公开(公告)号：US11596671B2

公开(公告)日：2023-03-07

申请号：US16777630

申请日：2020-01-30

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Franziska Horling ,  Johannes Lengler ,  Friedrich Scheiflinger

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.

公开(公告)号：US20240269241A1

公开(公告)日：2024-08-15

申请号：US18569950

申请日：2022-06-14

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner

IPC: A61K38/37 ,  A61K9/00 ,  A61K31/573 ,  A61K48/00 ,  C12N15/86 ,  G01N33/68 ,  G01N33/86

CPC classification number: A61K38/37 ,  A61K9/0019 ,  A61K31/573 ,  A61K48/005 ,  C12N15/86 ,  G01N33/6854 ,  G01N33/86 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2800/22 ,  G01N2800/52

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.

公开(公告)号：US11999798B2

公开(公告)日：2024-06-04

申请号：US17404459

申请日：2021-08-17

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Dirk Voelkel ,  Robert Pachlinger ,  Hanspeter Rottensteiner ,  Alfred Weber ,  Andrea Engelmaier

IPC: C07K16/36 ,  C07K16/00 ,  G01N33/573 ,  G01N33/86

CPC classification number: C07K16/36 ,  C07K16/005 ,  G01N33/573 ,  G01N33/86 ,  C07K2317/21 ,  C07K2317/33 ,  C07K2317/34 ,  C07K2317/35 ,  C07K2317/55 ,  C07K2317/76 ,  C07K2317/92 ,  C07K2319/21 ,  C07K2319/43 ,  G01N2333/9645

Abstract: Provided herein are anti-Factor IX Padua binding constructs, e.g., antibodies and antigen-binding fragments thereof. Related polypeptides, conjugates and kits are also provided. The inventions may be used in methods of detecting Factor IX Padua in a sample.

公开(公告)号：US11707536B2

公开(公告)日：2023-07-25

申请号：US16743850

申请日：2020-01-15

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Friedrich Scheiflinger

IPC: A61K48/00 ,  A61K31/711 ,  C07K14/755 ,  C12N15/86

CPC classification number: A61K48/0058 ,  A61K31/711 ,  C07K14/755 ,  C12N15/86 ,  C12N2750/14141

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.

公开(公告)号：US20220213172A1

公开(公告)日：2022-07-07

申请号：US17570187

申请日：2022-01-06

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Falko-Günter Falkner ,  Franziska Horling ,  Johannes Lengler ,  Hanspeter Rottensteiner ,  Friedrich Scheiflinger

IPC: C07K14/755 ,  A61K48/00 ,  C12N15/86

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.

公开(公告)号：US11185575B2

公开(公告)日：2021-11-30

申请号：US16567048

申请日：2019-09-11

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Alexandra Nathalie Kopic ,  Werner Hollriegl ,  Barbara Plaimauer ,  Hanspeter Rottensteiner ,  Eva-Maria Muchitsch

IPC: A61K38/48 ,  C12N9/64 ,  A61K9/00 ,  A61K9/19

Abstract: This invention relates to methods of subcutaneous administration of ADAMTS13 formulations to a treat a disease or condition associated with ADAMTS13 and VWF dysfunction. Furthermore, evidence of the unexpectedly high bioavailability of ADAMTS13 formulations administered subcutaneously is provided herein.

公开(公告)号：US11098132B2

公开(公告)日：2021-08-24

申请号：US16301962

申请日：2017-05-16

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Dirk Voelkel ,  Robert Pachlinger ,  Hanspeter Rottensteiner ,  Alfred Weber ,  Andrea Engelmaier

IPC: C07K16/36 ,  G01N33/573 ,  C07K16/00 ,  G01N33/86

Abstract: Provided herein are anti-Factor IX Padua binding constructs, e.g., antibodies and antigen-binding fragments thereof. Related polypeptides, conjugates and kits are also provided. The inventions may be used in methods of detecting Factor IX Padua in a sample.

公开(公告)号：US20240100128A1

公开(公告)日：2024-03-28

申请号：US18162978

申请日：2023-02-01

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Franziska Horling ,  Johannes Lengler ,  Friedrich Scheiflinger

IPC: A61K38/36 ,  A61K9/00 ,  A61K48/00 ,  A61P7/04

CPC classification number: A61K38/36 ,  A61K9/0019 ,  A61K48/0058 ,  A61P7/04

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.

公开(公告)号：US20230023826A1

公开(公告)日：2023-01-26

申请号：US17784093

申请日：2020-12-10

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: A61K48/00 ,  A61K31/573 ,  G01N33/86 ,  A61P7/04

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.

公开(公告)号：US11492388B2

公开(公告)日：2022-11-08

申请号：US16211201

申请日：2018-12-05

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Falko-Günter Falkner ,  Franziska Horling ,  Johannes Lengler ,  Hanspeter Rottensteiner ,  Friedrich Scheiflinger

IPC: C07K14/755 ,  A61K48/00 ,  C12N15/86

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.