The nanospresso device introduces precise drugs to hospital pharmacies

A new device for providing gene therapy could allow hospital pharmacies to produce personalized nanomedicine to order. This democratized approach to precision medicine, as published, can revolutionize the way hospitals treat rare diseases, even in low -range conditions.

Rare diseases affect millions around the world, but the universal model of drug development leaves patients with small treatment options. Currently, the European research project called Nanospresso is aimed at transferring balance in the grace of patients by increasing access to cheap genes and RNA therapy.

The prototype nanospresso device combines two proven technologiesnucleic acid Therapeutics and lipid nanoparticles-a production unit. Hospital pharmacists could use an unit to prepare sterile, to injecting nanomedicins adapted to the specific genetic abnormalities causing the patient’s condition, ignoring the need for centralized production of drugs.

Most drugs, including gene therapies, are produced in centralized facilities, a model that works on large volume drugs, but not in the case of rare states that affect a small number of patients. High production costs and reduced demand mean that many rare diseases remain untreated.

Leaded by prof. Raymonda Schiffeler from UMC Utrecht, Nanospresso can overthrow these blockers by decentralizing the production of nucleic acid nanomedicins to the local hospital. This can increase access to these therapies, even in low range settings.

Rare diseases together occupy our most important global health challenges in terms of dissemination. Their total influence emphasizes the urgent need for a platform, which allows hospitals to personalize internal drugs and on demand. By moving production to the care point, nanospresso can help introduce life -changing precise drugs within patient range. ”

Prof. Raymond Schiffelers at UMC Utrecht

Rare diseases affect 300 million people around the world-36 million in the EU itself with seven out of 10 people starting in childhood. Although each disease only affects a small population defined by the European Medicine Agency (EMA), because not more than five out of 10,000 people-from 5,000 to 8,000 such diseases.

“We are building a path for precise nanomedicine of nucleic acid,” says the first author of Dr. Marion Esteapé Senti, also from UMC Utrecht. “Nanospresso can revolutionize the way we treat rare diseases by introducing personalized medicine faster. User -friendly, inexpensive device can allow doctors to treat conditions whose conventional approaches cannot manage.”

From blanket to order

Nucleic acid therapies work through a specific focus on genetic instructions responsible for damaged protein production. They are also highly flexible. By changing only the sequence of RNA or DNA used, they can be directed to the treatment of various diseases-from inherited disorders and some cancers, to turn off key proteins in viral or bacterial cells to treat infection. The specificity of the drug can also reduce the probability and severity of side effects.

Financially financed by the Dutch scientific program, nanospresso uses small portable contributions that pharmacists can charge using a combination of lipid ingredients and nucleic acid therapy specific to the patient.

They are then mixed on site using a portable AKNE-AKIN micro-flow to espresso capsules adapted to the tastes of consumers. Lipid nanoparticles in the device protect the load of nucleic acid and help to achieve the right goal in the cell.

The result is precisely formulated, targeted nanomedicine, which is ready to inject. Because the system is small, independent and designed for use by hospital pharmacists, it can be implemented in clinics and hospitals around the world, enabling the creation of the most advanced drugs to order.

Questioning the model

Nanospresso raises questions about how the approach can match current healthcare systems and regulatory frames.

“The current model does not work for millions around the world and we believe that nanospresso will fill this gap in treatment. To this end, we are actively involved in discussions with drug regulators and programmers about how it happened for patients,” says Schiffelers.

The authors cite a historic precedent in pharmacies: Until the 20th century, pharmacists routinely prepared hand -adapted drugs in a process known as “joining”. They also cite the success of the use of similar nucleic acid platforms for manufacturing, for example, MRNA vaccines during the Covid-19 pandemic and say that modern progress in the micro-ranking with the closed system has enabled such a breakthrough as nanospresso.

“Our goal is to increase access to these therapies,” says Esteapé Senti. “Although nanospresso undermines a conventional medical approach, we are also involved with regulatory bodies to ensure compliance with quality, security and effectiveness Standards, tools and security. ”

System prototypes are currently in development, and the team actively deals with technological, scientific, medical and regulatory challenges. Future work will examine how nanospresso can be safely integrated with real healthcare conditions.

Project Nanospreso-NL received funds from the Netherlands of the Science Agenda, the Netherlands Organization of Scientific Research.