How gene therapy device could create personalised medicines for rare diseases

A new portable gene therapy device could allow hospital pharmacies to create personalised medicines on demand, a new study says.

Rare diseases affect more than 300 million people worldwide – 36 million in the EU alone – but they are often overlooked due to low patient numbers and the high costs associated with drug development.

Most medicines are made in factories and shipped to hospitals, but for rare conditions there often aren’t enough patients for companies to justify developing medicines in bulk.

Create medicines on demand

But the NANOSPRESSO project could allow pharmacists to create medicines for them on demand, according to a paper published in Frontiers in Science.

A pharmacist using the machine would put ingredients (such as genetic material and fats) into a small cartridge, which the device then mixes together in a very precise way. A tiny, targeted medicine can then be injected into the patient.

Prof Raymond Schiffelers, who led the project, said there is an “urgent need” for a way to make personalised medicines in hospitals and on demand in an affordable way.

“By shifting production to the point of care, NANOSPRESSO could help bring life-changing precision medicines within reach of patients.”

However, NANOSPRESSO faces significant hurdles before it can be seen in a healthcare setting anytime soon.

The medicines it creates will need to meet strict safety and quality standards, and regulators will need to decide how they will approve and monitor individualised treatments.

“NANOSPRESSO could revolutionise the way we treat rare diseases by bringing personalised medicine to more patients, faster,” says study author Dr Mariona Estapé Senti.

“The user-friendly, affordable device could let medics treat conditions that conventional approaches can’t manage.”

The study cites a historical precedent for pharmacies producing medicines – until the 20th century, pharmacists routinely prepared tailored medicines by hand.

They also cite the success of using similar nucleic acid platforms to produce mRNA vaccines during the COVID-19 pandemic, and say that modern advancements in closed-system microfluidics have enabled such breakthroughs such as this.