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RNA Therapeutics
10 February - 11 February 2025
RNA Therapeutics

SAE Media Group’s is proud to announce the 15th Annual RNA Therapeutics Conference, taking place on the 10-11 February 2025 in London, UK.


The RNA therapeutics industry has grown exponentially in recent years, largely attributed to the advancements in CRISPR RNA-editing technologies, and increased exposure of RNA vaccines following the COVID-19 pandemic. This upward trajectory is expected to continue, with 131 candidate therapies in clinical trials as of January 2024.


Don’t get left behind! Join us to discuss the latest advancements in RNA therapeutics with leaders from across the life science sector.
 

 Expect presentations and discussions addressing novel delivery methods and extra-hepatic delivery solutions, applications of AI for RNA sequence optimization and case studies of novel modalities progressing through the pipeline including circRNA, siRNA, miRNA, lncRNA and more. Further challenges that will be addressed include the lack of effective assays for in vivo analytical testing, including toxicity and immune-modulation considerations. We will also examine the rapidly changing regulatory landscape, identifying how the industry can adapt to meet changing guidelines and regulations.


Don't miss this opportunity to network with the pharma and biotech community of global experts in the UK and Europe; and help shape RNA therapeutic advancements! Our two-day agenda offers you peer-to-peer networking with Directors of key big pharma and biotech’s in the field, CEOs, Senior Scientists, Chief Medical Officers, Senior Vice Presidents and many more.
 

FEATURED SPEAKERS

Alessandro Bonetti

Alessandro Bonetti

Director, AstraZeneca
Christalyn Rhodes

Christalyn Rhodes

Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly
Ka-Wai  Wan

Ka-Wai Wan

Pharmaceutical Assessor, MHRA
Maren von Fritschen

Maren von Fritschen

Head - Regulatory Policy Europe, Moderna
Steven Hood

Steven Hood

Director, Oligonucleotide ADME Strategy, DMPK, GSK
Urs Langen

Urs Langen

Lab Head, Roche

Alessandro Bonetti

Director, AstraZeneca
Alessandro Bonetti

Dr. Alessandro Bonetti obtained his Master degree in Medical Biotechnologies at the University of Naples in 2001 and moved to Helsinki for his PhD studies where he investigated the genetic prediposition to multiple sclerosis in Nordic populations. He then moved to RIKEN Yokohama to study the biological role of long non-coding. During that time he developed numerous technologies to identify the functional relevance of non-coding transcripts in physiological and pathological conditions. Subsequently he joined AstraZeneca to lead the transcriptomic studies in the Oligonucleotide platform. In October 2022 he was appointed Principal Scientist to lead the non-coding initiative in AZ.

Christalyn Rhodes

Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly
Christalyn Rhodes

Christalyn Rhodes, PhD is currently the Associate Vice President Genetic Medicine, External Partnering and Collaborations for Eli Lilly and Company  here she identifies opportunities, manages deals, and innovates nuanced strategies and collaboration agreements to develop genetic medicine strategically aligned with Eli Lilly core therapeutic areas. Additionally, Christalyn serves as the Expert-In-Residence for Harvard Innovation Labs, on the board of University of Massachusetts Medical School’s Bridge Fund, as well as an advisor to the Nucleate organization where she has supported the launch and advancement of several startups ranging from idea stage and brainstorming to securing funding. Previously, Christalyn worked as a Venture Capital Associate for Flagship Pioneering where she co-founded a biotechnological platform company to create novel therapeutics in the space of personalized genomic medicine. Dr. Rhodes received a Ph.D. in Biological Sciences from Harvard University and a BA from the University of North Texas.

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

Scientific Advisor, NEUWAY Pharma
Ekkehard Leberer

Ekkehard serves as CEO 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

Chief Technology Officer, NeoVac
Heinrich Haas

Ph.D. in the group of Prof. Dr. Helmuth Möhwald at Johannes-Gutenberg Universität Mainz. Researched lipid membranes and organized bio-molecular systems. In pharmaceutical industry (Munich Biotech, Medigene, BioNTech) developed different types of nanoparticle products to clinical stage. Focus on advanced approaches for nanoparticle development and control.

Jay Sarkar

Researcher and Entrepreneur, Stanford University
Jay Sarkar

Dr. Sarkar has been in mRNA space for a little over a decade now. His work at Stanford University pioneered the use transiently expressed mRNAs to create an abridged epigenetic reprogramming regimen. This “transient or partial reprogramming” was shown to reset the age of a cell without erasing its identity. From this work, he spun out Turn Bio - the first company around this technology - and this approach has become a lead contender in longevity biotech space. He went on to found his current, new startup - reThink64 - that develops new paradigms for improving delivery of mRNAs and other large molecules.

Juha Monkare

Senior Scientist, Drug Product Development, Johnson & Johnson Innovative Medicine
Juha Monkare

Ka-Wai Wan

Pharmaceutical Assessor, MHRA
Ka-Wai  Wan

Dr Ka-Wai Wan is a pharmacist by training and graduated from The School of Pharmacy (University of London). She completed her PhD in protein and gene delivery using nanomedicine at the Welsh School of Pharmacy (Cardiff, UK) and worked as a research academic for over a decade prior to her transition to the MHRA as a pharmaceutical assessor. She now leads on the assessment of nano-based medicinal products, providing scientific and regulatory advice on medicines, particularly on nanomaterials used for both small and macromolecules delivery. This includes nucleic acids delivery using lipid nanoparticles for a wide variety of medical applications.

Loic Roux

Co-Founder, Deep Science Ventures
Loic Roux

Dr. Loïc Roux earned his PhD in Medicinal Chemistry from Aix-Marseille University, focusing on HIV nucleotide pro-drugs. In 2018, he joined Prof. Khvorova's lab at UMass Medical School to work on oligonucleotide stabilization and delivery, and later Prof. Wood's lab at Oxford University for peptide-oligonucleotide conjugates in muscular diseases. After a principal scientist position at PepGen Limited, he helped establish NATA, a UKRI initiative for nucleic acid therapeutics. Moving back to industry as Director of Lead Development at Ochrebio developing RNA therapeutics for liver diseases, he then founded, via the venture studio Deep Science Ventures, OligoTune Limited, a company aiming to revolutionised cancer treatment with RNA technology.

Lucas Bethge

Senior Scientist bei, Silence Therapeutics AG
Lucas Bethge

Lucas Bethge studied chemistry at the Humboldt-University zu Berlin (Germany). After he obtained his Ph.D. in 2011 in Bioorganic Chemistry, he first joined NOXXON Pharma. In 2016 he moved to Silence Therapeutics GmbH, a biotech company in Berlin that develops GalNAc-conjugated siRNA for therapeutic use. Currently, he is Vice-President and leading the Oligonucleotide Chemistry group at Silence Therapeutics. Lucas is an expert for the synthesis, conjugation, and analysis of oligonucleotides with over ten years of experience in industrial research and development at the interface between early research and clinical development.

Maren von Fritschen

Head - Regulatory Policy Europe, Moderna
Maren von Fritschen

Maren von Fritschen is a recognized leader in Regulatory Affairs with 20+ years in Pharma and MedTech. With a PhD in Natural Sciences, she is Head of Regulatory Policy Europe at Moderna, driving regulatory strategies for innovative technologies like mRNA. Maren built the regulatory arm at EUCOPE and was a foundational force behind PharmaLex, later founding AddOn Pharma as CEO. She's chaired DIA's Regional Advisory Council EMEA, contributed to TOPRA, and the STARS project. In the academic realm she is lecturing and mentoring master students at the University of applied sciences, Berlin. Additionally, she has established a comprehensive scientific network across leading Universities in Brazil, focusing on medication in primary health care.

Margaux Hubert

Senior Scientist - Computational Immunology, mRNA Center of Excellence, Sanofi
Margaux Hubert

Dr Margaux Hubert is an immunologist by training with a solid foundation in both experimental and computational research. She completed her PhD in immuno-ongology at the Cancer Research Center (CRCL) of Lyon. Through this first experience in research, she developed significant expertise in dendritic cells biology and their pivotal role in initiating immune responses against tumors. After graduating, she acquired complementary skills in bioinformatics and contributed to the identification of new immune biomarkers in tumors during a four-year postdoctoral fellowship in the Cancer Immunology Laboratory of Lyon (LICL). Bridging her knowledge in immunology with computational approaches, she then moved into a researcher position at the Center Léon Bérard anti-cancer hospital (CLB, Lyon, France). In 2024, she joined the mRNA Center of Excellence (CoE) at Sanofi, where she focuses on deciphering the mode of action of mRNA vaccines using both computational approaches and a strong expertise in immunology to analyze high-dimensional exploratory readouts from clinical and preclinical studies.

Matt Maciejewski

VP, Head of Data Science, Korro Bio
Matt Maciejewski

Matt Maciejewski is the VP and Head of Data Science at Korro Bio. With a PhD in computational biophysics from the NIH and University of Edinburgh, he completed postdoctoral fellowships at Cambridge and Novartis NIBR, where he innovated ML methods for cheminformatics. After an 8-year tenure at Pfizer, Matt now leads a team at Korro Bio, developing deep learning models to design chemically modified oligos for precise RNA editing. Overall, his work in machine learning and probabilistic modeling has significantly advanced disease understanding, drug design methods, and patient stratification.

Matt Maciejewski

VP, Head of Data Science, Korro Bio
Matt Maciejewski

Nagy Habib

Co-Founder and CMO , MiNA Therapeutics
Nagy Habib

For over three decades Nagy has been at the forefront of clinical research and clinical practise in cancer.
He pioneered the first clinical trial in the use of adenovirus and plasmid for the treatment of liver
cancer, as well as the use of plasmid gene therapy in hydrodynamic gene therapy delivery. He was the
first in Europe to perform a clinical trial with the use of adult CD34+ stem cells in patients with liver
failure and stroke.
Nagy is a founder and was the Head of R&D of Mina therapeutics whilst at Mina he was driving the
development of saRNA drug (a new class of medicines) which is currently being trialled in patients with
liver cancer in eight UK centres, and sites in Singapore and Taiwan (OUTREACH study, Clinical Trials.gov
ID NCT02716012) and in a second trial in patients with solid tumours (TIMEPOINT study,
ClinicalTrials.gov ID NCT 04105335) in the UK, USA, Europe, Singapore and Taiwan.
He has published widely in gene therapy, stem cell therapy, oligonucleotides, endoscopy and surgery.
Currently he is the CEO of Apterna Limited, a company focussed on novel oligonucleotide delivery and
Dawn Therapeutics specialising in gene therapy. Previously Nagy was founder and Chairman of
EMcision Limited (acquired by Boston Scientific Inc in 2018).
Nagy is Lead Clinician and Head of the Department of HPB Surgery at Imperial College London

Russell Hamilton

Associate Principal Scientist, AstraZeneca
Russell Hamilton

At AstraZeneca, my research focus is in the field of functional genomics, RNA structure, and epigenetics delving into the intricate mechanisms governing gene expression and regulation. My interdisciplinary approach explores the impact of these processes on disease development and paves the way for innovative therapeutic interventions.

Prior to joining AstraZeneca led a research group in the Department of Genetics, University of Cambridge and headed the bioinformatics facility in Centre for Trophoblast Research, University of Cambridge. Previously worked at CEGX (now Biomodal) developing epigenetics sequencing methodologies.
 

Soren Ottosen

VP of Research, Sixfold Bioscience
Soren Ottosen

Søren Ottosen is an early drug discovery program manager, strategy and project leader with 15 years of experience in identifying nucleic acid-based medicine candidates for clinical development, first at Santaris Pharma as a Senior Research Scientist and then at the Roche Innovation Center Copenhagen. While at Roche, Dr. Ottosen held a number of positions, including Senior Portfolio and Platform Development Leader and Senior Leader. Dr. Ottosen received his Bachelor of Science from the Institute of Molecular Biology at the University of Copenhagen, his Ph.D from the Department of Biochemistry and Molecular Biology at Michigan State University, and did his postdoctoral fellowship in the Department of Microbiology at NYU School of Medicine.

Steve Pascolo

Principal Investigator, University Hospital of Zurich and University of Zurich
Steve Pascolo

Trained as an immunologist at the Pasteur Institute (Paris, France), Dr. Steve Pascolo used mouse models to test and develop mRNA-based vaccines (direct injection of mRNA) during his post-doc in Tuebingen, Germany from 1998 till 2000. In 2000, he co-founded CureVac with Dr. Hoerr and Dr. von der Mulbe. Dr. Pascolo was Chief Scientific Officer (CSO) of the company from 2000 till 2006, developing the technology, implementing the worldwide first pharmaceutical (GMP) production of synthetic mRNA and starting in 2003 the worldwide first clinical studies where humans (including himself) got injections of in vitro transcribed mRNA. In 2006, he joined the oncology department of the University Hospital of Zurich, Switzerland and continued the development of immunotherapies based on RNA. In 2008, he founded Miescher Pharma to support this work. In 2013, he joined the dermatology department of the University Hospital of Zurich, Switzerland. In 2017, Dr. Pascolo implemented in the University of Zurich an academic mRNA platform https://www.cancer.uzh.ch/en/Research/mRNA-Platform.html. In collaboration with several research and clinical departments in Zurich he optimizes, tests and implements mRNA based vaccines and therapies.

Steven Hood

Director, Oligonucleotide ADME Strategy, DMPK, GSK
Steven Hood

Steve Hood received a PhD in Molecular Toxicology from the University of Surrey in 1993 and joined Glaxo Group Research as an Industrial Post doc in the Molecular Biology department in Greenford where he cloned and expressed CYPs in recombinant cell lines. Steve transferred to GlaxoWellcome at Ware in 1995 and became a manager in the new GSK DMPK Molecular Mechanism and Extrapolation Technology team in 2001. In 2010 Steve seconded to Scinovo and co-ordinated the GSK Oligo delivery (GOLD) project and was also Industrial co-lead of the IMI COMPACT consortium that ran from 2012-17 and evaluated delivery mechanisms for oligos and peptides. After a brief return to DMPK in 2015, Steve joined Bioimaging as a senior Scientific Director, responsible for the external imaging collaborations that are members of the Bioimaging Expertise Network (BEN). As part of this network, Steve was also Co-Director of the GSK Centre for Molecular Imaging (COMI) at the University of Illinois at Urbana Champaign, where he worked closely with Professor Stephen Boppart and his team.

In addition to his “day job”, Steve has spent most of the last 2 decades working on GSK’s diverse oligo portfolio and has supported projects ranging from inhaled SiRNAs, TLR antagonists, DMD exon skippers (Drisapersen) and ASOs for TTR (Innotersen) and HBV with Ionis (Bepirovirsen). More recently Steve has been involved with the Wave collaboration (Wve-006), the Jade HBV siRNA in licence project and the formation of the Boston Oligo hub.

In Jan 2024 Steve rejoined DMPK in and assume the role of Director of Oligo ADME Strategy. Steve will continue to develop the DMPK road map and train and mentor the next generation of DMPK Oligo Project Team Members and SMEs.
 

Urs Langen

Lab Head, Roche
Urs Langen

Urs Langen studied biochemistry at the University of Tübingen, Germany, before pursuing his PhD in vascular biology in the group of Prof. Ralf Adams at the Max Planck Institute in Münster, Germany. For his postdoctoral studies Urs joined the group of Prof. Chenghua Gu at Harvard Medical School in Boston, USA, where he investigated heterogeneity of the blood brain barrier in various brain areas. After completion of the postdoctoral project Urs joined Roche in Basel, Switzerland, where he has worked for 3 years as a senior scientist on drug delivery to the brain using the Roche Brainshuttle™ technology. Since 2024 Urs heads the Neurovascular Distribution Lab at Roche expanding his work on brain delivery of various drug modalities and leads multiple research projects in the dementia space. During his career Urs published numerous papers in high-impact peer-reviewed scientific journals including Nature and Nature Cell Biology.

Why you should attend:

  • Engage in the latest challenges in drug delivery and review the new and emerging alternatives to LNPs, that are broadening opportunities within tissue targeting.
  • Network with peers and industry experts to share ideas, discover novel solutions to some of the industry’s biggest challenges including biodistribution, controlling immunogenicity and increasing longevity of RNA therapeutic action.
  • Learn about novel modalities emerging in the space and the manufacturing and analytical strategies advancing development and synthesis of these RNA modalities.
  • Build your ecosystem within RNA therapeutics to uncover potential collaborators and keep pace with this rapidly-advancing field.

 Who should attend:

Any company striving to deliver optimal RNA therapies to patients in need.
If you or your company are working on or looking to break into any of the following areas, this conference is a must!

  • RNA therapeutic research and development
  • mRNA vaccines R&D
  • RNA therapeutic delivery methods
  • Analytical method development for oligonucleotide candidate testing
  • RNA manufacturing
  • RNA synthesis
  • Applications of AI within RNA vaccines and therapeutic development
     

Why not partner with us?
By sponsoring, you’ll position your brand for success. Don't miss out on the opportunity to elevate your visibility, credibility, and business prospects—consider sponsorship today!
Please contact Jack Grant at Jack.Grant@saemediagroup.com or call +44 (0) 20 7827 6113

Conference agenda

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8:00

Registration & Coffee

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9:00

Chairs' Opening Remarks

Christalyn Rhodes, Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly

Heinrich Haas, Chief Technology Officer, NeoVac

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9:10

RNA Therapeutics at Eli Lilly – recent strategy and advancements

Christalyn Rhodes, Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly

  • Explore the potential of novel backbone chemistry and AI guided oligonucleotide design to enhance the safety and potency of RNA based medicine.
  • Investigate the improvements and identification of ligand-based tissue specific targeting strategies for oligonucleotides, analogous to galnac in the liver, but for other tissues such as the lung, brain, or heart.
  • Evaluate novel routes of administration for oligonucleotides, such as transdermal delivery, that can overcome the barriers of stability, bioavailability, and immunogenicity.
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    9:50

    Driving innovation with mRNA platform technologies supported by regulatory provisions

    Maren von Fritschen, Head - Regulatory Policy Europe, Moderna

  • Regulatory understanding of platform technology in the EU and US and the need for global alignment
  • The value of mRNA platform technology for early provision of medicines to patients
  • Platform technology approaches today and in the future
  • The need for scientific platform technology guideline, specific for mRNA medicines

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    10:20

    Regulatory considerations for RNA-based medicinal products

    Ka-Wai Wan, Pharmaceutical Assessor, MHRA

  • Evolving landscape in the regulation of RNA-based medicinal products
  • Key considerations and challenges for product development
  • Support available for industry from the MHRA specifically on innovative medicines
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    10:50

    Regulatory joint Q&A

    Ka-Wai Wan, Pharmaceutical Assessor, MHRA

    Maren von Fritschen, Head - Regulatory Policy Europe, Moderna

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    11:10

    Morning break

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    11:40

    Translational Potential of Preclinical Models in Predicting Reactogenicity and Immunogenicity of mRNA Vaccines

    Margaux Hubert, Senior Scientist - Computational Immunology, mRNA Center of Excellence, Sanofi

  • Computational methods for accelerating mRNA vaccine R&D
  • Build an integrated translational model predicting immunogenicity and reactogenicity 
  • Ranking mRNA vaccine candidates
  • Preclinical assessment: translatability of models to the clinic
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    12:20

    Advancing computational approaches to accelerate discovery of novel RNA targets

    Russell Hamilton, Associate Principal Scientist, AstraZeneca

  • Leveraging computational approaches to target discovery
  • Gaining a deeper understanding of RNA sequence structure, to identify novel sequences for targeting
  • Identifying functional and structured regions of RNA to enable targetability assessment of RNAs with small molecules
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    13:00

    Networking Lunch

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    14:00

    Sixfold Bioscience: AI-powered RNA delivery

    Soren Ottosen, VP of Research, Sixfold Bioscience

  • Nucleic acid-based medicines have demonstrated great potential for novel therapeutic approaches in a few key tissues.
  • This promise is currently limited to diseases manifested in tissues either with natural uptake of nucleic acids, through complex routes of administration, or through a limited number of characterised ligand-conjugates
  • Sixfold is using AI to design effective delivery methods for currently poorly addressed cells and tissues beyond the currently targetable tissue space.
     
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    14:40

    ML-based Design of Oligonucleotides that Promote ADAR-Based RNA Editing

    Matt Maciejewski, VP, Head of Data Science, Korro Bio

  • We have developed a new few-shot deep learning methodology for oligo design.
  • With this method, we can design oligos that result in strong editing in new targets with 100-200 data points.
  • Our method outcompetes standard methods on numerous counts, including max editing that we can achieve, and consistency in improving editing in an active learning setting.

     

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    15:20

    Afternoon Break

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    15:50

    Taking an HBV oligo into Ph3 - Reflections from the ADME team

    Steven Hood, Director, Oligonucleotide ADME Strategy, DMPK, GSK

  • Understanding the plasma and tissue pharmacokinetics of a GalNAc targeted ASO and its major (unconjugated) metabolite.
  • A recognition that GalNAc targeting has a modest effect on the total liver burden of ASO but a profound effect on the cellular distribution within the liver.
  • A review of the Drug-Drug Interaction (DDI) risks with Bepirovirsen, to enable Phase 3 clinical trials.
  • Understanding why the non-targeted Bepirovirsen outperformed the GalNAc conjugated GSK3389404 in the clinic and subsequent development of novel cellular biodistribution and imaging methodologies.

     
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    16:30

    Characterization of siRNA product-related impurities by HILIC

    Lucas Bethge, Senior Scientist bei, Silence Therapeutics AG

  • The presentation will showcase the selection and synthesis of relevant siRNA product-related impurities.
  • Development of a chromatographic method for separation of impurities through HILIC will be demonstrated.
  • The ability of HILIC to complement other separation modes (AEX, IP-RP) will be discussed.
     
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    17:10

    Chairs' Closing Remarks and Close of Day One

    Christalyn Rhodes, Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly

    Heinrich Haas, Chief Technology Officer, NeoVac

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    8:30

    Registration & Coffee

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    9:00

    Chairs' Opening Remarks

    Heinrich Haas, Chief Technology Officer, NeoVac

    Christalyn Rhodes, Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly

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    9:10

    Assessing the current RNA Therapeutics Landscape

    Heinrich Haas, Chief Technology Officer, NeoVac

  • Overview of current challenges within the RNA therapeutics and vaccines space
  • Recent advancements, breakthroughs and novel technologies
  • Future perspectives – where is the field headed
  •  

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    9:50

    Beyond Antibody Delivery – Expanding the Cargo Space of the Brainshuttle™ to ASOs

    Urs Langen, Lab Head, Roche

  • Design and generation of Brainshuttle-ASO conjugates for CNS delivery of ASOs after I.V.administration
  • Broad biodistribution of Brainshuttle-ASOs in the brain
  • Robust target knockdown of target in brain after intravenous Brainshuttle-ASO administration
     
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    10:30

    Morning Break

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    11:00

    Evaluation and selection of RNA lipid nanoparticle mixing systems: an industry perspective (provisional)

    Juha Monkare

    Juha Monkare, Senior Scientist, Drug Product Development, Johnson & Johnson Innovative Medicine

  • Overview of methods to prepare RNA-LNPs
  • Review of available mixing systems and their key differences
  • Selection criteria and process to choose the mixing system matching the user requirements
     
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    11:40

    Drug Routers: A Novel Paradigm for RNA Drug Delivery

    Jay Sarkar, Researcher and Entrepreneur, Stanford University

  • Comparison of current carrier-based approaches vs a new networking strategy for delivery
  • Demonstration and topological characterization of network formation
  • Showcase of the variety of applications and unique production features
  • Delivery profiling in novel tissues in animal models.
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    12:20

    Networking Lunch

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    13:20

    Exploring novel indications for LNP-mediated extra-hepatic RNA therapeutic delivery

    Daniel Tondera, Head of Biology, Pantherna Therapeutics GmbH

  • Exploration of novel LNPs for extra-hepatic targeting
  • Working backwards – identifying cell types that can be successfully entered, before establishing the specific disease indications this could impact
  • Case study examples of exploratory delivery of mRNA to novel tissues for prevention of edema
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    14:00

    Reviewing the advances in novel RNA modalities

  • How do we see the RNA therapeutic landscape evolving in years to come? Will mRNA and RNAi continue to dominate the market or will new players enter the game? 
  • Reviewing the latest RNA modalities breaking into the field: saRNA, circRNA, siRNA, lncRNA , RNA editing – which modality has the most potential in treating disease 
  • Looking forward – which modalities overcome extra-hepatic delivery and drive advancements in RNA therapeutics
  • Ekkehard Leberer, Scientific Advisor, NEUWAY Pharma

    Loic Roux, Co-Founder, Deep Science Ventures

    Soren Ottosen, VP of Research, Sixfold Bioscience

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    14:40

    Afternoon Break

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    15:10

    LncRNA – strategy and recent breakthroughs at AZ

    Alessandro Bonetti, Director, AstraZeneca

  • Overview of the non-coding field
  • Non-coding elements as novel pharmacological targets
  • Developing a technological pipeline for identification and validation of non-coding pharmacological targets
  • RNA therapeutics targeting non-coding elements
     
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    15:50

    mRNA vaccines – the latest advancements in fighting infectious disease and cancer

    Steve Pascolo, Principal Investigator, University Hospital of Zurich and University of Zurich

  • Clinical developments from mRNA vaccine candidates targeting infectious diseases and cancer
  • New synthetic mRNA formats
  • Assessing the mRNA landscape - what challenges remain and what potential does this modality have to impact patients worldwide

     
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    16:30

    RNA activation and bio-distribution

    Nagy Habib, Co-Founder and CMO , MiNA Therapeutics

  • RNA activation using small activating RNA [saRNA] can be useful in the treatment of myeloid-rich resistant cancer as well as in rare genetic diseases
  • TfR aptamer can lead to brain biodistribution of nucleic acids as well as lipid nano-particles
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    17:10

    Chairs' Closing Remarks and Close of Day Two

    Heinrich Haas, Chief Technology Officer, NeoVac

    Christalyn Rhodes, Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations , Eli Lilly


    Director
    AstraZeneca
    Associate Vice President - Genetic Medicine - External Partnerships and Academic Collaborations
    Eli Lilly
    Head of Biology
    Pantherna Therapeutics GmbH
    Scientific Advisor
    NEUWAY Pharma
    Chief Technology Officer
    NeoVac
    Researcher and Entrepreneur
    Stanford University
    Senior Scientist, Drug Product Development
    Johnson & Johnson Innovative Medicine
    Pharmaceutical Assessor
    MHRA
    Co-Founder
    Deep Science Ventures
    Senior Scientist bei
    Silence Therapeutics AG
    Head - Regulatory Policy Europe
    Moderna
    Senior Scientist - Computational Immunology, mRNA Center of Excellence
    Sanofi
    VP, Head of Data Science
    Korro Bio
    VP, Head of Data Science
    Korro Bio
    Co-Founder and CMO
    MiNA Therapeutics
    Associate Principal Scientist
    AstraZeneca
    VP of Research
    Sixfold Bioscience
    Principal Investigator
    University Hospital of Zurich and University of Zurich
    Director, Oligonucleotide ADME Strategy, DMPK
    GSK
    Lab Head
    Roche

    VENUE

    Hilton London Kensington

    179-199 Holland Park Avenue, London, United Kingdom

    At the heart of the Holland Park district, our hotel is 10 minutes from Westfield London shopping center. We're blocks from Shepherd's Bush Underground station, linking to central London, and Kensington Palace and Gardens are two miles from us. Enjoy 24-hour access to our fitness center.

     
    Join us in WestEleven for hearty buffet breakfast, a great way to start the day! Our Avenue Bar and Lounge serves light bites throughout the day as well as a delicious, seasonal dining menu.”

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    Hilton London Kensington

    179-199 Holland Park Avenue
    London W11 4UL
    United Kingdom

    Hilton London Kensington

    At the heart of the Holland Park district, our hotel is 10 minutes from Westfield London shopping center. We're blocks from Shepherd's Bush Underground station, linking to central London, and Kensington Palace and Gardens are two miles from us. Enjoy 24-hour access to our fitness center.

     
    Join us in WestEleven for hearty buffet breakfast, a great way to start the day! Our Avenue Bar and Lounge serves light bites throughout the day as well as a delicious, seasonal dining menu.”
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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
    Registered in England - SMi Group Ltd trading as SAE Media Group




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