Skip to content

8:20 am Chair’s Opening Remarks

  • Joseph Lee Senior Director, Analytical Development, Gene Therapies, PTC Therapeutics

IMPROVING CURRENT ANALYTICAL METHODS TO GIVE MORE CONSISTENT & ACCURATE RESULTS

Synopsis

Session Chaired By:

Linda Engle, Principal Scientist, Technical Development/Associate Director, CMC Gene Therapy Portfolio, Biogen

8:30 am Overview of Challenges in rAAV Custom Assay Design

  • Harald Messer Scientist l, Manager, Analytical Development, AGTC

Synopsis

  • Understanding approaches such as rational design for improved risk assessment on key project systems in ADA
  • Recognizing sources of inherent variability in potency assay development for cell & gene therapy products
  • Assimilating exploratory and bio-pharmaceutics development in potency assay work with complex MOA

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 30μL of sample

9:15 am Sharing the Various Molecular Testing Components Required to Support Product Characterization

  • Joseph Lee Senior Director, Analytical Development, Gene Therapies, PTC Therapeutics

Synopsis

  • Investigating the components required to support analytical development
  • Beyond ID and titer – understanding the characteristics that need to be
    defined to work towards product safety
  • Discussing the factors influencing decisions on in-house and external
    development

9:45 am How TEM Based Image Analysis Can Reveal Process Impact on Critical Quality Attributes

Synopsis

Innovative Transmission electron microscopy (TEM) – based image analysis
combines the benefit of quantitative data and visual evidence. This powerful
technology is made accessible through automated analyses simplifying the
work process, producing powerful data faster that supports decision making
in viral vector based drug development and manufacturing.

10:15 am Morning Refreshments & Networking

ENSURING ACCURACY IN VECTOR GENOME TITERING TO ESTABLISH CORRECT & REPRODUCIBLE DOSING

Synopsis

Session Chaired By:

Svetlana Bergelson, Director, Technical Development, Biogen

11:00 am Contrasting qPCR and ddPCR Methods for Vector Titering

  • Vivian Choi Head of Global Gene Therapy Research, Takeda

Synopsis

  • What’s the best method to use? Investigating the advantages and disadvantages of qPCR and ddPCR at various stages of development
  • How to use titer methods to support the process as well as releasing the drug
  • Understanding how to successfully transition from early stage qPCR to ddPCR for commercial material: how to address differences through comparability studies and meet regulatory expectations

11:30 am PCR-based Approaches for Determining AAV Genome Titer in Support of Gene Therapy Development

  • Pete Clarner Senior Associate Scientist, Gene Therapy Accelerator Unit, Biogen

Synopsis

  • The criticality of genome titer assays at all different stages of development
  • The advantages and disadvantages of ddPCR and qPCR for gene therapy vector titering and development support
  • Strategies for method crossover and when to apply a platform

12:00 pm Lunch & Networking

BEYOND AAV: INVESTIGATING ANALYTICAL STRATEGIES TO EVALUATE PLASMIDS & LENTIVIRAL VECTORS

1:15 pm Plasmid DNA Control Strategy Drug Substance-Product vs Critical Raw Material

Synopsis

  • Specifications, Comparability, Potency assays, Stability, etc.
  • Method & Process Validation
  • Safety Risk vs Business Risk
  • Current Regulatory Guidance

1:45 pm Panel Discussion: Contrasting Analytical Requirements for AAV and Lentiviral Vectors

Synopsis

  • Discussing the relative advantages and disadvantages of lentiviral and AAV vector approaches in a variety of indications
  • Understanding how analytical strategies differ in the context of different viral vectors
  • Case studies detailing similarities and differences in the analytical approaches required for the analysis of different vectors

2:15 pm Afternoon Refreshments & Networking

IMPROVING CURRENT INFECTIVITY ASSAYS TO GIVE MORE CONSISTENT RESULTS

Synopsis

Session Chaired By:

Svetlana Bergelson, Director, Technical Development, Biogen

2:45 pm A Novel Approach to Measuring AAV Infectivity With ddPCR Technology

Synopsis

  • Design, development and qualification of a new AAV infectivity assay by ddPCR
  • Head-to-head comparison with the existing TCID50 method
  • Discussing pros and cons of the new infectivity assay

3:15 pm Relative Infectivity as a Reliable Alternative to the TCID50 Assay

  • Win Den Cheung Associate Director, Analytical Development, REGENXBIO

Synopsis

  • Understanding the limitations of the TCID50 infectious titer assay
  • Advantages of the relative infectivity method over the TCID50 assay
  • Utility of the relative infectivity method in a variety of applications to support early development

3:45 pm Chair’s Closing Remarks

  • Joseph Lee Senior Director, Analytical Development, Gene Therapies, PTC Therapeutics