Aptar Pharma and Aceso Advance Inhaled CF Therapy

Aptar Pharma has partnered with Aceso Therapeutics to support the development of ACT-101, an antisense oligonucleotide (ASO) candidate being developed as a potential treatment for cystic fibrosis (CF).

The collaboration will focus on advancing ACT-101 toward clinical development, with Aptar Pharma’s specialist inhalation development business, Nanopharm, leading formulation development and device assessment activities.

ACT-101 is designed to target disease mechanisms linked to cystic fibrosis. The companies plan to explore direct delivery of the ASO therapy to the lungs, an approach that could help deliver treatment closer to the site of disease.

Cystic fibrosis is a rare inherited condition that can cause serious damage to the lungs, digestive system and other organs. The disease affects around 100,000 people worldwide and continues to have significant unmet treatment needs.

Gemma Budd, General Manager of Nanopharm, said the partnership allows the company to apply its formulation and inhalation expertise to an oligonucleotide-based therapy.

Under the collaboration, Nanopharm will support Aceso’s clinical development plans by working on the formulation and delivery aspects of ACT-101. Its experience in inhaled and nasal drug product development is expected to play an important role as the program progresses toward clinical evaluation.

Thomas Tran, CEO and Co-Founder of Aceso Therapeutics, said the partnership is intended to accelerate the development of ACT-101 and assess its potential as a lung-delivered treatment for cystic fibrosis.

The work also builds on Aptar Pharma’s broader biologics compatibility program. The program is evaluating how complex biological medicines, including nucleic acids, peptides and proteins, interact with pulmonary and nasal drug delivery platforms.

Aptar Pharma is also studying nanoparticle-based delivery systems across different formulations and delivery formats. The company said these efforts are aimed at supporting the development of next-generation inhaled biologic medicines, from early research through clinical development.

For Aceso Therapeutics, the collaboration represents another step toward advancing its ASO platform and bringing ACT-101 closer to clinical testing. The companies will now work on formulation and device development activities to support the next stages of the program.