IP Library › Granted Patent US 12,318,357
Granted Patent B2
US 12,318,357 · App. 16/873,866 · Granted Jun 3, 2025

Small molecule drugs and related methods for treatment of diseases related to TDP-43, alpha-synuclein, huntingtin's protein and tau protein oligomer formation

Inventors: Ambuj K. Singh (Santa Barbara, CA); Christian A. Lang (Santa Barbara, CA)
Assignee: ACELOT, INC.
A61K31/137A61K31/167A61K31/277A61K31/357A61K31/40A61K31/452A61K31/495A61K31/5375
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Quick Facts
Patent No.
US 12,318,357
App. No.
16/873,866
Granted
Jun 3, 2025
Kind
B2
Abstract

The present invention provides small molecule drugs and pharmaceutical compositions for the treatment and prevention of diseases related to the formation of certain types of oligomers in a subject. More specifically, the drugs and compositions reduce or prevent the formation of oligomers formed from tau protein, TDP-43, Huntingtin's protein and/or alpha-synuclein. It further provides a method of reducing formation of or disrupting TDP-43, alpha-synuclein, Huntingtin's protein and/or tau protein oligomers in a subject, the method comprising the step of administering to the subject in need thereof a therapeutically effective amount of a pharmaceutical composition.

Claims (8)

1. A method of treating a disease in a subject in need thereof, wherein the disease is caused by deposits of TDP-43 in the brain of the subject and is selected from the group consisting of sporadic amyotrophic lateral sclerosis, frontotemporal dementia, chronic traumatic encephalopathy, progressive supranuclear palsy, corticobasal degeneration, Parkinson's disease, Huntington's disease, limbic-predominant age-related TDP-43 encephalopathy, and motor neuron diseases, the method comprising administering to the subject a therapeutically effective amount of a compound, wherein the compound is one of:

or a pharmaceutically acceptable salt of any of the foregoing.

2. The method of claim 1 , wherein the compound is one of:

or a pharmaceutically acceptable salt of any of the foregoing.

3. A method of treating a disease in a subject in need thereof, wherein the disease is caused by deposits of TDP-43 in the brain of the subject and is selected from the group consisting of sporadic amyotrophic lateral sclerosis, frontotemporal dementia, chronic traumatic encephalopathy, progressive supranuclear palsy, corticobasal degeneration, Parkinson's disease, Huntington's disease, limbic-predominant age-related TDP-43 encephalopathy, and motor neuron diseases, the method comprising administering to the subject a therapeutically effective amount of a compound selected from the group consisting of

and a pharmaceutically acceptable salt of any of the foregoing.

4. The method of claim 3 , wherein the subject has sporadic amyotrophic lateral sclerosis.

5. The method of claim 3 , wherein the compound is:

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 31, 2020
From: SINGH, AMBUJ K.; LANG, CHRISTIAN A.
To: ACELOT, INC.
Reel/Frame 053548/0377 →
Continuity (2)
Provisional Application 62922316 · Aug 2, 2019
Related Publication 20210052519A1 · Feb 25, 2021
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