SpliSense Secures $13 Million for Cystic Fibrosis Drug Trial

The capital injection follows promising clinical data where SPL84 demonstrated a mean absolute improvement of 10 percentage points in ppFEV1. In that study, 70% of participants showed improved lung function, marking the first clinical proof-of-concept for an inhaled antisense oligonucleotide targeting pulmonary disease. SpliSense CEO Gili Hart noted that the support reinforces the potential of their RNA-based platform to address significant unmet medical needs.

Currently, the company is conducting a Phase 2b study involving approximately 40 participants across the United States, Europe, and Israel. This trial evaluates the drug in combination with standard CFTR modulators, with topline results expected in the second half of 2027. Beyond this lead candidate, the company is leveraging its proprietary platform to develop treatments for other conditions, including chronic obstructive pulmonary disease and idiopathic pulmonary fibrosis. SPL84 has already secured Fast Track and Orphan Drug designations from the FDA, alongside an EMA PRIME designation.