Workshop Day
Workshop A: 9am – 12pm
Innovations in Partial Capsid Characterisation: From Tools & Techniques to Regulatory Perspectives
Synopsis
Characterising partial capsids in gene therapy is becoming increasingly crucial for optimising product quality and overcoming regulatory concerns. Understanding exact capsid content aids in fine-tuning process development, ensuring efficient gene delivery, minimising immunogenicity, and altogether advancing safer, more effective gene therapies.
This workshop will set out to include:
- Comparing traditional and novel techniques for partial capsid extended characterisation
- Discussing methods for quantifying the severity of partial capsids and streamlining the response to analytic readouts • Characterising batch-batch partial capsid comparability
- Determining methodologies for appropriate reference standard production and discussing mechanisms to enhance lab to lab reproducibility
- Developing robust and universal safety thresholds for partial capsid percentages
- Understanding regulatory perspectives on partial capsid characterisations
12:00 pm Lunch Break
Workshop B: 1pm-4pm
Advancing CQA Assessments for Gene Therapy
Synopsis
Ensuring that novel CQAs (Critical Quality Attributes) for gene therapies are addressed is pivotal in ensuring the safety, efficacy, and consistency of emerging products. This workshop will take a look at some of the less conventional CQAs and assess their relative importance when planning an early analytical strategy.
This workshop will set out to include:
- An introduction to novel characterisation data on AAV mutation studies
- Analysis of real-world case studies highlighting successful applications of advanced CQA assessments
- Discussion of challenges faced during CQA assessments and strategies to overcome them
- Understanding regulatory requirements and compliance considerations for CQA assessments in gene therapy
- Brainstorming alternative analytical approaches to CQA assessment for collaborative problem-solving