Pulmonary delivery of mRNA

Patent metadata

Field	Value
Publication number	US-9597413-B2
Application number	US-201414308554-A
Country	US
Kind code	B2
Filing date	Jun 18, 2014
Priority date	Jun 8, 2011
Publication date	Mar 21, 2017
Grant date	Mar 21, 2017

Abstract

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

First claim

The invention claimed is:

1. A method of delivery of messenger RNA (mRNA) for in vivo expression of protein, comprising
administering via inhalation from a nebulizer to a subject a composition comprising an mRNA encoding full-length exogenous CFTR protein such that the administering of the composition results in in vivo expression of the full-length exogenous CFTR protein encoded by the mRNA in lung cells of the subject, wherein the composition further comprises polyethylenimine (PEI).

2. The method of claim 1 , wherein the mRNA comprises a non-naturally occurring nucleotide.

3. The method of claim 1 , wherein the mRNA comprises pseudouridine.

4. A method of delivery of messenger RNA (mRNA) for in vivo expression of protein, comprising
administering via inhalation from a nebulizer to a subject a composition comprising an mRNA encoding full-length exogenous CFTR protein such that the administering of the composition results in in vivo expression of the full-length exogenous CFTR protein encoded by the mRNA in lung cells of the subject,
wherein the mRNA encoding the full-length exogenous CFTR protein is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

5. The method of claim 4 , wherein the mRNA comprises a non-naturally occurring nucleotide.

6. The method of claim 4 , wherein the mRNA comprises pseudouridine.

Assignees

• Shire Human Genetic Therapies

Inventors

• Guild Braydon Charles
• Derosa Frank
• Heartlein Michael

Classifications

• A61P5/00

  Drugs for disorders of the endocrine system · CPC title

• A61P7/06

  Antianaemics · CPC title

• A61P3/00

  Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) · CPC title

• A61P1/16

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