Interim Biomarker and Bioanalytical data from Imagine-1: A Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability, and Efficacy of PBGM01 in Subjects GM1 Gangliosidosis. Gene and cell therapies are making headways in recent years for bringing new hope to many patients with severe diseases and have no cure. However, the development of such therapies has proved challenging, and require concerted efforts in regulatory approaches, clinical study design and technical solutions. This presentation will discuss biomarker and bioanalytical strategy and challenges supporting gene therapy. The interim clinical, biomarker and bioanalytical data from the Passage Bio ongoing gene therapy trial Imagine-1 for treating patients with GM1 gangliosidosis will be presented.
Learning Objectives:
Upon completion, participants will be able to list multiple examples of biomarker assays used in support of a lysosomal enzyme gene therapy program.
Upon completion, participants will be able to describe how selection of biomarkers and bioanalytical assays for a gene therapy program connected to clinical objectives.
Upon completion, participants will be able to list examples of challenges inherent to implementation of biomarkers and bioanalysis for pediatric rare disease gene therapy programs.
