Conference Day Two
Thursday 14th September 2023
8:30 am Morning Coffee
9:15 am Chair’s Opening Remarks
9:20 am Supercharging Potency Assay Development to Establish the Required Breadth of Assays Capable of Presenting Total Functionality
9:30 am Highlighting the Breadth of Potency Assays Available to Industry to Initiate a Matrix Approach
Synopsis
- Determining what the right potency assays are for LNPs and selecting accordingly following characterization
- Considering the specifics of the payload when selecting the potency assays
- Building the breadth of assays to accommodate the different complexes
10:00 am Rationalizing Appropriate Reference Material to Establish Batch to Batch Consistency
Synopsis
• Recruiting a suitable reference material for all potency assays to serve as a basis of evaluation
• Digesting reference standard data to extract the potency information
• Adopting a matrix approach to reference material to mirror that of the potency assay design
10:30 am Roundtable Discussion – Honing Phase to Phase Potency Assay Considerations to Accommodate the Shifts in Required Output
Synopsis
- Managing expectations of the regulatory bodies for potency assays from phase to phase
- Spotlighting the shifts in focus and the desired outcomes from each assay at varying stages of development
- Considering the variations in cell-based potency assays and the implications of this heterogeneity
11:15 am Morning Refreshments & Networking
11:30 am Revealing Advancements in Technology to Improve Efficiency of Analytical Methods
11:45 am Roundtable Discussion – Developing a Robust Characterization Assay Panel to Support GMP Release Assays and CQA Assessment
Synopsis
- Discussing the requirements of a strong characterization assay panel
- Defining GMP standards in relation to LNP Drug Products
- Overviewing CQA criteria with an understanding of the assays available to clearly present the data with an appreciation of the gaps in the industry as it stands
12:15 pm Enlightening the Strategies of Characterizing Co-formulated LNPs to Ensure Precision of Analytics
Synopsis
- Outlining the future scope of characterization methods with co-formulated LNPs
- Differentiating the distribution of the two payloads across the LNP to understand encapsulation variance
- Addressing the efficiency of endosomal release with 2 payloads to maximize cellular uptake and subsequent translation
12:45 pm Panel Discussion – Leveraging an Array of Imaging Tools Synergistically to Create a Detailed Profile of the LNP Complex
Synopsis
- Combining variable sources of LNP imagery to collate a holistic analytical profile of the LNP
- Interpreting LNP images to differentiate internal structures of the LNP without ambiguity
- Furthering LNP imaging technology with the potential of labelling techniques alongside existing techniques
1:30 pm Lunch & Networking
1:59 pm Spotlighting Endosomal Release & Cellular Uptake to Quantify the Efficiency of Payload Delivery
2:30 pm Roundtable Discussion – Discovering the Metrics for Cellular Uptake to Improve Understanding of LNP Delivery Efficiency
Synopsis
- Outlining the mechanisms of action for cellular uptake with an understanding of LNP endosomal release
- Overcoming the distribution variance of payload across the LNPs to maximise targeted cellular uptake
- Understanding cell selectivity when characterizing the LNP with an appreciation of the target cell
3:00 pm Turbocharging Assay Development to Comprehensively Measure Endosomal Release
Synopsis
- Creating metrics for endosomal release to form the basis of a thorough potency assay
- Benchmarking successful endosomal release to standardize efficient payload delivery
- Maximizing the efficiency of expression with the aim of reducing the dosage required of the LNP
3:30 pm Audience Discussion – The Future of LNP – What is Next for Characterization & Analytical Development Research?
Synopsis
- Reflecting on key lessons learnt from the summit to create a clear plan for future analytics
- Debating the technological advancements required to drive development to the next stage
- Reaffirming goals for the industry and realistic progression expected by 2024