JP2024527525A5 - - Google Patents

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Publication number
JP2024527525A5
JP2024527525A5 JP2023579195A JP2023579195A JP2024527525A5 JP 2024527525 A5 JP2024527525 A5 JP 2024527525A5 JP 2023579195 A JP2023579195 A JP 2023579195A JP 2023579195 A JP2023579195 A JP 2023579195A JP 2024527525 A5 JP2024527525 A5 JP 2024527525A5
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JP
Japan
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composition according
lnp composition
level
lnp
administration
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Pending
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JP2023579195A
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English (en)
Japanese (ja)
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JP2024527525A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/034454 external-priority patent/WO2022271780A1/en
Publication of JP2024527525A publication Critical patent/JP2024527525A/ja
Publication of JP2024527525A5 publication Critical patent/JP2024527525A5/ja
Pending legal-status Critical Current

Links

JP2023579195A 2021-06-22 2022-06-22 肝遺伝子のインビボ編集のための方法 Pending JP2024527525A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202163202744P 2021-06-22 2021-06-22
US63/202,744 2021-06-22
US202163202812P 2021-06-25 2021-06-25
US63/202,812 2021-06-25
US202163263466P 2021-11-03 2021-11-03
US63/263,466 2021-11-03
US202163264435P 2021-11-22 2021-11-22
US63/264,435 2021-11-22
US202263314878P 2022-02-28 2022-02-28
US63/314,878 2022-02-28
PCT/US2022/034454 WO2022271780A1 (en) 2021-06-22 2022-06-22 Methods for in vivo editing of a liver gene

Publications (2)

Publication Number Publication Date
JP2024527525A JP2024527525A (ja) 2024-07-25
JP2024527525A5 true JP2024527525A5 (https=) 2025-07-22

Family

ID=82655316

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023579195A Pending JP2024527525A (ja) 2021-06-22 2022-06-22 肝遺伝子のインビボ編集のための方法

Country Status (10)

Country Link
US (1) US20250269062A1 (https=)
EP (1) EP4359531A1 (https=)
JP (1) JP2024527525A (https=)
KR (1) KR20240038705A (https=)
AU (1) AU2022296523A1 (https=)
CA (1) CA3224995A1 (https=)
IL (1) IL309055A (https=)
MX (1) MX2023015213A (https=)
TW (1) TW202327626A (https=)
WO (1) WO2022271780A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP3083556B1 (en) 2013-12-19 2019-12-25 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
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
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
BR112019015220B1 (pt) 2017-09-29 2023-10-03 CF&I Steel L.P. D/B/A EVRAZ Rocky Mountain Steel Método para unir trilhos de aço com entrada de calor por solda controlada
EP3688161A1 (en) 2017-09-29 2020-08-05 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
US20220009878A1 (en) * 2018-10-02 2022-01-13 Intellia Therapeutics, Inc. Ionizable amine lipids
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
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
WO2021046265A1 (en) * 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
EP4073249A1 (en) * 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing

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