In a not-so-distant future, pharmacists at a local hospital may be able to manufacture and deliver nanomedicines tailored to the specific needs of patients with a rare disease diagnosis. According to a paper published today in Frontiers in Science, this could be possible thanks to a new device designed to boost access to gene therapy at a fraction of the cost.
The device is called NANOSPRESSO, a name inspired by the convenience and customization that espresso capsule machines have brought to coffee enthusiasts. Its aim is to subvert the current one-size-fits-all model of drug manufacturing; while efficient for high-volume medicines such as vaccines and painkillers, this model fails when it comes to rare diseases, where high development costs and a small market size often result in extreme pricing and provide low incentives for drug development.
“The current model doesn’t work for millions around the world, and we believe NANOSPRESSO will fill that treatment gap,” said Raymond Schiffelers, PhD, professor at the University Medical Center (UMC) Utrecht and senior author of the study. “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. By shifting production to the point of care, we could help bring life-changing precision medicines within reach of patients.”
Currently in the prototype phase, the device consists of small, portable cartridges that can be loaded with nucleic acids and lipids. These are mixed on-site using microfluidics technology, resulting in the self-assembly of lipid nanoparticles that protect the nucleic acid cargo and deliver it to the cellular target once administered to a patient. In a hospital setting, this device would allow pharmacists to prepare sterile, injectable medicines containing a DNA or RNA sequence tailored to the specific genetic abnormality causing a patient’s condition.
“We’re building a path to precision nucleic acid nanomedicine,” said Mariona Estapé Senti, PhD, postdoctoral researcher at UMC Utrecht and first author of the study. “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.”
The developers are part of an international consortium seeking to tackle the complex challenge of treating orphan diseases and safely integrating a new model of care into a real world clinical setting. “We’re actively engaging in discussions with regulators and drug developers about how to make this happen for patients,” said Schiffelers.
In favor of this novel approach to bespoke gene therapy manufacturing, Schiffelers and colleagues cite historical precedents of “compounding,” a practice common until the 20th century where pharmacists regularly prepared tailored prescription medicines by hand. The researchers also draw similarities between their platform and those used during the COVID-19 pandemic to produce messenger RNA vaccines, with the added benefit that local production would bypass the need for complex logistics of low temperature transport and storage. This decentralized manufacturing model would also follow in the steps of CAR-T cell therapy manufacturing, which is increasingly transitioning from centralized to local production.
In the future, the technology behind NANOSPRESSO could find applications in other fields beyond rare disease. The research team sees great promise in precision oncology applications, where therapeutic interventions increasingly seek to target by the unique genetic makeup of a patient’s tumor. On-demand mRNA and gene therapy manufacturing could also offer the speed necessary to respond to the emergence of local variants of pathogens such as coronaviruses or Ebola.
