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公开(公告)号：US11946077B2
公开(公告)日：2024-04-02
申请号：US17905617
申请日：2021-06-04
申请人： EmendoBio Inc.
发明人： Lior Izhar , Liat Rockah , Nadav Marbach Bar , Nurit Meron
IPC分类号： C12N9/22
CPC分类号： C12N9/22 , C12N2310/20
摘要： The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-8 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
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公开(公告)号：US20230212562A1
公开(公告)日：2023-07-06
申请号：US17927663
申请日：2021-05-28
申请人： EmendoBio Inc.
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/111 , C12N15/1135 , C12N2310/20 , C12N2320/34
摘要： RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20246 and compositions, methods, and uses thereof.
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公开(公告)号：US11666641B2
公开(公告)日：2023-06-06
申请号：US17594761
申请日：2020-04-30
申请人： EmendoBio Inc.
发明人： David Baram , Lior Izhar , Asael Herman , Liat Rockah , Nadav Marbach-Bar , Nurit Meron , Joseph Georgeson
IPC分类号： C12N9/22 , A61K38/46 , A61K31/7105 , C12N15/11 , C12N15/90
CPC分类号： A61K38/465 , A61K31/7105 , C12N9/22 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2800/80
摘要： The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence of SEQ ID NO: 3 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
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公开(公告)号：US20210032663A1
公开(公告)日：2021-02-04
申请号：US17041661
申请日：2019-03-22
申请人： EMENDOBIO INC.
发明人： David Baram , Lior Izhar , Asael Herman , Rafi Emmanuel , Michal Golan Mashiach , Joseph Georgeson
摘要： RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-1313 and compositions, methods, and uses thereof.
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公开(公告)号：US20240350665A1
公开(公告)日：2024-10-24
申请号：US18291051
申请日：2022-07-21
申请人： EmendoBio Inc.
发明人： Rafi EMMANUEL
CPC分类号： A61K48/005 , C12N7/00 , C12N9/22 , C12N15/111 , C12N2310/20 , C12N2730/10121 , C12N2730/10162
摘要： RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-18936 and compositions, methods, and uses thereof.
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公开(公告)号：US12031149B2
公开(公告)日：2024-07-09
申请号：US16833119
申请日：2020-03-27
申请人： EmendoBio Inc.
发明人： David Baram , Lior Izhar , Asael Herman , Rafi Emmanuel , Michal Golan Mashiach , Joseph Georgeson
CPC分类号： C12N15/907 , A61K48/00 , A61P37/00 , C12N9/22 , C12N15/11 , C12N15/111 , C12N15/1136 , C12N2310/20 , C12N2800/80
摘要： RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6601 and compositions, methods, and uses thereof.
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公开(公告)号：US20220298495A1
公开(公告)日：2022-09-22
申请号：US17617897
申请日：2020-06-12
申请人： EmendoBio Inc.
发明人： Lior Izhar , Noam Diamant , Yuliya Zilberman
摘要： The present invention provides a novel gene editing composition comprising at least one fusion protein, which comprises a retrotransposon-encoded protein portion linked to a CRISPR nuclease portion, and an RNA template molecule comprising an insert template sequence.
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公开(公告)号：US20220154157A1
公开(公告)日：2022-05-19
申请号：US17428253
申请日：2020-02-04
申请人： EmendoBio Inc.
发明人： David Baram , Lior Izhar , Asael Herman , Liat Rockah , Nadav Marbach Bar , Rafi Emmanuel
摘要： The present invention is directed to, inter glia, composition and methods for genome editing. Specifically, a non-naturally occurring SpCas9 variant having an amino acid substitution at position K929, H930, or at both position K929 and H930.
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公开(公告)号：US20210371857A1
公开(公告)日：2021-12-02
申请号：US16341820
申请日：2017-10-30
申请人： EmendoBio Inc.
发明人： David Baram , Lior Izhar , Rafi Emmanuel
摘要： The present invention relates to compositions and methods for increasing the rate of site specific insertion of a donor DNA sequence to the genome. More specifically, the method introduces a donor DNA template containing at least one transcription factor binding site to a cell in order to favor specific insertion of a donor template sequence at a target site by homology directed repair (HDR).
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公开(公告)号：US20210155929A1
公开(公告)日：2021-05-27
申请号：US17090804
申请日：2020-11-05
申请人： EmendoBio Inc.
发明人： David Baram , Lior Izhar , Asael Herman , Rafi Emmanuel , Michal Golan Mashiach , Joseph Georgeson
IPC分类号： C12N15/113 , C12N15/90 , C12N9/64
摘要： Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) and which cell is heterozygous at one or more polymorphic sites selected from the group consisting of: rs10424470, rs4807932, rs10414837, rs376107533, rs3761010, rs10413889, rs3761007, rs10409474, rs3761005, rs351107, rs3761001, rs740021, rs781452480, rs371057361, rs570466264, rs1041904080, rs7250194, rs17216649, rs199720952, rs6510983, rs17223066, rs7255385, rs112639467, rs141213775, rs28591229, rs10469327, rs3834645, rs1683564, rs71335276, and rs8107095, the method comprising introducing to the cell a composition comprising: a CRISPR nuclease or a sequence encoding the CRISPR nuclease; and a first RNA molecule comprising a guide sequence portion having 17-30 nucleotides, wherein a complex of the CRISPR nuclease and the first RNA molecule affects a double strand break in the mutant allele of the ELANE gene the method optionally further comprising introduction of a second RNA molecule comprising a guide sequence portion capable of complexing with a CRISPR nuclease, wherein the complex of the second RNA molecule and CRISPR nuclease affects a second double strand break in the ELANE gene.
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