This session will review our understanding of potentiator mechanism of action and compare the effects that potentiators, such as ivacaftor (VX-770), have on different mutant CFTR proteins. Conclusions that allow for determination of candidates for current potentiator therapies will be drawn. Differences in mutant and wild-type CFTR thermal sensitivity will be discussed. Finally, the impact of potentiators on CFTR correction will be addressed.
Describe effects of potentiators on the function of different mutant CFTR proteins.
Evaluate effects of potentiators on mutant CFTR thermal stability.
Discuss effects of potentiators on correction of mutant CFTR.
Senior Director, Clinical Trials,
Cincinnati Children`s Hospital Medical Center