Senior Scientist Sangamo Therapeutics Brisbane, California
AAV-based gene therapy provides a viable therapeutic approach for treatment of lysosomal storage disorders (LSDs) with enzyme deficiency. From drug development perspective, characterization of gene therapy drug pharmacokinetics takes on distinct definition and require a complex and integrated bioanalytical strategy. This presentation focuses on assay considerations for quantitating transgene protein expression as a PD readout in AAV-mediated gene therapy. Two LSD enzyme activity assays will be utilized as study cases to discuss challenges and solutions for measuring transgene enzyme activity in plasma and tissue matrices.
Learning Objectives:
Unique challenges for measuring transgene protein (enzyme) in the context of preclinical and clinical studies
Transgene enzyme activity assay development and optimization
Practical solutions for quantitating transgene enzyme activity
