DAY ONE - Wednesday July 17, 2024
7:50 am Check in & Coffee
8:50 am Chair’s Opening Remarks
ENSURING PHASE-APPROPRIATE POTENCY ASSAY DEVELOPMENT
9:00 am Determining the Relative Importance of Identity Vs Functional Potency Assays Throughout Early to Late-Stage Potency Assay Development
Synopsis
- Evaluating the calculated risk involved in initiating potency assay development early in clinical phases (phases 1 and 2) to mitigate comparability challenges
- Identifying the importance of early identity potency assays for CMC compliance
- Highlighting the increasing expectations from regulators for potency assessment at all phases of clinical development
9:30 am (CASE STUDY) Understanding Potency Method Development, Bridging & Life Cycle Strategies
Synopsis
- Presenting a case study of assay development, including MOA-based potency assay design, optimization and robustness studies, and data analysis
- Communicating mechanisms of method qualification and the setting of acceptance criteria
- Extracting learnings from case-study examples of potency method life-cycle management
10:00 am (CASE STUDY) (NEW DATA) Bridging Potency Assays in Gene Therapy Clinical Development: Methods for Adjusting the Potency Matrix Between Clinical Phases
Synopsis
- Presenting case-study examples of both, a protein expression and viral entry assay to determine their relative importance across developmental phases
- Highlighting the increasing expectations from regulators for potency assessment at all phases of clinical development to determine acceptable differences in assay readouts between clinical phases
- How should you adapt your potency matrix approach through the early to latestage transition?
10:30 am Developing a Cell-Based Matrix Approach for AAV Gene Therapy
Synopsis
- Understanding the variability of TCID50
- Optimizing a function potency assay: challenges and lessons learned
- Incorporating a protein expression assay into a potency strategy during product development
11:00 am Speed Networking & Refreshments
Synopsis
This speed networking session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the gene therapy potency assay field and establish meaningful business relationships to pursue for the rest of the conference.
UNDERSTANDING REGULATORY EXPECTATIONS TO GUIDE POTENCY ASSAY PANEL SELECTION
12:00 pm (CASE STUDY) Can we Negotiate with Regulators? Outlining Lessons Learnt from Successful Regulatory Interactions to Help Facilitate Compromise
Synopsis
- A real-world example of an end-end potency assay design strategy that resulted in a regulatory approval
- Discussing how feedback from regulators was incorporated into potency assay design as the drug product moved towards commercialization
- Outlining how interactions with regulatory authority can ultimately lead to compromise in potency assay expectations
12:30 pm Optimizing Potency Assay Selection for Release Panels: Aligning Mechanism of Action/s & strategies
Synopsis
- Choosing appropriate potency assays for release panels
- Understanding the importance of continuously monitoring diverse potency attributes throughout clinical development
- Emphasizing the correlation between selected potency readouts and product critical quality attributes to
1:00 pm Networking Lunch
HOW DO OTHER CRITICAL QUALITY ATTRIBUTES IMPACT POTENCY ASSAY DEVELOPMENT?
2:00 pm Using Potency Readouts to Establish Critical Quality Attributes & Inform Vector Characterization Priorities
Synopsis
- Comparing the relative importance of AAV CQAs compared to mAbs
- Determining how the biophysical characteristics of AAV can affect potency readouts
2:30 pm (NEW DATA) Outlining the Utility of a Well-Performing Gene Therapy Functional Bioassay for Effective CQA Determination & Product Understanding
Synopsis
- Description/Development of functional bioassay
- Utilization of bioassays for CQA determination and for product understanding
3:00 pm ROUNDTABLE: Discussing the Influence of Post-Translation Modifications & Other Manufacturing Considerations on Gene Therapy Vector Potency to Better Define Relevant CQAs
Synopsis
Roundtable discussions include a larger focus on group discussion. A moderator will introduce the session topic and attendees then split into groups to discuss a series of predetermined agenda points. At the end, all groups report back on their discussions, and findings are collated.
This roundtable will cover:
- Understanding how post-translational modifications and other manufacturing considerations can impact vector functionality and cellular entry capacity
- Discussing the complexities of assessing modifications’ effects on potency within the context of biological variability
3:30 pm Afternoon Networking Break & Poster Session
DEVELOPING PROTOCOLS FOR EFFECTIVE RESOURCE MANAGEMENT & COMMUNICATION WITH CROS
4:00 pm Ensuring Appropriate Documentation Handling & Resource Management to Ensure a Smooth Transition During Tech Transfer
Synopsis
- Discussing the necessity to qualify every element of potency assays during the tech transfer process
- Exploring effective resource management techniques essential for successful tech transfer
- Highlighting proactive measures to anticipate and address potential hurdles during tech transfer, such as identifying bottlenecks, contingency planning, and fostering collaboration
4:30 pm Optimizing Number of Potency Assays Performed to Accelerate Commercialization without Draining Resources
Synopsis
- Balancing the quantity of potency assays performed with their relative time and resource strain
- Prioritizing assay development based on scientific rationale and regulatory expectations (including absolute potency assays, employing signal enhancers, and utilizing orthogonal methods)
- How to invest in relevant assays upfront to optimize resource use and mitigate financial risk
- To what extent can we streamline assay selection to accelerate commercialization?
5:00 pm PANEL: Partnering & Communicating Effectively with CROs – Finding a Company That Understands Your Specific Scientific Needs & Potency Assay Requirements
Synopsis
Moderated by Shashwat Mishra, Principal Scientist – Analytical Development, Regeneron
- Overcoming the bottlenecks of lengthy GMP training periods and high CRO turnover rates
- Analysing the impact of limited CRO capabilities and the need to reduce assay complexity for a smoother tech transfer
- Balancing timelines between gene therapy developers and CROs to expedite development processes