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公开(公告)号：US20240238387A1
公开(公告)日：2024-07-18
申请号：US18351633
申请日：2023-07-13
CPC分类号： A61K38/465 , A61K48/0058 , C12N9/22 , C12N15/52 , C12N15/861 , C12N15/907 , A61P27/00 , C12N2750/14143
摘要： Disclosed are recombinant meganucleases engineered to recognize and cleave recognition sequences present in a mutant RHO P23H allele. The invention further relates to the use of such recombinant meganucleases in methods for treating retinitis pigmentosa, wherein the mutant RHO P23H allele is preferentially targeted, cleaved, and inactivated.
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公开(公告)号：US20240200046A1
公开(公告)日：2024-06-20
申请号：US18556593
申请日：2022-04-22
发明人： Carlos T. Moraes , James Jefferson Smith , Derek Jantz , Ginger H. Tomberlin , John Morris , Wendy Shoop
CPC分类号： C12N9/22 , A61K9/127 , A61K38/465 , A61K48/005 , C12N15/86 , C07K2319/07 , C12N2750/14143
摘要： Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in combination with mitochondrial transit peptides in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been modified or edited.
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公开(公告)号：US11993793B2
公开(公告)日：2024-05-28
申请号：US18360517
申请日：2023-07-27
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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4.发明公开公开(公告)号：US20240035011A1
公开(公告)日：2024-02-01
申请号：US18465727
申请日：2023-09-12
发明人： James Jefferson Smith , Janel Lape , Victor Bartsevich , Hui Li
CPC分类号： C12N9/22 , A61K47/6929 , A61K9/1617 , C12N15/86 , C12N2015/8518
摘要： The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases can exhibit at least one optimized characteristic, such as enhanced specificity and/or efficiency of indel formation, when compared to the first-generation meganuclease HBV 11-12x.26. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or hepatocellular carcinoma.
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公开(公告)号：US20230295590A1
公开(公告)日：2023-09-21
申请号：US18161560
申请日：2023-01-30
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143 , C12N2750/14145
摘要： Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been having modified or edited.
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公开(公告)号：US20230279349A1
公开(公告)日：2023-09-07
申请号：US17820425
申请日：2022-08-17
IPC分类号： C12N5/0783 , C12N15/11 , C12N15/113
CPC分类号： C12N5/0636 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/141 , C12N2310/531
摘要： The present invention encompasses genetically-modified immune cells (and populations thereof) expressing a microRNA-adapted shRNA (shRNAmiR) that reduces the expression of a target endogenous protein. Methods for reducing the expression of an endogenous protein in an immune cell are also provided wherein the method comprises introducing a shRNAmiR that targets the endogenous protein. Using shRNAmiRs for knocking down the expression of a target protein allows for stable knockdown of expression of endogenous proteins in immune cells.
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公开(公告)号：US20230193230A1
公开(公告)日：2023-06-22
申请号：US17924352
申请日：2021-05-11
IPC分类号： C12N9/22 , C12N15/86 , C12N15/11 , C12N15/90 , A61K48/00 , A61P27/02 , A61K31/7105 , A61K38/46
CPC分类号： C12N9/22 , C12N15/86 , C12N15/11 , C12N15/907 , A61K48/005 , A61P27/02 , A61K31/7105 , A61K38/465 , C12N2750/14143 , C12N2750/14145 , C12N2800/80 , C12N2310/20
摘要： Disclosed are recombinant meganucleases engineered to bind and cleave a recognition sequence present in a mutant RHO P23H allele. The invention further relates to the use of such recombinant meganucleases in a method for treating retinitis pigmentosa, wherein the mutant RHO P23H allele is preferentially targeted, cleaved, and inactivated.
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公开(公告)号：US11680254B2
公开(公告)日：2023-06-20
申请号：US16606856
申请日：2018-04-20
CPC分类号： C12N9/22 , C12N15/86 , A61K48/00 , C12N2750/14143 , C12N2800/80
摘要： The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within the human PCSK9 gene. The present invention also encompasses methods for using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of cholesterol-related disorders, such as hypercholesterolemia. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating cholesterol-related disorders, such as hypercholesterolemia.
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9.发明申请公开(公告)号：US20230053176A1
公开(公告)日：2023-02-16
申请号：US17931896
申请日：2022-09-13
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US11384335B2
公开(公告)日：2022-07-12
申请号：US17511022
申请日：2021-10-26
发明人： Aaron Martin , Jon E. Chatterton
IPC分类号： C12N15/85 , C12N5/0783 , C12N15/11 , C12N15/113
摘要： The present invention encompasses genetically-modified immune cells (and populations thereof) expressing a microRNA-adapted shRNA (shRNAmiR) that reduces the expression of a target endogenous protein. Methods for reducing the expression of an endogenous protein in an immune cell are also provided wherein the method comprises introducing a shRNAmiR that targets the endogenous protein. Using shRNAmiRs for knocking down the expression of a target protein allows for stable knockdown of expression of endogenous proteins in immune cells.
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