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RNA Therapeutics

SAE Media Group announces the 11th Annual RNA Therapeutics Conference in London on February 19th - 20th 2020.


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

The global antisense and RNA therapeutics market size is expected to expand at a compound annual growth rate (CAGR) of 8.6% between 2019 and 2025, when it is projected to reach $1.81 billion. RNA-based drugs, including short interfering RNAs and antisense oligonucleotides, are particularly promising examples of this newer class of biologics. For over two decades, researchers have been trying to overcome major challenges for utilizing such RNAs in a therapeutic context, including intracellular delivery, stability, and immune response activation. Furthermore, the recent advent of CRISPR, an RNA-guided gene-editing technology, as well as new strides in the delivery of messenger RNA transcribed in vitro, have triggered a major expansion of the RNA-therapeutics field.


This year, we will maintain our focus on the latest research and developments on delivery system technologies that aim to tackle more difficult targets within the human body. This overarching topic warrants high focus and therefore we will, for the first time in the series, introduce a focus day on Oligonucletide Delivery Systems. Our main conference will focus on current developments in the area of mRNA therapeutics in immunotherapy and vaccination. We will also cover a fair bit of regulation, such as continuing upon patenting novel products, release of new 2020 regulations in the clinical trials arena, as well as ensuring compliance. Finally, a significant section that tackles novel and emerging therapies as well as the latest research findings from academia will also be present in this year’s agenda.


Join us in February 2020, as SAE Media Group’s RNA Therapeutics Conference brings together industry experts from leading RNA therapeutics companies to discuss the challenges for clinical translation of RNA-based therapeutics, with an emphasis on recent advances in delivery technologies, and present an overview of the applications of RNA-based drugs for modulation of gene and protein expression, and genome editing.
 

 

FEATURED SPEAKERS

Bo Rode Hansen

Bo Rode Hansen

President & CEO, Genevant Sciences
Christian Plank

Christian Plank

Managing Director, CSO, Ethris GmbH
Christiane Niederlaender

Christiane Niederlaender

Director, AMBR Consulting Ltd
Christopher Bunker

Christopher Bunker

Sr. Director of Business Development, Advanced Cell Diagnostics, A Bio-Techne Brand
David  Blakey

David Blakey

Chief Scientific Officer, MiNA Therapeutics
David  Henshall

David Henshall

Professor of Molecular Physiology & Neuroscience, RCSI
Ekkehard Leberer

Ekkehard Leberer

Senior Director, Sanofi
Heinrich Haas

Heinrich Haas

VP RNA Formulation and Drug Delivery , BioNTech AG
Jeffrey Ulmer

Jeffrey Ulmer

Head, Preclinical R&D US, GSK
Kirsty Wydenbach

Kirsty Wydenbach

Deputy Unit Manager, Clinical Trials Unit, Medicines & Healthcare products Regulatory Agency (MHRA)
Klaus Giese

Klaus Giese

CEO, Pantherna Therapeutics GmbH
Michel Janicot

Michel Janicot

CDO, InteRNA Technologies BV
Robin Shattock

Robin Shattock

Professor of Mucosal Infection and Immunity, Imperial College London
Shalini  Andersson

Shalini Andersson

Senior Director and Head of New Modalities, AstraZeneca
Simon Newman

Simon Newman

CSO, Nanogenics Ltd
Steve Pascolo

Steve Pascolo

Founder and CEO, Miescher Pharma GmbH
Steven Powell

Steven Powell

CEO, eTheRNA
Surender Vashist

Surender Vashist

RNA Engineering Scientist, Medimmune

Bo Rode Hansen

President & CEO, Genevant Sciences
Bo Rode Hansen

Bo Rode Hansen, MBA, PhD joined Genevant in October 2018, taking on the roles of President and Chief Executive Officer in November 2018. Previously, Bo served as Global Head of RNA Therapeutics and General Manager of the Roche Innovation Center Copenhagen. Prior to Roche, he was Executive Vice President and Head of Drug Discovery & Alliance Management at Santaris Pharma (acquired by Roche). Over the course of his career, Bo has led over 50 RNA therapeutic discovery programs resulting in 10 clinical candidates and over 40 patents and patent applications. He holds a PhD from Kobenhavns Universitet, an MS in Biochemistry from the University of Copenhagen and a BS from Universita Degli Studi di Pavia.

Christian Plank

Managing Director, CSO, Ethris GmbH
Christian Plank

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

Christiane Niederlaender

Director, AMBR Consulting Ltd
Christiane Niederlaender

Christiane has spent 12+ years in medicines, tissue, cell and gene therapy regulation. From 2011 to August 2019 she has worked at the MHRA, the UK’s medicine regulator, where she has assessed all classes of biological medicines at both UK and EU level and acted as the UK representative at the European Medicines Agency’s (EMA) committee for advanced therapies (CAT). Christiane was the Rapporteur/ Co-Rapporteur for several advanced therapy medicinal products (ATMPs) and many other biological product applications, including biosimilars. She acted as the Rapporteur and drafting group member for CAT guidelines. Christiane was actively involved in the MHRA's Brexit preparations and has in-depth knowledge of the MHRA no-deal provisions.
Prior to joining the MHRA, Christiane worked for the UK competent authority responsible for the European Tissues and Cells Directive as an assessor and inspector. She has a Ph.D. and research background in cell-signalling, cancer and stem cells and obtained a law degree in 2005.
Christiane has set up her own consultancy company in September 2019.
 

Christopher Bunker

Sr. Director of Business Development, Advanced Cell Diagnostics, A Bio-Techne Brand
Christopher Bunker

Dr Christopher Bunker is the Sr. Director of Business Development at Advanced Cell Diagnostics (ACD) in Newark, California. Dr Bunker did his doctoral research in Biological Chemistry at Harvard University and a Leukaemia Society of America postdoctoral fellowship in molecular genetics at University of California, San Francisco. He was a senior scientist in cancer functional genomics at Genome Therapeutics before joining Cell Signaling Technology at its inception in 2000. Dr. Bunker received an M.B.A from Boston University, and then joined Advanced Cell Diagnostics, where he has led its assay services business for the past nine years.

David Blakey

Chief Scientific Officer, MiNA Therapeutics
David  Blakey

I have a degree in Biochemistry from Oxford University, a PhD from London University and then worked as a Post Doc at the ICRF in London. In 1987 I joined AstraZeneca where I championed the company’s move into therapeutic antibodies and led the collaboration that developed Imfinzi.
I was appointed Chief Scientist in Oncology in 2008 and in my last 4-5 years at AstraZeneca I led a global cross therapy area team on oligonucleotide-based therapies. In October 2015, continuing my career in Oligonucleotide Therapeutics, I joined MiNA Therapeutics as their CSO and lead research on their small activating RNA platform.

David Henshall

Professor of Molecular Physiology & Neuroscience, RCSI
David  Henshall

David C. Henshall is Professor of Molecular Physiology & Neuroscience and Director of the FutureNeuro Research Centre at the Royal College of Surgeons in Ireland. He obtained his PhD in neuropharmacology from the University of Edinburgh, UK and undertook postdoctoral research at the University of Pittsburgh. His research focuses on the role of small noncoding RNAs in the development of brain diseases including epilepsy with special focus on microRNAs as diagnostic biomarkers and therapeutic targets. He is past-President of Neuroscience Ireland and the Chair of the International League Against Epilepsy’s Task Force on Genetics/Epigenetics.

 

David Deutsch

Pharmaceutical Assessor, MHRA
David Deutsch

Ekkehard Leberer

Senior Director, 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.
 

Heinrich Haas

VP RNA Formulation and Drug Delivery , BioNTech AG
Heinrich Haas

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

Jeffrey Ulmer

Head, Preclinical R&D US, GSK
Jeffrey Ulmer

Jeffrey B. Ulmer, Ph.D is Head, Preclinical R&D US based in Rockville, MD, USA. During his 30-year career in vaccines R&D at Merck Research Laboratories, Chiron Corporation, Novartis Vaccines and GSK, his leadership roles and responsibilities have included: Platform Technology Leader (DNA, RNA vaccines), Project Leader (Tuberculosis, SARS), Department Head (Immunology & Cell Biology), US Site Head for Research, Global Function Head (External Research), and Program Head (Technical R&D). He received his Ph.D. in biochemistry from McGill University and completed his postdoctoral training in the laboratory of Nobel laureate Dr. George Palade in the Department of Cell Biology at Yale University School of Medicine. He has published over 200 scientific articles and is an inventor on 11 patents.

Kirsty Wydenbach

Deputy Unit Manager, Clinical Trials Unit, Medicines & Healthcare products Regulatory Agency (MHRA)
Kirsty Wydenbach

 

Dr Kirsty Wydenbach is a Senior Medical Assessor and the Deputy Unit Manager in the Clinical Trials Unit, having joined the MHRA in 2009. She has been involved in the UK regulation of clinical trials across all therapy areas and all phases of development, including trials for chemical and biological products, Advanced Therapy Products and many first-in-man studies. She has also been involved in European discussions aiming to establish an EU harmonised approach to clinical trials, particularly for Developmental Safety Update Reports (DSURs) and Reference Safety Information (RSI). She was also an EMA expert for the update of the First-in-Human guideline. More recent EU priorities have included the new Clinical Trial Regulation and Kirsty currently sits on the safety subgroup of the Clinical Trials Facilitation Group (CTFG) that is working on how safety reporting aspects will be implemented. Other recent work has included collaboration with external industry groups and regulators regarding adaptive and novel trial designs: she is leading on this aspect for the MHRA in order to implement that aspect of the Life Sciences Industrial Strategy, and was a contributor to the EU CTFG “Recommendation Paper on the Initiation and Conduct of Complex Clinical Trials”.

Klaus Giese

CEO, Pantherna Therapeutics GmbH
Klaus Giese

Dr. Giese is a recognized expert with over 27 years of relevant experience including 17 years at the executive level (CEO, CSO). He has built research facilities and has managed research teams of up to 50 FTEs in both U.S. and Europe. Dr. Giese has broad knowledge in directing translational research into clinical development programs. He has a consistent record of managing collaborations with multinational companies across both the pharmaceutical and biotechnology sector as well as in academic institutions. He was instrumental in multiple financing transactions as well as M&A, divestments and strategic restructurings.

Michel Janicot

CDO, InteRNA Technologies BV
Michel Janicot

Michel holds a Ph.D. degree in Biochemistry (Paris VII. France) and was Postdoctoral Fellow in the Dept. of Biological Chemistry (M. Daniel Lane) at The Johns Hopkins University (Baltimore, MD. USA). He has over 25-years hands-on experience in Oncology preclinical and early clinical pharmaceutical drug development in increasing leadership and managerial roles at Rhone-Poulenc Rorer (Sanofi) and subsequently, at Janssen (Johnson & Johnson). Since 2012, Michel is the founder and Managing Director of JMi ONConsulting, a Brussels-based consulting firm specialized in providing services and guidances for research and development programs in Oncology, Inflammation, Immunology, Vaccines, COPD, MS, Diabetes, Fibrosis and Ophthalmology.

Robin Shattock

Professor of Mucosal Infection and Immunity, Imperial College London
Robin Shattock

Robin Shattock is a Professor of Mucosal Infection and Immunity at Imperial College, London. The main focus of his research is the investigation of the mechanisms of mucosal infection and development of novel vaccine strategies. He has secured funding from the MRC-UK, European Commission, EPSRC, CEPI, MRC and the Bill and Melinda Gates Foundation. He is the scientific director of the European AIDS Vaccine Initiative (EAVI2020) that coordinates a program of EU funded vaccine research across 22 institutions. He is the Director or the Imperial EPSRC Future Manufacturing Research Hub (FVMR) with the focus of advancing the manufacturing and deployment of vaccines.

 

Shalini Andersson

Senior Director and Head of New Modalities, AstraZeneca
Shalini  Andersson

Shalini is currently Chief Scientist, New Therapeutic Modalities, in AstraZeneca and responsible for AstraZeneca Biopharmaceuticals therapeutic modality strategy that will enable expansion of druggable target space. 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 drives several collaborations with external partners and academic groups across the globe.
 

Prior to this role, Shalini held several leadership roles within Cardiovascular, renal and metabolism.

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 45 peer reviewed articles, 2 book chapters and 4 patents.
 

Simon Newman

CSO, Nanogenics Ltd
Simon Newman

As Chief Scientific Officer of NanoGenics, Simon is responsible for the ongoing development of the in-house product pipeline and the continued development of the LipTide nucleic acid delivery technology. Simon has led oncology drug discovery projects and new company spinouts at Imperial College London for ten years. He has published over 60 peer-reviewed papers, which have been cited over 2000 times. Simon has worked with Ipsen, Sterix, and Oxandia Ltd and was Director of Research for Target Ovarian Cancer.

 

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.

Steven Powell

CEO, eTheRNA
Steven Powell

Steven has broad experience in several life science sectors in scientific, investment and commercial roles. He began his career with Beecham Pharmaceuticals (now GSK) and subsequently held senior roles with both public and private biotechnology and healthcare companies. He was CEO of London Stock Exchange listed KS Biomedix PLC from 1999 until its acquisition by Xenova in 2003, CEO of AIM (LSE) listed Plethora Solutions PLC until 2011, CEO of AIM listed Cambridge Cognition PLC and, latterly, CEO of Macrophage Pharma Limited. He is currently CEO of eTheRNA Immunotherapies NV, an immunotherapy company utilising mRNA technologies.
Over the last decade, he has specialised in the translation of complex scientific projects from research into commercial environments as an investor and in corporate development and operational management roles. Steven has overseen clinical trials at all stages of development from Phase I to III in oncology, urology, infectious disease and inflammation with two therapeutic programmes culminating in regulatory approvals.
He was an operational partner and adviser to Gilde Healthcare, a Dutch domiciled venture capital fund, an adviser to the Cancer Pioneer Fund (UK) and a Trustee and R&D committee member of a UK charity supporting research for the treatment of paediatric and adult brain tumours. Steven was educated in the UK, was awarded a Ph.D from the University of Aberdeen and was a visiting fellow at the University of Manchester.
 

Surender Vashist

RNA Engineering Scientist, Medimmune
Surender Vashist

Surender worked for 15 years on positive strand RNA viruses in academia and industry. Majority of this time was spent on studying the relationship of the functions of viral genome with the sequence and the structure adopted by genomic RNA. He studied elements of viral genome that help it translate efficiently in the hostile cellular environment. Using this experience and knowledge, Surender helped successfully build an internal platform for mRNA therapeutics, focused on infectious diseases, in AstraZeneca. He is now continuing to work on improving the platform by manipulating various cis- and trans- acting elements of the mRNA.

sponsors

Conference agenda

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8:30

Registration & Coffee

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9:00

Chair's Opening Remarks

Shalini Andersson

Shalini Andersson, Chief Scientist New Therapeutic Modalities, AstraZeneca

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9:10

Optimizing Nanoparticle mediated delivery of siRNAs to treat cancer and fibrosis

David Evans

David Evans, Chief Scientific Officer, Sirnaomics, Inc

• Presenting a polypeptide nanoparticle mediated delivery system for siRNA therapeutics to treat cancer and fibrosis
• The benefits of the ability to deliver multiple siRNAs within the tumour microenvironment will be discussed
• Leveraging this capability to improve outcomes with Immune Checkpoint inhibitors
• Improved GMP manufacturing approaches using microfluidics for clinical development
 

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9:50

Development of a light-triggered liposomes for intracellular oligonucleotide delivery to the retina

Enrico Mastrobattista

Enrico Mastrobattista, Professor of Pharmaceutical Biotechnology & Delivery, Utrecht University

• Inherent challenges in oligonucleotide-based drug delivery modes
• Achieving an efficient and safe intracellular liposomal delivery system activated by light
• Discuss the methods, results and opportunities
• Discuss an ex vivo perfused eye model for ocular delivery of ON
 

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10:30

Morning Coffee

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11:00

Design of Safe and Potent Nucleic Acid Therapeutics

Ed Yaworski

Ed Yaworski, VP, Pharmaceutical Development, Genevant Sciences

• Review of important elements of lipid nanoparticle technology
• Abrogation of common NA-LNP tolerability concerns
• Optimization of systemic mRNA therapeutics
 

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11:40

Addressing the challenge of chronic dosing via traditional LNP-based delivery for mRNA therapeutics

Shalini Andersson

Shalini Andersson, Chief Scientist New Therapeutic Modalities, AstraZeneca

• Clinical appeal of lipid nanoparticle delivery systems and other competing modes of delivery
• Challenges in production and screening of LNP candidates for effective translation to biotherapeutics
• Complications of chronic dosing and acceptable tolerability profiles
• Showcasing the development of a tolerable subcutaneous administration of mRNA via functionalized LNPs
 

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12:20

Networking Lunch

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13:20

Exploring the development and progress of BioNTech mRNA therapeutic delivery platforms

Heinrich Haas

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

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14:00

Facilitated delivery of self-amplifying mRNA vaccines

Jeffrey Ulmer

Jeffrey Ulmer, Head, Preclinical R&D US, GSK

• Cationic nanoemulsion delivery of mRNA
• Lipid nanoparticle delivery of mRNA
• Mechanism of action of facilitated delivery of mRNA vaccines
 

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14:40

Overcoming lung barriers to siRNA delivery in cystic fibrosis through tailored lipid/polymer hybrid nanoparticles

Francesca Ungaro

Francesca Ungaro, Associate Professor, University Of Napoli Federico II

• Non-Cellular and cellular barriers to inhaled siRNA in cystic fibrosis
• Strategies to engineer nanoparticles to overcome lung barriers
• In vitro and in vivo efficacy of inhalable hybrid lipid/polymer nanoparticles for siRNA delivery in cystic fibrosis
 

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15:20

Afternoon Tea

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15:50

Aiming at delivering RNA to tumors using intra-venous injections

Steve Pascolo

Steve Pascolo, Founder and CEO, Miescher Pharma GmbH

• Types of anti-cancer RNA requiring delivery to tumors
• Reported and original formulations: biodistribution in tumor bearing mice
• In vivo efficacy of formulated anti-cancer RNA

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16:30

Advanced microscopy methods to monitor RNA encapsulation and nanoparticle stability in biological environments

Katrien Remaut

Katrien Remaut, Associate Professor, Ghent University

• Fluorescence correlation spectroscopy (FCS) to monitor RNA (mRNA, siRNA) encapsulation in nanoparticles
• RNA release and degradation in biological fluids
• Intracellular dissociation and degradation kinetics
 

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17:10

Panel Discussion: Discussing the ongoing challenges of oligonucleotide delivery

• Questions around conjugation versus encapsulation; benefits and drawbacks
• Details and concerns behind the interactions between payloads and delivery vehicles
• Factors influencing the ratio between payload and delivery vehicle – what is an optimal ratio?
• Lipid-based encapsulation versus other nanoparticles
• Regulatory implications regarding production and quality; CMC and manufacturing
• Where is the next success in extrahepatic delivery?
 

Heinrich Haas

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

David Evans

David Evans, Chief Scientific Officer, Sirnaomics, Inc

David  Blakey

David Blakey, Chief Scientific Officer, MiNA Therapeutics

Jeffrey Ulmer

Jeffrey Ulmer, Head, Preclinical R&D US, GSK

Ed Yaworski

Ed Yaworski, VP, Pharmaceutical Development, Genevant Sciences

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18:10

Chair’s Closing Remarks and Close of Focus Day

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8:30

Registration & Coffee

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9:00

Chair's Opening Remarks

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

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9:10

Current progress of RNA therapeutics and industry overview: setting the scene

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

• Revisiting the essential concepts behind RNA therapy
• A brief history of RNA therapeutics and key technologies in the market
• Considerations in formulation design and delivery
• Future outlook
 

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9:50

mRNA Therapeutics for Pulmonary Disease

Christian Plank, Managing Director, CSO, Ethris GmbH

• Stabilized non-immunogenic mRNA (SNIM®RNA)
• Formulations for mRNA delivery
• Pulmonary delivery of mRNA
• mRNA transcript therapy for primary ciliary dyskinesia (PCD)
 

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10:30

Morning Coffee

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11:00

Rapid, Highly Sensitive Tissue Distribution and Pharmacodynamic Analysis of Therapeutic Nucleic Acids with RNAscope® and BaseScope™ in situ Hybridization Assay Platforms

Christopher Bunker, Sr. Director of Business Development, Advanced Cell Diagnostics, A Bio-Techne Brand


 • RNAscope and BaseScope in situ hybridization (ISH) platforms are utilized widely for high resolution tissue-based and cell-specific analysis of therapeutic nucleic acids and endogenous target mRNA expression in preclinical and clinical development. ACD’s ISH technologies molecular detection of any mRNA, ASO, siRNA, miRNA, as well as splice variants, gene edits, and point mutations. Advances in RNAscope, BaseScope and new miRNAscope technologies will be discussed in relation to therapeutic nucleic acid development.

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11:40

mRNA Therapeutics for Lung Indications

Klaus Giese, CEO, Pantherna Therapeutics GmbH

• PTXmRNA® - Novel mRNA molecules
• PTXLNPs® - Novel delivery vehicles
• Lung indications with high medical need
 

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12:20

Networking Lunch

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13:20

Small activating RNAs(saRNAs) – a novel therapeutic class of oligonucleotides with broad therapeutic potential

David Blakey, Chief Scientific Officer, MiNA Therapeutics

• Description of saRNA technology and broad applicability to a range of therapeutic targets
• Update on MiNA’s lead saRNA product MTL-CEBPA – preclinical and clinical data
• Emerging data on new therapeutic strategies with saRNAs
 

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14:00

Development of mRNA Formulations for Clinical Application

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

• Application of mRNA for therapeutic intervention
• Nanoparticulate carrier systems for RNA
• Manufacturing and Control of mRNA Nanotherapeutics
 

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14:40

Immunotherapeutic RNAs to treat cancer

Steve Pascolo, Founder and CEO, Miescher Pharma GmbH

• Optimisation of the production/design of mRNA
• Evaluating the anti-cancer efficacy of immunotherapeutic mRNAs (vaccines and gene therapies)
• Implementation and testing of immunochemotherapeutic RNA
 

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15:20

Afternoon Break

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15:50

Breaking down the immune barrier in cancer by systemic mRNA immunization

Steven Powell, CEO, eTheRNA

• The importance of Design-of-Experiments for the design of Lipid nanoparticle formulations
• The role of the lipid nanoparticle composition to govern immunogenicity
• The contribution of TriMix in the induction of high T-cell responses
• LNP-formulated mRNA can induce a multifunctional T-cell phenotype
• Systemic immunization leads to a T cell-inflamed tumor microenvironment
 

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16:30

A success story for AstraZeneca’s internal mRNA therapeutics platform: Responding quickly to pandemic

Surender Vashist, RNA Engineering Scientist, Medimmune

• Development of mRNA therapeutics platform @AZ.
• Optimisation of mRNA molecules for increased expression and reduced immunogenicity.
• Using our expertise in antibody production and mRNA therapeutics platform for quick and efficient response to Pandemic : DARPA P3 program

 

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17:10

Chair’s Closing Remarks and Close of Day One

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8:30

Registration & Coffee

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9:00

Chair's Opening Remarks

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

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9:10

MiRNA Therapeutics: From Discovery to the Bedside

Ekkehard Leberer, Senior Director, Sanofi

• The non-coding genome makes up 98.8% of the human genome. Most of this non-coding genome is transcribed into non-coding RNAs that may play an important role in cellular regulation in health and disease; these non-coding RNAs could be novel targets for future medicines.
• MicroRNAs are short non-coding RNAs that regulate biochemical pathways and networks of pathways by the mechanism of RNA interference (RNAi). MicroRNA-21 has been implicated in multiple organs as a microRNA associated with fibrotic diseases and cancer.
• The presentation will summarize the opportunities and challenges of developing microRNA-based drugs and will illustrate the successful generation of an anti-fibrotic microRNA-based therapeutic approach by targeting microRNA-21 with an antisense oligonucleotide (anti-miR-21). This microRNA-based drug is now in a phase 2 clinical trial for a fibrotic kidney disease called Alport Syndrome.
 

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9:50

MicroRNA Replacement Therapy: Myth or Reality?

Michel Janicot, CDO, InteRNA Technologies BV

• miRNAs - Mother Nature has already done most of the job! ...
• miRNAs as novel therapeutic intervention in Oncology
• miRNA replacement therapy (miRNA mimics)
 

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10:30

Morning Coffee

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11:00

NanoGenics; Using a peptide nanoparticle, LipTide, to develop a siRNA product portfolio

Simon Newman, CSO, Nanogenics Ltd

• How does the peptide nanoparticle, LipTide resemble an artificial virus?
• Delivering siRNA with LipTide across multiple administration routes
• Case study; Targeting the MRTF pathway to develop siRNA anti-fibrotic drugs
 

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11:40

Optimising saRNA lipid nanoparticle formulation through a DoE approach on clinically relevant skin explants

Robin Shattock, Professor of Mucosal Infection and Immunity, Imperial College London

• The promise of self-amplifying mRNA in the field of biotherapeutics: benefits and drawbacks
• The challenges in linear translation from animal to human models
• Utilising Design of Experiments factors of complexing lipid entity, lipid nanoparticle dose, lipid and particle concentration, and ratio of zwitterionic and cationic lipids to optimise saRNA-induced fluorescence in skin explants
• Broad application of the system in molecular and formulation design of clinically relevant oligonucleotide biotherapeutics
 

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12:20

Networking Lunch

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13:20

MicroRNA inhibitors as therapeutics for common and rare brain diseases

David Henshall, Professor of Molecular Physiology & Neuroscience, RCSI

• Short overview of brain diseases that represent suitable indications for treatment using antagomirs
• Current pipeline of LNA-based oligonucleotides in epilepsy and rare diseases
• Future prospects – delivery challenges, precision targeting using microRNA therapeutics for genetic disease and “network” approaches
 

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14:00

The Development of Nucleic Acid Delivery Platforms; a pan-RNA approach

Bo Rode Hansen, President & CEO, Genevant Sciences

• At Genevant, we are pursuing a modality agnostic “pan-RNA” approach to develop a diverse pipeline of RNA therapeutics; including delivery of RNAi payloads targeting both viral and endogenous gene targets, and mRNA in vaccine applications.
• Differentiated pan-RNA approach encompassing RNAi, mRNA and gene editing
• Industry-leading lipid nanoparticle (LNP) and ligand conjugate delivery platforms enable “best-fit” modality
 

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14:40

Afternoon Tea

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15:10

The upcoming regulatory updates in the EU clinical trials arena

Kirsty Wydenbach, Deputy Unit Manager, Clinical Trials Unit, Medicines & Healthcare products Regulatory Agency (MHRA)

• Upcoming 2020 regulatory updates affecting EU clinical trials
• Revisiting the regulatory environment and a prescription on how to interact with regulators
• Updates on regulatory expectations on reporting clinical trial data
 

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15:50

Current findings from pharmaceutical assessments and regulatory expectations in oligonucleotide-based product manufacturing

David Deutsch

David Deutsch, Pharmaceutical Assessor, MHRA

• Revisions to current guidelines in oligonucleotide-based product development and manufacturing
• Prescriptive guidelines to drug component sourcing, formulation and analysis
• Learning outcomes from current assessments of pharmaceutical licensing applications
 

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16:30

When RNA therapeutics are biological medicines: regulatory considerations for the EU

Christiane Niederlaender, Director, AMBR Consulting Ltd

• Classifications of RNA therapeutics within the medicines framework
• RNAs as vaccines and gene therapies and the implications for regulatory assessment and development
• RNAs in CRISPR approaches and the CMC considerations
• Key regulatory issues as highlighted by case studies
 

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17:10

Chair’s Closing Remarks and Close of Day Two


President & CEO
Genevant Sciences
Managing Director, CSO
Ethris GmbH
Director
AMBR Consulting Ltd
Sr. Director of Business Development
Advanced Cell Diagnostics, A Bio-Techne Brand
Chief Scientific Officer
MiNA Therapeutics
Professor of Molecular Physiology & Neuroscience
RCSI
Pharmaceutical Assessor
MHRA
Senior Director
Sanofi
VP RNA Formulation and Drug Delivery
BioNTech AG
Head, Preclinical R&D US
GSK
Deputy Unit Manager, Clinical Trials Unit
Medicines & Healthcare products Regulatory Agency (MHRA)
CEO
Pantherna Therapeutics GmbH
CDO
InteRNA Technologies BV
Professor of Mucosal Infection and Immunity
Imperial College London
Senior Director and Head of New Modalities
AstraZeneca
CSO
Nanogenics Ltd
Founder and CEO
Miescher Pharma GmbH
CEO
eTheRNA
RNA Engineering Scientist
Medimmune

Sponsors

Exhibitors

Supporting Media Partners

Supporters

Focus Days

Oligonucleotide Delivery Systems Focus Day

Oligonucleotide Delivery Systems Focus Day

Copthorne Tara Hotel
18 February 2020
London, United Kingdom

2020 Attendee List

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2020 RNA Therapeutics Brochure

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Speaker Interview with Surender Vashist

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Speaker Interview with Steve Pascolo

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Past Attendee List (2018 - 2019)

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Speaker Interview with David Henshall

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Speaker Interview with Simon Newman

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RNA Infographic

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Speaker Interview with Christiane Niederlaender

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2020 Speaker List

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'RNAi therapy in hATTR amyloidosis' by Dr Ali Murad - Alnylam

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'Small Activating RNA- From Bench to Bedside' by David Blakey - MINA Therapeutics

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'Self amplifying mRNA technology to accelerate vaccines R&D' by Jeffrey Ulmer - GSK

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'Enabling Delivery for Genetic Medicines' by Steffen Panzner - Lipocalyx

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RNA Therapeutics Programme

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Sponsors


Bio-Techne

Sponsors
http://www.bio-techne.com

Bio-Techne® unites some of the most referenced brands in Life Sciences: R&D Systems™, Novus Biologicals™, Tocris™, ProteinSimple™ and Advanced Cell Diagnostics™ (ACD). Advanced Cell Diagnostics (ACD), a Bio-Techne brand, is proud to be supporting the 11th Annual RNA Therapeutics Conference, to assist in the development of RNA Therapeutics.

The highly specific and sensitive single-molecule RNAscope® and BaseScope™ RNA ISH technologies from ACD, address key questions about therapeutic gene delivery vectors (both viral and non-viral) and therapeutic transgene expression. With the ability to visualize RNA at single molecule and single cell resolution, RNAscope technology is a solution for both the development and production of RNA therapeutics. As a reproducible and consistent assay to validate gene therapies between and within laboratories, it can be used to measure potency of vectors through quantification of copy numbers and transgene expression. The RNA ISH technology can also be a powerful tool in the understanding of vector and transgene expression with morphological context in tissue allowing differentiation of transgene from endogenous mRNAs, confirmation of gene-editing based therapies (CRISPR/Cas9) and off target effects.

With constant development of our technology and for new applications RNAscope technologies, Advanced Cell Diagnostics (ACD), a Bio-Techne brand, is proud to be supporting the 11th Annual RNA Therapeutics Conference, to assist in the development of RNA Therapeutics. Please join us at our booth to explore how our products and services can assist in your therapeutic discovery and development.



New England Biolabs, Inc.

Sponsors
https://international.neb.com/

For over 40 years, New England Biolabs, Inc. has led the industry in the discovery and production of molecular biology reagents. In addition to products for genomic research, NEB continues to expand its product offerings into areas related to PCR, gene expression, sample prep for next gen sequencing, cellular analysis, synthetic biology and RNA analysis.

Exhibitors


Aldevron

Exhibitors
http://www.aldevron.com

Aldevron serves the biotechnology industry with custom production of nucleic acids, proteins, and antibodies. Thousands of clients use Aldevron-produced plasmids, RNA and gene editing enzymes for projects ranging from discovery research to clinical trials to commercial applications. These products are critical raw materials and key components in commercially available drugs and medical devices. Aldevron specializes in GMP manufacturing and is known for inventing the GMP-Source™ quality system. Company headquarters are in Fargo, North Dakota, with additional facilities in Madison, Wisconsin, and Freiburg, Germany. To learn more, visit www.aldevron.com.


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.


Inabata France SAS

Exhibitors
https://inabata.fr/

INABATA France belongs to the Japanese Group INABATA CO.,LTD. which has 130 years of history in business.

Distribution of:

- Active Pharmaceutical Ingredients

- Advanced Intermediates

- Specialities



Intertek

Exhibitors
http://www.intertek.com

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


Nippon Fine Chemical Co., Ltd.

Exhibitors
https://www.nipponseika.co.jp/en/business/lipid/

Nippon Fine Chemical, aware of the future of such lipids, has developed new processes for synthesizing and purifying phospholipids and is supplying them on an industrial scale. These products - used Worldwide – are highly regarded as raw materials for preparing liposomes, pulmonary surfactants, and the design of drugs.

Our production is…

Purity Phospholipids and Cholesterol

Functional Lipids

Presome as Lipid Mixture and Liposome



Thermo Fisher Scientific

Exhibitors
http://www.thermofisher.com/

As a worldwide leader in serving science, Thermo Fisher Scientific invites you to partner with us to reach your RNA therapeutic development goals. Explore our portfolio of high-quality TheraPure reagents designed for your oligonucleotide and mRNA synthesis to help you move your project from development to manufacturing and commercialization. Accelerate your productivity and innovation with expertise, services, and support from Thermo Fisher Scientific.

Media Partners


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


Pharma Journalist

Supporting Media Partners
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.


IPI

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

IPI – International Pharmaceutical Industry Established by professionals with over 30 years experience in the Pharmaceutical and Life sciences publishing sectors. We have identified the needs of these dynamic industries, and have listened carefully to our readers and advertisers. With strong collaboration between Pharmaceutical and Life sciences Industry Associations we have created a global distribution network. We give you a promise to become a reliable extension of your marketing and communication arm. If necessary we will integrate our expertise with your needs. Incorporating new and innovative communication methods we will help to highlight your expertise.


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


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.


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

Media Partners


Drug Discovery Today

Supporters
http://www.drugdiscoverytoday.com/

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

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