JP2021534755A5 - - Google Patents
Info
- Publication number
- JP2021534755A5 JP2021534755A5 JP2021509821A JP2021509821A JP2021534755A5 JP 2021534755 A5 JP2021534755 A5 JP 2021534755A5 JP 2021509821 A JP2021509821 A JP 2021509821A JP 2021509821 A JP2021509821 A JP 2021509821A JP 2021534755 A5 JP2021534755 A5 JP 2021534755A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- composition according
- viral vector
- dmpk
- gene
- 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.)
- Withdrawn
Links
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024083610A JP2024105681A (ja) | 2018-08-22 | 2024-05-22 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| JP2025132404A JP2025166094A (ja) | 2018-08-22 | 2025-08-07 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862721105P | 2018-08-22 | 2018-08-22 | |
| US62/721,105 | 2018-08-22 | ||
| PCT/US2019/047779 WO2020041634A1 (en) | 2018-08-22 | 2019-08-22 | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024083610A Division JP2024105681A (ja) | 2018-08-22 | 2024-05-22 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021534755A JP2021534755A (ja) | 2021-12-16 |
| JP2021534755A5 true JP2021534755A5 (enExample) | 2022-08-19 |
Family
ID=69591460
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021509821A Withdrawn JP2021534755A (ja) | 2018-08-22 | 2019-08-22 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| JP2024083610A Pending JP2024105681A (ja) | 2018-08-22 | 2024-05-22 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| JP2025132404A Pending JP2025166094A (ja) | 2018-08-22 | 2025-08-07 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024083610A Pending JP2024105681A (ja) | 2018-08-22 | 2024-05-22 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| JP2025132404A Pending JP2025166094A (ja) | 2018-08-22 | 2025-08-07 | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US12492402B2 (enExample) |
| EP (1) | EP3841209A4 (enExample) |
| JP (3) | JP2021534755A (enExample) |
| AU (2) | AU2019326551A1 (enExample) |
| CA (1) | CA3110466A1 (enExample) |
| WO (1) | WO2020041634A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2023228712A1 (en) | 2022-03-04 | 2024-10-17 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising engineered small nuclear rna (snrna) |
| WO2023196862A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| 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 |
| WO2024086633A1 (en) * | 2022-10-19 | 2024-04-25 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dm1 protein kinase (dmpk) compositions thereof, and methods of use |
| AR131170A1 (es) * | 2022-12-02 | 2025-02-19 | Humble Bee Ltd | Polipéptido recombinante |
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2025137219A1 (en) * | 2023-12-21 | 2025-06-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| EP4692348A1 (en) * | 2024-08-07 | 2026-02-11 | Nocturna Therapeutics SL | Artificial rnas for treating repeat expansion disorders |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| FR2874384B1 (fr) * | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| PL381824A1 (pl) * | 2007-02-22 | 2008-09-01 | Instytut Chemii Bioorganicznej Pan W Poznaniu | Sekwencja siRNA, wektor, cel molekularny dla reagentów siRNA i wektorów wprowadzanych do komórek i tkanek, sposób oceny specyficzności wyciszania zmutowanego transkryptu, sposób badania oddziaływań enzymów szlaku interferencji RNA z transkryptami oraz zastosowanie sekwencji siRNA i wektora |
| US9080170B2 (en) * | 2010-03-17 | 2015-07-14 | Association Institut De Myologie | Modified U7 snRNAs for treatment of neuromuscular diseases |
| EP2595663A4 (en) | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| WO2012109667A1 (en) * | 2011-02-12 | 2012-08-16 | University Of Iowa Research Foundation | Therapeutic compounds |
| CA2907072A1 (en) * | 2012-03-16 | 2013-09-19 | Valerion Therapeutics, Llc | Antisense conjugates for decreasing expression of dmpk |
| TW201536329A (zh) * | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| ES2905181T3 (es) | 2015-05-01 | 2022-04-07 | Prec Biosciences Inc | Deleción precisa de secuencias cromosómicas in vivo |
| ES2830256T3 (es) | 2015-09-14 | 2021-06-03 | Nationwide Childrens Hospital Inc | Productos de virus recombinante y procedimientos de inhibición de expresión de miotilina |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| CA3019832C (en) | 2016-04-02 | 2023-05-09 | Research Institute At Nationwide Children's Hospital | Modified u6 promoter system for tissue specific expression |
-
2019
- 2019-08-22 JP JP2021509821A patent/JP2021534755A/ja not_active Withdrawn
- 2019-08-22 CA CA3110466A patent/CA3110466A1/en active Pending
- 2019-08-22 WO PCT/US2019/047779 patent/WO2020041634A1/en not_active Ceased
- 2019-08-22 US US17/269,741 patent/US12492402B2/en active Active
- 2019-08-22 EP EP19852799.6A patent/EP3841209A4/en active Pending
- 2019-08-22 AU AU2019326551A patent/AU2019326551A1/en not_active Abandoned
-
2024
- 2024-05-22 JP JP2024083610A patent/JP2024105681A/ja active Pending
-
2025
- 2025-08-07 JP JP2025132404A patent/JP2025166094A/ja active Pending
- 2025-11-04 US US19/379,523 patent/US20260049320A1/en active Pending
- 2025-12-19 AU AU2025283617A patent/AU2025283617A1/en active Pending