DAY ONE - Wednesday | May 28, 2025
7:30 am Registration & Coffee
7:55 am Chair’s Opening Remarks
08:00 – 10:00 | MORNING MASTERMIND
Navigating Regulatory Requirements for Gene Therapy Potency Assays
Synopsis
This session delves into the complexities of regulatory strategies for gene therapy potency assays, providing an in-depth exploration of both early and ongoing regulatory engagement. Participants will learn how to build effective relationships with regulatory bodies and prepare for regulatory meetings and submissions.
Designed to equip participants with the knowledge and skills to navigate the regulatory landscape effectively, this session offers insights and practical applications that extend beyond the main conference. With contributions from experts whose gene therapy products are at various stages of clinical development, attendees are guaranteed to gain valuable insights that can be applied to their current or upcoming regulatory applications and assay development strategies.
Developing & Validating Potency Assays Strategies to Ensure Robustness & Reliability
10:00 am Morning Refreshments & Speed Networking
Synopsis
Connect with all your fellow peers! This is your opportunity to network and forge new contacts with experts in assay development. Join speed networking to meet a variety of attendees at the summit, and exchange details to catch up later in the event.
11:00 am Evaluating Strategies for Creating & Validating Multiple Potency Assays
Synopsis
- Designing assays for different therapeutic modalities to enhance robustness
- Ensuring assay reproducibility to maintain consistency
- How can new technologies be integrated for improved assay development?
11:30 am Incorporating Matrix Approaches in Assay Development
Synopsis
- Understanding what matrix approaches are and their critical role in gene therapy potency assays
- Utilizing matrix approaches to standardize assay conditions, reduce variability, and enhance reproducibility
- Exploring various techniques and tools for implementing matrix approaches, including case studies of successful applications
12:00 pm Lunch Break
Elucidating the Mechanism of Action of Gene Therapies to More Easily Establish Product Potency
1:00 pm Demonstrating Mitochondrial Targeting in Gene Therapy: A Case Study on Potency Assay Development
Synopsis
- Aligning MoA studies with regulatory expectations to facilitate approval
- Documenting MoA for regulatory submissions to ensure compliance
- What can we learn from case studies of successful MoA-based approvals?
1:30 pm Expert Discussion: Understanding & Mimicking the Mechanism of Action In Vitro
Synopsis
- How can we develop in vitro models that accurately reflect in vivo conditions?
- What are the key biomarkers for MoA, and how can they enhance assay relevance?
- Discussing advanced imaging and analytical techniques for precise measurement
2:00 pm Afternoon Break & Poster Session
Phase-Appropriate Potency Assay Development by Tailoring Assays for Every Stage of Gene Therapy
3:00 pm Session Reserved for Program Partner
3:30 pm Implementing Equivalence Testing for an Early Phase Program
Synopsis
- An approach to setting phase-appropriate parallelism criteria
- Ensuring that the criteria is a meaningful reflection of a visual assessment
- Life cycle management of parallelism criteria set in early phase