Molecure has confirmed the in vitro activity of another molecule binding to a new mRNA target within the mRNA discovery platform

– In December 2023, Molecure confirmed the inhibition of protein translation with compounds targeting the mRNA in an in vitro proof-of-concept (PoC) using a cell-based assay 

– This month, Molecure confirmed inhibition of the protein translation function for another mRNA target. Another small molecule discovered and synthesized by Molecure showed efficacy in an in vitro assay, expanding the number of selective binders in its mRNA discovery platform. 

– The achievement of the PoC stage for another molecule and a second target in the mRNA platform provides evidence of the effectiveness of Molecure’s strategy to identify mRNA-binding compounds with therapeutic potential 

– Strong biopharmaceutical industry interest in Molecure’s mRNA targeting technology increases the  commercial potential of Molecure’s mRNA discovery platform and the likelihood of signing a collaboration or partnership agreement. 


Warsaw, 12th June 2024 – Molecure S.A. (‘Molecure’, WSE ticker: MOC), a biotechnology company discovering and developing drugs to the clinical stage that leverages its globally unique expertise in medicinal chemistry and biology to search for and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA translation for the treatment of multiple incurable diseases, has confirmed in vitro the ability to bind to another mRNA target and demonstrated efficacy of a second molecule in inhibiting the translation mechanism of a pathological protein using compounds that target the mRNA encoding that protein. This is another success in the development of the mRNA discovery platform and one of the Company’s strategic goals for 2023-2025. 


„The discovery of another class of molecules binding to a second mRNA biological target, confirmed in in vitro assays, is the result of our team’s tremendous commitment and innovative approach to finding new molecules that modulate the translational function of mRNA. In December 2023, we obtained positive in vitro results for the first class of our molecules, and just a few months later we confirmed translational inhibition for another molecule targeting a different mRNA target. This attests to the broad scientific competence of our team and the high quality of our drug design processes, which we are improving through the use of advanced AI tools and computational methods. In the mRNA-targeting small molecule technology, we are in the European and even global vanguard and see great commercial potential for our mRNA discovery platform.  At the June BIO International Convention in San Diego, we had many meetings with representatives of companies interested in collaborating on discovering and developing small molecules targeting mRNA. Most of them were BigPharma representatives. We see many large partnership deals in this space signed at very early stages of drug development. In line with our strategy, this should allow us to intensify our partnering discussions and increase the likelihood of establishing a commercially attractive collaboration with a large industry partner. We are very pleased with multiple discussions we had during the recent BIO in the US with potential partners regarding our research program pipeline” – said dr Zbigniew Zasłona, CSO Molecure S.A. 


Molecure in the area of the mRNA platform is considering several commercialisation models, including a service model based on generating revenue from collaborating on the development of mRNA targets selected by the partner, which, through the use of a platform supported by AI tools, the Company can validate and then design and optimise molecules that interact with these targets. Another model is the classic licensing to a partner of Molecure’s newly discovered molecules that directly model mRNA function. Molecure is one of the few biotech companies in the world that develops small-molecule drugs that directly interact with mRNA targets.  


About Molecure S.A. 

Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical stage, leveraging its own unique expertise in medicinal chemistry and biology to search for and develop first-in-class small-molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, may provide therapies for many incurable diseases. 

Molecure has generated a diverse portfolio of seven distinct programmes with support from leading academic research institutions around the world, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan and the International Institute of Molecular and Cell Biology in Warsaw, Poland (MIBMiK). 

The most advanced drug candidate developed by Molecure is OATD-01, a first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis.  

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer, whose Phase I clinical trial has begun with first patient administration in Q1 2023. 

Molecure’s headquarters and laboratories are located in Warsaw and Lodz. The company is listed on the Warsaw Stock Exchange (ticker: MOC). 

Detailed information can be found at:  

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA