3rd Gene Therapy Potency Assay Summit 2025

Topics

• Statistical Analysis of Key Data Packages to Achieve Regulatory Approval
• Understanding the Regulators to Reach Clinical Success
• Establishing an Optimum Robust Potency Assay Strategy to Avoid Regulatory/Clinical Setbacks
• Breaking Boundaries: Setting Gold Standard Regulatory Requirements
• Roadmap for Developing Robust Potency Assays for Accelerated Regulatory Approval
• Establishing an Optimum Robust Potency Assay Strategy to Avoid Regulatory/Clinical Setbacks
• Establishing Strategies to Develop Potency Assays to Ensure Transferability from Pre-Clinical to Clinical
• Performing the Potency Matrix
• Roadmap for Developing Robust Potency Assays for Accelerated Regulatory Approval
• Overcoming Reference Material Restraints
• Phase Appropriate Adaptations to The Reference Standard to Ensure Transferability of Potency Assays Through the Product Cycle
• Reference Standards in Cell Based Potency Assays
• Afternoon Refreshments & Poster Session
• Understanding Biological Variability Between Cell-Based Assays and How to Overcome Them
• When and How to Use Animal Models as an Alternative to Measuring Potency
• Investigating Potency Assays with Unknown MOA’s and the Relevant Biological Function to Accurately Measure Potency
• Establishing an Optimum Robust Potency Assay Strategy to Avoid Regulatory/Clinical Setbacks
• Breaking Boundaries: Setting Gold Standard Regulatory Requirements
• Roadmap for Developing Robust Potency Assays for Accelerated Regulatory Approval
• Establishing an Optimum Robust Potency Assay Strategy to Avoid Regulatory/Clinical Setbacks
• Establishing Strategies to Develop Potency Assays to Ensure Transferability from Pre-Clinical to Clinical
• Performing the Potency Matrix
• Roadmap for Developing Robust Potency Assays for Accelerated Regulatory Approval
• Overcoming Reference Material Restraints
• Phase Appropriate Adaptations to The Reference Standard to Ensure Transferability of Potency Assays Through the Product Cycle
• Reference Standards in Cell Based Potency Assays
• Afternoon Refreshments & Poster Session
• Understanding Biological Variability Between Cell-Based Assays and How to Overcome Them
• When and How to Use Animal Models as an Alternative to Measuring Potency
• Investigating Potency Assays with Unknown MOA’s and the Relevant Biological Function to Accurately Measure Potency
• Improving Cell Based Assays to Produce Repeatable and Reliable Results
• The Essential Attributes to Consider During Development, Optimization and Qualification of Cell-Based MoA Potency Assay for AAV Gene Therapy Products
• Quantitative, Image-Based Assays for the Functional Evaluation of RNA and Gene Therapies Targeting Muscle Disorders
• Cell Line Development Strategies for AAV Potency Assays
• Improving Cell Based Assays to Produce Repeatable and Reliable Results
• What Does Long Term Programme Management Look Like for Potency as The Life Cycle of Your Product Evolves?
• Best Practices, Platform Methods and Automation Tools for Successful mRNA Expression Assays for AAV Gene Therapy
• Appropriate Qualification Data to Meet Regulatory Requirements
• What Does Long Term Programme Management Look Like for Potency as The Life Cycle of Your Product Evolves?
• Understanding Biological Variability Between Cell-Based Assays and How to Overcome Them
• Improving accuracy of potency assays to produce reliable results