Developers partner to advance inhaled CF treatment toward human trials

Two drug developers are teaming up to advance toward clinical trials an inhaled treatment targeting the disease mechanisms underlying cystic fibrosis (CF).

The U.S.-based Aptar Pharma and Aceso Therapeutics, headquartered in France, announced they will collaborate on developing ACT-101, an experimental RNA therapy. Unlike traditional drugs that target proteins in the body after they are made, RNA therapies work earlier, focusing on biological pathways, as a means to treat disease.

The program will be supported by Nanopharm, an Aptar company that will lead formulation development and conduct device assessment activities, Aceso said in a press release detailing the plans. After completion of preclinical studies, Aceso plans to submit a clinical trial application to conduct clinical studies in humans. That submission is expected in late 2027.

“By partnering with Nanopharm, Aceso Therapeutics aims to accelerate the development of ACT-101 toward clinical evaluation, exploring its potential through direct delivery to the lung,” said Thomas Tran, Aceso’s cofounder and CEO. “This collaboration marks an important milestone for Aceso and for the continued development of potential new approaches for cystic fibrosis.”

Gemma Budd, Nanopharm’s general manager, said the collaboration will allow the company to apply its “formulation and inhalation science expertise” to ACT-101.

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CF is caused by genetic mutations in the CFTR gene, which result in an absent or dysfunctional CFTR protein. That protein helps control the movement of salt and water in and out of cells. A lack of CFTR protein leads to the accumulation of thick, sticky mucus in various organs, particularly the lungs and digestive tract, causing most CF symptoms.

ACT-101 uses a novel approach

ACT-101, in the pipeline at Aceso, is designed to increase the production of the CFTR protein. It uses an antisense oligonucleotide, a short piece of genetic material to prevent CFTR messenger RNA (mRNA) — the molecule carrying genetic instructions to make the protein — from being broken down. By stabilizing CFTR mRNA, ACT-101 may allow cells to produce more of their own CFTR protein.

Because the approach does not target specific CFTR mutations, it could potentially benefit people with CF whose mutations are not eligible for currently available CFTR modulators.

The investigational therapy was developed using Aceso’s PTGR (Post-Transcriptional Gene Restoration) platform, designed to restore gene activity by focusing on RNA interactions.

Preclinical studies showed that ACT-101 increased CFTR levels and activity in cells derived from people with CF carrying several clinically relevant mutations. That included F508del, the most common CF-causing mutation. According to the company, CFTR functional activity increased by as much as 60%.

Per Aceso, ACT-101 could be used alongside existing CFTR modulators and offer a potential treatment option for CF patients who have a limited or no response to current treatments.