Gene Therapy Delivery Device Could Let Hospitals Create Personalized Nanomedicines On-Demand
The new device aims to democratize precision medicine, expanding the ability of low-resource hospitals to treat rare diseases.
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A compact new device could change how gene and RNA therapies are made and delivered.
The portable microfluidic system, known as NANOSPRESSO, could allow hospital pharmacists to formulate nanomedicines tailored to individual patients on demand. By localizing production at the point of care, the NANOSPRESSO device could extend precision treatments to more people, including those in low-resource settings.
Described in Frontiers in Science, the prototype device combines two established technologies: nucleic acid therapeutics and lipid nanoparticles. This configuration allows the system to package genetic material into injectable nanomedicines that directly target the molecular cause of a patient's disease.
A decentralized approach for rare conditions
Traditional models of drug manufacturing are centralized, a structure that suits common diseases but limits treatment access for rare conditions. These illnesses, while individually uncommon, affect around 300 million people globally. Many remain untreated due to the high cost and complexity of developing bespoke drugs for small patient populations.
The NANOSPRESSO project, led by researchers at the University Medical Center Utrecht, offers an alternative. It enables production of nucleic acid nanomedicines in small batches directly within hospitals. This not only sidesteps the need for industrial-scale facilities but also reduces logistical barriers, potentially opening the door to broader access for patients with rare or underserved conditions.
From concept to cartridge
The device operates using single-use cartridges preloaded with lipid components. Pharmacists can add nucleic acids that have been customized to the genetic mutation causing a patient's condition. These are then processed on-site using a microfluidic mixing system, creating lipid nanoparticles that encapsulate the genetic material. These particles improve delivery by protecting the therapeutic RNA or DNA and guiding it to the intended site of action within cells.
Nucleic acid therapeutics
These therapies use strands of RNA or DNA to modify genetic activity in cells. They can block faulty instructions, supplement missing ones, or interfere with harmful proteins. Unlike traditional drugs, they are designed at the molecular level for high specificity.
Lipid nanoparticles
Tiny, fat-based particles used to protect and deliver RNA or DNA into cells. They help the genetic material reach its target in the body and are essential for the success of many nucleic acid-based treatments, including some vaccines.
Such flexibility allows the same platform to target a range of conditions by simply altering the nucleic acid sequence. Potential targets include inherited disorders, certain cancers, and infectious diseases.
Integrating technology into care
The NANOSPRESSO device is designed to be compact and user-friendly, with the aim of enabling use in clinical settings without the need for specialist training in drug manufacturing. The system's closed design also helps to maintain sterility, a critical requirement for injectable products.
The project team is currently refining the prototype and working through regulatory considerations. Key questions include how this decentralized model will be integrated into existing healthcare systems, and how to ensure that products meet established safety and quality standards. The researchers are engaging with regulators and stakeholders to address these issues.
This approach draws on a long history of in-hospital compounding – where pharmacists prepare medicines for individual patients – as well as recent advances in nucleic acid delivery systems, such as those used in mRNA COVID-19 vaccines.
Next steps
Prototypes of the NANOSPRESSO system are in development with support from the Netherlands Science Agenda and the Netherlands Organization for Scientific Research. Future work will evaluate the system’s performance, safety, and compatibility with real-world clinical workflows.
By allowing pharmacists to create therapies tailored to the genetic profile of each patient, this decentralized technology could broaden access to precision treatments that are otherwise economically unviable under current production models.
“Rare diseases collectively rank among our top global health challenges in terms of prevalence. Their combined impact underscores the urgent need for a platform that lets hospitals personalize medicines in-house and on-demand affordably,” said project leader Prof Raymond Schiffelers, of UMC Utrecht. “By shifting production to the point of care, NANOSPRESSO could help bring life-changing precision medicines within reach of patients.”
Reference: Estapé Senti M, Ceccaldi A, Luciani M, et al. NANOSPRESSO: toward personalized, locally produced nucleic acid nanomedicines. Front Sci. 2025;3:1458636. doi: 10.3389/fsci.2025.1458636
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