JP2022525429A5 - - Google Patents

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Publication number
JP2022525429A5
JP2022525429A5 JP2021557650A JP2021557650A JP2022525429A5 JP 2022525429 A5 JP2022525429 A5 JP 2022525429A5 JP 2021557650 A JP2021557650 A JP 2021557650A JP 2021557650 A JP2021557650 A JP 2021557650A JP 2022525429 A5 JP2022525429 A5 JP 2022525429A5
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JP
Japan
Prior art keywords
lipid
composition
administered
mol
guide
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Granted
Application number
JP2021557650A
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English (en)
Japanese (ja)
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JP7636338B2 (ja
JP2022525429A (ja
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Priority claimed from PCT/US2020/025533 external-priority patent/WO2020198706A1/en
Publication of JP2022525429A publication Critical patent/JP2022525429A/ja
Publication of JP2022525429A5 publication Critical patent/JP2022525429A5/ja
Priority to JP2025021006A priority Critical patent/JP2025081416A/ja
Application granted granted Critical
Publication of JP7636338B2 publication Critical patent/JP7636338B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021557650A 2019-03-28 2020-03-27 コルチコステロイドを含む、ttr遺伝子編集およびattrアミロイドーシスを治療するための組成物および方法、またはその使用 Active JP7636338B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025021006A JP2025081416A (ja) 2019-03-28 2025-02-12 コルチコステロイドを含む、ttr遺伝子編集およびattrアミロイドーシスを治療するための組成物および方法、またはその使用

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962825676P 2019-03-28 2019-03-28
US201962825637P 2019-03-28 2019-03-28
US62/825,637 2019-03-28
US62/825,676 2019-03-28
PCT/US2020/025533 WO2020198706A1 (en) 2019-03-28 2020-03-27 Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025021006A Division JP2025081416A (ja) 2019-03-28 2025-02-12 コルチコステロイドを含む、ttr遺伝子編集およびattrアミロイドーシスを治療するための組成物および方法、またはその使用

Publications (3)

Publication Number Publication Date
JP2022525429A JP2022525429A (ja) 2022-05-13
JP2022525429A5 true JP2022525429A5 (enExample) 2023-12-15
JP7636338B2 JP7636338B2 (ja) 2025-02-26

Family

ID=70334147

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021557650A Active JP7636338B2 (ja) 2019-03-28 2020-03-27 コルチコステロイドを含む、ttr遺伝子編集およびattrアミロイドーシスを治療するための組成物および方法、またはその使用
JP2025021006A Pending JP2025081416A (ja) 2019-03-28 2025-02-12 コルチコステロイドを含む、ttr遺伝子編集およびattrアミロイドーシスを治療するための組成物および方法、またはその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025021006A Pending JP2025081416A (ja) 2019-03-28 2025-02-12 コルチコステロイドを含む、ttr遺伝子編集およびattrアミロイドーシスを治療するための組成物および方法、またはその使用

Country Status (10)

Country Link
US (1) US20230035659A1 (enExample)
EP (1) EP3946285A1 (enExample)
JP (2) JP7636338B2 (enExample)
KR (1) KR20220004984A (enExample)
CN (1) CN113874004A (enExample)
AU (1) AU2020248337A1 (enExample)
CA (1) CA3134544A1 (enExample)
CO (1) CO2021014562A2 (enExample)
MX (1) MX2021011690A (enExample)
WO (1) WO2020198706A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
EP4259792A1 (en) * 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
EP4322920A1 (en) * 2021-04-17 2024-02-21 Intellia Therapeutics, Inc. Lipid nanoparticle compositions
CA3220738A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
WO2022271780A1 (en) * 2021-06-22 2022-12-29 Intellia Therapeutics, Inc. Methods for in vivo editing of a liver gene
WO2023230587A2 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
KR20260094824A (ko) * 2023-10-25 2026-06-23 씨에스피씨 종콰이 팔마씨우티컬 테크놀로지 (스자좡) 컴퍼니 리미티드 고효율, 저오프타겟 유전자 편집 도구
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 (22)

* 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
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
EP0760008A1 (en) 1994-05-19 1997-03-05 Dako A/S Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
CA2754043A1 (en) * 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
PT2931898E (pt) * 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
ES2887254T3 (es) 2013-03-08 2021-12-22 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
KR20160097338A (ko) * 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 crispr-cas 시스템의 조성물 및 방법 및 용도
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
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
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
DK4276189T3 (da) 2015-09-21 2026-05-26 Trilink Biotechnologies Llc Initierende oligonukleotidprimere med kappe til syntesering af 5'-ende RNA'er med kappe
ES3030658T3 (en) 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018067447A1 (en) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Improved methods for identifying double strand break sites
EP3688162B1 (en) 2017-09-29 2024-03-06 Intellia Therapeutics, Inc. Formulations
IL311278B2 (en) * 2017-09-29 2026-05-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing
TWI904069B (zh) * 2017-09-29 2025-11-11 美商英特利亞醫療公司 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法

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