

    16:00 Regulating nano therapeutics and commercial interests

    Sarah  Cowlishaw

    Sarah Cowlishaw, Associate , Covington & Burling Llp

  • Nanomedicine or device: Challenging the regulatory pathway and status
  • Developing Risk Management Plans: Appropriate governance and special considerations for data and safety
  • Determining intended use and therapeutic side effects: How are liabilities treated?
  • Companion diagnostics and monitoring treatment effects.
  • 16:40 Panel discussion: Pushing towards and clinical success and market authorisation

  • Comparative review of delivery systems and striving beyond the liver
  • Addressing liabilities and side effects of nanoparticles
  • Advancements and insights in by-passing physiological barriers between the route of administration to the target
  • Balancing ethical concerns and regulatory challenges on the road to market
  • How can the regulatory framework be improved to adapt with the evolution of nanotechnology?
  • Nagy Habib

    Nagy Habib, Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics

    Nigel Horscroft

    Nigel Horscroft, Director, Alliance Management, CureVac

    Ryszard Kole

    Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

    17:20 Chairman’s Closing Remarks and Close of Day One

    Ryszard Kole

    Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Nagy Habib

    Nagy Habib, Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics

    9:10 OPENING ADDRESS: RNA Activation with small activating RNA: liver application

    Nagy Habib

    Nagy Habib, Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics

    RNA activation using small oligonucleotide is an emerging technology that allows genetic up regulation and protein expression without the use of gene therapy. This new technique relies on the use of a double strand oligonucleotide that targets both the promoter and enhancer region and leads to increased expression of messenger RNA in most cells. 

    We exploited this technique to up regulate a liver transcription factor, CEBPa.  This is one of the master regulators of the hepatocytes increases hepatic differentiation.  In vivo administration of saRNA CEBPa led to improvement in liver function in models of liver failure, as well as non-alcoholic steatohepatitis (NASH). This positive data will be the basis for the technique to be applied clinically.

    9:50 Keynote address: Keynote address: How does the functionalisation of nanoparticles improve cell delivery

    Claude Paul Malvy

    Claude Paul Malvy, Co-head of research group: Chemical Vectorology of anticancer drugs, , Gustave Roussy Institute

    Case study: Functionalised nanoparticles associated to oligonucleotides with antibodies and aptamers

    10:30 Morning Coffee

    11:00 Novel mRNA therapeutics: development from basic research to clinical

    Heinrich Haas

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

    •          Therapeutic approaches based on mRNA
    •          Optimization of the mRNA stability and activity
    •          Formulation and delivery strategies for mRNA
    •         Process development and GMP manufacturing of mRNA therapeutics
    •         Clinical applications
     

    11:40 Time to consider Freedom to Operate?

    James Legg

    James Legg, European and UK Patent Attorney , Boult Wade Tennant

  • What is Freedom to Operate?
  • What are the risks?
  • Types of patent infringement and defences available
  • Mitigating the risks - patent transactions and offensive options
  • 12:20 Networking Lunch

    13:30 Novel properties sought from chemically-modified oligonucleotide molecules

    C.I. Edvard Smith

    C.I. Edvard Smith, Professor, Karolinska Institutet

  • Methods of mRNA repair
  • Methods for characterization of endocytosis and intracellular delivery
  • Clinical trial progresses and future perspectives
  • 14:10 Aptamers as delivery vehicles targeting tumour cells

    David Bunka

    David Bunka, CTO , Aptamer group

  • Automated selection of cell targeted aptamers
  • RNAi uses in literature with aptamers
  • 14:50 Afternoon Tea

    15:30 Panel discussion: Decentralising research and more collaboration

    Nagy Habib

    Nagy Habib, Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics

  • Identifying opportunistic areas of collaboration to accelerate clinical development and manufacture
  • One size does not fit all: Combinatorial approaches in RNA therapeutics
  • 16:10 RNAi and tumor microenvironment

    Amotz Shemi

    Amotz Shemi, CEO, Silenseed


    - siRNA against mutated KRAS is effective in pancreatic cancer (clinical evidence) - toward phase 2
    - prolonged contineues release of siRNA modifies tumor microenvironment and accelerates drug distribution
    - the LODER nano is now in development for delivery of RNAi-based drugs
     

    16:50 Chairman’s Closing Remarks and Close of Day Two

    Nagy Habib

    Nagy Habib, Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics

    +

    FEATURED SPEAKERS

    Christianne Rijcken

    Christianne Rijcken

    CSO, Cristal Therapeutics
    Nagy Habib

    Nagy Habib

    Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics
    Ryszard Kole

    Ryszard Kole

    Distinguished Scientist, Sarepta Therapeutics

    Amotz Shemi

    CEO, Silenseed
    Amotz Shemi

    Ben Berkhout

    Head of the Laboratory of Experimental Virology , University of Amsterdam
    Ben Berkhout

    C.I. Edvard Smith

    Professor, Karolinska Institutet
    C.I. Edvard Smith

    Christianne Rijcken

    CSO, Cristal Therapeutics
    Christianne Rijcken

    Christine Schuberth-Wagner

    SVP Research, Rigontec GmbH
    Christine Schuberth-Wagner

    Claude Paul Malvy

    Co-head of research group: Chemical Vectorology of anticancer drugs, , Gustave Roussy Institute
    Claude Paul Malvy

    David Bunka

    CTO , Aptamer group
    David Bunka

    Heinrich Haas

    VP RNA Formulation and Drug Developement , BioNTech AG
    Heinrich Haas

    James Legg

    European and UK Patent Attorney , Boult Wade Tennant
    James Legg

    Kai Wilkens

    Senior Director Europe, Advanced Cell Diagnostics SRL
    Kai Wilkens

    Mustafa Diken

    Deputy Vice President Immunotherapies and Preclinical Research , BioNTech AG
    Mustafa Diken

    Nagy Habib

    Professor of Surgery, Chairman and Co-Founder, Imperial College London, MiNA Therapeutics
    Nagy Habib

    Nigel Horscroft

    Director, Alliance Management, CureVac
    Nigel Horscroft

    Ryszard Kole

    Distinguished Scientist, Sarepta Therapeutics
    Ryszard Kole

    Sarah Cowlishaw

    Associate , Covington & Burling Llp
    Sarah  Cowlishaw

    Sponsors and Exhibitors

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    Official Publication

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    Q&A With Sarepta Therapeutics

    Download

    An Interview with Dr Kai Wilkens, European Senior Director for Sales & Marketing, Advanced Cell Diagnostics

    Download

    Attendee List RNA Therapeutics 2016

    Download

    NEW INTERVIEW with the Gustave Roussy Institute

    Download

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    Pharmaceutical-technology.com is the only website focusing specifically on issues relevant to pharmaceutical professionals working with technology, be it development, engineering, IT or production. Pharmaceutical-technology.com brings you the latest in industry projects and updates, along with the news, views and trends that leading professionals – from senior executives to manufacturing managers and heads of procurement – require to stay on top of their field.


    Inderscience Publishers

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    http://www.inderscience.com

    Inderscience is a dynamic, leading, independent journal publisher. The company disseminates the latest research across the broad fields of science, engineering and technology; management, public and business administration; environment, ecological economics and sustainable development; computing, ICT and internet/web services, and related areas. Inderscience offers over 35 years' experience in publishing and has succeeded in building a substantial collection approaching 400 high-quality peer-reviewed international research journals in both online and print formats. We offer a variety of ways to keep up-to-date with the latest published leading-edge research while our online collection represents a fully-searchable digital archive of around 60,000 articles.


    Bioscreening

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    http://www.bioscreening.com



    Labhoo

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    http://www.labhoo.com



    Antibodies Online

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    http://www.antibodies-online.com

    antibodies-online.com facilitates researchers to find the appropriate antibodies for their respective research project. As an independent and multi-vendor marketplace for research antibodies and thanks to the cooperation with more than 130 different manufacturers, a large selection of Antibodies (over 700,000), ELISA Kits (over 130,000), Secondary Antibodies (17,000), Proteins (130,000), Peptides and Isotype Controls is available. By supporting the Independent Validation Initiative and the Resource Identification Initiative, antibodies-online.com also addresses the important topics of transparent product quality and scientific reproducibility. In addition scientists receive news and detailed information about the individual antibodies from databases such as the PubMed and others.


    International Pharmaceutical Industry

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    http://www.ipimediaworld.com

    IPI gives you an insight into all areas of Biopharm, Medical, Pharmaceutical and Healthcare. IPI provides a proven supportive means of communication to the Pharmaceutical, Bio Pharmaceutical, Nutraceutical and Medical Devices industry, incorporating the latest in research and technology innovations, regulatory guidelines, marketing and communication strategies.


    PharmaVOICE

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    http://www.pharmavoice.com

    PharmaVOICE magazine, and its supporting VIEW publications, provide commentary about the challenges and trends impacting the life-sciences industry, covering a range of issues from molecule through market. PharmaVOICE's more than 41,000 U.S.-based subscribers are also kept abreast of the latest trends through additional media resources, including WebSeminars, Podcasts, Videocasts, and White Papers.


    British Pharmacological Society

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    http://www.bps.ac.uk

    The British Pharmacological Society (BPS) is the primary UK learned society concerned with research into drugs and the way they work. Our members work in academia, industry, regulatory agencies and the health services, and many are medically qualified. The Society covers the whole spectrum of pharmacology, including laboratory, clinical, and toxicological aspects. Clinical pharmacology is the medical speciality dedicated to promoting safe and effective use of medicines for patient benefit. Clinical pharmacologists work as consultants in the NHS and many hold prominent positions in UK Universities.

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    Holiday Inn Kensington Forum

    97 Cromwell Road
    London SW7 4DN
    United Kingdom

    Holiday Inn Kensington Forum

    Holiday Inn Kensington Forum is perfectly situated in one of London’s most luxurious and beautiful areas within South Kensington. 

    The hotel is just 2 minutes walk from Gloucester Road tube station for convenient travel to Hyde Park, London Eye, Tower Bridge plus more of London’s top attractions. There are also easy and direct links to some major transport hubs including Victoria, Kings Cross St Pancras, Paddington and Heathrow. 

    This distinctive hotel in south London has so much to offer to make all guests really feel at home. The latest Holiday Inn relaunch is not just about the new look and feel for the hotel but to offer guests more benefits during their stay including a pillow menu for extra comfort during their sleep and a curved shower rail for more spacious feel. 

    In addition to our 906 rooms, all business guests can take advantage of our meeting and conference facilities including High Speed Internet Access and unlimited Starbucks coffee at The Academy. Our hotel’s professional event planners are on board to help take the hard work and stress away from planning your next event. 

    So whether you in London on business or pleasure, make the Holiday Inn London Kensington Forum your first choice of hotel and book your accommodation for our lowest internet rate guarantee. 

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    WHAT IS CPD?

    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.

    CPD AND PROFESSIONAL INSTITUTES

    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.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    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.

    GLOBAL CPD

    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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    Contact SMi GROUP LTD

    UK Office
    Opening Hours: 9.00 - 17.30 (local time)
    SMi Group Ltd, 2nd Floor South, Harling House, 47-51 Great Suffolk Street, London, SE1 0BS, United Kingdom, Link to Map
    Tel: +44 (0) 20 7827 6000 Fax: +44 (0) 20 7827 6001
    Website: http://www.smi-online.co.uk Email: events@smi-online.co.uk
    Registered in England No: 3779287 VAT No: GB 976 2951 71




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