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Oligonucleotide therapeutics - the emerging medicine class - are harnessing the therapeutic benefit of targeting genetic material via antisense, mRNA, RNAi, saRNA and siRNA. Their market growth: CAGR of 13.7% projected to reach USD 8.2 billion by 2024 is driven by their potential to provide more efficacious and less toxic alternatives to small molecules. They bring about the ability to affect targets that have been considered “non-draggable”. Heavy investments in oligonucleotide therapeutic discovery have created an emerging market need for drug delivery technologies.
 

SMi’s inaugural Oligonucleotide Therapeutics and Delivery conference is here to keep you updated. By bringing together leading representatives of pharmaceutical, biotechnology and academic institutions, the 2-day conference will provide first-hand information on the latest clinical trial candidates and a platform for exchanging ideas for tackling the biggest challenge: DELIVERY. From optimizing particle size to choosing the right nanocarrier systems, the conference will address delivery to non-hepatocyte cells such as cancerous tissues and overview the most successful platforms. Discussions will be centered around improving targeted therapy uniting the field’s biggest players to share their opinions.
 

Established on the success of our RNA Therapeutics series, we look forward to welcoming you at Oligonucleotide Therapeutics and Delivery to join the conversation around maximizing the potential of oligo-based treatments.

  • Gain first-hand insight into oligonucleotide therapy clinical success of the latest developments for novel agents
  • Listen to case studies presenting the latest candidates undergoing pre-clinical and clinical research
  • Deepen your understanding of crucial delivery methods and available platforms for non-hepatocytic delivery
  • Explore the chemistry of oligonucleotide therapeutics and examine novel applications of antisense oligonucleotides in the AstraZeneca Deep Dive
  • Engage in the latest innovations of oligonucleotide therapeutics with inisights into immunochemotherapeutics, ocular indications and Duchenne Muscular Dystrophy

 

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

  • RNA Biology/Discovery
  • Novel Therapeutic Modalities
  • Innovation Technologies
  • C-level Scientific Executives
  • 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 Chair's Opening Remarks

Nagy Habib

Nagy Habib, Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
View Bio

9:10 Evolution, challenges and potential of RNA Therapeutics

Michelle Lynn Hall

Michelle Lynn Hall, Senior Director, Eli Lilly & Co - New Therapeutic Modalities
View Bio

• Background on RNA Therapeutics –what it encompasses, current
status and pipeline
• Challenges surrounding development of RNA Therapeutics
• Promising future of RNA therapeutics
 

9:45 Case study: Automating the discovery and development of RNA therapeutics

Maria Luisa Pineda

Maria Luisa Pineda, CEO and Co-founder, Envisagenics
View Bio

• Envisagenics’ SpliceCore platform integrates proprietary machine learning algorithms, high performance computing, and RNA-splicing analytics to identify disease-specific isoforms that will function as therapeutic targets
• Proof-of-concept program in Triple Negative Breast Cancer (TNBC) – discovery of TNBC specific splicing isoforms
• Machine learning driven methodologies to designing and developing RNA therapeutics targeting TNBC specific splicing isoforms
• Streamlined experimental validations of SpliceCore designed RNA therapeutics
 

10:20 Morning Coffee

10:55 Oligonucleotides as a therapeutic option for Duchenne Muscular Dystrophy (DMD)

Andre  Muller-York

Andre Muller-York, Senior Medical Affairs Director, Sarepta Therapeutics Germany GmbH

• Background for the oligonucleotides PMOs / PPMOs and the MoA for RNA-exon skipping
• I.v. PMOs / PPMOs for RNA-exon skipping in DMD
• Clinical results for the PMOs / PPMOs from the Sarepta pipeline in DMD
 

11:30 siRNA Therapeutics for Ocular Indications

Elena Feinstein

Elena Feinstein, Chief Scientific Officer, Quark Pharmaceuticals

• Advances in platform technology
• Proof of concept data from non-clinical studies in ocular indications will be discussed
• Exploring proof of concept data from clinical studies in ocular indications
 

12:05 Immunochemotherapeutic oligonucleotides for cancer treatment

Steve Pascolo

Steve Pascolo, Founder and CEO, Miescher Pharma GmbH
View Bio

• Introducing the immunochemotherapeutic oligonucleotide approach
• Highlighting success in oncology mouse models to date
• Addressing challenges of delivery and maximising the EPR effect
• Vaccination with RNA
• Discussing next steps of the programs and evaluating potential delivery solutions
 

12:40 Networking Lunch

13:40 Introduction of non-chiral phosphorodithioates into Locked Nucleic Acids

Meiling LI

Meiling LI, Senior Scientist , F. Hoffmann-La Roche Ltd.
View Bio

• RNA therapeutics have clearly demonstrated their medical benefit.
• Phosphorothioates have been extensively profiled and cover most of the clinically investigated entities.The complexity of diastereoisomers have resulted in the great challenges in understanding PK/PD of stereomixed oligonucleotides.
• Introduction of non-chiral phosphorodithioates dramatically reduces the diastereomeric complexity and can potentially improve PK/PD of oligonucleotides.
• Combinations of phosphorodithioates and stereodefined motives will allow to rapidly identify highly potent one single phosphorothioate LNA isomer, which is supported by recent in vitro and in vivo data.
 

14:15 The UK Nucleic Acid Therapy Accelerator (NATA)

Loic Roux

Loic Roux, Head of Chemistry, NATA
View Bio

• A background and introduction to the aims of the NATA in supporting the development of synthetic RNA therapeutics
• The NATA Hub: a world leading research institute to support the RNA therapeutics community, providing dedicated capability in oligo synthesis and biological screening
• The NATA’s upcoming funding calls to address barriers in the manufacture and the delivery of RNA therapeutics
• Opportunities to partner with the NATA Hub; Q&A session
 

14:50 Formulation strategies for the development of injectable Spiegelmer drug product solutions

Stefan Vonhoff

Stefan Vonhoff, VP Chemistry&Manufacturing, NOXXON Pharma AG
View Bio

• Introducing the Spiegelmer technology approach and NOXXON’s clinical development program
• Defining a target product profile for injectable Spiegelmer drug product solutions
• Overcoming development challenges for Spiegelmers drug product solutions
• Establishing quality control methods for Spiegelmer drug product manufacturing and release in accordance with guidelines
 

15:25 Afternoon Tea

15:55 Exploring the chemistry of oligonucleotide therapeutics

Jimmy Weterings

Jimmy Weterings, Principal Scientist, AstraZeneca
View Bio

• Examining further advances in chemistry that are in the pipeline of oligonucleotide drug candidates
• Considering the possible effect of delivery systems that may provide extra footholds to improve the potency and specificity of oligonucleotide drugs
• Focusing on strategies to imbue antisense oligonucleotides with more drug-like properties and their applicability to other nucleic acid therapeutics
 

16:30 Fluorescent Base Analogs (FBAs) in Gapmer Technology: Stealth Labeling of Antisense

Tom Baladi

Tom Baladi, Postdoctoral Research Fellow, AstraZeneca
View Bio

• Outline of the design and assets of FBAs: modifying nucleobases to gain fluorescence properties without using external dyes
• Synthesis of FBA phosphoramidites and incorporation into ASOs
• Synthesis of FBA triphosphates and enzymatic incorporation into mRNA
• Assessment of structural, biological and photophysical properties of labelled constructs.
 

17:05 Chair’s Closing Remarks and Close of Day One

Nagy Habib

Nagy Habib, Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
View Bio

8:30 Registration & Coffee

9:00 Chair's Opening Remarks

Nagy Habib

Nagy Habib, Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
View Bio

9:10 Treating diseases with antibody oligonucleotide conjugates: Combining the selectivity of antibodies with the specificity of oligonucleotide drugs

Arthur A Levin

Arthur A Levin, Chief Scientific Officer, Avidity Biosciences Inc
View Bio

• Antibody oligonucleotide conjugates utilize the selectivity of antibody delivery and the specificity of oligonucleotide therapeutics to treat diseases
• AOC1001 is designed to treat myotonic dystrophy type 1
• Other AOCs are in development to address muscle disease and other disorders
 

9:45 Delivery of Novel siRNA constructs for treating cancer

David Evans

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

• Creating siRNA payloads for cancer treatment
• Understanding the challenges of cell-specific delivery: how to get size and efficacy right?
• Choosing the appropriate delivery system
• Presenting success and future steps

10:20 Morning Coffee

10:55 Recent clinical progress with RNAi loaded KRAS-LODER for pancreatic tumour

Amotz Shemi

Amotz Shemi, CEO, Silenseed

• Introducing the RNAi loaded KRAS-LODER for targeting KRAS mutations
• Overviewing recent clinical success in phase II trials
• Presenting phase II clinical trials results including primary and secondary endpoints
• Outlining next steps of programme

11:30 INT-1B3 (miR-193a-3p mimic): from bench-to-bedside

Michel Janicot

Michel Janicot, CDO, InteRNA Technologies BV

• Focus on microRNA R&D
• Success (bench-to-bedside) story for Oncology therapeutic intervention
• Preclinical to first in human study transition
 

12:05 Targeted Delivery of C/EBPa-saRNA by RNA Aptamers

Nagy Habib

Nagy Habib, Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
View Bio

• Understanding the principles of RNA activation and possibilities of therapeutic benefit
• Harnessing small activating RNAs for the treatment of Pancreatic ductal adenocarcinoma
• Evaluating aptamers and appropriate means of targeted delivery
• Looking at lessons learnt from using saRNA for oncological purposes
 

12:40 Networking Lunch

13:40 Small activating RNAs – a novel therapeutic class of oligonucleotides

Matthew Catley

Matthew Catley, Research Director, MiNa Therapeutics Ltd
View Bio

• Description of saRNA technology to upregulate transcription and broad applicability to a range of therapeutic targets
• Generation of a lead candidate saRNA to HNF4a for liver disease
• Update on MiNA’s lead saRNA clinical agent MTL-CEBPA – Clinical PD and efficacy
 

14:15 MicroRNA Therapeutics: Targeting the Pathways of Human Disease

Ekkehard Leberer

Ekkehard Leberer, Senior Director, R&D Alliance Management, Sanofi
View Bio

• MicroRNAs are short non-coding RNAs that regulate biochemical pathways by RNA interference (RNAi). Dysregulation of microRNAs is associated with many diseases.
• MicroRNA-21 has been implicated with fibrotic diseases and cancer.
• The presentation shows the development of an anti-fibrotic antisense oligonucleotide (anti-miR-21) for the treatment of a genetic fibrotic kidney disease called Alport Syndrome, and summarizes the potential of anti-miR-21 as a drug to treat hepatocellular carcinoma.
 

14:50 The tool-box approach to improve the performance of siRNA-platform technology

Adrien Weingartner

Adrien Weingartner, Principal Scientist, Group Leader Drug Delivery, Silence Therapeutics AG

• Interplay of design and function
• Stability and duration of action
• Linker design and valency of ligands
• Is there room for additional improvements?
 

15:25 Afternoon Tea

15:55 Targeted cytoplasmic delivery of oligonucleotides

Guy Hermans

Guy Hermans, Chief Executive Officer, Sapreme Technologies B.V.
View Bio

• Sapreme’s SPT001 compound improves cellular delivery of non-permeable biomolecules such as nucleic acids by up to 1000 fold by enhancing their escape from the endosome
• SPT001 conjugation to a targeting ligand can drive payload internalization and cytoplasmic delivery in a cell- or tissue specific manner – further improving the therapeutic window of any given antisense oligo or siRNA
• We will highlight recent proof-of-principle studies on nucleic acid delivery in vitro and in vivo
 

16:30 Clinical development of AsiDNA, a first in class decoy agonist oligonucleotide targeting DNA damage response in tumor cells

Wael Jdey

Wael Jdey, Head of Biology, Onxeo S.A.
View Bio

• Introduction to platON™: proprietary chemistry platform based on a library of decoy agonist oligonucleotides which generates disruptive compounds acting on intracellular DNA-binding targets
• Introduction to AsiDNA the leading decoy agonist generated from platON and targeting DNA damage response function
• Recent preclinical highlights: AsiDNA abrogates resistance to multiple anti-cancer targeted therapies
• Recent clinical development of AsiDNA
• Outlining next steps of clinical development
 

17:05 Chair’s Closing Remarks and Close of Day Two

Nagy Habib

Nagy Habib, Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
View Bio

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FEATURED SPEAKERS

Adrien Weingartner

Adrien Weingartner

Principal Scientist, Group Leader Drug Delivery, Silence Therapeutics AG
Amotz Shemi

Amotz Shemi

CEO, Silenseed
Andre  Muller-York

Andre Muller-York

Senior Medical Affairs Director, Sarepta Therapeutics Germany GmbH
Maria Luisa Pineda

Maria Luisa Pineda

CEO and Co-founder, Envisagenics
Meiling LI

Meiling LI

Senior Scientist , F. Hoffmann-La Roche Ltd.
Nagy Habib

Nagy Habib

Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
Stefan Vonhoff

Stefan Vonhoff

VP Chemistry&Manufacturing, NOXXON Pharma AG
Suzan Hammond

Suzan Hammond

Research Assistant, University Of Oxford
Tom Baladi

Tom Baladi

Postdoctoral Research Fellow, AstraZeneca
Wael Jdey

Wael Jdey

Head of Biology, Onxeo S.A.

Adrien Weingartner

Principal Scientist, Group Leader Drug Delivery, Silence Therapeutics AG
Adrien Weingartner

Alexey Wolfson

CEO, Advirna
Alexey Wolfson

Amotz Shemi

CEO, Silenseed
Amotz Shemi

Andre Muller-York

Senior Medical Affairs Director, Sarepta Therapeutics Germany GmbH
Andre  Muller-York

Arthur A Levin

Chief Scientific Officer, Avidity Biosciences Inc
Arthur A Levin

Arthur A Levin

Chief Scientific Officer, Avidity Biosciences Inc
Arthur A Levin

Arthur Levin is a leader in RNA therapeutics field. He serves as CSO at Avidity Biosciences. Previously, he held Senior level positions at mRiragen, Santaris Pharma, and Ionis Pharmaceuticals. He played key roles in developing numerous oligonucleotides including the first approved antisense drugs and the first microRNA-targeted therapeutic in clinical trials. He has three decades of experience in drug development from discovery through drug registration. Dr. Levin has published over 60 scientific articles, including highly cited reviews. He is on the scientific advisory boards of multiple institutions. Art received a doctorate in toxicology from the University of Rochester.
 

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 built scientific research teams focused on target validation, drug discovery and high throughput screening of small molecule and siRNA libraries to identify lead candidates for development in Oncology and other indications. David has more than 15 years’ experience in the RNAi field – building genome scale screening capabilities for target identification in Oncology and other indications at TGEN and Dharmacon prior to cofounding Sirnaomics to focus on developing siRNA as a therapeutic. David previously worked at the Frederick National Lab for Cancer Research and in the Boston area with Millennium Pharmaceuticals and Serono Pharmaceuticals.

Ekkehard Leberer

Senior Director R&D Alliance Management, Sanofi
Ekkehard Leberer

Industry Park Hoechst, D-65926 Frankfurt, Germany

Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986), and conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada. He then obtained the Habilitation for Professor of Biochemistry at the University of Konstanz, Germany (1992). 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.
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. Since 2012, he is Global Alliance Manager for R&D at Sanofi, Frankfurt. 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 (www.compact-research.org), Brussels. He serves as a member in Scientific Advisory Boards of several biotechnology companies, and he is the Head of the Supervisory Board of BioM, Munich.
His 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.
 

Ekkehard Leberer

Senior Director, R&D Alliance Management, Sanofi
Ekkehard Leberer

Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986), and conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada. He then obtained the Habilitation for Professor of Biochemistry at the University of Konstanz, Germany (1992). 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. 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. Since 2012, he is Global Alliance Manager for R&D at Sanofi, Frankfurt. 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 (www.compact-research.org), Brussels. He serves as a member in Scientific Advisory Boards of several biotechnology companies, and he is the Head of the Supervisory Board of BioM, Munich. His 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.
 

Guy Hermans

Chief Executive Officer, Sapreme Technologies B.V.
Guy Hermans

Guy joined Sapreme as CEO in 2020. He brings in over 15 years of experience in antibody fragment discovery and early development, as well as antibody discovery technology development.
In his previous position as CSO at Isogenica Ltd., he led internal technology and product development, as well as drive the scientific interactions with licensees and other partners. The re-engineering of the company and its product portfolio allowed it to become consistently profitable, and multiple leads discovered there have now entered the clinic.
Prior to this, Guy was Research Fellow at Ablynx where he conceived of many of its internal drug development programs and led a number of them through preclinical development. He was also responsible for several technology development programs, addressing challenging target classes or enabling novel applications for domain antibodies.
During his academic training, Guy did research in the pathogenesis and immunotherapy of autoimmune and inflammatory disorders, ranging from cell-based immunotherapy clinical trials in Multiple Sclerosis patients to target discovery and validation work at Stanford University Medical School.
 

Guy Hermans

Chief Executive Officer, Sapreme Technologies B.V.
Guy Hermans

Guy joined Sapreme as CEO in 2020. He brings in over 15 years of experience in antibody fragment discovery and early development, as well as antibody discovery technology development.
In his previous position as CSO at Isogenica Ltd., he led internal technology and product development, as well as drive the scientific interactions with licensees and other partners. The re-engineering of the company and its product portfolio allowed it to become consistently profitable, and multiple leads discovered there have now entered the clinic.
Prior to this, Guy was Research Fellow at Ablynx where he conceived of many of its internal drug development programs and led a number of them through preclinical development. He was also responsible for several technology development programs, addressing challenging target classes or enabling novel applications for domain antibodies.
During his academic training, Guy did research in the pathogenesis and immunotherapy of autoimmune and inflammatory disorders, ranging from cell-based immunotherapy clinical trials in Multiple Sclerosis patients to target discovery and validation work at Stanford University Medical School.
 

Jimmy Weterings

Principal Scientist, AstraZeneca
Jimmy Weterings

Jimmy Weterings

Principal Scientist, AstraZeneca
Jimmy Weterings

Jimmy has had a tight bond with Oligonucleotide Therapeutics since 2002, performing research on PNA in the van Boom group at Leiden University and targeted ASO conjugates at Prosensa. During his Ph.D (Bio-organic Chemistry) at Leiden University he synthesised and studied CpG and other TLR ligand containing conjugates. At Cenix BioScience, he developed targeted siRNA conjugates for CNS delivery. And at Cristal Therapeutics he lead various projects on the nanomedicine delivery of oligonucleotide in oncology. In 2019, Jimmy joined AstraZeneca, (Gothenburg, SE) fully dedicating himself to the further advancement of (targeted) oligonucleotide therapeutics. Jimmy (co-)authored 11 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.

 

Loic Roux

Head of Chemistry, NATA
Loic Roux

Dr. Loïc Roux received his Ph.D in Medicinal Chemistry from Aix-Marseille University in France. His work focused on the development of new nucleotides pro-drugs against HIV focusing on the phosphorus chemistry. He joined Prof. Khvorova's lab (UMass Med School, MA, USA) in 2018 working on the stabilization and delivery of fully chemically modified oligonucleotides for neurodegenerative diseases and then Prof. Wood's lab (University of Oxford, UK) working on peptide-oligonucleotides conjugates for muscular degenerative diseases. After a position as principal scientist at PepGen Limited promoting a peptide delivery platform for nucleic acid delivery, he is currently the Head of Chemistry of NATA, a new UKRI initiative aiming to promote the development of nucleic acid therapeutics.

Loic Roux

Head of Chemistry, NATA
Loic Roux

Maria Luisa Pineda

CEO and Co-founder, Envisagenics
Maria Luisa Pineda

Dr. Maria Luisa Pineda is the co-founder and CEO of Envisagenics, Inc. a biotech spinout of Cold Spring Harbor Laboratory that integrates proprietary machine learning algorithms, high performance computing, and RNA-splicing analytics to identify disease-specific splicing variants as therapeutic targets. Envisagenics has leveraged its SpliceCore® software platform for therapeutic development with its first pipeline of assets in triple negative breast cancer. Dr. Pineda received her PhD from the prestigious Watson School of Biological Sciences at Cold Spring Harbor Laboratory, then she acquired investment experience in technology and life-science startup companies at Canrock Ventures and Golden Seeds, LLC. Under Dr. Pineda’s leadership, Envisagenics has acquired nearly $2.5M in funding from the National Institutes of Health Small Business Innovation Research (SBIR) program and pre-seed investments from Topspin Partners and the Long Island Emerging Technology Fund, and from Breakout Labs, a Peter Thiel foundation. Envisagenics has raised over $5M from investors including Third Kind Venture Capital (3KVC), Cosine, LLC (NYC biotech investors), Dolby Family Ventures, Dynamk Capital, Madrona Venture Group, M12, NY Empire State Development (ESD), and SV Angel. Envisagenics has won several prestigious competitions such as the JLABS Artificial Intelligence for Drug Discovery QuickFire Challenge and was named the North American Winner of the Innovate.AI competition. More recently, Dr. Pineda secured Envisagenics’ first oncology partnership with a large pharmaceutical company. Dr. Pineda was named one of the top 20 Latinas in NYC by El Diario, NYC’s oldest Spanish Language newspaper and is an alumna of the Stanford Latino Entrepreneur Leaders Program developed by the Latino Business Action Network and the Stanford Graduate School of Business.

Mark Edbrooke

Industrial Advisor , Nucleic Acid Therapy Accelerator
Mark Edbrooke

Mark Edbrooke is an Independent Scientific Consultant specialising in oligonucleotide therapeutics and macromolecule delivery. He worked for 20 years in GSK where he ran a transnational functional genomics department, and latterly a Discovery Performance Unit (DPU) focused on the development of therapeutic siRNAs and antisense oligonucleotides. He then joined AstraZeneca (AZ) to work on therapeutic antisense, microRNA, and modified RNA. He now consults for numerous UK-based universities, as well as for the NATA, a government-funded accelerator for nucleic acid therapeutics.

Matthew Catley

Research Director, MiNa Therapeutics Ltd
Matthew Catley

Dr Matthew Catley completed his PhD studies of airway signalling pathways involved in lung disease with Peter Barnes at Imperial college. He completed further post-doctoral studies on the pharmacology of inhaled therapeutics with Maria Belvisi at Imperial College. Moving to UCB/Celltech he worked on monoclonal antibodies for the treatment of autoimmune and inflammatory disorders. In 2012 he moved to AstraZeneca in Gothenburg to help establish and run the preclinical bioscience department. After a year at Covance, Matthew Joined MiNA therapeutics in 2019 as Research Director. Matthew has developed a range of therapeutic modalities including small molecules, antibodies, peptides and oligonucleotides.

Meiling LI

Senior Scientist , F. Hoffmann-La Roche Ltd.
Meiling LI

Dr. Meiling Li is currently working as a senior scientist in RNA therapeutics in Hoffmann La Roche, Basel, Switzerland. Her research focuses on the modifications of the backbones in terms of stereodefined phosphorothioates, phosphorodithioates and other highly interested modifications. Dr. Li received her PhD from University of Oxford (UK) in organic synthetic chemistry. She then worked at ETH Zurich as a postdoc on nucleoside chemistry. In 2017 she moved to Hoffmann La Roche as a scientist in RNA therapeutics research department. Dr. Li has been involved in different projects of drug development and contributed greatly to the understanding of structure activity relationship (SAR) of oligonucleotide drugs.

Michel Janicot

Chief Development Officer, InteRNA Technologies BV
Michel Janicot

Michelle Lynn Hall

Senior Director, Eli Lilly & Co - New Therapeutic Modalities
Michelle Lynn Hall

Michelle Lynn Hall is currently the Senior Director of New Therapeutic Modalities at Eli Lilly, where she leads a team of engineers and scientists in the discovery of nucleic-acid-based therapies. Immediately prior to joining Lilly, Michelle worked at Moderna, where she led a team of modelers studying mRNA therapeutics design and lipid nanoparticle delivery. She received her Ph.D. from Columbia University where she developed a novel method to predict chemical reactivity and applied these to skills to a diverse set of problems ranging from materials science to biocatalysis. She then completed a joint postdoc at Novartis Institutes for Biomedical Research and Yale University, where she studied computer-aided drug design. She subsequently worked at Schrödinger where she focused on molecular simulations, machine learning, and chemical biology. She is passionate about equity, diversity, inclusion, and awareness in science, technology, engineering and math (STEM) and has served as a mentor, champion and advocate for women and minorities throughout her career. As a native Texan, she has intolerably strong opinions about BBQ.

Michelle Lynn Hall

Senior Director, Eli Lilly & Co - New Therapeutic Modalities
Michelle Lynn Hall

Nagy Habib

Professor of Surgery, Imperial College, Co-founder MiNA therapeutics, Imperial College London
Nagy Habib

Nagy Habib is a leading translational researcher on liver cancer and its treatment. He pioneered the first clinical trial in the use of adenovirus and plasmid for the treatment of liver cancer, as well as the use of plasmid gene therapy in hydrodynamic gene delivery. He is the first person in the West to publish a clinical trial on the use of adult bone marrow derived stem cells for the treatment of patients with liver insufficiency.

He has published on the evolution of molecular biology of tumours from oncogene, tumour suppressor gene, epigenetic modification, gene therapy, stem cell therapy, RNA (Ribonucleic acid) and saRNA (small activating RNA) and RNA aptamers.

He is the inventor of, and was co-author of the first publication about the use for radiofrequency in devices used in liver surgery (Habib™ 4X), as well as interventional endoscopy (Habib™ EndoHPB – HPB: Hepatopancreatobiliary) and interventional radiology (Habib™ Percutaneous HPB, Habib™ VesCoag, Habib™ VesOpen).

Nagy is currently Lead Clinician and Head of the Department of HPB Surgery at Imperial College London. In June 2007 he was also appointed Pro Rector for Commercial Affairs at the university. He holds a gold award from the Advisory Committee for Clinical Excellence, which is given in recognition of exceptional contributions from NHS consultants, and he was named as one of Britain’s top surgeons in December 2011 by the Saturday Times Magazine. In December 2012, he was awarded the TAKREEM AWARD for Science and Technology for scientific work in liver cancer and the technological development of the Habib 4X surgical device. In 2015, Nagy was awarded a Foreign Membership of the prestigious French Académie Nationale de Chirurgie.
 

Stefan Vonhoff

VP Chemistry&Manufacturing, NOXXON Pharma AG
Stefan Vonhoff

Stefan Vonhoff, Ph.D. is VP Chemistry&Manufacturing at NOXXON Pharma AG, Berlin, Germany, a biotech company developing Spiegelmers – enantiomeric aptamers – for various therapeutic indications in the oncology area.
Stefan holds a Ph.D. in chemistry from University College London and received post-doctoral training in the Department of Chemistry at the ETH Zurich. He joined NOXXON in 1999 to establish the manufacturing process for L-nucleoside phosphoramidites and pegylated L-RNA aptamers. Today, his main responsibility is to coordinate external manufacturing and QC/QP-release activities for starting materials, drug substance, parenteral drug product and clinical trial supply.
 

Steve Pascolo

Founder and CEO, Miescher Pharma GmbH
Steve Pascolo

Trained as an immunologist at the Pasteur Institute (Paris, France), I used mouse models to test and develop mRNA-based vaccines (direct injection of mRNA) during my post-doc in Tuebingen, Germany. In 1999, I co-founded CureVac with Dr. Hoerr and Dr. von der Mulbe. I was CSO of the company from 2000 till 2006, developing the technology, implementing the worldwide first GMP production of mRNA and performing the worldwide first clinical studies where humans got injections of in vitro transcribed mRNA. In 2006 I joined the oncology department of the University Hospital of Zurich, Switzerland and continued the development of immunotherapies based on RNA. In 2008 I founded Miescher Pharma to support this work. In 2017, we implemented in Zurich an mRNA platform https://www.cancer.uzh.ch/en/Research/mRNA-Platform.html. In collaboration with several research and clinical departments in Zurich we test different mRNAs capable to enhance immunity against cancer. In addition, we developed modified RNA oligonucleotides to perform immunochemotherapies.

Suzan Hammond

Research Assistant, University Of Oxford
Suzan Hammond

Suzan Hammond, PhD, is a career research scientist with experience focused on neuromuscular genetic disorders. Dr Hammond received her degree from Northwestern University studying in vivo modelling for the neurodegenerative disease spinal muscular atrophy. As a research scientist at the University of Oxford, Suzan focuses on patient unmet needs by developing delivery vehicles for antisense oligonucleotides towards the brain, spinal cord and skeletal muscle. She works with academic and industrial collaborators to advance her work to early clinical development. Dr Hammond’s work has been featured on the Naked Scientist Podcast and Zone in with Zon for trending nucleic acid research.

Tom Baladi

Postdoctoral Research Fellow, AstraZeneca
Tom Baladi

Tom Baladi was awarded his Ph.D. in Medicinal Chemistry in 2016 following his work on the design and development of selective small molecule kinase inhibitors at the Paris-Saclay University. For his second post-doctoral fellowship, he joined in 2018 the Wilhelmsson research group at Chalmers University (Gothenburg, Sweden) as part of the FoRmulaEx platform, a Swedish industry/academia consortium. He now focuses on the synthesis of fluorescent nucleobase analogs and their incorporation into various nucleotide-based modalities for in vitro imaging, to enable studies of drug-delivery systems. His work is performed at AstraZeneca Gothenburg, another active member of FoRmulaEx.

Vera Brinks

Director Pharmaceutics, ProQR Therapeutics
Vera Brinks

Vera Brinks is director pharmaceutics at ProQR Therapeutics, Leiden, the Netherlands. With her team she is responsible for early and late phase oligonucleotide DP development. Vera received her PhD in 2009 in the field of biopharmaceutics at the Leiden University (The Netherlands). Before joining ProQR 6 years ago, Vera has worked as postdoctoral fellow at the FDA (CDER, DTP), and as postdoctoral researcher at the Utrecht University (the Netherlands) for 6 years. She has over 25 publications in the field of therapeutic proteins, biosimilars and formulation-related quality attributes.

Wael Jdey

Head of Biology, Onxeo S.A.
Wael Jdey

As a scientist with an interest in health and disease, in particularly cancer, I aim to contribute to the understanding and targeting of this complex disease. My background in oncology, cell biology, signaling pathways and DNA repair showed me that interdisciplinary knowledge is the key in tackling complex diseases such cancer; Inter-group collaboration and team work are definitely key components to success. I am always looking to evolve in a stimulating environment where my skills and interdisciplinary knowledge can participate in the development of solutions for a medical unmet need to improve patient life quality.

Exhibitors

Official Media Partner

Supporting Media Partners

Workshops

Target Discovery for RNA Therapeutics
Workshop

Target Discovery for RNA Therapeutics

Holiday Inn Kensington Forum
21st September 2021
London, United Kingdom

VENUE

Holiday Inn Kensington Forum

97 Cromwell Road , London, United Kingdom

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

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

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

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

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

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 SMi Group. SMi 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

Speaker Interview - Maria Luisa Pineda, Envisagenics

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RNA infographic

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P-328 RNA Past Atendee list (P-311)

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David Blakey - MiNA therapeutics, Past presentation

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Christopher Bunker- Bio-techne, Past presentation

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Heinrich Haas - BioNTech, Past presentation

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Surender Vashist - AstraZeneca, Past presentation

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Ethris, Past presentation

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Dr Simon Newman - NanoGenics, Past presentation

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Davis C. Henchall - Future neuro, Past presentation

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Dr Kirsty Wydenbach - MHRA, Past presentation

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David Deutsch - MHRA, Past presentation

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Bo Rode Hansen - GENEVANT, Past presenation

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Dr. Michel Janicot - InteRNA Technologies, Past presentation

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Brochure 2020

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Short Programme

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Full programme

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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.


GenScript

Exhibitors
https://www.genscript.com/

GenScript is a world leader in biotechnology reagent services, providing life sciences services and products to over 200,000 scientists in over 100 countries worldwide. Established in 2002 in New Jersey, United States, the company was one of the first to commercialize gene synthesis as well as establish fully integrated capabilities for custom peptide synthesis, complex protein expression and engineering, custom antibody development and engineering, in vitro/in vivo pharmacology, as well as a variety of other research-focused catalogue products.

After almost two decades of rapid growth, the company has expanded its business in recent years into the fields of immunotherapy, CDMO and microbiology to further its core mission of making people and nature healthier through biotechnological innovation.

With global support from its loyal customers and over 2600 employees located across the globe, GenScript continues to strive towards their vision of being the most reliable biotech company in the world, in service of a better and healthier future.



Tosoh Bioscience

Exhibitors
https://www.tosohbioscience.com/

Tosoh Bioscience is an acknowledged global leader in liquid chromatography with a focus on bioseparations. Our team of chromatography experts enables our biopharma partners to provide safe and efficient therapies against life threatening diseases. Our portfolio encompasses SEC instruments and a comprehensive line of media and process development, HPLC, and UHPLC columns. The products are used in R & D, process development, downstream processing, and quality control of biologics, biosimilars or biobetters. Typical applications comprise the purification of therapeutic proteins and oligonucleotides at lab, pilot, and commercial scale, as well as their characterization and QC analysis by (U)HPLC. Tosoh Bioscience is part of the Tosoh Group, a Japanese chemical and specialty products group with over 100 companies worldwide and a workforce of about 13000 people.

Media Partners


Pharma Journalist

Official Media Partner
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.

Media Partners


World Pharma News

Supporting Media Partners
http://www.worldpharmanews.com/



Pharmacircle

Supporting Media Partners
http://www.pharmacircle.com

PharmaCircle is an innovative knowledge management company serving the current and future global leaders in the Pharmaceutical and Biotechnology related industries. PharmaCircle is a one stop information and analysis source for pipeline, products, clinical trials, drug delivery technologies, deals and acquisitions, company financials, venture capital investments, product sales, pharmaceutical services, news, patents and more….


ASD MEDIA

Supporting Media Partners
http://www.asd-network.com

Professionals in the Aerospace & Defence market use the ASD Media internet platforms to:
  • Be informed on the latest market developments; www.asd-network.com
  • Find the latest business news; www.asd-network.com
  • Find the upcoming events; www.asd-network.com
  • Find companies and organizations; www.asdsource.com
  • Distribute news globally. www.asdwire.com
The ASD Media platforms are well known and used intensively, your company is able to benefit from these large numbers of users. Be informed with ASD-Network; Create exposure for your company with ASDSource, distribute your news with ASDWire or advertise with us, build your brand and increase traffic to your company’s website.

For more detailed information please contact with: ASD MEDIA
Stefan.koopman@asdmedia.nl


pharmaphorum

Supporting Media Partners
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


Drug Discovery Today

Supporting Media Partners
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.


Gene Therapy Net

Supporting Media Partners
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


Pharmaceutical Technology

Supporting Media Partners
http://www.pharmaceutical-technology.com

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


Technology Networks

Supporting Media Partners
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


Farmavita

Supporting Media Partners
https://farmavitar.com

FarmavitaR+ is the professional network of experts and service providers. Network is gathering local consultants from 90 countries in Europe, Asia, North America, Latin America, Australia and Africa. Management of international, multi-centre projects is our core competence. FarmavitaR+ is providing solutions related to pharmaceutical, medical device, food supplement and cosmetic products. Scope of services is related to solutions for product development, quality assurance, clinical trials, product registration, portfolio analysis, lifecycle management, vigilance/risk management, pricing/reimbursing, market access and promotional compliance. FarmavitaR+ is brand name of Farmavita Regulanet Ltd. Visit https://farmavitar.com for more information. Outsource anything you can think of!


SciDoc Publishers

Supporting Media Partners
http://www.scidoc.org

SciDoc Publishers is a major source provider of e-journals in the field of Science, Technology and Medicine (STM). The nature of journals - Open Access and Peer-reviewed. We are aimed with a sole motive in making a mark in the field of Open Access, by propagating the knowledge to the scientific community. Our prime concern involves, the knowledge to reach millions of readers and give them access to scientific publications - online.


Pharmalicensing

Supporting Media Partners
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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Holiday Inn Kensington Forum

97 Cromwell Road
London SW7 4DN
United Kingdom

Holiday Inn Kensington Forum

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

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

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

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

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

HOTEL BOOKING FORM

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Description

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WHAT IS CPD?

CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

CPD AND PROFESSIONAL INSTITUTES

There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

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

Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

As a formal provider of CPD certified activities, SMI Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

GLOBAL CPD

Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

CPD Certificates

We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@smi-online.co.uk

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