The Nanospresso-NL project commenced in February 2023 with a significant kick-off event in Utrecht, marking the start of an ambitious journey towards creating personalized treatments for orphan diseases. This meeting was more than just a starting point—it was a commitment by a consortium of experts to meet complex healthcare needs through innovative nanomedicine.

Nanomedicine for Unmet Clinical Needs

Nanospresso-NL is pioneering a platform unlike any other—a testament to tailored healthcare where the one-size-fits-all approach can’t be used. Our technology is envisioned to craft individualized nucleic-acid based nanomedicines (mRNA – LNP) at the bedside—a beacon of hope for patients grappling with rare genetic disorders and orphan diseases. We’re targeting conditions where the demand for personalized medicine is not just beneficial but critical—for diseases such as methylmalonic acidemia (MMA) and long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHAD), where each patient’s treatment can be as unique as their DNA.

A consortium representing all the key actors involved

The kick-off illuminated the diverse expertise within our consortium, showcasing a collective of innovators and visionaries, among which:

• UMC Nanomedicine, our Coordinator
• Saxion, spearheading technological advances in microfluidic design and nanoengineering.
• Solstice Pharmaceuticals, with its critical pharmaceutical expertise vital to nanoformulation development.
• Karolinska Institute and SINTEF, contributing top-tier scientific research capabilities.
• UMC Hospital Pharmacy and Wilhelmina Children’s Hospital, notably ensuring our solution will fit with real-world clinical settings.
• The Centre for Pharmaceutical Policy and Regulation at Utrecht University, Leiden University, and Erasmus University, notably providing some key aspects of the legal and policy framework.
• And the European Technology Platform on Nanomedicine (ETPN), playing a key role in aligning our scientific objectives with broader European healthcare strategies and ensuring our innovation, communication and dissemination aligns with industry standards.

NWO’s Strategic Support

Reflecting on the reasons behind the project’s endorsement, the Dutch Research Council NWO recognized Nanospresso-NL for its potential to significantly impact both the scientific community and society. The funding was awarded based on the project’s interdisciplinary consortium that brings together diverse knowledge, the inclusion of patient organizations to ensure the relevance of the research, and a work plan characterized by well-integrated, complementary tasks. This support from NWO has been a foundational stone in our progress over the past months.

The Kick-off in Retrospect and a long journey ahead

Looking back at the kick-off, the diverse expertise and enthusiasm of the consortium were evident. Discussions and presentations by partners laid out a roadmap for our technology development and set clear objectives for addressing the needs of patients with orphan diseases.

Since the kick-off, Nanospresso-NL has been diligently working towards developing a platform capable of producing single-dose, patient-specific nanomedicines. Our mission is to tailor treatments to the unique genetic makeup of individuals facing diseases like MMA and LCHAD.

As we launch our website, we stand at the threshold of new possibilities in nanomedicine. With the support of NWO and collaboration among leading institutions and organizations, we are poised to make significant strides in personalized healthcare for orphan diseases. Join us on this path as we share updates, breakthroughs, and the transformative impact of our work.