Purpose-built library specializing in small molecule RNA drug discovery

High-throughput screening (HTS) with an RNA splicing focused library makes obtaining hit compounds in a short period of time and with a high success probability possible. The compounds were designed and synthesized by our medicinal chemists with the aim of affecting for RNA splicing. 

Library Concept

Approximately 1,700 compounds, which focused on pharmacophore with the intention of binding to RNA splice sites, were designed and synthesized by our medicinal chemists. Access to chemical spaces which have mostly been unfilled in the existing library is possible.

Library with a focus on quality

・Lead-like and diverse molecular weight distributions
・High drug-likeness
・Compound library with a wide range of basicity

High hit rate and selectivity

Representative compounds of each chemotype were selected from the library and allowed to act on cultured cells overnight. The effect on RNA splicing by each compound was assessed using Real Time PCR. As a result, compounds which affected RNA splicing were found in the SMN2 gene used as control and in two selected genes, A and B, with hit rates exceeding 10%. Moreover, several selective compounds for each gene could be identified.