AT-01

AT-01 is a first-in-class antibody-drug conjugate (ADC) developed by Alethio Therapeutics, a spinout from the University of Oxford (formerly known as Alethiomics). The drug was discovered using the proprietary ARTEMIS discovery engine and is designed to target a novel protein that is uniquely upregulated on mutant stem cells and fibrosis-driving megakaryocytes in myeloproliferative neoplasms (MPNs). AT-01 is specifically engineered to penetrate fibrotic bone marrow to selectively target and eliminate disease-driving cells while sparing healthy tissue. Preclinical data have demonstrated the elimination of targeted cells and disease-modifying potential, supporting the advancement of the program toward IND/CTA-enabling studies in 2026.