Iduronidase stabilizers and uses thereof

Abstract

Disclosed herein are compounds that stabilize recombinant human α-iduronidase (rh-α-IDUA) activity, and their uses in the treatment and/or prophylaxis of lysosomal storage diseases (LSDs), such as mucopolysaccharidosis type I (MPS1). The compound disclosed herein has the structure of formula (I), wherein, n is an integral between 0 and 2; X1 is O or —NH; X2 is O, —NH or —NRa, in which Ra is C1-10alkyl; Y is H or wherein m and p are independently 0 or 1; X3 is S, O, or —NH; X4 is O, —NH, methylene, or —CH2(C1-10)alkyl; and A is aryl, heteroaryl, or heterocyclyl optionally substituted with one or more substituent selected from the group consisting of halo, hydroxyl, amino and phosphate.

Dependencies are inferred from the claim text.