Aptar Pharma and Aceso Therapeutics Collaborate to Advance Inhaled ASO Therapy for Cystic Fibrosis
Aptar Pharma, a global provider of drug delivery, dosing, protection technologies and development services, has announced a new collaboration with Aceso Therapeutics, a biotechnology company focused on developing innovative treatments for cystic fibrosis (CF). The partnership will support the development of ACT-101, an antisense oligonucleotide (ASO) candidate being investigated as a potential therapy designed to address underlying disease mechanisms associated with cystic fibrosis.
The collaboration brings together Aceso Therapeutics’ focus on novel genetic medicines with the formulation and inhalation development expertise of Nanopharm, Aptar Pharma’s specialist inhalation development services business. Under the agreement, Nanopharm will lead formulation development and device assessment activities intended to support Aceso Therapeutics’ clinical development plans for ACT-101.
The collaboration represents an important step in the development of potential nucleic acid-based treatments for cystic fibrosis and highlights the growing interest in directly delivering advanced therapeutic molecules to the lungs.
Addressing the Underlying Mechanisms of Cystic Fibrosis
Cystic fibrosis is a rare inherited genetic disorder that can cause progressive and severe damage to multiple organs, particularly the lungs and digestive system. The disease affects approximately 100,000 people worldwide, creating a continuing need for innovative therapeutic approaches.
CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These genetic abnormalities can interfere with the production or function of the CFTR protein, disrupting the movement of salt and water across cell membranes. In the lungs, this can contribute to the accumulation of thick and sticky mucus, recurrent infections, inflammation and progressive deterioration of respiratory function.
Existing therapies have improved outcomes for many people living with cystic fibrosis, particularly treatments designed to address specific CFTR mutations. Nevertheless, significant unmet medical needs remain, including for patients who cannot benefit sufficiently from currently available approaches.
This continuing need has encouraged researchers and biotechnology companies to investigate therapies capable of addressing disease biology at a deeper level.
ACT-101 is an antisense oligonucleotide candidate being developed by Aceso Therapeutics with the goal of targeting disease mechanisms associated with cystic fibrosis. ASOs are short strands of synthetic nucleic acids designed to interact with specific RNA sequences and influence the production of proteins. Their ability to modulate gene expression has generated considerable interest as a therapeutic strategy across a range of genetic and rare diseases.
Focus on Direct Delivery to the Lung
A central component of the ACT-101 development strategy is the potential for direct delivery to the lungs. Because the respiratory system is one of the primary organs affected by cystic fibrosis, inhaled administration may provide a way to deliver the therapeutic candidate directly to the site where it is intended to act.
Developing an inhaled nucleic acid medicine, however, involves a number of technical challenges. The formulation must maintain the stability and activity of the therapeutic molecule while also being suitable for aerosolization and delivery through an appropriate inhalation device.
The interaction between the formulation and delivery system is another important consideration. Characteristics such as particle size, aerosol performance, device compatibility, dose consistency and deposition within the respiratory tract can all influence the effectiveness of an inhaled medicine.
Nanopharm will contribute its specialized expertise to these areas as Aceso Therapeutics progresses the ACT-101 program toward clinical evaluation.
Nanopharm to Lead Formulation and Device Activities
As part of the collaboration, Nanopharm will undertake formulation development and device assessment activities to help support the clinical development roadmap for ACT-101.
Nanopharm specializes in inhalation and nasal drug product development and provides expertise intended to help companies move complex drug candidates through early development and toward clinical testing.
For an ASO program such as ACT-101, formulation development represents a particularly important step. Nucleic acid-based therapeutics can have different physical and chemical requirements compared with conventional small-molecule medicines. Ensuring that an ASO remains appropriately formulated and can be efficiently delivered through an inhalation system is therefore an essential part of the development process.
The collaboration allows Aceso Therapeutics to access Nanopharm’s technical capabilities while continuing to focus on the advancement of its ASO platform.
Combining Biotechnology and Drug Delivery Expertise
Gemma Budd, General Manager of Nanopharm, an Aptar Pharma company, said cystic fibrosis continues to represent a serious disease with significant unmet needs. She highlighted the opportunity for Nanopharm to apply its formulation and inhalation science expertise to an innovative oligonucleotide program.
The partnership reflects an increasingly important trend in pharmaceutical development: successful development of advanced therapies often depends not only on the therapeutic molecule itself but also on how that molecule is formulated and delivered.
This is particularly relevant for biologics and nucleic acid-based medicines. Molecules such as ASOs, peptides, proteins and other complex therapeutics can present specialized delivery challenges. Conventional drug delivery approaches may not always be appropriate, making specialized formulation and device development an important part of the overall development strategy.
Through Nanopharm, Aptar Pharma can provide expertise focused specifically on inhaled and nasal drug delivery, potentially helping developers address these challenges at earlier stages of development.
Aceso Therapeutics Seeks to Accelerate ACT-101 Development
Thomas Tran, CEO and Co-Founder of Aceso Therapeutics, said the company expects the collaboration with Nanopharm to help accelerate ACT-101 toward clinical evaluation.
According to Tran, the partnership will allow Aceso Therapeutics to explore the potential of delivering ACT-101 directly to the lungs while benefiting from Nanopharm’s experience in inhaled and nasal drug product development.
The broader scientific capabilities available within Aptar Pharma were also identified as an important factor in the collaboration. For Aceso Therapeutics, access to expertise covering formulation, inhalation technologies and drug delivery systems could help support the continued development of its ASO platform.
The collaboration marks a development milestone for the biotechnology company as it advances ACT-101 and evaluates new therapeutic approaches for cystic fibrosis.
Broader Aptar Pharma Biologics Compatibility Program
The work being undertaken for ACT-101 forms part of Aptar Pharma’s broader efforts to understand the compatibility of complex biologic molecules with its pulmonary and nasal drug delivery platforms.
Through its biologics compatibility program, Aptar Pharma is systematically evaluating how different complex therapeutic molecules interact with its delivery technologies across a range of formulations and delivery formats.
The program includes biologic modalities such as nucleic acids, peptides and proteins, as well as nanoparticle-based systems associated with these therapeutic approaches.
This type of evaluation is becoming increasingly relevant as pharmaceutical pipelines expand beyond traditional small-molecule drugs. Advanced therapies can require delivery technologies that are capable of preserving the properties of the active pharmaceutical ingredient while achieving appropriate deposition and dosing.
By investigating these interactions, Aptar Pharma aims to build knowledge that can support the development of next-generation pulmonary and nasal medicines.
Growing Interest in Inhaled Biologic Medicines
The pharmaceutical industry has shown increasing interest in inhaled delivery as a route for administering complex medicines. The lungs provide a large surface area and direct access to the respiratory system, making pulmonary administration attractive for certain therapeutic applications.
However, developing inhaled biologics requires specialized knowledge. The formulation must be engineered for delivery through an inhalation device, and the resulting aerosol must have characteristics appropriate for deposition in the intended region of the respiratory tract.
For nucleic acid medicines, these considerations can become even more complex. ASOs and other genetic medicines may require specialized formulation strategies to protect the active molecule and facilitate effective delivery.
The ACT-101 collaboration provides an example of how drug delivery specialists and biotechnology companies can work together to address these challenges.
Supporting Development From Early Feasibility Through Clinical Stages
Aptar Pharma’s approach extends across different stages of drug development, from early feasibility assessments through clinical development. The collaboration with Aceso Therapeutics is consistent with this model, with Nanopharm providing development expertise as ACT-101 progresses toward potential clinical evaluation.
Early formulation and device assessments can provide valuable information for developers as they establish the characteristics of a future drug product. Identifying formulation or device-related challenges early in development may help inform subsequent development decisions and reduce potential delays as programs advance.
For Aceso Therapeutics, these activities are expected to support its broader clinical development roadmap for ACT-101.
At the same time, the collaboration provides Nanopharm with an opportunity to apply its inhalation expertise to an emerging class of genetic medicines.
Potential Significance for Cystic Fibrosis Treatment Development
The development of ACT-101 comes against the backdrop of continued efforts to expand treatment options for people living with cystic fibrosis.
While advances in CF care have transformed outcomes for many patients, the genetic nature and complexity of the disease mean that new therapeutic strategies remain important. Treatments designed to influence disease mechanisms at the molecular level could potentially complement existing approaches or address populations with limited therapeutic options.
ASO technology is one of several emerging approaches being investigated to modify genetic and molecular pathways underlying disease. The potential to administer such therapies directly to the lungs adds another dimension to their development.
However, ACT-101 remains a development-stage candidate, and additional research and clinical evaluation will be necessary to determine its safety, effectiveness and potential role in cystic fibrosis treatment.
A Strategic Collaboration in Advanced Drug Delivery
The partnership between Aptar Pharma and Aceso Therapeutics illustrates the increasing importance of specialized drug delivery capabilities in the development of innovative medicines.
As pharmaceutical pipelines increasingly include ASOs, RNA-based medicines, proteins, peptides and other complex therapeutics, developers must consider delivery systems alongside the therapeutic molecule itself. Formulation stability, device compatibility, aerosol performance and efficient delivery can all influence whether an advanced medicine can progress successfully toward clinical use.
Through Nanopharm, Aptar Pharma brings specialized expertise in inhaled and nasal drug development to the ACT-101 program. Aceso Therapeutics, meanwhile, contributes its focus on ASO-based therapeutic development for cystic fibrosis.
Together, the companies aim to advance the ACT-101 program toward clinical evaluation while exploring the potential benefits of direct pulmonary delivery.
The collaboration also reinforces Aptar Pharma’s broader commitment to supporting the next generation of inhaled biologic medicines. By combining formulation science, device assessment and delivery technology development, the company is seeking to help pharmaceutical and biotechnology developers translate complex therapeutic concepts into practical drug products.
For Aceso Therapeutics, the partnership represents another step toward advancing ACT-101 and its ASO platform. For the wider cystic fibrosis community, the program reflects continued scientific efforts to explore new approaches that could ultimately address important unmet needs in this rare genetic disease.
About Aceso Therapeutics
Based in Montpellier, France, Aceso Therapeutics builds on over a decade of academic research and continues to develop the 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. Learn more at www.aceso-therapeutics.com.
About Aptar Pharma
Aptar Pharma is part of AptarGroup, Inc., a global leader in drug delivery, dosing and protection technologies, and consumer product dispensing. Aptar partners with the world’s top healthcare and consumer brands to deliver medicines and create exceptional user experiences. Serving diverse markets, from pharmaceutical to beauty to food and beverage, Aptar combines market expertise with proprietary design, engineering and science to develop innovative solutions that help improve lives worldwide.
Nanopharm, an Aptar Pharma company, is a specialist pharmaceutical product development organization focused on orally inhaled and nasal drug products (OINDPs). Nanopharm applies fundamental science and engineering rigor to accelerate the development of complex inhaled therapies, from early feasibility through to clinical supply.