JP2024534575A5 - - Google Patents

Info

Publication number
JP2024534575A5
JP2024534575A5 JP2024518410A JP2024518410A JP2024534575A5 JP 2024534575 A5 JP2024534575 A5 JP 2024534575A5 JP 2024518410 A JP2024518410 A JP 2024518410A JP 2024518410 A JP2024518410 A JP 2024518410A JP 2024534575 A5 JP2024534575 A5 JP 2024534575A5
Authority
JP
Japan
Prior art keywords
sequence
uridine
mol
mrna
adenosine
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
Application number
JP2024518410A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024534575A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/044453 external-priority patent/WO2023049299A2/en
Publication of JP2024534575A publication Critical patent/JP2024534575A/ja
Priority to JP2025171713A priority Critical patent/JP2026012737A/ja
Publication of JP2024534575A5 publication Critical patent/JP2024534575A5/ja
Pending legal-status Critical Current

Links

JP2024518410A 2021-09-22 2022-09-22 Pcsk9又はangptl3の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法 Pending JP2024534575A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025171713A JP2026012737A (ja) 2021-09-22 2025-10-10 Pcsk9又はangptl3の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163247236P 2021-09-22 2021-09-22
US63/247,236 2021-09-22
US202163255333P 2021-10-13 2021-10-13
US63/255,333 2021-10-13
US202263389679P 2022-07-15 2022-07-15
US63/389,679 2022-07-15
PCT/US2022/044453 WO2023049299A2 (en) 2021-09-22 2022-09-22 Gene editing of pcsk9 or angptl3 and compositions and methods of using same for treatment of disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025171713A Division JP2026012737A (ja) 2021-09-22 2025-10-10 Pcsk9又はangptl3の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

Publications (2)

Publication Number Publication Date
JP2024534575A JP2024534575A (ja) 2024-09-20
JP2024534575A5 true JP2024534575A5 (cg-RX-API-DMAC7.html) 2025-10-21

Family

ID=85721146

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2024518410A Pending JP2024534575A (ja) 2021-09-22 2022-09-22 Pcsk9又はangptl3の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法
JP2025171713A Pending JP2026012737A (ja) 2021-09-22 2025-10-10 Pcsk9又はangptl3の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025171713A Pending JP2026012737A (ja) 2021-09-22 2025-10-10 Pcsk9又はangptl3の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

Country Status (10)

Country Link
US (1) US20240390520A1 (cg-RX-API-DMAC7.html)
EP (1) EP4405470A4 (cg-RX-API-DMAC7.html)
JP (2) JP2024534575A (cg-RX-API-DMAC7.html)
KR (2) KR20250160515A (cg-RX-API-DMAC7.html)
AU (1) AU2022349449A1 (cg-RX-API-DMAC7.html)
CA (1) CA3232742A1 (cg-RX-API-DMAC7.html)
GB (4) GB2702733A (cg-RX-API-DMAC7.html)
IL (1) IL311607A (cg-RX-API-DMAC7.html)
MX (1) MX2024003570A (cg-RX-API-DMAC7.html)
WO (1) WO2023049299A2 (cg-RX-API-DMAC7.html)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018519811A (ja) * 2015-06-29 2018-07-26 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. 修飾crispr rna及び修飾単一crispr rnaならびにその使用
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
JP7646552B2 (ja) 2019-01-31 2025-03-17 ビーム セラピューティクス インク. 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2025137465A1 (en) * 2023-12-21 2025-06-26 Beam Therapeutics Inc. Base editors with modified editing windows
WO2025167914A1 (zh) * 2024-02-07 2025-08-14 益杰立科新加坡有限公司 通过表观编辑多靶点治疗疾病的方法
WO2026143185A1 (en) 2024-12-23 2026-07-02 Ensoma Aps Engineered rna-guided dna-binding polypeptides

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8691750B2 (en) * 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
KR101696704B1 (ko) * 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
AU2016211517B2 (en) * 2015-01-28 2021-05-13 Caribou Biosciences, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
US11279928B2 (en) * 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
WO2017070632A2 (en) * 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
AU2018224387B2 (en) * 2017-02-22 2024-08-08 Crispr Therapeutics Ag Compositions and methods for gene editing
JP7832761B2 (ja) * 2018-06-08 2026-03-18 インテリア セラピューティクス,インコーポレイテッド 遺伝子編集のための修飾されたガイドrna
BR102019009665A2 (pt) * 2018-12-21 2022-02-08 Jacques P. Tremblay Modificação da proteína precursora beta amiloide (app) através da edição de base usando o sistema crispr/cas9
CA3174708A1 (en) * 2020-03-04 2021-09-10 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
US12115230B2 (en) * 2020-04-09 2024-10-15 Verve Therapeutics, Inc. Base editing of ANGPTL3 and methods of using same for treatment of disease

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