ORT001

ORT001 is a preclinical-stage small molecule inhibitor of deoxycytidine kinase (dCK), a key enzyme in the nucleotide salvage pathway. Developed by Orthos Therapeutics (a spinout from CNRS, INSERM, and the Institut Paoli-Calmettes), ORT001 targets metabolic vulnerabilities in proliferating cancer cells, which are highly dependent on nucleotide synthesis for survival. By blocking dCK, ORT001 disrupts the salvage pathway, and when combined with thymidine (which inhibits the de novo nucleotide synthesis pathway), it triggers synthetic lethality in cancer cells. The primary therapeutic focus for ORT001 is T-cell acute lymphoblastic leukemia (T-ALL), with potential applications in other cancers and T-cell-mediated autoimmune diseases.