AstraZeneca and Aptamer partner to enhance targeted siRNA therapies

AstraZeneca+and+Aptamer+partner+to+enhance+targeted+siRNA+therapies

The collaboration will support the development of Aptamer Group’s Optimer technology and AstraZeneca’s siRNA therapies. Image: Solskin / Shutterstock

Pharmaceutical giant AstraZeneca and British biotechnology company Aptamer Group hope to improve the efficacy of small interfacing RNA (siRNA) therapies using Aptamer’s Optimer delivery system.

The companies did not disclose financial details of the collaboration, but following the announcement on July 3, Aptamer shares rose 18% to $0.95 (£0.74).

The collaboration between the two UK-based companies will begin in the preclinical phase, starting with lab data before moving on to animal studies. The Optimer system has already shown efficacy in fibrotic liver in in vitro studies.

The companies did not want to confirm which indication they will investigate in the preclinical phase.

While the better-known and better-researched messenger RNA (mRNA), used to develop Covid-19 vaccines, carries genetic information for protein synthesis, siRNA specifically targets and precisely switches off specific genes.

However, a major challenge for drug developers is off-target efficacy, where the siRNA can bind to unintended mRNA targets, leading to unintended gene silencing. As a result, a more effective delivery system would be hugely beneficial.

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Aptamer claims its Optimer technology could represent a “paradigm shift” in the targeted delivery of siRNA molecules.

According to Dr. Arron Tolley, CTO of Aptamer Group, these types of partnerships with major pharmaceutical companies will help the company make rapid progress with its Optimer technology.

Tolley said: “It is great to see major pharmaceutical companies such as AstraZeneca interested in the Optimer technology and wanting to explore its potential as a non-viral vector.”

“Our in vitro dataset demonstrates that our Optimer delivery vehicle successfully overcomes challenges by selectively targeting activated hepatic stellate cells, the cells responsible for fibrotic liver disease, and not other cell types in the liver or other tissues. This allows the siRNA to be selectively delivered to the fibrotic cells, as opposed 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 of moving to animal studies.”

Tolley added that Optimer is also being evaluated by another top 15 pharmaceutical partner with the potential for in-licensing, and that the product is attracting interest from other companies in the sector.

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, kidney diseases, metabolic diseases, respiratory diseases and hepatitis C.

The first siRNA agent, Alnylam’s Onpattro (patisiran), received approval from the U.S. Food Drug Administration (FDA) in 2018. As of March 2024, the FDA had approved five other agents: Alnylam’s Givlaari (givosiran), Oxlumo (lumasiran) and Amvuttra (vutisiran), Novartis’ Leqvio (inclisiran), and Dicerna Pharmaceuticals’ Rivfloza (nedosiran).

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