Aptar Pharma entered a collaboration with Aceso Therapeutics to advance development of ACT-101, an antisense oligonucleotide candidate designed to target the underlying disease mechanisms of Cystic Fibrosis.

Cystic fibrosis is a rare, inherited disease that causes severe damage to the lungs, digestive system and other organs, and it affects approximately 100,000 people worldwide.² The collaboration pairs Aceso's oligonucleotide platform with Aptar Pharma's inhalation development expertise, aiming to move ACT-101 closer to clinical evaluation for a disease with significant unmet need.¹

What role will Nanopharm play?

The program will be supported by Nanopharm, Aptar Pharma's specialist inhalation development services business, which will lead formulation development and device assessment activities to support Aceso's clinical development roadmap for ACT-101.¹ That work positions Nanopharm to apply its inhalation science expertise directly to an early-stage oligonucleotide asset.

"Cystic fibrosis remains a devastating disease with significant unmet need," said Gemma Budd, general manager of Nanopharm, an Aptar Pharma company. "Partnering with Aceso Therapeutics enables us to apply our formulation and inhalation science expertise to an innovative oligonucleotide asset. We are proud to contribute our expertise to this early-stage development program."

"By partnering with Nanopharm, Aceso Therapeutics aims to accelerate the development of ACT-101 towards clinical evaluation, exploring its potential through direct delivery to the lung," said Thomas Tran, chief executive officer and co-founder of Aceso Therapeutics. "Nanopharm's proven track record in inhaled and nasal drug product development, combined with the wider scientific depth within Aptar Pharma, gives us confidence as we continue to advance our ASO platform. This collaboration marks an important milestone for Aceso and for the continued development of potential new approaches for cystic fibrosis."

How does this fit into Aptar Pharma's broader strategy?

The development activities Nanopharm is carrying out under the collaboration with Aceso Therapeutics complement Aptar Pharma's broader biologics compatibility program, which is systematically evaluating how complex biologic molecules, including nucleic acids, peptides and proteins, and their associated nanoparticle-based delivery systems, interact with Aptar Pharma's pulmonary and nasal delivery platforms across a range of formulations and delivery formats.¹

Together, the two initiatives underscore Aptar Pharma's commitment to enabling the next generation of inhaled biologic medicines, from early feasibility through clinical development, as Nanopharm continues to lead formulation and device work on ACT-101 alongside Aceso's broader clinical development roadmap for the program.¹

Pharmaceutical Executive recently spoke with K2 Therapeutics’ CEO Dr. Ying Huang about the growing shift towards globally sourced innovation and focus on global partnerships, where he noted that, “The first priority should always be addressing an unmet medical need. There are certain cancers for which no good treatment options exist, let alone a cure. There are diseases like Alzheimer's and Parkinson's where we can only slow the progression of symptoms. You always want to focus on conditions where the unmet need is significant — because just as with any product, you need to be offering something the market genuinely needs.”

Sources

1. Aptar and Aceso Therapeutics Announce Collaboration to Advance Inhaled Antisense Oligonucleotide Therapy for Cystic Fibrosis Aptar Pharmaceuticals September 7, 2026, https://www.businesswire.com/news/home/20260907721569/en/Aptar-and-Aceso-Therapeutics-Announce-Collaboration-to-Advance-Inhaled-Antisense-Oligonucleotide-Therapy-for-Cystic-Fibrosis
2. Cystic fibrosis Mayo Clinic Date accessed September 8, 2026, https://www.mayoclinic.org/diseases-conditions/cystic-fibrosis/symptoms-causes/syc-20353700