Developing an AI Powered Platform for RNA-drug discovery

Monika Gullerova, Professor of Molecular Medicine and Tutor in Medicine, has secured seed funding to develop an AI powered platform for RNA-drug discovery. A team of four will work on the Raiden project, the aim of which is to achieve >95% predictive ability and to spin out a company that will lead in the field of RNA targeting by small compounds for new generation of therapies.

The Raiden project addresses a significant unmet need in drug discovery by focusing on small non-coding RNAs (ncRNAs), which play a crucial role in gene regulation and are frequently implicated in diseases like cancer and drug resistance. Despite their importance, targeted therapeutics for ncRNAs remain underdeveloped, relying on oligonucleotide therapies, which face challenges such as delivery, stability, off target effects and heavy modifications, creating a gap in RNA-focused drug discovery, especially for small molecules.

The Gullerova Lab's solution, Raiden, is an AI-Enabled RNA-Drug Interaction Discovery Platform that leverages machine learning and deep learning techniques to identify small molecules that bind to specific RNAs. Raiden uniquely combines two innovative approaches: (1) integration of 3D RNA structural features to identify "druggable" regions and (2) experimental validation of its predictions, generating real-world data that continuously refines the platform’s accuracy.

Unlike competitors that focus predominantly on proteins or messenger RNA, Raiden is specifically designed to target small non-coding RNA. The Gullerova Lab's dual-modality approach differentiates us by integrating advanced 3D RNA structure analysis with a robust library of small compounds, setting a new standard for precision and scalability in RNA-targeted drug discovery.

The Gullerova Lab published the following papers in 2024:

Tetrameric INTS6-SOSS1 complex facilitates DNA:RNA hybrid autoregulation at double-strand breaks in Nucleic Acids Research

From silence to symphony: transcriptional repression and recovery in response to DNA damage in Transcription

TIRR regulates mRNA export and association with P-bodies in response to DNA damage in Nucleic Acids Research

The GATAD2B-NuRD complex drives DNA:RNA hybrid-dependent chromatin boundary formation upon DNA damage in EMBO Press

Tyrosine 1-phosphorylated RNA polymerase II transcribes PROMPTs to facilitate proximal promoter pausing and induce global transcriptional repression in response to DNA damage in Genome Research

The ARID1A-METTL3-m6A axis ensures effective RNase H1-mediated resolution of R-loops and genome stability in Cell Reports