公开(公告)号：US20240376495A1

公开(公告)日：2024-11-14

申请号：US18688682

申请日：2022-09-02

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Peter COLOSI ,  Vincent LEONARD ,  Silvia RAMIREZ ,  Justin ISHIDA ,  Yu-Shan TSENG ,  Teague STERLING

IPC: C12N15/86 ,  A61K48/00 ,  C07K14/005 ,  C12N7/00

Abstract: The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.

公开(公告)号：US20240318156A1

公开(公告)日：2024-09-26

申请号：US18573298

申请日：2022-06-24

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Daniel WENDT ,  Mika AOYAGI-SCHARBER ,  Roger LAWRENCE, ,  Manuel LOPEZ

IPC: C12N9/24 ,  A61K38/00

CPC classification number: C12N9/2402 ,  C12Y302/01052 ,  A61K38/00

Abstract: Disclosed herein are recombinant β-hexosaminidase variant a subunits that form a β-hexosaminidase variant a subunit homodimer that have optimized properties for use in treating Tay-Sachs disease or Sandhoff Disease.

公开(公告)号：US20240200096A1

公开(公告)日：2024-06-20

申请号：US18479761

申请日：2023-10-02

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Peter COLOSI ,  Justin ISHIDA ,  Silvia RAMIREZ

IPC: C12N15/86 ,  A61K45/06 ,  A61P7/00

CPC classification number: C12N15/86 ,  A61P7/00 ,  A61K45/06 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/48 ,  C12N2830/85

Abstract: The present disclosure relates, in general, to methods for readministering, or redosing, a subject having undergone a first gene therapy regimen with a second, or subsequent, administration of a gene therapy regimen, wherein the first gene therapy vector and second gene therapy vector comprise different AAV capsids but carry a transgene or polynucleotide useful to treat the same disease or disorder.

公开(公告)号：US20240124849A1

公开(公告)日：2024-04-18

申请号：US18273430

申请日：2022-01-27

Applicant: BioMarin Pharmaceutical Inc.

Inventor： Vishal AGRAWAL ,  Teresa CHRISTIANSON ,  Francisco Javier FEMENIA ,  Santosh G. PANDE

IPC: C12N7/00 ,  C07K14/015 ,  C12N15/86

CPC classification number: C12N7/00 ,  C07K14/015 ,  C12N15/86 ,  C12N2750/14151

Abstract: The present invention provides modified alphaviruses and compositions, methods, and kits for preparing and using, in particular AAV viral particles pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics.

公开(公告)号：US11918633B2

公开(公告)日：2024-03-05

申请号：US17747697

申请日：2022-05-18

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Debra Lounsbury

IPC: C12N9/88 ,  A61K38/51 ,  A61P3/00

CPC classification number: A61K38/51 ,  A61P3/00 ,  C12Y403/01024

Abstract: Phenylalanine ammonia-lyase (PAL) variants with a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL for therapeutic uses, including the treatment of adolescent subjects having PKU.

公开(公告)号：US20230322864A1

公开(公告)日：2023-10-12

申请号：US18063589

申请日：2022-12-08

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Peter Cameron COLOSI ,  Michael LOCHRIE ,  Robert NG

IPC: C07K14/005 ,  C12N15/86 ,  A61P21/00

CPC classification number: C07K14/005 ,  C12N15/86 ,  A61P21/00 ,  C12N2750/14122 ,  C12N2750/14143

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：US20230192799A1

公开(公告)日：2023-06-22

申请号：US17642150

申请日：2020-09-16

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Daniel J. Wendt ,  Geoffrey Berguig ,  Karol Estrada ,  Jonathan LeBowitz

IPC: C07K14/58 ,  A61K47/54 ,  A61P19/08

CPC classification number: C07K14/58 ,  A61K47/542 ,  A61P19/08 ,  A61K38/00

Abstract: The present disclosure, relates, in general, to stable variants of C-type natriuretic peptide (CNP) and uses thereof to treat bone-related disorders.

公开(公告)号：US20230089374A1

公开(公告)日：2023-03-23

申请号：US17751144

申请日：2022-05-23

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Bing WANG ,  Qi CHAO

IPC: C07D249/04 ,  C07D401/12 ,  C07D401/14 ,  C07D403/12 ,  C07D405/12 ,  C07D409/12 ,  C07D417/12

Abstract: Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO). Such diseases or disorders include, for example, disorders of glyoxylate metabolism, including primary hyperoxaluria, that are associated with production of excessive amounts of oxalate.

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：US11345904B2

公开(公告)日：2022-05-31

申请号：US16078546

申请日：2017-02-24

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Saeed Moshashaee ,  Jason K. Pinkstaff ,  Adam Shaywitz ,  Natalie Ciaccio

IPC: C12N9/26 ,  C12N9/24 ,  A61K47/65 ,  A61P25/28 ,  A61K9/00 ,  A61K38/43 ,  A61K47/26

Abstract: The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lysosomal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.