A new portable gene therapy device could allow hospital pharmacies to create personalised medicines on demand for rare disease patients, a new study suggests.
The technology could reduce reliance on centralised drug manufacturing by enabling pharmacists to prepare treatments directly at the bedside.
This approach may address the challenge of treating rare diseases, which affect more than 300 million people worldwide — including 36 million in the EU — but are often overlooked due to small patient populations and high development costs.
The system combines genetic material — such as DNA or RNA, which instructs cells to produce therapeutic proteins — with fats in a precisely controlled process to produce injectable treatments tailored to individuals.
Led by professor Raymond Schiffelers, the NANOSPRESSO project uses microfluidics — a technique that handles tiny volumes of fluid through microscopic channels — to mix ingredients inside a sealed cartridge. The resulting nanometre-scale particles can then be administered directly to patients.
“By shifting production to the point of care, NANOSPRESSO could help bring life-changing precision medicines within reach of patients,” said professor Schiffelers, who highlighted an “urgent need” for affordable methods to create patient-specific treatments in hospitals.
Most medicines are manufactured in bulk at central facilities and shipped to hospitals, but this is rarely viable for rare conditions where the number of patients is too small to justify large-scale production.
Study contributor Dr Mariona Estapé Senti said: “NANOSPRESSO could revolutionise the way we treat rare diseases by bringing personalised medicine to more patients, faster.
The user-friendly, affordable device could let medics treat conditions that conventional approaches can’t manage.”
The study draws historical comparisons, noting that until the 20th century, pharmacists routinely prepared medicines by hand for individual patients before industrial manufacturing became standard.
The authors also point to the successful use of similar nucleic acid platforms in developing mRNA vaccines during the COVID-19 pandemic as evidence that this kind of gene-based technology can be scaled effectively.
However, the researchers acknowledge that regulatory challenges remain.
Any medicines produced would need to meet strict safety and quality standards, and new frameworks may be needed to govern therapies made on site for individual patients.
The device builds on closed-system microfluidics, which keeps the formulation process sealed from outside contamination, supporting safer preparation of gene-based medicines in clinical settings.
