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公开(公告)号：US20240318156A1
公开(公告)日：2024-09-26
申请号：US18573298
申请日：2022-06-24
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Daniel WENDT , Mika AOYAGI-SCHARBER , Roger LAWRENCE, , Manuel LOPEZ
CPC classification number: C12N9/2402 , C12Y302/01052 , A61K38/00
Abstract: Disclosed herein are recombinant β-hexosaminidase variant a subunits that form a β-hexosaminidase variant a subunit homodimer that have optimized properties for use in treating Tay-Sachs disease or Sandhoff Disease.
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