The Nanospresso device brings precision medicine to hospital pharmacies

The Nanospresso device brings precision medicine to hospital pharmacies

A new gene therapy delivery device could lead to hospital pharmacies making personalized nanomedicines available to order. This democratized approach to precision medicine, as published inLimits in sciencecould revolutionize how hospitals treat rare diseases, even in low-resource settings.

Rare diseases affect millions worldwide, but the one-size-fits-all drug development model leaves patients with few treatment options. Now a European research project called Nanospresso aims to tip the balance in patients' favor by increasing access to low-cost tailored genetics and RNA therapies.

The prototype Nanospresso device combines two proven technologies-nucleic acid therapeutics and lipid nanoparticles-into a portable manufacturing unit. Hospital pharmacists could use the unit to prepare sterile, injectable nanomedicines tailored to the specific genetic abnormality causing the patient's condition, circumventing the need for centralized drug production.

Most drugs, including gene therapies, are manufactured in centralized facilities, a model that works for high-volume drugs but not for rare diseases that each affect a small number of patients. High manufacturing costs and limited demand mean that many rare diseases remain untreated.

Led by Prof. Raymond Schiffers at UMC Utrecht, Nanospresso could undermine these blockers by decentralizing the production of nucleic acid nanomedicines to the local hospital. This could increase access to these therapies even in low-resource settings.

Rare diseases are part of our global global health challenges in terms of prevalence. Their combined impact underscores the urgent need for a platform that allows hospitals to personalize medications in-house and on-demand. By moving production to the point of care, Nanospresso could help bring life-changing precision medicines within patients' reach. “

Prof. Raymond Schiffer at UMC Utrecht

Rare diseases affect 300 million people worldwide - 36 million in the EU alone - with seven in ten starting in childhood. Although each disease only affects a small population described by the European Medicines Agency (EMA) as no more than five in 10,000 people, there are between 5,000 and 8,000 such diseases.

“We are building a path to precision nucleic acid nanomedicine,” says first author Dr. Mariona Estapé Senti, also from UMC Utrecht. "Nanospresso could revolutionize the way we treat rare diseases by bringing personalized medicine to more patients. The easy-to-use, affordable device could enable medical professionals to treat diseases that conventional approaches cannot."

From ceiling to tailor-made

Nucleic acid therapies specifically target the genetic instructions responsible for defective protein production. They are also very adaptable. By only changing the sequence of the RNA or DNA used, they can be instructed to treat various diseases and certain cancers, to deactivate important proteins in viral or bacterial cells to treat infections. The specificity of the medicine can also reduce the likelihood and severity of side effects.

Funded by the Netherlands Science Agenda, Nanospresso uses small portable cartridges that pharmacists can load with a combination of lipid components and patient-specific nucleic acid therapies.

These are then mixed on-site using a portable microfluidic device to create espresso pods tailored to the consumer's taste. The lipid nanoparticles in the device protect the nucleic acid charge and thus help to reach the correct target in the cell.

The result is a precisely formulated, targeted nanomedicine that is ready for injection. Because the system is small, self-contained and designed by hospital prosthetists, it could be used in clinics and hospitals around the world, allowing the most advanced medicines to be used in hospital settings.

Challenge the model

Nanospresso raises questions about how the approach fits into current healthcare systems and regulatory frameworks.

"The current model is not working for millions around the world, and we believe Nanospresso will fill this treatment gap. To this end, we are actively engaged in discussions with regulators and drug developers about how to do this for patients," says Schifflers.

The authors cite historical precedents in pharmacies: By the 20th century, pharmacists routinely prepared customized medications in a process known as “compounding.” They also cite success in using similar nucleic acid platforms to generate mRNA vaccines, for example, during the Covid-19 pandemic, and say modern advances in closed-system microfluidics have enabled such breakthroughs as Nanospresso.

“Our goal is to increase access to these therapies,” says Estapé Senti. “Although Nanospresso challenges the conventional medical approach, we also engage with regulatory authorities to ensure compliance with quality, safety and effectiveness standards, tools and protections.”

The system is currently in prototype form and the team is actively addressing the technological, scientific, medical and regulatory challenges. Future work will explore how Nanospresso could be safely integrated into real-world healthcare settings.

The Nanospresso-NL project received funding from the Netherlands Science Agenda, Netherlands organization for scientific research.

Sources: