Science
TARGETING THE "UNDRUGGABLES"
Only a quarter of the proteins implicated in human diseases are currently druggable by traditional approaches for developing small-molecules compounds. Thus, new approaches directly targeting messenger RNAs of disease genes open up unprecedented opportunities for drug development. At Rgenta, we develop novel methods to mine the wealth of genomics data to identify high-efficacy and high-specificity RNA target sites. We also build focused compound libraries likely to recognize the RNA target sites and modulate their function, particularly those mediated by the regulatory proteins. Our unique approach has resulted in new lead compounds for previously undruggable targets in a range of human diseases.
Rgenta's Novel Approach
Traditional Approach
Rgenta's Approach
Selective modulation of RNA splicing and processing
Rgenta’s integrative RNA-targeting discovery platform
Thousands of unique mRNA/RBP/spliceosome interactions in target genes identified for drug intervention.
Robust HTS assays specifically designed for RNA/RBP interactions.
Custom designed RNA targeting selectivity panel to drive selectivity.
RNA-targeting small molecule design principles and multi-parameter optimization.
Target Discovery Engine
Data-driven target discovery platform integrates RNA regulation across tissues and cell types to exploit the biological modulation for therapeutic benefit.
Lead Discovery Engine
Robust assays tailored specifically for the mechanism of action of interest and custom-designed compound libraries enable rapid screening and lead generation.
Lead Optimization Engine
Lead optimization platform applies medicinal chemistry expert knowledge and multi-parameter optimization design principles to improve lead compound pharmacological properties.
