公开(公告)号：US10851058B2

公开(公告)日：2020-12-01

申请号：US15740437

申请日：2016-07-01

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Shripad Bhagwat ,  Gregory Luedtke ,  Alexander Bridges

IPC: C07D223/08 ,  C07D223/12 ,  C07D451/04 ,  C07D451/06 ,  C07D205/04 ,  C07D207/12 ,  C07D207/14 ,  C07D211/46 ,  C07D211/58 ,  A61P25/28 ,  C07D211/54 ,  C07D451/02

Abstract: Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).

公开(公告)号：US20190263775A1

公开(公告)日：2019-08-29

申请号：US16264505

申请日：2019-01-31

Applicant: BioMarin Pharmaceutical Inc.

Inventor： Bing Wang ,  Daniel Chu ,  Alexander James Bridges

IPC: C07D319/18 ,  C07D305/08 ,  C07D405/14 ,  C07D311/58 ,  C07D333/24 ,  C07D333/60 ,  C07D405/06 ,  C07D471/04 ,  C07D471/08 ,  C07D487/08 ,  C07B59/00 ,  C07D309/12 ,  C07D309/10 ,  C07D211/46 ,  C07D213/56 ,  C07D213/61 ,  C07D215/06 ,  C07D235/08 ,  C07D277/30 ,  C07D295/13 ,  C07D491/056 ,  C07D307/81 ,  C07D307/79 ,  C07D213/64 ,  C07D213/46 ,  C07D295/12 ,  C07D277/62 ,  C07D209/04 ,  C07D277/22 ,  C07D265/30 ,  C07D205/04 ,  C07D417/12 ,  C07D413/12 ,  C07D409/12 ,  C07D407/12 ,  C07D405/12 ,  C07D235/06 ,  C07D333/62 ,  C07D231/56 ,  C07D333/16 ,  C07D311/12

Abstract: Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).

公开(公告)号：US20190216905A1

公开(公告)日：2019-07-18

申请号：US16368091

申请日：2019-03-28

Applicant: BioMarin Pharmaceutical Inc.

Inventor： Thomas Lester ,  Saeed Moshashaee ,  Augustus O. Okhamafe ,  Charles A. O'Neill

IPC: A61K38/48 ,  A61K9/08 ,  A61M5/14 ,  C12N9/48 ,  A61K47/02 ,  A61K9/00

Abstract: Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.

公开(公告)号：US10308608B2

公开(公告)日：2019-06-04

申请号：US16047179

申请日：2018-07-27

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Shripad Bhagwat ,  Gregory Luedtke ,  Alexander Bridges

IPC: C07D205/04 ,  C07D207/12 ,  C07D207/14 ,  C07D211/46 ,  C07D211/58 ,  C07D223/08 ,  C07D223/12 ,  C07D451/04 ,  C07D451/06 ,  C07D451/02

Abstract: Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).

公开(公告)号：US20190134005A1

公开(公告)日：2019-05-09

申请号：US16308414

申请日：2017-06-09

Applicant: BioMarin Pharmaceutical Inc.

Inventor： Bing Wang

IPC: A61K31/4166 ,  A61K31/4178

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 ceramide galactosyltransferase (CGT), such as, for example, lysosomal storage diseases. Examples of lysosomal storage diseases include, for example, Krabbe disease and Metachromatic Leukodystrophy.

公开(公告)号：US20180370987A1

公开(公告)日：2018-12-27

申请号：US16046382

申请日：2018-07-26

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Vincent Jacques ,  James R. Rusche ,  Norton P. Peet ,  Jasbir Singh

IPC: C07D513/04 ,  C07D401/06 ,  C07D211/34 ,  C07D403/06 ,  C07D471/04 ,  C07D263/32 ,  C07D231/12 ,  C07D277/593 ,  C07D261/08

Abstract: This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).

公开(公告)号：US20180362472A1

公开(公告)日：2018-12-20

申请号：US16009311

申请日：2018-06-15

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Vincent Jacques ,  James R. Rusche

IPC: C07D231/56 ,  C07D401/06

CPC classification number: C07D231/56 ,  C07D401/06

Abstract: Disclosed herein are compounds of formula (I), and methods of inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3) using compounds of formula (I).

公开(公告)号：US20180208606A1

公开(公告)日：2018-07-26

申请号：US15860741

申请日：2018-01-03

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Vincent Jacques ,  James R. Rusche ,  Norton P. Peet ,  Jasbir Singh

IPC: C07D513/04 ,  C07D401/06 ,  C07D471/08 ,  C07D231/12 ,  C07D471/04 ,  C07D403/10 ,  C07D211/78 ,  C07D451/02 ,  C07D277/30 ,  C07D205/06 ,  C07D305/10 ,  C07D211/70 ,  C07D277/34 ,  C07D263/32 ,  C07D261/08 ,  C07D403/06

Abstract: This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).

公开(公告)号：US20180207239A1

公开(公告)日：2018-07-26

申请号：US15880002

申请日：2018-01-25

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： SHERRY BULLENS ,  STUART BUNTING ,  TIANWEI CHOU ,  AUGUSTUS O. OKHAMAFE ,  CHRISTOPHER P. PRICE ,  DANIEL J. WENDT ,  CLARENCE YAP

IPC: A61K38/22 ,  A61K47/26 ,  A61K47/18 ,  A61K47/12 ,  A61K9/00 ,  A61K38/17 ,  A61K9/19 ,  A61K47/10

Abstract: The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.

公开(公告)号：US09908899B2

公开(公告)日：2018-03-06

申请号：US15337354

申请日：2016-10-28

Applicant: BIOMARIN PHARMACEUTICAL INC.

Inventor： Vincent Jacques ,  James R. Rusche ,  Norton P. Peet ,  Jasbir Singh

IPC: A61K31/397 ,  A61K31/46 ,  A61K31/4427 ,  A61K31/445 ,  A61K31/337 ,  C07D513/04 ,  C07D263/32 ,  C07D471/04 ,  C07D231/12 ,  C07D261/08 ,  C07D211/70 ,  C07D403/10 ,  C07D403/06 ,  C07D211/78 ,  C07D451/02 ,  C07D305/10 ,  C07D205/06 ,  C07D401/06 ,  C07D471/08 ,  C07D277/34 ,  C07D277/30

CPC classification number: C07D513/04 ,  C07D205/06 ,  C07D211/70 ,  C07D211/78 ,  C07D231/12 ,  C07D261/08 ,  C07D263/32 ,  C07D277/30 ,  C07D277/34 ,  C07D305/10 ,  C07D401/06 ,  C07D403/06 ,  C07D403/10 ,  C07D451/02 ,  C07D471/04 ,  C07D471/08

Abstract: The compounds provided herein are phenyl analine amides and are useful for inhibiting histone deacteylase (“HDAC”) enzymes, such as HDAC1, HDAC2, and HDAC3.