The discovery of biomarkers that track with the correction of CFTR and/or disease progression can address many of the present goals of transforming patient care. For example, ex vivo markers such as primary cell organoid or monolayer responses to therapies that modulate CFTR function can help us enhance personalization of care and allow us to tailor CFTR modulator therapy based on patient cell responses to drug. In patients, markers of CFTR correction such as sweat chloride are being standardized, and markers of disease progression and/or manifestations such as inflammation are under study. The session will highlight a number of approaches to biomarker discovery and application to foster a discussion of the questions we can answer using this approach to improve care.
Summarize about innovative approaches to identify novel
biomarkers that are associated with markers of disease progression in CF.
Identify the most promising biomarkers to sensitively detect the
biologic activity of new therapeutics and enable CF drug development.
Define the role of biomarkers in therapeutic drug development
and the need for appropriate studies that will enable qualification and