ADAR1 inhibitor (Accent Therapeutics)

Accent Therapeutics is developing a preclinical-stage small molecule inhibitor of ADAR1 (Adenosine deaminase acting on RNA 1) for the treatment of solid tumors, including head and neck squamous cell carcinoma (HNSCC) and non-small cell lung cancer (NSCLC), particularly those resistant to PD-(L)1 checkpoint inhibitors. ADAR1 is an enzyme that catalyzes the deamination of adenosine to inosine in double-stranded RNA (dsRNA), a process that prevents the activation of innate immune sensors like MDA5 and PKR. By inhibiting ADAR1, the drug promotes the accumulation of dsRNA, triggering a type I interferon response and subsequent immune-mediated cell death in tumor cells with high intrinsic interferon signaling. This mechanism aims to sensitize "cold" tumors to immunotherapy and provide a monotherapy option for specific subsets of cancers.

Other names

ADAR1 small molecule inhibitorADAR-1 small molecule inhibitorADAR 1 small molecule inhibitorADAR1 programADAR-1 programADAR 1 program