mRNA Lightning platform for the discovery of selective small molecule mRNA regulatory drugs

Abstract

Anima Biotech is advancing mRNA Lightning, a novel platform for the discovery of selective mRNA small molecules. Anima’s differentiated approach combines high-scale, automated, phenotypic cell-based screens with deep learning image analysis algorithms developed for mRNA regulation. Anima’s pipeline projects will be presented, demonstrating how mRNA-regulation target space is amenable to selective modulation by small molecules. The mRNA regulation target space includes proteins involved in RNA processing post-transcription and ends in mRNA translation by ribosomes. Anima’s mRNA translation modulators are not targeting the ribosomes or directly bind to mRNA but rather target a novel target space, proteins involved in the regulation of mRNA translation. By targeting these newly discovered cellular mechanisms that regulate mRNA biology, Anima discovered highly selective drugs against a broad range of challenging and seemingly undruggable targets. The small molecules have been shown to be tissue and/or disease specific, as they are targeting proteins that have specific biological roles in coordinating and regulating the translation of individual mRNAs and pathways.