Our Science

Science at the Heart of RNA

Only a quarter of the proteins implicated in human disease are currently druggable by traditional small molecule approaches. Thus, new approaches directly targeting messenger RNAs of disease genes open up unprecedented opportunities for drug development.


At Rgenta, we have developed novel methods to mine the wealth of genomics data to identify high-efficacy and high-specificity RNA target sites. We have also built focused compound libraries designed to recognize the RNA target sites and modulate their function, particularly those mediated by regulatory proteins that regulate RNA splicing. Our unique approach enables us to generate new lead compounds for previously undruggable targets in a range of human diseases.

Rgenta's Novel Approach

Rgenta’s innovative approach aims to stop pathogenic proteins before they are made. By designing small molecules that can modulate splicing events and introduce signals that cause an RNA message to be destroyed by the cell’s machinery through nonsense-mediated decay (NMD), we can eliminate production of the pathogenic proteins. Our small molecules can be designed to enable targeted modulation of master regulators in biological pathways to tackle highly relevant but previously undruggable targets across disease areas. 

Targeting RNA and RNA-Binding Protein Interactions to Regulate Splicing and Gene Expression

Mechanism of Action


  • Regulate alternative splicing
  • Degrade target RNA through NMD
  • Eliminate pathological target protein


Differentiated Approach


  • Access tissues difficult for RNA targeting oligo therapies
  • Unlock targets undruggable at protein level

Rgenta’s Leading RNA-Targeting Discovery Platform

Our proprietary platform is industry leading and designed to select RNA targets that have a high probability of success for small molecule regulation.

>500Tb Genomic Data and Advanced Analysis Pipeline

Used to identify Plausible RNA-RBP Interactions

Thousands of unique mRNA/RBP/spliceosome interactions in target genes identified for drug intervention


Robust HTS assays and chemical libraries specifically designed for RNA/RBP interactions



RNA-targeting small molecule design principles and multi-parameter optimization

RSwitch Technology for Regulatable Gene and Cell Therapy

Rgenta has developed a regulatable gene therapy system called RSwitch, that can fine-tune the transgene levels in gene and cell therapy applications using an oral small molecule drug. We are looking to partner with groups developing gene and cell therapies across a wide variety of therapeutic indications.


RSwitch encodes a “dimmer” switch that makes the expression of transgene dependent on the administration of an oral small molecule drug that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein.


Rgenta has demonstrated the RSwitch system’s feasibility in vitro and in vivo, achieving dose-dependent expression of reporter transgenes following small molecule administration. Our RSwitch technology has broad applications across a range of genetic therapies.


Broad Applications of Rgenta's RSwitch Technology

Contact us at info@rgentatx.com to learn more about Rgenta’s technology.