JPWO2023049299A5 - - Google Patents

Info

Publication number
JPWO2023049299A5
JPWO2023049299A5 JP2024518410A JP2024518410A JPWO2023049299A5 JP WO2023049299 A5 JPWO2023049299 A5 JP WO2023049299A5 JP 2024518410 A JP2024518410 A JP 2024518410A JP 2024518410 A JP2024518410 A JP 2024518410A JP WO2023049299 A5 JPWO2023049299 A5 JP WO2023049299A5
Authority
JP
Japan
Prior art keywords
sequence
seq
item
composition
uridine
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
JP2024534575A5 (https=
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 JPWO2023049299A5 publication Critical patent/JPWO2023049299A5/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 (3)

Publication Number Publication Date
JP2024534575A JP2024534575A (ja) 2024-09-20
JPWO2023049299A5 true JPWO2023049299A5 (https=) 2025-10-21
JP2024534575A5 JP2024534575A5 (https=) 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 (https=)
EP (1) EP4405470A2 (https=)
JP (2) JP2024534575A (https=)
KR (2) KR20250160515A (https=)
AU (1) AU2022349449A1 (https=)
CA (1) CA3232742A1 (https=)
GB (3) GB2700667A (https=)
IL (1) IL311607A (https=)
MX (1) MX2024003570A (https=)
WO (1) WO2023049299A2 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4545544A3 (en) * 2015-06-29 2025-10-08 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE 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
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
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

Family Cites Families (3)

* 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
EP3250691B9 (en) * 2015-01-28 2023-08-02 Caribou Biosciences, Inc. Crispr hybrid dna/rna polynucleotides and methods of use
JP7109784B2 (ja) * 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したCas9蛋白質

Similar Documents

Publication Publication Date Title
JP7765837B2 (ja) シュタルガルト病の処置におけるヌクレオチド脱アミノ化のためのアンチセンスオリゴヌクレオチド
US20250154504A1 (en) Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
US20220127609A1 (en) Antisense oligonucleotides for nucleic acid editing
EP1737879B1 (en) Aptamer-mediated intracellular delivery of therapeutic oligonucleotides
UA130104C2 (uk) Композиції на основі ліпідних наночастинок
CA3118559A1 (en) Lipid nanoparticle formulations
JP2008512097A (ja) アプタマー医薬品化学
GB2628238A (en) Gene editing of PCSK9 or ANGPTL3 and compositions and methods of using same for treatment of disease
JP2002511851A (ja) 肝細胞の遺伝的病変を修正するためのリコンビナジェニックオリゴヌクレオ塩基のin vivoでの使用
JPWO2023049299A5 (https=)
CN120129532A (zh) 稳定的脂质或类脂质纳米颗粒悬浮液
JP2008512098A (ja) フォン・ビルブラント因子に対するアプタマー、および血栓性疾患の処置剤としてのその使用
JP2025527583A (ja) 脂質ナノ粒子の組成物
EP3420093B1 (en) Micelle based system nuclease encapsulation for in-vivo gene editing
EP4688143A1 (en) Antisense oligonucleotides for the treatment of liver disease
JPWO2022256440A5 (https=)
US20220288237A1 (en) Compositions and methods for modulating apolipoprotein b (apob) gene expression
JP2026508879A (ja) 治療用オリゴヌクレオチドの局所送達
JPWO2021195467A5 (https=)
CN118109463B (zh) 靶向PCSK9基因的siRNA及其应用
Zhou et al. Case study of antisense oligonucleotides for duchenne muscular dystrophy
WO2026052111A1 (zh) 一种多聚mRNA
KR20260052816A (ko) 치료학적 올리고뉴클레오티드의 국소 전달
WO2025155911A1 (en) Antisense oligonucleotides for the treatment of hurler syndrome
WO2025165840A1 (en) Compositions and methods of treating cardiovascular disease