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1. WO2011005799 - RNA CONTAINING MODIFIED NUCLEOTIDES AND USE THEREOF IN VACCINES

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[ EN ]

Claims

1. A self-replicating RNA molecule comprising at least two nucleosides that each,

independently, comprise at least one chemical modification.

2. The self-replicating RNA molecule of claim 1 , wherein the at least two modified

nucleosides are components of modified nucleotides in which the nitrogenous base comprises the chemical modification.

3. The self-replicating RNA molecule of claim 2, wherein about 0.01% to about 25% of the nucleotides in the self-replicating RNA molecule are modified nucleotides.

4. The self-replicating RNA molecule of claim 2, wherein about 0.01% to about 25% of the nucleotides that contain uracil, cytosine, adenine, or guanine in the self-replicating RNA molecule are modified nucleotides.

5. The self-replicating RNA molecule according to any one of claims 1-4, wherein the

nucleosides that comprise at least one chemical modification or the modified nucleotide comprises a nucleoside selected from the group consisting of dihydrouridine,

methyladenosine, methylcytidine, methylguanosine, methyluridine, methylpseudouridine, thiouridine, deoxycytodine, and deoxyuridine.

6. The self-replicating RNA molecule according to any one of claims 1-5, wherein the self- replicating RNA molecule comprises at least about 4kb.

7. The self-replicating RNA molecule according to any one of claims 1-6, wherein said self- replicating RNA molecule encodes at least one antigen.

8. The self-replicating RNA molecule of claim 7, wherein the antigen is a viral, bacterial, fungal or protozoan antigen.

9. The self-replicating RNA molecule of any one of claims 1-8, wherein the chemical modifications are, independently, selected from the group consisting of hypoxanthine, inosine, 8-oxo-adenine, 7-substituted derivatives thereof, dihydrouracil, pseudouracil, 2- thiouracil, 4-thiouracil, 5-aminouracil, 5-(Ci-Ce)-alkyluracil, 5-methyluracil, 5 -(C2-C6)- alkenyluracil, 5-(C2-C6)-alkynyluracil, 5-(hydroxymethyl)uracil, 5-chlorouracil, 5- fluorouracil, 5-bromouracil, 5 -hydroxy cytosine, 5-(Ci-C6)-alkylcytosine, 5- methylcytosine, 5-(C2-C6)-alkenylcytosine, 5-(C2-C6)-alkynylcytosine, 5-chlorocytosine, 5-fluorocytosine, 5-bromocytosine, N2-dimethylguanine, 7-deazaguanine, 8-azaguanine, 7-deaza-7-substituted guanine, 7-deaza-7-(C2-C6)alkynylguanine, 7-deaza-8-substituted guanine, 8-hydroxyguanine, 6-thioguanine, 8-oxoguanine, 2-aminopurine, 2-amino-6- chloropurine, 2,4-diaminopurine, 2,6-diaminopurine, 8-azapurine, substituted 7- deazapurine, 7-deaza-7-substituted purine, 7-deaza-8-substituted purine, hydrogen (abasic residue), and any combination thereof.

10. A pharmaceutical composition comprising a self-replicating RNA molecule according to any one of claims 1-9 and a pharmaceutically acceptable carrier and/or a

pharmaceutically acceptable vehicle.

11. The pharmaceutical composition of claim 10, further comprising at least one adjuvant.

12. The pharmaceutical composition of claim 10 or 11, further comprising a cationic lipid, a liposome, a cochleate, a virosome, an immune-stimulating complex, a microparticle, a microsphere, a nanosphere, a unilamellar vesicle, a multilamellar vesicle, an oil-inwater emulsion, a water-in-oil emulsion, an emulsome, and a polycationic peptide, or a cationic nanoemulsion.

13. The pharmaceutical composition of any one of claims 10-12, wherein the self-replicating RNA molecule is encapsulated in, bound to or adsorbed on a cationic lipid, a liposome, a cochleate, a virosome, an immune-stimulating complex, a microparticle, a microsphere, a nanosphere, a unilamellar vesicle, a multilamellar vesicle, an oil-inwater emulsion, a water-in-oil emulsion, an emulsome, and a polycationic peptide, a cationic nanoemulsion and combinations thereof.

14. A method for the prevention and/or treatment of an infectious disease comprising

administering an effective amount of a pharmaceutical composition according to any one of claims 10-13.

15. A method for inducing an immune response in a subject comprising administering to the subject an effective amount of a pharmaceutical composition according to any one of claims 10-13.

16. A method of vaccinating a subject, comprising administering to the subject a

pharmaceutical composition according to any one of claims 10-13.

17. A method for inducing a mammalian cell to produce a protein of interest, comprising the step of contacting the cell with a pharmaceutical composition according to any one of claims 10-13, under conditions suitable for the uptake of the self-replicating RNA molecule by the cell, thereby inducing a mammalian cell to produce a protein of interest.

18. A method for gene delivery comprising administering to a pharmaceutical composition according to any one of claims 10-13.