Gene therapy holds promise for treating a wide range of diseases by precisely altering the genetic composition of cells. In order for genetic drugs to be clinically successful, sophisticated delivery systems are required. At present adeno-associated virus (AAV) and lipid nanoparticle (LNP) represent the lead viral and non-viral vectors for gene therapy drugs. However, analytical characterization of AAVs and LNPs has been challenging due to their complex composition and structure.

Many C&GT analytical development resources are focused on leveraging established techniques that are already mainstays in the biopharmaceutical industry, including charge heterogeneity and purity analyses. Maurice combines the industry gold standard icIEF technology with CE-SDS capabilities to address these critical quality attributes.

In this webinar, our panelists will discuss the application of Maurice for the characterization of AAVs and LNPS using either cIEF or CE-SDS detection schemes. You will learn how Maurice can be used to measure surface charge of an LNP-based mRNA vaccine- for confirmation of LNP identity in a manufacturing setting, and for stability assessment for process and formulation development. You will also learn how to assess purity, stability and concentration of AAVs.

Speakers:

John Loughney

John Loughney
Director, Vaccine Analytical Research & Development (VAR&D)
Merck & Co., Inc.




Chris Heger

Chris Heger
Senior Manager, Applications Science
ProteinSimple, a Bio-Techne brand




Presentations:

  • John Loughney: Development of an imaged capillary isoelectric focusing method for characterizing the surface charge of mRNA lipid nanoparticle vaccines.
  • Chris Heger: Characterization of Gene Therapy tools, including AAVs, using Maurice