08:00 Check-In & Coffee
8:50 am Chair’s Opening Remarks
9:00 am Industry Leader’s Fireside Chat: Navigating the Current & Future Landscape of Gene Therapy Analytical Development with Industry Veterans
Synopsis
- Delve into a discussion on the latest developments in gene therapy analytical methods, focusing on the unique challenges and regulatory frameworks specific to the European market
- Examine how analytical strategies in Europe are evolving to balance scientific rigour with regulatory alignment, ensuring assays meet both local and international standards
- Gain insights into new technologies transforming gene therapy analytics, with a particular focus on navigating the European regulatory environment and preparing for future advancements in manufacturing and clinical trials
OPTIMISING ASSAY DEVELOPMENT FOR CONSISTENCY & ROBUSTNESS ACROSS GENE THERAPY FORMULATIONS
9:50 am Tackling Key Considerations of Platform Assays & Methods According to Recent Guidance
Synopsis
- Understand how platform assays ensure standardised methods for reliable and reproducible results across various formulations and batches
- Learn how minor formulation tweaks may stay within the same platform, but significant changes could require validation and regulatory re-submission
- Discuss how modifications to critical quality attributes or biological outcomes often trigger the need for new validation and full regulatory submission
10:20 Morning Break & Speed Networking
Synopsis
As this community re-unites for the 6th time, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections.
11:20 am Roundtable Discussion: Understanding Essential Assays for Gene Therapy Drug Approval – What You Need & Why
Synopsis
In this roundtable, you will gain practical insights into developing and implementing critical assays like potency, identity, and purity for gene therapy drug products, ensuring quality and safety. You will also learn how to align assay development with evolving regulatory requirements, enhancing product consistency and ensuring compliance throughout the product lifecycle.
- Understanding Key Assays for Gene Therapy: Explore the essential assays needed for the development of gene therapy drug products (DP), including potency, identity, and purity assays, and why each is critical for ensuring product quality and safety
- Assay Development & Implementation: Dive into the best practices for developing and implementing these assays, from early-phase exploratory work to late-stage, commercial-ready methods
- Regulatory Alignment & Strategy: Discuss how to align assay development with evolving regulatory requirements, ensuring assays meet both scientific and compliance standards throughout the product lifecycle
12:00 pm Translating Viral Vector Vaccine Success to Gene Therapy Advancements
Synopsis
- Leveraging J&J’s decade of experience in optimising viral vector formulations, improving stability and enhancing the efficacy of gene therapy products
- Adapting novel analytical techniques from viral vector vaccine work to develop robust, scalable methods tailored to gene therapy needs
- Using predictive modelling and cutting-edge strategies from vaccine development to mitigate risks and ensure product consistency in gene therapy
12:30 pm Navigating the Challenges of VG Titer Assays in Gene Therapy: Accuracy, Sensitivity, & Standardisation
Synopsis
Discuss the challenges of distinguishing between full and empty viral particles in VG titer assays and the impact this has on the reliability of potency assessments for gene therapies
- Explore how the sensitivity and specificity of VG titer assays can be affected by sample quality, contamination, assay method, and strategies to mitigate these issues for more accurate titer measurements
- Address the lack of universal standardisation across VG titer assay platforms and the importance of assay validation to meet regulatory requirements, ensuring reproducibility and consistency across manufacturing sites
1:00 pm 1:00 Lunch Break & Networking
2:00 pm Tech Focused Discussions: Building Your Next-Gen Gene Therapy Analytical Toolbox for Consistency & Enhanced Testing Capabilities
Synopsis
You will be able to choose a table based on the analytical technology that you are most interested in. You’ll have 30 minutes to engage with the experts in focused discussions on the benefits, challenges and real-world applications of advanced analytical techniques.
- Exploring next-gen tools like NGS, mass spectrometry, cryoTEM, HPLC, Bio-layer interferometry (BLI), and flow cytometry for enhanced potency testing, vector characterisation, and stability analysis; and their pivotal role in gene therapy advancements
- How tools like LC-MS, qPCR, and bioassays can help address scaling issues during the transition from small-scale research to large-scale commercial manufacturing, ensuring batch-to-batch consistency and regulatory compliance
- Practical insights on transferring analytical methods, such as ICP-MS, elisa-based assays, and protein aggregation assays, from R&D to manufacturing, ensuring smooth tech transfer without compromising assay performance, data integrity, or regulatory alignment
OPTIMISING BIOPHYSICAL CHARACTERISATION & STABILITY TESTING TO ENHANCE QUALITY
2:30 pm Addressing Challenges in the Characterisation & Stability of AAV Vectors – A Biophysical Perspective
Synopsis
- Examine the essential biophysical methods for evaluating AAV vectors in early clinical stages, focusing on determining full and empty particle ratios and selecting the appropriate techniques based on sample quality and development phase
- Discuss insights into choosing the right biophysical techniques based on specific sample conditions, highlighting the importance of monitoring
- Elaborate on regulatory aspects and how to implement new ICH Q2(R2) and Q14 guidelines
3:00 Afternoon Break & Poster session
Synopsis
This is your opportunity to contribute to the conversation and share your cutting-edge
research with this community, while discovering exciting work carried out by your peers.
4:00 pm Unmasking the Hidden Threat of Viral Vector Process Contaminants with Improved Characterisation Techniques
Synopsis
- Address contaminants introduced by vector production which may affect the purity and safety of the final product
- Uncover how tools like mass spectrometry, ELISA, and qPCR are essential for accurately detecting and characterising these contaminants at various stages of production
- Characterisation supports regulatory requirements and helps implement control strategies to minimise contamination risks, ensuring the consistency and safety of gene therapies
4:30 pm Confronting the Challenges of Viral Vector Genome Integrity for Consistent Gene Therapy Outcomes
Synopsis
- Understanding accurate detection of viral vector genome integrity is crucial to ensure the vector carries the correct genetic material without mutations, deletions, or rearrangements that could impact therapeutic efficacy or safety
- Address how variability in the efficiency of viral packaging and transduction processes can lead to inconsistencies in genome integrity, requiring robust methods to quantify and control this variation across different production lots and stages
- Ensuring viral vector genome integrity involves complying with stringent regulatory standards, necessitating the development of validated, reproducible assays to guarantee that the gene therapy product consistently meets quality and safety requirements throughout its lifecycle