公开(公告)号：US20230357134A1

公开(公告)日：2023-11-09

申请号：US18245235

申请日：2021-09-16

Applicant: UCL BUSINESS LTD

Inventor： Vittorio BELLOTTI ,  Alessandra CORAZZA ,  Palma Patrizia MANGIONE ,  Mark Brian PEPYS ,  Christopher SWAIN ,  Graham Walter TAYLOR

IPC: C07C229/58 ,  C07D237/24 ,  C07D213/80 ,  C07D263/57 ,  C07D231/12 ,  C07D263/34 ,  A61P43/00

CPC classification number: C07C229/58 ,  C07D237/24 ,  C07D213/80 ,  C07D263/57 ,  C07D231/12 ,  C07D263/34 ,  A61P43/00

Abstract: The present invention relates to compounds for stabilising the native tetrameric form of transthyretin and protecting it from proteolytic cleavage; compounds for use in the prevention and treatment of transthyretin amyloidosis; and agents and medicaments comprising such compounds. The compounds are based on a general structure A-L-B, wherein A is a group of formula (II) or of formula (III): or of formula (IV) or of formula (V) B is a group of formula (III), (IV), or (V), or a group of formula (VI) or a group of formula —R10Z, wherein: Z is selected from —CO2R′, —CONR′R″, —SO2R′ wherein R′ and R″ are independently H or C1-C4 alkyl; and R10 is a C1-C4 alkylene or alkenylene group; and L represents a linker group which is a saturated or unsaturated chain of 5 to 13 carbon atoms.