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Shifa Precision (a division of Shifa Biomedical) is developing a small molecule inhibitor of Proprotein convertase subtilisin/kexin type 9 (PCSK9) for the treatment of dyslipidemia and the lowering of LDL cholesterol. This candidate, identified as Approach 2 in the company's pipeline, utilizes a novel mechanism of action by inhibiting the autocatalytic processing of PCSK9. PCSK9 is synthesized as a zymogen that must undergo intramolecular cleavage in the endoplasmic reticulum to become functional and be secreted. By blocking this maturation step, the compound prevents the production of functional PCSK9, thereby increasing the density of LDL receptors on the cell surface and enhancing the clearance of LDL-C from the blood. The program is currently in the preclinical stage of development.
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