Pre-Conference Workshop Day
November 27, 2023
8:00 am Registration & Morning Refreshments
BIOASSAYS
MOLECULAR BIOLOGY
Workshop B 9:00 – 12:00 pm
Breaking Down the Analytical Toolbox for Characterizing Genomic Integrity
Synopsis
- Contrasting the pros and cons of different analytical techniques to quantitively characterize genome integrity
- Discussing regulatory attitudes and best methods for transitioning between characterization technologies
- Overcoming gene therapy-specific challenges – what is the best tool to use to characterize unknown and partial capsid genomes?
PHYSICOCHEMICAL PROPERTIES
Workshop C 9:00 – 12:00 pm
Bringing Semi-Quantitative Empty/Full Assays to All Stages of the Manufacturing Process
Synopsis
- Discussing empty/full method requirements for release testing vs. in process testing
- Comparing empty/full readouts from samples tested on traditional and new empty/full methods
- Contrasting empty/full readouts from samples tested in upstream and downstream buffers of a manufacturing process
QUALITY & PROCESS DEVELOPMENT
Workshop D 9:00 – 12:00 pm
Applying Biologics Development Practices to Gene Therapy Products: Platform Methods & Quality by Design
Synopsis
- Outlining lessons from biologics to generate platform methods for gene therapy analytics
- How well do QbD principles fit when applied to gene therapy analytical development?
- Breaking down practices to adapt to the realities of gene therapy – adjusting a round hole to fit a square peg
12:00 pm Lunch & Networking
Workshop E 1:00 – 4:00 pm
Where to Start – How to Set Up Potency Assay Development for Success
Synopsis
- Laying out the timeline for potency assay development
- Dealing with a large number of variables with no clear flowchart for potency assay development
- What avenues are the best to try initially, and how do know you are on the right path?
- Streamlining team ideas to focus on a common goal for assay development
- Utilizing a back-up assay approach to develop assays in parallel outside of supply chain restrictions
Workshop F 1:00 – 4:00 pm
Utilizing NGS for enhanced product understanding of AAV gene therapy vectors
Synopsis
- Â High-level overview of NGS and rAAV gene therapy
- Overview of NGS for rAAV product understanding
- Interactive workshop relating to rAAV multiattribute measurements by NGS
- High-level overview of rAAV NGS analysis and validation strategies
Workshop G 1:00 – 4:00 pm
Identifying General Standards for the Assessment of Gene Therapies
Synopsis
- Outlining different interpretations of terminology and the impact on comparability conversations within analytical consortia
- Identifying current gaps and needs in gene therapy standards
- Linking different interpretations to lack of standardization across different assay measurements and the impact on regulatory expectations
- What are the best practices to achieve industry consensus and standardization?Â
Workshop H 1:00 – 4:00 pm
Validating Tools for the Quality Control Environment
Synopsis
- Identifying which emerging tools are fit for purpose in newer modalities like gene therapy
- Exploring case studies of assay and method qualification and validation: what should constitute the validation data package
- Case study: optimizing ddPCR assay for determination of vector genome titer in AAV
- Hearing examples for those in late-stage development and exploring strategies for analytical development and quality control collaboration and harmonization
4:00 pm Close of Pre-Conference Workshop Day
To Access the Main 2-day Agenda, Download the Full Event Guide HERE