8:20 am Registration & Morning Coffee

9:20 am Chairs Opening Remarks

9:30 am Defining Criteria for Quality Control Comparison Analysis in Lentiviral Vectors


• Optimizing your quality control assessments for lentiviral vectors

• Navigating the specific challenges of cytotoxicity when dealing with lentiviral vectors

• Understanding your product and process is key in establishing the product profile and CQAs for comparability


10:30 am Rethinking the Potency Paradigm for Gene Therapy Products


• Highlighting and targeting the current challenges in developing appropriately robust potency assays for gene therapy products

• Illuminating developments in potency assays measuring target function

• Discussing how you can implement more robust potency assays in your own comparability assessments

11:00 am First-Principles Biophysical Techniques are Critical in Preventing Interpretation Errors that can Confound Comparability Studies

  • Ronald Toth Senior Scientist - Characterization, Sanofi


'Jingle' fallacies common in gene therapy analytics are discussed – e.g., the difference between mass and number weightings is often not appreciated when considering constructs such as "%empty" and "%HMW". The concept of "assay poise" is introduced – e.g., how well "posed" are the techniques SEC and DLS to measure High Molecular Weight when their separation axes are by diameter and not by mass? How well "posed" are A260/A230 methods to measure %empty? SV-AUC is presented as a first-principles technique with true mass-based separation enabling it to bridge between techniques with mass and number weightings and to distinguish between species that are truly High Molecular Weight and species that are simply "High-Diameter". DLS and Nanosight are presented as first-principles techniques capable of bridging between techniques producing number and mass weighted particle size distributions. As a final example of a jingle fallacy, the wildly different implementations of DLS offered by vendors are discussed with an emphasis on comparing the regularization algorithms offered by Malvern and Wyatt for the purposes of quantifying AAV High-Diameter Species.

11:30 am Morning Refreshments

12:00 pm Developing High Throughput Appropriate Tools for Process Comparability

  • Ivan Budyak Director - Analytical Development & Biophysical Characterization, Eli Lilly & Co.


• Accentuating recent development in appropriate analytical tools for comparing processes

• Addressing gaps in current analytical tools for advancement in the future

• Determining which analytical tools will complement your comparability assessments of process change


12:30 pm Extended Characterization of AAV Capsids by Mass Spectrometry to Determine Critical Quality Attributes

  • Marco Thomann Principal Scientist and Group Leader, Roche Diagnostics GmbH


• Challenges in characterizing AAV capsids

• How mass spectrometry can be used in extended characterization

• Determining CQAs from mass spectrometry

1:00 pm Lunch

2:00 pm Quantifying & Characterizing AAV Gene Therapy Products Using Transmission Electron microscopy & Molecular Methods

  • David Dobnik Senior Associate - Research, National Institute of Biology


• Optimizing processes for quantifying and characterizing AAV products to speed up comparability assessments

• Describing both upstream and downstream optimization tactics to streamline quantification and characterization

• Outlining the benefits of using TEM and molecular methods for quantification and characterisation of AAV gene therapy products

2:30 pm Benchmarking the State of Regulatory Expectations on Stability & Release Characterization


• Hearing about the current expectations from regulators on stability and release characterization

• Outlining the best methods for stability and release characterization to meet these expectations

• Using results from these tests and guidance from regulators to store batches

3:00 pm End of Conference Day Two