Pre-clinical characterisation of oligonucleotide-based drug delivery systems for gene therapies
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Date
2020-07-16
Authors
Mirón-Barroso, Sofía
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Abstract
Cancer is a leading cause of death worldwide and the need for more effective and specific treatments is evident. Oligonucleotide therapeutics, such as siRNAs, have shown great promise in the treatment of cancer. Nevertheless, the main constraint preventing these therapeutics from being in the clinic is the lack of safe and effective drug delivery systems.
RNA nanotechnology is an emerging field in which RNA strands are computationally designed to self-assemble into RNA nanoparticles with predictable sizes and shapes. Sixfold Bioscience Ltd. aims to develop Programmable Oligonucleotide Drug Delivery Systems (PODS) that can selectively target cancer cells and efficiently deliver siRNA therapeutics. Here we show the pre-clinical assessment of PODS. Physicochemical characterisation and self-assembly properties were evaluated, the safety profile was assessed and functionalization with different moieties such as endosomal escape molecules, fluorophores, and therapeutic moieties was attempted.
PODS are promising siRNA delivery systems since they appear to be stable in physiological conditions and to be safe in relevant cell lines. Functionalization using click chemistry strategies is feasible but further optimisation is needed. Successful development of this therapeutic delivery system will provide a solution to change the way cancer is currently treated.
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Keywords
Gene therapy, siRNA delivery, Nanomedicine