New findings strengthen RNAi cancer fight

By Kirsty Barnes

- Last updated on GMT

Related tags: Rna interference

Scientists at Atugen, a German-based firm, have successfully used a
new RNA interference (RNAi) technology to reduce tumour growth and
spread, according to new research published.

RNAi technology is hailed as the greatest medical advancement since antibiotics, with the potential to cut out the genes that cause diseases such as cancer.

Atugen is a subsidiary of European biopharma company SR Pharma - one of the key players in the development of this cutting-edge technology.

RNAi technology has so far been used to silence several of the critical gene products involved in carcinogenesis, which has generated significant antiproliferative and/or proapoptotic effects in many cell-culture systems and preclinical animal models. However, a major obstacle for translating this RNAi technology from a mere research tool into a realistic therapeutic strategy remains the efficient delivery of these small molecules to the targeted cell type in vivo​.

Atugen's scientists now claim that using its new siRNA-lipoplex (AtuPLEX) technology, they have uncovered the requirement of siRNA formulation to allow functional cellular uptake and delivery in vivo​ after intravenous administration.

"Functional delivery has finally been recognised as the key issue for future therapeutic applications of RNAi,"​ said Iain Ross, executive chairman of SR Pharma.

Details of Atugen's development and systemic application of AtuPLEX are now available in two articles published in the latest online issue of Gene Therapy​ journal; titled "A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium"​ and "RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy."

According to the first article, proof of concept studies using Atugen's proprietary stabilised siRNAs (AtuRNAi), formulated with Atugen's novel cationic lipid (AtuFECT) and other components of the AtuPLEX, demonstrated target specific and RNAi-mediated knockdown for loss of function analysis in vivo.

These results were further verified when Atugen scientists successfully applied the AtuPLEX for inhibiting tumour growth and metastases using appropriate in vivo​ tumour models, according to data shown in the second article.

"AtuPLEX has all the required features to become a novel therapeutic modality for a wide range of human diseases,"​ said Ross.

Although other significant obstacles such as incomplete suppression of target genes, nonspecific immune responses and the so-called off-target effects, need to be overcome before this RNAi technology can be successfully translated into the clinical arena, much progress is being made, and these latest findings represent a significant advancement.

The development of new RNAi-based therapeutics and their entry into early phase clinical trials can be now expected in the very near future.

Related topics: Ingredients

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