NANOSPRESSO is transforming the way we deliver advanced treatments for rare diseases by making them right at the hospital, tailored to each patient. Using technologies like nanomedicine, mRNA, and microfluidics, NANOSPRESSO ensures that personalised medicines are produced on-site, quickly and affordably, bringing life-saving treatments directly to those who need them most.
Develop safer, more effective delivery methods for nucleic acid therapies, such as mRNA encapsulated in lipid nanoparticles (LNPs).
Create microfluidic platforms capable of precision mixing for on-site production, allowing for rapid assembly of treatment cartridges.
Deploy rapid, patient-specific treatments for rare diseases directly at the point of care, reducing the time between diagnosis and treatment.
Aim to shift pharmaceutical production from centralised facilities to local, decentralised hubs using automated, scalable microfluidic systems.
Novel platforms like microfluidic precision mixing bridge the gap between laboratory research and clinical application, enabling effective nanomedicine delivery for rare diseases or RNA therapies. This fosters a community effect, allowing specialists to collaborate effectively.
Provide tools for on-site production of LNP-based medicines tailored to individual patient needs, reducing reliance on mass-produced drugs.
Encourage new partnerships focused on localised production, supporting the growth of local biotech industries capable of integrating decentralised technologies.
NANOSPRESSO aims to engage with complex regulatory frameworks for nano-enabled cell and gene therapies, and compounding ATMPs. It aims not only to meet compliance but also to contribute to regulatory science, benefiting other stakeholders interested in these fields.
Localization of nucleic acid therapies aims at reducing manufacturing and scale-up costs, avoiding logistical challenges like cold chain requirements while ensuring consistent quality. This approach can attract pharmaceutical interest by offering a cost-effective solution for rare diseases.
Create new jobs in biotechnology, healthcare, and microfluidics; support local research institutions in developing customized treatments.
Proof of concept for integrated microfluidic production of mRNA therapies within cartridge-based systems, establishing partnerships.
Key Actions: Establish prototypes, validate LNP encapsulation, form partnerships with hospitals and technology providers.
Initiation and progress of clinical trials, demonstrating efficacy and safety, and early regulatory engagement.
Key Actions: Start Phase 1 and 2 trials, generate safety and efficacy data, collaborate with regulatory authorities.
Completion of clinical trials, regulatory pathway establishment, and adoption of hospital-based production.
Key Actions: Complete Phase 3 trials, secure regulatory approval, implement decentralised production.
NANOSPRESSO aims at democratising access to cutting-edge nucleic acid therapies, reducing both geographical and financial barriers. This aligns with the Dutch Research Agenda (NWA) goals, emphasising societal challenges. For details, see NWO's Cluster Questions.
The NANOSPRESSO-NL project was granted for several reasons:
The high potential to create scientific and societal breakthroughs.
The high-quality consortium that is interdisciplinary and knowledge chain wide.
The involvement of patient organisations in the consortium.
The high quality of the workplan with well-fitting complementary work packages.
NWA methodology guides the social impact of NANOSPRESSO. Research questions were derived from a public questionnaire identifying healthcare disparities. By involving patient advocacy groups and healthcare providers, Nanospresso ensures outcomes align with public needs.
Step 1: Formulate research questions based on societal input.
Step 2: Develop microfluidic technology for on-site production, enabling local healthcare providers.
Step 3: Maintain engagement with patient communities to refine treatment protocols.
NANOSPRESSO aims at reducing health disparities by enabling hospitals in low-resource settings to produce advanced treatments. This decentralisation ensures that underserved regions have access to nucleic acid therapies.
NWO emphasises projects that provide measurable social benefits. NANOSPRESSO commits to indicators of success, like increased patient access, improved outcomes, and enhanced quality of life.
NANOSPRESSO is funded by the Dutch Research Agenda (NWA) ORC 2020/21 program. For more information about our partners, funding, and legal notices, please visit our contact page and follow us on social media.