IP Library › Granted Patent US 12,403,132
Granted Patent B2
US 12,403,132 · App. 17/924,601 · Granted Sep 2, 2025

Methods of treating bone marrow failure syndromes and compositions for use in the same

Inventors: Kathleen Miho Sakamoto (Stanford, CA); Mark Wilkes (Stanford, CA)
Assignee: The Board of Trustees of the Leland Stanford Junior University
A61K31/4375A61K31/155A61K36/258A61P7/00
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Quick Facts
Patent No.
US 12,403,132
App. No.
17/924,601
Granted
Sep 2, 2025
Kind
B2
Abstract

Methods of treating a subject for a bone marrow failure syndrome, e.g., DBA, are provided. Aspects of the methods include administering to the subject an effective amount of a Nemo-Like Kinase (NLK) inhibitor. Also provided are compositions that find use in practicing embodiment of the invention.

Claims (13)

1. A method of treating a subject diagnosed as having a bone marrow failure syndrome, the method comprising:

administering to the subject an effective amount of a nemo-like kinase (NLK) inhibitor to treat the subject for the bone marrow failure syndrome.

2. The method according to claim 1 , wherein the inhibitor comprises a small molecule.

3. The method according to claim 2 , wherein the small molecule is selected from the group consisting of: OTS167, INK128, AST487, BIRB796, SB203580, SD208, SB431542 and Galunisertib.

4. The method according to claim 3 , wherein the small molecule is OTS167.

5. The method according to claim 1 , wherein the NLK inhibitor comprises an NLK specific binding member.

6. The method according to claim 5 , wherein the NLK specific binding member comprises an antibody or binding fragment thereof.

7. The method according to claim 1 , wherein the NLK inhibitor comprises an NLK expression inhibiting agent.

8. The method according to claim 7 , wherein the NLK expression inhibiting agent comprises a nucleic acid.

9. The method according to claim 8 , wherein the nucleic acid is a micro-RNA.

10. The method according to claim 9 , wherein the micro-RNA is miR-26a, miR-208 or miR-181.

11. The method according to claim 7 , wherein the NLK expression inhibiting agent is metformin or ginsenoside Rb1.

12. The method according to claim 1 , wherein administration of the NLK inhibitor increases erythroid expansion.

Assignments (2)
CONFIRMATORY LICENSE Recorded Dec 19, 2023
From: STANFORD UNIVERSITY
To: UNITED STATES GOVERNMENT
Reel/Frame 066072/0149 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 10, 2022
From: SAKAMOTO, KATHLEEN MIHO; WILKES, MARK
To: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
Reel/Frame 061724/0930 →
Continuity (2)
Provisional Application 63046877 · Jul 1, 2020
Related Publication 20230172917A1 · Jun 8, 2023
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