KR20200058509A - Attr 아밀로이드증의 ttr 유전자 편집 및 치료를 위한 조성물 및 방법 - Google Patents

Attr 아밀로이드증의 ttr 유전자 편집 및 치료를 위한 조성물 및 방법 Download PDF

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KR20200058509A
KR20200058509A KR1020207012163A KR20207012163A KR20200058509A KR 20200058509 A KR20200058509 A KR 20200058509A KR 1020207012163 A KR1020207012163 A KR 1020207012163A KR 20207012163 A KR20207012163 A KR 20207012163A KR 20200058509 A KR20200058509 A KR 20200058509A
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composition
seq
sequence selected
sequence
ttr
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Korean (ko)
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아티 마헨드라 프라카쉬 칸졸리아
쇼부 오다테
제시카 린 시쳐
레이날드 마이클 레스카뷰
월터 스트랩스
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인텔리아 테라퓨틱스, 인크.
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020207012163A 2017-09-29 2018-09-28 Attr 아밀로이드증의 ttr 유전자 편집 및 치료를 위한 조성물 및 방법 Pending KR20200058509A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762566236P 2017-09-29 2017-09-29
US62/566,236 2017-09-29
US201862671902P 2018-05-15 2018-05-15
US62/671,902 2018-05-15
PCT/US2018/053382 WO2019067872A1 (en) 2017-09-29 2018-09-28 COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR

Publications (1)

Publication Number Publication Date
KR20200058509A true KR20200058509A (ko) 2020-05-27

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KR1020207012163A Pending KR20200058509A (ko) 2017-09-29 2018-09-28 Attr 아밀로이드증의 ttr 유전자 편집 및 치료를 위한 조성물 및 방법

Country Status (16)

Country Link
US (4) US20200248180A1 (https=)
EP (1) EP3688161A1 (https=)
JP (3) JP2021500864A (https=)
KR (1) KR20200058509A (https=)
CN (2) CN118530993A (https=)
AU (2) AU2018338787C1 (https=)
BR (1) BR112020005287A2 (https=)
CA (1) CA3077251A1 (https=)
CO (1) CO2020005116A2 (https=)
IL (3) IL311170B1 (https=)
MX (2) MX2020003608A (https=)
MY (1) MY203849A (https=)
PH (1) PH12020550364A1 (https=)
SG (1) SG11202002565YA (https=)
TW (1) TWI904069B (https=)
WO (1) WO2019067872A1 (https=)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
MX2021011757A (es) * 2019-03-28 2021-12-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la expresión de polipéptidos.
JP7631215B2 (ja) * 2019-03-28 2025-02-18 インテリア セラピューティクス，インコーポレイテッド Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法
AU2020248337A1 (en) * 2019-03-28 2021-11-04 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
EP4073249A1 (en) * 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
JP2023548005A (ja) * 2020-10-28 2023-11-15 ノヴォ ノルディスク アー／エス 抗トランスサイレチン抗体およびその使用方法
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
CN112795595A (zh) * 2020-12-24 2021-05-14 中山大学 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统
WO2022246266A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
KR20240038705A (ko) 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
US20250127927A1 (en) * 2021-08-23 2025-04-24 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CN116064598B (zh) * 2021-10-08 2024-03-12 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
WO2023185697A2 (en) * 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
CN118853659A (zh) * 2023-04-14 2024-10-29 深圳瑞吉生物科技有限公司 提高mRNA翻译蛋白的输出量的核酸及其应用
WO2024259135A1 (en) 2023-06-13 2024-12-19 Intellia Therapeutics, Inc. Assays for analysis of ribonucleic acid (rna) molecules
CN120225668A (zh) * 2023-10-25 2025-06-27 石药集团中奇制药技术（石家庄）有限公司 一种高效低脱靶的基因编辑工具
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する２′修飾オリゴヌクレオチド
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ
US6974667B2 (en) 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
US7244764B2 (en) * 2003-06-23 2007-07-17 Neurochem (International) Limited Methods and compositions for treating amyloid-related diseases
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
EP2323667A4 (en) 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
MX360460B (es) 2008-10-20 2018-11-05 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
PL2563920T3 (pl) 2010-04-29 2017-08-31 Ionis Pharmaceuticals, Inc. Modulacja ekspresji transtyretyny
EP3730618A1 (en) 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3031921B1 (en) * 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
DK3456831T3 (da) * 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
CN105683379A (zh) * 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
WO2015183025A1 (ko) * 2014-05-28 2015-12-03 주식회사 툴젠 표적 특이적 뉴클레아제를 이용한 표적 dna의 민감한 검출 방법
SI3161128T1 (sl) * 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Postopki in sestavki za ciljano genetsko spremembo in postopki uporabe
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
CN120842296A (zh) 2015-09-21 2025-10-28 垂林克生物技术有限公司 用于合成5’-加帽rna的组合物和方法
JP6799058B2 (ja) * 2015-09-21 2020-12-09 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． アレル選択的な遺伝子編集およびその使用
BR112018008971A2 (pt) * 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
EP3429632B1 (en) * 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
LT3436077T (lt) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas

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CA3077251A1 (en) 2019-04-04
EP3688161A1 (en) 2020-08-05
CN118530993A (zh) 2024-08-23
SG11202002565YA (en) 2020-04-29
CN111417728A (zh) 2020-07-14
JP7707140B2 (ja) 2025-07-14
CO2020005116A2 (es) 2020-05-15
IL325460A (en) 2026-02-01
AU2018338787A1 (en) 2020-04-16
AU2018338787B2 (en) 2025-05-08
US20230257747A1 (en) 2023-08-17
JP2023029966A (ja) 2023-03-07
AU2018338787C1 (en) 2025-10-23
NZ762942A (en) 2024-10-25
TWI904069B (zh) 2025-11-11
IL273317A (en) 2020-04-30
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US20230118592A1 (en) 2023-04-20
PH12020550364A1 (en) 2021-02-15
US11795460B2 (en) 2023-10-24
JP2021500864A (ja) 2021-01-14
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US20250207130A1 (en) 2025-06-26
US20200248180A1 (en) 2020-08-06
US11965165B2 (en) 2024-04-23
IL311170A (en) 2024-04-01
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BR112020005287A2 (pt) 2020-09-24
TW201932479A (zh) 2019-08-16

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