Carboxylated 2′-amino-LNA nucleotides and oligonucleotides comprising the same
AssigneeBioNTech Delivery Technologies GmbH
InventorSteffen Panzner
Life of this patent
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Abstract
The invention proves an oligonucleotide comprising one or more carboxylated 2′-amino-LNA nucleotide units. The invention also provides a method of transfecting cells with the oligonucleotide, a method of treating a human or animal by therapy using the oligonucleotide, and a pharmaceutical composition comprising the oligonucleotide.
Claims20
An oligonucleotide comprising from 3 to 100 nucleotide units, wherein at least one nucleotide unit independently copmrises a carboxylated 2′-amino locked nucleic acid (LNA) nucleotides of formula (1′)
- wherein:
- B is a nucleobase,
- each of L1 is independently an internucleoside linkage, a bond to a neighboring nucleotide unit or a terminal moiety selected from the group consisting of —OH, phosphate, and a linker for the conjugation of the oligonucleotide to another molecule, and
- R is a carboxylated substituent comprising one or two carboxyl group(s) and wherein R comprises from 6 to 40 carbon atoms and from 0 to 3 heteroatoms selected from the group consisting of O, N, and S.
An oligonucleotide, comprising from 3 to 100 nucleotide units, wherein at least one nucleotide unit independently comprises a 2′-amino locked nucleic acid (LNA) nucleotide of structure (11) to (16), wherein Y is oxygen, or Y is absent when the carbon attached to Y is a methylene group:
- (11)
- wherein x is 1, 2, 3, 4, 5, 7, 9, 11 or 13;
- (12)
- wherein the hydrocarbyl moiety is an oleoyl residue;
- (13)
- wherein x is 2, 3, 4, 5, 6, 7, 8, 10, 12 or 14;
- (14)
- wherein x is 1, 2, 3, 4, 5, 6, 7, 8, 9, 11 or 13 and Z is H or a methyl, ethyl, propyl, butyl, pentyl or hexyl group or a constitutional isomer thereof and the combined number of carbon atoms in x and Z is from 1 to 13;
- (15)
- wherein x is 1, 2, 3, 4, 5,6, 7, 8, 9, 11 or 13 and Z is H or a methyl, ethyl, propyl, butyl, pentyl or hexyl group or a constitutional isomer thereof and the combined number of carbon atoms in x and Z is from 1 to 13;
- and
- (16)
- wherein x is 1, 2, 3, 4, 5, 6, 7, 8, 9, 10 or 12 and Z is H or a methyl, ethyl, propyl, butyl, pentyl or hexyl group or a constitutional isomer thereof and the combined number of carbon atoms in x and Z is from 1 to 13.
Dependencies are inferred from the claim text.