IP Library › Granted Patent US 12,297,432
Granted Patent B2
US 12,297,432 · App. 17/842,478 · Granted May 13, 2025

Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity

Inventors: Nessan Anthony Bermingham (Cambridge, MA); Brian R. Bettencourt (Cambridge, MA); Pei Ge (Cambridge, MA)
Assignee: Takeda Pharmaceuticals U.S.A., Inc.
C12N15/113C12N2310/11C12N2310/315C12N2310/321C12N2310/3341C12N2310/335C12N2310/346C12N2320/35
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Quick Facts
Patent No.
US 12,297,432
App. No.
17/842,478
Granted
May 13, 2025
Kind
B2
Abstract

The present disclosure features useful compositions and methods to treat nucleotide repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with MSH3 activity.

Claims (9)

1. A single-stranded oligonucleotide of 15-30 linked nucleotides in length, wherein the oligonucleotide is at least 95% complementary to at least 15 contiguous nucleobases at positions 2685-2714 of SEQ ID NO: 614, or a pharmaceutically acceptable salt thereof, wherein the oligonucleotide comprises at least one alternative nucleobase, or a pharmaceutically acceptable salt thereof.

2. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises at least one alternative internucleoside linkage, or a pharmaceutically acceptable salt thereof.

3. The oligonucleotide of claim 2 , wherein the at least one alternative internucleoside linkage is a phosphorothioate internucleoside linkage.

4. The oligonucleotide of claim 1 , wherein the alternative nucleobase is 5′-methylcytosine, pseudouridine, or 5-methoxyuridine.

5. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises at least one alternative sugar moiety, or a pharmaceutically acceptable salt thereof.

6. The oligonucleotide of claim 5 , wherein the alternative sugar moiety is 2′-OMe or a bicyclic nucleic acid.

7. The oligonucleotide claim 1 , wherein the oligonucleotide is selected from the group consisting of Antisense Oligo Nos. 289-384 of Table 3, or a pharmaceutically acceptable salt thereof.

8. The oligonucleotide of claim 1 , wherein the oligonucleotide is Antisense Oligo No. 289 of Table 3, or a pharmaceutically acceptable salt thereof.

9. A pharmaceutical composition comprising one or more of the oligonucleotides, or pharmaceutically acceptable salts thereof, of claim 1 and a pharmaceutically acceptable carrier or excipient.

Assignments (3)
PATENT ASSIGNMENT AGREEMENT Recorded Nov 3, 2023
From: TRIPLET ABC, LLC
To: TAKEDA PHARMACEUTICALS U.S.A., INC.
Reel/Frame 065453/0515 →
PATENT ASSIGNMENT AGREEMENT Recorded Nov 3, 2023
From: TRIPLET THERAPEUTICS, INC.
To: TRIPLET ABC, LLC
Reel/Frame 065453/0549 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 14, 2022
From: BERMINGHAM, NESSAN ANTHONY; BETTENCOURT, BRIAN R.; GE, PEI
To: TRIPLET THERAPEUTICS, INC.
Reel/Frame 060506/0965 →
Continuity (3)
Continuation 17337172 · Jun 2, 2021
Provisional Application 63034319 · Jun 3, 2020
Related Publication 20230068672A1 · Mar 2, 2023
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