公开(公告)号：US20220402974A1

公开(公告)日：2022-12-22

申请号：US16319877

申请日：2017-07-25

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Peter Cameron Colosi ,  Michael LOCHRIE ,  Robert NG

IPC: C07K14/005 ,  C12N15/86

Abstract: The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.

公开(公告)号：US10463718B2

公开(公告)日：2019-11-05

申请号：US15294310

申请日：2016-10-14

Applicant: BIOMARIN PHARMACEUTICAL INC. ,  UCL BUSINESS PLC

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny McIntosh ,  Edward Tuddenham

IPC: A01N63/00 ,  A61K38/37 ,  A61K48/00 ,  C12N7/00 ,  C07K14/755 ,  C12N15/86

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

公开(公告)号：US11406690B2

公开(公告)日：2022-08-09

申请号：US16588130

申请日：2019-09-30

Applicant: BIOMARIN PHARMACEUTICAL INC. ,  UCL BUSINESS PLC ,  ST. JUDE CHILDREN'S RESEARCH HOSPITAL

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny McIntosh ,  Edward Tuddenham ,  Andrew Davidoff

IPC: A01N63/00 ,  A61K38/37 ,  A61K48/00 ,  C12N7/00 ,  C07K14/755 ,  C12N15/86

Abstract: The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs. The disclosure also provides methods of improving the birth rate of multiplexed genetically modified animals. In some embodiments, the present closure is concerned with the generation and utilization of porcine cells in which porcine endogenous retroviral (PERV) elements have been inactivated. In sonic embodiments, the PERV-free or PERV-reduced porcine cells are cloned to produce porcine embryos. In some embodiments, the PERV-free or PERV-reduced embryos may be grown into adult swine from which organs and/or tissues may be extracted and used for such purposes as xenotransplantation into non-porcine animals such as humans.

公开(公告)号：US20170095538A1

公开(公告)日：2017-04-06

申请号：US15294310

申请日：2016-10-14

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny McIntosh ,  Edward Tuddenham

IPC: A61K38/37 ,  A61K48/00 ,  C07K14/755 ,  C12N15/86 ,  C12N7/00

CPC classification number: A61K38/37 ,  A61K48/0058 ,  A61K48/0066 ,  C07K14/755 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14132 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14151 ,  C12N2800/22 ,  C12N2830/008

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

公开(公告)号：US20170087219A1

公开(公告)日：2017-03-30

申请号：US15274046

申请日：2016-09-23

Applicant: BioMarin Pharmaceutical Inc.

Inventor： Stuart Bunting ,  Peter Cameron Colosi ,  Erno Pungor

IPC: A61K38/37 ,  G01N33/68 ,  C12N7/00 ,  A61K47/26 ,  A61K47/10 ,  A61K48/00 ,  A61K47/02

Abstract: The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.

公开(公告)号：US20240058423A1

公开(公告)日：2024-02-22

申请号：US18324594

申请日：2023-05-26

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Stuart Bunting ,  Peter Cameron Colosi ,  Erno Pungor

IPC: A61K38/37 ,  A61K39/12 ,  C07K14/755 ,  A61K47/02 ,  A61K47/10 ,  A61K47/26 ,  A61K48/00 ,  C12N7/00 ,  G01N33/68

CPC classification number: A61K38/37 ,  A61K39/12 ,  C07K14/755 ,  A61K47/02 ,  A61K47/10 ,  A61K47/26 ,  A61K48/0008 ,  A61K48/0075 ,  C12N7/00 ,  G01N33/6854 ,  C12N2710/16044 ,  C12N2750/14141 ,  C12N2750/14121 ,  C12N2750/14143 ,  G01N2333/015

Abstract: The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.

公开(公告)号：US20220339262A1

公开(公告)日：2022-10-27

申请号：US17854286

申请日：2022-06-30

Applicant: BIOMARIN PHARMACEUTICAL INC. ,  UCL BUSINESS LTD ,  ST. JUDE CHILDREN'S RESEARCH HOSPITAL

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny McIntosh ,  Edward Tuddenham ,  Andrew Davidoff

IPC: A61K38/37 ,  A61K48/00 ,  C12N7/00 ,  C07K14/755 ,  C12N15/86

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

公开(公告)号：US20200061161A1

公开(公告)日：2020-02-27

申请号：US16675841

申请日：2019-11-06

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Stuart Bunting ,  Peter Cameron Colosi ,  Erno Pungor

IPC: A61K38/37 ,  G01N33/68 ,  C12N7/00 ,  A61K48/00 ,  A61K47/26 ,  A61K47/10 ,  A61K47/02 ,  C07K14/755 ,  A61K39/12

Abstract: The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.

公开(公告)号：US09504762B2

公开(公告)日：2016-11-29

申请号：US14482648

申请日：2014-09-10

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny Mcintosh ,  Edward Tuddenham

IPC: A01N63/00 ,  A01N65/00 ,  A61K48/00 ,  C12N7/00 ,  C07K14/755 ,  C12N15/86

CPC classification number: A61K38/37 ,  A61K48/0058 ,  A61K48/0066 ,  C07K14/755 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14132 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14151 ,  C12N2800/22 ,  C12N2830/008

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

Abstract translation: 本发明提供改进的腺相关病毒（AAV）因子VIII（FVIII）载体，包括产生功能性因子VIII多肽的AAV FVIII载体和具有高表达活性的AAV FVIII载体。

公开(公告)号：US12239692B2

公开(公告)日：2025-03-04

申请号：US17854286

申请日：2022-06-30

Applicant: BIOMARIN PHARMACEUTICAL INC. ,  UCL BUSINESS LTD ,  ST. JUDE CHILDREN'S RESEARCH HOSPITAL

Inventor： Peter Cameron Colosi ,  Amit Nathwani ,  Jenny McIntosh ,  Edward Tuddenham ,  Andrew Davidoff

IPC: A01N63/00 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755 ,  C12N7/00 ,  C12N15/86

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.