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公开(公告)号:US20240417706A1
公开(公告)日:2024-12-19
申请号:US18394385
申请日:2023-12-22
Applicant: EDITAS MEDICINE, INC.
Inventor: Jennifer Leah GORI , Luis A. BARRERA , Edouard AUPEPIN DE LAMOTHE-DREUZY , Jack HEATH
IPC: C12N9/22 , A61K31/7105 , A61K38/46 , C12N15/11
Abstract: Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
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公开(公告)号:US12110545B2
公开(公告)日:2024-10-08
申请号:US16475862
申请日:2018-01-05
Applicant: Editas Medicine, Inc.
Inventor: Georgia Giannoukos , Christopher Wilson , Dawn Ciulla
IPC: C12Q1/6869 , G16B20/50 , G16B30/10
CPC classification number: C12Q1/6869 , G16B20/50 , G16B30/10
Abstract: The present disclosure relates to methods of analyzing activity of nucleases, such as ZFNs, TALENs or CRISPR-associated nucleases, in particular their cutting at “off-target” loci.
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公开(公告)号:US12031132B2
公开(公告)日:2024-07-09
申请号:US17019154
申请日:2020-09-11
Applicant: EDITAS MEDICINE, INC.
Inventor: Jennifer Leah Gori , Edouard Aupepin De Lamothe-Dreuzy , Jack Heath , John Anthony Zuris , KaiHsin Chang
IPC: C12N15/113 , A61K9/00
CPC classification number: C12N15/113 , A61K9/0009 , C12N2310/313
Abstract: Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
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公开(公告)号:US20240207448A1
公开(公告)日:2024-06-27
申请号:US18555716
申请日:2022-04-15
Applicant: EDITAS MEDICINE, INC.
Inventor: Benjamin Aryeh DINER , Deepak REYON , Mariacarmela ALLOCCA
CPC classification number: A61K48/005 , A61P27/02 , C12N9/22 , C12N15/11 , C12N15/86 , G01N33/6848 , C12N2310/20 , C12N2750/14143
Abstract: CRISPR/RNA-guided nuclease-related compositions and methods for treatment of RHO-associated retinitis pigmentosa, e.g., autosomal-dominant retinitis pigmentosa (adRP).
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5.
公开(公告)号:US20240167059A1
公开(公告)日:2024-05-23
申请号:US18520878
申请日:2023-11-28
Applicant: Editas Medicine, Inc.
Inventor: Cecilia Cotta-Ramusino , Carrie M. Margulies
IPC: C12N15/90 , C12N5/0735 , C12N5/074 , C12N5/0789 , C12N9/22 , C12N15/10 , C12N15/113 , C12N15/86
CPC classification number: C12N15/902 , C12N5/0606 , C12N5/0647 , C12N5/0696 , C12N9/22 , C12N15/102 , C12N15/113 , C12N15/86 , C12N2310/20
Abstract: Disclosed herein are genome editing systems and related methods which allow for the detection and quantitative measurement of all possible on-target gene editing outcomes, including targeted integration. The compositions and methods described herein rely on the use of donor templates comprising a 5′ homology arm, a cargo, a one or more priming sites, a 3′ homology arm, and optionally stuffer sequence.
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公开(公告)号:US20240141386A1
公开(公告)日:2024-05-02
申请号:US18161806
申请日:2023-01-30
Applicant: EDITAS MEDICINE, INC.
Inventor: Morgan Lee MAEDER , Rina J. MEPANI , Michael STEFANIDAKIS
IPC: C12N15/90 , A61K9/00 , A61K35/761 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/86
CPC classification number: C12N15/907 , A61K9/0048 , A61K35/761 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/86 , C12N2310/20 , C12N2320/32 , C12N2330/50 , C12N2330/51 , C12N2750/14143 , C12N2800/80
Abstract: Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.
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7.
公开(公告)号:US20240067992A1
公开(公告)日:2024-02-29
申请号:US18450549
申请日:2023-08-16
Applicant: Editas Medicine, Inc.
Inventor: Alexandra GLUCKSMANN , Deborah PALESTRANT , Louis Anthony TARTAGLIA , Jordi MATA-FINK , Agnieszka Dorota CZECHOWICZ
IPC: C12N15/90 , A61K38/46 , A61K47/54 , A61K48/00 , C12N9/22 , C12N9/96 , C12N15/11 , C12N15/82 , C12N15/85
CPC classification number: C12N15/907 , A61K38/465 , A61K47/549 , A61K48/005 , C12N9/22 , C12N9/96 , C12N15/11 , C12N15/8216 , C12N15/85 , C12N15/902 , C12N2310/3513 , C12N2320/33 , C12Y301/00 , Y02A50/30
Abstract: Methods and compositions useful in targeting a payload to or editing target nucleic acid are disclosed herein.
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公开(公告)号:US20230038993A1
公开(公告)日:2023-02-09
申请号:US17165821
申请日:2021-02-02
Applicant: EDITAS MEDICINE, INC.
Inventor: Morgan Lee MAEDER , Rina J. MEPANI , David A. BUMCROT , Shen SHEN , Michael STEFANIDAKIS
Abstract: Compositions and methods for treatment of CEP290 related diseases are disclosed.
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公开(公告)号:US20230002760A1
公开(公告)日:2023-01-05
申请号:US17572403
申请日:2022-01-10
Applicant: Editas Medicine, Inc.
Inventor: Gordon Grant Welstead , Jennifer Leah Gori , Jack Michael Heath , David A. Bumcrot
IPC: C12N15/11 , C12N15/113 , A61P7/00 , A61K35/17 , C12N5/0783 , C12N9/22 , C12N9/96
Abstract: CRISPR/Cas-related compositions and methods which provide for efficient gene editing of eukaryotic cells using modified gRNAs.
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公开(公告)号:US20220389460A1
公开(公告)日:2022-12-08
申请号:US17840152
申请日:2022-06-14
Applicant: EDITAS MEDICINE, INC. , THE BROAD INSTITUTE, INC. , THE UNIVERSITY OF IOWA RESEARCH FOUNDATION , MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventor: Feng ZHANG , Deborah PALESTRANT , Beverly DAVIDSON , Jordi MATA-FINK , Edgardo RODRIGUEZ , Alexis BORISY
IPC: C12N15/90 , C12N15/63 , C12N15/11 , C12N15/113 , C12N9/22
Abstract: Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.
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