公开(公告)号：US20210332447A1

公开(公告)日：2021-10-28

申请号：US17313777

申请日：2021-05-06

申请人： uniQure IP B.V.

发明人： Jacek LUBELSKI ,  Wilhelmus Theodorus Johannes Maria Christiaan HERMENS

IPC分类号： C12Q1/70 ,  C12Q1/6851 ,  C12Q1/6869

摘要： The current invention relates to nucleic acid impurities in a composition comprising a parvoviral vector. In particular, the current invention shows that DNA impurities are not randomly encapsulated within a parvoviral virion. The invention therefore relates to a method for identifying and quantifying a nucleic acid impurity in a composition comprising a parvoviral vector. Finally, the current invention relates to method of determining whether a composition comprising a parvoviral vector is regarded as clinically pure.

公开(公告)号：US20190153473A1

公开(公告)日：2019-05-23

申请号：US16179648

申请日：2018-11-02

申请人： uniQure IP B.V.

发明人： Wilhelmus Theodorus Johannes Maria Christiaan HERMENS ,  Saskia Jacoba Petronella HAAST ,  Dennis Johan BIESMANS ,  Andrew Christian BAKKER

IPC分类号： C12N15/86 ,  C12N7/00 ,  C07K14/005

CPC分类号： C12N15/86 ,  C07K14/005 ,  C12N7/00 ,  C12N2710/14043 ,  C12N2710/14044 ,  C12N2710/14143 ,  C12N2710/14144 ,  C12N2710/14152 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14151 ,  C12N2750/14152 ,  C12N2800/105 ,  C12N2800/50

摘要： 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.

公开(公告)号：US20170356008A1

公开(公告)日：2017-12-14

申请号：US15124139

申请日：2015-03-10

申请人： UniQure IP B.V.

发明人： Jacek LUBELSKI ,  Sebastiaan Menno BOSMA ,  Harald Peter Albert PETRY ,  Wilhelmus Theodorus Johannes Maria Christiaan HERMENS

IPC分类号： C12N15/86 ,  C07K14/005 ,  C12N7/00

CPC分类号： C12N15/86 ,  C07K14/005 ,  C12N7/00 ,  C12N2710/14043 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2799/026 ,  C12N2830/008

摘要： 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. The insect cell further comprises a second nucleotide sequence comprising at least one AAV inverted terminal repeat (ITR) nucleotide sequence; a third nucleotide sequence comprising a Rep52 or a Rep40 coding sequence operably linked to expression control sequences for expression in an insect cell; and, a fourth nucleotide sequence comprising a Rep78 or a Rep68 coding sequence operably linked to expression control sequences for expression in an insect cell. The invention further relates to adeno-associated viral vectors with an altered ratio of the viral capsid proteins.