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On September 4, 2025, CF (CF) recorded a trading volume of $0.30 billion, a 64.83% increase from the previous day, ranking 356th in market activity. The stock closed down 0.70%, reflecting mixed investor sentiment amid developments in its therapeutic pipeline.
Splisense’s investigational therapy SPL84, targeting cystic fibrosis (CF) caused by the 3849+10 kb C-to-T mutation, advanced to Phase 2 clinical trials. The inhaled antisense oligonucleotide aims to correct splicing errors in the CFTR gene, enabling production of functional protein. Interim safety data from the first 12 patients showed no severe adverse effects, while up to 70% of participants demonstrated improved lung function as measured by ppFEV1 compared to placebo. The therapy’s mechanism involves binding to mutated mRNA to restore protein function, addressing a rare genetic mutation affecting a subset of CF patients.
Positive safety results and preliminary efficacy signals have bolstered confidence in the treatment’s potential. Splisense’s CEO highlighted the milestone as the first demonstration of an inhaled antisense oligonucleotide showing efficacy in pulmonary diseases. However, the therapy remains in early-stage trials, with larger-scale data required to confirm long-term benefits and broader applicability.
Backtesting results indicate that CF’s recent performance aligns with historical patterns observed during similar phases of clinical advancements, though market reactions remain subject to regulatory and trial outcome uncertainties.

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