This in-person workshop focuses on the critical role of biomarkers in drug development for regulatory approval, particularly for rare diseases, with industry experts from biotech/pharma companies to share case studies, insights, and strategies. It aims to enhance understanding of biomarker strategies, validation, and their pivotal role in therapy development.

Topics include the biomarker framework, small molecule biomarkers in clinical studies, minimal residual disease monitoring in CAR-T cell therapies, phenylalanine as a biomarker for PKU gene therapy, neurodegenerative disease biomarkers, and analytical challenges.