Cell & Gene Therapy Optimization, a Framework: Assessing efficacy and safety with dynamic biomarkers

Cell- and gene-based therapies hold enormous potential for biomedical transformation, enabling new treatment modalities for traditionally difficult-to-treat diseases. Unlocking this potential, however, has proven challenging due to heterogeneity in necessary target engagement and drug response across patients, as well as from potential adverse events related to delivery modalities. Small molecule biomarkers, which read out the dynamic changes that occur after drug exposure, are a non-invasive means to better assess therapeutic response and predict adverse events throughout the course of cell and gene therapy development.

In this eChalk Talk we discuss how high throughput mass spectrometry can be used to broadly capture thousands of dynamic, non-genetic cell & gene therapy biomarkers and identify those that signal pharmacodynamic response, drug-target engagement, and toxicity events, both early after treatment and over time.