November 19-21 | Boston, MA, USA

Workshop A: 1 place remaining!

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Day Two

8:20 am Chair’s Opening Remarks

IMPROVING CURRENT ANALYTICAL METHODS TO GIVE MORE CONSISTENT & ACCURATE RESULTS

8:30 am Analytical Development Strategies for AAV Gene Therapy programs

  • Patrick Starremans Associate Director, Analytical Sciences & Head of Process Analytics, Voyager Therapeutics

Synopsis

  • Considerations for development of analytical assays
    – Assay throughput: In-process vs Release
    – To qualify or not to qualify?
    – In-house use vs outsourcing models
  • Development strategies for cell-based potency assays
    – Phase appropriate development
    – (case study)
  • Switching gears
    – Adapting to program and assay changes
    – (case study)

9:00 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

9:30 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

10:00 am Morning Refreshments & Networking

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

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

1:00 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

1:30 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

2:00 pm Afternoon Refreshments & Networking

IMPROVING CURRENT INFECTIVITY ASSAYS TO GIVE MORE CONSISTENT RESULTS

2:30 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

3:00 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

3:30 pm Chair’s Closing Remarks

3:45 pm Close of Conference