The Cystic Fibrosis Foundation has supplied $13m of funding to SpliSense, helping the biotech continue development of its inhaled drug candidate designed to treat the disease.
SpliSense’s SPL84, an antisense oligonucleotide (ASO) therapy, is being developed for patients with cystic fibrosis carrying the 3849+10kb C→T mutation.
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The drug recently demonstrated positive results in a Phase IIa trial (NCT06429176). Enrolled patients were those harbouring the mutation, with some cohorts involving patients already receiving standard-of-care (SoC) cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The Israeli biotech said that SPL84 showcased favourable safety and encouraging efficacy in the trial. This included up to 70% of treated patients showing an improvement in lung function.
There are close to 40,000 children and adults living with cystic fibrosis in the US. The genetic condition, caused by a faulty CFTR gene, leads to airways clogging up with sticky mucus, making it difficult to clear bacteria. This causes frequent infections and impaired lung function. CFTR modulators are approved for patients with specific mutations and are primarily administered as oral tablets. Vertex Pharmaceutical’s Trikafta (elexacaftor/tezacaftor/ivacaftor) is the best-selling drug in this class, generating $10.31bn in 2025 sales.
According to SpliSense, its Phase IIa results are the first to show proof-of-concept for an inhaled ASO therapy. SPL84 is designed to correct the splicing defect caused by the 3849+10kb C→T mutation in the CFTR gene. The therapy is administered directly to the lungs through inhalation, representing targeted delivery of the therapy. SPL84 has received fast track and orphan drug designations from the US Food and Drug Administration (FDA), as well as a PRIME designation from the European Medicines Agency (EMA) – all three providing streamlined regulatory benefits.
With financial backing from the Cystic Fibrosis Foundation now secured, the biotech will now turn towards its ongoing Phase IIb study. The randomised, placebo-controlled study is expected to enrol approximately 40 patients across sites in the US, Europe and Israel, with topline results expected in the second half of 2027.
SpliSense CEO, Gili Hart, said: “We are honoured to receive this investment from the Cystic Fibrosis Foundation, one of the world’s leading organisations advancing innovative therapies for people with cystic fibrosis. We believe this commitment reflects both the strength of our Phase II clinical data and the potential of SPL84 to become a transformative treatment option for patients, paving the way also for earlier candidates in our pipeline developed for additional lung diseases to advance into the clinic.”
SpliSense is not the only company to work with the Cystic Fibrosis Foundation this year. In March, the charity entered a research agreement with AI-driven techbio company Antiverse to develop antibodies targeting the extracellular region of the CFTR protein.
