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The SAE Media Group is proud to announce the
Oligonucleotide Therapeutics and Delivery

21st - 22nd September, 2022 | London, UK
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Oligonucleotide therapeutics - the emerging medicine class - are harnessing the therapeutic benefit of targeting genetic material utilising oligonucleotide conjugates, antisense oligonucleotides (ASOs), mRNA and RNAi. Their market growth is driven by their potential to provide more efficacious and less toxic alternatives to small molecules as well as their ability to affect targets that have been considered “non-druggable”. Despite recent technological advances, achieving efficient oligonucleotide delivery, particularly to extrahepatic tissues, remains a major translational limitation and thus investments in oligonucleotide therapeutic discovery have created an emerging market need for drug delivery technologies


The Oligonucleotide Therapeutics and Delivery conference reveals the latest in oligonucleotide discovery and delivery, with presentations on targeted delivery mechanisms, oligonucleotide chemistries and important clinical advances. Global experts from big pharma and leading biotechs will explore the emerging therapeutic applications of oligonucleotides with case studies surrounding the discovery and development of oligonucleotide therapeutics as well as CMC and regulatory standpoints. The two-day conference will bring you high-quality insights and industry connections on the latest clinical trial candidates and a platform for exchanging ideas for tackling the biggest challenge: DELIVERY. 
 

 

  • Listen to case studies presenting on pre-clinical and clinical research in areas such as oncology and what can be learned for future clinical trials
  • Deepen your understanding of oligonucleotide therapeutic delivery and explore the latest innovations in extrahepatic oligonucleotide delivery and target specificity
  • Understand the regulatory and CMC environment surrounding oligonucleotide therapeutic development
  • Explore developments in oligonucleotide chemistries and examine developments in novel applications of antisense oligonucleotides and siRNA
  • Engage in the latest innovations in oligonucleotide therapeutics such as utilising AI machine learning to drive the discovery of novel therapeutics
     

Executives, Directors, VPs, Heads, Principals, Managers of:

RNA Biology/Discovery
Novel Therapeutic Modalities
Innovation Technologies
Formulation and Drug Delivery
Clinical Research and Development
Cell Biology

 

Accanis Therapeutics; Advanced Cell Diagnostics – A Bio-Techne Brand; Aescap 2.0; Alnylam Pharmaceuticals UK & Ireland; AmpTec GmbH; Aseptic Technologies SA; AstraZeneca; Bachem AG; BianoGMP; BioNTech IMFS GmbH; BioNTech RNA Pharmaceuticals; Boehringer Ingelheim Pharma GmbH & Co. KG; Calithera Biosciences; CEPI - Coalition for Epimemic Prepardness Innovation; Chemgenes Corporation; Curevac Ag; Dicerna Therapeutics.; Duke University School of Medicine; Ethris GmbH; Exicure Inc.; F.Hoffmann-La Roche AG; GSK; Horizon Discovery; InteRNA Technologies BV; Intertek; Lipocalyx; MedImmune; MHRA; MiNA Therapeutics; N4 Pharma UK Limited; New England Biolabs; NHS Blood and Transplant Authority; Nitto Avecia; NOF Europe GmbH; Nouscom SRL; ProQR Therapeutics; RCSI; RCSI Royal College of Surgeons in Ireland,; RNA Medco; ROCHE; Roche Diagnostics Deutschland GmbH; Roche Innovation Center; Russell Publishing Ltd; Sanofi; Sanofi-Aventis Deutschland GmbH; Takeda Development Centre Europe Ltd.; TCR Solutions; The Italian Institute of Technology; The RNA Medicines Company; Thermo Fisher Scientific; University Medicine Mainz Hospital; University Of Portsmouth; University Of Surrey; Yuria Pharma;

 

Conference programme

8:00 Registration & Coffee

9:00 Chairman's Opening Remarks

Ekkehard Leberer

Ekkehard Leberer, Senior Consultant, Elbicon
View Bio

Jimmy Weterings

Jimmy Weterings, Principal Scientist, AstraZeneca
View Bio

9:10 Developing Oligonucleotides into Therapies: Opportunities and Challenges

  • Introduction to oligonucleotide therapeutics and the current landscape
  • Therapeutic promise of oligonucleotide modalities
  • Outlining some of the key challenges in oligonucleotide therapeutics including extra-hepatic delivery
  • Exploring some of the emerging delivery solutions and future opportunities
     
  • Shalini Andersson

    Shalini Andersson, Vice President Oligonucleotide Discovery, AstraZeneca
    View Bio

    9:50 The UK Nucleic Acid Therapy Accelerator (NATA)- One Year On

    Nick Lench

    Nick Lench, Executive Director, Nucleic Acid Therapy Accelerator (NATA)
    View Bio

  • Introduction to the aims and mission of NATA in supporting the development of RNA Therapeutics and the long-term vision of the organization
  • Insight into the NATA’s current focus on short oligonucleotides and case studies on siRNAs and ASOs
  • The NATA’s funding calls to address challenges in the delivery of RNA
  •  

    10:30 Morning Coffee

    11:00 Current CMC Regulatory Challenges in ASO Development

    Chris Chorley

    Chris Chorley, Associate Director, Global Regulatory CMC, Biogen
    View Bio

  • Overview of key CMC regulatory challenges during the clinical development phase
  • Regulatory strategies for use of platform data and prior knowledge
     
  • 11:40 Molecular Mechanisms of Antisense Oligonucleotides Cytotoxicity

    Sergio Leone

    Sergio Leone, Research Associate, MRC Toxicology Unit, University Of Cambridge
    View Bio

  • Introduction to the mechanistic action of ASOs in cells after delivery
  • Insight into in vivo and in vitro models for the study of ASO cytotoxicity
  • Exploring molecular mechanisms of ASO cytotoxicity to better design future ASO therapeutics
  • Discussion of balancing safety and efficacy of ASOs
  • 12:20 Application of Isotope Labelled Oligonucleotides to Imaging and Bioanalysis

    Rouven Stulz

    Rouven Stulz, Senior Scientist, AstraZeneca
    View Bio

  • Labelling Strategy
  • Intracellular imaging of oligonucleotide conjugates
  • Quantification of oligonucleotide conjugates in endosomes
  • Other applications

     

  • 13:00 Networking Lunch

    14:00 Upregulation of Protein Expression via ASO Therapy

    Eric Lim

    Eric Lim, Director, Platform, Discovery Research, Stoke Therapeutics, Inc
    View Bio

  • Introduction to Stoke’s propriety research platform TANGO
  • Exploring the design of ASOs that bind to pre-mRNA and help the target genes to produce more protein
  • Outline of future activities and challenges
  •  

    14:40 Exploring Modern Oligonucleotide Therapeutics for the Treatment of Muscle Diseases

    Stefano Zanotti

    Stefano Zanotti, Senior Director, Head of Neuromuscular Research, Dyne Therapeutics
    View Bio

  • Introduction to the FORCETM platform
  • Discussion of PMO chemistry and antisense oligonucleotide-mediated exon skipping as a therapeutic approach
  • Application of the FORCETM platform for the treatment of Duchenne muscular dystrophy
  • Insight into DYNE-251 and naked PMO delivery in NHP
     
  • 15:20 Afternoon Tea

    15:50 Delivery of Novel siRNA Constructs for Treating Cancer

    David Evans

    David Evans, Chief Scientific Officer, Sirnaomics, Inc
    View Bio

  • Creating novel siRNA constructs for cancer treatment
  • Introduction to polypeptide nanoparticle delivery system (PNP-IT)
  • STP705- Sirnaomic’s lead oncology siRNA therapeutic targeting TGF-β1/ COX-2
  • Discussion of novel siRNA therapeutics targeting other oncology indications
     
  • 16:30 The Power of Artificial Intelligence: Discovery of Novel Therapeutics through RNA Splicing analysis

    Martin Akerman

    Martin Akerman, CTO & Co-Founder, Envisagenics
    View Bio

  • Introduction to Envisagenics’ AI-driven drug discovery platform, SpliceCore®
  • Leveraging RNA splicing expertise and the SpliceCore platform to identify novel RNA splicing events for drug development
  • Current limitations of AI/ML and how best to approach a successful translation into the downstream research and development
  • 17:10 ADAR-based RNA Editing for Therapy

    Thorsten Stafforst

    Thorsten Stafforst, Professor of RNA Biochemistry, University Of Tubingen

  • Introduction to the concept of RNA base editing
  • Discussion of the CLUSTER approach to achieve editing with high precision and efficiency
  • Looking into the future of drug development in the field of RNA base editing

     

  • 17:50 Chairman's Closing Remarks and Close of Day One

    Ekkehard Leberer

    Ekkehard Leberer, Senior Consultant, Elbicon
    View Bio

    Jimmy Weterings

    Jimmy Weterings, Principal Scientist, AstraZeneca
    View Bio

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Ekkehard Leberer

    Ekkehard Leberer, Senior Consultant, Elbicon
    View Bio

    Jimmy Weterings

    Jimmy Weterings, Principal Scientist, AstraZeneca
    View Bio

    9:10 Oligonucleotide Delivery: challenges and opportunities

    Matthew Wood

    Matthew Wood, Professor of Neuroscience, Deputy Head Medical Science Division (Innovation), Director of MDUK Oxford Neuromuscular Centre, Director of Oxford Harrington Rare Disease Centre, University of Oxford Department of Physiology
    View Bio

  • While oligonucleotide therapies have enormous therapeutic potential, significant challenges in drug delivery to many extra-hepatic cell/tissue types remain
  • This is particularly the case for neurological and neuromuscular disorders where limited efficacy and poor oligonucleotide delivery to affected tissues including skeletal muscle, cardiac muscle and the central nervous system, represent both major challenges and opportunities
  • A wide range of delivery technologies are being advanced to address this challenge, including bio-conjugates and nanotechnologies
  • Current progress and future prospects will be discussed
  • 9:50 Clinical Development of ApTOLL- An Aptamer to Reduce Inflammation

    Macarena Hernandez- Jimenez

    Macarena Hernandez- Jimenez, Chief Scientific Officer, aptaTargets S.L.
    View Bio

  • A background to AptaTargets and introduction to ApTOLL: an aptamer designed to block TLR4, a crucial receptor involved in the inflammatory response triggered in different diseases
  • Demonstration of efficacy and safety of ApTOLL in experimental models
  • Exploring the results of a first-in-human clinical trial of ApTOLL
  • Outlining the steps going forward
     
  • 10:30 Morning Coffee

    11:00 Introduction to RNA activation and its use to restore normal function to cells

    Nagy Habib

    Nagy Habib, Associate Director, Co-Founder, CMO and Head of R&D, MiNA Therapeutics Ltd
    View Bio

  • Discussion of increasing duration of oligonucleotide drugs and why this matters 
  • Utilising saRNA in oncology and RNA gene activation in the brain
  • Update on the clinical progress of MTL-CEBPA: an investigative drug that targets tumour associated macrophages to favourably alter the tumour microenvironment
  • Exploring how to predict clinical response with saRNAs
     
  • 11:40 Antisense oligonucleotide clinical trials in Huntington’s disease: what can we learn?

    Sarah Tabrizi

    Sarah Tabrizi, Director of UCL Huntington’s disease centre, UCL Institute of Neurology
    View Bio

  • Overview of oligonucleotide therapies in development for Huntington’s disease
  • Details of the preclinical to clinical development of Tominersen, an ASO targeting HTT
  • Possible reasons for the failure of the phase 3 GENERATION-HD1 clinical trial of Tominersen
  • Lessons we need to learn for future clinical trials of oligonucleotides in neurodegenerative diseases
  • 12:20 Networking Lunch

    13:20 Approaches towards the next generation of RNA Delivery Systems

    Heinrich Haas

    Heinrich Haas, Vice President Formulation & Drug Delivery, BioNTech
    View Bio

  • Introduction to the fundamentals of RNA delivery
  • Insight into the design of RNA delivery systems and new delivery technologies arising in R&D
  • Discussion of CMC and GMP criteria for successful clinical translation of RNA delivery systems
  • Exploring the development opportunities of various next generation RNA delivery systems
  • 14:00 Protein-based Nanocapsules for therapeutic RNA Delivery

    Oliver Ernst

    Oliver Ernst, Managing Director, Chief Operating Officer, Neuway Pharma GmbH
    View Bio

  • Introduction to NEUWAY’s scalable and versatile delivery technology (EnPC®) offering a broad range of therapeutic applications
  • CNS Delivery Option:
  • Discussion of utilising naturally derived protein capsules to deliver mRNA across the blood brain barrier and the benefits of this
  • Exploring the development of innovative therapeutics for treatment of brain diseases and CNS-related genetic diseases
  • Future opportunities in the targeted delivery of RNA therapeutics
     
  • 14:40 RNA therapeutics modalities and targeted delivery approaches for developing transformational medicines for patients

    Tamar Grossman

    Tamar Grossman, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceuticals
    View Bio

  • Introduction to the development of siRNA therapeutics
  • Discussion of the design of novel carriers to improve extra-hepatic targeting
  • Insight into siRNA delivery to the CNS
     
  • 15:20 Afternoon Tea

    15:50 Miniaturized RNAi Triggers Composed of Single Oligonucleotides

    Dmitry Samarsky

    Dmitry Samarsky, Chief Technology Officer, Sirnaomics
    View Bio

  • Introduction to GalAhead: Sirnaomic’s GalNAc-RNAi therapeutic platform
  • Miniaturized RNAi (mxRNA) as a key technological component of the platform
  • Validation of technology in vivo and in vitro
  • Progress report on GalAhead based programs
     
  • 16:30 Spherical Nucleic Acids (SNAs) Facilitate Efficient Local Delivery for Neurological Indications

    Bart Anderson

    Bart Anderson, Senior Director R&D, Exicure
    View Bio

  • Delivery of SNAs to the CNS leads to broad CNS penetration and uptake in critical neuronal cell types
  • Higher persistence in the CNS leads to prolonged efficacy
  • SNA targeting SCN9A / Nav1.7 for treating chronic pain
     
  • 17:10 Chairman’s Closing Remarks and Close of Day Two

    Ekkehard Leberer

    Ekkehard Leberer, Senior Consultant, Elbicon
    View Bio

    Jimmy Weterings

    Jimmy Weterings, Principal Scientist, AstraZeneca
    View Bio

    +

    FEATURED SPEAKERS

    Bart Anderson

    Bart Anderson

    Senior Director R&D, Exicure
    Chris Chorley

    Chris Chorley

    Associate Director, Global Regulatory CMC, Biogen
    David Evans

    David Evans

    Chief Scientific Officer, Sirnaomics, Inc
    Dmitry Samarsky

    Dmitry Samarsky

    Chief Technology Officer, Sirnaomics
    Eric Lim

    Eric Lim

    Director, Platform, Discovery Research, Stoke Therapeutics, Inc
    Heinrich Haas

    Heinrich Haas

    Vice President Formulation & Drug Delivery, BioNTech
    Macarena Hernandez- Jimenez

    Macarena Hernandez- Jimenez

    Chief Scientific Officer, aptaTargets S.L.
    Martin Akerman

    Martin Akerman

    CTO & Co-Founder, Envisagenics
    Matthew Wood

    Matthew Wood

    Professor of Neuroscience, Deputy Head Medical Science Division (Innovation), Director of MDUK Oxford Neuromuscular Centre, Director of Oxford Harrington Rare Disease Centre, University of Oxford Department of Physiology
    Nagy Habib

    Nagy Habib

    Associate Director, Co-Founder, CMO and Head of R&D, MiNA Therapeutics Ltd
    Nick Lench

    Nick Lench

    Executive Director, Nucleic Acid Therapy Accelerator (NATA)
    Oliver Ernst

    Oliver Ernst

    Managing Director, Chief Operating Officer, Neuway Pharma GmbH
    Rouven Stulz

    Rouven Stulz

    Senior Scientist, AstraZeneca
    Sarah Tabrizi

    Sarah Tabrizi

    Director of UCL Huntington’s disease centre, UCL Institute of Neurology
    Sergio Leone

    Sergio Leone

    Research Associate, MRC Toxicology Unit, University Of Cambridge
    Shalini Andersson

    Shalini Andersson

    Vice President Oligonucleotide Discovery, AstraZeneca
    Stefano Zanotti

    Stefano Zanotti

    Senior Director, Head of Neuromuscular Research, Dyne Therapeutics
    Tamar Grossman

    Tamar Grossman

    Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceuticals
    Thorsten Stafforst

    Thorsten Stafforst

    Professor of RNA Biochemistry, University Of Tubingen

    Bart Anderson

    Senior Director R&D, Exicure
    Bart Anderson

    Bart Anderson, Ph.D., is the Senior Director, R&D at Exicure, where he leads preclinical discovery and development efforts across all mechanisms of action and therapeutic areas. Bart background training and experience are in molecular biology, genetics, neuroscience, and nucleic acid therapeutics. Since joining the company, Exicure’s preclinical Spherical Nucleic Acid (SNA) oligonucleotide compounds progressed into clinical trials using antisense-mediated mRNA reduction in dermatology as well as immunostimulation (TLR9-agonist) in immuno-oncology, and investigation expanded to include splice-modulating oligonucleotides and neurological disorders. Current areas of emphasis are neurology indications using antisense oligonucleotides (ASOs) for RNase H-mediated RNA knockdown and splice modulation.

    Chris Chorley

    Associate Director, Global Regulatory CMC, Biogen
    Chris Chorley

    Chris has worked in the UK pharmaceutical industry for approximately 15 years, beginning his career as a Research Scientist for AstraZeneca, before moving into regulatory affairs in 2011. He held positions of increasing responsibility with Pharmalink Consulting and Ipsen, before joining Biogen in 2020, where he now leads the CMC regulatory activities for the ASO early development portfolio. Chris hold a Masters degree in Chemistry from the University of York, UK.

    David Evans

    Chief Scientific Officer, Sirnaomics, Inc
    David Evans

    Dr. Evans obtained his BSc and PhD from Imperial College, London. Over the last 3 decades he has been responsible for building and leading scientific research teams focused on target validation and drug discovery in Oncology and other indications. David has more than 18 years’ experience in the RNAi field – building genome scale screening capabilities for target identification at TGEN and Dharmacon prior to cofounding Sirnaomics to focus on developing siRNA as a therapeutic. David previously worked in the Boston area at Millennium Pharmaceuticals and Serono Pharmaceuticals.


     

    Dmitry Samarsky

    Chief Technology Officer, Sirnaomics
    Dmitry Samarsky

    Dmitry Samarsky, PhD has been at the inception of RNAi technology and drug development - starting in 2001 as Director of Technology Development at Sequitur (acquired by Invitrogen) and (in 2005) as Director of Technology Development at Dharmacon (now part of GE). He then served as VP of Technology Development at RXi Pharmaceuticals, USA (2007-2011), SVP of Technology and International Business Development at RiboBio, China (2011-2016) and, most recently, as Chief Scientific Officer at Silence Therapeutics, Germany/UK (2016-2018). Dr. Samarsky has authored more than 40 scientific papers, articles, book chapters, patents, and patent applications. He has been an invited speaker at more than 100 international conferences, and currently serves on the Scientific Advisory Boards for the OTS (Oligonucleotide Therapeutics Society). Dr. Samarsky received his doctorate in biochemistry and molecular biology from University of Massachusetts, Amherst (1998), followed by a postdoctoral position as an H. Arthur Smith Fellow for Cancer Research in Michael Green's lab at University of Massachusetts Medical School (1998-2001).

     

    Ekkehard Leberer

    Senior Consultant, Elbicon
    Ekkehard Leberer

    Dr. Leberer received his diplomas in biology, chemistry and physics and his Ph.D. in biochemistry at the University of Konstanz, Germany. He conducted post-doctoral training in molecular biology at the Banting and Best Institute, University of Toronto, Canada, and he obtained the Habilitation for Professor of Biochemistry at the University of Konstanz. From 1989-1998, he served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. He was also Adjunct Professor at McGill University, Montreal.

    His academic research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the co-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.

    Since joining the pharmaceutical industry in 1998, Dr. Leberer carried out various managing roles in Hoechst Marion Roussel, Aventis and Sanofi (Frankfurt and Munich,
    Germany), including responsibilities in functional genomics, biological sciences, external innovation, alliance management, and pre-clinical and clinical development of various therapeutic modalities encompassing small molecule drugs and biopharmaceuticals (peptides, antibodies and
    oligonucleotides). In addition, from 2012-2018, he has been the Scientific Managing Director of the Innovative Medicines Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes, Brussels (www.imi.europa.eu).

    In March 2021, Dr. Leberer initiated ELBIOCON Life Sciences Consulting, Munich (www.elbiocon.com), offering his broad experience in pharmaceutical drug research and
    development to clients in academia and biotechnology. He has served as a member in Scientific Advisory Boards of several biotechnology companies, and he is the Head of the Supervisory Board of BioM, Munich (www.bio-m.org).
     

    Eric Lim

    Director, Platform, Discovery Research, Stoke Therapeutics, Inc
    Eric Lim

    With a background in Computer Science and Mathematics, Eric received his Ph.D. in Molecular Biology, Cell Biology, and Biochemistry from Fairbrother Lab at Brown University. His work focused on using a combination of computational biology and high throughput genomics techniques to identify functional splicing elements in the genome. Eric has a decade of post-postdoctoral experience in the biotech industry and is currently the Head of Bioinformatics at Stoke Therapeutics, where he leads a team of computational scientists in leveraging oligonucleotides as a modality to treat the underlying cause of genetic diseases. Before Stoke, he was a co-investigator of a team at the Children's Hospital of Philadelphia to support research into potential treatments for an aggressive form of pediatric leukemia. His prior research also involved the development of preclinical cancer genomics in identifying preferential anti-tumor activities of a small molecule in blood cancers and solid tumors. Eric has over 24 publications and patents in the field of RNA splicing and his work has led to numerous awards, including the 40 Under 40 in Cancer.

    Heinrich Haas

    Vice President Formulation & Drug Delivery, BioNTech
    Heinrich Haas

    Heinrich has his professional focus on development of pharmaceutical products in the field of nanotechnology and drug & RNA delivery. In his Ph.D. in the group of Prof. Dr. Helmuth Möhwald at Johannes-Gutenberg Universität Mainz and academic career in Italy and Brazil he researched lipid membranes and organized bio-molecular systems. Having moved to pharmaceutical industry he developed nanoparticle products with application in cancer, inflammatory diseases autoimmune diseases and other indications. At BioNTech, he is the scientific lead of the formulation and analytics department, which develops RNA therapeutics based on a broad scope of delivery technologies.

     

    Jimmy Weterings

    Principal Scientist, AstraZeneca
    Jimmy Weterings

    Jimmy has had a tight bond with Oligonucleotide Therapeutics since 2002, performing research on Peptide Nucleic Acid (PNA) in the van Boom group at Leiden University and targeted SSO conjugates at Prosensa. During his Ph.D (Bio-organic Chemistry) at Leiden University, the Netherlands he generated and studied CpG Oligonucleotide and other Toll-like receptor targeting ligand containing conjugates. At Cenix BioScience, he developed siRNA conjugates for targeted CNS delivery. And at Cristal Therapeutics he lead various projects on the nanomedicine delivery of oligonucleotide in oncology. In 2019, Jimmy joined AstraZeneca in Sweden, fully dedicating himself to the further advancement of (targeted) oligonucleotide therapeutics.
    Jimmy (co-)authored 13 articles and holds several patents for drug delivery enabling technologies, including the most recent one on TMTHSI, a promising new conjugation compound for oligonucleotide and antibody conjugates.

     

    Macarena Hernandez- Jimenez

    Chief Scientific Officer, aptaTargets S.L.
    Macarena Hernandez- Jimenez

    PhD in Biomedicine and expert in Pharmacovigilance with more than 15 years of experience in Neuroscience and Pharmacology in cerebrovascular diseases. PhD and postdoctoral positions in topics related to cerebral hypoxia-ischemia at the hospital setting and in cerebral ischemia and Pharmacology at the Complutense University, where developed the investigation of large translational nonclinical studies as part of the Stroke Spanish Investigation Consortium. Currently, Associated Professor of Pharmacology and Neurosciences at Complutense University and Scientific Director of aptaTargets focused on nonclinical studies and clinical trials required to develop a new aptameric therapy for the treatment of stroke and COVID-19 patients.

    Martin Akerman

    CTO & Co-Founder, Envisagenics
    Martin Akerman

    Martin Akerman is the Co-founder and CTO of Envisagenics. He is the inventor of the SpliceCore® platform, Envisagenics’ flagship AI technology for the discovery of splicing-based therapeutic targets. Martin trained as a post doctorate fellow with Dr. Adrian Krainer from Cold Spring Harbor Laboratory. He received his PhD in Bioinformatics from Technion, Israel Institute of Technology. As a first-time entrepreneur, Martin represented Envisagenics in winning the J&J AI for Drug Discovery QuickFire challenge in 2017 and Microsoft’s Innovate.AI challenge in 2018. His goal is to combine cutting-edge computation with RNA domain expertise to develop innovative drugs for cancer and genetic diseases.

    Matthew Wood

    Professor of Neuroscience, Deputy Head Medical Science Division (Innovation), Director of MDUK Oxford Neuromuscular Centre, Director of Oxford Harrington Rare Disease Centre, University of Oxford Department of Physiology
    Matthew Wood

    MATTHEW WOOD F.Med.Sci. is Professor of Neuroscience and Deputy Head of the Medical Sciences Division at the University of Oxford. He directs the Laboratory of RNA biology and Neuromuscular Disease investigating development of RNA-based medicines for neuromuscular disease focusing on the development of advanced generation antisense oligonucleotides for Duchenne muscular dystrophy and related neuromuscular conditions. He is currently Director of MDUK Oxford Neuromuscular Centre and Director of the Oxford Harrington Rare Disease Centre. He has pioneered the development of novel drug delivery systems including peptide and exosome-based technologies for RNA delivery.

    He is a co-founder of the biotech spin-outs Evox Therapeutics and PepGen, and has recently led a major UK national initiative to establish a UK Nucleic Acid Therapy Accelerator (NATA).
     

    Nagy Habib

    Associate Director, Co-Founder, CMO and Head of R&D, MiNA Therapeutics Ltd
    Nagy Habib

    Nagy is a founder and Head of R&D of MiNA Therapeutics and Head of Department of HPB Surgery at Imperial College London. For over three decades he has been at the forefront of clinical research and clinical practice in cancer.


    Currently, he is driving the development of an saRNA drug which is currently being trialed in patients with liver cancer in eight UK centres, and sites in Singapore and Taiwan (OUTREACH study, ClinicalTrials.gov ID NCT02716012) and in a second trial in patients with solid tumours (TIMEPOINT study, ClinicalTrials.gov ID NCT 04105335) in the UK, USA, Europe, Singapore and Taiwan.

    Nick Lench

    Executive Director, Nucleic Acid Therapy Accelerator (NATA)
    Nick Lench

    Nick is a co-founder of Congenica Limited, a genomic medicine and digital health company based at the Wellcome Genome Campus, Cambridge and held positions of COO and CSO prior to his appointment at NATA. Nick has worked in academia, industry and the NHS and has extensive experience in personalised medicine and clinical diagnostics. Prior to Congenica, Nick was Director of the NE Thames Regional Genetics Service at Great Ormond Street Hospital for Children, London. Nick was awarded a personal chair in Medical Genetics at Cardiff University, is currently an honorary reader in Genetics and Genomic Medicine at UCL and a fellow of the Royal College of Pathologists.

    Oliver Ernst

    Managing Director, Chief Operating Officer, Neuway Pharma GmbH
    Oliver Ernst

    Dr. Ernst joined NEUWAY in November 2018 as Managing Director, also leading business development, strategic partnering and financing, with strong background in management of innovation and intellectual property. Prior to NEUWAY, Oliver had gained over ten years of professional experience in the field of intellectual property and innovation management ranging from private practice to large enterprise. Oliver graduated with a degree in biotechnology engineering from the Technical University Berlin and received his PhD at the Fraunhofer Institute for Biomedical Engineering, Potsdam (IBMT).


     

    Peter Oliver

    Head of Biology, Nucleic Acid Therapy Accelerator
    Peter Oliver

    Rouven Stulz

    Senior Scientist, AstraZeneca
    Rouven Stulz

    Rouven Stulz received his Bachelors and Masters degree from the university of Bern before moving to Gothenburg to pursue his PhD in a collaboration between Karolinska Institute and AstraZeneca. His work focused on the development of synthetic strategies for the isotope labelling of oligonucleotides and their application to imaging and bioanalysis. He also worked on the synthesis of modified nucleotides with the goal to increase the therapeutic potential of antisense oligonucleotides. Currently he is part of AstraZeneca’s oligonucleotide discovery program as a Senior Scientist and is working on the development of novel nucleoside analogues.

    Sarah Tabrizi

    Director of UCL Huntington’s disease centre, UCL Institute of Neurology
    Sarah Tabrizi

    Sarah Tabrizi is Director of the UCL Huntington’s Disease (HD) Centre, Joint Head of Department Neurodegenerative Disease at the UCL Queen Square Institute of Neurology, a Principal Investigator at the UK Dementia Research Institute, and Consultant Neurologist at the National Hospital for Neurology and Neurosurgery. In addition to a basic bench science programme focussing on basic cellular mechanisms of neurodegeneration in HD that can be harnessed for therapeutics, she also leads a large translational research programme that is working towards finding effective disease-modifying treatments for HD. She was global clinical PI on the world’s first gene targeting study for HD using anti-sense oligonucleotide therapy.

    Sergio Leone

    Research Associate, MRC Toxicology Unit, University Of Cambridge
    Sergio Leone

    Sergio Leone studied Biology at the University of Palermo (Italy) and obtained hid PhD in 2016 from the University of Zurich (Switzerland) studying the role of a long non-coding RNA in the regulation of ribosomal RNA genes silencing and embryonic stem cells differentiation. Moving as postdoc in the lab of Ritwick Sawarkar at the Max Planck Institute for Epigenetics and Immunobiology in Freiburg (Germany), he started his research on RNA binding of molecular chaperones. In 2020 Sergio moved to the MRC Toxicology Unit in Cambridge continuing his work on protein-RNA interaction and on molecular mechanisms of oligonucleotide therapeutics cytotoxicity.

    Shalini Andersson

    Vice President Oligonucleotide Discovery, AstraZeneca
    Shalini Andersson

    Shalini is currently Vice President Oligonucleotide Discovery in BioPharmaceuticals R&D. Shalini has also the strategic responsibility for explorative activities and build of capabilities in the new therapeutic modalities area.


    Shalini has broad experience in Pharmaceutical research as well as of strategic and management roles and steers several collaborations with external partners and academic groups across the globe.
    Prior to the current position, Shalini has held several leadership roles within Medicinal Chemistry and Drug Metabolism & Pharmacokinetics in Cardiovascular, renal and metabolism therapy area.

    Shalini is also leading the Targeted Drug Delivery platform projects and is involved in collaborations focused on developing RNA Therapeutics with Ionis Pharma, Silence Therapeutics and VaxEquity. Shalini is member of the board of the Oligonucleotide Therapeutics Soc. as well as of the scientific advisory group for NATA (UK) and member of the steering group for OligoNova (Sweden).

    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 60 peer reviewed articles and 5 patents.
     

    Stefano Zanotti

    Senior Director, Head of Neuromuscular Research, Dyne Therapeutics
    Stefano Zanotti

    Stefano Zanotti is Head of Neuromuscular Research at Dyne Therapeutics, where he oversees preclinical development of treatments for genetically-driven muscular diseases. Stefano dedicated his career to rare genetic disorders. Prior to joining Dyne, Stefano led pre-clinical research programs in multiple rare diseases as Director of Rare Renal and Musculoskeletal Diseases at Sanofi. Previously, Stefano was an Assistant Professor at the UCONN School of Medicine, where he conducted NIH-funded research on genetic disorders of the skeletal system.
    Stefano has authored more than 50 research articles and reviews in major scientific journals and received multiple awards for his contributions to skeletal biology.
     

    Tamar Grossman

    Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceuticals
    Tamar Grossman

    Dr. Tamar R. Grossman is the Global Head of RNA and Targeted Therapeutics at Janssen Pharmaceutical Companies of Johnson and Johnson. Tamar is responsible for the delivery of RNA based development candidates across all therapeutic areas. Previously, she was a Senior Director and Head of Rare Diseases at Arcturus Therapeutics where she developed RNA therapeutics across different RNA technologies. Prior to Arcturus, Tamar was the Director of Translational Medicine at Ionis Pharmaceuticals where she led various antisense oligonucleotide research programs to clinical development. She received her PhD in Biochemistry from Tel-Aviv University and complete postdoctoral research training at the UCSD.

    Thorsten Stafforst

    Professor of RNA Biochemistry, University Of Tubingen
    Thorsten Stafforst

    Thorsten is Professor of Nucleic Acid Biochemistry at the Interfaculty Institute of Biochemistry at the University of Tübingen. He is a trained chemist and chemical biologist. In 2011, he started his independent lab in Tübingen. Since 2016, he is permanent professor in Tübingen supported by a Heisenberg-Professorship (DFG). His lab works on site-directed RNA editing strategies and their exploitation in life sciences and medicine. He is supported by major funding from the ERC, the DFG and the VolkswagenStiftung.


     

    Exhibitors

    Supporters

    Workshops

    Oligonucleotide Therapies- Overcoming the Challenges of Delivery
    Workshop

    Oligonucleotide Therapies- Overcoming the Challenges of Delivery

    Copthorne Tara Hotel
    20th September 2022
    London, United Kingdom

    Managing CMC Activities for the Development of Oligonucleotide Therapeutics
    Workshop

    Managing CMC Activities for the Development of Oligonucleotide Therapeutics

    Copthorne Tara Hotel
    20th September 2022
    London, United Kingdom

    VENUE

    Copthorne Tara Hotel

    Scarsdale Place, Kensington, London, United Kingdom

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

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

     

    A number of our clients have been approached by third party organisations offering to book hotel rooms. We would advise that you do not book through them as they are not representing the SAE Media Group. SAE Media Group books all hotel rooms directly. If you are approached by a third party organisation then please contact us before making any bookings. If you have already booked a hotel room using a third party organisation, we would highly recommend contacting the hotel you were booked into to ensure a booking has been made for you. We would also advise you to please check the terms and conditions of the booking carefully.
    HOTEL BOOKING FORM

    Oligonucleotide Therapeutics and Delivery Brochure 2022

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    Conference Co-Chair Letter

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    Workshop B Agenda

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    Workshop A Agenda

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    Speaker Bios

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    Past Attendees

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    Past Presentation - Guy Hermans, Sapreme Technologies

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    Past Presentation - Art Levin, Avidity Biosciences

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    Past Presentation - Dr André Müller-York, Sarepta Therapeutics

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    Exhibitors


    ChemGenes

    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.

    Media Partners


    Bio Pharma Trend

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    https://www.biopharmatrend.com/

    BiopharmaTrend is a market and business intelligence platform for drug discovery and biotech industry, focusing on emerging areas: artificial intelligence in pharmaceutical R&D, "digitalization" of the pharma industry, and technology/business trends in pre-clinical and clinical drug development. We also publish articles, commentaries and opinions contributed by top industry professionals.


    On Drug Delivery

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    http://www.ondrugdelivery.com/subscribe

    ONdrugDelivery provides 30,000 readers throughout global pharma/biopharma, with the latest, most pertinent industry information and intelligence about the drug delivery-related ideas, technologies, services and products the sector is developing. Established in 2004, and trusted throughout the industry for its high quality contributions from leading drug delivery experts, ONdrugDelivery is a must for pharma / biotech industry professionals who need to know what’s going on in drug delivery. Subscribe online today to the print or free digital edition: www.ondrugdelivery.com/subscribe.


    Pharma Journalist

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    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.


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    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


    Pharmalicensing

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    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.


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    http://www.genomeweb.com

    GenomeWeb LLC is an independent, privately-held online and print publisher based in New York. Since 1997, GenomeWeb has served the global community of scientists, technology professionals, and executives who use and develop the latest advanced tools in molecular biology research and molecular diagnostics.

    GenomeWeb’s editorial mission is to serve readers with exclusive, in-depth coverage of the technology, institutions, and scientists that make up the worldwide research enterprise of molecular biology. We operate the largest online news organization focused on advanced research tools in genomics, proteomics, and bioinformatics.

    GenomeWeb readers can be found in major biopharmaceutical companies, top research universities, biomedical institutes, and government laboratories.


    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


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    MedNous is a print publication and website about medical innovation in Europe. It carries exclusive interviews with companies that are at the forefront of medical technology, as well as contributor articles from prominent practitioners. Our mission is to identify significant advances in medicine and to explain how this innovation is being commercialised. In doing so, we talk to venture capitalists about what products and platforms they are supporting. We report on how regulators cope with the accelerating pace of innovation. And we regularly cover the latest developments in the discovery and development of new medical concepts in the area of antibodies, vaccines, small molecules, regenerative medicine and nanomedicines. MedNous combines the English word for medicine with the Greek word for intellect. And those with nous are readers of our publication. Visit our website: www.mednous.com


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    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.


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    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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