Invention Grant
- Patent Title: Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression
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Application No.: US16777630Application Date: 2020-01-30
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Publication No.: US11596671B2Publication Date: 2023-03-07
- Inventor: Hanspeter Rottensteiner , Franziska Horling , Johannes Lengler , Friedrich Scheiflinger
- Applicant: Takeda Pharmaceutical Company Limited
- Applicant Address: JP Osaka
- Assignee: Takeda Pharmaceutical Company Limited
- Current Assignee: Takeda Pharmaceutical Company Limited
- Current Assignee Address: JP Osaka
- Agency: Morgan, Lewis & Bockius LLP
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K38/36 ; A61P7/04 ; A61K9/00

Abstract:
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor IX variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia B. In some embodiments, the present disclosure provides methods for dosing a hemophilia B patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor IX polypeptide.
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