IP Library › Granted Patent US 12,377,073
Granted Patent B2
US 12,377,073 · App. 17/288,178 · Granted Aug 5, 2025

Treatments for charcot-marie-tooth disease

Inventors: Robert W. Burgess (Bar Harbor, ME); Emily Spaulding (Bar Harbor, ME)
Assignee: The Jackson Laboratory
A61K31/4162A61K31/404A61K45/06A61P21/00A61P25/00C12N15/113
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Quick Facts
Patent No.
US 12,377,073
App. No.
17/288,178
Granted
Aug 5, 2025
Kind
B2
Abstract

Provided herein, in some embodiments, are methods and compositions for the treatment of Charcot-Marie-Tooth disease, comprising an inhibitor of a GCN2 pathway component.

Claims (10)

1. A method comprising administering to a subject an inhibitor of expression and/or activity of GCN2, wherein the subject has Charcot-Marie-Tooth (CMT) disease.

2. The method of claim 1 , wherein the CMT disease is selected from CMT1, CMT2, CMT2D, diCMT, CMT2W, and CMT2N.

3. The method of claim 1 , wherein the subject is a human.

4. The method of claim 1 , wherein the inhibitor inhibits expression of GCN2.

5. The method of claim 1 , wherein the inhibitor inhibits activity of GCN2.

6. The method of claim 1 , wherein the inhibitor is a selected from polypeptides, polynucleotides, and small component drugs.

7. The method of claim 6 , wherein the inhibitor is an antibody.

8. The method of claim 6 , wherein the inhibitor is a programmable nuclease.

9. The method of claim 6 , wherein the inhibitor is RNA interference component or an antisense RNA component.

10. The method of claim 6 , wherein the inhibitor is a small component drug selected from the group consisting of A-92 (triazolo[4,5-d]pyrimidine derivative), indirubin-3-monoxime, SP600125, and a Syk inhibitor.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 12, 2022
From: BURGESS, ROBERT; SPAULDING, EMILY
To: THE JACKSON LABORATORY
Reel/Frame 061385/0422 →
Continuity (3)
Provisional Application 62751388 · Oct 26, 2018
Related Publication 20210393591A1 · Dec 23, 2021
Related Publication 20220387387A2 · Dec 8, 2022
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