Conference Day One
Wednesday, 25 September
7:45 am Check In & Refreshments
8:45 am Chair’s Opening Remarks
Magnifying the Structural Complexities of LNPs Identified Throughout Pre-Clinical Research
9:00 am Exploring the Vast Array of Lipids for LNP Formulation with Insights into Lipid Characterisation to Optimise Drug Delivery
Synopsis
- Revealing the impact of lipid components on LNP size, structure, stability, PKa, encapsulation efficiency and endosomal release
- Unravelling the crucial roles of ionisable cationic lipids in LNP encapsulation and subsequent endosomal release
- Employing sophisticated screening techniques to create a comprehensive lipid profile, reducing the risk of LNP instability and impurities
9:30 am Addressing the Translatability and Delivery Outcomes of Lipid Nanoparticles Across a Spectrum of Research Models
Synopsis
- Achieving an equilibrium between potency and toxicity during LNP screening
- Translating toxicity observations across various models to human in vivo trials to guarantee product safety
- Leveraging in vitro experimental insights to establish a foundational understanding for translation into in vivo settings
10:00 am Optimizing scale-up to GMP manufacturing of RNA-LNPs
10:30 am Morning Refreshments & Speed Networking
Creating a Delivery Vehicle Capable of Pinpoint Targeted Delivery to Avoid Liver Leakage
11:30 am Evaluating the Role of Lipid Components in Targeted Delivery, Specifically Acknowledging the Helper Lipid as a Key Component to Success
Synopsis
Brand New Data!
- Investigating lipid components in isolation to understand the extent of the role it has on targeted delivery
- Reviewing the location of the lipid component and how this impacts delivery outcomes, including internal or external pretrusion from the surface of the lipid nanoparticles
- Spotlighting the mechanisms of action as a result of the helper lipid and how this can be used for optimal LNP formulations
12:00 pm Showcasing Innovative Research to Develop a Targeted Delivery Vehicle to Increase Efficiency of Drug Delivery
Synopsis
- Reviewing the traditional approaches to formulate an LNP with targeted delivery in mind
- Outlining novel steps to create an LNP capable of extra-hepatic delivery to reach the previously unreachable, widening the therapeutic potential
- Evaluating the risk of safety and toxicity with novel LNP formulations to maximise targeted delivery whilst reducing adverse effects
12:30 pm Explore the latest analytical tools for precise characterization of lipid nanoparticles (LNPs)
Synopsis
Gain insights into how LCMS is utilized to profile impurities in ionizable lipids used in constructing LNPs. Additionally, learn how CE is employed to study encapsulation efficiency, profile mRNA impurities, and assess the purity of plasmid building blocks. Finally, discover how LCMS is advancing to delve into the structure of oligonucleotides and mRNA
1:00 pm Lunch & Networking
Spotlighting Toxicity & Immunogenicity to Develop an LNP with Comprehensive Safety Profiles
2:00 pm Foreseeing Challenges with Immunogenicity Through Robust Analysis of LNPs to Unpick Factors Behind Immune Response
Synopsis
- Understanding of LNP immunogenicity and toxicity read-outs in relation to a variety of therapeutic types and disease indications
- Advancing immunogenicity models to minimise anti-PEG response, as well as alternatives to reduce adverse effects
- Altering LNP design and raw materials to combat immunogenicity challenges
2:30 pm Panel Discussion: Overviewing the Greatest Risks Posed by Lipid Nanoparticles to Mitigate Adverse Affect Occurence
Synopsis
- Sharing insights into the adverse affects recorded across the literature and triaging the common causes of such events
- Spotlighting steps of formulation which require greatest attention to control the toxicity profile of the LNP
- Considering bioaccumulation of LNPs and the types of therapeutics and target sites which increase this risk
3:15 pm Comprehensive LNP Characterization from Cargo to Target Modification Using Nano-Flow Cytometry
Synopsis
- Rapid single particle measurements of LNPs RNA cargo and surface targeting moieties can be easily achieved with the NanoAnalyzer
- Thousands of individual LNPs are assessed for their size, concentration, RNA presence and quantity, surface ligand density delivered in a simultaneous measurement
- Strengthen formulation design, improve down-selection of leading preparations and identify characteristics which lead to superior formulations
3:30 pm Afternoon Refreshments & Networking
Driving the New Wave of Manufacturing Techniques to Ensure Safety and Efficiency of Production
4:00 pm Experimenting with the Shear Stress Placed Upon the Lipid Nanoparticle in Accordance with the LNP Design and Malleability
Synopsis
Brand New Data!
- Manipulating shear boundaries to optimise manufacturing capabilities whilst minimising damage to LNP
- Screening an LNP to profile the potential of damage across a spectrum of shear stress levels
- Visualising the extent of shear stress damage to quantify batch viability throughout manufacture
4:30 pm Manufacturing of RNA-LNP-loaded Dry Powders for the treatment of Pulmonary Diseases
Synopsis
- Introducing a platform technology for the production of Nano-Embedded in Microparticles (NEMs)
- NEMs as a dry powder formulation suitable for pulmonary delivery
- Illustration of the NEM potential in an in vivo proof-of-concept (PoC) study and in ex vivo human lung grafts in an allergic asthma model, targeting GATA3 which plays a pivotal role in the pathophysiology of severe type-2 asthma