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US 12,338,438 B2Granted
Conjugated oligonucleotide compounds, methods of making and uses thereof
AssigneeE-Therapeutics PLC
InventorsAhmad Ali Mortazavi, Viviana Mannella, Muthusamy Jayaraman
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Abstract
The present invention relates to novel conjugated oligonucleotide compounds, which are suitable for therapeutic use. Additionally, the present invention provides methods of making these compounds, as well as methods of using such compounds for the treatment of various diseases and conditions.
Claims19
A compound of Formula (I):
- wherein:
- R1 at each occurrence is independently selected from the group consisting of hydrogen, methyl, and ethyl;
- R2 is fluoro or hydroxy;
- X1 and X2 at each occurrence are independently selected from the group consisting of methylene, oxygen, and sulfur;
- m is an integer of from 1 to 6;
- n is an integer of from 1 to 10;
- q, r, s, t, v are independently integers from 0 to 4, with the proviso that:
- (i) q and r cannot both be 0 at the same time;
- and
- (ii) s, t, and v cannot all be 0 at the same time;
- Z is:
- wherein:
- Z1, Z2, Z3, and Z4 are independently at each occurrence oxygen or sulfur;
- and
- one of the bonds between P and Z2, and P and Z3, is a single bond and the other bond is a double bond;
- and
- is:
- wherein:
- A1 is hydrogen;
- a is an integer of 2 or 3;
- and
- b is an integer of 2 to 5.
19 more elements
A compound having the structure of:
- (i) Formula (II):
- (ii) Formula (IV):
- (iii) Formula (III):
- or
- (iv) Formula (V):
- wherein
- is
- wherein:
- A1 is hydrogen;
- a is an integer of 2 or 3;
- and
- b is an integer of 2 to 5;
- optionally wherein the oligonucleotide comprises one or more degradation protective moieties at one or more ends.
10 more elements
A compound having the structure of:
- (i) Formula (VIII):
- (ii) Formula (X):
- (iii) Formula (IX):
- or
- (iv) Formula (XI):
2 more elements
Dependencies are inferred from the claim text.