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SPL84 and Trikafta® exhibit comparable and additive effects in patient-derived HBE cells carrying the 3849+10kb C→T/F508del CFTR variants
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DOI：10.1016/j.jcf.2026.06.005.png)
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Abstract
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• SPL84 corrects the CFTR splicing defect caused by the 3849+10kb C→T mutation and restores CFTR function in patient-derived airway epithelial cells. • SPL84 successfully completed phase 2a study demonstrating it safety and efficacy following weekly inhaled treatment in pwCF carrying 3849+10kb C→T mutation. • SPL84 demonstrates CFTR functional rescue comparable to Trikafta® in HBE cells from pwCF carrying the 3849/F508del variants. • SPL84 and Trikafta® combination therapy resulted in a significant and consistent additive improvement in CFTR activity across multiple independent patient-derived bronchial epithelial cell donors, with mean activity approximately doubling relative to either treatment alone. • The additive effect can potentially lead to a further significant improvement in patient’s lung function, breaking of the observed modulators ceiling effect, further supporting SPL84 clinical development in a phase 2b study evaluated in combination with Trikafta®.
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