The US FDA has granted fast track designation to Rznomics' RZ-001 for the treatment of hepatocellular carcinoma (HCC).
In frame translation initiation sites within coding sequences drive the expression of N-terminally truncated proteoforms.
Inhibition of nonsense-mediated decay turns immunologically cold tumors hot by increasing the amount of mutant RNA leading to an increase in T-cell-targetable neoantigens on the cell surface.