Local preparation of high-quality, personalised nucleic acid nanomedicines
Orphan 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, accounting for 3.5% – 5.9% of the worldwide population. Orphan diseases have significant impact as they are often chronic, progressive and life-threatening. The majority of rare diseases influence life expectancy and lead to physical, emotional and 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 relevance under normal market conditions.
The majority (72%) of orphan diseases is genetic and the cause can be relatively easily addressed by compensating for the defective gene. Administering mRNA for the defective of missing protein is an effective treatment for many rare diseases with a metabolic origin. This mRNA needs to be packaged within lipid nanoparticles (LNPs) to protect the mRNA and to target the mRNA to the right organ, mainly the liver.
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 research group Applied Nanotechnology focuses on designing and fabricating a microfluidic chip and cartridge to generate LNPs which serve as the nanomedicine to treat rare diseases. Moreover, the generated LNPs will be characterized for their morphology and performance. As an intern you will focus on the design and fabrication of microfluidic chips to generate monodisperse LNPs with a size < 100 nm. Different key parameters will be tested (channel dimension, flow rate, lipid and mRNA concentration) to analyse how they effect the morphology of LNPs. Moreover, experiments will be performed to determine the amount of encapsulated mRNA and the transfection efficacy within cells.
Project duration:
Spring 24/25 (February 2025 – July 2025)
Educational programs:
– Biomedical Lab Research (BML)
– Chemistry (CH)
Interested, please send your motivation letter and CV before the 8th of December 2024 to Floor Wolbers (f.wolbers@saxion.nl)