Molecular constructs with targeting and effector moieties for treating central nervous system diseases

摘要

The present disclosure provides various molecular constructs having a targeting element and an effector element. Methods for treating various diseases using such molecular constructs are also disclosed.

主权项

1. A molecular construct comprising a first linker unit and a second linker unit, wherein,
the first linker unit comprises,
a first center core comprising a plurality of amine groups,a first linking arm linked to the first center core,optionally, a first connecting arm linked to the first linking arm,a first element linked to the first linking arm or the first connecting arm, andoptionally, a first coupling arm linked to the first center core; and the second linker unit comprises,
a second center core comprising a plurality of amine groups,a second linking arm linked to the second center core,optionally, a second connecting arm linked to the second linking arm,a second element linked to the second linking arm or the second connecting arm, andoptionally, a second coupling arm linked to the second center core; wherein,the first and second linker units are coupled to each other via CuAAC reaction, SPAAC reaction or iEDDA reaction occurred between any of the followings: the first and second center cores, the first coupling arm and the second center core, the first and second coupling arms, or the first center core and the second coupling arm; wherein the first element is an scFv specific for transferrin receptor, and the second element is interferon-β (IFN-β), fingolimod, fingolimod phosphate, or an scFv specific for integrin α4 or β-amyloid.