BioInnovation Institute (BII), an international non-profit foundation, incubating and accelerating world-class life science research, today announces that it has accepted a new project into its Bio Studio program to develop a small RNA-based platform technology for treating monogenic diseases caused by reduced protein levels, primarily haploinsufficiency disorders, which constitute a significant unmet medical need. The project, named smartRNA will be led by Søren Lykke-Andersen.
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 customizable, designed to specifically target the disease-associated healthy pool of mRNA for stabilization. This stabilization delays mRNA degradation, allowing additional rounds of translation, resulting in increased levels of the functional protein. The technology has platform potential, as it leverages a universal small RNA pathway to target generic mRNA features.
Thus, the aim of the smartRNA project in BII’s Bio Studio program is 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 1M EUR 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.
During the 3-year BII Bio Studio program, the objective of smartRNA is to deliver an in vitro proof-of-concept, a pseudo in vivo proof-of-principle, and in vitro benchmarking against established technologies.
Principal Investigator Søren Lykke-Andersen is 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, and more than 15 years of experience supervising students and technicians.
Søren Lykke-Andersen, Principal Investigator at the Aarhus University 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 customizable small RNA-based platform technology to achieve this”.
Anja Mølhart Høg, Entrepreneur-in-Residence heading up the smartRNA project, added: “We are excited to welcome the smartRNA project to BII’s Bio Studio program. smartRNA is addressing an area with a significant unmet need – for the vast majority of haploinsufficiency diseases, there is a lack of disease-modifying treatment options. This innovative approach has the potential to transform the treatment landscape for haploinsufficiency diseases and to make a significant societal impact, offering hope and improved outcomes for patients worldwide through personalized medicine and precision therapeutics for rare diseases”.
Read more about the smartRNA project here.
The Bio Studio program is a recently established BII program with the ambition to build and run a leading life science company creation facility in Europe. This latest project joins collaborations announced last year with KU Leuven, University of Copenhagen, European Molecular Biology Laboratory (EMBL) in Heidelberg, Imperial College London, and Institute of Protein Design at University of Washington.