JPWO2022103852A5 - - Google Patents

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Publication number
JPWO2022103852A5
JPWO2022103852A5 JP2023528056A JP2023528056A JPWO2022103852A5 JP WO2022103852 A5 JPWO2022103852 A5 JP WO2022103852A5 JP 2023528056 A JP2023528056 A JP 2023528056A JP 2023528056 A JP2023528056 A JP 2023528056A JP WO2022103852 A5 JPWO2022103852 A5 JP WO2022103852A5
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JP
Japan
Prior art keywords
rna
target
editing
guide
positions
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
JP2023528056A
Other languages
English (en)
Japanese (ja)
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JP2024500279A5 (https=
JP2024500279A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/058799 external-priority patent/WO2022103852A1/en
Publication of JP2024500279A publication Critical patent/JP2024500279A/ja
Publication of JP2024500279A5 publication Critical patent/JP2024500279A5/ja
Publication of JPWO2022103852A5 publication Critical patent/JPWO2022103852A5/ja
Pending legal-status Critical Current

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JP2023528056A 2020-11-11 2021-11-10 Rna編集組成物及び使用方法 Pending JP2024500279A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063112452P 2020-11-11 2020-11-11
US63/112,452 2020-11-11
US202063119754P 2020-12-01 2020-12-01
US63/119,754 2020-12-01
US202163153070P 2021-02-24 2021-02-24
US63/153,070 2021-02-24
US202163178159P 2021-04-22 2021-04-22
US63/178,159 2021-04-22
US202163193373P 2021-05-26 2021-05-26
US63/193,373 2021-05-26
PCT/US2021/058799 WO2022103852A1 (en) 2020-11-11 2021-11-10 Rna-editing compositions and methods of use

Publications (3)

Publication Number Publication Date
JP2024500279A JP2024500279A (ja) 2024-01-09
JP2024500279A5 JP2024500279A5 (https=) 2024-11-18
JPWO2022103852A5 true JPWO2022103852A5 (https=) 2024-11-18

Family

ID=81601658

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023528056A Pending JP2024500279A (ja) 2020-11-11 2021-11-10 Rna編集組成物及び使用方法

Country Status (6)

Country Link
US (1) US20230399635A1 (https=)
EP (1) EP4244358A4 (https=)
JP (1) JP2024500279A (https=)
AU (1) AU2021378789A1 (https=)
CA (1) CA3174139A1 (https=)
WO (1) WO2022103852A1 (https=)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
AU2022301997A1 (en) * 2021-06-29 2024-01-04 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
EP4441219A2 (en) * 2021-12-01 2024-10-09 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides for rna editing targeting lrrk2
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
JP2025525564A (ja) * 2022-07-18 2025-08-05 エフ. ホフマン-ラ ロシュ アーゲー オリゴヌクレオチドの編集
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性dnaベクター
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
AR131145A1 (es) 2022-11-24 2025-02-19 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 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
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 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
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024226860A2 (en) * 2023-04-26 2024-10-31 Shape Therapeutics Inc. Small nuclear rna processing hairpins for small rna payloads
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025015091A1 (en) * 2023-07-11 2025-01-16 Shape Therapeutics Inc. Serpina1-targeting engineered guide rnas and polynucleotides
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
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
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
WO2026006476A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Abca4 vectors and engineered guide rnas
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
EP3475424A1 (en) * 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
EP4389889A3 (en) * 2017-10-06 2024-09-04 Oregon Health & Science University Compositions and methods for editing rna
AU2019336793B2 (en) * 2018-09-06 2024-11-21 The Regents Of The University Of California RNA and DNA base editing via engineered ADAR recruitment
WO2021113270A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
CN115777020A (zh) * 2020-04-22 2023-03-10 塑造治疗公司 使用snrna组分的组合物和方法

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