Synonyms: compound 1 [WO2018064632A1] | VX-659 | VX659
Compound class:
Synthetic organic
Comment: Bamocaftor (VX-659) is a next-generation CFTR corrector class molecule [2]. It is structurally related to elexacaftor and like other corrector molecules was designed to improve CFTR protein processing and trafficking in the presence of the F508del mutation. Bamocaftor's mechanism of action is different from that of the first generation CFTR corrector tezacaftor and its functional and clinical effects appear to be additive [2]. As such a triple combination therapeutic with tezacaftor, izacaftor (a CFTR potentiator that increases CFTR channel gating activity) and bamocaftor increases CFTR function beyond that achieved by current standard of care dual therapy tezacaftor/izacaftor, and improves clinical symptoms in patients with cystic fibrosis with one or two F508del alleles [3].
![]() Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖ |
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