8:30 am Check-In & Coffee
9:25 am Chair’s Opening Remarks
9:30 am Panel: Cracking the Code on Potency: Defining, Measuring, & Validating Functional Activity in Gene Therapy Products
Synopsis
- What Does Potency Really Mean in Gene Therapy?: Addressing the challenge of linking in vitro assay results to in vivo clinical efficacy for diverse modalities (AAV, LV, gene editing, mRNA)
- Assay Design Under Regulatory Scrutiny: Navigating FDA/EMA expectations for potency assays as a critical element of IND, BLA, and RMAT submissions
- Toward Meaningful Metrics: Strategies to move beyond surrogate markers and design potency assays that reflect true biological function and therapeutic relevance
10:30 am Session Reserved for NanoMosaic
11:00 am Morning Break & Networking
Bioassays & Molecular Biology
Enhancing Analytical Methods for Gene Therapy Product Release & Compliance
12:00 pm Characterizing Novel CRISPR Editors for Research & Therapeutics
Synopsis
- Developing and evaluating assays for protein engineering and therapeutic development
- Assays for novel and engineered editors to establish data sets
- Editor characterization and data sets for regulatory filings, clinical trials, and long-term follow-up studies
12:30 pm Advancing Residual DNA Characterization with A Multiplexed Digital PCR-Based Approach to Size Profiling
Synopsis
- Development of a dPCR assay for precise sizing of residual DNA in gene therapy products
- Analytical strategies to distinguish and quantify short vs long DNA fragments for improved quality assessments
- How expanded size characterization can support regulatory guidance and CMC filings beyond current standards.
1:00 pm Lunch Break & Networking
2:00 pm Standardizing the Future: USP Standards for Advancing Analytical Quality for Gene Therapy
Synopsis
- USP’s new AAV reference standards and analytical reference material
- Review of collaborative study data using USP AAV standards
- USP’s documentary standards supporting gene therapy
- Future USP gene therapy reference standards
2:30 pm Addressing the Challenge of ReplicationCompetent Virus Analytics in Gene Therapy
Synopsis
- Understanding Replication-Competent Viruses: Exploring the potential risks and safety concerns associated with replicationcompetent viruses in gene therapy applications
- Analytical Methods for Detecting RCVs: Overview of bioanalytical techniques, including PCR-based methods, qPCR, and cell-based assays for identifying replicationcompetent viruses in viral vector preparations
- Regulatory Considerations and Guidelines: Discussing the FDA’s and other regulatory agencies’ guidelines for evaluating and mitigating risks associated with replication-competent viruses, and ensuring patient safety in gene therapy products
Physicochemical Properties
Innovation, Risk, & Readiness in Physicochemical Analysis
12:00 pm Round-table: From R&D to QC: Validating Novel Physicochemical Methods
Synopsis
- Translating Innovation into QC: Strategies for adapting cuttingedge R&D methods for routine, validated QC use
- Regulatory Considerations: Expectations for method transfer, validation, and documentation to meet compliance standards
- Data Pipelines and Bioinformatics: Building efficient workflows for data analysis, integration, and regulatory support
12:30 pm Decoding LNPs: Advanced Analytical Strategies for Precise Characterization
Synopsis
- Establishing critical quality attributes (CQAs): Defining key parameters like size, encapsulation efficiency, and polydispersity.
- Innovative methods in LNP analysis
- Regulatory readiness: Ensuring analytical robustness and reproducibility to support IND and commercial submissions.
2:00 pm Stability Science for Reliable Shelf Life & Commercial Success: The Mystery of AAV Instability – Bridging the Scientific Gaps in Vector Degradation
Synopsis
- Unknowns that Impact Decisions: Current processes and storage strategies are built on incomplete data. Without a clear understanding of how AAV degrades, teams are flying blind when it comes to long-term stability and risk assessment
- Degradants–The Invisible Variables: What actually happens to AAV particles under stress? Without characterization of degradation products, it’s difficult to predict or explain batch variability, affecting product consistency and compliance
- The Downstream Implications: These knowledge gaps complicate everything from formulation and fill-finish decisions to regulatory submissions, where expectations around stability and shelf-life justification are rising
2:30 pm Decoding AAV Degradation: Building a Framework for Stability-Indicating Assays
Synopsis
- Explore degradation pathways under different storage conditions (frozen, refrigerated, room temperature)
- Investigate how titer loss correlates with capsid integrity and genome degradation
- Define criteria for selecting effective stability-indicating assays and extending retest dates
3:00 pm Afternoon Break & Networking
4:00 pm Harmonizing Global Regulatory Expectations for CMC in Gene Therapy: Challenges & Solutions
Synopsis
- Navigating Global Regulatory Frameworks: Exploring the differences in CMC (Chemistry, Manufacturing, and Controls) requirements across regions and the need for global alignment in gene therapy
- Efforts to Harmonize Standards: Discussing ongoing initiatives and collaborations aimed at standardizing CMC expectations to reduce inconsistencies and streamline approvals worldwide
- Overcoming Regulatory Barriers: Identifying key challenges in aligning regulatory expectations and strategies for overcoming them to ensure smoother global product development and market access
4:30 pm Navigating RMAT Designation: Aligning Analytical Development with Accelerated Regulatory Pathways
Synopsis
- Strategic Planning for Analytical Development with RMATs: Key data and validation approaches that supported the application
- Lessons from FDA Interactions: Insights into regulatory expectations and how early alignment helped shape development
- Impact on Program Strategy: How the RMAT designation influenced timelines, priorities, and cross-functional collaboration