Aptamer and AstraZeneca to determine the efficacy of targeted siRNA delivery vehicle

Published: 3-Jul-2024

AstraZeneca is providing Aptamer with siRNAs to test if its Optima fibrotic liver technology can successfully facilitate the delivery and cell integration of therapeutic RNA

Aptamer Group, a provider of nucleic acid binders, has entered into a novel agreement with AstraZeneca to evaluate the suitability of its Optima fibrotic liver delivery vehicles for the targeted delivery of siRNA.

The vehicles have shown promise in recent studies, and AstraZeneca will provide the siRNA necessary to facilitate the company’s next research phase, which will explore the technology’s potential in delivering these functional RNAs to the liver.

 

Enhancing cell internalisation of siRNAs with a non-viral vector

Aptamer’s Optimer-based delivery vehicle, which is specifically designed for fibrotic liver cell delivery, will be trialled with therapeutic siRNAs as its cargo.

Aptamer will conduct in-house experiments to determine the feasibility of the technology, which — if successful — will be presented to AstraZeneca for evaluation. 

The therapeutic implications of siRNA are vast, but delivering the functional RNA to specific cell types and tissues while also achieving internalisation has been a significant challenge. 

Therefore, the Optimer technology could facilitate the accurate, targeted and efficient delivery of siRNA molecules, potentially leading to the development of novel compounds that offer significant advantages over currently available tissue-targeting therapies. 


Chief Technical Officer of Aptamer Group, Dr Arron Tolley said: "Targeted delivery to specific cell types remains a critical unmet need in many applications within the tissue targeting space and — as such — has been part of our strategic focus to support the development of optimised nucleic acid binders. Our initial dataset in Optimer targeted delivery has raised significant interest from multiple parties, and determining the technology’s efficacy in animal models will derisk the Optimer delivery platform and bring us closer to delivering targeted and effective gene therapies for patients.”

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