spliesense-cystic-fibrosis-foundation-spl84-phase2b

SpliSense has secured up to $13 million in funding from the Cystic Fibrosis Foundation to support the continued development of its lead candidate SPL84 in cystic fibrosis, following positive Phase 2a clinical results and the ongoing initiation of a global Phase 2b study.

The funding agreement will support the development of SPL84, an inhaled antisense oligonucleotide (ASO) therapy designed to correct a splicing defect caused by the 3849+10kb C→T mutation in the CFTR gene. The mutation is associated with a subgroup of people with cystic fibrosis who continue to experience unmet need despite standard-of-care CFTR modulator therapies.

According to SpliSense, SPL84 is designed to promote production of functional CFTR protein by targeting mutant CFTR RNA directly in the lungs, enabling localised delivery of treatment to the primary site of disease.

The company said the investment follows positive Phase 2a data, which demonstrated favourable safety and encouraging efficacy signals, including improvements in lung function in up to 70% of treated participants and a mean absolute improvement of 10 percentage points in ppFEV1 compared with placebo. SpliSense described the results as the first clinical proof-of-concept for an inhaled antisense oligonucleotide therapy in a pulmonary disease.

Gili Hart, chief executive officer of SpliSense, said: “SPL84 has the potential to address a significant medical need for people living with cystic fibrosis who carry the 3849+10kb C→T mutation.”

She added: “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 CF. We believe this commitment reflects both the strength of our Phase 2 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.”

The ongoing Phase 2b study is a randomised, placebo-controlled trial evaluating SPL84 in people with cystic fibrosis carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR modulators. The study is expected to enrol approximately 40 participants across sites in the United States, Europe and Israel, with topline results anticipated in the second half of 2027.

SpliSense said the funding and clinical progress further validate its inhaled ASO platform and support broader development of its respiratory pipeline, which includes programmes targeting chronic obstructive pulmonary disease, non-cystic fibrosis bronchiectasis, asthma and idiopathic pulmonary fibrosis.

The announcement also highlights continued interest from non-profit and disease-focused foundations in supporting mid-stage development of precision RNA-based therapies in rare disease populations, particularly where existing treatment options remain limited.

As the programme progresses through Phase 2b, SPL84 represents one of the more advanced inhaled RNA-based approaches in development for cystic fibrosis, a disease area that continues to evolve despite advances in CFTR modulator therapies.