8:15 am Virtual Coffee & Networking

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

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

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

Synopsis

  • 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

Synopsis

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

10:00 am Morning Break & Virtual Networking Time

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

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

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

Synopsis

• Infectivity, expression, and functional potency are three major biological activities of viral vectors. Do we need all three for each programme?
• When and how can we use infectivity for in vitro potency assessment?
• Implementation of a novel ddPCR-based method for measuring infectivity
• Relative transduction versus infectious titer assays

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

Synopsis

• Process manufacturing and Quality Control of AAV vectors
• VG titers- qPCR vs ddPCR
• Evaluation of genome integrity with 2D ddPCR

12:30 pm Lunch & Networking

Navigating the Regulatory Space

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

Synopsis

  • 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 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

3:15 pm Afternoon Virtual Networking & Refreshments

Synopsis

Coffee Q&A Session with Louise Bisset, Senior Biopharmaceutical Quality Assessor, Medicines & Healthcare Products Regulatory Agency

INTRODUCING NEXT GENERATION SEQUENCING FOR CHARACTERISING IMPURITIES

4:15 pm 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

4:45 pm End of Virtual Summit