MG-006

MG-006 is a small molecule inhibitor of the Sec61 translocon, developed by Gate Bioscience as a tool compound for investigating the treatment of immunoglobulin light chain (AL) amyloidosis. The Sec61 translocon is the primary channel through which proteins enter the endoplasmic reticulum for secretion. In AL amyloidosis, plasma cells produce misfolded light chains that form toxic amyloid fibrils. MG-006 blocks the co-translational translocation of these light chains, thereby preventing their secretion and inducing proteotoxic stress and apoptosis in the diseased plasma cells. Preclinical data presented at ASH 2024 demonstrated that MG-006 effectively reduces cell viability in AL amyloidosis cell lines and exhibits synergistic effects when combined with the proteasome inhibitor bortezomib.