DAY ONE - Wednesday July 17, 2024
7:00 am Check-In & Light Breakfast
7:50 am Chair’s Opening Remarks
ENSURING PHASE-APPROPRIATE POTENCY ASSAY DEVELOPMENT
8:00 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
8:30 am Cell and Image-Based Assays for the Functional Evaluation of Gene Therapies Targeting Muscle Disorders
Synopsis
- Describing image-based bioassays suitable for measuring activity and functionality of gene therapy products targeting multiple muscle disorders, including DMD and DM1
- The MyoScreen Platform enhances the standardization of myotube culture compared to conventional culture plates, allowing for the reduction of inherent variability in cell-based assays, and high throughput imaging and analysis of specific proteins
- Described bioassays provide insights into the structure-function relationship of therapeutics and can be customized as potency assays, from early CMC development to lot release
9:00 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?
9:30 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
10:00 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
11:00 am (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
11:30 am 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
12:00 pm ROUNDTABLE: Unpacking the Details of the New FDA Guidance: “Potency Assurance for Cellular & Gene Therapy Products” – Changes, Learnings & Implications
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:
- Outlining the major changes made to the new FDA guidance to understand the reasoning behind the updates
- Making comparisons with the previous guidance and identifying significant shifts or emphasis changes
- Discussing how the new guidelines should be interpreted and implemented to guide novel assay design approaches
- Introducing case-study examples of the new guidance in practice
- Discussing potential hurdles and opportunities in implementing revised guidelines
- Outlining how the new guidance may influence your potency assay strategy going forward
12:45 pm Networking Lunch
HOW DO OTHER CRITICAL QUALITY ATTRIBUTES IMPACT POTENCY ASSAY DEVELOPMENT?
1:45 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
2:15 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
2:45 pm ROUNDTABLE: Optimizing the Number of Potency Assays Performed to Accelerate Commercialization Without Draining Resources
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:
- Meeting regulatory requirements for potency assessment
- The roadmap to get there: having phase-appropriate potency assays at the ready for critical milestones
- Being smart about assigning resources while developing a potency matrix
3:30 pm Afternoon Networking Break & Poster Session
DEVELOPING PROTOCOLS FOR EFFECTIVE RESOURCE MANAGEMENT & COMMUNICATION WITH CROS
4:15 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