JP2022516515A5 - - Google Patents
Info
- Publication number
- JP2022516515A5 JP2022516515A5 JP2021538230A JP2021538230A JP2022516515A5 JP 2022516515 A5 JP2022516515 A5 JP 2022516515A5 JP 2021538230 A JP2021538230 A JP 2021538230A JP 2021538230 A JP2021538230 A JP 2021538230A JP 2022516515 A5 JP2022516515 A5 JP 2022516515A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- nucleotide sequence
- sequence described
- variant
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024219757A JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862786670P | 2018-12-31 | 2018-12-31 | |
| US62/786,670 | 2018-12-31 | ||
| PCT/US2019/069048 WO2020142479A1 (en) | 2018-12-31 | 2019-12-31 | Dux4 rna silencing using rna targeting crispr-cas13b |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024219757A Division JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022516515A JP2022516515A (ja) | 2022-02-28 |
| JP2022516515A5 true JP2022516515A5 (https=) | 2023-01-05 |
Family
ID=69376003
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538230A Pending JP2022516515A (ja) | 2018-12-31 | 2019-12-31 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
| JP2024219757A Pending JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024219757A Pending JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US12545909B2 (https=) |
| EP (1) | EP3906308A1 (https=) |
| JP (2) | JP2022516515A (https=) |
| KR (1) | KR20210110345A (https=) |
| AU (1) | AU2019419494B2 (https=) |
| CA (1) | CA3124963A1 (https=) |
| IL (1) | IL284447B2 (https=) |
| WO (1) | WO2020142479A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| EP4087489B1 (en) | 2020-01-08 | 2025-05-14 | Vitruvia Holdings Inc. | Methods and computing system for processing ultrasound image to determine health of subdermal tissue |
| WO2021188390A1 (en) | 2020-03-19 | 2021-09-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| CA3189065A1 (en) | 2020-09-11 | 2022-03-17 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
| IL303230A (en) * | 2020-11-30 | 2023-07-01 | Res Inst Nationwide Childrens Hospital | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
| CA3220338A1 (en) * | 2021-05-25 | 2022-12-01 | Yiannis SAVVA | Engineered guide rnas and polynucleotides |
| WO2022266324A1 (en) * | 2021-06-17 | 2022-12-22 | Epicrispr Biotechnologies, Inc. | Systems and methods for regulating aberrant gene expressions |
| KR20240032971A (ko) * | 2021-07-08 | 2024-03-12 | 테나야 테라퓨틱스, 인코포레이티드 | 유전자 요법을 위한 최적화된 발현 카세트 |
| CN118265544A (zh) * | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| WO2025137547A1 (en) * | 2023-12-22 | 2025-06-26 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating facioscapulohumeral muscular dystrophy |
Family Cites Families (39)
| 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 |
| 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 |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| 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 |
| USH2191H1 (en) * | 2000-10-24 | 2007-06-05 | Snp Consortium | Identification and mapping of single nucleotide polymorphisms in the human genome |
| 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 |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| EP3192880B1 (en) * | 2010-08-18 | 2019-10-09 | Fred Hutchinson Cancer Research Center | Nucleic acid agents for use in treating facioscapulohumeral dystrophy (fshd) |
| WO2012109570A1 (en) * | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| EP2736539B1 (en) * | 2011-07-25 | 2017-08-23 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| US10538763B2 (en) * | 2015-01-16 | 2020-01-21 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of DUX4 |
| CN116814590A (zh) * | 2015-10-22 | 2023-09-29 | 布罗德研究所有限公司 | Vi-b型crispr酶和系统 |
| US10828377B2 (en) * | 2015-10-26 | 2020-11-10 | The University Of Tokyo | Method for determining presence or absence of suffering from malignant lymphoma or leukemia, and agent for treatment and/or prevention of leukemia |
| ES3034857T3 (en) * | 2015-11-12 | 2025-08-25 | The Res Institute At Nationwide Childrens Hospital | Methods of treating muscular dystrophy |
| JP6966463B2 (ja) * | 2016-02-26 | 2021-11-17 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 組換えウイルス産物及びｄｕｘ４エクソンスキッピングを誘導するための方法 |
| CA3019832C (en) * | 2016-04-02 | 2023-05-09 | Research Institute At Nationwide Children's Hospital | Modified u6 promoter system for tissue specific expression |
| EP3515506B1 (en) * | 2016-09-23 | 2023-09-13 | University of Massachusetts | Silencing of dux4 by recombinant gene editing complexes |
| US11160823B2 (en) * | 2016-11-07 | 2021-11-02 | University Of Massachusetts | Therapeutic targets for facioscapulohumeral muscular dystrophy |
| JP2020511141A (ja) * | 2017-03-15 | 2020-04-16 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系 |
| AU2018251801B2 (en) | 2017-04-12 | 2024-11-07 | Massachusetts Institute Of Technology | Novel type VI CRISPR orthologs and systems |
| EP3640329A1 (en) * | 2018-10-18 | 2020-04-22 | Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) | Gene editing based cancer treatment |
| IL303230A (en) * | 2020-11-30 | 2023-07-01 | Res Inst Nationwide Childrens Hospital | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
| US20240093191A1 (en) * | 2021-02-03 | 2024-03-21 | Research Institute At Nationwide Children's Hospital | Compositions and methods for treating disease associated with dux4 overexpression |
-
2019
- 2019-12-31 EP EP19845752.5A patent/EP3906308A1/en active Pending
- 2019-12-31 WO PCT/US2019/069048 patent/WO2020142479A1/en not_active Ceased
- 2019-12-31 AU AU2019419494A patent/AU2019419494B2/en active Active
- 2019-12-31 IL IL284447A patent/IL284447B2/en unknown
- 2019-12-31 KR KR1020217023871A patent/KR20210110345A/ko active Pending
- 2019-12-31 JP JP2021538230A patent/JP2022516515A/ja active Pending
- 2019-12-31 CA CA3124963A patent/CA3124963A1/en active Pending
- 2019-12-31 US US17/419,171 patent/US12545909B2/en active Active
-
2024
- 2024-12-16 JP JP2024219757A patent/JP2025032342A/ja active Pending
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