8:00 am Online Login

8:30 am Characterisation of AAV Stocks by High-throughput Sequencing

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

Synopsis

• Utilising next generation sequencing to characterise residual DNA
• Exploring genome identity and integrity
• Discussing sequencing biases

8:50 am Accurate & Sensitive Identification of Nucleic Acid Impurities in AAV Vector Samples by High-Throughput Sequencing

  • David Dobnik Scientific Associate, Head of Laboratory for Therapeutic Viruses, National Institute of Biology

Synopsis

  • Using HTS to identify all nucleic acid impurities present in AAV vectors
  • Highlighting differences between short and long read platforms
  • Case study on AAV vectors, focusing on encapsidated impurities
  • Discussing the limitations of this method and its regulatory implications

9:10 am AAV Titration ELISAs: a AAV ELISAs: a Robust & Reliable Tool for Capsid Quantification & Reliable Tool for AAV Quantification

Synopsis

• Comparison between PROGEN’s ELISA and other analytical methods
• Technical parameters of PROGEN´s Titration ELISA
• Procedure of ELISA calibration

9:40 am Process & Product Impurities

Synopsis

Talk details to be confirmed.

10:00 am Live Q&A & Panel Discussion

  • David Dobnik Scientific Associate, Head of Laboratory for Therapeutic Viruses, National Institute of Biology
  • Eduard Ayuso Team Leader, Innovative Vectorology, Scientific Director Translational Vector Core (CPV), INSERM
  • Sushmita ‘Mimi’ Roy Executive Director, Analytical Sciences, BioMarin
  • Katharina Hammer Director, Quality Control, PROGEN

10:30 am Morning Break & Discover Booths

ASSAY VALIDATION IN LINE WITH CURRENT REGULATORY GUIDELINES

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

Synopsis

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

11:20 am 5 Minute Break

11:25 am 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 Scientist, bluebird bio

Synopsis

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

12:00 pm Lunch & Networking

Detecting, Characterising & Avoiding Aggregates

12:30 pm Characterising Aggregates: Exploring Methods for Transfer into QC

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

Synopsis

  • 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

12:50 pm Protein or Not? Advanced High Throughput Aggregate Analysis with the Aura

Synopsis

In protein-based formulations, distinguishing aggregated API from other particle types is important for understanding the root cause of instability. Until now, existing methods have been either unreliable or too cumbersome and difficult to use in many workflows

Here we introduce the Aura, a 96-well low-volume aggregate and particle imaging system that can rapidly size, count, and characterize particles and identify them as proteins, non-proteins, hydrophobic, or other types of molecules.

1:20 pm Formulation & Manufacturing Process Optimization to Prevent Aggregation in Final Drug Product

Synopsis

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

1:40 pm Panel Q&A & Panel Discussion

2:00 pm Coffee Q&A Session

  • Louise Bisset Senior Biopharmaceutical Quality Assessor , Medicines & Healthcare Products Regulatory Agency

3:00 pm Final Speed Networking Session

Synopsis

Don’t forget to refresh your browser and make the most of your final opportunity to meet your colleagues!

4:00 pm Time for 1-2-1 Private Calls & Explore the Exhibition Booths

4:30 pm End of Summit