Article
SFX-01 is therapeutic against myeloproliferative disorders caused by activating mutations in Shp2.
EMBO molecular medicine - 1 Aug 2025
Cho Hyun-Ju, Smith Joy, Switzer Christopher H, Louka Eleni, Charles Rebecca L, Prysyazhna Oleksandra, Schroder Ewald, Fernandez-Caggiano Mariana, de Jesus Daniel Simoes, Eminaga Seda, Yin Xiaoke, Yang Xiaoping, Lynham Steven, Mayr Manuel, Morales Valle, Bianchi Katiuscia, Rajeeve Vinothini, Cutillas Pedro R, Mead Adam J, Eaton Philip
Abstract excerpt
Activating mutations of Src homology-2 domain-containing protein tyrosine phosphatase-2 (Shp2) cause multiple childhood conditions for which there is an unmet therapeutic need, including juvenile myelomonocytic leukemia (JMML) and Noonan syndrome. SFX-01, an α-cyclodextrin-stabilized sulforaphane complex currently in clinical development, covalently adducts cysteine residues. Using unbiased proteomics, its...
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