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US 2022/0054524 A1Published application
New conjugated nucleic acid molecules and their uses
AssigneeOnxeo
InventorsBRIAN SPROAT, CHRISTELLE ZANDANEL, FRANÇOISE BONO, ALEXANDRE SIMON
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Abstract
The present invention relates to new nucleic acid molecules of therapeutic interest, in particular for use in the treatment of cancer.
Claims25
1-25. (canceled)
A conjugated nucleic acid molecule comprising a double-stranded nucleic acid moiety, the 5′ end of the first strand and the 3′ end of the complementary strand being linked together by a loop, and optionally a molecule facilitating the endocytosis which is linked to the loop,
- wherein
- the length of the double-stranded nucleic acid moiety is from 10 to 20 base pairs;
- the sequence of the double-stranded nucleic acid moiety has less than 80% sequence identity to any gene in a human genome;
- the double-stranded nucleic acid moiety comprises deoxyribonucleotides and up to 30% of ribonucleotides or modified deoxyribonucleotides with respect to the total number of nucleotides of the nucleic acid molecule;
- and
- the loop has a structure selected from one of the following formulae:
- —O—P(X)OH—O—{[(CH2)2—O]g—P(X)OH—O}r—K—O—P(X)OH—O—{[(CH2)2—O]h—P(X)OH—O—}s (I)
- with r and s being independently an integer 0 or 1;
- g and h being independently an integer from 1 to 7 and the sum g+h being from 4 to 7;
- with K being
- with i, j, k and 1 being independently an integer from 0 to 6;
- or
- —O—P(X)OH—O—[(CH2)d—C(O)—NH]b—CHR—[C(O)—NH—(CH2)e]c—O—P(X)OH—O— (II)
- with b and c being independently an integer from 0 to 4, and the sum b+c is from 3 to 7;
- d and e being independently an integer from 1 to 3;
- and
- with R being -Lf-J,
- X being O or S, L being a linker and f being an integer being 0 or 1, and J being a molecule facilitating the endocytosis or being H.
15 more elements
Dependencies are inferred from the claim text.