Analytical Development 101 Day

Monday, November 29 2021 | 10.00AM - 3.00PM EST

Looking to understand basic principles and familiarize yourself with key terms and technologies? 

Look no further than the 101 Day at Gene Therapy Analytical Development where you will learn the important foundations to the analytical development field, preparing you and increasing your confidence in the discussion that lie in the deeper-dive days ahead!  

Chair:

Cecilia Della Valle

Cecilia Della Vale
Associate Director, Analytical Science & Technology (AS&T)
PTC Therapeutics

An Introduction to Gene Therapy

  • Basic biology of the vectors and the importance of analytics (AV, AAV & lentiviral) 
  • Exploring different vector serotypes  
  • What are the main challenges with analytics with respect to other biologics? 
  • Describing the high-level challenges of GTs compared to monoclonal antibodies and relevant challenges, such as potency and impurities 

A Case study of Common Analytical Hurdles

  • Introduction to how the analytics link to the process
  • Timing – how is the product release and shipment with the timeline
  • Getting analytics done and the product out the door in time
  • Interactions of gene therapy departments with regards to key assays
Liz Higgins

Elizabeth Higgins
Independent Consultant

What are the Assays?

An introduction to the individual assays used in gene therapy analytical development, discussing the advantages and limitations of each, and how they compare to protein analytics

Bioassays

  • An introduction to the bioassays required: what does each assay mean?
  • What technologies are being used for analysis?
  • In vivo vs in vitro bioassays
  • Highlighting standards and controls
Vesselin Mitaksov

Vess Mitaksov
Senior Principal Scientist
Pfizer

Molecular Biology

  • PCR-based applications for the characterization of aav gene therapy vectors  
  • Brief overview of qPCR vs. ddPCR  
  • Applying PCR-based methods to characterize AAV vectors 
Peter Clarner

Peter Clarner
Scientist, Gene Therapy Accelerator Unit
Biogen

Analytical Chemistry Techniques

  • An introduction to analytical chromatography techniques
  • Limitations and advantages to the techniques used for vector characterization