This WP advances RNA-based therapies and diagnostics through innovative modeling, genetic circuit design, drug discovery, and testing methodologies. It develops computational models, engineered RNA devices, and novel drug compounds, leading to pre-clinical studies that demonstrate the efficacy and safety of these interventions.

Computational Modeling
Computational models predict RNA structures and functions of RNA-based systems, including toxicity forecasts for non-coding RNA variants from large cohort sequencing. Efforts also focus on designing aptamers, discovering binding sites, improving screenings of RNA-RNA and RNA-protein interactions, and experimentally determining RNA crystal and cryoEM structures in complex with small molecule modulators, especially for large RNAs. In the field of RNA–RNA interactions, RIME was developed as an AI-based tool capable of accurately predicting interacting RNAs, revealing the key role of repetitive sequences in gene regulation and nervous system development.

Drug Discovery and Targeting
Compounds targeting RNA structures are identified and optimized, using RNA-binding molecules to address specific disease-relevant pathways. This supports the creation of precise therapeutic agents.

Bioprinting and Screening
Bioprinters and bioinks are developed for disease modeling, enabling high-throughput screening of RNA-based drugs in 3D bioprinted tissues to accelerate validation.

Pre-clinical Testing
In vitro and in vivo testing evaluates formulated (modified) RNA, including pharmacokinetics and toxicology profiles. Small ncRNA signatures are explored for diagnostic purposes in assessing anti-neuroinflammatory therapies.