Aceso Therapeutics is an early-stage biotech based in Montpellier, France, developing next-generation therapies for cystic fibrosis, a rare and severe lung disease. Despite current triple therapies, many patients remain ineligible or insufficiently responsive, representing a major unmet medical need and significant market opportunity.

ACT-101 is a first-in-class inhaled antisense oligonucleotide that takes an upstream approach by increasing CFTR mRNA levels, leading to higher protein production and improved CFTR function. This differentiated mechanism offers potential as both a standalone therapy and in combination with existing treatments. Preclinical data in patient-derived epithelia show a significant increase in CFTR activity, potentially reaching clinically meaningful levels, with no toxicity observed in vivo, supporting a favorable safety profile. The program is advancing under regulatory guidance toward a IND submission planned for end-2027 to initiate Phase 1 clinical trials.

Aceso is preparing €2M fundraising complete preclinical development, aiming for Phase 2 in 2031 and partnership.