Most modern cystic fibrosis (CF) drugs — the “CFTR modulators” that transformed care — work by fixing the faulty CFTR protein after the cell has made it. A new collaboration is trying to intervene one step earlier, at the RNA stage.
Drug-delivery specialist Aptar Pharma — through its inhalation division Nanopharm — is teaming with Aceso Therapeutics to advance ACT-101, an inhalable antisense oligonucleotide aimed at the most common CF mutation, F508del. Aptar will lead formulation development and device work.
How it works
ACT-101 is a short piece of engineered RNA that uses a patented mechanism of post-transcriptional regulation — acting on the messenger RNA to improve the resulting CFTR protein’s function before it’s fully made, rather than propping up a defective protein afterward. Because CF is a lung disease, delivering it by direct inhalation gets the therapy straight to the target tissue.
Why it matters
CF affects roughly 100,000 people worldwide, and while modulators help many, they don’t work for every mutation or patient — so an RNA-level approach could broaden who can be treated. The caveat: this is advanced preclinical work. Aceso plans to file an IND with the FDA in 2027 to begin first-in-human trials, so real clinical proof is still years away. “Cystic fibrosis remains a devastating disease with significant unmet need,” said Nanopharm’s general manager.