JPWO2022195074A5 - - Google Patents
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- Publication number
- JPWO2022195074A5 JPWO2022195074A5 JP2023557131A JP2023557131A JPWO2022195074A5 JP WO2022195074 A5 JPWO2022195074 A5 JP WO2022195074A5 JP 2023557131 A JP2023557131 A JP 2023557131A JP 2023557131 A JP2023557131 A JP 2023557131A JP WO2022195074 A5 JPWO2022195074 A5 JP WO2022195074A5
- Authority
- JP
- Japan
- Prior art keywords
- pkp2
- fold
- gene therapy
- isoform
- nucleic acid
- 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
- 101000583179 Homo sapiens Plakophilin-2 Proteins 0.000 claims 22
- 102100030348 Plakophilin-2 Human genes 0.000 claims 22
- 230000001225 therapeutic effect Effects 0.000 claims 20
- 239000013598 vector Substances 0.000 claims 17
- 210000004413 cardiac myocyte Anatomy 0.000 claims 15
- 238000001415 gene therapy Methods 0.000 claims 15
- 238000009472 formulation Methods 0.000 claims 14
- 239000000203 mixture Substances 0.000 claims 14
- 102000001708 Protein Isoforms Human genes 0.000 claims 12
- 108010029485 Protein Isoforms Proteins 0.000 claims 12
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 11
- 150000007523 nucleic acids Chemical group 0.000 claims 11
- 239000013603 viral vector Substances 0.000 claims 10
- 210000004027 cell Anatomy 0.000 claims 7
- 208000031229 Cardiomyopathies Diseases 0.000 claims 4
- 239000003937 drug carrier Substances 0.000 claims 4
- 239000000546 pharmaceutical excipient Substances 0.000 claims 4
- 230000000747 cardiac effect Effects 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 101000764260 Homo sapiens Troponin T, cardiac muscle Proteins 0.000 claims 1
- 102100026893 Troponin T, cardiac muscle Human genes 0.000 claims 1
- 210000002064 heart cell Anatomy 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163163393P | 2021-03-19 | 2021-03-19 | |
| US63/163,393 | 2021-03-19 | ||
| PCT/EP2022/057145 WO2022195074A2 (en) | 2021-03-19 | 2022-03-18 | Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024512483A JP2024512483A (ja) | 2024-03-19 |
| JPWO2022195074A5 true JPWO2022195074A5 (https=) | 2025-03-27 |
| JP2024512483A5 JP2024512483A5 (https=) | 2025-03-27 |
Family
ID=81384690
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023557131A Pending JP2024512483A (ja) | 2021-03-19 | 2022-03-18 | 遺伝子治療組成物及び不整脈原性右室心筋症の治療 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240042059A1 (https=) |
| EP (1) | EP4308172A2 (https=) |
| JP (1) | JP2024512483A (https=) |
| KR (1) | KR20230159471A (https=) |
| CN (1) | CN117715660A (https=) |
| AU (1) | AU2022239846A1 (https=) |
| BR (1) | BR112023018944A2 (https=) |
| CA (1) | CA3212043A1 (https=) |
| IL (1) | IL305959A (https=) |
| MX (1) | MX2023011035A (https=) |
| TW (1) | TW202300180A (https=) |
| WO (1) | WO2022195074A2 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| JP2023538519A (ja) | 2020-08-07 | 2023-09-08 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 |
| US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
| IL301675A (en) | 2020-10-09 | 2023-05-01 | Tenaya Therapeutics Inc | Methods and preparations for placophilin-2 gene therapy |
| CN115851751A (zh) * | 2022-12-21 | 2023-03-28 | 百世诺（北京）医疗科技有限公司 | 肥厚型心肌病变异基因tnnt2及其应用 |
| WO2025189238A1 (en) * | 2024-03-12 | 2025-09-18 | Murdoch Children's Research Institute | Treatment methods for muscle-associated diseases |
| WO2025238184A1 (en) * | 2024-05-15 | 2025-11-20 | Stichting Amsterdam UMC | Scn10a-short gene therapy to restore cardiac impulse conduction and to protect against cardiac arrhythmia |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12186369B2 (en) * | 2017-09-20 | 2025-01-07 | The Regents Of The University Of California | Gene therapy strategy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy |
| US12098385B2 (en) * | 2017-09-20 | 2024-09-24 | The Regents Of The University Of California | Gene therapy strategy to restore cardiac electrical and structural function in arrhythmogenic right ventricular cardiomyopathy |
| CN109004092A (zh) | 2018-06-29 | 2018-12-14 | 云谷（固安）科技有限公司 | 有机电致发光器件和有机电致发光装置 |
| KR20220066914A (ko) * | 2019-09-20 | 2022-05-24 | 유씨엘 비즈니스 리미티드 | 부정맥 유발성 우심실 심근병증의 유전자 요법 조성물 및 치료 |
| JP2023538519A (ja) * | 2020-08-07 | 2023-09-08 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 |
| US11781156B2 (en) * | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
| IL301675A (en) * | 2020-10-09 | 2023-05-01 | Tenaya Therapeutics Inc | Methods and preparations for placophilin-2 gene therapy |
-
2022
- 2022-03-18 KR KR1020237034942A patent/KR20230159471A/ko active Pending
- 2022-03-18 MX MX2023011035A patent/MX2023011035A/es unknown
- 2022-03-18 CN CN202280034403.0A patent/CN117715660A/zh active Pending
- 2022-03-18 JP JP2023557131A patent/JP2024512483A/ja active Pending
- 2022-03-18 WO PCT/EP2022/057145 patent/WO2022195074A2/en not_active Ceased
- 2022-03-18 CA CA3212043A patent/CA3212043A1/en active Pending
- 2022-03-18 TW TW111110077A patent/TW202300180A/zh unknown
- 2022-03-18 AU AU2022239846A patent/AU2022239846A1/en active Pending
- 2022-03-18 US US18/282,061 patent/US20240042059A1/en active Pending
- 2022-03-18 IL IL305959A patent/IL305959A/en unknown
- 2022-03-18 EP EP22718072.6A patent/EP4308172A2/en active Pending
- 2022-03-18 BR BR112023018944A patent/BR112023018944A2/pt unknown
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