JPWO2021242903A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021242903A5 JPWO2021242903A5 JP2022572560A JP2022572560A JPWO2021242903A5 JP WO2021242903 A5 JPWO2021242903 A5 JP WO2021242903A5 JP 2022572560 A JP2022572560 A JP 2022572560A JP 2022572560 A JP2022572560 A JP 2022572560A JP WO2021242903 A5 JPWO2021242903 A5 JP WO2021242903A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- guide rna
- rna
- engineered
- 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
Links
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2023527354A JP2023527354A (ja) | 2023-06-28 |
| JPWO2021242903A5 true JPWO2021242903A5 (https=) | 2024-06-03 |
| JP2023527354A5 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)
| 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 |
| WO2023278436A1 (en) * | 2021-06-29 | 2023-01-05 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| AU2022301997A1 (en) * | 2021-06-29 | 2024-01-04 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| CA3236391A1 (en) * | 2021-10-26 | 2023-05-04 | Yiannis SAVVA | 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 |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| 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 |
| CN121152627A (zh) * | 2023-03-31 | 2025-12-16 | 时夕(广州)生物科技有限公司 | 一种与rna编辑靶位点形成编辑底物的序列 |
| WO2024256620A1 (en) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
| 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 |
| WO2025128134A1 (en) * | 2023-12-14 | 2025-06-19 | National Taiwan University Hospital | Crispri targeting alpha-synuclein |
| 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10676737B2 (en) * | 2014-12-17 | 2020-06-09 | 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 |
| US11274300B2 (en) * | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| WO2018213708A1 (en) * | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| US20210130800A1 (en) * | 2017-10-23 | 2021-05-06 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
-
2021
- 2021-05-26 CA CA3177380A patent/CA3177380A1/en active Pending
- 2021-05-26 AU AU2021280283A patent/AU2021280283A1/en active Pending
- 2021-05-26 EP EP21733326.9A patent/EP4158024A2/en active Pending
- 2021-05-26 US US17/925,871 patent/US20230193279A1/en active Pending
- 2021-05-26 JP JP2022572560A patent/JP2023527354A/ja active Pending
- 2021-05-26 WO PCT/US2021/034323 patent/WO2021242903A2/en not_active Ceased
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2021242903A5 (https=) | ||
| US20230263910A1 (en) | Aav vectors targeted to the central nervous system | |
| US20250161485A1 (en) | Aav capsid variants and uses thereof | |
| WO2021216853A1 (en) | Compositions and methods using snrna components | |
| JP2025148448A (ja) | アデノ随伴ウイルスベクター変種 | |
| GB2590880A (en) | RNA and DNA base editing via engineered ADAR recruitment | |
| SG11202111102SA (en) | Novel aav capsids and compositions containing same | |
| EP3748004A1 (en) | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy | |
| JP2013531471A5 (https=) | ||
| KR20210058806A (ko) | Rna 표적화 융합 단백질 조성물 및 사용 방법 | |
| EP3129485A2 (en) | Crispr/cas-related methods and compositions for treating cystic fibrosis | |
| JPWO2021113270A5 (https=) | ||
| KR20230051208A (ko) | 신규 aav 캡시드 및 이를 함유하는 조성물 | |
| CN115151646A (zh) | 调节核酸序列 | |
| EP4547690A1 (en) | Aav capsid variants and uses thereof | |
| WO2025038430A1 (en) | Aav capsid variants and uses thereof | |
| WO2024011112A1 (en) | Aav capsid variants and uses thereof | |
| JP2021534794A5 (https=) | ||
| JP6473438B2 (ja) | プロモーター組成物 | |
| JPWO2020047268A5 (https=) | ||
| JPWO2022028472A5 (https=) | ||
| WO2025090858A1 (en) | Methods for identifying aav capsid variants with desired characteristics | |
| WO2026055323A1 (en) | Therapeutic regulation of scn2a splicing | |
| JP2018033353A (ja) | 遺伝子発現制御のための発現制御核酸分子およびその用途 |