Analytical Development 101 Day

Monday, November 29 2021 | 10.00AM - 2.00PM 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!  

10.00 Next Generation Analytical Characterization of Gene Therapy Vectors

  • On top of the challenges GT companies are already facing in developing the analytical "tool kit" for gene therapy vectors, more companies will be facing new challenges in meeting Late Phase expectations for analytical characterization and release testing
Liz Higgins

Elizabeth Higgins
Vice President CMC
NysnoBio

 

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

10.30 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

11.00 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

11. 30 Lunch Break

12. 30 Round Table Discussion 

Cecilia Della Valle

Chair:
Cecilia Della Valle

Associate Director
PTC Therapeutics

Liz Higgins

Elizabeth Higgins
Vice President CMC
NysnoBio

 

Peter Clarner

Peter Clarner
Scientist, Gene Therapy Accelerator Unit
Biogen

Vesselin Mitaksov

Vess Mitaksov
Senior Principal Scientist
Pfizer