- Cystic Fibrosis Foundation funding follows positive SPL84 Phase 2a clinical results demonstrating the first-ever clinical proof-of-concept for an inhaled antisense oligonucleotide therapy in a lung disease
- Funding supports the ongoing global Phase 2b study evaluating SPL84 in people with cystic fibrosis carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR modulators; topline results expected in H2 2027
JERUSALEM, June 16, 2026 /PRNewswire/ — SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced that it has entered into a funding agreement with the Cystic Fibrosis Foundation, under which the Foundation will provide up to $13 million to support the continued clinical development of SPL84, SpliSense’s lead inhaled antisense oligonucleotide (ASO) therapy for cystic fibrosis (CF).
The funding follows SpliSense’s positive Phase 2a clinical results for SPL84, which demonstrated favorable safety and encouraging efficacy, including improvement in lung function in up to 70% of treated participants and an estimated mean absolute improvement of 10 percentage points in ppFEV1 versus placebo. These results represented the first-ever clinical proof-of-concept for an inhaled ASO therapy in a pulmonary disease.
“SPL84 has the potential to address a significant medical need for people living with cystic fibrosis who carry the 3849+10kb C→T mutation,” said Gili Hart, Ph.D., Chief Executive Officer of SpliSense. “We are honored to receive this investment from the Cystic Fibrosis Foundation, one of the world’s leading organizations advancing innovative therapies for people with CF. We believe this commitment reflects both the strength of our Phase 2 clinical data and the potential of SPL84 to become a transformative treatment option for patients, paving the way also for earlier candidates in our pipeline developed for additional lung diseases to advance into the clinic.”
Dr. Hart continued, “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.”
The ongoing Phase 2b study is designed to evaluate the safety, tolerability, and efficacy of SPL84 in people with cystic fibrosis carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR modulators. The randomized, placebo-controlled study is expected to enroll approximately 40 participants across sites in the United States, Europe and Israel. Topline results are anticipated in H2 2027 (NCT06429176).
The positive Phase 2 results and the initiation of the Phase 2b program further validate SpliSense’s proprietary inhaled ASO platform and support advancement of the Company’s broader pipeline, 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).
About SPL84
SPL84 is an inhaled antisense oligonucleotide designed to correct the splicing defect caused by the 3849+10kb C→T mutation in the CFTR gene. The 3849+10kb C→T mutation represents a clinically meaningful CF population with continued unmet need despite available CFTR modulator therapies. By targeting the mutant CFTR RNA, SPL84 promotes production of functional CFTR protein. The therapy is administered directly to the lungs through inhalation, enabling targeted delivery to the primary site of disease. SPL84 has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration and was recently granted EMA PRIME designation as well.
About Cystic Fibrosis (CF)
CF is a genetic multisystem disorder that originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed in the lungs as well as in other tissues. The past decade has seen a dramatic change in CF care with the approval of new CFTR modulators. However, approved CFTR modulators do not support all people with CF and do not offer a cure for the disease. Thus, new strategies of therapeutic development are essential to address partially responsive and non-responsive people with CF, specifically those carrying the 3849+10 Kb C->T splicing mutation.
About SpliSense
SpliSense is a clinical-stage biotechnology company developing ASO-based therapies for pulmonary diseases. The Company’s proprietary inhaled ASO platform is designed to address the root causes of severe pulmonary disorders through modulation of gene expression and RNA processing. SpliSense is advancing a pipeline of programs in cystic fibrosis, muco-obstructive lung diseases (COPD, Asthma and NCFB) and idiopathic pulmonary fibrosis.
For additional information, please visit www.splisense.com.
Company Contact:
Tsipi Haitovsky
Global Media Liaison
+972-52-5989-892
tsipi@splisense.com
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