8:15 am Chair’s Opening Remarks

Detecting, Characterising & Avoiding Aggregates

8:30 am Characterising Aggregates: Exploring Methods for Transfer into QC

  • Franz Schnetzinger Director, Quality Control & Chemical Manufacturing Control Analytical Development, Gyroscope


  • Orthogonal methods for rAAV vector aggregate characterisation
  • Impact of sample matrix and material preparation on test result
  • Advantages and disadvantages of DLS, SEC-HPLC, nsTEM, and other methods
  • Finding suitable controls and transferring methods into QC

9:00 am Automated, Low-Volume Subvisible Particle Aggregate Analysis with the HORIZON System


  • Subvisible particle analysis is a key predictor of stability and safety and is an essential parenteral drug quality metric. Assessing vector particle aggregates in the subvisible range is especially challenging in AAV formulation development where limited precious sample is available
  • The HORIZON® system from Halo Labs employs Backgrounded Membrane Imaging (BMI) technology as a solution for detecting and quantifying subvisible vector particle aggregates in low volume, high throughput format
  • BMI is fully automated, fluidics-free, and uses only 25μL of sample

9:30 am Formulation & Manufacturing Process Optimization to Prevent Aggregation in Final Drug Product


  • Case study on one of UniQure’s drug products
  • Agglomeration in a manufacturing process
  • Process changes/optimisation to prevent agglomeration

10:00 am Morning Refreshments & Networking


11:00 am Validation of Safety Assays Required for Regulatory Submission


  • Qualification during product life cycle
  • Validation/ matrix qualification and system suitability
  • Compendial vs new NAT methods

11:30 am Using Infectivity Assays as a Surrogate for Potency Assessment of Viral Vectors

  • Marina Feschenko Director, Analytical Development- Gene Therapy/Biologics, Biogen

12:00 pm Applications of ddPCR for the Characterisation of AAV Vectors

12:30 pm Lunch & Networking

Navigating the Regulatory Space

2:00 pm Development & Validation of Analytical Methods for Gene Therapy Commercialization


  • Analytical method development and validation strategies
  • Meeting the challenges of short timelines and material availability within a GMP framework
  • Present relevant case studies

2:30 pm Regulatory Panel Discussion: Expected Regulatory Requirements for Analytics Depending on the Product’s Stage in Clinical Development

  • Joseph Lee Executive Director, Analytical Development, PTC Therapeutics
  • Jaap Twisk Senior Director, Analytical Development, UniQure
  • Louise Bisset Senior Biopharmaceutical Quality Assessor , Medicines & Healthcare Products Regulatory Agency
  • Greg Saia Senior Scientis, bluebird bio


Hear different regulatory experts share their opinions on the industry’s most important challenges, including:

  • Producing relevant potency assay data
  • The use of alternative infectivity assays
  • Setting reference standards
  • Novel techniques – validation and acceptance
  • Different world regions: do different agencies have different thresholds?
  • Commenting on the specific lentiviral vector regulatory challenges
  • When to engage regulators regarding the analytic plan

3:15 pm Afternoon Networking & Refreshments


4:15 pm Next Generation Sequencing

  • Eduard Ayuso Team Leader, Innovative Vectorology, Scientific Director Translational Vector Core (CPV), INSERM

4:45 pm Chair’s Closing Remarks