SpliSense has secured a funding commitment of up to $13 million from the Cystic Fibrosis Foundation to support the continued clinical development of its lead investigational therapy, SPL84, a novel inhaled antisense oligonucleotide (ASO) treatment for cystic fibrosis (CF).
The clinical-stage biotechnology company announced the agreement following encouraging Phase 2a clinical data for SPL84, which demonstrated favorable safety and promising efficacy signals in patients carrying the 3849+10kb C→T mutation, a genetic variant associated with cystic fibrosis. The funding is expected to help advance the therapy through later-stage clinical studies and potentially toward regulatory approval.
According to SpliSense, the Phase 2a trial produced encouraging outcomes, including improvements in lung function in up to 70% of treated participants. The company also reported an estimated mean absolute improvement of 10 percentage points in percent predicted forced expiratory volume in one second (ppFEV1) compared with placebo. The findings marked what the company described as the first clinical proof-of-concept for an inhaled antisense oligonucleotide therapy in a pulmonary disease.
SPL84 is designed to address the underlying genetic defect in patients with the 3849+10kb C→T mutation, a population that continues to face unmet medical needs despite advances in cystic fibrosis treatment. The therapy is administered through inhalation, enabling direct delivery to the lungs, the primary organ affected by the disease.
Gili Hart, Chief Executive Officer of SpliSense, said the investment reflects confidence in both the company’s clinical results and the broader potential of its RNA-based therapeutic platform. Hart noted that the support from the Cystic Fibrosis Foundation, one of the leading organizations dedicated to advancing innovative treatments for people living with CF, will help accelerate the development of SPL84 and support future programs targeting additional lung diseases.
The company is currently conducting a Phase 2b clinical trial evaluating the safety, tolerability, and efficacy of SPL84 in cystic fibrosis patients who carry the 3849+10kb C→T mutation and are receiving standard-of-care CFTR modulators. The randomized, placebo-controlled study is expected to enroll approximately 40 participants across clinical sites in the United States, Europe, and Israel.
Top-line data from the Phase 2b study are anticipated during the second half of 2027. The trial is registered under ClinicalTrials.gov identifier NCT06429176.
Beyond SPL84, SpliSense is leveraging its proprietary inhaled ASO platform to develop additional therapies for respiratory diseases. Its pipeline includes SPL5AC, which is being investigated for muco-obstructive conditions such as chronic obstructive pulmonary disease (COPD), non-cystic fibrosis bronchiectasis, asthma, and cystic fibrosis. The company is also advancing SPL5B, a candidate targeting idiopathic pulmonary fibrosis (IPF).
The new funding agreement strengthens SpliSense’s financial position and highlights growing interest in RNA-based medicines for pulmonary disorders. As the company advances its clinical programs, the support from the Cystic Fibrosis Foundation could play a significant role in accelerating the development of therapies aimed at addressing genetic and chronic lung diseases that continue to present major treatment challenges.