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公开(公告)号：WO2021144649A2
公开(公告)日：2021-07-22
申请号：PCT/IB2021/000008
申请日：2021-01-15
Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED
IPC: A61K48/00 , C12N15/86 , A61K48/005 , A61K48/0058 , A61K48/0066 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2800/22 , C12N2830/008 , C12N2830/42 , C12N2830/48 , C12N9/0071 , C12Y114/16001
Abstract: The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid and a codon-optimized sequence encoding a human phenylalanine hydroxylase (PAH) enzyme. The disclosure also provides a method of treating a subject having phenylketonuria (PKU), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid, and a promoter operably linked to a nucleic acid sequence that encodes PAH, and wherein administering results in a decrease in phenylalanine level in the subject.
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Patent Agency Ranking2.发明申请
公开(公告)号：WO2022264040A1
公开(公告)日：2022-12-22
申请号：PCT/IB2022/055518
申请日：2022-06-14
Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED
Inventor： ROTTENSTEINER, Hanspeter
IPC: C07K14/755 , A61K38/37 , A61K48/00 , A01K2217/075 , A01K2227/105 , A01K2267/0381 , A61K48/005 , A61K48/0058 , C12N2750/14143 , C12N2800/22
Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.
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3.发明公开
公开(公告)号：EP4073106A2
公开(公告)日：2022-10-19
申请号：EP20842356.6
申请日：2020-12-10
Applicant: Takeda Pharmaceutical Company Limited
Inventor： ROTTENSTEINER, Hanspeter , HOELLRIEGL, Werner
IPC: C07K14/755 , A61K48/00 , C12N15/86 , C12N9/64 , C12N15/864 , A61K38/37
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公开(公告)号：EP4330270A2
公开(公告)日：2024-03-06
申请号：EP22732631.1
申请日：2022-04-29
Inventor： HAAR, Janina , GRIMM, Dirk , MULLER, Oliver , RAPTI, Kleopatra , ROTTENSTEINER, Hanspeter , HOELLRIEGL, Werner
IPC: C07K14/015 , C12N15/35 , C12N5/10 , C12N15/864 , A61K48/00
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5.发明公开
公开(公告)号：EP4355768A1
公开(公告)日：2024-04-24
申请号：EP22747747.8
申请日：2022-06-14
Applicant: Takeda Pharmaceutical Company Limited
Inventor： ROTTENSTEINER, Hanspeter
IPC: C07K14/755 , A61K38/37 , A61K48/00
CPC classification number: A01K2217/07520130101 , A01K2227/10520130101 , A01K2267/038120130101 , A61K38/37 , C07K14/755 , C12N2750/1414320130101 , C12N2800/2220130101 , A61K48/0058 , A61K48/005
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公开(公告)号：EP4090382A2
公开(公告)日：2022-11-23
申请号：EP21709460.6
申请日：2021-01-15
Applicant: Takeda Pharmaceutical Company Limited
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