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 The other half of success: What if the real barrier to ATMP success isn’t clinical — but analytical?
Synopsis
- What if one of the main causes of delays in ATMP development wasn’t clinical… but analytical?
- Why can working with a CRO make the difference in ATMPs development?
- What services can a CRO specialized in ATMPs offer you?
11:45 am Advancing potency assays through quantitative TEM: Accurate analysis of AAV vector content
Synopsis
- Transmission Electron Microscopy (TEM) as a comprehensive tool for evaluating many critical quality attributes (CQAs) with a single assay for gene therapy vectors like AAVs, adenovirus, LVVs, and more.
- Combined with QuTEM’s image analysis methodology, TEM enables quantitative assessment of AAV vector content, including full, empty, partially filled, and overfilled capsids, as well as clusters/aggregates in one run.
- QuTEM’s GMP TEM services support vector development and strengthens CQA assessments, ensuring vector quality throughout manufacturing and release
12:00 pm Considerations for Potency Assay Development for Gene Editing Products
Synopsis
- Overview of matrix approach for potency assay development
- Review of available regulatory guidance and expectations for phased-based approach to potency assay development
- Tools and considerations when measuring biological cascade of RNA & LNP delivery in-vitro
- Challenges associated with potency assay development for gene editing products
12:30 pm Lunch Break
Elucidating the Mechanism of Action of Gene Therapies to More Easily Establish Product Potency
1:30 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?
2:00 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
2:30 pm Afternoon Break & Poster Session
Phase-Appropriate Potency Assay Development by Tailoring Assays for Every Stage of Gene Therapy
3:30 pm Don’t Be Late: Functional Potency Assays as the FDA’s New Gatekeeper
Synopsis
- Functional Potency Assays Are No Longer Optional—They’re Expected
- Curi Bio enables longitudinal functional + analytical readouts
- Validation of a human 3D engineered neuromuscular junction for potency assessment
3:40 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
4:10 pm Chair’s Closing Remarks
4:15 pm Drinks Reception
Synopsis
Wrap up the day with a relaxed networking session—reconnect with familiar faces and spark new conversations with fellow analytical and regulatory experts driving gene therapy forward.