Patents Assigned to Uniqure IP B.V.
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Patent number: 10837027Abstract: The present invention relates to the production of adeno-associated viral vectors in insect cells. The insect cells therefore comprise a first nucleotide sequence encoding the adeno-associated virus (AAV) capsid proteins, whereby the initiation codon for translation of the AAV VP1 capsid protein is a non-ATG, suboptimal initiation codon and wherein the coding sequence for one or more amino acid residues have been inserted between the suboptimal translation initiation codon and the codon encoding the amino acid residue that corresponds to the amino acid residue at position 2 of the wild type capsid amino acid sequence of which the first amino acid residue is alanine, glycine valine, aspartic acid or glutamic acid.Type: GrantFiled: March 10, 2015Date of Patent: November 17, 2020Assignee: UNIQURE IP B.V.Inventors: Jacek Lubelski, Sebastiaan Menno Bosma, Harald Peter Albert Petry, Wilhelmus Theodorus Johannes Maria Christiaan Hermens
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Publication number: 20200339992Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.Type: ApplicationFiled: July 15, 2020Publication date: October 29, 2020Applicant: Uniqure IP B.V.Inventors: Pavlina Stefanova KONSTANTINOVA, Jana MINIARIKOVÁ
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Patent number: 10767180Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.Type: GrantFiled: November 16, 2018Date of Patent: September 8, 2020Assignee: UNIQURE IP B.V.Inventors: Pavlina Stefanova Konstantinova, Jana Miniariková
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Publication number: 20200199625Abstract: The current invention relates to gene therapy approaches for the treatment of SCA3, in particular RNAi based gene therapy approaches utilizing a total knockdown approach. The inventors provide for selected target regions and/or target sequences for which highly efficient knockdown of the ATXN3 gene expression can be advantageously obtained in human neuronal cells and in mouse models relevant for SCA3.Type: ApplicationFiled: November 14, 2019Publication date: June 25, 2020Applicant: uniQure IP B.V.Inventors: Melvin Maurice EVERS, Pavlina Stefanova KONSTANTINOVA, Raygene Michael MARTIER
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VECTORS WITH MODIFIED INITIATION CODON FOR THE TRANSLATION OF AAV-REP78 USEFUL FOR PRODUCTION OF AAV
Publication number: 20200131535Abstract: The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions. The insect cells preferably comprise a first nucleotide sequence encoding the parvoviral rep proteins whereby the initiation codon for translation of the parvoviral Rep78 protein is a suboptimal initiation codon that effects partial exon skipping upon expression in insect cells. The insect cell further comprises a second nucleotide sequence comprising at least one parvoviral (AA V) inverted terminal repeat (ITR) nucleotide sequence and a third nucleotide sequence comprising a sequences coding for the parvoviral capsid proteins.Type: ApplicationFiled: January 10, 2020Publication date: April 30, 2020Applicant: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan Hermens, Saskia Jacoba Petronella Haast, Dennis Johan Biesmans, Andrew Christian Bakker -
Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
Patent number: 10533188Abstract: The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions. The insect cells preferably comprise a first nucleotide sequence encoding the parvoviral rep proteins whereby the initiation codon for translation of the parvoviral Rep78 protein is a suboptimal initiation codon that effects partial exon skipping upon expression in insect cells. The insect cell further comprises a second nucleotide sequence comprising at least one parvoviral (AAV) inverted terminal repeat (ITR) nucleotide sequence and a third nucleotide sequence comprising a sequences coding for the parvoviral capsid proteins.Type: GrantFiled: November 2, 2018Date of Patent: January 14, 2020Assignee: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan Hermens, Saskia Jacoba Petronella Haast, Dennis Johan Biesmans, Andrew Christian Bakker -
Publication number: 20190338254Abstract: Nucleic acids encoding Parvoviral Rep proteins with a mutated nuclear localization signal (NLS) are provided. Also provided is a nucleic acid comprising a nucleotide sequence encoding a Parvoviral Rep protein with a mutated zinc finger domain and a nucleic acid comprising a nucleotide sequence encoding a Parvoviral Rep protein comprising an amino acid mutation at position 43, 57, 79, 97, 120, 179, 305, 484, 493 or 571 with reference to SEQ ID NO: 2. Nucleic acid constructs and cells, such as insect cells, comprising the nucleic acids are provided as well as a method for producing a recombinant Parvoviral virion using the nucleic acids.Type: ApplicationFiled: July 17, 2019Publication date: November 7, 2019Applicant: uniQure IP B.V.Inventors: Yvet NOORDMAN, Jacek LUBELSKI, Andrew Christian BAKKER
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Publication number: 20190269800Abstract: Upon administration of rAAV vectors the humoral immune response (neutralizing antibodies) is the first barrier that needs to be overcome. Surprisingly it was found that by using immunoadsorption for depletion of immunoglobulins from the blood (plasma), subjects can be highly efficiently treated with rAAV vectors, i.e. obtain highly efficient transduction after rAAV vector administration, in spite of the presence of high levels of nAb.Type: ApplicationFiled: April 15, 2019Publication date: September 5, 2019Applicant: uniQure IP B.V.Inventor: Valerie FERREIRA
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Patent number: 10400221Abstract: Nucleic acids encoding Parvoviral Rep proteins with a mutated nuclear localization signal (NLS) are provided. Also provided is a nucleic acid comprising a nucleotide sequence encoding a Parvoviral Rep protein with a mutated zinc finger domain and a nucleic acid comprising a nucleotide sequence encoding a Parvoviral Rep protein comprising an amino acid mutation at position 43, 57, 79, 97, 120, 179, 305, 484, 493 or 571 with reference to SEQ ID NO: 2. Nucleic acid constructs and cells, such as insect cells, comprising the nucleic acids are provided as well as a method for producing a recombinant Parvoviral virion using the nucleic acids.Type: GrantFiled: January 5, 2018Date of Patent: September 3, 2019Assignee: Uniqure IP B.V.Inventors: Yvet Noordman, Jacek Lubelski, Andrew Christian Bakker
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Publication number: 20190218523Abstract: The present invention relates to a separation of viruses of an essentially spherical shape from viruses with a rod-like shape that are comprised in a sample, wherein the sample comprising the viruses is subjected to filtration.Type: ApplicationFiled: March 20, 2019Publication date: July 18, 2019Applicant: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan HERMENS, James Patrick SMITH
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Publication number: 20190203183Abstract: The present invention relates to production of proteins in insect cells whereby repeated coding sequences are used in baculoviral vectors. In particular the invention relates to the production of parvoviral vectors that may be used in gene therapy and to improvements in expression of the viral rep proteins that increase the productivity of parvoviral vectors.Type: ApplicationFiled: December 11, 2018Publication date: July 4, 2019Applicant: uniQure IP B.V.Inventors: Andrew Christian BAKKER, Wilhelmus Johannes Theodorus Maria Christiaan HERMENS
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VECTORS WITH MODIFIED INITIATION CODON FOR THE TRANSLATION OF AAV-REP78 USEFUL FOR PRODUCTION OF AAV
Publication number: 20190153473Abstract: The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions. The insect cells preferably comprise a first nucleotide sequence encoding the parvoviral rep proteins whereby the initiation codon for translation of the parvoviral Rep78 protein is a suboptimal initiation codon that effects partial exon skipping upon expression in insect cells. The insect cell further comprises a second nucleotide sequence comprising at least one parvoviral (AAV) inverted terminal repeat (ITR) nucleotide sequence and a third nucleotide sequence comprising a sequences coding for the parvoviral capsid proteins.Type: ApplicationFiled: November 2, 2018Publication date: May 23, 2019Applicant: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan HERMENS, Saskia Jacoba Petronella HAAST, Dennis Johan BIESMANS, Andrew Christian BAKKER -
Publication number: 20190144860Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.Type: ApplicationFiled: November 16, 2018Publication date: May 16, 2019Applicant: uniQure IP B.V.Inventors: Pavlina Stefanova Konstantinova, Jana Miniariková
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Patent number: 10286087Abstract: Upon administration of rAAV vectors the humoral immune response (neutralizing antibodies) is the first barrier that needs to be overcome. Surprisingly it was found that by using immunoadsorption for depletion of immunoglobulins from the blood (plasma), subjects can be highly efficiently treated with rAAV vectors, i.e. obtain highly efficient transduction after rAAV vector administration, in spite of the presence of high levels of nAb.Type: GrantFiled: December 15, 2017Date of Patent: May 14, 2019Assignee: UNIQURE IP B.V.Inventor: Valerie Ferreira
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Patent number: 10253301Abstract: The present invention relates to a separation of viruses of an essentially spherical shape from viruses with a rod-like shape that are comprised in a sample, wherein the sample comprising the viruses is subjected to filtration.Type: GrantFiled: December 8, 2017Date of Patent: April 9, 2019Assignee: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan Hermens, James Patrick Smith
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Patent number: 10190098Abstract: The present invention relates to production of proteins in insect cells whereby repeated coding sequences are used in baculoviral vectors. In particular the invention relates to the production of parvoviral vectors that may be used in gene therapy and to improvements in expression of the viral rep proteins that increase the productivity of parvoviral vectors.Type: GrantFiled: September 30, 2015Date of Patent: January 29, 2019Assignee: UniQure IP B.V.Inventors: Andrew Christian Bakker, Wilhelmus Theodorus Johannes Maria C Hermens
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Publication number: 20190008909Abstract: The present invention relates to means and method for AAV based gene therapies in humans. In particular, the present invention relates to the treatment of human patients that may be suspected to have antibodies directed against the AAV intended for use in the treatment.Type: ApplicationFiled: July 10, 2018Publication date: January 10, 2019Applicant: uniQure IP B.V.Inventors: Bart Antonius NIJMEIJER, Valerie FERREIRA
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Patent number: 10174321Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.Type: GrantFiled: December 23, 2015Date of Patent: January 8, 2019Assignee: UNIQURE IP B.V.Inventors: Pavlina Stefanova Konstantinova, Jana Miniariková
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Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
Patent number: 10138496Abstract: The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions. The insect cells preferably comprise a first nucleotide sequence encoding the parvoviral rep proteins whereby the initiation codon for translation of the parvoviral Rep78 protein is a suboptimal initiation codon that effects partial exon skipping upon expression in insect cells. The insect cell further comprises a second nucleotide sequence comprising at least one parvoviral (AA V) inverted terminal repeat (ITR) nucleotide sequence and a third nucleotide sequence comprising a sequences coding for the parvoviral capsid proteins.Type: GrantFiled: May 14, 2018Date of Patent: November 27, 2018Assignee: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan Hermens, Saskia Jacoba Petronella Haast, Dennis Johan Biesmans, Andrew Christian Bakker -
VECTORS WITH MODIFIED INITIATION CODON FOR THE TRANSLATION OF AAV-REP78 USEFUL FOR PRODUCTION OF AAV
Publication number: 20180258448Abstract: The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions. The insect cells preferably comprise a first nucleotide sequence encoding the parvoviral rep proteins whereby the initiation codon for translation of the parvoviral Rep78 protein is a suboptimal initiation codon that effects partial exon skipping upon expression in insect cells. The insect cell further comprises a second nucleotide sequence comprising at least one parvoviral (AA V) inverted terminal repeat (ITR) nucleotide sequence and a third nucleotide sequence comprising a sequences coding for the parvoviral capsid proteins.Type: ApplicationFiled: May 14, 2018Publication date: September 13, 2018Applicant: uniQure IP B.V.Inventors: Wilhelmus Theodorus Johannes Maria Christiaan HERMENS, Saskia Jacoba Petronella HAAST, Dennis Johan BIESMANS, Andrew Christian BAKKER