This session initially focuses on establishing a short coming of the recently approved small molecule modulators VX-809 and VX-770. These molecules fail to restore the defective membrane distribution of F508del-CFTR. Then, the session emphasizes the need for finding new approaches to find new and more effective therapies by targeting different aspects of CFTR trafficking and recycling. Finally, the session ends with showing that the mouse model is not effective in reflecting drug efficacy in human subjects.
Demonstrate the lack of effectiveness of VX-809 and VX-770 in the restoration of the defective membrane distribution of F508del-CFTR.
Create the need for the identification of new potential therapies targeting different aspects of mutant CFTR trafficking and internalization.
Create a difference in the response of mouse and human models to newly developed treatments when defining drug efficacy.