The four speakers and a introductory presentation in this session will focus on presenting new information on how small molecule modulators of CFTR folding and function interact with the CFTR protein using biochemical,cell biological, structural and chemical approaches. This information is central to understanding the mechanism of action of these modulators, the site of their action on CFTR, and which disease-causing genotypes should respond to specific modulators or combinations thereof.
Summarize the mechanism of action of both approved, and developmental small molecule therapies that modulate specific mutant CFTR targets.
Summarize how structural, biochemical, cell biological and chemical approaches can be employed to determine the site of action of CFTR modulators.
Distinguish the rationale for treating specific genotypes with small molecule therapies with specific mechanisms of action.