# US 11,433,027 B2 · Dry powder composition comprising long-chain RNA > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 11,433,027 B2 - Document ID: US11433027B2 - Status: Granted - Kind: B2 - Application number: 16/998,259 - Filed: 2020-08-20 - Published: 2020-12-10 - Granted: 2022-09-06 - Earliest claimed: 2015-05-20 - Assignees: CureVac AG - Applicants: CureVac AG - Inventors: Fabian Johannes Eber; Benyamin Yazdan Panah; Stefanie Sewing; Thomas Ketterer; Thorsten Mutzke; Tilmann Roos; Michael Sonntag; Michael Wiggenhorn; Katharina Kolland - Web page: https://patentgrep.com/patent/US11433027B2 ## CPC - A61K 9/16: Agglomerates; Granulates; Microbeadlets {; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction (A61K9/20 takes precedence if the final form is a tablet; microspheres with drug-free outer coating, microcapsules A61K9/50; mixture of different granules, microcapsules, (coated) microparticles A61K9/5084; nanoparticles A61K9/51)} - A61K 9/1682: {Processes} - A61K 39/145: Orthomyxoviridae, e.g. influenza virus - A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered} - A61K 48/0091: {Purification or manufacturing processes for gene therapy compositions} - A61P 31/00: Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics - A61P 31/04: Antibacterial agents - A61P 31/12: Antivirals - A61P 33/02: Antiprotozoals, e.g. for leishmaniasis, trichomoniasis, toxoplasmosis - A61P 35/00: Antineoplastic agents - A61P 37/06: Immunosuppressants, e.g. drugs for graft rejection - A61P 37/08: Antiallergic agents (antiasthmatic agents A61P11/06; ophthalmic antiallergics A61P27/14) - A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy - A61K 2039/53: {DNA (RNA) vaccination} - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS ## 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. ## Claims ### Claim 1 A dry powder composition comprising a long-chain RNA molecule comprising at least 30 nucleotides in a complex with a cationic or polycationic compound, wherein said dry powder: (i) has a residual moisture content of less than 7% (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. ### Claim 2 (depends on 1) The dry powder composition according to claim 1, wherein the average sphericity of the particles in the composition is in a range from 0.7 to 1.0. ### Claim 3 (depends on 1) The dry powder composition according to claim 1, wherein the composition has a residual moisture content of 5% (w/w) or less. ### Claim 4 (depends on 1) The dry powder composition according to claim 1, wherein the long-chain RNA molecule is present in the form of a free long-chain RNA molecule, or in the form of a complex comprising the long-chain RNA molecule. ### Claim 5 (depends on 1) The dry powder composition according to claim 1, wherein the long-chain RNA molecule is in a complex with a cationic or polycationic lipid. ### Claim 6 (depends on 1) The dry powder composition according to claim 1, wherein the long-chain RNA molecule is a single-stranded RNA molecule. ### Claim 7 (depends on 1) The dry powder composition according to claim 1, wherein the long-chain RNA molecule comprises at least one open reading frame (ORF) encoding a protein or a peptide. ### Claim 8 (depends on 7) The dry powder composition according to claim 7, wherein the long-chain RNA molecule is an mRNA molecule. ### Claim 9 (depends on 7) The dry powder composition according to claim 7, wherein the long-chain RNA molecule comprises at least 200 nucleotides. ### Claim 10 (depends on 9) The dry powder composition according to claim 9, wherein the long-chain RNA molecule comprises at least one modification. ### Claim 11 (depends on 9) The dry powder composition according to claim 9, wherein the dry powder composition comprises at least one excipient. ### Claim 12 (depends on 9) The dry powder composition according to claim 9, wherein the dry powder is made by a method of spray-drying. ### Claim 13 (depends on 12) The dry powder composition according to claim 12, wherein the method of spray-drying comprises the steps of: (a) providing a liquid comprising the long-chain RNA molecule to be spray-dried; and (b) spray-drying the liquid of step (a) with a spray-drying device having a drying gas inlet and a drying gas outlet, wherein the drying gas has a temperature at the inlet of at least 85° C., wherein the drying gas has a temperature at the outlet of at least 50° C. ### Claim 14 (depends on 11) The dry powder composition according to claim 11, wherein the at least one excipient selected from a cryoprotectant, a lyoprotectant, and a bulking agent. ### Claim 15 (depends on 13) The dry powder composition according to claim 13, wherein the liquid of step (a) comprises a spray-drying-compatible solvent. ### Claim 16 (depends on 13) The dry powder composition according to claim 13, wherein the liquid of step (a) is atomized and a droplet resulting from the atomization of the liquid is characterized by a mass median aerodynamic diameter of 300 nm to 200 μm. ### Claim 17 (depends on 13) The dry powder composition according to claim 13, wherein the liquid of step (a) further comprises a suspending agent and/or osmolality of about 200 mosmol/l to about 400 mosmol/l. ### Claim 18 (depends on 9) A pharmaceutical composition comprising the dry powder composition according to claim 9. ### Claim 19 (depends on 18) The pharmaceutical composition according to claim 18, further comprising at least one pharmaceutically acceptable excipient. ### Claim 20 (depends on 18) The pharmaceutical composition according to claim 18, wherein the dry powder is formulated for administration by inhalation. ### Claim 21 (depends on 18) The pharmaceutical composition according to claim 18, wherein the dry powder is formulated for pulmonary delivery. ### Claim 22 (depends on 9) The dry powder composition according to 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. ### Claim 23 (depends on 14) The dry powder composition according to claim 14, wherein the at least one excipient comprises a free carbohydrate. ### Claim 24 (depends on 23) The dry powder composition according to claim 23, wherein the at least one excipient comprises lactose, sucrose or trehalose. ### Claim 25 (depends on 9) The dry powder composition according to claim 9, wherein the long-chain RNA molecule is 500 to 20,000 nucleotides in length.