Chapter 19 – Biomarkers

Regenerative medicine is a new area of translational science that comprises different emerging technologies such as nanotechnology, innovative biomaterials, genetic engineering, and cellular therapeutics. While potential clinical indications as well as the market size are huge, any application still needs to prove that its clinical efficacy and its deployment are sustainable. In the context of translational regenerative medicine, biomarkers not only characterize the function, quality, and safety/toxicity of a product (potency) but also help identify those patients or cohorts who would most likely benefit from such an innovative treatment modality (prediction or stratification) and allow efficacy in those patients who have been identified as responders to be monitored. This is of pivotal importance for the development of drugs or drug candidates that have not yet been approved to foster the early decision on how to go forward and where to stop the development of less promising compounds or technologies. Diverse and numerous technologies to identify and measure biomarkers are currently available. The “omics” platforms, modern imaging technologies, and other technologies can be used as tools to measure many biomarkers in vivo and ex vivo. Intelligent clinical trial design is another valuable tool to validate and deploy biomarkers and to accelerate cost-saving drug development, in particular in regenerative medicine.