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RNA Therapeutics
20 February - 21 February 2019
RNA Therapeutics

SAE Media Group announces the 10th Annual RNA Therapeutics Conference in London on February 20th - 21st 2019.


The field of RNA therapeutics is rapidly expanding, and the potential for using RNA drugs for personalised medicines and immunotherapy, as well as to address genetic, infectious and chronic diseases will ensure the continued development of RNA therapeutics for years to come.


Join us in February 2019, as SAE Media Group’s RNA Therapeutics Conference brings together industry experts from leading RNA therapeutics companies to discuss the challenges for clinical translation of RNA-based therapeutics, with an emphasis on recent advances in delivery technologies, and present an overview of the applications of RNA-based drugs for modulation of gene and protein expression, and genome editing.
 

 

Discover the clinical progress of Spherical Nucleic Acids at Exicure

Understand Sanofi’s strategies to deliver therapeutic oligonucleotides across biobarriers

Learn from ProQR how to plan for oligonucleotide supply from personalized medicine to large scale markets

Explore the development of small activating RNA – from bench to bedside with MiNA Therapeutics

Gain insight into the discovery and development of self-amplifying mRNA vaccines at GSK
 

FEATURED SPEAKERS

Ali Murad

Ali Murad

Director, Alnylam Pharmaceuticals UK & Ireland
Andre Gerber

Andre Gerber

Professor, RNA Biology, University Of Surrey
David  Blakey

David Blakey

Chief Scientific Officer, MiNA Therapeutics
David Giljohann

David Giljohann

CEO, Exicure Inc.
Ekkehard Leberer

Ekkehard Leberer

Senior Director , Sanofi-Aventis Deutschland GmbH
Hans Kistemaker

Hans Kistemaker

Associate Director, ProQR
Henrich Haas

Henrich Haas

Vice President RNA Formulation & Drug Delivery, BioNTech RNA Pharmaceuticals
Ingmar Hoerr

Ingmar Hoerr

Founder and Chairman of the Supervisory Board, CureVac Ag
Jeffrey Ulmer

Jeffrey Ulmer

Head Preclinical R&D, GSK
Markus  Mandler

Markus Mandler

Chief Scientific Officer, Accanis Biotech F&E GmbH & Co KG
Paul Agris

Paul Agris

Consulting Professor, Department of Medicine, Duke University School of Medicine
Shalini  Andersson

Shalini Andersson

Senior Director and Head of New Modalities, AstraZeneca

Ali Murad

Director, Alnylam Pharmaceuticals UK & Ireland
Ali Murad

Ali leads medical activities for Alnylam Pharmaceutical’s amyloidosis franchise in the UK & Ireland. Alnylam is leading the translation of RNA interference into a new class of innovative medicines.

Prior to Alnylam, Ali held leadership roles in the UK and internationally at Novartis. Before this, he was an Engagement Manager in McKinsey & Company’s London office, where he worked with pharmaceutical companies on strategic projects.

Ali is a physician and has previously worked in the NHS, both as a clinician and research fellow. His medical degree is from the University of Oxford, where he was a Hobson Mann scholar.
 

Andre Gerber

Professor, RNA Biology, University Of Surrey
Andre Gerber

André studied Biochemistry at the University of Zurich and obtained a PhD in Cell Biology from the University of Basel for his work on adenosine deaminases acting on RNA. He completed post-doctoral studies at Stanford University, where he started to explore RNA-protein interactions on a global scale. During 2004-2011, he was an independent research group leader at the ETH Zurich, further investigating RNA-protein interaction networks with genomics and proteomics means in different model organisms. Since 2012, he is Professor of RNA biology at the Faculty of Health and Medical Sciences, University of Surrey, UK.

Bob Brown

Chief Scientific Officer, Senior Vice President, Dicerna Pharmaceuticals Inc.
Bob Brown

Christian Plank

Managing Director, CSO, Ethris GmbH
Christian Plank

Christian Plank is co-founder and chief technology officer of Ethris GmbH located in the Munich metropolitan area in Germany. Christian Plank holds a PhD in biochemistry from the University of Vienna, Austria. After a postdoctoral fellowship with Prof. Francis C. Szoka at the University of California, San Francisco, he returned to Europe to become a professor at the Technical University of Munich, Germany. He has been a principal investigator and coordinator of numerous grant projects that have focused on non-viral nucleic acid delivery and nanomagnetic drug targeting. He is a member of the Munich Center of Nanosciences (CeNS) and of the "Nanosystems Initiative Munich" (NIM) cluster of excellence.

Christoph Rosenbohm

Head of Discovery Operations, Roche Innovation Center
Christoph  Rosenbohm

As Head of Synthesis & Optimization, RMR Christoph is responsible for leading the Oligonucleotides synthesis team and two Lead Design & Synthesis teams and as a member of the RMR-LT he participates in defining and setting the strategy for RMR.

Christoph holds a Master of Science and a Ph.D. in Organic Chemistry from the University of Southern Denmark (the lab of Prof. Jesper Wengel - one of the inventors of LNA). After his PhD, Christoph worked as a post-doctoral fellow at the Danish Technical Univerisity. In addition Christoph holds an executive MBA in Technology, Market and Organisation from Copenhagen Business School.

Christoph started his biotech career in Cureon as chemist with the task of synthesizing the LNA monomers. The company was later merged with Pantheco A/S to create Santaris Pharma A/S in 2003, where he become group leader for the chemistry team. Later the group was joined with CMC, Bioinformatics, PK and Pharmacolgy to form the Research Operation Department for which he became the director. A position he held until the acquisition by Roche in 2014.
 

Daniel Zucker

Business Development Manager, NOF Europe GmbH
Daniel Zucker

Daniel Zucker completed a Ph.D. at the field of drug delivery and biochemistry at the Hebrew University of Jerusalem in Israel in 2010. Next, he went for a postdoc at the Technical University of Denmark, at the Centre for Nanomedicine and Theranostics. Afterwards, he worked as a senior scientist for RNA delivery at BioNTech RNA Pharmaceuticals GmbH in Germany. Currently, he is a business development manager for drug delivery systems at NOF Europe GmbH in Germany.
During his career, he worked on lipid and polymer based nanoparticles and gather experise in reseach, formulation development, regulatory affairs, and business development.
Daniel is a co-author of 7 peer-reviewed articles, 3 patent applications, and 1 book in the field of drug delivery.
 

David Blakey

Chief Scientific Officer, MiNA Therapeutics
David  Blakey

I have a degree in Biochemistry from Oxford University, a PhD from London University and then worked as a Post Doc at the ICRF in London. In 1987 I joined AstraZeneca where I championed the company’s move into therapeutic antibodies and led the collaboration that developed Imfinzi.
 

I was appointed Chief Scientist in Oncology in 2008 and in my last 4-5 years at AstraZeneca I led a global cross therapy area team on oligonucleotide-based therapies. In October 2015, continuing my career in Oligonucleotide Therapeutics, I joined MiNA Therapeutics as their CSO and lead research on their small activating RNA platform.
 

David Giljohann

CEO, Exicure Inc.
David Giljohann

Dr. Giljohann has served as CEO of Exicure since 2013. Dr. Giljohann obtained his Ph.D. in 2009 from Northwestern University under the direction of Dr. Chad A. Mirkin where he developed oligonucleotide-modified nanoparticles, including NanoFlare™, and Spherical Nucleic Acid (SNA™) constructs. Dr. Giljohann has contributed to over 25 manuscripts and over 100 patents and applications.

Ekkehard Leberer

Senior Director , Sanofi-Aventis Deutschland GmbH
Ekkehard Leberer

Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986). He conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada, and then became a Professor of Biochemistry at the University of Konstanz, Germany (1992). He is currently responsible for R&D Alliance Management at Sanofi, and is the Scientific Managing Director of the Innovative Medicine Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes (www.compact-research.org).
Since joining Hoechst Marion Roussel in 1998, Dr. Leberer carried out various managing roles in this company, Sanofi’s predecessor companies and Sanofi itself, including responsibilities in functional genomics, biological sciences and external innovation for oligonucleotide-based therapeutics. He has also served as Head of Biotechnology Germany and a member of the Scientific Review Committee of Aventis Pharma Germany.
Prior to joining pharmaceutical industry, Dr. Leberer served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. His research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the principal discoverer of the p21 activated protein kinase (PAK) family of cell signalling proteins and of novel virulence-inducing genes in pathogenic fungi. He is co-author of more than 60 publications in prestigious peer-reviewed journals including Nature and Science.

 

Hans Kistemaker

Associate Director, ProQR
Hans Kistemaker

Hans Kistemaker studied chemistry at the University of Groningen. He performed his bachelor and master research internships under the supervision of Nobel laureate Prof. Dr. Ben Feringa. He then moved to the bio-organic synthesis group of Prof. Dr. Hermen Overkleeft and Prof. Dr. Gijs van der Marel at the University of Leiden, where he performed his PhD research under supervision of Dr. Dima Filippov. He graduated with honors on his research titled ‘Synthesis of well-defined ADP-Ribosylated biomolecules’. He joined ProQR Therapeutics, a drug development company focusing on severe genetic disorders, in 2015 and he is now an Associate Director within the CMC department responsible for all the chemistry and Drug Substance manufacturing activities within ProQR.

Henrich Haas

Vice President RNA Formulation & Drug Delivery, BioNTech RNA Pharmaceuticals
Henrich Haas

Heinrich Haas has more than 20 years of experience in academic research and industrial pharmaceutical development. After he received his Ph.D. in physical chemistry, Dr. Haas researched lipid membranes and organized biomolecular systems. His professional focus is on colloidal/nanoparticulate formulations for targeted drug delivery with therapeutic and diagnostic applications. Before joining BioNTech RNA Pharmaceuticals GmbH (former Ribological GmbH), he was responsible for a variety of projects in biopharmaceutical research and development, ranging from the exploration of novel colloidal therapeutic and diagnostic carriers to up-scaling and development of market-compliant manufacturing methods for liposome products. After joining BioNTech RNA Pharmaceuticals GmbH in 2010, he helped build the formulation development and analytics unit, which develops formulations for delivery of RNA and small molecules. He has an active record of publications in peer-reviewed journals and patent applications in the field of drug delivery.

Ingmar Hoerr

Founder and Chairman of the Supervisory Board, CureVac Ag
Ingmar Hoerr

CEO and co-founder of the biopharmaceutical company CureVac AG, incorporated in 2000, is Dr. Ingmar Hoerr. His entrepreneurship was motivated by a surprising discovery during his doctoral research at the University of Tuebingen. Experiments conducted for his research showed that the mRNA molecule class can be injected into tissue directly and is capable of expressing genes into proteins effectively as well as generating a strong specific immune response, contrary to what had previously been believed. From this key discovery, the founders built up a company that was the first ever to treat a human subject with an mRNA drug and that is now a leading German “unicorn” in the research and development of mRNA-based drugs.


Ingmar Hoerr received his PhD from the University of Tübingen and his MBA from Danube University, Krems, Austria. He currently advises the European Commission as member of the High Level Group of Innovators in developing a European Innovation Council and is juror of the founder prize Weconomy.
 

Jeffrey Ulmer

Head Preclinical R&D, GSK
Jeffrey Ulmer

Dr. Jeffrey Ulmer received his B.Sc. with honors from the Department of Chemistry at the University of Regina and was the recipient of the Merit Award of the Society of Chemical Industry of Canada. He received his Ph.D. in biochemistry from McGill University and completed his postdoctoral training in the laboratory of Nobel laureate Dr. George Palade in the Department of Cell Biology at Yale University School of Medicine. At Merck Research Laboratories, Chiron Corporation and Novartis Vaccines he conducted seminal studies on nucleic acid vaccines and novel vaccine adjuvants/delivery systems. He has published over 200 scientific articles and is an inventor on 11 patents. Dr. Ulmer is on the editorial boards of Expert Opinion on Biological Therapy, Human Vaccines, and Expert Review of Vaccines, and serves on several external scientific advisory boards and committees. During his 28 year career in vaccines R&D, his leadership roles and responsibilities have included: Platform Technology Leader (DNA, RNA vaccines), Project Leader (Tuberculosis, SARS), Department Head (Immunology & Cell Biology), US Site Head for Research, and Global Function Head (External Research). He is currently Head, Preclinical R&D, GSK Vaccines, where he leads a diverse team of scientists in the discovery and development of viral and bacterial vaccines, and technology innovation.

Kai Wilkens

Senior Director Europe ACD, Advanced Cell Diagnostics SRL
Kai Wilkens

Dr. Kai Wilkens completed his Biology studies and PhD thesis in Molecular Biology in Bochum working in the field of Gene Expression Control. He did clinical research for a couple of years and after that held different positions in several Life Science companies pioneering innovative technologies and mainly enabling new approaches in translational and clinical research. These included MWG Biotech, Panomics/Affymetrix, Mesoscale Discovery and now Advanced Cell Diagnostics, a Bio-Techne brand.

Kirsty Wydenbach

Deputy Unit Manager, Clinical Trials Unit, MHRA
Kirsty Wydenbach

Having joined the MHRA in 2009, Kirsty is a Senior Medical Assessor and the Deputy Unit Manager in the Clinical Trials Unit. She has been involved in the UK regulation of clinical trials across all therapy areas and all phases of development, including trials for chemical and biological products, Advanced Therapy Products and many first-in-man studies. She has also been involved in European discussions aiming to establish an EU harmonised approach to clinical trials, particularly for Developmental Safety Update Reports (DSURs) and Reference Safety Information (RSI). More recent EU priorities have included the new Clinical Trial Regulation and Kirsty currently sits on the safety subgroup of the Clinical Trials Facilitation Group (CTFG) that is working on how safety reporting aspects will be implemented. She is also the co-chair for safety for the EU NTC Curriculum for European regulatory agency assessors and was an EMA expert for the update of the First-in-Human guideline. Other recent work has included collaboration with external industry groups and regulators regarding adaptive and novel trial designs: she is an author on the upcoming ECMC consensus paper on complex innovative trials and a contributor to the EU CTFG discussions on adaptive design trials.

Markus Mandler

Chief Scientific Officer, Accanis Biotech F&E GmbH & Co KG
Markus  Mandler

Dr. Mandler is CSO of ACCANIS, bringing with him over 10 years of collaborative research and management experience spanning academia and industry. He completed his PhD at Boehringer Ingelheim´s Institute of Molecular Pathology in Vienna in 2003 working on skin development and especially focused on appendages including hair, feathers, and teeth. Dr. Mandler is the former head of the Neurodegeneration Department at AFFiRiS where he was responsible for developing several immunotherapeutics from preclinic into early clinical development. He has authored numerous publications in high impact journals, several patents and has received multiple national and international research grants for his work.

Paul Agris

Consulting Professor, Department of Medicine, Duke University School of Medicine
Paul Agris

Paul Agris founded The RNA Institute, a global alliance of top RNA biomedical researchers creating tools for RNA applications. Formerly, Agris was Head of NCSU’s Department of Biochemistry and founded and led the RNA Society of North Carolina for more than a decade. He was an Assistant, Associate and full Professor in the Division of Biological Sciences and Department of Medicine at the University of Missouri-Columbia. Agris received his graduate degree from MIT, and was a postdoctoral fellow at Yale. For over 50 years, he has conducted funded RNA science leading to publications, patents and the founding of two companies.

Shalini Andersson

Senior Director and Head of New Modalities, AstraZeneca
Shalini  Andersson

Shalini is currently the Senior Director and Head of New Therapeutic Modalities, Cardiovascular, Renal and Metabolic Innovative Medicines unit (CVRM IMED) in AstraZeneca.
Shalini has broad experience in Pharmaceutical research as well as strategic and management roles. Shalini drives several collaborations with external partners including an open innovation collaboration with Ionis Pharmaceuticals and the delivery of modified RNA to man with Moderna Therapeutics. Shalini is also involved in several academic collaborations across the globe.
Prior to this role, Shalini held several leadership roles within CVRM Drug Metabolism & Pharmacokinetics and in Medicinal Chemistry.

Shalini received her PhD in 1989 at the University of Linköping, Sweden and has held various research and teaching positions at the University of Linköping prior to moving to industry. She is the author or co-author of over 35 peer reviewed articles, 2 book chapters and 4 patents.
 

Steffen Panzner

Managing Director, Lipocalyx
Steffen Panzner

Dr. Panzner is an expert in oligonucleotide delivery; author of more than 35 patent families and numerous scientific publications covering novel concepts for oligonucleotide delivery such as the Viromer® (virus-mimicking polymer) and the Smarticles® (amphoteric liposome) technology. Products based on these technologies are in phase 1 clinical trials or earlier stages of development.
 

Dr. Panzner was Founder and CSO of Novosom AG between 1999 and 2010 and is Founder and CEO of Lipocalyx GmbH which started 2011. He also is a consultant and CMC expert for pharma and biotech companies as well as for VC firms.
 

sponsors

Conference agenda

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8:30

Registration & Coffee

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9:00

Chairman's Opening Remarks

Ekkehard Leberer, Senior Director , Sanofi-Aventis Deutschland GmbH

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9:10

Spherical nucleic acids: Clinical progress

David Giljohann, CEO, Exicure Inc.

• Localization to endosome permits large numbers of SNA’s to be delivered with low toxicity
• SNAs can be used to knockdown genes or interest, or activate immune pathways productively
      1. AST-008 TLR9 agonist clinical trial progress will be discussed
      2. XCUR-17, Topical antisense in psoriasis will be discussed

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9:50

LNA therapeutics: Recent developments & discovery concepts

Christoph Rosenbohm, Head of Discovery Operations, Roche Innovation Center

• Locked nucleic acid (LNA) has over the last fifteen years become a leading RNA therapeutic modality and is today one important reason for the high interest in oligonucleotide medicines
• The combination of LNA nucleosides and novel phosphorothioate chemistries enables bespoke tailoring of LNA drug designs
• Such new drug designs and new discovery concepts have improved RNA therapeutic drug discovery significantly
• The presentation will review the recent developments, and also show how this modality is constantly improved by novel nucleotide modifications, designs, modelling and delivery technologies

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10:30

Morning Coffee

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11:00

Update on the regulatory environment for clinical trials

Kirsty Wydenbach, Deputy Unit Manager, Clinical Trials Unit, MHRA

• Novel trial designs
• Overcoming common governing pitfalls
• Updates of new and upcoming regulations and how you may be affected
 

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11:40

Towards mRNA therapeutics for skin diseases

Markus Mandler , Chief Scientific Officer, Accanis Biotech F&E GmbH & Co KG

• Skin offers various opportunities with regard to development of mRNA-based therapeutics: diseases with validated molecular targets/attractive markets and direct access facilitating quantification of mRNA expression/clinical activity
• mRNA is a new drug format capable of exceeding existing protein-based therapeutics.
• ACCANIS develops proprietary IVT-mRNAs addressing validated targets for specific skin conditions
• We systematically modified specific IVT-mRNAs and tested the most interesting ones in ex vivo and in vivo skin model systems varying formulation and delivery
 

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12:20

Networking Lunch

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13:20

SPOTLIGHT: Self-amplifying mRNA vaccines

Jeffrey Ulmer, Head Preclinical R&D, GSK

• Induction of broad and potent immune responses
• Synthetic production methods
• Amenable to rapid response against newly emerging infectious diseases
• Potential disruptive technology to streamline vaccine discovery and development

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14:00

Pharmaco-Kinetics of RNA Therapy monitored in situ

Kai Wilkens, Senior Director Europe ACD, Advanced Cell Diagnostics SRL

  • Single copy detection of any RNA molecule in situ
  • High Specificity: Discriminate between endogenous and codon optimized seq
  • Various sample types – FFPE, fresh & fixed frozen,
  • Various assay formats – chromogenic, fluorescent, multiplex, automated, manual
  • Any species – any target – any tissue
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    14:40

    Profiling the rearrangement of RNA-binding proteins on cancer-related mRNAs identifies modulators of drug sensitivity

    Andre Gerber, Professor, RNA Biology, University Of Surrey

    • Cisplatin induces post-transcriptional regulation of a cancer-related mRNA
    • New biochemical approach to capture particular mRNAs and bound proteins
    • RNA-binding proteins modulate drug sensitivity of cancer cells

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    15:20

    Innovative mRNA vaccines

    Ingmar Hoerr, Founder and Chairman of the Supervisory Board, CureVac Ag

    • Introducing CureVac’s RNActive® prophylactic vaccine technology
    • Development of mRNA-based vaccines designed to prevent influenza and malaria infection
    • mRNA platform enables the cost-effective and fast manufacturing of vaccines to prevent diseases
    • Advantages include: flexible applications, rapid production and potential to address several global vaccine challenges

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    16:00

    Afternoon Tea

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    16:30

    Review of the engineering and application of a novel antibiotic against a unique RNA target that prevents resistance

    Paul Agris, Consulting Professor, Department of Medicine, Duke University School of Medicine

    • Target selection unique to Gram positive pathogens
    • Screening tools for putative small molecule, RNA binding antibiotics against Gram positive pathogens and their biofilms
    • Target prevents emergence of resistance
    • Low Cytotoxicity and Toxicity 
     

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    17:10

    Development of the novel GalXC™ RNAi therapeutics for the treatment of chronic liver diseases

    Bob Brown

    Bob Brown, Chief Scientific Officer, Senior Vice President, Dicerna Pharmaceuticals Inc.

    • GalXC technology platform uses RNAi to inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of selected genes
    • Potential to treat diseases by silencing previously inaccessible drug targets
    • Development of novel RNAi therapies for non-alcoholic steatohepatitis and other chronic liver diseases

     

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    17:50

    Chairman’s Closing Remarks and Close of Day One

    Ekkehard Leberer, Senior Director , Sanofi-Aventis Deutschland GmbH

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    8:30

    Registration & Coffee

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    9:00

    Chair's Opening Remarks

    Shalini Andersson, Senior Director and Head of New Modalities, AstraZeneca

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    9:10

    Development of formulations for delivery of RNA and small molecules

    Henrich Haas, Vice President RNA Formulation & Drug Delivery, BioNTech RNA Pharmaceuticals

    • Technical challenges of systemic delivery of vaccine antigens into dendritic cells (DCs)
    • DCs can be targeted precisely and effectively in vivo using intravenously administered RNA-lipoplexes (RNA-LPX)
    • Using well-known lipid carriers by optimally adjusting net charge, without the need for functionalization of particles with molecular ligands
    • Future Perspectives

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    9:50

    Delivery of RNA in vivo using Redox responsive and ionizable lipids (COATOSOME® SS series)

    Daniel Zucker, Business Development Manager, NOF Europe GmbH

    •             Reducing toxicity of lipids by designing biodegradable structures.
    •             Improving transfection of RNA by using lipids that degrade themselves fast in the intracellular environment.
    •             RNA delivery in vivo to specific organs by lipid nanoparticles.
    •             Experimental comparison between COATOSOME® SS series and other lipids for RNA transfection
     

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    10:30

    Morning Coffee

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    11:00

    Advances in targeted delivery of anti-sense oligonucleotide therapeutics to pancreatic ß-cells for regenerative approaches in Type 2 Diabetes

    Shalini Andersson, Senior Director and Head of New Modalities, AstraZeneca

    • Advantages of targeted delivery approaches based on GLP1-conjugation
    • GLP1 peptide structure activity relationship with respect to gene knock-down in pancreatic β-cells
    • Biodistribution of GLP1-conjugated and unconjugated anti-sense oligonucleotides(ASOs) and therapeutic window
    • Possibility to utilise GLP1-ASO conjugates for regenerative approaches in Type 2 Diabetes

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    11:40

    Delivering therapeutic oligonucleotides across biobarriers: Opportunities and challenges in drug development

    Ekkehard Leberer, Senior Director , Sanofi-Aventis Deutschland GmbH

    • Oligonucleotides have a huge pharmacological potential but their widespread therapeutic application is limited due to pharmacokinetic and drug disposition limitations at both the tissue and cellular level.
    • The presentation will address these delivery limitations and summarize the work of a European consortium (Innovative Medicines Initiative COMPACT Consortium) of pharma companies and academic partners to improve nanocarrier-based delivery technologies that can overcome these limitations.

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    12:20

    Delivering mRNA using polymeric an liposomal carriers

    Steffen Panzner, Managing Director, Lipocalyx

  • Presentation and differentiation of polymer and liposome technologies
  • Parameters for optimization towards in vivo applicability
  • Data from preclinical models
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    12:40

    Networking Lunch

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    13:40

    Small activating RNA – from bench to bedside

    David Blakey, Chief Scientific Officer, MiNA Therapeutics

    • Novel MOA of small activating RNAs to upregulate gene expression
    • Preclinical studies demonstrating MOA and therapeutic activity
    • Clinical experience with MTL-CEBPA – the first saRNA in the clinic. 
     

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    14:20

    mRNA therapeutics for localised aplications

    Christian Plank, Managing Director, CSO, Ethris GmbH

    • Stabilized non-immunogenic mRNA (SNIM®RNA)
    • Formulations for mRNA delivery
    • Pulmonary delivery
    • Transcript-activated implants for bone regeneration
     

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    15:30

    Planning for oligonucleotide supply: personalized medicine to large scale markets

    Hans Kistemaker, Associate Director, ProQR

    • Securing oligonucleotide drug substance supply
    • Early vs late phase
    • Small scale (ultra-orphan) vs large scale markets

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    16:00

    Afternoon Tea

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    16:30

    Case study: RNAi therapy for hATTR amyloidosis

    Ali Murad, Director, Alnylam Pharmaceuticals UK & Ireland

    • hATTR amyloidosis is an orphan condition caused by the deposition of mutant TTR protein into tissues, particularly the nervous system and cardiac system. It causes severe morbidity and mortality
    • Reducing the production of mutant TTR protein through RNA based therapeutics presents a novel way to treat this disease and this talk will focus on the role of an RNAi based therapy
     

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    16:50

    Chair’s Closing Remarks and Close of Day Two

    Shalini Andersson, Senior Director and Head of New Modalities, AstraZeneca


    Director
    Alnylam Pharmaceuticals UK & Ireland
    Professor, RNA Biology
    University Of Surrey
    Chief Scientific Officer, Senior Vice President
    Dicerna Pharmaceuticals Inc.
    Managing Director, CSO
    Ethris GmbH
    Head of Discovery Operations
    Roche Innovation Center
    Business Development Manager
    NOF Europe GmbH
    Chief Scientific Officer
    MiNA Therapeutics
    CEO
    Exicure Inc.
    Senior Director
    Sanofi-Aventis Deutschland GmbH
    Associate Director
    ProQR
    Vice President RNA Formulation & Drug Delivery
    BioNTech RNA Pharmaceuticals
    Founder and Chairman of the Supervisory Board
    CureVac Ag
    Head Preclinical R&D
    GSK
    Senior Director Europe ACD
    Advanced Cell Diagnostics SRL
    Deputy Unit Manager, Clinical Trials Unit
    MHRA
    Chief Scientific Officer
    Accanis Biotech F&E GmbH & Co KG
    Consulting Professor, Department of Medicine
    Duke University School of Medicine
    Senior Director and Head of New Modalities
    AstraZeneca
    Managing Director
    Lipocalyx

    Sponsors and Exhibitors

    Official Media Partner

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    2019 ATTENDEE LIST

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    [PAST PRESENTATION] - BIONTECH

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    [PAST PRESENTATION] - ProQR

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    Brochure

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    2019 AGENDA

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    [CHAIR LETTER]

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    INTERVIEW WITH Andre Gerber, Professor, RNA Biology, University Of Surrey

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    CUREVAC FOUNDER & Chairman of the Supervisory Board INTERVIEW

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    RNA Symposium Flyer

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    [PAST SPEAKER INTERVIEW] - EKKEHARD LEBERER, SANOFI

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    [PAST SPEAKER INTERVIEW] - Professor Nagy Habib, MiNA Therapeutics

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    [PAST SPEAKER INTERVIEW] - Dr. Noreen Henig, ProQR Therapeutics

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    [2019 speaker list]

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    Sponsors and Exhibitors


    ChemGenes

    Sponsors and Exhibitors
    http://www.chemgenes.com

    ChemGenes, an ISO 9001 certified company established in 1981, is the industry leader in manufacturing oligonucleotide synthesis reagents and has consistently provided the highest quality phosphoramidites and solid supports in the market. Our facility, just outside of Boston/Cambridge Massachusetts USA, is setup for bulk manufacturing of therapeutic grade phosphoramidite and solid support DNA/RNA synthesis products for GMP grade oligonucleotide manufacturing. Additionally, ChemGenes carries the widest variety of modified phosphoramidites and supports currently used in oligonucleotide synthesis including Microarray Technology, Oligonucleotide Therapeutics, Oligonucleotide Based Probes and other areas of Nucleic Acid research. ChemGenes remains devoted to providing you with invaluable customer service and comprehensive technical support.


    Intertek

    Sponsors and Exhibitors
    http://www.intertek.com

    With nearly 20 years’ experience in regulatory-driven, phase-appropriate application of orthogonal analytical techniques for oligonucleotides and a heritage of supporting GMP manufacturing and regulatory submissions, our expertise can support your development from initial characterization and CMC, through to bioanalytical services and long-term quality control including Method Development/Validation, Identity/Assay, GMP Release/Stability Testing, Physico-chemical, Structure, Sequence & Chain Length Characterisation, Cell-Based Assays, Purity & Impurities Analysis, ICH Stability Studies and Extractables / Leachables.


    NOF Corporation

    Sponsors and Exhibitors
    http://nofeurope.com/

    NOF CORPORATION, the leading commercial supplier of drug delivery products through R&D to commercial scale, provides activated PEGs for protein modification, single molecular PEGs for ADCs, Polysorbate 80 for formulation of biological medicines, PEG lipids and ionizable lipids for lipid nanoparticle (LNP) formulations. NOF's LNP platform composed of COATSOME® SS Series, novel biodegradable ionizable lipid, demonstrates superior endosomal escape and provides high protein expression with extremely low systemic toxicity. The platform can be applied to gene delivery, gene editing, nucleic acid vaccine and in vitro transfection. It is capable of encapsulating pDNA, siRNA and mRNA, and delivering these to various organs and tissues. NOF’s LNP platform is becoming adopted in clinical planning by NOF collaborators. We would like to include URL for our website. https://nofeurope.com/

    Media Partners


    The RNA Institute

    Official Media Partner
    https://www.rna.albany.edu/

    Mission: The Institute is focused on disseminating the technologies, tools, methods and materials through collaborations with researchers in a breadth of disciplines and foci critical to the advancement of scientific capabilities. RNA is ephemeral and challenging to work with but its potential in medical sciences and human health is invaluable.

    Media Partners


    World Pharma News

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    http://www.worldpharmanews.com/



    American Pharmaceutical Review

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    http://www.americanpharmaceuticalreview.com

    Start receiving your free copy today. American Pharmaceutical Review is a review of the drug pipeline from the late discovery phase through manufacturing. American Pharmaceutical Review prides itself on having the most relevant, unbiased and informative editorial in the industry. You will find that all of our editorial is highly technical and written by government agencies, consultants, academics, and large pharmaceuticals companies. American Pharmaceutical Review covers several key topics that are important to drug production:Separations and purification, Drug Delivery, Biopharmaceutical Processing, Biopharmaceutical Development, Formulation development, Manufacturing, Microbiology, Instrumentation, Spectroscopy


    Journal for Clinical Studies – Your Resource for Multisite Studies & Emerging Markets

    Supporters
    http://www.JforCS.com

    Published bi-monthly.JCS is a unique journal, dedicated to providing information to the global pharmaceutical, biotechnology, medical devices and contract research organisations. JCS details practical and theoretical operational procedures, challenges, validatory and regulatory guidelines when conducting trials on a multisite basis and particularly within the emerging markets, naïve patient population, and remote access areas. JCS provides country by country objectives and uniquely brings you experiences in therapeutic areas of Liver diseases, kidney diseases, insect borne diseases, malnutrition and under nutrition. JCS is led by a strong editorial advisory board sourced out for their experiences; you will get the most practical insight for your global studies. JCS invites you to join us, write for us, feature your experiences with us, advertise your capabilities with us, and ask our advisory board for suggestions and guidelines. Let us make health care available to all.


    GBI

    Supporters
    http://www.gbihealth.com/

    GBI is an information, applications, and services company focused on the healthcare industry. Leveraging data from across the healthcare value chain, GBI creates web and mobile applications that enable individuals and organizations to make better decisions and communicate more effectively.


    Pharma Journalist

    Supporters
    http://www.pharmajournalist.com

    Pharma Journalist is a product of Kellen Media. Pharma Journalist is one of the leading website covering the need of global Pharmaceutical Industry. Articles like latest news, trends, analysis, market report, press releases, whitepapers, case studies, etc. published on pharmajournalist.com helps Industry professionals and decision makers to stay on the top of this fast-paced industry. Pharma Journalist aims of providing fast and informative articles to its readers and subscribers.


    Biocompare

    Supporters
    http://www.biocompare.com

    Biocompare.com is the leading resource for up-to-date product information, product reviews, and new technologies for life scientists. Biocompare combines an in-depth knowledge of life science products and new technologies with the power of the Internet to offer scientists the most dynamic, relevant, and innovative media-based marketplace for life science information.


    Labcompare

    Supporters
    http://www.labcompare.com

    Labcompare is a product of American Laboratory, and is already included in the AL logo on your website. I have attached the standalone Labcompare logo and info is below, but if you do add this one separately, could you exchange the American Lab logo with the one attached? For laboratory managers and researcher scientists across analytical chemistry and applied sciences who need to find product solutions, advice, and access scientific tools. Labcompare provides an industry-leading, comprehensive, online resource of product information to support the laboratory’s needs. Labcompare supports every step of the buying journey by focusing on core and new product technology, supplies, tips, and buyer’s guides.


    Bentham Science

    Supporters
    http://www.benthamscience.com/

    Bentham Science Publishers is a major STM journal publisher of 130 plus print and online journal. Out of these, 40 journals have already registered good IMPACT FACTORS as per Journal Citation Reports® 2017. These titles have extensive readership mostly in Europe and North America. For a detailed profile please visit our website at http://www.benthamscience.com. Besides, Bentham Science publishes eBooks in all areas of Science, Technology and Medicine. Our eBooks provide professionals, academicians, corporate researchers, graduates and undergraduates worldwide with the most current information in their subject areas of interest. Our eBooks are also available in the ePub and Kindle formats besides the PDF edition here http://ebooks.benthamscience.com/. Bentham is offering attendees of this conference discounts on its publication. For more information click here


    SelectScience

    Supporters
    http://www.selectscience.net/register?utm_source=Media-Partner&utm_medium=Website&utm_campaign=SMI

    SelectScience is an independent, online information resource for the worldwide scientific community, and the home of trusted information for laboratory scientists. Discover the latest drug discovery and development technologies, products and techniques with product reviews, videos, application notes and news articles. Become a member for free today.


    CLocate

    Supporters
    http://www.clocate.com

    Clocate.com is a leading international directory for worldwide conferences and exhibitions. Clocate.com is equipped with a unique and comprehensive search that helps you find easily any event in any category or location. Each event includes detailed information, like, description, dates, location, map, prices, link to the official event's website and more...


    Future Science Group

    Supporters
    http://bit.ly/2A5IXar

    [Therapeutic Delivery Journal] Therapeutic Delivery offers both academics and industry professionals broad and comprehensive coverage of all aspects of the evolving drug delivery field. The journal features cutting-edge research and reviews, opinion articles on the hottest topics and a monthly industry update, written by key players in the therapeutic delivery community.


    Pharmavision

    Supporters
    http://www.pharmavision.co.uk

    PharmaVision offers a consultancy service providing independent pharmaceutical thematic research to the healthcare industry, the investment community and competitive intelligence specialists. We perform due diligence research and provide expert commentary in healthcare. Our reports combine scientific analysis in drug delivery, R&D technologies and pharmaceutical products including patient modelling, product/technology forecasts and market trends evaluation.


    Contract Biotechnology

    Supporters
    http://www.contract-biotechnology.com

    Contract-Biotechnology.com is a web-based platform for laboratory outsourcing solutions. It is an online R&D matching tool that connects Scientifics and service and product providers worldwide. The platform Contract-Biotechnology.com would help you in the process of finding the right partner saving time and money, because with one single and secure application you would be able to receive multiple quotes quickly, keeping your contact information confidential. Contract-Biotechnology.com is the new Collaboration Network Model for Discovery Research and Development. Contract-Biotechnology.com team has extensive experience working for pharmaceuticals, biotechs, universities and academic research institutes and can help you addressing your key gaps.


    Labiotech.eu

    Supporters
    https://goo.gl/DRE2Gp

    Labiotech.eu is the leading digital media covering the European Biotech industry. Over 100,000 monthly visitors use it to keep an eye on the business and innovations in biotechnology. Hope you'll enjoy reading our stories!


    Pharmalicensing

    Supporters
    http://www.pharmalicensing.com

    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.


    Labsave

    Supporters
    http://www.labsave.com

    Labsave is the leading savings website for Laboratory Equipment and Lab Supplies. We work closely with the top suppliers in the world to bring you the best products at the most competitive prices. We are constantly striving to secure the biggest and most exclusive offers that you won’t find anywhere else! http://www.labsave.com/


    Swiss Biotech Association

    Supporters
    http://www.swissbiotech.org/

    The Swiss Biotech Association (SBA) is the national industry association for biotechnology, including pharmaceuticals, diagnostics, agriculture, food, cosmetics, environmental biotechnology, and specialty chemicals. Members are companies active in modern biotechnology, such as R&D, Production, Marketing and Sales, Finance, Services and Consulting. SBA provides a networking platform for Life Science clusters, academic and federal institutions the like. Founded in March 1998, the Association grows steadily.


    Drug Discovery Today

    Supporters
    http://www.drugdiscoverytoday.com/

    Drug Discovery Today covers the whole of the preclinical drug discovery process. The reviews are cutting edge, written by experts in their respective fields and cover all aspects of drug discovery from genomic and proteomic approaches, computational drug design, medicinal chemistry and the translation of these sciences to therapies.


    International Pharmaceutical Industry (IPI)

    Supporters
    http://ipimediaworld.com/

    IPI – International Pharmaceutical Industry IPI – was established to fill in the void for effective marketing and communication between all stakeholders in the Life sciences sector globally. Edited by our carefully selected editorial advisory panel, and extensive research network, IPI provides a proven supportive means of communication to the pharmaceutical, bio pharmaceutical, nutraceutical and medical devices industry the latest in research and technology innovations, regulatory guidelines, marketing and communication strategies which will enable them to be more efficient, bring products to market faster, reduce cost and make healthcare accessible to all.


    Drug Target Review

    Supporters
    http://www.drugtargetreview.com

    Drug Target Review’s quarterly magazine, website and annual events program provides high quality content with peer-written articles that are submitted exclusively by the world’s most respected scientists in their field. This attracts a committed base of readers, users and delegates made up of senior decision-makers from the life science and drug discovery industries at the top pharmaceutical companies, as well as academics and scientists from the top research institutes across the globe.


    pharmaphorum

    Supporters
    http://www.pharmaphorum.com

    pharmaphorum drives innovation within the pharmaceutical industry, by bringing healthcare together through a suite of media services that help produce and disseminate thought leadership, combined with an online platform for communicating messages to a global audience.Visit www.pharmaphorum.com


    Medical Device Network

    Supporters
    http://www.medicaldevice-network.com/

    Medicaldevice-network.com is your one stop for information on the medical device industry, covering everything along the supply chain from biomaterials and materials solutions to electronics, packaging, motors and motion control, outsourcing and more. Through our international team of journalists, we cover diagnosis, prevention, monitoring, standards, treatment and technology – all you need as a professional in the medical industry to stay abreast of the latest developments in your field.


    Gate2Biotech

    Supporters
    http://www.gate2biotech.com

    Gate2Biotech is a portal that unites biotechnological community in Central Europe. It covers all the news in the field of biotechnology. Thanks to the the portal the companies engaged in the field can easily search for research institutions and other partners to solve technical problems they are facing and here they can also present their services offer to potential partners from the Czech Republic and abroad. Gate2Biotech portal serves as a bridge connecting scientific and commercial sector. It incorporates offices and support organizations dedicated to promoting transfer of innovative technologies into a unified communication platform of Czech and international biotechnologies. As a mediator of information it draws attention to biotechnologies and support of their application and transfer into practice. It also functions as a tool for encouraging non-professionals (especially scientists and students) towards innovative business or applied research.


    Biosave

    Supporters
    http://www.Biosave.com

    Biosave.com brings together the best Life Science promotions, product releases, featured products, publications, videos and events from 100’s of leading Life Science suppliers. Updated 24 hours a day, Biosave is constantly uploading special promotions and exciting product news that you won’t find anywhere else. Our goal is to provide our users with the most exclusive product information to help reach that all important purchasing decision.


    Absave

    Supporters
    http://www.absave.com

    Absave.com is the leading savings website for Antibodies and Immunological Products. Search our extensive database of Antibodies, Kits, Proteins & Peptides, Reagents, etc. to find the best savings!


    Gene Therapy Net

    Supporters
    http://www.genetherapynet.com

    Gene Therapy Net is the information resource for basic and clinical research in gene therapy, and the site serves as a network in the exchange of gene therapy information and breaking news items. Visitors can keep track of the latest scientific papers, conference announcements, gene therapy jobs, regulations and guidelines


    Technology Networks

    Supporters
    http://go.technologynetworks.com/subscribe-to-newsletters

    Founded in 2000, Technology Networks is established as the leading news provider for life science and drug discovery professionals. In addition, we provide unique content including webcasts, videos, application notes and posters from recent conferences. Our portfolio now includes around 30 focussed scientific communities, all of which are accessible free of charge within TechnologyNetworks.com

    Copthorne Tara Hotel

    Scarsdale Place
    Kensington
    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.

     

    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
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