Pharma giant AstraZeneca and UK biotech Aptamer Group are hoping to bolster the efficacy of small interfacing RNA (siRNA) therapies, using Aptamer’s Optimer delivery system.
The companies did not disclose the financials behind the collaboration but following the 3 July announcement, Aptamer’s shares were up by 18% to $0.95 (£0.74).
Go deeper with GlobalData
The collaboration between the two UK-based companies will begin in pre-clinical stages, starting with lab data, before moving to animal studies. The Optimer system has already shown to be effective in fibrotic liver in in-vitro studies.
The companies would not confirm which indication they will be investigating at pre-clinical stages.
While the more known and investigated messenger RNA (mRNA), used to develop the Covid-19 vaccinations, carries genetic information for protein synthesis, siRNA targets and silences specific genes with precision.
A huge challenge that has faced drug developers however is off-target effects, where the siRNA may bind to unintended mRNA targets, leading to unintended gene silencing. As a result, a more effective delivery system would be hugely beneficial.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataAptamer claims that its Optimer technology could represent a ‘paradigm shift’ in the targeted delivery of siRNA molecules.
CTO of Aptamer Group, Dr. Arron Tolley, said that these sorts of partnerships with big pharma will help the company make rapid progress with its Optimer technology.
Tolley said: “It is great to see large pharma businesses like AstraZeneca interested in Optimer technology and wanting to explore its potential as a non-viral vector.”
“Our in-vitro dataset shows our Optimer delivery vehicle successfully overcomes challenges, selectively targeting activated hepatic stellate cells, the cells responsible for fibrotic liver disease, and not other cell types within the liver or other tissues. This enables the siRNA to be selectivity delivered into the fibrotic cells, relative to normal liver cells. The collaboration was initiated based on these results, and we will evaluate the performance of their siRNA molecule with our Optimer delivery vehicle for fibrotic liver, with the intention to move to animal studies.”
Tolley added that Optimer is also being evaluated by a different top 15 pharma partner with the potential for licensing, and is gaining interest from other companies across the industry.
siRNA can be used as a therapeutic approach in various diseases, including oncology, fibrosis, neurological disorders, HIV, arthritis, Crohn’s disease, ulcerative colitis, cardiovascular diseases, renal disease, metabolic disorders, respiratory diseases, and hepatitis C.
The first siRNA agent, Alnylam’s Onpattro (patisiran), received US Food Drug Administration (FDA) approval in 2018. By March 2024, the FDA had approved five other agents, Alnylam’s Givlaari (givosiran), Oxlumo (lumasiran) and Amvuttra (vutisiran), Novartis’s Leqvio (inclisiran), Dicerna Pharmaceuticals Rivfloza (nedosiran).
