公开(公告)号：US20240148665A1

公开(公告)日：2024-05-09

申请号：US18495741

申请日：2023-10-26

Applicant: University of Virginia Patent Foundation

Inventor： Anuradha Illendula ,  Samir Zuberi ,  Alex Powell

IPC: A61K9/51 ,  A61K31/7105 ,  A61K47/10 ,  A61K47/24 ,  A61K47/26 ,  A61K47/28

CPC classification number: A61K9/5123 ,  A61K31/7105 ,  A61K47/10 ,  A61K47/24 ,  A61K47/26 ,  A61K47/28

Abstract: Provided are compositions that include compositions of galactosyl-conjugated lipid nanoparticles (LNPs) encapsulating one or more active agents. In some embodiments, the galactosyl-conjugated LNPs have a lipid component having D-Lin-MC3-DMA, ALC-0315 and SM-102, cholesterol. DSPC and DOPE, and DMG-2000-PEG. In some embodiments, the GalNAc-conjugated LNP has one or more galactosyl moieties bioconjugated to cholesterol present with a lipid component of the GalNAc-conjugated LNP. Also provided are methods for treating and/or preventing diseases, disorders, and/or conditions associated with undesirable PCSK9 gene expression, optionally a cardiovascular disease, disorder, or condition, including but not limited to atherosclerosis and/or thrombosis, and sepsis, septic shock, cytokine storm, or sequelae thereof.