期刊论文SPL84 and trikafta® exhibit comparable and additive effects in patient-derived HBE cells carrying the 3849+ 10kb C→ T/F508del CFTR variants复制

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Y Cheng, YS Oren, J Harrington, A Hatton…
Journal of Cystic …, 2026
Elsevier
Abstract Background The 3849+ 10kb C→ T (3849) splicing variant in the CFTR gene creates a cryptic exon containing a stop codon, reducing wild-type mRNA and producing non-functional protein. Although Trikafta®(ETI) is approved for people with cystic fibrosis (pwCF) carrying this variant, clinical responses are often modest and variable, underscoring the need for therapies that directly target the 3849 splicing defect. SPL84 is an inhaled antisense oligonucleotide (ASO) designed to restore normal splicing. In a Phase 2a study …