08:00 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 Scaling Gene Therapy Production & Overcoming Challenges in Quality, Yield, & Cost-Effectiveness
Synopsis
- Maintaining Product Quality at Scale: Strategies for ensuring consistent product quality, controlling variability, and validating analytical methods during largescale production
- Achieving Consistent Yields: Approaches to optimise production processes, improve reproducibility, and minimise batch-to-batch variation in viral vector production
- Cost-Effective Manufacturing Solutions: Identifying scalable and cost-efficient production methods, including process optimisation, automation, and alternative production platforms
10:20 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 Harmonising Standards for Safety & Efficacy Testing in Gene Therapies: Collaborative Approaches to CQAs
Synopsis
- Development of International Standards: Exploring MHRA’s collaboration with WHO to create and implement global benchmarks for gene therapy safety and efficacy
- Advancing Harmonisation: Methodologies for harmonising approaches to control testing and critical quality attribute (CQA) measurement
- Innovations in R&D: Insights from MHRA activities focused on safety and efficacy research to support standardised gene therapy testing
12:20 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 Panel Discussion: 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
2:15 Afternoon Break & Networking
3:15 pm OPTIMISING MANUFACTURING EFFICIENCIES WITH INNOVATIONS IN AUTOMATION & REAL-TIME PROCESS MONITORING
3:20 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
3:50 pm Enhancing Manufacturing with Process Analytical Technologies (PAT) in Real-Time Product Yield Monitoring
Synopsis
- Real-Time Process Monitoring: PAT tools like NIR spectroscopy, Raman spectroscopy, and mass spectrometry allow for continuous, real-time monitoring of key process parameters, offering immediate insights into product yield and composition, reducing the need for time-consuming batch testing
- Accelerated Decision-Making: The integration of these technologies empowers manufacturers to make data-driven decisions quickly, minimising delays and enabling more agile production workflows that respond to fluctuations in process variables
- Optimised Process Efficiency: By facilitating early detection of deviations and providing more accurate yield predictions, PAT helps in streamlining the manufacturing process, improving