- 专利标题： Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
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申请号： US15274046申请日： 2016-09-23
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公开(公告)号： US10512675B2公开(公告)日： 2019-12-24
- 发明人: Stuart Bunting , Peter Cameron Colosi , Erno Pungor
- 申请人： BioMarin Pharmaceutical Inc.
- 申请人地址： US CA Novato
- 专利权人： BIOMARIN PHARMACEUTICAL INC.
- 当前专利权人： BIOMARIN PHARMACEUTICAL INC.
- 当前专利权人地址： US CA Novato
- 代理机构： Marshall, Gerstein & Borun LLP
- 主分类号： A61K38/37
- IPC分类号： A61K38/37 ; A61K48/00 ; C07K14/755 ; C12N7/00 ; C12N15/867 ; C12N15/00 ; A61K47/02 ; A61K47/10 ; A61K47/26 ; G01N33/68 ; A61K39/12
摘要：
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
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