Tuesday 16th September - Main Conference Day OneTuesday, 16 September 2025 - ET (Eastern Time, GMT-05:00)

As gene therapy evolves beyond AAV-based delivery systems, new analytical challenges arise in ensuring genome integrity, optimizing therapeutic dosing, and mitigating immunogenicity risks. The industry is shifting toward alternative delivery methods such as CRISPR-based therapies, lipid nanoparticles (LNPs), plasmids, and synthetic vectors, requiring advanced analytical approaches to maintain product quality, efficacy, and regulatory compliance. This session will explore: Genome integrity analysis beyond traditional capsid characterization, leveraging long-read sequencing and multiplex digital PCR to assess payload stability and completeness, optimizing viral vectors for complete genomes, reducing dosing requirements, improving patient outcomes, and lowering production costs, second-dose strategies for non-AAV gene therapies, addressing durability, re-dosing challenges, and immunogenicity considerations, emerging analytical approaches for non-AAV delivery systems, including mRNA-based CRISPR therapies, LNP formulations, and synthetic gene vectors, key regulatory considerations for characterizing non-viral gene therapy platforms, ensuring alignment with evolving global standards.

Regulatory expectations for potency and release testing are evolving, particularly with the rise of advanced biologics such as CGTs, ADCs, and bispecific antibodies. Companies must navigate challenges in assay standardization, surrogate potency methods, and comparability assessments while ensuring compliance with global regulatory frameworks. This session, led by industry experts and regulatory specialists, will provide practical insights into meeting the latest standards, including ICH Q6B (Specifications for Biotechnological/Biological Products), USP General Chapter <1032> (Design and Development of Biological Assays), and FDA guidance on potency testing for gene therapy products. Attendees will gain actionable strategies for improving assay validation, reducing variability, and streamlining regulatory approval pathways.

Round Table Session 1: Tackling Variability in Cell-Based Bioassays: Strategies for Achieving Consistent and Reliable Results

Cell-based bioassays are crucial for potency testing, yet their inherent variability can lead to batch failures and regulatory delays. This roundtable explores best practices and innovative strategies for achieving consistent and reliable results. Standardization efforts are critical for improving accuracy and global harmonization of potency testing.

Key discussion points:

Optimizing assay conditions to enhance batch-to-batch reproducibility.-

• Data-driven approaches, utilizing statistical modelling and real-time monitoring, to minimize variability.
• Leveraging AI tools for unbiased cell analysis, automated gating in flow cytometry, and improved data insights
• Understanding and meeting regulatory expectations to ensure compliance and reduce rework.
• The impact of AI-driven automation for increasing throughput while maintaining assay integrity
• Case studies showcasing successful strategies for minimizing variability and improving assay performance.

Round Table Session 2: Accelerating Product Release – Overcoming Challenges in Real-Time Release Testing (RTRT)

Real-Time Release Testing (RTRT) is a transformative approach to batch release, offering faster turnaround times, cost reduction, and improved process control. However, many companies struggle with RTRT implementation, regulatory acceptance, and aligning process analytical technology (PAT) with manufacturing workflows Participants will gain practical insights into integrating RTRT into their workflows, helping to accelerate product release and enhance overall biomanufacturing efficiency.

Key Discussion Points:

• Leveraging PAT and advanced data analytics to enable RTRT in GMP environments
• Overcoming regulatory and technical barriers to RTRT adoption
• Case studies on successful RTRT implementation, demonstrating how companies have improved efficiency and compliance.