NANOSPRESSO-NL: Local preparation of high-quality, personalized nucleic acid nanomedicines

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.