Key points from article :
A new study published in Frontiers in Science introduces a groundbreaking device called NANOSPRESSO, designed to enable hospital pharmacies to produce personalised gene therapies on demand. Led by Prof. Raymond Schiffelers, the project aims to address a major gap in treatment for the 300 million people worldwide living with rare diseases, many of whom are left without effective therapies due to the low number of patients and the high costs of large-scale drug manufacturing.
The device works by mixing genetic material and lipids inside a small cartridge, producing tiny, precisely tailored medicines that can be injected directly into the patient. Unlike traditional manufacturing, which happens in large factories, NANOSPRESSO allows for point-of-care production—meaning treatments can be made right in the hospital for individual patients. This could significantly speed up access to life-changing medicines for those with conditions that are currently underserved.
While the technology holds promise, it faces several regulatory and quality assurance hurdles. Authorities will need to create frameworks to monitor these on-demand therapies, ensuring they meet strict safety and efficacy standards. Still, Dr. Mariona Estapé Senti, a co-author of the study, believes NANOSPRESSO has the potential to revolutionise rare disease treatment, citing the historical precedent of pharmacy-compounded medicines and the recent success of mRNA vaccines during the COVID-19 pandemic as evidence that such a shift is possible.
Overall, this portable device reflects a broader trend toward personalised, decentralised medicine—offering hope for faster, more equitable treatments in the future.
