RNAble is developing novel compounds that alter RNA stability through modulation of RNA epitranscriptomics for the treatment of specific cancers and rare genetic diseases with nonsense mutations.
RNA transcripts undergo modifications that are recognized by specific RNA-binding proteins dictating their stability.
Professor Rotem Karni, RNAble inventor, discovered that targeting components of the RNA epitranscriptomic machinery with small molecules resulted in the stabilization of several RNAs, including unstable nonsense mutated transcripts which are degraded by the nonsense-mediated decay machinery.
This discovery opens the possibility of restoring the expression of mutated genes essential to rare diseases and suppressing the growth of specific cancers.