8.00 Check-In & Coffee
ASSAY DEVELOPMENT
QC READINESS
WORKSHOP A
WORKSHOP B
9:00 am Strategic Assay Development: When to Invest, Implement, & Innovate Advanced Analytical Methods in Cell & Gene Therapy
Synopsis
- This workshop explores the integration of advanced analytical methods, such as NGS, ddPCR, and mass spectometry, into assay development for cell and gene therapies. Gain insights into balancing cost, regulatory requirements, and outsourcing to optimise analytical strategies for innovative therapies.
- Timing & Cost-Effectiveness: Identify when to integrate advanced assays like NGS, ddPCR, or Raman spectroscopy into assay development pipelines while balancing cost and practicality
- Regulatory Readiness: Discuss how regulatory requirements influence assay design and when to elevate exploratory methods like mass spectrometry, capillary electrophoresis, or flow cytometry to standard release criteria
- Access & Feasibility: Explore the role of CROs in assay development and how to navigate outsourcing challenges for advanced analytical methods
9:00 am Choosing Between qPCR vs. dPCR vs. ddPCR for Robust Analytics & Regulatory Compliance
Synopsis
In gene therapy development, accurate quantification of viral vectors is critical for assessing potency and ensuring therapeutic efficacy. This discussion will explore the strengths and limitations of qPCR, dPCR, and ddPCR, and how selecting the right method impacts early versus late-stage gene therapy processes.
- PCR Method Comparison Pros & Cons: Overview of qPCR, dPCR, and ddPCR for gene therapy, focusing on their use in viral vector quantification and potency testing
- Early vs. Late-Stage Assay Selection: Why early-stage programmes favour dPCR/ddPCR, while late-stage programmes often rely on qPCR due to established workflows
- Pitfalls of qPCR in Late-Stage Gene Therapy: Challenges of using qPCR for AAV titration and potency in late-stage programmes, and how dPCR/ddPCR can improve accuracy and regulatory compliance
12:00 Lunch Break
WORKSHOP C
WORKSHOP D
1:00 pm Navigating the Ongoing Challenges of Potency Assays in Gene Therapy for Regulatory Alignment & Enhanced Validation
Synopsis
The complexities surrounding potency testing, from early-stage assay workarounds to long-term validation in compliance with evolving regulatory requirements, continue to be a significant hurdle for the industry, and this workshop will provide insights into overcoming these challenges while aligning with best practices.
Clarifying Potency Assay Purpose & Misconceptions: Delve into the recurring misunderstandings about potency testing, and the true purpose of potency assays in ensuring lot-to-lot consistency
Early-Stage Assay Challenges & Workarounds: Examine how early-stage gene therapy programmes navigate the lack of fully validated potency assays by utilising a combination of functional and binding assays to maintain product release standards
Long-Term Potency Validation & Regulatory Alignment: Discuss the complexities of validating potency assays throughout the gene therapy lifecycle, balancing scientific consistency with regulatory requirements to ensure clinical efficacy is meaningfully linked to assay results
1:00 pm From Bench to Bedside: Mastering QC & CMC Alignment to Meet Evolving Regulatory Standards
Synopsis
Ensuring QC readiness and CMC alignment is critical to the successful development and regulatory approval of gene therapies. As regulatory standards evolve, especially with the complexities of viral vector production, maintaining consistency and meeting compliance requirements at each stage—from early development to clinical trials-is essential.
- Timing & Implementation of QC Assays: Identifying the optimal stages to introduce specific QC assays that align with development milestones and meet evolving regulatory requirements, ensuring both timely and compliant assessment
- Addressing Evolving Regulatory Standards & CMC Alignment: Discussing the integration of ICH Q14, FDA, and EMA guidance into the CMC strategy, with a focus on maintaining QC readiness throughout the development phases, particularly during late-stage development and clinical trials
- Ensuring Consistency & Robustness of Viral Vectors: Tackling challenges in viral vector production, ensuring batch-tobatch consistency, and developing fit-for-purpose analytical methods for potency assays, while meeting both global and European