08:00am Check-In & Coffee
8:55 am Chair’s Opening Remarks
9:00 am Presentation & Roundtable Discussion: A Review of Gene Therapy Release Assays & Their Utility
Synopsis
- Are all assays used for release of AAV products telling us confirming a product quality attribute?
- Are the fundamentals for these methods sound?
- Are we interpreting the data from these assays correctly?
- Is it too late to do something about this now?
SCALABILITY & COMMERCIALISATION CONSIDERATIONS IN ANALYTICAL DEVELOPMENT FOR GENE THERAPY PRODUCTS
9:50 am Considerations for analytical methods used for DSP characterisation
Synopsis
- Establishing Robust and Reproducible Analytical Methods: Ensuring accuracy and consistency in characterizing downstream processing (DSP) steps to meet regulatory and quality requirements.
- Key Analytical Techniques for Purity, Potency, and Safety Assessment: Evaluating critical quality attributes (CQAs) of gene therapy products, including residual impurities, vector integrity, and biological activity.
- Overcoming Challenges in Method Development and Validation: Addressing sensitivity, scalability, and regulatory expectations to optimize DSP characterization for clinical and commercial applications.
10:20am Morning Break & Networking
11:20 am Roundtable Discussion: Overcoming Tech Transfer Hurdles to Maintain Product Quality, Consistency & Regulatory Compliance
Synopsis
- Discuss best practices for transferring manufacturing processes between teams or sites to ensure consistency, quality, and regulatory compliance without delaying timelines
- Explore the key challenges of scaling up gene therapy production and strategies for minimising bottlenecks to maintain efficient timelines
- Highlight the importance of collaboration across R&D, manufacturing, and quality control teams to ensure smooth tech transfer and timely product development
REGULATORY COMPLIANCE & CMC ALIGNMENT FOR STREAMLINED APPROVAL
11:50 am Research & Development Into Gene Therapy Standards
Synopsis
- The structural and functional complexity of CGT, along with its long-term persistence and efficacy, necessitates additional risk considerations.
- Assessing CGT critical quality attributes (CQAs) requires the characterization of multiple parameters.
- A unified and standardized approach to ATMP CQA assessment is essential for harmonization across the field.
12:20pm Lunch Break & Networking
1:20 pm Navigating Late-Stage Changes in Analytical Development to Ensure Regulatory Compliance & Mitigate Risks
Synopsis
- Explore the technical and regulatory hurdles faced when making changes to gene therapy products at late development stages, including changes to the viral vector, formulation, or assay systems, and how these can affect product consistency and regulatory compliance
- Discuss strategies for assessing and mitigating risks when making late-stage changes, particularly regarding how those changes might impact the quality, potency, or stability of the gene therapy product
- Review the regulatory landscape surrounding late-stage modifications, including the necessity of re-submissions, updates to the manufacturing process, or additional stability data to align with agency requirements
1:40 pm Aging Like Fine Champagne: Ensuring Consistent Quality with Reference Standards
Synopsis
- Just as a fine champagne maker relies on multiple vintages to achieve consistent quality, manufacturers must employ both primary and working reference standards to ensure product consistency throughout its lifecycle
- Highlight the working reference standard’s role in bridging gaps when the primary standard no longer reflects the product’s original characteristics, ensuring uninterrupted quality assurance
- Explore how planned transitions from primary to working reference standards, supported by stability testing, maintain long-term product quality and compliance, much like ensuring consistent champagne flavour over decades
2:10 pm Fireside Chat: Overcoming Challenges in Validation & Clinical Relevance of Potency Assays to Demonstrate Efficacy
Synopsis
- Potency Assay Validation Challenges: Validating potency assays, especially cell-based ones, is difficult due to their product-specific nature and the need to establish reliable biomarkers or functional outputs to demonstrate efficacy
- Unique Assays for Unique Products: Potency assays are often unique to each gene therapy product, requiring adaptation of academic or preliminary assays for use in clinical development while ensuring reproducibility and reliability
- Linking Potency to Clinical Response: The critical challenge lies in correlating in vitro potency measurements with actual clinical outcomes, ensuring that the assay’s predictive power aligns with therapeutic efficacy and safety in patients
2:50pm Afternoon Break & Networking
OPTIMISING MANUFACTURING EFFICIENCIES WITH INNOVATIONS IN AUTOMATION & REAL-TIME PROCESS MONITORING
3:20 pm Addressing challenges in conjugated AAV quality attributes
Synopsis
- Aligater Platform technology on a new generation of bio-conjugated vectors
- Developing an analytical toolbox and applying orthogonal methods to characterize bio-conjugated vectors.
3:50 pm Implementing Automation from Cell Culture to Assay Execution for Enhanced Efficiency
Synopsis
- Explore emerging trends in automation for gene therapy, such as fully automated PCR systems and novel applications in cell culture automation that go beyond traditional liquid handling or plate setup
- Discuss the potential of automated systems for handling large-scale cell culture processes, including laying down plates and other complex tasks that are traditionally labour-intensive
- Highlight advancements in automating assay workflows, focusing on novel techniques that improve throughput, consistency, and efficiency in gene therapy development