Profiling the CFTR Variant Selectivity and Off-Target Interactions of VX-121
Ashish R. Jhangiani, John A. Olson, Austin Tedman, Catherine Foye, JaNise J Jackson, Ashlyn G Winters, J Renfrew White, Mia Perfetti, Grace M Abell, Crissey D. Cameron, Liudmyla Arifova, Brianna Corman, J. Paul Robinson, Charles P. Kuntz, Kaitlyn V. Ledwitch, , Jonathan P. Schlebach
This study evaluates VX-121's efficacy across 232 rare CFTR variants, finding it generally more effective than VX-445 but with one variant showing reduced selectivity.
bioRxiv · Jun 8, 2026 · PreprintPharmacology & Toxicology