BioAnalytical Scientist Sangamo Therapeutics Richmond, California
The increasing prevalence of in vivo gene therapy with recombinant adeno-associated virus brings many new bioanalytical challenges. For lysosomal storage diseases, gene therapy offers a next generation treatment beyond traditional enzyme replacement therapy (ERT). Due to long term treatment with ERT, many patients exhibit anti-ERT antibodies which are pre-existing antibodies for gene therapy. To detect treatment-boosted antibodies to transgene product, drug tolerance is a big bioanalytical hurdle because the continuous expression of transgene protein from gene therapy treatment making it impossible to collect washout samples. This presentation will discuss novel assay optimization to improve assay sensitivity and drug tolerance to appropriately assess treatment-boosted neutralizing anti-transgene protein antibodies in supporting clinical trials.
Learning Objectives:
Understand current gene therapy approaches to treating lysosomal storage diseases (LSDs)
Recognize difficulties in detecting pre-existing anti-lysosomal enzyme antibodies after treatment with gene therapy
Learn new approaches to increase the ability to appropriately detect and monitor anti-transgene antibodies
