Challenges of developing ADA and concentration (PK or PD) assays for lysosomal transgene enzymes on an automated immunoassay platform

Gene therapy can be used to permanently correct genetic disorders by delivering a functional copy of the gene into the nucleus of affected cells or alternative cells, which then release the functional protein into systemic circulation.

One of the biggest challenges faced by the bioanalytical sector is the lack of a true reference material. As a result, in most assays, an alternative commercially available therapeutic (for example, an enzyme replacement therapy (ERT) product) can be used as a surrogate for the transgene enzyme. This webinar discusses approaches taken in order to monitor the concentration of the expressed transgene product and any associated immunogenicity.

If you have problems viewing the webinar, please contact maritha.lundin@gyrosproteintech.com