Conference Day One

Tuesday, December 10, 2024

8:25 am Chair’s Opening Remarks

Showcasing Clinical Immune Monitoring & Analyzing Data to Advance Non-Clinical Safety Testing

8:30 am Case Study: Considerations for the Measurement of LNP Immunogenicity & Review of Potential Impact in Clinical Programs

Synopsis

  • Anti-LNP Immunogenicity considerations, measurement, and impact
  • Considerations of immunogenicity to LNP (e.g. PEG), what and how to measure PEG reactivity
  • Potential impact (with example) of immune responses to LNP

Building In Vitro Tools to Advance Prediction of Clinical Immunogenicity & Toxicity

9:00 am Harnessing Effective In Vitro Tools to Measure LNP Immunogenicity

Synopsis

  • Evaluating which tools are most appropriate to measure various immune components
  • Combining assays to improve efficiency of immunogenicity testing
  • Reviewing which immune measurements and data are most vital depending on target organ
  • Defining signal correlation between non-clinical assays and humans to measure accuracy

9:30 am Utilizing Machine Learning to Improve LNP Safety and Toxicity Profile

Synopsis

  • Developing computational models to screen LNP formulations and predict immunogenicity
  • Leveraging artificial intelligence and machine learning to accelerate LNP development
  • Interpreting learnings to influence design and delivery of safer LNP molecules

10 am Morning Break & Speed Networking

11:00 am Building Optimized LNP Molecules to Deliver Vaccines that Balance Immunogenicity & Reactogenicity

Synopsis

  • Determining baseline reactogenicity to characterize immunogenic effect
  • Designing LNP structures that strike the balance between desired immune response and unwanted adverse effects
  • Optimizing delivery specificity to mediate targeted immune activation while minimizing off-target effects

11:30 am Optimizing Ionizable Lipid & LNP Properties to Improve Safety

  • Sean Semple Vice President - Preclinical Development, Acuitas Therapeutics

Synopsis

  • LNP toxicity profiles
  • Impact of lipid, cargo and LNP properties on safety parameters
  • Correlations with pharmacokinetics and tissue distribution

12:00 pm Lunch Break & Networking

Redefining LNP Design to Combat Immunogenicity & Guarantee Specificity

1:00 pm Fireside Chat: Defining Best-Practice Methods to Understand the Impact of Formulation & Delivery Approaches on LNP Immunogenicity & Toxicity

  • Maja Sedic Senior Director, Toxicology, Orna Therapeutics
  • Kevin Sun Assistant Professor, Icahn School of Medicine at Mount Sinai

Synopsis

  • Discussing how to utilize novel methods, such as screening, computational methods and AI, to uncover and understand immune response to LNPs
  • Unravelling how dose and delivery method can impact safety profiles, and more specifically immunogenicity vs toxicity
  • Distinguishing how methods may vary by modality, approach or payload

2 pm Afternoon Break & Poster Session

Synopsis

This is the perfect opportunity to contribute to the conversation and share your cutting

edge research with this community while discovering exciting work carried out by your

peers. Get in touch if you would like to be involved!

Optimizing Non-Clinical & Translational Approaches to Assess LNP Safety Risk

3:00 pm Non-Clinical Testing Strategy of an RNA Lipid Formulation as Platform Approach

Synopsis

  • Non-clinical testing
  • RNA lipid formulation
  • Platform approach

3:30 pm Preclinical Safety Assessment of LNP-Formulated Drugs & Vaccines

Synopsis

  • Determining the most effective control to measure base immunogenicity of LNP
  • Reviewing how different cargos might interact with nanoparticle characteristics and subsequently alter safety profile
  • Testing immunogenicity with and without cargo to determine if cargo or payload impacts LNP safety

4:00 pm Translational Challenges in LNP-mRNA Systemic Delivery

Synopsis

Talk Details TBC

4:30 pm Close of Conference Day One