JPWO2020041634A5 - - Google Patents
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- Publication number
- JPWO2020041634A5 JPWO2020041634A5 JP2021509821A JP2021509821A JPWO2020041634A5 JP WO2020041634 A5 JPWO2020041634 A5 JP WO2020041634A5 JP 2021509821 A JP2021509821 A JP 2021509821A JP 2021509821 A JP2021509821 A JP 2021509821A JP WO2020041634 A5 JPWO2020041634 A5 JP WO2020041634A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- composition
- 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.)
- Pending
Links
- 239000013603 viral vector Substances 0.000 claims description 54
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 claims description 42
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 claims description 22
- 239000002773 nucleotide Substances 0.000 claims description 19
- 125000003729 nucleotide group Chemical group 0.000 claims description 19
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 16
- 210000000234 capsid Anatomy 0.000 claims description 16
- 108090000623 proteins and genes Proteins 0.000 claims description 16
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 15
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 15
- 108091033319 polynucleotide Proteins 0.000 claims description 11
- 102000040430 polynucleotide Human genes 0.000 claims description 11
- 239000002157 polynucleotide Substances 0.000 claims description 11
- 102000001253 Protein Kinase Human genes 0.000 claims description 10
- 108091026823 U7 small nuclear RNA Proteins 0.000 claims description 10
- 108060006633 protein kinase Proteins 0.000 claims description 10
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 9
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 8
- 241000713666 Lentivirus Species 0.000 claims description 8
- 102100022437 Myotonin-protein kinase Human genes 0.000 claims description 8
- 241000700584 Simplexvirus Species 0.000 claims description 8
- 108091036066 Three prime untranslated region Proteins 0.000 claims description 8
- 241000700618 Vaccinia virus Species 0.000 claims description 8
- 241000700605 Viruses Species 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 241000701161 unidentified adenovirus Species 0.000 claims description 8
- 241000701447 unidentified baculovirus Species 0.000 claims description 8
- 241001430294 unidentified retrovirus Species 0.000 claims description 8
- 230000003274 myotonic effect Effects 0.000 claims description 6
- 239000004055 small Interfering RNA Substances 0.000 claims description 6
- 230000002452 interceptive effect Effects 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 230000001256 tonic effect Effects 0.000 claims description 2
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2021534755A JP2021534755A (ja) | 2021-12-16 |
| JP2021534755A5 JP2021534755A5 (https=) | 2022-08-19 |
| JPWO2020041634A5 true JPWO2020041634A5 (https=) | 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 (https=) |
| EP (1) | EP3841209A4 (https=) |
| JP (3) | JP2021534755A (https=) |
| AU (2) | AU2019326551A1 (https=) |
| CA (1) | CA3110466A1 (https=) |
| WO (1) | WO2020041634A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2025507958A (ja) | 2022-03-04 | 2025-03-21 | ロックアネイビオ， インコーポレイテッド | 操作された核内低分子ＲＮＡ（ｓｎＲＮＡ）を含む組成物および方法 |
| CA3247417A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY |
| WO2024006770A1 (en) * | 2022-06-27 | 2024-01-04 | Astellas Gene Therapies, Inc. | Compositions and methods for the treatment of myotonic dystrophies |
| EP4604970A1 (en) * | 2022-10-19 | 2025-08-27 | 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 |
| JP2026511976A (ja) * | 2023-04-05 | 2026-04-14 | ジェンザイム・コーポレーション | Ｄｍ－１筋強直性ジストロフィーの標的遺伝子療法 |
| 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 |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| 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 |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | 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 |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved 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 |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| 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 |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| 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 |
| CA2792696C (en) | 2010-03-17 | 2020-01-07 | Association Institut De Myologie | Modified u7 snrnas for treatment of neuromuscular diseases |
| WO2012012467A2 (en) * | 2010-07-19 | 2012-01-26 | Isis Pharmaceuticals, Inc. | Modulation of nuclear-retained rna |
| ES2739804T3 (es) | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| US9610362B2 (en) * | 2012-03-16 | 2017-04-04 | 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 |
| US20180344817A1 (en) * | 2015-05-01 | 2018-12-06 | Precision Biosciences, Inc. | Precise deletion of chromosomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases |
| WO2017048732A1 (en) | 2015-09-14 | 2017-03-23 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibition of expression of myotilin |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| BR112018070249A2 (pt) | 2016-04-02 | 2019-01-29 | Research Institute At Nationwide Children's Hospital | sistema promotor de u6 modificada para expressão específica de tecido |
-
2019
- 2019-08-22 EP EP19852799.6A patent/EP3841209A4/en active Pending
- 2019-08-22 WO PCT/US2019/047779 patent/WO2020041634A1/en not_active Ceased
- 2019-08-22 JP JP2021509821A patent/JP2021534755A/ja not_active Withdrawn
- 2019-08-22 US US17/269,741 patent/US12492402B2/en active Active
- 2019-08-22 CA CA3110466A patent/CA3110466A1/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
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