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RNA Therapeutics
15 February - 16 February 2016
RNA Therapeutics

SAE Media 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/

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

Conference agenda

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8:30

Registration & Coffee

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9:00

Chairman's Opening Remarks

Ryszard Kole

Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

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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.
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    9:50

    A new class of RNA therapeutics – RIG-I

    Christine Schuberth-Wagner

    Christine Schuberth-Wagner, SVP Research, Rigontec GmbH

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    10:30

    Morning Coffee

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    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
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    11:40

    RNA vaccines development and optimising mRNA therapeutics and delivery

    Mustafa Diken

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

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    12:20

    Networking Lunch

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    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
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    14:10

    Keynote Address: Rational design including functionalization of nanoparticles for improved cell delivery

    Christianne Rijcken

    Christianne Rijcken, CSO, Cristal Therapeutics

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    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
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    15:30

    Afternoon Tea

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    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.
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    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

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    17:20

    Chairman’s Closing Remarks and Close of Day One

    Ryszard Kole

    Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

    clock

    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

    Nagy Habib

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

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    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.

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    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

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    10:30

    Morning Coffee

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    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
     

    clock

    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
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    12:20

    Networking Lunch

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    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
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    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
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    14:50

    Afternoon Tea

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    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
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    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
     

    clock

    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


    CEO
    Silenseed
    Head of the Laboratory of Experimental Virology
    University of Amsterdam
    Professor
    Karolinska Institutet
    CSO
    Cristal Therapeutics
    SVP Research
    Rigontec GmbH
    Co-head of research group: Chemical Vectorology of anticancer drugs,
    Gustave Roussy Institute
    CTO
    Aptamer group
    VP RNA Formulation and Drug Developement
    BioNTech AG
    European and UK Patent Attorney
    Boult Wade Tennant
    Senior Director Europe
    Advanced Cell Diagnostics SRL
    Deputy Vice President Immunotherapies and Preclinical Research
    BioNTech AG
    Professor of Surgery, Chairman and Co-Founder
    Imperial College London, MiNA Therapeutics
    Director, Alliance Management
    CureVac
    Distinguished Scientist
    Sarepta Therapeutics
    Associate
    Covington & Burling Llp

    Sponsors and Exhibitors

    Official Media Partner

    Official Publication

    Supporters

    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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    Advanced Cell Diagnostics

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    Take advantage of single copy detection of specific transcripts in the tissue with RNAscope® - validate your RNAi approach within the morphological context for virtually ANY gene from ANY genome in ANY tissue. ACD’s RNAscope® Technology is capable of detecting RNA biomarkers in situ at single molecule sensitivity.

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    RNA Biology

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    RNA Biology, provides a platform for presenting and discussing cutting-edge RNA research. RNA Biology brings together a multidisciplinary community of scientists working within: transcription and splicing, post-transcriptional regulation of gene expression, non-coding RNAs, RNA localization, translation and catalysis by RNA, structural biology, bioinformatics, and RNA in disease and therapy.


    Transcription

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    Pharmalicensing (www.pharmalicensing.com) is the premier biopharmaceutical Open Innovation resource designed for professionals involved with partnering, licensing and business development worldwide. Actively supporting all forms of partnering and in- and out-licensing activities, Pharmalicensing utilizes the unique online Showcase Profiling & Discover services, as well as its renowned Partnering Search service leveraging its global network of industry experts, to enable companies to identify and engage with appropriate partners around the world. Pharmalicensing is actively utilized by many tens of thousands of industry professionals each month. Pharmalicensing is a division of Cognis Group, Inc.


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    Weekly Update newsletter and News Bulletins cover the application of informatics, IT and computer science in biomedical research and drug discovery. As the life sciences become an increasingly quantitative discipline, Bio-IT World provides topical news coverage and analysis of cutting-edge technologies to handle the data deluge in petascale computing and the tools to deliver individualized medicine.

    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. 

    HOTEL BOOKING FORM

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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, SAE Media 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@saemediagroup.com

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    Contact SAE Media Group

    UK Office
    Opening Hours: 9.00 - 17.30 (local time)
    SAE Media Group , Ground Floor, India House, 45 Curlew Street, London, SE1 2ND, United Kingdom
    Tel: +44 (0) 20 7827 6000 Fax: +44 (0) 20 7827 6001
    Website: http://www.smgconferences.com Email: events@saemediagroup.com
    Registered in England - SMi Group Ltd trading as SAE Media Group




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