JP2021500864A5 - - Google Patents

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Publication number
JP2021500864A5
JP2021500864A5 JP2020517500A JP2020517500A JP2021500864A5 JP 2021500864 A5 JP2021500864 A5 JP 2021500864A5 JP 2020517500 A JP2020517500 A JP 2020517500A JP 2020517500 A JP2020517500 A JP 2020517500A JP 2021500864 A5 JP2021500864 A5 JP 2021500864A5
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JP
Japan
Prior art keywords
composition
seq
guide
ttr
subject
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
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JP2020517500A
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English (en)
Japanese (ja)
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JP2021500864A (ja
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Publication date
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Priority claimed from PCT/US2018/053382 external-priority patent/WO2019067872A1/en
Publication of JP2021500864A publication Critical patent/JP2021500864A/ja
Publication of JP2021500864A5 publication Critical patent/JP2021500864A5/ja
Priority to JP2022194564A priority Critical patent/JP7707140B2/ja
Priority to JP2025112164A priority patent/JP2025163016A/ja
Pending legal-status Critical Current

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JP2020517500A 2017-09-29 2018-09-28 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法 Pending JP2021500864A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022194564A JP7707140B2 (ja) 2017-09-29 2022-12-06 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法
JP2025112164A JP2025163016A (ja) 2017-09-29 2025-07-02 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法

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

Related Child Applications (1)

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JP2022194564A Division JP7707140B2 (ja) 2017-09-29 2022-12-06 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法

Publications (2)

Publication Number Publication Date
JP2021500864A JP2021500864A (ja) 2021-01-14
JP2021500864A5 true JP2021500864A5 (enExample) 2021-11-04

Family

ID=63858214

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2020517500A Pending JP2021500864A (ja) 2017-09-29 2018-09-28 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法
JP2022194564A Active JP7707140B2 (ja) 2017-09-29 2022-12-06 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法
JP2025112164A Pending JP2025163016A (ja) 2017-09-29 2025-07-02 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022194564A Active JP7707140B2 (ja) 2017-09-29 2022-12-06 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法
JP2025112164A Pending JP2025163016A (ja) 2017-09-29 2025-07-02 Ttr遺伝子編集およびattrアミロイドーシスの治療用の組成物および方法

Country Status (15)

Country Link
US (4) US20200248180A1 (enExample)
EP (1) EP3688161A1 (enExample)
JP (3) JP2021500864A (enExample)
KR (1) KR20200058509A (enExample)
CN (2) CN118530993A (enExample)
AU (2) AU2018338787C1 (enExample)
BR (1) BR112020005287A2 (enExample)
CA (1) CA3077251A1 (enExample)
CO (1) CO2020005116A2 (enExample)
IL (2) IL311170A (enExample)
MX (1) MX2020003608A (enExample)
MY (1) MY203849A (enExample)
PH (1) PH12020550364A1 (enExample)
SG (1) SG11202002565YA (enExample)
WO (1) WO2019067872A1 (enExample)

Families Citing this family (21)

* 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
EP3585162B1 (en) 2017-09-29 2023-08-30 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
EP3946598A1 (en) * 2019-03-28 2022-02-09 Intellia Therapeutics, Inc. Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent
EP3946285A1 (en) * 2019-03-28 2022-02-09 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof
WO2020198641A2 (en) * 2019-03-28 2020-10-01 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for polypeptide expression
AU2020286382A1 (en) * 2019-06-04 2021-11-04 Regeneron Pharmaceuticals, Inc. Non-human animals 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
WO2022125968A1 (en) 2020-12-11 2022-06-16 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
MX2023015213A (es) 2021-06-22 2024-03-19 Intellia Therapeutics Inc Metodos para la edicion in vivo de un gen hepatico.
WO2023028469A2 (en) * 2021-08-23 2023-03-02 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
CN118159654A (zh) 2021-09-20 2024-06-07 阿尔尼拉姆医药品有限公司 抑制素亚基βE(INHBE)调节剂组合物及其使用方法
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
TW202417017A (zh) * 2022-06-30 2024-05-01 香港商正基基因科技有限公司 具有化學修飾的導引rna
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
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
CN120265763A (zh) * 2023-10-25 2025-07-04 上海津曼特生物科技有限公司 靶向ttr的基因编辑组合物
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
KR940703846A (ko) 1991-12-24 1994-12-12 비. 린네 파샬 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides)
AU2522095A (en) 1994-05-19 1995-12-18 Dako A/S Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis
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
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
EP2323667A4 (en) 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
WO2010048228A2 (en) 2008-10-20 2010-04-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
JP5723378B2 (ja) 2009-11-03 2015-05-27 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. トランスサイレチン(ttr)を阻害する脂質製剤化組成物及び方法
JP5896175B2 (ja) 2010-04-29 2016-03-30 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. トランスサイレチン発現の調節
AU2012340159B2 (en) 2011-11-18 2017-09-07 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
BR112015013784A2 (pt) * 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
DK3553174T3 (da) 2012-12-17 2025-08-04 Harvard College Rna-guided modificering af humant genom
EA201891018A1 (ru) 2013-03-08 2018-09-28 Новартис Аг Липиды и липидные композиции для доставки активных агентов
SG10201808935WA (en) * 2013-04-16 2018-11-29 Regeneron Pharma Targeted modification of rat genome
BR112015027322A8 (pt) 2013-05-01 2018-01-02 Isis Pharmaceuticals Inc Compostos antissenso conjugados e sua utilização
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
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
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
WO2015200805A2 (en) * 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
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
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
HUE065704T2 (hu) 2015-09-21 2024-06-28 Trilink Biotechnologies Llc Készítmények és módszerek 5-sapkával ellátott RNS-ek szintetizálására
EP3353298B1 (en) * 2015-09-21 2023-09-13 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
BR112018008971A2 (pt) * 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
WO2017093804A2 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
US11083799B2 (en) * 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
JP7245651B2 (ja) * 2016-03-30 2023-03-24 インテリア セラピューティクス,インコーポレイテッド Crispr/cas構成成分のための脂質ナノ粒子製剤
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
TWI835719B (zh) 2016-12-08 2024-03-21 美商英特利亞醫療公司 經修飾之嚮導rna

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