Workshop Day

Workshop A: 9am – 12pm

Innovations in Partial Capsid Characterisation: From Tools & Techniques to Regulatory Perspectives

  • David Dobnik Senior Research Associate, National Institute of Biology
  • Ishtiaq khaliq Lead Gene Therapy Analytics Molecular Biology Methods, UCB

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

  • Marco Thomann Principal Scientist - Gene Therapy & Technical Development Analytics, Roche

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