IP Library › Granted Patent US 12,152,244
Granted Patent B2
US 12,152,244 · App. 17/586,175 · Granted Nov 26, 2024

Selective antisense compounds and uses thereof

Inventors: Michael Oestergaard (Carlsbad, CA); Punit P. Seth (Carlsbad, CA); Eric E. Swayze (Encinitas, CA)
Assignee: Ionis Pharmaceuticals, Inc.
C12N15/113C07H21/02C12N2310/11C12N2310/3125C12N2310/313C12N2310/315C12N2310/3231C12N2310/3341C12N2310/335C12N2310/341C12N2310/345C12N2310/346C12N2320/34
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Quick Facts
Patent No.
US 12,152,244
App. No.
17/586,175
Granted
Nov 26, 2024
Kind
B2
Abstract

The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

Claims (14)

1. A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the nucleobase sequence of the modified oligonucleotide comprises at least 8 contiguous nucleobases of the nucleobase sequence recited in SEQ ID NOs: 251, 268, or 490-500, and wherein the modified oligonucleotide has a sugar motif selected from eeeee-d9-eeeee, ekk-d9-kke, eekk-d8-kkeee, ekek-d8-kekee, ekk-d8-kkeee, eeekk-d7-kkeee, or eekek-d7-kekee; wherein k is a cEt sugar moiety, e is a 2′-OCH 2 CH 2 OCH 3 ribosyl sugar moiety, and d is a 2′-deoxyribosyl sugar moiety.

2. The compound of claim 1 , wherein the nucleobase sequence of the modified oligonucleotide is 100% complementary to SEQ ID NO: 1.

3. The compound of claim 1 , wherein the modified oligonucleotide has a eeeee-d9-eeeee sugar motif.

4. The compound of claim 1 , wherein the modified oligonucleotide has a ekk-d9-kke sugar motif.

5. The compound of claim 1 , wherein the modified oligonucleotide has a eekk-d8-kkeee sugar motif.

6. The compound of claim 1 , wherein the modified oligonucleotide has a ekek-d8-kekee sugar motif.

7. The compound of claim 1 , wherein the modified oligonucleotide has a ekk-d8-kkeee sugar motif.

8. The compound of claim 1 , wherein the modified oligonucleotide has a eeekk-d7-kkeee sugar motif.

9. The compound of claim 1 , wherein the modified oligonucleotide has a eekek-d7-kekee sugar motif.

10. The compound of claim 1 , wherein the modified oligonucleotide comprises at least one phosphorothioate internucleoside linkage.

11. The compound of claim 1 , wherein the compound is conjugated.

12. A pharmaceutical composition comprising the compound of claim 1 and a pharmaceutically acceptable carrier or diluent.

13. The pharmaceutical composition of claim 12 , wherein the pharmaceutically acceptable diluent is phosphate-buffered saline (PBS).

14. A method of ameliorating a symptom of Huntington's disease, comprising administering the compound of claim 1 to a subject in need thereof.