公开(公告)号：US20220243201A1

公开(公告)日：2022-08-04

申请号：US17576562

申请日：2022-01-14

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Ajay Maghodia ,  Philip Zakas

IPC: C12N15/113 ,  A61K31/713 ,  A61K31/7105 ,  A61P7/04 ,  C07K14/755 ,  C12N15/86 ,  C12N7/00

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.

公开(公告)号：US20220106383A1

公开(公告)日：2022-04-07

申请号：US17479705

申请日：2021-09-20

Applicant: Bioverativ Therapeutics Inc.

Inventor： Ekta Seth CHHABRA ,  John KULMAN ,  Tongyao LIU

IPC: C07K14/755 ,  A61K38/37

Abstract: The present invention provides a chimeric molecule comprising a VWF protein fused to a heterologous moiety via a VWF linker. The invention provides an efficient VWF linker that can be cleaved in the presence of thrombin. The chimeric molecule can further comprise a polypeptide chain comprising a FVIII protein and a second heterologous moiety, wherein the chain comprising the VWF protein and the chain comprising the FVIII protein are associated with each other. The invention also includes nucleotides, vectors, host cells, methods of using the chimeric proteins.

公开(公告)号：US11266720B2

公开(公告)日：2022-03-08

申请号：US16270302

申请日：2019-02-07

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Jennifer A. Dumont ,  Susan Low ,  Alan J. Bitonti ,  Glenn Pierce ,  Alvin Luk ,  Haiyan Jiang ,  Byron McKinney ,  Matt Ottmer ,  Jurg Sommer ,  Karen Nugent ,  Lian Li ,  Robert Peters

IPC: C07K14/755 ,  A61K38/37 ,  A61K47/64 ,  A61K47/68 ,  C07K16/46

Abstract: The present invention provides methods of administering Factor VIII; methods of administering chimeric and hybrid polypeptides comprising Factor VIII; chimeric and hybrid polypeptides comprising Factor VIII; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells.

公开(公告)号：US20220064622A1

公开(公告)日：2022-03-03

申请号：US17378200

申请日：2021-07-16

Applicant: Bioverativ Therapeutics Inc.

Inventor： Glenn PIERCE ,  Samantha TRUEX ,  Robert T. PETERS ,  Haiyan JIANG ,  Mark BRADER

IPC: C12N9/64 ,  C07K14/745 ,  C07K16/36

Abstract: The present invention provides methods of administering long-acting Factor IX; methods of administering long-acting, chimeric and hybrid polypeptides comprising Factor IX; and methods of producing such chimeric and hybrid polypeptides using cells.

公开(公告)号：US11225650B2

公开(公告)日：2022-01-18

申请号：US15619196

申请日：2017-06-09

Applicant: Bioverativ Therapeutics Inc.

Inventor： Glenn Pierce ,  Samantha Truex ,  Robert T. Peters ,  Haiyan Jiang ,  Mark Brader

IPC: A61K38/36 ,  C12N9/64 ,  C07K14/745 ,  C07K16/36 ,  A61K38/00

Abstract: The present invention provides methods of administering long-acting Factor IX; methods of administering long-acting, chimeric and hybrid polypeptides comprising Factor IX; and methods of producing such chimeric and hybrid polypeptides using cells.

公开(公告)号：US20210355473A1

公开(公告)日：2021-11-18

申请号：US17222636

申请日：2021-04-05

Applicant: Bioverativ Therapeutics Inc.

Inventor： Joe SALAS ,  Robert PETERS ,  Alan BITONTI

IPC: C12N9/64 ,  C07K14/745 ,  C07K16/28 ,  C07K14/755 ,  C12N9/96 ,  C07K16/18

Abstract: Chimeric clotting factors which localize the therapeutic to sites of coagulation (e.g., by being targeted to platelets or being activatable at sites of coagulation), have reduced clearance rates, have improved manufacturability, have reduced thrombogenicity, have enhanced activity, or have more than one of these characteristics are described as are methods for making chimeric clotting factors and methods for improving hemostasis using these clotting factors.

公开(公告)号：US20210115425A1

公开(公告)日：2021-04-22

申请号：US17060759

申请日：2020-10-01

Applicant: Bioverativ Therapeutics Inc.

Inventor： Siyuan TAN ,  Robert T. PETERS ,  Tongyao LIU

IPC: C12N9/64

Abstract: The present invention provides codon optimized Factor IX sequences, vectors and host cells comprising codon optimized Factor IX sequences, polypeptides encoded by codon optimized Factor IX sequences, and methods of producing such polypeptides. The present invention also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor IX nucleic acid sequence or the polypeptide encoded thereby.

公开(公告)号：US20210023185A1

公开(公告)日：2021-01-28

申请号：US17032354

申请日：2020-09-25

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Glenn PIERCE ,  Samantha TRUEX ,  Robert T. PETERS ,  Haiyan JIANG

IPC: A61K38/48 ,  A61P7/04 ,  A61K47/64 ,  A61K38/38 ,  A61K39/395 ,  C07K16/18 ,  C12N9/64 ,  C07K14/76 ,  A61K9/00 ,  C12N9/96

Abstract: The present invention provides methods of administering Factor IX; methods of administering chimeric and hybrid polypeptides comprising Factor IX; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells.

公开(公告)号：US10898554B1

公开(公告)日：2021-01-26

申请号：US17032354

申请日：2020-09-25

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Glenn Pierce ,  Samantha Truex ,  Robert T. Peters ,  Haiyan Jiang

IPC: A61K38/36 ,  A61K38/48 ,  A61P7/04 ,  A61K47/64 ,  A61K38/38 ,  A61K39/395 ,  C07K16/18 ,  C12N9/64 ,  C07K14/76 ,  A61K9/00 ,  C12N9/96

Abstract: The present invention provides methods of administering Factor IX; methods of administering chimeric and hybrid polypeptides comprising Factor IX; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells.

公开(公告)号：US10881742B2

公开(公告)日：2021-01-05

申请号：US15991629

申请日：2018-05-29

Applicant: Bioverativ Therapeutics Inc.

Inventor： Jennifer A. Dumont ,  Susan Low ,  Alan J. Bitonti ,  Glenn Pierce ,  Alvin Luk ,  Haiyan Jiang ,  Byron McKinney ,  Matt Ottmer ,  Jurg Sommer ,  Karen Nugent ,  Lian Li ,  Robert T. Peters

IPC: A61K38/37 ,  A61K47/68 ,  A61K47/60 ,  C07K14/755 ,  G01N33/86

Abstract: The present invention provides methods of administering Factor VIII (processed FVIII, single chain FVIII, or a combination thereof); methods of administering chimeric and hybrid polypeptides comprising Factor VIII; chimeric and hybrid polypeptides comprising Factor VIII; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells.