XFOREST Therapeutics

What it does

Company Overview – xFOREST Therapeutics xFOREST Therapeutics is a Kyoto University iPS cell institute spin-out founded in 2020, dedicated to overcoming “undruggable” diseases through RNA-targeted small molecule therapeutics. By unlocking the therapeutic potential of RNA structures, we aim to deliver novel treatment options for patients with few existing therapies. Our proprietary FOREST platform enables high-throughput, structure-based discovery of RNA-binding small molecules, a previously inaccessible domain for small molecule drugs The platform’s core engine, MatrixFOREST, biochemically profiles binding interactions across thousands of RNA structural motifs and tens of thousands of compounds. This "library × library" approach generates single-nucleotide resolution binding profiles, enabling efficient discovery of high-affinity binders (KD < 100 nM) and guiding SAR-based lead optimization. SpliceVerse, our complementary platform, screens splicing modulators and their mechanisms of action (MoA) through multiplexed assays. It supports high-throughput selectivity evaluation and provides detailed mechanistic insights at the nucleotide level resolution. xFOREST’s approach solves key limitations of conventional RNA drug discovery, which typically relies on low-throughput, target-by-target methods. Our large-scale interaction data enables the identification of drug-like, RNA-selective chemical clusters, forming the foundation for next-generation RNA-targeted drug libraries. We are collaborating with five leading Japanese pharmaceutical companies including Takeda, Daiichi-Sankyo and Astellas across therapeutic areas such as oncology, neuroscience, immunology, and rare diseases. In parallel, we continue expanding our RNA-focused compound library and internal lead discovery programs, establishing xFOREST as a pioneering platform company in RNA-targeted therapeutics.