Zurich, Switzerland & Paris, France – [Insert Date, e.g., October 26, 2026] – In a significant move set to redefine the therapeutic landscape for Cystic Fibrosis (CF), drug delivery innovator Aptar Pharma has announced a strategic collaboration with Aceso Therapeutics. The partnership aims to accelerate the development of Aceso’s groundbreaking ACT-101, an inhalable antisense oligonucleotide (ASO) designed to tackle the fundamental disease mechanisms of CF. This collaboration leverages Aptar’s specialized inhalation development services, Nanopharm, to pave the way for a novel therapy that promises an earlier, more targeted intervention than existing CF modulators.
n
Cystic Fibrosis, a severe genetic disorder, currently affects approximately 100,000 individuals worldwide, according to the American Lung Association (ALA). It is characterized by the production of abnormally thick and sticky mucus that clogs the lungs, obstructs the pancreas, and can damage other organs. While significant strides have been made in CF treatment, particularly with the advent of CFTR modulators, a substantial unmet need persists for therapies that offer broader applicability, fewer systemic side effects, and even more fundamental correction of the disease pathology. ACT-101 represents a potential leap forward by targeting the disease at an earlier, post-transcriptional stage, offering hope for a new generation of CF patients.
n
Main Facts: A New Frontier in CF Treatment
n
The core of this collaboration centers on ACT-101, a first-in-class inhalable antisense oligonucleotide developed by French biotech Aceso Therapeutics. This innovative biologic is specifically engineered to treat the underlying cause of CF by modulating gene expression, rather than solely correcting misfolded proteins. Aptar Pharma, through its expert inhalation development subsidiary Nanopharm, will play a crucial role in bringing ACT-101 to fruition. Nanopharm’s responsibilities will encompass comprehensive formulation development and meticulous device assessment activities, ensuring that ACT-101 can be effectively and safely delivered directly to the lungs – the primary site of CF pathology.
n
ACT-101 stands out in the crowded CF therapeutic space due to its unique mechanism. While current CFTR modulators primarily focus on improving the function of the defective CFTR protein that has already been produced, ACT-101 is designed to intervene a step earlier. It employs a patented RNA mechanism that targets post-transcriptional regulation, aiming to correct or significantly improve CFTR protein function before the protein is fully formed and potentially misfolded. This approach holds particular promise for CF patients carrying the prevalent F508del mutation, which accounts for a significant portion of CF cases globally. By directly addressing the RNA, ACT-101 seeks to ensure that the cellular machinery produces a functional CFTR protein from the outset, offering a more upstream therapeutic strategy.
n
The partnership underscores a shared vision between Aptar and Aceso: to bring transformative, patient-centric therapies to market. For Aceso, leveraging Nanopharm’s specialized expertise in inhaled drug product development is critical for navigating the complexities of delivering a delicate ASO directly to the pulmonary system. For Aptar, this collaboration further solidifies its position as a leading partner in the development of advanced biologic therapies, particularly those requiring sophisticated inhalation solutions.
n
Chronology: Charting the Path to Clinical Trials
n
ACT-101 is currently in advanced preclinical development, a crucial stage where its safety, efficacy, and optimal delivery parameters are rigorously evaluated in laboratory and animal models. The promising results from these preclinical studies have emboldened Aceso Therapeutics to outline an ambitious, yet achievable, timeline for its progression into human trials.
n
Aceso has publicly stated its intention to submit an Investigational New Drug (IND) filing for ACT-101 with the U.S. Food and Drug Administration (FDA) in 2027. This IND filing is a pivotal regulatory milestone, representing a comprehensive application detailing all preclinical data, manufacturing information, and the proposed clinical trial design. A successful IND clearance by the FDA would then pave the way for the initiation of first-in-human (Phase 1) clinical trials for ACT-101. These initial trials are designed primarily to assess the safety, tolerability, and pharmacokinetic profile of the drug in human volunteers, typically healthy individuals or a small cohort of CF patients.
n
Should the Phase 1 trials yield positive results, ACT-101 would then progress through subsequent phases of clinical development (Phase 2 and Phase 3), involving larger patient populations to further evaluate efficacy and safety. The journey from preclinical development to market approval is arduous and lengthy, often spanning many years. However, the collaborative efforts between Aceso and Aptar, particularly Nanopharm’s role in optimizing formulation and delivery, are expected to streamline this process, minimizing potential hurdles and accelerating the drug’s trajectory toward patients. The strategic decision to target an IND filing in 2027 reflects a confident and well-planned development roadmap, indicative of the robust preclinical data supporting ACT-101’s potential.
n
Supporting Data: Unpacking the Science and the Need
n
Cystic fibrosis is caused by mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, which provides instructions for making the CFTR protein. This protein acts as a channel on the surface of cells, primarily involved in transporting chloride and bicarbonate ions across cell membranes. When the CFTR protein is defective or absent due to genetic mutations, ion transport is disrupted, leading to the production of thick, sticky mucus in various organs, most notably the lungs and pancreas. This mucus clogs airways, leading to chronic infections, inflammation, and progressive lung damage, and impairs digestive enzyme release, causing malabsorption and malnutrition.
n
The F508del mutation is the most common CF-causing mutation, present in approximately 90% of individuals with CF and in 50% of people with CF worldwide, with two copies of the mutation. This mutation results in a misfolded CFTR protein that is prematurely degraded by the cell, preventing it from reaching the cell surface to perform its function.
n
Current Therapeutic Landscape and Unmet Needs:nOver the past decade, the introduction of CFTR modulators, such as ivacaftor (Kalydeco), lumacaftor/ivacaftor (Orkambi), tezacaftor/ivacaftor (Symdeko), and elexacaftor/tezacaftor/ivacaftor (Trikafta), has revolutionized CF treatment. These small molecule drugs directly target the defective CFTR protein, helping it to fold correctly, reach the cell surface, and/or function more efficiently. Trikafta, in particular, has demonstrated remarkable efficacy for patients with at least one F508del mutation, significantly improving lung function and quality of life for many.
n
However, even with these advancements, significant unmet needs persist:
n
- n
- Non-responders: Not all patients benefit from current modulators, particularly those with rare or nonsense mutations.
- Residual Disease: While highly effective, current modulators do not fully restore CFTR function to normal levels, meaning patients may still experience some progression of lung damage and other complications over time.
- Systemic Side Effects: As orally administered drugs, CFTR modulators have systemic exposure, leading to potential off-target effects and drug-drug interactions.
- Cost: The high cost of these therapies remains a significant barrier to access in many parts of the world.
- Early Intervention: The progressive nature of CF lung disease emphasizes the need for even earlier and more fundamental interventions.
n
n
n
n
n
n
The Promise of ACT-101 and ASOs:nACT-101, as an antisense oligonucleotide (ASO), offers a fundamentally different approach. ASOs are short, synthetic single-stranded DNA or RNA molecules that can selectively bind to messenger RNA (mRNA) or pre-mRNA. By binding to specific RNA sequences, ASOs can modulate gene expression, either by blocking the production of a problematic protein, enhancing the production of a deficient protein, or altering RNA splicing. In the context of CF, ACT-101’s "first-in-class RNA mechanism targeting post-transcriptional regulation" suggests it aims to correct the genetic instructions after transcription (DNA to RNA) but before translation (RNA to protein). This could involve mechanisms like:
n
- n
- Correcting Splicing: Ensuring the correct mRNA transcript is produced, leading to a functional CFTR protein.
- Stabilizing mRNA: Preventing the premature degradation of CFTR mRNA, allowing more functional protein to be made.
- Modulating Translation: Directly influencing the cellular machinery to produce a correctly folded and functional CFTR protein.
n
n
n
By intervening at the RNA level, ACT-101 aims to prevent the production of misfolded or non-functional CFTR protein from the very beginning, a step earlier than modulators that attempt to fix an already flawed protein. The inhaled delivery route is also critical, allowing the ASO to be directly deposited into the lungs, where the primary damage of CF occurs. This local delivery minimizes systemic exposure, potentially reducing side effects and allowing for lower, more concentrated doses at the site of action.
Aptar Pharma and Nanopharm’s Expertise:
Aptar Pharma is a global leader in drug delivery systems, specializing in devices for nasal, pulmonary, and injectable applications. Its subsidiary, Nanopharm, is particularly renowned for its deep scientific expertise in inhalation and nasal drug product development. Nanopharm’s capabilities include:
- Advanced Formulation Development: Designing optimal formulations for complex molecules like ASOs to ensure stability, bioavailability, and effective aerosolization.
- Device Assessment and Selection: Identifying and optimizing the most suitable inhalation device (e.g., nebulizer, dry powder inhaler, metered-dose inhaler) for a given formulation to maximize drug delivery to the lungs.
- Aerosol Science and Characterization: Precisely characterizing the aerosol properties (e.g., particle size distribution) to ensure consistent and targeted lung deposition.
- Preclinical and Clinical Support: Providing comprehensive data and expertise to support regulatory filings and clinical trial design.
The collaboration with Aceso aligns perfectly with Aptar’s broader biologics compatibility program. This initiative focuses on understanding how complex biologic molecules – including nucleic acids (like ASOs), peptides, and proteins – interact with Aptar’s delivery platforms across various formulations and formats. By actively engaging with partners like Aceso, Aptar is not only advancing specific therapies but also building a deeper scientific understanding of the challenges and solutions for delivering next-generation biologics via inhalation.
Official Responses: Voices from the Collaboration
The significance of this partnership and the promise of ACT-101 were highlighted by key figures from both collaborating organizations.
Thomas Tran, CEO and co-founder of Aceso Therapeutics, expressed profound optimism regarding the collaboration: “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.” Tran further emphasized the broader impact, stating, “This collaboration marks an important milestone for Aceso and for the continued development of potential new approaches for cystic fibrosis.” His statements underscore Aceso’s strategic decision to leverage external expertise to de-risk and expedite the development of their innovative asset.
Gemma Budd, General Manager of Nanopharm, echoed this enthusiasm, highlighting the critical unmet need that ACT-101 seeks to address. “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,” Budd remarked. Her comments emphasize Nanopharm’s commitment to applying its specialized capabilities to address serious diseases and contribute to the development of truly transformative therapies. The synergy between Aceso’s cutting-edge biologic and Nanopharm’s delivery expertise is expected to be a powerful engine for progress.
Implications: Reshaping the Future of CF Therapy
The collaboration between Aptar Pharma and Aceso Therapeutics carries profound implications for the future of cystic fibrosis treatment and the broader field of genetic medicine.
For CF Patients:
If successful, ACT-101 could offer a significant new therapeutic option, particularly for those who do not fully benefit from current CFTR modulators or those seeking a more fundamental correction of the disease. The direct lung delivery mechanism could lead to more localized efficacy with potentially fewer systemic side effects, improving patient safety and compliance. By intervening earlier in the disease cascade, ACT-101 could potentially slow or even halt the progression of lung damage more effectively, preserving lung function and significantly enhancing the quality of life and life expectancy for CF patients. This could be particularly impactful for younger patients, where early intervention can have a lifelong benefit.
For ASO Therapy in Lung Diseases:
The successful development of an inhaled ASO for CF would represent a major validation for this therapeutic modality in respiratory diseases. It would open doors for exploring ASOs for other lung conditions driven by genetic defects or abnormal protein production, such as alpha-1 antitrypsin deficiency, idiopathic pulmonary fibrosis, or even severe asthma. This collaboration could set a precedent for how complex nucleic acid therapies are formulated and delivered directly to the lungs, stimulating further innovation in the field.
For Aptar Pharma and Nanopharm:
This partnership strengthens Aptar’s strategic position in the rapidly growing market for biologics and advanced therapies. By demonstrating its capability to facilitate the delivery of sophisticated molecules like ASOs, Aptar enhances its reputation as a preferred partner for biotech and pharmaceutical companies developing next-generation treatments. It also validates Nanopharm’s specialized expertise in inhalation development, reinforcing its role at the forefront of aerosol science and drug delivery innovation. The insights gained from developing ACT-101 will undoubtedly feed into Aptar’s broader biologics compatibility program, fostering a deeper understanding of the challenges and opportunities in delivering various complex molecules.
Challenges and Opportunities:
While the potential is immense, the path forward is not without challenges. The development of inhaled biologics, especially ASOs, involves complex hurdles related to stability, formulation, nebulization efficiency, and cellular uptake within the lung. Ensuring patient adherence to an inhaled regimen and demonstrating long-term safety and efficacy will be crucial during clinical trials. The competitive landscape for CF therapies is also dynamic, with ongoing research into gene editing and other novel approaches.
However, the focused strategy of ACT-101, targeting the F508del mutation with an earlier intervention mechanism via direct lung delivery, positions it uniquely. The combined scientific rigor and development expertise of Aceso and Aptar provide a robust foundation to navigate these challenges. This collaboration is not just about a single drug; it represents a commitment to pushing the boundaries of what is possible in treating one of the most challenging genetic diseases, heralding a future where targeted, inhaled biologic therapies could significantly improve patient outcomes. The anticipated IND filing in 2027 will be a eagerly watched milestone, signaling the transition of this promising science into tangible hope for CF patients worldwide.
