JP2023527354A5 - - Google Patents

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Publication number
JP2023527354A5
JP2023527354A5 JP2022572560A JP2022572560A JP2023527354A5 JP 2023527354 A5 JP2023527354 A5 JP 2023527354A5 JP 2022572560 A JP2022572560 A JP 2022572560A JP 2022572560 A JP2022572560 A JP 2022572560A JP 2023527354 A5 JP2023527354 A5 JP 2023527354A5
Authority
JP
Japan
Prior art keywords
aav
manipulated
item
guide rna
vector
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
JP2022572560A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023527354A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/034323 external-priority patent/WO2021242903A2/en
Publication of JP2023527354A publication Critical patent/JP2023527354A/ja
Publication of JP2023527354A5 publication Critical patent/JP2023527354A5/ja
Pending legal-status Critical Current

Links

JP2022572560A 2020-05-26 2021-05-26 標的ｒｎａを修飾するための組成物及び方法 Pending JP2023527354A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063030166P 2020-05-26 2020-05-26
US63/030,166 2020-05-26
US202063112329P 2020-11-11 2020-11-11
US63/112,329 2020-11-11
US202063119921P 2020-12-01 2020-12-01
US63/119,921 2020-12-01
US202163153175P 2021-02-24 2021-02-24
US63/153,175 2021-02-24
US202163178059P 2021-04-22 2021-04-22
US63/178,059 2021-04-22
PCT/US2021/034323 WO2021242903A2 (en) 2020-05-26 2021-05-26 Compositions and methods for modifying target rnas

Publications (2)

Publication Number Publication Date
JP2023527354A JP2023527354A (ja) 2023-06-28
JP2023527354A5 true JP2023527354A5 (https=) 2024-06-03

Family

ID=76502872

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022572560A Pending JP2023527354A (ja) 2020-05-26 2021-05-26 標的ｒｎａを修飾するための組成物及び方法

Country Status (6)

Country Link
US (1) US20230193279A1 (https=)
EP (1) EP4158024A2 (https=)
JP (1) JP2023527354A (https=)
AU (1) AU2021280283A1 (https=)
CA (1) CA3177380A1 (https=)
WO (1) WO2021242903A2 (https=)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
CA3223109A1 (en) * 2021-06-29 2023-01-05 Richard Thomas SULLIVAN Engineered guide rnas and polynucleotides
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
EP4423274A2 (en) * 2021-10-26 2024-09-04 Shape Therapeutics Inc. Rna-editing compositions and methods of use
EP4441219A2 (en) * 2021-12-01 2024-10-09 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides for rna editing targeting lrrk2
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025524566A (ja) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Ａｄａｒ媒介ｒｎａ編集のためのオリゴヌクレオチドおよびその使用
US20260027237A1 (en) 2022-07-15 2026-01-29 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
AU2023390632A1 (en) 2022-12-09 2025-07-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
WO2024137991A2 (en) * 2022-12-23 2024-06-27 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
EP4689110A1 (en) 2023-03-24 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
EP4691475A1 (en) * 2023-03-31 2026-02-11 Recorna (Guangzhou) Biotechnology Co., Ltd. Sequence for forming editing substrate together with rna editing target site
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
TW202523678A (zh) * 2023-12-14 2025-06-16 國立臺灣大學 ＣＲＩＳＰＲＩ標靶α－突觸核蛋白
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
GB202405143D0 (en) 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3234134B1 (en) * 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
US10370667B2 (en) * 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
EP3571300A1 (en) * 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
US11866697B2 (en) * 2017-05-18 2024-01-09 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2019084062A1 (en) * 2017-10-23 2019-05-02 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION

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