Precision Medicine By Targeting Rare Human PCSK9 Variants for Cholesterol Treatment

摘要

The invention relates to human targets of interest (TOI), anti-TOI ligands, kits compositions and method.

主权项

1. A method of reducing cholesterol level or maintaining previously reduced cholesterol level in a human in need thereof, the method comprising administering to said human an antibody or antibody fragment that specifically binds a proprotein convertase subtilisin/kexin type 9 (PCSK9) that comprises a C-terminal domain comprising a mutation I474V or E670G in SEQ ID NO: 1,
wherein the antibody or antibody fragment comprises a VH domain derived from the recombination of a human VH segment, a human D gene segment and a human JH segment, the human VH segment encoding the framework 1 of SEQ ID NO: 40, and wherein said human comprises a nucleotide sequence encoding said PCSK9 that comprises a C-terminal domain comprising a mutation E670G in SEQ ID NO: 1.