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US 2026/0200830 A1Published application
Compositions and methods for reducing tactile dysfunction, anxiety, and social impairment
AssigneePresident and Fellows of Harvard College
InventorsDavid D. Ginty, Lauren L. Orefice, Jinbo Lee
Life of this patent
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Abstract
The present invention features novel peripherally-restricted isoguvacine analogs with reduced blood brain barrier permeability and methods of use thereof for reducing tactile dysfunction, social impairment, and anxiety in a subject diagnosed with Autism Spectrum Disorder, Rett syndrome, Phelan McDermid syndrome, or Fragile X syndrome.
Claims22
1-3. (canceled)
A compound having the structure of Formula (II):
- wherein
- n=1, 2, 3, 4, 5, 6, 7, or 8;
- each of R1 and R2 is, independently, hydrogen, deuterium, optionally substituted C1-6 alkyl, or optionally substituted C3-6 cycloalkyl wherein R1 and R2 are covalently linked;
- R3 is substituted C1-6 alkyl;
- and
- each R4 is, independently, hydrogen, deuterium, halogen, C1-4 alkoxy, C1-6 alkyl, —CF3, —CH3S, —CH3SO2, or —NO2;
- or a pharmaceutically acceptable salt thereof.
4 more elements
5-22. (canceled)
A compound having the structure of Formula (I):
- wherein
- n=1, 2, 3, 4, 5, 6, 7, or 8;
- each of R1 and R2 is, independently, hydrogen, deuterium, optionally substituted C1-6 alkyl, or optionally substituted C3-6 cycloalkyl wherein R1 and R2 are covalently linked;
- each R4 is, independently, hydrogen, deuterium, halogen, C1-4 alkoxy, C1-6 alkyl, —CF3, —CH3S, —CH3SO2, or —NO2;
- and
- A is a carboxylic acid biomimetic of formula:
- or pharmaceutically acceptable salt thereof.
4 more elements
Dependencies are inferred from the claim text.