Results mark first evidence of potential clinical benefit of antisense oligonucleotide therapy in a pulmonary disease
Encouraging safety profile with no identified safety signals to date
Improvement in lung function observed in up to 70% of participants treated with SPL84
The readout validates SpliSense’ platform, supporting advancement of the Company’s additional pulmonary programs, for muco-obstructive diseases and idiopathic pulmonary fibrosis, into the clinic in early 2026
JERUSALEM, Sept. 3, 2025 /PRNewswire/ — SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced positive results from SPL84-002, its ongoing global Phase 2 study evaluating SPL84, the Company’s lead antisense oligonucleotide (ASO), for the treatment of people with cystic fibrosis (CF) carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the CFTR gene