CGT4983

CGT4983 is an orally bioavailable, potent, and highly selective small molecule inhibitor of the **JAK2 V617F** mutation, currently under development by Cogent Biosciences for the treatment of myeloproliferative neoplasms (MPNs). The V617F mutation in the Janus kinase 2 (JAK2) gene is a primary driver of diseases such as polycythemia vera, essential thrombocythemia, and myelofibrosis. Unlike existing JAK inhibitors that non-selectively target both wild-type and mutant JAK2, CGT4983 is engineered to specifically inhibit the mutant form while sparing wild-type JAK2 signaling. This selectivity is intended to avoid the hematologic toxicities, such as anemia and thrombocytopenia, that often limit the clinical utility of non-selective inhibitors. By providing a wider therapeutic window, CGT4983 aims to achieve deeper molecular responses and improved safety for patients with MPNs.

Other names

JAK2 V617F inhibitorJAK-2 V617F inhibitorJAK 2 V617F inhibitorwild-type-sparing JAK2 V617F mutant-selective inhibitor