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Methods for Directing Lipid Nanoparticles in Vivo

Application US20260078381A1 Kind: A1 Mar 19, 2026

Assignee

Vertex Pharmaceuticals Incorporated

Inventors

Gregoriy Aleksandrovich Dokshin, Souvik Biswas

Abstract

Compositions and methods for enhancing payload-based gene therapy by increasing the percentage of payload delivered to a non-liver target in a subject by blocking binding of LDL to LDL receptors (LDLR) in the liver, and then administering a payload-based therapy targeting a non-liver tissue.

CPC Classifications

C12N 15/1138 C12N 2310/14 C12N 2310/351 C12N 2310/3515

Filing Date

2025-11-25

Application No.

19400453