Cystic Fibrosis Foundation Invests $13M in Israeli Biotech SpliSense

Why It Matters

An inhaled ASO could broaden treatment options beyond oral CFTR modulators, offering a targeted therapy for mutation subsets currently underserved and potentially reshaping the cystic fibrosis market.

Key Takeaways

• Cystic Fibrosis Foundation invests $13 M in SpliSense for inhaled ASO therapy.
• SPL84 showed 70% of patients improved lung function in Phase IIa trial.
• Therapy earned FDA fast‑track, orphan status and EMA PRIME designation.
• Phase IIb study will enroll ~40 patients, results expected H2 2027.

Pulse Analysis

Cystic fibrosis remains one of the most challenging genetic lung diseases in the United States, affecting roughly 40,000 patients. While oral CFTR modulators such as Vertex’s Trikafta have transformed outcomes for many, they only address a subset of mutations and require daily pill intake. An inhaled antisense oligonucleotide (ASO) offers a fundamentally different delivery route, targeting the defective gene directly in the airway epithelium. This approach could expand therapeutic options to patients whose mutations are not amenable to existing modulators and reduce systemic exposure.

SpliSense’s lead candidate, SPL84, is designed to correct the splicing error caused by the 3849+10 kb C→T mutation. In a Phase IIa study, up to 70 % of participants experienced measurable improvements in lung function, and safety signals were favorable. The program has already secured fast‑track and orphan drug status from the FDA and PRIME designation from the EMA, accelerating regulatory pathways. The recent $13 million grant from the Cystic Fibrosis Foundation not only validates the early data but also funds the transition to a Phase IIb trial slated for 2027.

The infusion of nonprofit capital into a late‑stage biotech underscores a growing trend of disease‑focused foundations de‑risking innovative therapeutics. If SPL84 confirms its efficacy in the upcoming Phase IIb, it could capture a niche market alongside high‑selling modulators, potentially adding billions to the CF therapeutic landscape. Moreover, success would pave the way for SpliSense’s pipeline of inhaled ASO treatments targeting other pulmonary conditions, reinforcing the strategic value of inhalation as a delivery platform. Investors and pharma partners will be watching closely as the data emerge.

Deal Summary

The Cystic Fibrosis Foundation announced a $13 million funding commitment to Israeli biotech SpliSense to advance its inhaled antisense oligonucleotide therapy SPL84 for cystic fibrosis. The grant will support the company’s ongoing development, including a Phase IIb study slated for 2027. The funding underscores the foundation’s support for innovative treatments targeting the 3849+10 kb C→T mutation.