ADAR1 inhibitor (Zα-targeting)

ADAR1 inhibitor (Zα-targeting) refers to a series of small molecule inhibitors developed to selectively target the Zα domain of the p150 isoform of Adenosine Deaminase Acting on RNA (ADAR1). ADAR1 is an enzyme responsible for A-to-I RNA editing, which prevents the activation of the MDA5-mediated innate immune response by endogenous double-stranded RNA (dsRNA). The p150 isoform is specifically induced by interferon and contains a unique Zα domain that binds to Z-DNA and Z-RNA. By disrupting this binding, these inhibitors prevent ADAR1 from masking immunogenic dsRNA, thereby promoting an anti-tumor immune environment and inducing apoptosis in ADAR1-dependent tumors. These candidates were identified through the Multi-layer Ultra-large Screening and Exploration (M.U.S.E) pipeline and are being investigated for the treatment of oral squamous cell carcinoma (OSCC), particularly in Asian populations where the disease burden is high.

Other names

Zα-targeting ADAR1 inhibitorp150 ADAR1 inhibitorp-150 ADAR1 inhibitorp 150 ADAR1 inhibitor