Drug delivery specialist Aptar Pharma is collaborating with Aceso Therapeutics to advance development for the latter’s ACT-101 – an inhalable antisense oligonucleotide (ASO) designed to treat the underlying disease mechanisms of cystic fibrosis (CF).
Under the pair-up, Nanopharm, Aptar’s specialist inhalation development services business, will lead formulation development and device assessment activities to support Aceso’s clinical development roadmap for ACT-101.
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The French biotech’s biologic is under development as a CF therapy inhaled directly into the lungs. The ASO uses a patented, first-in-class RNA mechanism targeting post-transcriptional regulation to correct or improve CFTR protein function and is specifically optimised for CF patients bearing the F508del mutation. While there are several inhaled therapies for CF already approved, ACT-101 is designed to intervene a step earlier than current modulators.
Currently in advanced preclinical development, Aceso has previously said it plans to make an investigational new drug (IND) filing for ACT-101 with the US Food and Drug Administration (FDA), paving the way towards the initiation of first-in-human trials of the ASO, in 2027.
“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. 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,” said Aceso Therapeutics’ CEO and co-founder, Thomas Tran.
“This collaboration marks an important milestone for Aceso and for the continued development of potential new approaches for cystic fibrosis,” Tran added.
Aptar highlighted that its collaboration with Aceso complements its broader biologics compatibility programme, which is evaluating how complex biologic molecules including nucleic acids, peptides and proteins, and their associated nanoparticle-based delivery systems interact with its pulmonary and nasal delivery platforms across a range of formulations and delivery formats.
Gemma Budd, general manager of Nanopharm, commented: “Cystic fibrosis remains a devastating disease with significant unmet need. Partnering with Aceso Therapeutics enables us to apply our formulation and inhalation science expertise to an innovative oligonucleotide asset.”
CF causes severe damage to the lungs, digestive system and other organs and affects approximately 100,000 people worldwide, as per the American Lung Association (ALA).
