A new portable gene therapy device could change how hospitals create personalized medicines, according to a recent study. This device, known as NANOSPRESSO, allows pharmacists to mix and produce tailored treatments on demand, especially for patients with rare diseases.
Rare diseases impact over 300 million people worldwide, including 36 million in the European Union. Unfortunately, these conditions often get overlooked because they affect small numbers of people, making it hard for companies to justify the high costs of developing new drugs.
Typically, medicines are manufactured in large factories and then distributed to hospitals. However, for rare diseases, the demand often isn’t enough to support bulk production. The NANOSPRESSO project aims to solve this problem by enabling pharmacists to create medications as needed. A pharmacist would simply load ingredients, like genetic materials and fats, into a small cartridge. The device then mixes these components precisely, allowing for the creation of targeted treatments that can be injected directly into patients.
Professor Raymond Schiffelers, who led the project, emphasized the urgent need for affordable personalized medicine in hospitals. He believes that by moving production to the point of care, NANOSPRESSO can help make essential treatments accessible to more patients.
Despite its potential, NANOSPRESSO still faces significant challenges before it can be used in hospitals. The medications produced must meet strict safety and quality standards, and regulatory bodies will need to establish guidelines for approving and monitoring these individualized treatments.
Dr. Mariona Estapé Senti, another author of the study, noted that this device could revolutionize how we treat rare diseases, speeding up access to personalized medicine. She pointed out that similar technologies have already shown success, such as the use of mRNA platforms for COVID-19 vaccines. The advancements in microfluidics technology have made projects like NANOSPRESSO possible.
Historically, pharmacists prepared custom medicines by hand until the 20th century, and this new device could bring that practice back into modern healthcare. As the demand for personalized treatments grows, innovations like NANOSPRESSO could play a crucial role in addressing the needs of patients with rare conditions.