Information about how VX809 works with F508del (He et al. 2012):

What needs to occur with F508del:
The channel needs to be assembled properly, stable at the cell surface and function well at the cell surface.

What occurs with VX809 and F508del:
– VX809 rescued the assembly (folding) at body temperature.
– The VX809 rescued CFTR has a short lifetime (decreased stability) and loses channel activity at body temperature.
– Other medications which stabilise the CFTR could increase the CFTR rescue, these could be combined with VX809 or similar medications that help assembly.

How it interacts:
– Where VX809 acts on the CFTR is unknown, there is only circumstantial evidence that it interacts directly with the protein, possibly during its assembly in the endoplasmic reticulum.
– There are several possible binding sites including the NBD1/CL4 interface. This area has also been targeted by computer models to identify additional correctors.



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1. Class 2 Mutations & F508del, 3. Vertex - VX809 & VX661