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.
The European definition of an orphan disease is synonym to a rare disease; 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, most of them (72%) have a genetic basis. Orphan 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. Moreover, 70% have a pediatric onset.
Unfortunately, there is a lack of effective personalized treatment options for many patients with (ultra-)orphan 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 and the challenging approval and reimbursement landscape also make it hard to make a profit.
As 72% of orphan diseases are genetic, administering mRNA for the defective of missing protein is for instance an effective cure for many rare diseases. mRNA and gene therapy technologies belong to the advanced therapy medicinal products (ATMPs) in the medical field and hold the promise to cure acute fatal diseases as well as chronic illness, however, the market is confronted with high development costs and long regulatory trajectories. This makes ultra-orphan diseases challenging for return-on-investment.
Therefore, 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. The technology that will be researched and developed in NANOSPRESSO-NL will enable local production of nucleic acid therapeutics based on the specific needs of an individual patient. This personalized approach especially improves the quality of care for patients with (ultra)orphan diseases. The availability of affordable and effective therapies for this group of patients is regularly threatened by high drug development costs and market failure, resulting in high prices or stopping development prematurely. This is especially true for patients that live in developing countries, and the novel production method could improve the availability of advanced nucleic acid therapeutics in such countries by facilitating their on-site production.
The Nanospresso-NL project is led by prof. Raymond Schiffelers (CDL Research, UMCU). The total (international) consortium consists of renowned partners which have their expertise in the medical, pharmaceutical, nanomedicine, technical, societal and regulatory field.
The research group Applied Nanotechnology (Saxion) together with the University of Twente and the company Solstice will focus on developing and validating the technological part (‘Machine’). An easy-to-use and cheap microfluidic cartridge will be designed and developed that contains all the components (mRNA, excipients, microfluidic chip, …) to enable rapid mixing and harvesting of the sterile end-product (i.e. the nanomedicine), which can then be administered to the patient. Research question will focus on analyzing the stability of the solutions in the blisters (mRNA, excipients) as well as designing and fabricating different microfluidic devices to know more about the mechanisms by which liposomes are formed and obtain knowledge what the effect and mutational relation is of the key parameters (such as channel size, flow rates and lipid concentration), that determine liposome size and size dispersity.
- Biomedical Lab Research (for the core task Research and Innovation)
Interested, please send your motivation letter and CV before the 4th of July 2022 to
Floor Wolbers, senior researcher Research Group Applied Nanotechnology
Project leader Nanospresso-NL