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US 2026/0306465 A1Published application
Compounds for treating hereditary spastic paraplegia
AssigneeChildren's Medical Center Corporation
InventorsDarius Ebrahimi-Fakhari, Mustafa Sahin, Jed L. Hubbs, Masato Satoh
Life of this patent
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Abstract
Provided herein are compounds that modulate Autophagy Related 9 A (ATG9A) trafficking and/or increase autophagic flux. Also provided are pharmaceutical compositions comprising the compounds, and methods of treating neurological disease or disorder, such as hereditary spastic paraplegia (HSP).
Claims34
A compound of Formula (I):
- or a pharmaceutically acceptable salt thereof, wherein:
- each occurrence of R1 is, independently, halogen, substituted or unsubstituted acyl, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted heteroaliphatic, substituted or unsubstituted carbocyclyl, substituted or unsubstituted heterocyclyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, a nitrogen protecting group when attached to a nitrogen atom, —ORA, —N(RA)2, —SRA, —CN, —SCN, —C(═NRA)RA, —C(═NRA)ORA, —C(═NRA)N(RA)2, —C(═O)RA, —C(═O)ORA, —C(═O)N(RA)2, —C(═O)NRAS(O)2RA, —NO2, —NRAC(═O)RA, —NRAC(═O)ORA, —NRAC(═O)N(RA)2, —NRAC(═NRA)N(RA)2, —OC(═O)RA, —OC(═O)ORA, —OC(═O)N(RA)2, —NRAS(O)2RA, OS(O)2RA, —S(O)2NRAC(O)RA, —S(O)2N(RA)2, —S(O)2ORA or —S(O)2RA;
- or two R1 groups are joined to form a substituted or unsubstituted carbocyclyl ring, a substituted or unsubstituted aryl ring, a substituted or unsubstituted heterocyclyl ring, or a substituted or unsubstituted heteroaryl ring;
- t is 0 or a positive integer;
- and
- each occurrence of RA is, independently, hydrogen, substituted or unsubstituted acyl, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted heteroaliphatic, substituted or unsubstituted carbocyclyl, substituted or unsubstituted heterocyclyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, a nitrogen protecting group when attached to a nitrogen atom, an oxygen protecting group when attached to an oxygen atom, or a sulfur protecting group when attached to a sulfur atom, or two RA groups are joined to form a substituted or unsubstituted heterocyclyl ring, or a substituted or unsubstituted heteroaryl ring;
- wherein, when present, each occurrence of R1 is bound to any substitutable atom of the compound.
4 more elements
Dependencies are inferred from the claim text.