JP2021522836A5 - - Google Patents
Info
- Publication number
- JP2021522836A5 JP2021522836A5 JP2020563947A JP2020563947A JP2021522836A5 JP 2021522836 A5 JP2021522836 A5 JP 2021522836A5 JP 2020563947 A JP2020563947 A JP 2020563947A JP 2020563947 A JP2020563947 A JP 2020563947A JP 2021522836 A5 JP2021522836 A5 JP 2021522836A5
- Authority
- JP
- Japan
- Prior art keywords
- viral vector
- vector according
- promoter
- nucleic acid
- rna
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862671769P | 2018-05-15 | 2018-05-15 | |
| US62/671,769 | 2018-05-15 | ||
| PCT/US2019/032423 WO2019222354A1 (en) | 2018-05-15 | 2019-05-15 | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021522836A JP2021522836A (ja) | 2021-09-02 |
| JP2021522836A5 true JP2021522836A5 (enExample) | 2022-05-24 |
Family
ID=68540673
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020563947A Pending JP2021522836A (ja) | 2018-05-15 | 2019-05-15 | スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20210269825A1 (enExample) |
| EP (1) | EP3793566A4 (enExample) |
| JP (1) | JP2021522836A (enExample) |
| KR (1) | KR20210010549A (enExample) |
| CN (1) | CN112469421A (enExample) |
| AU (1) | AU2019268346A1 (enExample) |
| BR (1) | BR112020023298A2 (enExample) |
| CA (1) | CA3098249A1 (enExample) |
| CL (1) | CL2020002955A1 (enExample) |
| CO (1) | CO2020015239A2 (enExample) |
| MA (1) | MA51938B1 (enExample) |
| MX (1) | MX2020012269A (enExample) |
| PH (1) | PH12020551913A1 (enExample) |
| SG (1) | SG11202011151VA (enExample) |
| WO (1) | WO2019222354A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| US12630842B2 (en) | 2020-05-13 | 2026-05-19 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| KR20240169111A (ko) * | 2022-04-06 | 2024-12-02 | 젠자임 코포레이션 | Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법 |
| US20250352672A1 (en) * | 2022-05-13 | 2025-11-20 | University Of Washington | Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting |
| TW202409289A (zh) * | 2022-06-27 | 2024-03-01 | 美商安斯泰來基因治療股份有限公司 | 用於治療肌強直性營養不良之組合物及方法 |
| WO2024211480A2 (en) * | 2023-04-05 | 2024-10-10 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2024229164A2 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025038795A1 (en) * | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025122536A1 (en) * | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| TW202615058A (zh) * | 2024-06-12 | 2026-04-16 | 美商艾拉倫製藥股份有限公司 | 失養強直性肌肉蛋白質激酶（ｄｍｐｋ）ｉｒｎａ組成物及其使用方法 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5977333A (en) * | 1992-02-06 | 1999-11-02 | Massachusetts Institute Of Technology | DNA sequence encoding the myotonic dystrophy gene and uses thereof |
| US20050042646A1 (en) * | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| ES2756326T3 (es) * | 2010-07-19 | 2020-04-27 | Ionis Pharmaceuticals Inc | Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK) |
| AU2015370903B2 (en) * | 2014-12-24 | 2021-06-17 | Uniqure Ip B.V. | RNAi induced huntingtin gene suppression |
| WO2017079291A1 (en) * | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| IL302455A (en) * | 2016-03-01 | 2023-06-01 | Univ Florida | Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa |
| MX381216B (es) * | 2016-09-30 | 2025-03-12 | Regeneron Pharma | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
| WO2020029303A1 (zh) * | 2018-08-10 | 2020-02-13 | 富士通株式会社 | 一种通信方法、装置和通信系统 |
-
2019
- 2019-05-15 JP JP2020563947A patent/JP2021522836A/ja active Pending
- 2019-05-15 KR KR1020207036197A patent/KR20210010549A/ko not_active Abandoned
- 2019-05-15 MA MA51938A patent/MA51938B1/fr unknown
- 2019-05-15 SG SG11202011151VA patent/SG11202011151VA/en unknown
- 2019-05-15 US US17/054,474 patent/US20210269825A1/en not_active Abandoned
- 2019-05-15 BR BR112020023298-0A patent/BR112020023298A2/pt not_active Application Discontinuation
- 2019-05-15 AU AU2019268346A patent/AU2019268346A1/en not_active Abandoned
- 2019-05-15 CN CN201980047374.XA patent/CN112469421A/zh active Pending
- 2019-05-15 MX MX2020012269A patent/MX2020012269A/es unknown
- 2019-05-15 WO PCT/US2019/032423 patent/WO2019222354A1/en not_active Ceased
- 2019-05-15 CA CA3098249A patent/CA3098249A1/en active Pending
- 2019-05-15 EP EP19803882.0A patent/EP3793566A4/en not_active Withdrawn
-
2020
- 2020-11-10 PH PH12020551913A patent/PH12020551913A1/en unknown
- 2020-11-13 CL CL2020002955A patent/CL2020002955A1/es unknown
- 2020-12-03 CO CONC2020/0015239A patent/CO2020015239A2/es unknown