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RNA Therapeutics

SAE Media Group’s 14th Annual Conference
RNA Therapeutics
8-9 February 2023 | London, UK
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SAE Media Group is proud to announce the 14th Annual RNA Therapeutics Conference taking place on 8-9 February 2023

RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious and chronic diseases. This is reflected with the value of the global antisense & RNA therapeutics market expected to reach USD 1.81 billion by 2025, growing at a CAGR of 7.5%. With Covid-19 rapidly accelerating the RNA landscape through massive regulatory successes, 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, there has been considerable progress in the development of RNA medicines and a major expansion of the RNA-therapeutics field.

In 2023, we will see the return of SAE’s RNA Therapeutics conference to a live in-person event, showcasing an update on mRNA therapeutic applications, coupled with an industry outlook of the field. We will also explore the latest trends in novel RNA therapies, including developments in AI modelling and small molecule modulation of RNA. Further topics to be examined comprise of novel RNA modalities encompassing self-amplifying RNA, saRNA, circular RNA, lnc RNA and many more.

Don't miss this opportunity to network with the pharma and biotech community of global experts in the UK and Europe; the two-day agenda offers you peer-to-peer networking with Directors of novel RNA therapies, CEOs, Senior and expert scientists, Chief Medical Officers, Senior Vice Presidents and many more.
 

FEATURED SPEAKERS

Christian Plank

Christian Plank

Chief Technology Officer, Ethris GmbH
Covadonga Paneda

Covadonga Paneda

Chief Development Officer, Altamira Therapeutics
Daniel Lorenz

Daniel Lorenz

Associate Director of Research, Eclipse BioInnovations
Daniel Tondera

Daniel Tondera

Head of Biology, Pantherna Therapeutics GmbH
Ekkehard Leberer

Ekkehard Leberer

Life Sciences Consultant, Elbicon
Heinrich Haas

Heinrich Haas

VP Formulation & Drug Delivery, BioNTech
Jack Wei

Jack Wei

Senior Director, Sirnaomics, Inc
Joseph Martinelli

Joseph Martinelli

Executive Director, Eli Lilly
Lykke Pedersen

Lykke Pedersen

Head of RNA Therapeutics, Abzu Aps
Mark Cunningham

Mark Cunningham

Head of Operations, Nucleoic Acid Therapy Accelerator
Martin Akerman

Martin Akerman

Chief Technology Officer, Envisagenics
Nicole Datson

Nicole Datson

Chief Development Officer, Vico Therapeutics
Oliver Ernst

Oliver Ernst

Managing Director, NEUWAY Pharma GmbH
Rok Sekirnik

Rok Sekirnik

Head of Process Development, mRNA pDNA, Sartorius BIA Separations
Samir Ounzain

Samir Ounzain

CEO, HAYA Therapeutics
Shalini Andersson

Shalini Andersson

Vice President Oligonucleotide Discovery, AstraZeneca
Tamar Grossman

Tamar Grossman

Vice President, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceutical Companies of Johnson & Johnson
Venkatesh Pilla Reddy

Venkatesh Pilla Reddy

Director of Clinical Pharmacology and Pharmacometrics, Astrazeneca
Virginia Castilla-Llorente

Virginia Castilla-Llorente

Associate Director, Evox Therapeutics Ltd
Yaniv Erlich

Yaniv Erlich

CEO, Eleven Therapeutics

Christian Plank

Chief Technology Officer, Ethris GmbH
Christian Plank

Christian Plank is co-founder and chief technology officer of Ethris GmbH which is 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 principal investigator and coordinator of numerous grant projects that have focused on non-viral nucleic acid delivery and nanomagnetic drug targeting. He is the author of more than 170 publications and co-inventor of numerous patents in the field of nucleic acid delivery.

Covadonga Paneda

Chief Development Officer, Altamira Therapeutics
Covadonga Paneda

Covadonga Pañeda, Ph.D. has over 15 years of experience in leadership and management of biotechnology companies. Before joining Altamira in April 2022, she spent seven years managing Sylentis’ R&D efforts, a clinical stage RNAi company, two years leading the translational activities of start-up companies Isquaemia Biotech and Mirnax Biosens; and most recently, she served as Director of Development at LiMM Therapeutics, a French neuroimmune biopharmaceutical company.
Dr. Pañeda holds a Ph.D. in Biochemistry and Molecular Biology from the Faculty of Medicine at Universidad Autónoma, Madrid, Spain, and a Master in Management of Biotechnology Companies awarded by Fundación Genoma España, Madrid, Spain.
 

Daniel Lorenz

Associate Director of Research, Eclipse BioInnovations
Daniel Lorenz

Dan Lorenz, PhD is the Associate Director of Research at Eclipsebio, focusing on emerging technologies to support RNA Therapeutics. Before being recruited to Eclipse, Dan completed his post doc in the lab of Gene Yeo at the University of California, San Diego where he focused on CLIP based technologies for discovering RNA binding sites and RNA modifications using long-read platforms. Prior to UCSD, Dan received his doctorate in chemical biology from University of Michigan, where his work was largely focused on high throughput screen methodologies.

Daniel Tondera

Head of Biology, Pantherna Therapeutics GmbH
Daniel Tondera

Daniel Tondera is an experienced expert in the field of RNA based therapeutics.
Before joining Pantherna Therapeutics, he was Group Leader at Silence Therapeutics where he was investigating the potential of using miRNAs and mRNA-based gene editing technologies for systemic LNP-mediated therapies. Prior to this he held positions as Group Leader at Cenix Bioscience in Dresden/Germany and as Senior Scientist at CRT in London/UK.
Daniel studied biochemistry at the University of Potsdam/Germany and received his PhD from the University of Potsdam in collaboration with Silence Therapeutics followed by postdoctoral training at the University of Geneva/Switzerland and at the Dana-Farber-Cancer Institute in Boston/MA.
 

Ekkehard Leberer

Life Sciences Consultant, Elbicon
Ekkehard Leberer

Ekkehard serves as an independent life sciences consultant at ELBIOCON (www.elbiocon.com) with more than 30 years of experience in biotechnology and pharmaceutical industry. He headed research teams and held managerial positions at the Biotechnology Research Institute of the National Research Council (NRC) of Canada in Montreal and in Big Pharma at Hoechst Marion Roussel, Aventis and Sanofi in Martinsried/Munich and Hoechst/Frankfurt. He was educated in chemistry and molecular biology at the University of Konstanz (Ph.D. and Habilitation in biochemistry) and University of Toronto (PostDoc at the Banting and Best Department of Medical Research). He is co-author of more than 60 publications in prestigious peer-reviewed journals including Nature and Science.
His scientific and managerial activities included responsibilities in functional genomics, genetic engineering, biological sciences, pre-clinical and clinical development of various therapeutic modalities encompassing small molecule drugs and biopharmaceuticals (peptides, antibodies, oligonucleotides, and mRNAs), drug delivery, portfolio management, external innovation, alliance management and business strategies.
Ekkehard served as a member in Scientific Advisory Boards of several biotechnology companies, and he was the Head of the Supervisory Board of BioM, Munich (www.bio-m.org). He is a member in the life sciences consulting network NewGuide (www.newguide.net).
 

Heinrich Haas

VP Formulation & Drug Delivery, BioNTech
Heinrich Haas

Heinrich Haas
Vice President RNA Formulation & Dug Delivery
BioNTech RNA Pharmaceuticals GmbH, An der Goldgrube 12, 55131 Mainz, Germany

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

Jack Wei

Senior Director, Sirnaomics, Inc
Jack Wei

Dr. Wei is a senior director at Sirnacomics, Inc, where he oversees preclinical drug development using GalAhead technology. Jack joined Sirnacomics with more than 16 years drug discovery experience both in academic research institutes including Johns Hopkins and Lieber Institute for Brain Development and major pharmaceutical companies Merck and Schering-Plough.

Joseph Martinelli

Executive Director, Eli Lilly
Joseph Martinelli

Lykke Pedersen

Head of RNA Therapeutics, Abzu Aps
Lykke Pedersen

Lykke was awarded her PhD in Biophysics from the University of Copenhagen and conducted research at the Niels Bohr Institute. Although her education is in Physics, she's a Bioinformatician at heart and has over 8 years' experience working in the pharmaceutical industry. Lykke is skilled in machine learning, mathematical modeling, statistics, and gene expression profiling, and she is an expert in R programming.

Lykke is Head of RNA Therapeutics at Abzu, the deep tech startup that accelerates exploration and enables scientists to bring drugs to market faster.
 

Mark Cunningham

Head of Operations, Nucleoic Acid Therapy Accelerator
Mark Cunningham

Mark graduated from the University of Hertfordshire and started his career in recombinant protein expression & purification at start-up and biotech companies in England. In 1994, Mark moved to the United States where he contributed towards therapeutic monoclonal antibody development for the Janssen R&D arm of Johnson & Johnson. After a 20-year career with J&J as a scientific and operational leader, Mark moved back to the UK where he joined the UKRI’s Nucleic Acid Therapy Accelerator (NATA) as Head of Operations.

Martin Akerman

Chief Technology Officer, Envisagenics
Martin Akerman

Dr. Martin Akerman is the inventor of SpliceCore®, Envisagenics’ flagship platform born of his vision of applying machine learning to RNA information and discovering new drug targets in areas of unmet need. Martin trained as a postdoctoral fellow with Dr. Adrian Krainer at Cold Spring Harbor Laboratory, where he helped in the development of Spinraza®, the first FDA-approved RNA therapeutic for treating Spinal Muscular Atrophy. Dr. Akerman received his PhD in Bioinformatics from Technion, Israel Institute of Technology, where he studied how RNA splicing can boost functionality of the human genome and trigger diseases.

Nicole Datson

Chief Development Officer, Vico Therapeutics
Nicole Datson

Nicole Datson has held the position of Chief Development Officer at VICO Therapeutics since the company was founded in 2019. Prior to joining VICO, she served as Senior Director Drug Discovery and Early Preclinical Development at BioMarin Nederland and was head of preclinical development at Prosensa Therapeutics. She has more than a decade of expertise in developing antisense oligonucleotide-based therapies for neuromuscular and neurodegenerative genetic disorders and has headed VICO’s VO659 programme from early R&D through nonclinical safety studies to the first clinical trial.
She obtained her PhD in molecular genetics at Leiden University.
 

Oliver Ernst

Managing Director, NEUWAY Pharma GmbH
Oliver Ernst

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

Rok Sekirnik

Head of Process Development, mRNA pDNA, Sartorius BIA Separations
Rok Sekirnik

Rok Sekirnik graduated in Chemistry at University of Oxford, where he also completed his PhD. He has worked in the areas of mAb, vaccine and gene therapy vector development in various roles, including Principal Scientist at Novartis and Project Manager at Batavia Biosciences. His team at BIA Separations is working on developing high yielding, cost-effective purification approaches for pDNA and mRNA production.

Samir Ounzain

CEO, HAYA Therapeutics
Samir Ounzain

Samir Ounzain is a molecular biologist with over 15 years of experience exploring the dark matter of the genome and its roles in development and disease. Prior to founding HAYA Therapeutics, Samir was a Project Leader and Research Fellow at the Lausanne University Hospital (CHUV), where his research efforts directly led to the discovery of hundreds of novel heart-enriched lncRNAs, most notably the lncRNAs CARMEN, Meteor and Wisper.

Senne Dillen

Drug Product & Substance Senior Scientist, eTheRNA immunotherapies NV
Senne Dillen

Shalini Andersson

Vice President Oligonucleotide Discovery, AstraZeneca
Shalini Andersson

Shalini is Vice President Oligonucleotide Discovery in BioPharmaceuticals R&D, AstraZeneca. She is a member of the Management Team for Discovery Sciences as well as of the Review Board for drug projects in all therapy areas in BioPharmaceuticals R&D.
Shalini is also leading Targeted Drug Delivery within Cardiovascular, Renal and metabolic diseases and several collaborations with academic research groups and biotechs in the nucleic therapeutics field.
Shalini received her PhD in 1989 at the University of Linköping, Sweden. Prior to joining AstraZeneca in 1997, she was Associate Professor in Organic and Analytical Chemistry at the University of Linköping, Sweden. She is the author or co-author of over 60 peer reviewed articles and 5 patents.
Shalini is member of several boards including the Board of Oligonucleotide Therapeutics Society, the Member of Board of Directors for OligoNova, the Swedish national center for research and development of therapeutic oligonucleotides and of the Scientific Advisory Group for Nucleic Acid Therapy Accelerator (NATA, UK).
 

Tamar Grossman

Vice President, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceutical Companies of Johnson & Johnson
Tamar Grossman

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

Venkatesh Pilla Reddy

Director of Clinical Pharmacology and Pharmacometrics, Astrazeneca
Venkatesh Pilla Reddy

Dr. Venkatesh Pilla Reddy, PhD is Director, Clinical Pharmacology and Quantitative Pharmacology function of AstraZeneca, Cambridge UK. He is currently supporting COVID-19 Projects (AZ Vaccine and EVUSHELD) as a Global Clinical Pharmacology Lead. He obtained his PhD in Pharmacometrics through a unique industry collaboration program between Pfizer, Janssen Pharmaceuticals and Merck via TI Pharma in the Netherlands. His PhD work was focused on PKPD M&S of antipsychotic drugs. He subsequently worked at Quantitative Pharmacology and Pharmacometrics group in Merck & Co before joining AstraZeneca Early Oncology function. Venkatesh provided Model-Informed Drug Development support to various oncology projects. He has been influential in gaining acceptance and addressing questions from regulatory agencies for EVUSHELD (Tixagevimab and Cilgavimab), Olaparib and Osimertinib programs. He has participated on MIDD related panel discussion with the European Medicines Agency and the US Food and Drug Administration (FDA). Venkatesh currently holds the Deputy Topic Leader position at the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) Management Committee to work on the ICH M12 DDI guidelines, and co-leads various cross-industry working groups such as IQ TALG, ISOP and ISSX M&S. Over 20 years of his industry carrier, he has published > 60 peer review research articles, 2 book chapters.

Virginia Castilla-Llorente

Associate Director, Evox Therapeutics Ltd
Virginia Castilla-Llorente

Virginia has over 13 years of experience working in RNA, first as an academic from a more basic research approach, and, for the last 5 years, in biotech, as Research Associate Director in Evox Therapeutics. Prior to this, Virginia did a postdoc studying miRNA mediated gene silencing in Memorial Sloan Kettering Cancer Centre, followed by a second postdoc focused on the understanding of protein-mRNA interactions in UCL and The Francis Crick Institute. Virginia has authored a number of scientific publications and patents on the aforementioned fields. Virginia earned her PhD in Molecular Biology in Centro de Biologia Molecular Severo Ochoa, UAM.

Yaniv Erlich

CEO, Eleven Therapeutics
Yaniv Erlich

Dr. Yaniv Erlich is the co-founder and CEO of Eleven Therapeutics. Prior to this position, he was the CSO of MyHeritage, a consumer genomics company (acquired by Francisco Partners), an Associate Professor of Computer Science at Columbia University and a Principal Investigator at the Whitehead Institute, MIT. Dr. Erlich received his bachelor’s degree from Tel-Aviv University, Israel (2006) and a PhD from the Watson School of Biological Sciences at Cold Spring Harbor Laboratory (2010). Dr. Erlich’s research interests are computational human genetics. Dr. Erlich is a TEDMED speaker (2018), the recipient of DARPA’s Young Faculty Award (2017), the Burroughs Wellcome Career Award (2013), Harold M. Weintraub award (2010), the IEEE/ACM-CS HPC award (2008), and he was selected as one of 2010 Tomorrow’s PIs team of Genome Technology.

sponsors

Conference agenda

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8:00

Registration & Coffee

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9:00

Chairman's Opening Remarks

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9:10

Developing Oligonucleotides into Therapies: Opportunities and Challenges

Shalini Andersson, Vice President Oligonucleotide Discovery, AstraZeneca

  • Introduction to oligonucleotide therapeutics and the current landscape
  • Therapeutic promise of oligonucleotide modalities
  • Outlining some of the key challenges in oligonucleotide therapeutics including extra-hepatic delivery
  • Exploring some of the emerging delivery solutions and future opportunities
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    9:50

    Tailoring delivery systems for next generation mRNA therapeutics

    Heinrich Haas, VP Formulation & Drug Delivery, BioNTech

  • Nanoparticle technologies for nucleotide and drug delivery systems
  • Structural and functional coherencies within selected delivery systems
  • Novel approaches and tailored solutions for future mRNA therapeutics
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    10:30

    Chromatography in mRNA Production: From Analytics to Purification

    Rok Sekirnik, Head of Process Development, mRNA pDNA, Sartorius BIA Separations

  • Case studies of leveraging the power of at-line HPLC monitoring for process development to increase purity and yield of mRNA production.
  • Approaches to optimize plasmid purifi cation and linearization, IVT reaction and mRNA purifi cation will be described, including purifi cation methods to achieve >95% supercoiled plasmid purity, optimize
    linearization reaction, and increase of IVT reaction yield from 3-5 mg/mL to 10-12 mg/mL.
  • Use of single use bioreactors to produce multi-gram amounts of mRNA  will be demonstrated.
  • Oligo dT-based affi nity purifi cation will be described with approaches to optimize dynamic binding capacity to reach up to 6 g/L.
  • Multimodal chromatography and reverse-phase chromatography cases will be discussed for capture of non-polyadenylated RNA, and removal of dsRNA.
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    11:00

    Morning Coffee

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    11:30

    mRNA Delivery to the CNS: Targeting Neurological Disease

    Oliver Ernst, Managing Director, NEUWAY Pharma GmbH

  • EnPC® as drug delivery technology providing for treatment of multiple disorders delivering a broad range of therapeutic modalities
  • Discussion of utilising naturally derived protein capsules to deliver mRNA across the blood brain barrier and resulting benefits
  • Exploring the development of innovative therapeutics for treatment of brain diseases and CNS-related genetic diseases
  • Future opportunities in the targeted delivery of RNA therapeutics
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    12:10

    The therapeutic potential of mRNA in the restoration of endothelial cell function

    Daniel Tondera, Head of Biology, Pantherna Therapeutics GmbH

  • Exploring Next-generation PTX_LNP™ formulations for delivering PTXmRNA™ to various tissues and cell types
  • Reviewing PTXcLNP™ enabling target protein expression in the vascular endothelium of the lung via the intravenous delivery
  • Case study: mRNA therapy to prevent lung oedema in Acute Respiratory Distress Syndrome
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    12:50

    Getting the message across - Addressing challenges in RNA delivery using customized LNPs

    Senne Dillen

    Senne Dillen, Drug Product & Substance Senior Scientist, eTheRNA immunotherapies NV

  • The 4 pillars of a successful RNA therapeutic
  • Optimizing Drug Substance processes to increase tolerability
  • LNPs tailored to target sites and administration methods
  • Facilitating logistics through the development of lyophilizable formulations
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    13:20

    Networking Lunch

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    14:20

    Role of Clinical Pharmacology in the Development of Novel RNA Modalities

    Venkatesh Pilla Reddy, Director of Clinical Pharmacology and Pharmacometrics, Astrazeneca

  • Delving into model informed therapeutic development for novel therapies including self-amplifying RNA technology
  • Reviewing application and benefi ts of Clinical Pharmacology in AAV, mRNA and self-amplifying RNAs based therapies
  • Next steps: assessing the current fi eld and future work required for approval of novel RNA vaccines and immunotherapies
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    15:00

    Drug and Target Discovery with Explainable AI

    Lykke Pedersen, Head of RNA Therapeutics, Abzu Aps

  • What is “explainable” artificial intelligence? An intro for scientists and researchers
  • Why is “explainability” necessary for pharma and life science?
  • An RNA therapeutics use case: Applying explainable AI to augment understanding
  • The future: How to harness the power of explainable AI to reshape drug development
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    15:30

    Afternoon Tea

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    16:00

    AI/ML developed RNA therapeutics for neurodegenerative diseases

    Martin Akerman, Chief Technology Officer, Envisagenics

  • Overcoming in-silico modeling challenges for neurodegenerative diseases
  • How Envisagenics’ SpliceCore platform can be used in designing antisense oligonucleotides
  • Translational R&D from in-silico to in-vitro for potential ASO candidates in neurodegenerative diseases
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    16:40

    Optimizing RNAi applications through novel AI technologies

    Yaniv Erlich, CEO, Eleven Therapeutics

  • Utilising massively parallel combinatorial searches and AI algorithms to unleash the full power of RNAi
  • Reviewing the structure-activity relationship (SAR) of RNAi molecules to enhance drug safety and efficacy
  • Developing the next generation of RNAi therapeutics to silence the disease-causing genes

     

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    17:20

    Chairman’s Closing Remarks and Close of Day One

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    17:30

    End of Day One Networking Reception

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

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    9:10

    Genetic medicine: state of the art, frontiers and current research

    Joseph Martinelli

    Joseph Martinelli, Executive Director, Eli Lilly

  • Addressing key challenges at the forefront of the next generation of genetic medicines
  • The role of Nucleic acid innovation in the development of RNA therapeutics
  • Exploring delivery innovation as a crucial component in the advancement of RNA therapies
     
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    10:30

    OligoPhore and SemaPhore for extrahepatic delivery of therapeutic RNA

    Covadonga Paneda, Chief Development Officer, Altamira Therapeutics

  • Description of OligoPhore and SemaPhore platforms for delivery therapeutic RNA
  • Rapid and extensive pH-dependent release of RNA from endosomal resulting in effective delivery into cells
  • Distribution of therapeutic RNA into extrahepatic tissues
  • Examples of effective RNA activity in animal models of disease
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    11:10

    Morning Coffee

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    11:40

    Exploring Extracellular Vesicles as delivery agents for RNA therapeutics

    Virginia Castilla-Llorente, Associate Director, Evox Therapeutics Ltd

  • An introduction into the therapeutic potentials of Extracellular Vesicles
  • Advancing the RNA therapeutic landscape through the Evox platform for mRNA, siRNA, and gene editing EV delivery
  • Future perspectives: next steps in the development of EVs for the delivery of RNA modalities
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    12:20

    Advancing RNA therapeutics through the development of long non-coding

    Samir Ounzain, CEO, HAYA Therapeutics

  • Exploring new directions in the identification of novel lncRNA targets
  • Reviewing antisense oligonucleotides in the targeting of disease generating lncRNAs
  • Case study: targeting the lncRNA Wisper, a cardiac tissue-enriched driver of fibrosis in the heart
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    13:00

    Exploring mRNA therapeutics in the treatment of chronic lung diseases

    Christian Plank, Chief Technology Officer, Ethris GmbH

  • Utilising mRNA for protein replacement therapies in respiratory and infectious diseases
  • Reviewing lipidoid nanoparticle formulation technologies
  • Caste study: SNIM RNA as a novel therapeutic platform
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    13:40

    Networking Lunch

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    14:40

    Panel Discussion: Advancing RNA therapeutics by improving drug-like properties of RNA modalities

  • Upcoming RNA-based medicines beyond vaccines: What will be the next products? What should industry be looking to work on?
  • Delivery systems beyond LNPs: Will LNPs do the job or do we need more for targeted delivery, such as: extrahepatic and organ-specific delivery, BBB and tumours?
  • Types of RNA: What are the emerging ‘flavors’ and what is promising - saRNA, modRNA, circularRNA?
  • Ekkehard Leberer, Life Sciences Consultant, Elbicon

    Heinrich Haas, VP Formulation & Drug Delivery, BioNTech

    Christian Plank, Chief Technology Officer, Ethris GmbH

    Shalini Andersson, Vice President Oligonucleotide Discovery, AstraZeneca

    Jack Wei, Senior Director, Sirnaomics, Inc

    Mark Cunningham, Head of Operations, Nucleoic Acid Therapy Accelerator

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    15:20

    Novel RNA modulating therapies for the treatment of genetic neurological diseases

    Nicole Datson, Chief Development Officer, Vico Therapeutics

  • Delving into the therapeutic benefits of targeting RNA modulation using antisense oligonucleotides
  • Specific challenges for antisense oligonucleotide therapeutics in the CNS space
  • Case study – VO659 targeting polyglutamine disorders, spinocerebellar ataxia type 3 and type 1 and Huntington’s disease
     
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    16:00

    Afternoon Tea

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    16:30

    Targeted RNA therapeutics approaches for developing transformational medicines for patients

    Tamar Grossman, Vice President, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceutical Companies of Johnson & Johnson

  • RNA modalities for therapeutics and vaccine development
  • Novel carriers for improved extra-hepatic targeting
  • CNS delivery of siRNA for neurological disease therapies
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    17:10

    GalAhead™: a novel therapeutic GalNAc-RNAi platform to downregulate single and multiple genes

    Jack Wei, Senior Director, Sirnaomics, Inc

  • Sirnaomics has developed novel proprietary GalNAc-RNAi therapeutic platform – GalAhead™
  • GalAhead comprises two key technological components – mxRNA™ (miniaturized RNAi triggers) and muRNA™ (multi-unit RNAi triggers)
  • mxRNAs are composed of single ~30 nt long oligonucleotides to downregulate individual genes
  • muRNAs are comprised of multiple oligonucleotides (2 or more) to simultaneously silence two or more targets
  • Data will be presented validating these two technologies both in vivo and in vitro, as well as progress report on quickly expanding GalAhead therapeutic pipeline
     
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    17:50

    Chairman’s Closing Remarks and Close of Day Two


    Chief Technology Officer
    Ethris GmbH
    Chief Development Officer
    Altamira Therapeutics
    Associate Director of Research
    Eclipse BioInnovations
    Head of Biology
    Pantherna Therapeutics GmbH
    Life Sciences Consultant
    Elbicon
    VP Formulation & Drug Delivery
    BioNTech
    Senior Director
    Sirnaomics, Inc
    Executive Director
    Eli Lilly
    Head of RNA Therapeutics
    Abzu Aps
    Head of Operations
    Nucleoic Acid Therapy Accelerator
    Chief Technology Officer
    Envisagenics
    Chief Development Officer
    Vico Therapeutics
    Managing Director
    NEUWAY Pharma GmbH
    Head of Process Development, mRNA pDNA
    Sartorius BIA Separations
    CEO
    HAYA Therapeutics
    Drug Product & Substance Senior Scientist
    eTheRNA immunotherapies NV
    Vice President Oligonucleotide Discovery
    AstraZeneca
    Vice President, Global Head of RNA and Targeted Therapeutics
    Janssen Pharmaceutical Companies of Johnson & Johnson
    Director of Clinical Pharmacology and Pharmacometrics
    Astrazeneca
    Associate Director
    Evox Therapeutics Ltd
    CEO
    Eleven Therapeutics

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    Exhibitors

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    Full Conference Agenda

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    Short Conference Agenda

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    Preliminary Attendee List

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    Exclusive Speaker Interview - Yaniv Erlich, CEO, Eleven Therapeutics

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    Past Attendee List

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    Press Release - AstraZeneca, Johnson & Johnson, Eli Lily and more at the RNA Therapeutics Conference 2023

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    RNA Therapeutics 2023 Brochure

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    Speaker Biographies

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    Conference Chair Invitation Letter - Heinrich Haas, BioNTech

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    Sponsors


    Abzu®

    Sponsors
    https://www.abzu.ai/

    Abzu® was born from the desire to challenge the fundamental assumptions of contemporary, black-box AI. Abzu’s pioneering technology, the QLattice®, is a research engine that accelerates exploration, enabling scientists to bring drugs to market faster.

    The QLattice introduces a new standard of interpretability and explainability to life science data sets. Through simple models and straightforward mathematical expressions, Abzu’s QLattice achieves a level of transparency and performance in machine learning that is disrupting domains previously dominated by opaque and cumbersome models.

    Founded in January 2018, Abzu is a deep tech startup with offices in Copenhagen, Denmark and Barcelona, Spain. Abzu derives its name from Ancient Sumerian, meaning “the source of the waters of wisdom”.

    Abzu has raised a total of €8,1M, and last closed a seed round in April 2021 for €4.9M.



    Eclipsebio

    Sponsors
    https://eclipsebio.com/?gclid=EAIaIQobChMI_pKAm9zi_AIVkr7tCh0gSQOREAAYASAAEgKL5PD_BwE

    Eclipsebio has developed multiple world-class RNA genomics products used by biopharma and academic customers around the world to accelerate the development of tomorrow’s key RNA genomics discoveries and medicines.

    Our innovative standard and customizable solutions bring new and deeper insights to the RNA interactome by providing more accurate, reproducible and useable data for our customers.

    Our products:

    - Identify new RNA regulatory and epigenetic mechanisms

    - Spur exciting new insights into mRNA

    - RNA binding protein and miRNA biology

    - Map new RNA drug targets to accelerate the development of safe and effective RNA medicines



    eTheRNA

    Sponsors
    http://www.etherna.be

    eTheRNA is an mRNA technology platform company with integrated capabilities including mRNA construct design and optimization, coupled with specialized expertise in designing and manufacturing customized lipid nanoparticles (cLNP) formulations tailored for the prevention and treatment of various pathological states, providing end-to-end solutions for next generation mRNA therapeutics. The company believes that customized LNPs are critical enablers in advancing mRNA technologies to new levels of prophylactic and therapeutic utility.



    Roche

    Sponsors
    https://custombiotech.roche.com/global/en/portfolio/mrna-therapeutics-raw-materials.html

    Roche CustomBiotech - We provide reliable, tailor-made and high-quality raw materials, kits and technologies to biopharma and diagnostics companies.

    With the CustomBiotech division Roche merged its innovative technologies, excellent knowledge, and decades of experience in the Biopharma and Diagnostics Industry. We work with many pioneering mRNA companies to develop and manufacture needed raw material solutions meeting the strict requirements in Biopharma. Our guiding tenet is to minimize variation of products built on our raw materials by ensuring excellent quality, high performance within narrow tolerances, and reliable delivery.



    Sartorius BIA Separations d.o.o.

    Sponsors
    https://www.biaseparations.com/

    Sartorius BIA Separations, develops and manufactures market leading CIM® monolithic chromatographic columns for purification and analysis of large biomolecules, such as viruses, plasmids, and mRNA, which are applied in cell and gene therapies. Sartorius BIA’s Cornerstone® Biomanufacturing Development Services are result of more than 20 years of hands-on experience with the most challenging biopharmaceutical products and offer a comprehensive approach of integrated process development solutions and novel technology designed to improve the robustness and yield of large biomolecules production, while improving the safety of therapeutic products. Sartorius BIA's technology for manufacturing-scale purification is already used in production of the first commercialized advanced therapeutics; Sartorius BIA Separations also has a keen presence with novel drug candidates in the clinical pipeline.


    Exhibitors


    euroAPI

    Exhibitors
    http://www.euroapi.com/en

    Euroapi is focused on reinventing active ingredient solutions to sustainably meet customers’ and patients’ needs around the world. We are a world leader in active pharmaceutical ingredients with some 200 products in our portfolio, offering a large span of technologies, while developing innovative molecules through our Contract Development and Manufacturing Organization (CDMO). Taking action for health by enabling access to essential therapies inspires our 3,350 people every day. With strong research and development capabilities and six manufacturing sites all located in Europe, Euroapi ensures API manufacturing of the highest quality to supply customers in more than 80 countries. EUROAPI is listed on Euronext. Find out more at www.euroapi.com


    PolyPeptide

    Exhibitors
    http://www.polypeptide.com/

    PolyPeptide is a focused Contract Development & Manufacturing Organization (CDMO) for peptide- and oligonucleotide-based active pharmaceutical ingredients. By supporting its customers mainly in pharma and biotech, it contributes to the health of millions of patients across the world. PolyPeptide offers products and services from pre-clinical through to commercial stages, including generics. Its active custom projects pipeline reflects the opportunities from novel drug therapies in development to fight both widespread and rare diseases. Dating back to 1952, PolyPeptide today runs a global network of six GMP-certified facilities in Europe, the U.S. and India with around 1,200 employees as per the middle of 2022. PolyPeptide’s shares (SIX: PPGN) are listed on SIX Swiss Exchange.



    RIBOPRO

    Exhibitors
    https://ribopro.eu/

    RIBOPRO is an innovative and fast-growing CDMO/CMO, providing premium (m)RNA technology and formulation services to the pharmaceutical and biotechnology industry. RIBOPRO designs and synthesizes high-performance mRNA for R&D and GMP purposes and delivers unconditional support from early research into clinical trials and commercialization. Through their in-house research, the company continuously develops both the manufacturing processes for high quality mRNA and lipid nanoparticles (LNPs) and develops novel technologies to boost the performance of the mRNA/gain control over the delivery. These technologies not only provide better outcomes, but also provide freedom to operate in an otherwise competitive marketplace.



    Symbiosis Pharmaceutical Services

    Exhibitors
    https://www.symbiosis-pharma.com/

    Symbiosis Pharmaceutical Services is a contract development and manufacturing organisation (CDMO) specialising in the GMP manufacture and sterile fill/finish of vials for clinical trials and commercial supply. Regulatory compliance, technical capability and operational flexibility are at its core.

    Manufacturing from a purpose-built FDA inspected and MHRA-licensed facility, the CDMO can handle products that require aseptic liquid filling and lyophilisation for a range of complex biologics, viral vectors for use in gene therapies and small molecule drugs.

    Offering fast access to manufacturing slots and accelerated release of drug product, Symbiosis is primed to meet demand for small-scale, fast-turnaround drug product sterile manufacturing.



    Tebu-bio

    Exhibitors
    https://www.tebu-bio.com/

    Our vision of helping to advance Life Sciences research every day, is embodied by providing a unique, holistic range of solutions to make life easier – easily and quickly accessible.

    The full scope of your needs is at the centre of our attention. Our experts provide reliable, multimodal solutions, encompassing our extensive range of products, in-house laboratory platforms, and our practical administrative and logistics services. You’ll find the right combination of answers to accelerate your daily work, in one place.

    Enjoy local access to global solutions, always within easy reach at one of our 9 local offices in Europe


    Media Partners


    Contract Biotechnology

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

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


    Gene Therapy Net

    Supporters
    http://www.genetherapynet.com

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


    World Pharma News

    Supporters
    http://www.worldpharmanews.com/



    Gate2Biotech

    Supporters
    http://www.gate2biotech.com

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


    American Pharmaceutical Review

    Supporters
    http://www.americanpharmaceuticalreview.com

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


    Pharmacircle

    Supporters
    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….


    Biocompare

    Supporters
    http://www.biocompare.com

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


    Mednous

    Supporters
    http://www.MedNous.com

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


    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.


    Pharmiweb

    Supporters
    http://www.pharmiweb.com

    Established in October 2003, PharmiWeb Solutions is an energetic, innovative provider of web-based solutions for the pharmaceutical sector, with customers such as AstraZeneca, Roche, Servier Laboratories, and Merck With solutions in three key areas: Online Publishing, e-Business and Mobile Computing, we deliver connectivity – between people and applications – unlocking islands of data and knowledge. We own some of the sector’s most innovative Online Publications: PharmiWeb.com, HospitalPharma.com and Detail-Direct.com. We are actively developing solutions for Mobile Computing platforms. For example, our PharmiTabTM e-pharmacy solution runs real-time patient data and prescribing transactions in a busy hospital environment, on a TabletPC, wireless client/server platform. e-Business solutions range from e-Detailing, to Online Market Research, all designed to increase reach, increase sales, and to reduce administration and costs.


    Technology Networks

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

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


    On Drug Delivery

    Supporters
    http://www.ondrugdelivery.com/subscribe

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


    Genomeweb L L C

    Supporters
    http://www.genomeweb.com

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

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

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


    Pharma Journalist

    Supporters
    http://www.pharmajournalist.com

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


    ASD MEDIA

    Supporters
    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

    Supporters
    http://www.pharmaphorum.com

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

    Copthorne Tara Hotel

    Scarsdale Place
    Kensington
    London W8 5SR
    United Kingdom

    Copthorne Tara Hotel

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

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

     

    HOTEL BOOKING FORM

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    WHAT IS CPD?

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

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

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

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

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

    CPD AND PROFESSIONAL INSTITUTES

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

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

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

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

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

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

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

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

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

    GLOBAL CPD

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

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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    Contact SAE Media Group

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




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