[EN] TARGETED IDURONATE-2-SULFATASE COMPOUNDS<br/>[FR] COMPOSÉS D'IDURONATE-2-SULFATASE CIBLÉS
申请人:ANGIOCHEM INC
公开号:WO2014082184A1
公开(公告)日:2014-06-05
The present invention is related to a compound that includes a lysosomal enzyme and a targeting moiety, for example, a compound that includes iduronate-2-sulfatase conjugated to Angiopep-2 through a linker formed by specific click chemistry reactions. In certain embodiments, these compounds, owing to the presence of the targeting moiety, can cross the blood-brain barrier or accumulate in the lysosome more effectively than the enzyme alone. The invention also features pharmaceutical compositions containing such compounds and methods for treating lysosomal storage disorders (e.g., mucopolysaccharidosis Type II) using such compounds.