Researchers in the U.K. have overthrown the orthodox view that childhood cancers have a low mutation burden, opening up new drug targets and opportunities for repurposing existing therapies. In particular, a high mutation rate is associated with a response to cancer immunotherapy. But although PD-1 checkpoint inhibitors are approved for treating pediatric cancers with a high level of microsatellite instability mutations, in general it is thought childhood tumors are not amenable to immunotherapy.

Hepatocellular carcinoma (HCC) is among the leading causes of cancer-related deaths worldwide due to lack of effective diagnosis at early stages and limited comprehension of its pathogenesis, thus limiting the development of effective treatments.

PRMT1 is a key enzyme that catalyzes asymmetric dimethylation of arginine residues and overexpressed in various cancers and inflammatory diseases. While current PRMT1 inhibitors lack specificity and effectiveness, targeted degradation of PRMT1 offers a potential strategy to treat PRMT1-driven conditions and explore its nonenzymatic roles.

Voltron Therapeutics Inc. has reported data from a study evaluating VTX-0P4.