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

  • Craig Jerome Bio-Assay Associate, Beacon Therapeutics
  • Nikhil Jain Senior Associate Scientist, Intellia Therapeutics

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

  • Margaret Butko Associate Director - Protein & Analytical Characterization, Adverum Biotechnologies

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

  • Scott Burger Principal, Advanced Cell & Gene Therapy
  • Zoe Yin Independent Consultant, CMC, Self Employed

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

  •  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

  • Dawn Henke Senior Manager - Technical Program, Standards Coordinating Body
  • Katie Zander Director, Standards Education & Outreach, Standards Coordinating Body

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? 

Workshop H
1:00 – 4:00 pm

Validating Tools for the Quality Control Environment

  • James Yuan Senior Scientist, PTC Therapeutics
  • Wei Cao Lead Scientist, Analytical Program Development, Spark Therapeutics

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