smartRNA project joins BII Bio Studio programme

RNA

A project focused on developing a small RNA-based platform technology for treating monogenic diseases, smartRNA, has joined the BioInnovation Institute’s (BII) Bio Studio programme.

The approach to achieve this involves delivering an engineered small artificial (smart)RNA into the cell, where it assembles with endogenous protein co-factors to form an enzymatic complex.

The smartRNA is customisable, designed to specifically target the disease-associated healthy pool of mRNA for stabilisation.

The technology has platform potential, as it leverages a universal small RNA pathway to target generic mRNA features.

The project aims to leverage this proprietary technology to develop treatments for currently untreatable disorders caused by haploinsufficiency or other monogenic diseases caused by reduced protein activity.

BII will support the project with an in-kind grant of up to €1 million per year for a project period of up to three years, as well as with business development expertise, intellectual property support, access to investor network, and both wet lab and office infrastructure.

The project will be led by Søren Lykke-Andersen, a Senior Scientist at Aarhus University (AU), working at the Department of Molecular Biology and Genetics. He leads a research group focused on RNA therapeutics and has extensive expertise in studying RNA, including practical laboratory work, advanced computational analysis of complex data sets.

He said: “There are around 350 confirmed and many more predicted haploinsufficiency diseases, including many serious conditions such as cancer, epilepsy, and developmental disorders. Thus, there is a high unmet medical need to develop effective methods to treat monogenic diseases caused by reduced protein levels, primarily haploinsufficiency disorders. With the support of BII, we will be able to translate our research into a superior customisable small RNA-based platform technology to achieve this.”

Diana Spencer, Senior Digital Content Editor, DDW

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