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

  • Zheng Meng Senior Director - Analytical Development & Chemistry Manufacturing Controls, Ultragenyx


  • 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


• 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


  • 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


  • 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


  • 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

  • Sixuan Li Postdoctoral research assistant, Pfizer
  • Zack Zhang Director, Analytical Development, Beam Therapeutics
  • Zheng Meng Senior Director - Analytical Development & Chemistry Manufacturing Controls, Ultragenyx


  • 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


  • 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


  • 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?


  • 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 

4:00 pm Chair’s Closing Remarks

4:15 pm End of Conference