Aptar and Aceso Therapeutics Partner to Advance Inhaled Antisense Oligonucleotide Therapy for Cystic Fibrosis
核心洞察
Aptar Pharma (搜索) and Aceso Therapeutics (搜索) announced a collaboration to advance ACT-101, an inhaled antisense oligonucleotide (ASO) targeting the underlying disease mechanisms of cystic fibrosis (搜索).
Nanopharm (搜索), Aptar's inhalation development services business, will lead formulation development and device assessment to support Aceso's clinical roadmap for ACT-101.
ACT-101 uses a first-in-class RNA mechanism targeting post-transcriptional regulation to correct CFTR (搜索) function, optimized for patients with the F508del mutation.
Aptar Pharma (搜索), a global leader in drug delivery and dosing technologies, has announced a collaboration with Aceso Therapeutics (搜索), a French biotech developing novel therapies for cystic fibrosis (搜索) (CF), to advance the development of ACT-101, an antisense oligonucleotide (ASO) candidate designed to target the underlying disease mechanisms of CF.
The program will be supported by Nanopharm (搜索), Aptar Pharma (搜索)'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.
A First-in-Class RNA Mechanism for CFTR Correction
ACT-101 is an inhalable antisense oligonucleotide developed as a CF therapy delivered directly into the lungs. The ASO uses a patented, first-in-class RNA mechanism targeting post-transcriptional regulation to correct or improve CFTR (搜索) protein function, and is specifically optimized for CF patients bearing the F508del mutation. While several inhaled therapies for CF are already approved, ACT-101 is designed to intervene a step earlier than current modulators.
The candidate is currently in advanced preclinical development. Aceso has previously indicated plans to make an investigational new drug (IND) filing for ACT-101 with the US Food and Drug Administration (FDA), paving the way toward the initiation of first-in-human trials of the ASO in 2027.
Addressing Significant Unmet Need
Cystic fibrosis (搜索) is a rare, inherited disease that causes severe damage to the lungs, digestive system and other organs, and affects approximately 100,000 people worldwide, according to the American Lung Association (ALA).
Gemma Budd, General Manager of Nanopharm (搜索), an Aptar Pharma (搜索) company, said, "Cystic fibrosis (搜索) remains a devastating disease with significant unmet need. Partnering with Aceso Therapeutics (搜索) 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."
Thomas Tran, CEO and Co-Founder of Aceso Therapeutics (搜索), said, "By partnering with Nanopharm (搜索), Aceso Therapeutics aims to accelerate the development of ACT-101 towards clinical evaluation, exploring its potential through direct delivery to the lung. Nanopharm's proven track record in inhaled and nasal drug product development, combined with the wider scientific depth within Aptar Pharma (搜索), 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 (搜索)."
Complementing a Broader Biologics Compatibility Program
The development activities carried out by Nanopharm (搜索) under the collaboration complement Aptar Pharma (搜索)'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. Together, these initiatives underscore the group's commitment to enabling the next generation of inhaled biologic medicines, from early feasibility through clinical development.
About the Partners
Based in Montpellier, France, Aceso Therapeutics (搜索) builds on over a decade of academic research and continues to develop its technology in collaboration with the PhyMedExp laboratory. With its proprietary PTGR platform designed to restore gene expression in genetic diseases, Aceso aims to build a leading RNA therapeutics company and transform treatment for patients underserved by current therapies.
Aptar Pharma (搜索) is part of AptarGroup, Inc., a global leader in drug delivery, dosing and protection technologies. Nanopharm (搜索), an Aptar Pharma company, is a specialist pharmaceutical product development organization focused on orally inhaled and nasal drug products (OINDPs), applying fundamental science and engineering rigor to accelerate the development of complex inhaled therapies from early feasibility through to clinical supply.
ACT-101 is an investigational candidate that has not been approved by any regulatory authority, and no conclusions regarding its safety or efficacy can be drawn at this stage.