KR102758180B1 - 합성 가이드 분자, 그와 관련된 조성물 및 방법 - Google Patents

합성 가이드 분자, 그와 관련된 조성물 및 방법 Download PDF

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KR102758180B1
KR102758180B1 KR1020237043357A KR20237043357A KR102758180B1 KR 102758180 B1 KR102758180 B1 KR 102758180B1 KR 1020237043357 A KR1020237043357 A KR 1020237043357A KR 20237043357 A KR20237043357 A KR 20237043357A KR 102758180 B1 KR102758180 B1 KR 102758180B1
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guide molecule
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KR20230175330A (ko
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짐 하일
스테파니 킹
샘 사코마노
스테이시 케이프하트
세실리아 페르난데스
하리하란 자야람
브루스 이튼
카린 젬스키 베리
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에디타스 메디신, 인코포레이티드
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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KR1020237043357A 2016-12-30 2017-12-29 합성 가이드 분자, 그와 관련된 조성물 및 방법 Active KR102758180B1 (ko)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662441046P 2016-12-30 2016-12-30
US62/441,046 2016-12-30
US201762492001P 2017-04-28 2017-04-28
US62/492,001 2017-04-28
KR1020197022278A KR102618864B1 (ko) 2016-12-30 2017-12-29 합성 가이드 분자, 그와 관련된 조성물 및 방법
PCT/US2017/069019 WO2018126176A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto

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KR20230175330A KR20230175330A (ko) 2023-12-29
KR102758180B1 true KR102758180B1 (ko) 2025-01-23

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KR1020197022278A Active KR102618864B1 (ko) 2016-12-30 2017-12-29 합성 가이드 분자, 그와 관련된 조성물 및 방법

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US (1) US20230111575A1 (cg-RX-API-DMAC7.html)
EP (1) EP3565895A1 (cg-RX-API-DMAC7.html)
JP (3) JP7167029B2 (cg-RX-API-DMAC7.html)
KR (2) KR102758180B1 (cg-RX-API-DMAC7.html)
CN (2) CN110249052B (cg-RX-API-DMAC7.html)
AU (2) AU2017388753A1 (cg-RX-API-DMAC7.html)
CA (1) CA3048434A1 (cg-RX-API-DMAC7.html)
MX (1) MX420023B (cg-RX-API-DMAC7.html)
WO (1) WO2018126176A1 (cg-RX-API-DMAC7.html)

Families Citing this family (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3116997B1 (en) 2014-03-10 2019-05-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
DK3507366T3 (da) 2016-09-01 2020-11-02 Proqr Therapeutics Ii Bv Kemisk modificeret, enkeltstrengede rna-modificerende oligonukleotider
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
CN110662554A (zh) 2017-02-28 2020-01-07 Vor生物制药股份有限公司 用于抑制谱系特异性蛋白质的组合物和方法
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US20190076814A1 (en) 2017-09-11 2019-03-14 Synthego Corporation Biopolymer synthesis system and method
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
AU2019333104A1 (en) 2018-08-28 2021-03-25 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
GB201901873D0 (en) * 2019-02-11 2019-04-03 Proqr Therapeutics Ii Bv Antisense oligonucleotides for nucleic acid editing
WO2020237217A1 (en) 2019-05-23 2020-11-26 Vor Biopharma, Inc Compositions and methods for cd33 modification
KR20220047380A (ko) 2019-08-28 2022-04-15 보르 바이오파마 인크. Cll1 변형을 위한 조성물 및 방법
CN114787352A (zh) 2019-08-28 2022-07-22 Vor生物制药股份有限公司 用于cd123修饰的组合物和方法
WO2022047165A1 (en) 2020-08-28 2022-03-03 Vor Biopharma Inc. Compositions and methods for cd123 modification
US20240110189A1 (en) 2020-08-28 2024-04-04 Vor Biopharma Inc. Compositions and methods for cll1 modification
US20230398219A1 (en) 2020-09-14 2023-12-14 Vor Biopharma Inc. Compositions and methods for cd38 modification
JP2023541458A (ja) 2020-09-14 2023-10-02 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ５修飾のための化合物および方法
EP4214318A1 (en) 2020-09-18 2023-07-26 Vor Biopharma Inc. Compositions and methods for cd7 modification
WO2022067240A1 (en) 2020-09-28 2022-03-31 Vor Biopharma, Inc. Compositions and methods for cd6 modification
US20230364146A1 (en) 2020-09-30 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd30 gene modification
JP2023548111A (ja) 2020-10-27 2023-11-15 ブイオーアール バイオファーマ インコーポレーテッド 造血悪性腫瘍を治療するための組成物および方法
WO2022094245A1 (en) 2020-10-30 2022-05-05 Vor Biopharma, Inc. Compositions and methods for bcma modification
US20230414755A1 (en) 2020-11-13 2023-12-28 Vor Biopharma Inc. Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors
KR20230126707A (ko) 2020-12-31 2023-08-30 보르 바이오파마 인크. Cd34 유전자 변형을 위한 조성물 및 방법
WO2022217086A1 (en) 2021-04-09 2022-10-13 Vor Biopharma Inc. Photocleavable guide rnas and methods of use thereof
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
WO2023015182A1 (en) 2021-08-02 2023-02-09 Vor Biopharma Inc. Compositions and methods for gene modification
WO2023049926A2 (en) 2021-09-27 2023-03-30 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
JP2024543369A (ja) 2021-11-09 2024-11-21 ブイオーアール バイオファーマ インコーポレーテッド Ｅｍｒ２修飾のための化合物及び方法
US20250179531A1 (en) 2022-02-25 2025-06-05 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
US20250295695A1 (en) 2022-04-04 2025-09-25 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
WO2024015925A2 (en) 2022-07-13 2024-01-18 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
EP4623081A2 (en) * 2022-11-23 2025-10-01 Prime Medicine, Inc. Split synthesis of long rnas
WO2024168312A1 (en) 2023-02-09 2024-08-15 Vor Biopharma Inc. Methods for treating hematopoietic malignancy
US20250006305A1 (en) * 2023-06-29 2025-01-02 Synthego Corporation Methods and systems for automated fraction selection in nucleic acid manufacturing
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160215275A1 (en) 2015-01-27 2016-07-28 Minghong Zhong Chemically Ligated RNAs for CRISPR/Cas9-lgRNA Complexes as Antiviral Therapeutic Agents
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2016182917A1 (en) 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
WO2016186745A1 (en) * 2015-05-15 2016-11-24 Ge Healthcare Dharmacon, Inc. Synthetic single guide rna for cas9-mediated gene editing

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013055690A1 (en) * 2011-10-10 2013-04-18 Kmt Waterjet Systems Inc. Gasketless high pressure connection
CN106459995B (zh) * 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3116997B1 (en) 2014-03-10 2019-05-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
WO2015148863A2 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3204513A2 (en) * 2014-10-09 2017-08-16 Life Technologies Corporation Crispr oligonucleotides and gene editing
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
EP4019975A1 (en) * 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3409776A4 (en) * 2016-01-30 2019-12-25 Bonac Corporation ARTIFICIAL SINGLE GUIDE RNA AND THEIR USE
EP3443088B1 (en) 2016-04-13 2024-09-18 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160215275A1 (en) 2015-01-27 2016-07-28 Minghong Zhong Chemically Ligated RNAs for CRISPR/Cas9-lgRNA Complexes as Antiviral Therapeutic Agents
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2016182917A1 (en) 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
WO2016186745A1 (en) * 2015-05-15 2016-11-24 Ge Healthcare Dharmacon, Inc. Synthetic single guide rna for cas9-mediated gene editing

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KR102618864B1 (ko) 2024-01-02
CN118345072A (zh) 2024-07-16
MX2019007750A (es) 2019-10-15
US20230111575A1 (en) 2023-04-13
JP7167029B2 (ja) 2022-11-08
CN110249052B (zh) 2024-04-12
AU2017388753A1 (en) 2019-07-11
KR20190110554A (ko) 2019-09-30
JP2022173349A (ja) 2022-11-18
JP2020503049A (ja) 2020-01-30
MX420023B (es) 2025-01-14
CN110249052A (zh) 2019-09-17
EP3565895A1 (en) 2019-11-13
JP7610559B2 (ja) 2025-01-08
CA3048434A1 (en) 2018-07-05
KR20230175330A (ko) 2023-12-29
AU2024205204A1 (en) 2024-09-05
WO2018126176A1 (en) 2018-07-05
JP2025016776A (ja) 2025-02-04

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