e-Therapeutics PLC said it expects to offer “early-stage business development opportunities to potential partners in the coming months” following successful lab testing of its lead technology.
The company uses small interfering RNA, sometimes known as silencing RNA, and couples, or ‘conjugates’ it with a drug technology that ensures it gets to the right cells in the liver.
These so-called GaINAc-siRNA ‘constructs’ were assessed as part of a characterisation study.
The research demonstrated e-Therapeutics’ approach was equivalent in performance to leading competitor programmes showing “deep and durable target gene knock-down”.
Gene knockdown is where gene proliferation is slowed or even stopped.
The evaluation also revealed GaINAc-siRNA constructs were successfully characterised and showed “reproducible performance across three target genes”.
“These excellent results show that our proprietary delivery system and siRNA chemistries are competitive relative to peer platforms,” said chief executive Ali Mortazavi.
“This is a material step in the company's ultimate goal of developing an in-house RNAi pipeline with future scope for early-stage partnering.
“Eleven new patent applications have been filed to protect these innovations. In parallel, we are developing the world's largest data resource in hepatocyte biology which will enable the identification and prosecution of novel therapeutic targets, both internally and with collaborators.”
e-Therapeutics has not said which exact disease areas it is targeting with its technology, though as intimated above its efforts are focused on the liver.
Its progress mirrors that of Silence Therapeutics, which has successfully developed its nascent gene silencing technology into industry partnerships with the potential to yield hundreds of millions of pounds in milestone payments.
In a separate announcement, e-Therapeutics released its interim results, which charted the company’s significant operational progress in the six months ended July 31 and beyond and, crucially, showed the business was in a financially stable position to kick on.
As at the period-end it was sitting on £31.6mln of cash following May’s placing and open offer of new stock. Research and development spend for the six months was £2.5mln, and, unsurprisingly, the group posted an operating loss of £3.5mln.