vanzacaftor (VX-121) / Vertex 
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  • ||||||||||  vanzacaftor (VX-121) / Vertex
    Differential effects of (S)- and (R)-vanzacaftor on BKCa potentiation and CFTR correction () -  Sep 4, 2024 - Abstract #NACFC2024NACFC_1016;    
    Incubation in (S)-VX-121 converts a population of band B F508del CFTR to band C, consistent with F508del CFTR correction, but (R)-VX-121 fails to correct F508del CFTR. (S)- and (R)-VX-121 both stimulate K+ secretion across HBECs and potentiate BKCa channels during whole-cell patch-clamp recording.
  • ||||||||||  vanzacaftor/tezacaftor/deutivacaftor (VX-121/VX-661/VX-561) / Vertex, vanzacaftor (VX-121) / Vertex
    Trial completion, Combination therapy:  A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis (clinicaltrials.gov) -  Jan 9, 2020   
    P2,  N=87, Completed, 
    This, alongwith more readily accessible material of a wide genotype range and greaterexpansion capability, makes the hPEM model an attractive platform forassessing CFTR functional rescue in response to novel therapeutic agents. Active, not recruiting --> Completed