公开(公告)号：US11926841B2

公开(公告)日：2024-03-12

申请号：US17481576

申请日：2021-09-22

申请人： SIRION BIOTECH GmbH

发明人： Ulrich Koszinowski ,  Simona Langer ,  Zsolt Ruzsics ,  Christian Thirion

IPC分类号： C07H21/04 ,  C12N15/86 ,  C12Q1/02 ,  C12Q1/6897 ,  C12Q1/70

CPC分类号： C12N15/86 ,  C12Q1/025 ,  C12Q1/6897 ,  C12Q1/70 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2830/002

摘要： The present invention relates to a cell comprising (aa) a nucleic acid comprising in 5′ to 3′ direction (i) at least one adeno-associated virus (AAV) inverted terminal repeat (ITR) sequence; (ii) a promoter which is capable of being activated by (a) helper polypeptide(s) and optionally (a) helper polynucleotide(s); and (iii) a transgenic coding sequence under the control of said promoter of (aa)(ii); and (ab) a nucleic acid comprising in 5′ to 3′ direction (i) a promoter which is capable of being activated by said helper polypeptide(s) and optionally said helper polynucleotide(s); and (ii) at least one AAV rep gene coding sequence under the control of said promoter of (ab)(i); wherein said cell does not comprise an AAV cap gene and/or is not able to express any AAV cap gene product.

公开(公告)号：US11155831B2

公开(公告)日：2021-10-26

申请号：US16073488

申请日：2017-01-30

申请人： SIRION BIOTECH GMBH

发明人： Ulrich Koszinowski ,  Simona Langer ,  Zsolt Ruzsics ,  Christian Thirion

IPC分类号： C12N7/00 ,  C12Q1/70 ,  C12N15/86 ,  C12Q1/6897 ,  C12Q1/02

摘要： The present invention relates to a cell comprising (aa) a nucleic acid comprising in 5′ to 3′ direction (i) at least one adeno-associated virus (AAV) inverted terminal repeat (ITR) sequence; (ii) a promoter which is capable of being activated by (a) helper polypeptide(s) and optionally (a) helper polynucleotide(s); and (iii) a transgenic coding sequence under the control of said promoter of (aa)(ii); and (ab) a nucleic acid comprising in 5′ to 3′ direction (i) a promoter which is capable of being activated by said helper polypeptide(s) and optionally said helper polynucleotide(s); and (ii) at least one AAV rep gene coding sequence under the control of said promoter of (ab)(i); wherein said cell does not comprise an AAV cap gene and/or is not able to express any AAV cap gene product.