公开(公告)号：US20210207138A1

公开(公告)日：2021-07-08

申请号：US17119806

申请日：2020-12-11

Applicant: BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

Inventor： Gerard Johannes Platenburg ,  Josephus Johannes De Kimpe ,  Judith Christina Theodora Van Deutekom ,  Garrit-Jan Boudewijn Van Ommen ,  Annemieke Aartsma-Rus

IPC: C12N15/113

Abstract: The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5′-GUGGCUAACAGAAGCU (SEQ ID NO:1), 5′-GGGAACAUGCUAAAUAC (SEQ ID NO:2), 5′-AGACACAAAUUCCUGAGA (SEQ ID NO:3), or 5′-CUGUUGAGAAA (SEQ ID NO. 4), and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.

公开(公告)号：US20190194656A1

公开(公告)日：2019-06-27

申请号：US16201951

申请日：2018-11-27

Applicant: BioMarin Technologies B.V.

Inventor： Peter Christian de Visser ,  Judith Christina Theodora van Deutekom

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/331 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2320/33 ,  C12N2310/3521

Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.

公开(公告)号：US20190177725A1

公开(公告)日：2019-06-13

申请号：US16283458

申请日：2019-02-22

Applicant: BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

Inventor： Josephus Johannes De Kimpe ,  Adriana Marie Rus ,  Gerard Johannes Platenburg ,  Judith Christina Theodora Van Deutekom ,  Garrit-Jan Boudewijn Van Ommen

IPC: C12N15/113 ,  A61K38/17 ,  A61K45/06 ,  A61K31/7088 ,  A61K31/56 ,  A61K31/58 ,  A61K31/573 ,  A61K31/57

Abstract: The. invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

公开(公告)号：US20190119679A1

公开(公告)日：2019-04-25

申请号：US16229821

申请日：2018-12-21

Applicant: BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

Inventor： Josephus Johannes De Kimpe ,  Adriana Marie Rus ,  Gerard Johannes Platenburg ,  Judith Christina Theodora Van Deutekom ,  Garrit-Jan Boudewijn Van Ommen

IPC: C12N15/113 ,  A61K31/57 ,  A61K38/17 ,  A61K45/06 ,  A61K31/7088 ,  A61K31/56 ,  A61K31/58 ,  A61K31/573

Abstract: The. invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

公开(公告)号：US10179912B2

公开(公告)日：2019-01-15

申请号：US15232493

申请日：2016-08-09

Applicant: BioMarin Technologies B.V.

Inventor： Peter Christian de Visser ,  Judith Christina Theodora van Deutekom

IPC: C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125

Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.

公开(公告)号：US09890379B2

公开(公告)日：2018-02-13

申请号：US14809483

申请日：2015-07-27

Applicant: BioMarin Technologies B.V.

Inventor： Josephus Johannes De Kimpe ,  Gerard Johannes Platenburg ,  Derick Gert Wansink

IPC: C12N15/11 ,  C12N15/113 ,  C12Q1/68 ,  A61K48/00

CPC classification number: C12N15/11 ,  A61K48/00 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/346 ,  C12Q1/6883 ,  C12Q2600/156 ,  C12N2310/3521

Abstract: The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.

公开(公告)号：US20170107512A1

公开(公告)日：2017-04-20

申请号：US15390836

申请日：2016-12-27

Applicant: BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

Inventor： Josephus Johannes De KIMPE ,  Adriana Marie RUS ,  Gerard Johannes PLATENBURG ,  Judith Christina Theodora Van DEUTEKOM ,  Garrit-Jan Boudewijn Van OMMEN

IPC: C12N15/113

CPC classification number: C12N15/113 ,  A61K31/56 ,  A61K31/57 ,  A61K31/573 ,  A61K31/58 ,  A61K31/7088 ,  A61K38/1719 ,  A61K45/06 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/313 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/33 ,  A61K2300/00

Abstract: The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.

公开(公告)号：US20210254071A1

公开(公告)日：2021-08-19

申请号：US17302500

申请日：2021-05-04

Applicant: BioMarin Technologies B.V.

Inventor： Judith Christina Theodora van Deutekom ,  Josephus Johannes de Kimpe ,  Gerard Johannes Platenburg

IPC: C12N15/113 ,  C12N15/11 ,  A61K48/00

Abstract: The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.

公开(公告)号：US11034956B2

公开(公告)日：2021-06-15

申请号：US16694980

申请日：2019-11-25

Applicant: BioMarin Technologies B.V.

Inventor： Judith Christina Theodora Van Deutekom ,  Josephus Johannes De Kimpe ,  Gerard Johannes Platenburg

IPC: C07H21/04 ,  C12N15/113 ,  C12N15/11 ,  A61K48/00

Abstract: The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.

公开(公告)号：US20210139904A1

公开(公告)日：2021-05-13

申请号：US17129117

申请日：2020-12-21

Applicant: BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

Inventor： Judith Christina Theodora Van Deutekom

IPC: C12N15/113 ,  A61K31/56 ,  A61K31/57 ,  A61K38/17 ,  A61K31/7088 ,  A61K45/06 ,  A61K31/573 ,  A61K31/58

Abstract: The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.