Local preparation of high-quality, personalised nucleic acid nanomedicines
Funding: NWA-ORC
Project duration:
Rare diseases are a condition that affects not more than 1 person per 2000 in the European population. There are between 5 000 and 8 000 rare diseases, currently affect 3.5% – 5.9% of the worldwide population. Rare diseases have significant impact as they are often chronic, commonly are progressive and can be life-threatening. The majority of orphan diseases affect life expectancy and lead to physical, emotional and/or psychosocial limitations with a wide range of disabilities when left untreated. Unfortunately, there is a lack of effective personalized treatment options for many patients with rare diseases which can be explained by challenging R&D combined with market failure. Developing medicines intended for small numbers of patients has little commercial incentive under normal market conditions.
Our consortium has all the capacity to break through this vicious circle. 72% of orphan diseases are genetic and the cause can be relatively easily addressed by compensating for the defective gene. Administering mRNA for the defective of missing protein is for instance an effective cure for many rare diseases, like MMA. mRNA and gene therapy technologies belong to the advanced therapy medicinal products (ATMPs). However, over the last decade, only 15 ATMPs have received approval in Europe. That is a stark contrast with the fact that there have been over 500 clinical trials using ATMPs in the EU since 2009. Unfortunately, the market is confronted with high development costs and long regulatory trajectories.
NANOSPRESSO-NL aims to research, develop and implement a technology for decentralized and small-scale production of high quality and affordable nucleic acid nanomedicines for the treatment of orphan diseases. We will deliver proof-of-concept by preparing our model NANOSPRESSO-NL Therapeutic (NNTx). NANOSPRESSO-NL has three main objectives addressed in three research lines:
- Technical development of a device that can reproducibly manufacture high quality personalised nucleic acid therapeutics to be operated in a hospital pharmacy.
- Validation, demonstration and operational feasibility of the technology in preclinical and clinical setting.
- Creation of the regulatory, legal and health economic conditions for the translation of NANOSPRESSO-NL.
The Applied Nanotechnology group is mainly involved in the technical development of the compounding technology. This comprises the selection, quality control and compatibility of the active pharmaceutical ingredient (API) and excipients for cartridge-based microfluidic self-assembly. Further, we work on designing the cartridge prototypes with special focus on integration with blisters, microfluidics and the production machine.
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