Liver specific delivery of messenger RNA

Patent metadata

Field	Value
Publication number	US-10576166-B2
Application number	US-201615092226-A
Country	US
Kind code	B2
Filing date	Apr 6, 2016
Priority date	Dec 1, 2009
Publication date	Mar 3, 2020
Grant date	Mar 3, 2020

Abstract

Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.

First claim

What is claimed is:

1. The cationic lipid:

Assignees

• Translate Bio Inc

Inventors

• Derosa Frank
• Heartlein Michael

Classifications

• A61P1/16

  for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics · CPC title

• C12N15/67

  General methods for enhancing the expression · CPC title

• A61K9/1271

  Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers (liposomes as conjugates {A61K47/6911}) · CPC title

• A61K9/0019

  Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00) · CPC title

• A61K48/005

  characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered · CPC title