Dear ETPN members, dear colleagues, we have received the excellent news that the project proposal NANOSPRESSO-NL will be funded by the Dutch Research Council with a 9M€ budget for the next five years. NANOSPRESSO-NL will develop a disruptive technology to make lipid nanoparticles for the delivery of RNA and DNA at the bedside, initially for orphan diseases. The ETPN will be participating in this great innovative Nanomedicine project, notably to optimize its communication and dissemination activities.
Concept of Nanospresso-NL
Nucleic acid therapeutics are revolutionizing medicine. However, manufacture, scale-up, distribution and stability are the new limiting factors. NANOSPRESSO-NL aims to lay the groundwork for personalized and local production in a hospital pharmacy of nucleic acid therapeutics. Most of the current state-of-the-art nucleic acid therapeutics, for example the clinically used COVID-19 mRNA vaccines, are prepared by careful and controlled mixing of excipients and nucleic acid that electrostatically and hydrophobically complex into the final nanomedicine formulation. Since all therapeutic nucleic acids are essentially the same molecule -only with different lengths and sequences- the production process to turn them into nanomedicines is uniquely qualified for platform design. As a result, quality control can be the production process rather than the end product, similar to a popular method of local high-quality espresso making. This would allow rapid affordable local development of personalized therapeutics without distribution and stability issues.
NANOSPRESSO-NL specifically addresses the current mismatch between personalized therapeutic strategies and industrial centralized large-scale manufacture of medicines. This large-scale manufacture is associated with significant investments which makes the business case for many orphan diseases and individualized therapies challenging.
NANOSPRESSO-NL aims to create a machine that allows single dose production of nucleic acid nanomedicines in a local hospital pharmacy near the patient to enable individualized therapeutics to be developed (such as mRNAs encoding neo-epitopes for immune therapy of cancer) and orphan diseases (such as enzyme replacement therapy for hereditary metabolic disorders) in a cost-efficient manner. This is complemented by a comprehensive analysis of the legal and regulatory framework in which such a production method will operate in addition to stakeholders’ landscape analysis at the national and European level.
Why NANOSPRESSO-NL is unique?
- accelerate translation of nucleic acid therapeutics to the clinic by addressing the legal, practical and technical challenges currently impeding the commercialisation of nucleic acid therapeutics.
- improve treatment options for patients with (ultra-)orphan diseases. Compounding of personalised therapies for individual patients is effective and efficient, mitigating limitations for therapies that have an unprofitable business case or suffer from monopolistic value-based pricing strategies.
- create an ecosystem based in NL for the clinical development for nucleic acid nanomedicine, reinforcing the European leadership in the field. The knowledge generated can be utilised in many therapeutic areas.
NANOSPRESSO-NL will be coordinated by Raymond Schiffelers, Professor of Nanomedicine at UMC Utrecht, and chairman of the ETPN. The consortium combines four academic knowledge institutions, four research institutions with applied and societal focus, two societal stakeholder groups, and seven co-funding companies to cover the full knowledge chain and key stakeholders.
- Universitair Medisch Centrum Utrecht
- Universiteit Twente
- Saxion Hogeschool
- Leids Universitair Medisch Centrum
- Princes Máxima Centrum
- Universiteit Utrecht
- Universiteit Leiden
- Solstice Pharmaceuticals B.V.
- CelluTx LLC USA
- Anjarium Biosciences Switzerland
- siTOOLs Biotech GmbH Germany
- Lipoid GmbH Germany
- Patiëntenvereniging Stofwisselingsziekten
- Instituut Verantwoord Medicijngebruik
- Karolinska Institutet, SINTEF
- Val d’Hebron Hospital
- National University Singapore
- European Technology Platform Nanomedicine
- Friedrich-Alexander- Universität Erlangen-Nürnberg
We look forward very much to starting the collaboration with these great partners to make NANOSPRESSO-NL a new milestone revolutionizing the way we think and make nanomedicine useful and accessible to patients in Europe. Stay tuned!