Thursday, June 6, 2013
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Cystic fibrosis (CF)
Cystic fibrosis transmembrane conductance regulator (CFTR)
vitro and cell
culture studies identified the molecular mechanism of action for CFTR
corrector compounds and suggest new combination approaches that could help
treat CF. In reconstituted phospholipid bilayers carrying recombinant
ΔF508 CFTR, the corrector compound VX-809
directly bound the protein and stabilized the interface between nucleotide
binding domain 1 and membrane spanning domains 1 and 2. In cultured cells and
samples from patients with CF that have the ΔF508 CFTR mutation, VX-809
plus either of two compounds that help stabilize protein folding increased
functional CFTR expression compared with any individual treatment. Next steps
include identifying drug-like stabilizers of NBD1 folding, which could be
combined with VX-809 or other CFTR correctors.
Pharmaceuticals Inc.'s VX-809, a CFTR corrector, is in
Phase III trials to treat ΔF508 mutant CF in combination with the CFTR
a CFTR corrector that is structurally related to VX-809, is in Phase II
trials in combination with Kalydeco to treat ΔF508 CF.
Vertex markets Kalydeco to treat CF.
Published online June 6, 2013
Patent and licensing status
Okiyoneda, T. et al.
Nat. Chem. Biol.; published online May 12, 2013;
Contact: Gergely L. Lukacs, McGill University, Montreal,
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