8:30am | Morning Registration & Coffee

9:20 am Chair’s Opening Remarks

  • Tao Niu Associate Director, Modeling & Simulations, Vertex Pharmaceuticals

Exploring the Effect of Composition of LNPs & Accurate Pre-Clinical Model Prediction Strategies

9:30 am Nanoparticle Single-Cell Multiomic Readouts Reveal That Cell Heterogeneity Influences LNP-Mediated mRNA Delivery

Synopsis

  • Exploring high-throughput LNP screening
  • Optimizing mRNA-based LNP delivery at single cell resolution
  • Researching biodistribution and functional delivery of an mRNA-carrying LNP and identify immunogenic LNPs

10:00 am Roundtable: Evaluating Pre-Clinical In Vitro Models for Safety of LNPs

  • Philippe Collin Director - Delivery Systems, Clinical Pharmacology & Safety Sciences, AstraZeneca

Synopsis

  • Understanding the influencing factors of safety – lipids, modality
  • Outlining performance and translatability of in vitro models
  • Maximizing the potential of gene engineering and high-throughput methodologies

10:45 am Understanding of Immune Recognition of Nucleic Acids on Application Nanoparticles

Synopsis

  • Different therapeutic nucleic acids (TNAs) can be organized in programmable nucleic acid nanoparticles (NANPs) for their simultaneous delivery to the diseased cells and further synchronized action
  • Understanding immunological properties of TNAs and NANPs to expedite their clinical translation
  • Delving into immunorecognition of NANPs defined by their architectural parameters that can be tuned to address specific therapeutic needs

VIRTUAL SESSION

11:15am | Morning Break & Speed Networking

12:00 pm Modifying Immunogenicity with a Multi-Cargo LNP System

  • Heather Denroche Director of Preclinical Development, Integrated Nanotherapeutics Inc.

Synopsis

  • Discussing how additional functionalities (e.g., immune modulation) are enabled in a multi-cargo LNP system
  • Reducing unwanted immune responses to LNP delivery systems
  • Developing immune tolerizing LNPs for disease applications

12:30 pm Immunological Properties of LNPs & Nucleic Acid-Based APIs

  • Marina Dobrovolskaia Director of Operations, Nanotechnology Characterization Lab, Frederick National Laboratory for Cancer Research

Synopsis

  • Exploring how the nanomaterial interacts with the immune system as an entity
  • Reviewing the immunological compatibility of the carrier and API
  • Understanding that the final formulation has unique properties that neither API or carrier have stand-alone

VIRTUAL SESSION

Measuring & Modulating Immunogenicity of LNP-Payload Composition

1:00pm | Networking Lunch

2:00 pm Translational Models to Evaluate LNP Immunogenicity & Reactogenicity

  • Joshua DiNapoli Global Project Head - mRNA Platform Development, Sanofi Pasteur

Synopsis

  • Reviewing models currently used in the field and the value they bring in ranking lipids for immuno and reactogenicity
  • Discussing new models that can be considered (e.g. MIMIC) and data supporting them
  • Exploring challenges in model translatability

Spotlight Session

2:30 pm Understanding Immune Responses Linked to Physio-Chemical Characteristics of Non-Viral Delivery Systems

  • Dr Neill Liptrott Reader in Pharmacology & Immunocompatibility, University of Liverpool

Synopsis

  • Discussing the need for robust physio-chemical characterisation of delivery systems, to link to biological effect
  • Evaluating the importance of route of administration for prioritizing immune assessment
  • Requirement of more complex, physiologically relevant, models to assess immune responses

VIRTUAL SESSION

3:00pm | Afternoon Networking Break

3:30 pm Integrating Tolerability Assessments into Early LNP Discovery

  • Cory Sago Formerly Senior Director, Head of LNP Discovery, Beam Therapeutics

4:00 pm Examining Immunological Profile of mRNA-LNP Therapeutics

Synopsis

  • Determining interplay of immune responses with compositions and
    characterization of LNP
  • Researching innate and adaptive immune systems of mRNA-LNP delivery
  • Outlining mRNA-LNP delivery and immunogenicity

VIRTUAL SESSION

4:30 pm Complement Activation Related Pseudoallergy Associated with LNPs

  • Peter Bedocs Senior Scientist, Defense & Veterans Center for Integrative Pain Management

Synopsis

  • Delving into drivers of complement activation: size, shape, and charge of your LNP
  • Understanding LNP interactions with complement proteins and subsequent activation of complement
  • Identifying and preventing the impacts of complement activation

5:00pm | Chair’s Closing Remarks & End of Day One

  • Tao Niu Associate Director, Modeling & Simulations, Vertex Pharmaceuticals