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RNA Therapeutics USA
October 23 - October 24, 2024
RNA Therapeutics USA

Enabling you to connect with key representatives from big pharma and biotech, and advance the future of RNA Therapeutics.
 

SAE Media Group’s 2nd annual RNA Therapeutics USA Conference will be back for 2024 in Boston to bring together experts from the genetic medicines field!

Dive into the latest advances in RNA therapeutics, analytical and control processes, and vaccine development. Access cutting edge innovations in delivery technologies for precision targeting with case studies on LNPs and more. Explore how AI can be used can be used to optimise drug discovery and delivery platforms, as well as formulation strategies for enhancing therapeutic success.

This year's RNA Therapeutics USA Conference promises to be an unmissable event! Featuring Pfizer's mRNA breakthroughs, Sanofi's delivery optimization insights, and case studies from Moderna and AstraZeneca, this event is your chance to stay ahead in RNA innovation, you won't want to miss it!

FEATURED SPEAKERS

Chelsea Thorn

Chelsea Thorn

Senior Scientist, Pfizer
Derek O Hagan

Derek O Hagan

Senior Advisor R&D, GSK
Heinrich Haas

Heinrich Haas

Chief Technology Officer, NeoVac
Hetal Patel

Hetal Patel

Senior Principle Scientist/Group Leader, Pfizer
Jay Sarkar

Jay Sarkar

Researcher and Entrepreneur, Stanford University
Jim Weterings

Jim Weterings

Vice President R&D USA RNA Therapeutics & Delivery, Sirnaomics
Kinkini Roy

Kinkini Roy

Associate Director, Aviceda Therapeutics
Martin Akerman

Martin Akerman

CTO & Co-Founder, Envisagenics
Neil Robertson

Neil Robertson

Principal Scientist, TransCode Therapeutics, Inc.
Richard Geary

Richard Geary

EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.
Sourav Choudhury

Sourav Choudhury

Head AAV Technologies, Sanofi
Steve Pascolo

Steve Pascolo

Founder and CEO, Miescher Pharma GmbH
Yue Hui

Yue Hui

Scientist, Moderna
Zimeng Wang

Zimeng Wang

Senior Scientist, AstraZeneca

Chelsea Thorn

Senior Scientist, Pfizer
Chelsea Thorn

Chelsea Thorn is a Senior Scientist at Pfizer within the BioTherapeutics Pharmaceutical Science Research & Development organization in Andover, MA, US. Dr Thorn is experienced in drug product design across biotherapeutic programs, with a particular interest in mRNA lipid nanoparticles. Her research is broadly focused on developing delivery solutions to advance novel therapeutics into the clinic and is driven by developing breakthroughs that can change patient's lives.
Dr Thorn completed a PhD (Pharmaceutical Science) in 2021 at the University of South Australia with key capabilities in biopharmaceutics, nanoparticle-based drug delivery, and bacterial infectious diseases. Her research developed a lipid-based nanoparticle system as a platform technology to innovate the efficacy of antimicrobials, resulting in a patent filing.
 

Confirmed Representative

, BioPhorum
Confirmed Representative

Derek O Hagan

Senior Advisor R&D, GSK
Derek O Hagan

Heinrich Haas

Chief Technology Officer, NeoVac
Heinrich Haas

Hetal Patel

Senior Principle Scientist/Group Leader, Pfizer
Hetal Patel

I am a group leader/Senior Principal Scientist at Pfizer Analytical Research and Development managing early and late-stage method validation and transfers.
I have a masters in organic chemistry, Ph.D. in protein chemistry/biochemistry from Rutgers University and Postdoctoral from Albert Einstein, NY. I pursued MBA in management from Babson (finance, leadership excellence, people management) during my tenure at Pfizer.
I have hands on experience working with mAbs, mRNA, small molecules, including small molecule synthesis, protein expression, variety of chromatographic/spectroscopic techniques, biochemical and biophysical characterization technologies, GMP inspections/audits, regulatory submissions/query responses. Lately, I have been actively leading implementation of ICH Q2 (R2) and ICH Q14 approaches around using prior knowledge and abbreviated validations in late-stage mRNA and mAb product development.
 

Jay Sarkar

Researcher and Entrepreneur, Stanford University
Jay Sarkar

Dr. Sarkar is a researcher and entrepreneur with a background in applied physics and electrical engineering, which he utilizes to develop systems biology solutions for medicine. He built his first company off his foundational PhD work at Stanford University, where he pioneered the use the mRNA modality for transiently reprogramming cell epigenetics to specifically reset cellular age. The approach has grown as one of the hottest in the longevity community, with some of the largest startup investments in biotech history. He has now progressed to studying the broader problem of new modality integration and distribution in a variety of tissues.

Jim Weterings

Vice President R&D USA RNA Therapeutics & Delivery, Sirnaomics
Jim Weterings

Jim has had a tight bond with Oligonucleotides since 2002, performing research on PNA (conjugates) in the van Boom group at Leiden University, the Netherlands and targeted SSO conjugates at Prosensa. During his Ph.D at Leiden University he synthesized and studied CpG Oligonucleotide and other TLR ligand containing conjugates. At Cenix BioScience in Belgium/Germany, he developed targeted siRNA conjugates for CNS delivery. And at Cristal Therapeutics, the Netherlands he led various projects on the (targeted) nanomedicine delivery of oligonucleotide in oncology. In 2019 he joined AstraZeneca in Sweden, to further the advancement of (targeted) oligonucleotide therapeutics. In December 2022, Jim joined Sirnaomics, USA where he now serves as Vice President R&D USA, driving efforts to deliver RNAi Therapeutics using GalNAc and Peptide Nanoparticle platforms.

 

Kinkini Roy

Associate Director, Aviceda Therapeutics
Kinkini Roy

After finishing PhD and postdoc study from University of Massachusetts Amherst, Kinkini Roy worked in industry for last 12 years in Boston Biotech as well as big corporation. Currently she is leading the program for the next-generation RNA therapeutics using a proprietary synthetic, high-affinity, multi-valent glycans that target extrahepatic cells and improve intracellular delivery and efficacy of siRNA, Anti- sense oligonucleotides, mRNA-based therapeutics, Crispr-Cas9 gene editing and lysosomal targeted protein degradation. Along with that she is leading the formulation development team and develop the formulation development strategy for pipeline development.

Martin Akerman

CTO & Co-Founder, 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.

Neil Robertson

Principal Scientist, TransCode Therapeutics, Inc.
Neil Robertson

Dr. Neil Robertson is a Principal Scientist at TransCode Therapeutics. His research is focused on the application of material science to solve biomedical questions and limitations. Over his career, he has advanced the field of material science for nucleic acid delivery with specific relevance to cancer. His expertise lies in the design and use of novel nanoparticle platforms for the detection and treatment of human pathologies in various disease models. His research is focused on the development of novel nanoparticle constructs for the detection and treatment of cancer. By building on past work and designing new iron oxide nanoparticles, he aims to more precisely target tumor tissue of interest. His goal is to create novel multi-modal imaging constructs which can be useful for both researchers and clinicians. Dr. Robertson received his Ph.D. in Chemistry from the University at Albany, SUNY under the guidance of Prof. Mehmet Yigit and his B.S. in Biochemistry from the University at Buffalo, SUNY.

Richard Geary

EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.
Richard Geary

Dr. Geary is the Executive Vice President and Chief Development Officer at Ionis Pharmaceuticals. He is responsible for preclinical development, clinical development and clinical-stage manufacturing for Ionis’ antisense medicines. Since joining Ionis in 1995, Dr. Geary has been involved in discovery and development including the regulatory submission of more than 50 investigational new drug applications to U.S. and other regulatory agencies. In his position as head of development, Dr. Geary has led six successful antisense medicine development programs through approvals in multiple jurisdictions.
During his tenure at Ionis, he has authored or co-authored 14 book chapters and more than 80 peer-reviewed manuscripts.
 

Sourav Choudhury

Head AAV Technologies, Sanofi
Sourav Choudhury

Sourav Choudhury is a Lab Head in the Genomics Medicine Unit at Sanofi, based in Framingham, MA. He joined Sanofi in 2020 to lead AAV Technologies Laboratory, a platform group focused on AAV capsid engineering and AAV immunology.
Sourav obtained his Ph.D. from UMASS Medical School for his work on development of novel CNS-tropic AAV capsids. He did his postdoctoral training in the laboratory of Feng Zhang at Broad Institute, demonstrating the first in vivo application of CRISPR nuclease Cas12a. Sourav has since worked in Cambridge-based early stage biotechs, first at Intellia Therapeutics designing AAV-based genome editing tools, and then at LogicBio Therapeutics, where he led early-discovery stage indication selection.
 

Steve Pascolo

Founder and CEO, Miescher Pharma GmbH
Steve Pascolo

Trained as an immunologist at the Pasteur Institute (Paris, France), Prof. Steve Pascolo started working on mRNA vaccine in 1998 in Tuebingen, Germany. In 2000, he co-founded CureVac and was Chief Scientific Officer (CSO) of the company, 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 left CureVac and continued the development of immunotherapies based on RNA at the University Hospital of Zurich. In 2008, he founded Miescher Pharma to support this work. In 2017, Prof. Pascolo implemented in the University of Zurich an academic mRNA platform. In collaboration with several research and clinical departments in Zurich he optimizes, tests and implements mRNA based vaccines and therapies.

Yue Hui

Scientist, Moderna
Yue Hui

I earned my Ph.D. degree in Chemical Engineering from Caltech under Professor David A. Tirrell. In September 2022, I joined Moderna as a Scientist in Formulation Discovery. Here, I lead research initiatives focused on LNP process fundamentals and innovation, as well as novel LNP systems for both hepatic and extra-hepatic delivery of nucleic acids.

Zimeng Wang

Senior Scientist, AstraZeneca
Zimeng Wang

Zimeng is a senior scientist of Advanced Drug Delivery, Pharmaceutical Sciences, AstraZeneca. His research in AZ includes formulation optimization to enhance the efficacy and safety of LNP vaccines, development of dry powder LNPs to improve RT stability, and development of LNPs for local/targeted delivery. Before joining AZ, he worked in Phosphorex Inc. on development of sustained release microspheres and targeted delivery nanoparticles. Zimeng got his PhD in University of Rhode Island focusing on development of dry powder aerosol nanocomposite microparticles for the treatment of pulmonary diseases.

Whether you're new to the RNA space or at the forefront of RNA innovation, our 2-day conference is designed to meet your needs with a comprehensive agenda exploring the latest in RNA research and development.

  • Engage, Collaborate, and Learn: Connect with peers to exchange ideas, collaborate on projects, and learn from both the successes and challenges in RNA discovery, development, delivery, and manufacturing.
  • Stay Ahead of the Curve: Dive into sessions covering breakthroughs in mRNA technology, innovative delivery systems, and cutting-edge therapeutic applications. Gain insights from leading pharmaceutical companies such as Pfizer, Moderna, Sanofi, and AstraZeneca.
  • Drive Innovation: Participate in discussions on the future of RNA therapeutics, including the role of AI and machine learning in drug discovery and development. Learn how to overcome current challenges and leverage new opportunities to stay competitive in this fast-evolving field.

Join us in October!
 

Who Should Attend?

Join us at RNA Therapeutics USA and immerse yourself in a hub of innovation and expertise. Engage directly with top-tier professionals including Directors, Heads, and Principal Scientists specializing in RNA Drug Delivery/Development, Genetic Medicine, Analytical Development, CMC/Manufacturing, and Drug Discovery. This event offers a rare platform to delve into the latest scientific breakthroughs, explore cutting-edge technologies, and discuss strategic insights with pioneers shaping the future of RNA therapeutics. Elevate your knowledge, spark new ideas, and discover collaborative opportunities that can accelerate your research and development projects.

sponsors

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 Anelise Longo at Anelise.Longo@saemediagroup.com or call +44 (0) 20 7827 6077

Conference agenda

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8:00

Registration & Coffee

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9:00

Chair's Opening Remarks

Richard Geary, EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.

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9:10

Opening Address: Advancements in Antisense Therapy for Neurological Disorders

Richard Geary, EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.

  • Utilising antisense technology to broaden therapeutic scope
  • Case studies: nusinersen (Spinraza) in spinal muscular atrophy (SMA) for spinal muscular atrophy and tofersen (Qalsody) in ALS with SOD1
  • mutations
  • Opportunities in applying antisense technology as potential therapy for other neurodegenerative diseases and neurodevelopmental disorders
  • clock

    9:50

    RNA-Template Directed Immunotherapy for Cancer

    Neil Robertson, Principal Scientist, TransCode Therapeutics, Inc.

  • A novel delivery system, called TTX, has been developed and validated both preclinically and in a clinical trial for the delivery of nucleic acids to cancer
  • The talk will highlight the application of the novel immunotherapeutic, TTX-RIGA, for cancer immunotherapy through pattern-recognition receptor (PRR) agonism of the RIG-I PRR
  • Preclinical results with TTX-RIGA show inhibition of secondary tumor growth in a model of melanoma
  • clock

    10:30

    Morning Coffee

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    11:00

    RNA Therapeutics development with AI/ML

    Martin Akerman, CTO & Co-Founder, Envisagenics

  • Introducing SpliceCore, an AI-driven platform developed by Envisagenics, tailored to uncover novel therapeutic targets for neurodegenerative diseases like ALS, leveraging the intricate process of alternative splicing which is crucial in neuronal function and disease
  • Decoding ALS with SpliceCore: We will explore the scientific premise of alternative splicing, and the use of AI/ML to develop splicing modulatory drugs to treat sporadic ALS
  • SpliceCore’s predictive power has been validated experimentally, demonstrating its efficacy in identifying novel antisense drug targets for neurodegeneration
  • clock

    11:40

    Advancing Gene Delivery Vehicles with AI/ML

    Sourav Choudhury, Head AAV Technologies, Sanofi

  • Insight into use of viral delivery platforms for improved delivery
  • Opportunities to advance capsid development and further understand capsid functioning through use of AI
  • Examples of machine learning guided capsid development
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    12:20

    Networking Lunch

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    13:20

    Key Considerations for Targeted Delivery of RNA

    Kinkini Roy, Associate Director, Aviceda Therapeutics

  • Importance of targeted delivery for maximizing therapeutic efficacy and minimizing off-target effects
  • Strategies and technologies to achieve controlled and efficient targeted RNA delivery
  • Active vs. Passive Targeting: Comparing targeting methods, their mechanisms, advantages, and applications in RNA therapeutics
  • Major obstacles in targeted RNA delivery and potential ways to address these challenges
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    14:00

    Development of 3-component LNP with fast shedding off PEG copolymer coating

    Zimeng Wang, Senior Scientist, AstraZeneca

  • PEG-lipid in LNP brings “PEG dilemma”: (1) PEG-lipid is essential to achieve desired LNP size during fabrication and maintain the colloidal stability during storage; (2) PEG-lipid could reduce the cell uptake and induce PEG-specific antibodies increasing the clearance rate of PEGylated LNP
  • PEG copolymer coating could be a potential solution to “PEG dilemma”, which maintains particle size during storage but sheds off quickly after administration to improve the in vivo efficacy
  • In this study, we optimized both the fabrication process and LNP composition to ensure desired physicochemical properties and efficacy of poloxamer 407 (P407) coated LNP
  • In vivo study using luciferase mRNA showed initial P407 coated formulation led to increased spleen and lung mRNA distribution comparing to the LNP with PEG-lipid. However, lower luciferase protein level in organs indicated that further optimization is required to improve the LNP efficacy
  • The optimized LNP with increased phospholipid ratio significantly improved the in vitro efficacy of P407 coated LNP
  • clock

    14:40

    Afternoon Break

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    15:10

    Novel Constructs for Network Mediated Large Molecule Drug Delivery

    Jay Sarkar, Researcher and Entrepreneur, Stanford University

  • Drug Routers and the Network Paradigm for Drug Delivery
  • Analysis of Router Induced Networks
  • Distributable Cargo and Cell Type Diversity
  • In Vivo Delivery and Continuing Studies
  • clock

    15:50

    The advantages and disadvantages of the various RNA delivery technologies

    Confirmed Representative

    Confirmed Representative, , BioPhorum

  • Current delivery systems can be unstable with sensitivity to shearing and temperature
  • New and emerging RNA delivery systems could provide more beneficial delivery systems with more agile and effi cient manufacturing methods
  • Technology could be adapted from different platforms to leverage new solutions. However, it is unclear what type of characterization should be performed, how risks are defined and what needs to be addressed for each batch
  • Regulatory guidelines are still in development for RNA technologies and the chemistry, manufacturing and control perspective is unclear
  • Understanding the comparative benefits and challenges across delivery systems can aid more informed decision making during delivery technology selection and development
  • clock

    16:30

    Chair’s Closing Remarks and Close of Day One

    Richard Geary, EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.

    clock

    8:30

    Registration & Coffee

    clock

    9:00

    Chair's Opening Remarks

    Richard Geary, EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.

    clock

    9:10

    GalAhead™ muRNA: A Proprietary GalNAc-RNAi Therapeutic Platform for simultaneous Downregulation of Multiple Genes

    Jim Weterings, Vice President R&D USA RNA Therapeutics & Delivery, Sirnaomics

  • Introduction to GalAhead™, Sirnaomics’ GalNAc-RNAi therapeutic platform
  • Validation of the GalAhead™ muRNA technology in vitro and in vivo
  • Progress report on muRNA GalAhead-based program
  • clock

    9:50

    mRNA process science and engineering: first steps towards a novel modality

    Yue Hui, Scientist, Moderna

  • mRNA as a new modality for vaccines and therapeutics
  • Fundamentals on LNP assembly: understanding the impact of fluid dynamics
  • A case study on CMV vaccine
  • clock

    10:30

    Morning Coffee

    clock

    11:00

    Formulation considerations for mRNA-lipid nanoparticles: vaccines and beyond

    Chelsea Thorn, Senior Scientist, Pfizer

  • Formulation aspects of lipid nanoparticles that enabled the success of mRNA as a vaccine
  • Challenges in formulation and process parameters during product development of mRNA-lipid nanoparticles
  • Considerations when designing mRNA-lipid nanoparticle drug products as a therapeutic vs. vaccine
  • clock

    11:40

    Practical Application of Platform Analytical Procedures in Late-Stage Development of mRNA

    Hetal Patel, Senior Principle Scientist/Group Leader, Pfizer

  • Practical applications of platform analytical procedures leveraging guidance from ICH Q2 (R2) and ICH Q14 and the challenges associated with this paradigm shift
  • Using prior knowledge, experience and historical data to expand the application of platform analytical procedures to reduce redundant work and enable a strong scientific justification for approach to an abbreviated validation in late-stage
  • How prior knowledge including method validation and transfer may be used to enable rapid commercial readiness with streamlined approach
  • clock

    12:20

    Networking Lunch

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    13:20

    Novel Lipid Technology Platforms for RNA Therapeutics and Vaccines

    Heinrich Haas

    Heinrich Haas, Chief Technology Officer, NeoVac

  • To fully exploit the potential of nucleotide-based pharmaceuticals, tailored delivery systems are required
  • Key tasks comprise control of activity, targeting selectivity and immunogenicity
  • Careful selection of lipids and lipid compositions allows to design nanoparticles for a given purpose
  • In-depth analytics and control strategies are of great importance for successful development
  • Here we present examples for controlled assembly for improved mRNA nanoparticle formats
  • clock

    14:00

    The role of adjuvants in vaccine development

    Derek O Hagan

    Derek O Hagan, Senior Advisor R&D, GSK

  • Reviewing the mechanisms and science behind adjuvants, and how they enhance immune responses
  • Exploring the potential of adjuvants to improve the performance of RNA vaccines
  • Future outlook: what will the next generation of adjuvants look like?
  • clock

    14:40

    Afternoon Break

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    15:10

    mRNA Vaccines: The past, present and future directions for advancing

    Steve Pascolo, Founder and CEO, Miescher Pharma GmbH

  • History of mRNA vaccines and how they have developed since COVID-19
  • Insight into the mode of action of synthetic vs natural mRNA vaccines
  • Effective formulation and production strategies to implement for mRNA vaccine development
  • Evaluating the current vaccines and therapies based on synthetic mRNA currently in the market and the key takeaways
  • clock

    15:50

    Fireside Chat: The Future of RNA Therapeutics: Innovations, Technologies, and Modalities

  • Exploring the potential next wave of RNA-based therapeutics poised to hit the market. What are the technologies and research areas that the industry should prioritize and develop?
  • Platforms for developing delivery vectors: latest platforms and strategies for creating effective and reliable delivery vectors
  • Novel and emerging RNA modalities that are expanding the therapeutic landscape, offering new possibilities for treatment
  • Future opportunities for furthering innovation and strategic collaborations
  • Sourav Choudhury, Head AAV Technologies, Sanofi

    Heinrich Haas

    Heinrich Haas, Chief Technology Officer, NeoVac

    Steve Pascolo, Founder and CEO, Miescher Pharma GmbH

    clock

    16:30

    Chair’s Closing Remarks and Close of Day Two

    Richard Geary, EVP, Chief Development Officer, Ionis Pharmaceuticals, Inc.


    Senior Scientist
    Pfizer
    BioPhorum
    Senior Advisor R&D
    GSK
    Chief Technology Officer
    NeoVac
    Senior Principle Scientist/Group Leader
    Pfizer
    Researcher and Entrepreneur
    Stanford University
    Vice President R&D USA RNA Therapeutics & Delivery
    Sirnaomics
    Associate Director
    Aviceda Therapeutics
    CTO & Co-Founder
    Envisagenics
    Principal Scientist
    TransCode Therapeutics, Inc.
    EVP, Chief Development Officer
    Ionis Pharmaceuticals, Inc.
    Head AAV Technologies
    Sanofi
    Founder and CEO
    Miescher Pharma GmbH
    Scientist
    Moderna
    Senior Scientist
    AstraZeneca

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