Israel-based clinical-stage biotechnology company SpliSense has secured up to $13 million in funding from the Cystic Fibrosis Foundation to support the continued clinical development of its lead therapy candidate, SPL84, an inhaled antisense oligonucleotide (ASO) therapy for cystic fibrosis (CF).
The investment follows positive SPL84 Phase 2a clinical results, which demonstrated a favourable safety profile and early signs of efficacy. According to the company, up to 70 per cent of treated participants showed improvements in lung function, with an estimated mean absolute increase of 10 percentage points in ppFEV1 compared with placebo.
The findings mark what SpliSense describes as “the first-ever clinical proof-of-concept for an inhaled ASO therapy in a pulmonary disease.”
Cystic fibrosis is a genetic, multisystem disorder caused by mutations in the CFTR gene, which produces a protein essential for regulating chloride transport in the lungs and other organs. Although CFTR modulator therapies have transformed treatment in recent years, they do not work for all people with CF and do not offer a cure for the disease.
SPL84 has been developed specifically for people with cystic fibrosis carrying the 3849+10kb C→T mutation — a patient group that continues to face unmet treatment needs despite existing therapies. The inhaled ASO is designed to correct the splicing defect caused by this mutation, enabling the body to produce functional CFTR protein. It is administered directly to the lungs through inhalation, enabling targeted delivery to the primary site of disease.
Commenting on the funding, SpliSense CEO Dr. Gili Hart said SPL84 has the potential to address a significant medical need for people living with this mutation and could also help accelerate development of earlier-stage candidates across the company’s broader respiratory pipeline.
“We appreciate the Foundation’s long-standing support of SpliSense. Their continued support of SPL84 and our RNA-based platform enables us to accelerate development toward later-stage clinical studies and potential registration,” she said.
SpliSense is currently running a Phase 2b randomised, placebo-controlled trial to evaluate the safety, tolerability, and efficacy of SPL84 in people with cystic fibrosis carrying the 3849+10kb C→T mutation who are already receiving standard CFTR modulator treatment.
The study is expected to enrol approximately 40 participants across sites in the United States, Europe and Israel. Topline results are anticipated in the second half of 2027.
SPL84 has received Fast Track and Orphan Drug designation from the US Food and Drug Administration and has also been granted PRIME designation by the European Medicines Agency.
Beyond cystic fibrosis, the company is also advancing therapies for other pulmonary conditions, including SPL5AC for muco-obstructive diseases such as chronic obstructive pulmonary disease (COPD), non-cystic fibrosis bronchiectasis (NCFB), asthma and cystic fibrosis, as well as SPL5B for idiopathic pulmonary fibrosis (IPF)
