RNAble is developing a small molecule modifier of RNA stability to treat cancer and rare diseases
Inhibition of specific epitranscriptomic modifiers can lead to stabilization of various RNA molecules. In the case of mutated RNA transcripts, such stabilization may prevent nonsense mediated decay (NMD). This can potentially lead to expression of partially functional proteins in the case of various rare diseases, or to increase in RNA load in NMD-dependent cancers.
AML, rare disease
Prof. Rotem Karni – Inventor
Amir Mor, PhD – CSO