Dry powder composition comprising long-chain RNA

Patent metadata

Field	Value
Publication number	US-12318486-B2
Application number	US-202217811808-A
Country	US
Kind code	B2
Filing date	Jul 11, 2022
Priority date	May 20, 2015
Publication date	Jun 3, 2025
Grant date	Jun 3, 2025

Abstract

The present invention is directed to a storage-stable formulation of long-chain RNA. In particular, the invention concerns a dry powder composition comprising a long-chain RNA molecule. The present invention is furthermore directed to methods for preparing a dry powder composition comprising a long-chain RNA molecule by spray-drying. The invention further concerns the use of such a dry powder composition comprising a long-chain RNA molecule in the preparation of pharmaceutical compositions and vaccines, to a method of treating or preventing a disorder or a disease, to first and second medical uses of such a dry powder composition comprising a long-chain RNA molecule and to kits, particularly to kits of parts, comprising such a dry powder composition comprising a long-chain RNA molecule.

First claim

The invention claimed is:

1. A dry powder composition comprising a long-chain RNA molecule that is 500 to 20,000 nucleotides in length and is formulated with a cationic or polycationic compound, wherein the long-chain RNA molecule comprises at least one open reading frame (ORF) encoding a protein, wherein:
(i) said dry powder has a residual moisture content of less than 2%-5% (w/w); and
(ii) comprises a plurality of particles having a median particle size, in a volume weighted distribution of the composition, in a range from 1 μm and 20 μm.

2. The dry powder composition according to claim 1 , said dry powder having a residual moisture content of less than 4% (w/w).

3. The dry powder composition according to claim 1 , wherein the median particle size in a volume weighted distribution is at least 1 μm.

4. The dry powder composition according to claim 1 , wherein the particles have an average sphericity in a range from 0.7 to 1.0.

5. The dry powder composition according to claim 1 , wherein the long-chain RNA molecule is an mRNA molecule.

6. The dry powder composition according to claim 1 , wherein the long-chain RNA molecule comprises at least one nucleotide modification.

7. The dry powder composition according to claim 1 , wherein the dry powder composition comprises at least one further excipient.

8. The dry powder composition according to claim 1 , wherein the long-chain RNA molecule is in a complex with a cationic or polycationic lipid.

9. A pharmaceutical composition comprising the dry powder composition according to claim 8 .

10. The pharmaceutical composition of claim 9 , further comprising at least one pharmaceutically acceptable excipient.

11. The pharmaceutical composition of claim 10 , wherein the dry powder is formulated for administration by inhalation.

12. The pharmaceutical composition of claim 10 , wherein the dry powder is formulated for pulmonary delivery.

13. The dry powder composition of claim 9 , wherein upon reconstitution of the dry powder in a suitable solvent, the complexed long-chain RNA molecule is present in the solvent in the form of nanoparticles having a size of about 50 to 500 nm.

14. The dry powder composition of claim 10 , wherein the at least one excipient comprises a free carbohydrate.

15. The dry powder composition of claim 14 , wherein the at least one excipient comprises lactose, sucrose or trehalose.

16. The dry powder composition of claim 15 , wherein the at least one excipient comprises sucrose.

Assignees

• CureVac SE

Inventors

• Eber Fabian Johannes
• Yazdan Panah Benyamin
• Sewing Stefanie
• Ketterer Thomas
• Mutzke Thorsten
• Roos Tilmann
• Sonntag Michael
• Wiggenhorn Michael
• Kolland Katharina

Classifications

• A61K2039/53

  DNA (RNA) vaccination · CPC title

• A61K48/00

  Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy · CPC title

• A61K39/145

  Orthomyxoviridae, e.g. influenza virus · CPC title

• A61K9/1682

  Processes · CPC title

• A61P35/00

  Antineoplastic agents · CPC title