SpliSense announced that it has secured up to $13 million in new funding from the Cystic Fibrosis Foundation to support an ongoing Phase 2 trial of SPL84 inhaled antisense oligonucleotide for the treatment of cystic fibrosis. The announcements says that the ongoing trial is Phase 2b; however, the official title cited in the study record says that it is Phase 2a. In 2024, the CFF provided $8.5 million for Phase 2 development of SPL84, which recently received early Priority Medicines designation from the EMA.
In September 2025, SpliSense announced data from a Phase 2a SPL84-002 trial in patients with a 3849+10Kb C>T mutation in the CFTR gene which it says “demonstrated favorable safety and encouraging efficacy.” According to the announcement, the ongoing trial is expected to enroll approximately 40 CF patients (64 according to the study record) who have the 3849+10kb C→T mutation, with results expected by the end of 2027.
SpliSense CEO Gili Hart commented, “SPL84 has the potential to address a significant medical need for people living with cystic fibrosis who carry the 3849+10kb C→T mutation. 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.”
Read the SpliSense press release
