Supplementary Figures:
Supplementary Figure 1 – Intestinal organoids (F508del/F508del)
respond positively to RDR01752. Organoids were incubated for 24 h with
DMSO (negative control), VX-809 (3.7 µM), VX-661 (5 µM) or RDR01752 (10
µM) and acutely stimulated with different concentrations of forskolin
(Fsk, 0.128 to 20 µM) with VX-770 (3 µM) or genistein (Gen, 50 µM).
Graph summarizing data of FIS expressed as the absolute area under the
curve (AUC; baseline = 100%, t = 60 min). N = 3.
Supplementary Figure 2 – RDR01752 is not additive to C18 in
rescuing F508del-CFTR traffic. (A) CFBE stably expressing
mCherry-Flag-F508del-CFTR were incubated for 48 h with the following
compounds individually or in combination: DMSO (negative control),
RDR01752 (10 µM), C18 (5 µM) and VX-770 (3 µM). (B)Immunostaining was performed and fluorescence images of extracellularly
exposed Flag-tags were quantified to determine CFTR PM expression
compared to the DMSO. N = 4. Vs. single corrector (white bars):
*P <0.05, ***P < 0.001. n.s.: not
significant.