RNA Biology and Beyond

RNA-based technologies have developed from an emerging field to established tools for drug discovery and offer a high potential for successful research programs in therapeutic areas such as neurodegeneration, oncology, and antiviral diseases.

We offer a broad panel of tools ranging from gene expression analysis, to screening of compounds for RNA-targeting drug discovery. Our iPRO (innovative Partner Research Organization) approach means we are open to apply our expertise to develop new projects tailored to our clients’ needs. Our units work in close collaboration with each other to ensure an efficient project transition from molecular biology to assay development and high-throughput screening campaigns.

RNA platforms and technologies

Our molecular biology unit is equipped with state-of-the-art technologies to evaluate gene expression level modulation by Real-Time PCR (TaqManTM and SYBR based-assays) from low to high-throughput applications, such as compound profiling or creening, using automated systems.

Understanding the effect of deregulated splicing events is a key step to treat several diseases such as cancer and neurodegenerative diseases. Our assay platforms allow the study of splicing modulation by using different approaches, including the use of a reporter tagging an endogenous gene expression (CRISPR-Cas9 technology), the use of a reporter tagging an exogenous gene (minigenes), TaqManTM-based detection and quantification of splicing variants and promoter activation assays.

Target validation by arrayed RNAi screenings can be carried out from a few to thousands of targets, in a miniaturized format (384-well plate), suitable for functional or image-based phenotypic readouts. Protocols can be adapted to a variety of cell lines (e.g., iPSC-derived cells; cancer cell lines and more).

A small molecule compound collection specifically designed to bind RNA is available. This collection is suitable to initiate discovery programs aimed at finding RNA splicing modulators, antiviral compounds, and RNA-protein interaction modulators.

  • Around 2.000 drug-like and lead-like compounds
  • High novelty & chemical diversity
  • Optimal starting point for MedChem programs

Our expertise encompasses promoter repression and activation analysis, assays to monitor the activity of DNA and chromatin modifying enzymes (enzymatic assays; DNA-protein interaction assay; RNA-protein interaction assay).

We use a fluorescence-based approach suitable as secondary assays for the identification of molecules directly intercalating nucleic acids. We offer our experience in customizing the assay conditions, including the design of long RNA molecules, or testing different RNA and DNA species.

We offer the customized development of HTS-grade enzymatic assays, transferable to clients or suitable to screen for RNase inhibitors or Hit-to-Lead follow-up activities performed also in-house.

By using a luciferase-based reporter system, we can develop safe cell-based viral-RNA replication assay (BSL-2 level) and perform compound profiling activity, for the identification of viral replication inhibitors.

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