公开(公告)号：US12091675B2

公开(公告)日：2024-09-17

申请号：US17296944

申请日：2019-07-15

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: C12N15/861 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755

CPC classification number: C12N15/861 ,  A61K38/37 ,  A61K48/0083 ,  C07K14/755 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0381

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：US20230211017A1

公开(公告)日：2023-07-06

申请号：US18145784

申请日：2022-12-22

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Friedrich Scheiflinger

IPC: A61K48/00 ,  A61P7/04

CPC classification number: A61K48/0066 ,  A61K48/0058 ,  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.

公开(公告)号：US20250043310A1

公开(公告)日：2025-02-06

申请号：US18797893

申请日：2024-08-08

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: C12N15/861 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755

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 VII polypeptide.

公开(公告)号：US11779635B2

公开(公告)日：2023-10-10

申请号：US17525759

申请日：2021-11-12

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

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

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

CPC classification number: A61K38/4886 ,  A61K9/0019 ,  A61K9/19 ,  C12N9/6489 ,  C12Y304/24087

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：US11564992B2

公开(公告)日：2023-01-31

申请号：US16748296

申请日：2020-01-21

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Juergen Siekmann ,  Stefan Haider ,  Hanspeter Rottensteiner ,  Peter Turecek

IPC: A61K47/64 ,  C07K1/107 ,  A61K47/60 ,  A61K47/54 ,  A61K47/61 ,  A61K47/59 ,  A61K51/08

Abstract: The invention relates to materials and methods of conjugating a water soluble polymer to an oxidized carbohydrate moiety of a therapeutic protein comprising contacting the oxidized carbohydrate moiety with an activated water soluble polymer under conditions that allow conjugation. More specifically, the present invention relates to the aforementioned materials and methods wherein the water soluble polymer contains an active aminooxy group and wherein an oxime or hydrazone linkage is formed between the oxidized carbohydrate moiety and the active aminooxy group on the water soluble polymer, and wherein the conjugation is carried out in the presence of a nucleophilic catalyst.

公开(公告)号：US11254731B2

公开(公告)日：2022-02-22

申请号：US16211202

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.