Patents Assigned to BioMarin Technologies B.V.
  • Patent number: 10533171
    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.
    Type: Grant
    Filed: March 24, 2017
    Date of Patent: January 14, 2020
    Assignee: BioMarin Technologies B.V.
    Inventors: Judith Christina Theodora Van Deutekom, Josephus Johannes De Kimpe, Gerard Johannes Platenburg
  • Patent number: 10246707
    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) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
    Type: Grant
    Filed: September 21, 2015
    Date of Patent: April 2, 2019
    Assignees: Biomarin Technologies B.V., Academisch Ziekenhuis Leiden
    Inventors: Gerard Johannes Platenburg, Josephus Johannes de Kimpe, Judith Christina Theodora van Deutekom, Garrit-Jan Boudewijn van Ommen, Annemieke Aartsma-Rus
  • Patent number: 10179912
    Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.
    Type: Grant
    Filed: August 9, 2016
    Date of Patent: January 15, 2019
    Assignee: BioMarin Technologies B.V.
    Inventors: Peter Christian de Visser, Judith Christina Theodora van Deutekom
  • Patent number: 9926557
    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 isolated 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.
    Type: Grant
    Filed: April 26, 2011
    Date of Patent: March 27, 2018
    Assignees: BioMarin Technologies B.V., Academisch Ziekenhuis Leiden
    Inventors: Josephus Johannes De Kimpe, Gerardus Johannes Platenburg, Judith Christina Theodora Van Deutekom, Annemieke Aartsma-Rus, Garrit-Jan Boudewijn Van Ommen
  • Patent number: 9890379
    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.
    Type: Grant
    Filed: July 27, 2015
    Date of Patent: February 13, 2018
    Assignee: BioMarin Technologies B.V.
    Inventors: Josephus Johannes De Kimpe, Gerard Johannes Platenburg, Derick Gert Wansink
  • Patent number: 9745576
    Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.
    Type: Grant
    Filed: October 23, 2014
    Date of Patent: August 29, 2017
    Assignee: BIOMARIN TECHNOLOGIES B.V.
    Inventors: Peter Christian de Visser, Susan Allegonda Maria Mulders
  • Patent number: 9528109
    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 isolated muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within the exon. The invention further relates to such molecule used in the method.
    Type: Grant
    Filed: November 14, 2014
    Date of Patent: December 27, 2016
    Assignees: BioMarin Technologies B.V., Academisch Ziekenhuis Leiden
    Inventors: Josephus Johannes De Kimpe, Adriana Marie Rus, Gerard Johannes Platenburg, Judith Christina Theodora Van Deutekom, Garrit-Jan Boudewijn Van Ommen
  • Patent number: 9499818
    Abstract: The invention relates to a method where a molecule is used for inducing and/or promoting the skipping of at least one of the exons 51-53, 55, 57 and 59 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient. The method comprising providing the cell and/or the patient with a molecule. The invention also relates to the molecule and its composition that is being used for inducing exon skipping.
    Type: Grant
    Filed: February 25, 2015
    Date of Patent: November 22, 2016
    Assignees: BioMarin Technologies, B.V., Academisch Ziekenhuis Leiden
    Inventor: Judith Christina Theodora van Deutekom
  • Patent number: 9243245
    Abstract: The invention provides means and methods for alleviating one or more symptom(s) of Duchenne Muscular Dystrophy and/or Becker Muscular Dystrophy. Therapies using compounds for providing patients with functional muscle proteins are combined with at least one adjunct compound for reducing inflammation, preferably for reducing muscle tissue inflammation, and/or at least one adjunct compound for improving muscle fiber function, integrity and/or survival.
    Type: Grant
    Filed: April 26, 2010
    Date of Patent: January 26, 2016
    Assignees: Academisch Ziekenhuis Leiden, BioMarin Technologies B.V.
    Inventors: Josephus Johannes De Kimpe, Gerard Johannes Platenburg, Judith Christina Theodora Van Deutekom, Annemieke Aartsma-Rus, Garrit-Jan Boudewijn Van Ommen