Line-1 inhibitors to treat disease
AssigneeTransposon Therapeutics, Inc.
InventorsClaudio STURINO, Malay DOSHI, Eckard WEBER, Michael G. CORDINGLEY
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Abstract
The present disclosure provides methods of treating or preventing a disease, disorder, or condition in a subject in need thereof, the methods comprising administering to the subject a therapeutically effective amount of a compound of Formula I: or a pharmaceutically acceptable salt or solvate thereof, or a tautomer thereof, wherein R1, R2, and B are defined as set forth in the specification.
Claims24
A method of treating or preventing a disease, disorder, or condition caused by a pathophysiological retrotransposon-associated process in a subject in need thereof, and/or treating or preventing a symptom of the disease, disorder, or condition, the method comprising administering to the subject a therapeutically effective amount of:
- with provisos that the disease, disorder, or condition is not (i) cancer;
- or (ii) an infectious disease.
A method of inhibiting a LINE-1 retrotransposition event that causes a disease, disorder, or condition in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of:
- with provisos that the disease, disorder, or condition is not (i) cancer;
- or (ii) an infectious disease.
3-5. (canceled)
A method, comprising administering a therapeutically effective amount of:
- wherein:
- (a) the subject has a disease, condition, or disorder;
- and
- (b) the disease, condition, or disorder is characterized as having an overexpression of retrotransposon RNA, retrotransposon reverse transcriptase, or retrotransposon DNA,
- with provisos that the disease, disorder, or condition is not (i) cancer;
- or (ii) an infectious disease.
7-27. (canceled)
29-31. (canceled)
44-49. (canceled)
A kit comprising:
- and instructions for administering the compound to a subject having a disease, condition, or disorder caused by a pathophysiological retrotransposon-associated process.
Dependencies are inferred from the claim text.