A revolutionary portable gene therapy device could transform how hospitals treat rare diseases by enabling on-demand production of personalized nanomedicines, according to research published in Frontiers in Science. The NANOSPRESSO project, led by Professor Raymond Schiffelers at UMC Utrecht, addresses a critical gap in treating the 300 million people worldwide affected by rare diseases—36 million in the EU alone.
Addressing the Rare Disease Treatment Gap
Rare diseases collectively represent one of the world's top global health challenges, with seven in 10 cases beginning in childhood. Despite affecting 5,000 to 8,000 different conditions, each disease impacts small populations—defined by the European Medicines Agency as no more than five in 10,000 people. This low patient volume makes traditional centralized drug manufacturing economically unfeasible for pharmaceutical companies.
"Rare diseases collectively rank among our top global health challenges in terms of prevalence," says Schiffelers. "Their combined impact underscores the urgent need for a platform that lets hospitals personalize medicines in-house and on-demand affordably."
Revolutionary Technology Platform
The NANOSPRESSO device combines two proven technologies—nucleic acid therapeutics and lipid nanoparticles—into a portable manufacturing unit. Hospital pharmacists load small cartridges with lipid components and patient-specific nucleic acid therapies, which are then mixed using a portable microfluidic device similar to espresso capsules tailored to consumers' tastes.
The system produces precisely formulated, targeted nanomedicines ready for injection. The lipid nanoparticles protect the nucleic acid cargo and help it reach the correct cellular target. By changing only the RNA or DNA sequence, the therapy can be directed to treat different diseases—from inherited disorders and certain cancers to infections by disabling crucial proteins in viral or bacterial cells.
"We're building a path to precision nucleic acid nanomedicine," says first author Dr. Mariona Estapé Senti from UMC Utrecht. "NANOSPRESSO could revolutionize the way we treat rare diseases by bringing personalized medicine to more patients, faster. The user-friendly, affordable device could let medics treat conditions that conventional approaches can't manage."
Decentralizing Medicine Production
The approach represents a fundamental shift from the current model where medicines are manufactured in centralized facilities and shipped to hospitals. This traditional system works for high-volume drugs but fails for rare conditions due to high manufacturing costs and limited demand, leaving many rare diseases untreated.
"By shifting production to the point of care, NANOSPRESSO could help bring life-changing precision medicines within reach of patients," Schiffelers explains. The portable, self-contained system designed for hospital pharmacists could be deployed in clinics and hospitals worldwide, including low-resource settings.
Historical Precedent and Modern Innovation
The researchers cite historical precedent for pharmacy-based medicine production—until the 20th century, pharmacists routinely prepared tailored medicines by hand through a process called 'compounding.' They also reference the successful use of similar nucleic acid platforms to produce mRNA vaccines during the COVID-19 pandemic, demonstrating that modern advancements in closed-system microfluidics have enabled such breakthroughs.
Regulatory and Implementation Challenges
Despite its promise, NANOSPRESSO faces significant hurdles before clinical implementation. The personalized medicines must meet strict safety and quality standards, and regulators need to develop new frameworks for approving and monitoring individualized treatments.
"The current model doesn't work for millions around the world, and we believe NANOSPRESSO will fill that treatment gap," says Schiffelers. "To that end, we're actively engaging in discussions with regulators and drug developers about how to make this happen for patients."
Estapé Senti adds, "Although NANOSPRESSO challenges the conventional medical approach, we are also engaging with regulatory authorities to ensure compliance with quality, safety, and efficacy standards, tools, and safeguards."
The team is currently developing prototypes and addressing the technological, scientific, medical, and regulatory challenges involved. Future work will explore safe integration of NANOSPRESSO into real-world healthcare settings. The project received funding from the Netherlands Science Agenda and the Netherlands Organization for Scientific Research.
