JP2023029966A5 - - Google Patents
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- JP2023029966A5 JP2023029966A5 JP2022194564A JP2022194564A JP2023029966A5 JP 2023029966 A5 JP2023029966 A5 JP 2023029966A5 JP 2022194564 A JP2022194564 A JP 2022194564A JP 2022194564 A JP2022194564 A JP 2022194564A JP 2023029966 A5 JP2023029966 A5 JP 2023029966A5
- Authority
- JP
- Japan
- Prior art keywords
- guide
- seq
- nucleotides
- sequence
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025112164A JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566236P | 2017-09-29 | 2017-09-29 | |
| US62/566,236 | 2017-09-29 | ||
| US201862671902P | 2018-05-15 | 2018-05-15 | |
| US62/671,902 | 2018-05-15 | ||
| PCT/US2018/053382 WO2019067872A1 (en) | 2017-09-29 | 2018-09-28 | COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR |
| JP2020517500A JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Division JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025112164A Division JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023029966A JP2023029966A (ja) | 2023-03-07 |
| JP2023029966A5 true JP2023029966A5 (enExample) | 2023-06-02 |
| JP7707140B2 JP7707140B2 (ja) | 2025-07-14 |
Family
ID=63858214
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Pending JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2022194564A Active JP7707140B2 (ja) | 2017-09-29 | 2022-12-06 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2025112164A Pending JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Pending JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025112164A Pending JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US20200248180A1 (enExample) |
| EP (1) | EP3688161A1 (enExample) |
| JP (3) | JP2021500864A (enExample) |
| KR (1) | KR20200058509A (enExample) |
| CN (2) | CN118530993A (enExample) |
| AU (2) | AU2018338787C1 (enExample) |
| BR (1) | BR112020005287A2 (enExample) |
| CA (1) | CA3077251A1 (enExample) |
| CO (1) | CO2020005116A2 (enExample) |
| IL (2) | IL311170A (enExample) |
| MX (1) | MX2020003608A (enExample) |
| MY (1) | MY203849A (enExample) |
| PH (1) | PH12020550364A1 (enExample) |
| SG (1) | SG11202002565YA (enExample) |
| WO (1) | WO2019067872A1 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| EP3585162B1 (en) | 2017-09-29 | 2023-08-30 | Regeneron Pharmaceuticals, Inc. | Rodents comprising a humanized ttr locus and methods of use |
| EP3946598A1 (en) * | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
| EP3946285A1 (en) * | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof |
| WO2020198641A2 (en) * | 2019-03-28 | 2020-10-01 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for polypeptide expression |
| AU2020286382A1 (en) * | 2019-06-04 | 2021-11-04 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| EP4073249A1 (en) * | 2019-12-11 | 2022-10-19 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
| JP2023548005A (ja) * | 2020-10-28 | 2023-11-15 | ノヴォ ノルディスク アー／エス | 抗トランスサイレチン抗体およびその使用方法 |
| WO2022125968A1 (en) | 2020-12-11 | 2022-06-16 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing involving deamination |
| CN112795595A (zh) * | 2020-12-24 | 2021-05-14 | 中山大学 | 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统 |
| MX2023015213A (es) | 2021-06-22 | 2024-03-19 | Intellia Therapeutics Inc | Metodos para la edicion in vivo de un gen hepatico. |
| WO2023028469A2 (en) * | 2021-08-23 | 2023-03-02 | The Board Of Trustees Of The Leland Stanford Junior University | Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells |
| CN118159654A (zh) | 2021-09-20 | 2024-06-07 | 阿尔尼拉姆医药品有限公司 | 抑制素亚基βE（INHBE）调节剂组合物及其使用方法 |
| CN116064598B (zh) * | 2021-10-08 | 2024-03-12 | 苏州艾博生物科技有限公司 | 冠状病毒的核酸疫苗 |
| WO2023185697A2 (en) * | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| TW202417017A (zh) * | 2022-06-30 | 2024-05-01 | 香港商正基基因科技有限公司 | 具有化學修飾的導引rna |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| CN118853659A (zh) * | 2023-04-14 | 2024-10-29 | 深圳瑞吉生物科技有限公司 | 提高mRNA翻译蛋白的输出量的核酸及其应用 |
| WO2024259135A1 (en) | 2023-06-13 | 2024-12-19 | Intellia Therapeutics, Inc. | Assays for analysis of ribonucleic acid (rna) molecules |
| CN120265763A (zh) * | 2023-10-25 | 2025-07-04 | 上海津曼特生物科技有限公司 | 靶向ttr的基因编辑组合物 |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| AU2522095A (en) | 1994-05-19 | 1995-12-18 | Dako A/S | Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis |
| US6974667B2 (en) | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
| US7244764B2 (en) * | 2003-06-23 | 2007-07-17 | Neurochem (International) Limited | Methods and compositions for treating amyloid-related diseases |
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| EP2323667A4 (en) | 2008-08-07 | 2012-07-25 | Isis Pharmaceuticals Inc | MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES |
| WO2010048228A2 (en) | 2008-10-20 | 2010-04-29 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of transthyretin |
| JP5723378B2 (ja) | 2009-11-03 | 2015-05-27 | アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | トランスサイレチン（ｔｔｒ）を阻害する脂質製剤化組成物及び方法 |
| JP5896175B2 (ja) | 2010-04-29 | 2016-03-30 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | トランスサイレチン発現の調節 |
| AU2012340159B2 (en) | 2011-11-18 | 2017-09-07 | Alnylam Pharmaceuticals, Inc. | RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases |
| EP2931898B1 (en) | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| BR112015013784A2 (pt) * | 2012-12-12 | 2017-07-11 | Massachusetts Inst Technology | aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas |
| DK3553174T3 (da) | 2012-12-17 | 2025-08-04 | Harvard College | Rna-guided modificering af humant genom |
| EA201891018A1 (ru) | 2013-03-08 | 2018-09-28 | Новартис Аг | Липиды и липидные композиции для доставки активных агентов |
| SG10201808935WA (en) * | 2013-04-16 | 2018-11-29 | Regeneron Pharma | Targeted modification of rat genome |
| BR112015027322A8 (pt) | 2013-05-01 | 2018-01-02 | Isis Pharmaceuticals Inc | Compostos antissenso conjugados e sua utilização |
| CN105683379A (zh) * | 2013-06-17 | 2016-06-15 | 布罗德研究所有限公司 | 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化 |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| WO2015183025A1 (ko) * | 2014-05-28 | 2015-12-03 | 주식회사 툴젠 | 표적 특이적 뉴클레아제를 이용한 표적 dna의 민감한 검출 방법 |
| WO2015200805A2 (en) * | 2014-06-26 | 2015-12-30 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modifications and methods of use |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| WO2016141224A1 (en) | 2015-03-03 | 2016-09-09 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| HUE065704T2 (hu) | 2015-09-21 | 2024-06-28 | Trilink Biotechnologies Llc | Készítmények és módszerek 5-sapkával ellátott RNS-ek szintetizálására |
| EP3353298B1 (en) * | 2015-09-21 | 2023-09-13 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| WO2017093804A2 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| US11083799B2 (en) * | 2016-03-16 | 2021-08-10 | Crispr Therapeutics Ag | Materials and methods for treatment of hereditary haemochromatosis |
| JP7245651B2 (ja) * | 2016-03-30 | 2023-03-24 | インテリア セラピューティクス，インコーポレイテッド | Ｃｒｉｓｐｒ／ｃａｓ構成成分のための脂質ナノ粒子製剤 |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| TWI835719B (zh) | 2016-12-08 | 2024-03-21 | 美商英特利亞醫療公司 | 經修飾之嚮導rna |
-
2018
- 2018-09-28 CN CN202410646325.0A patent/CN118530993A/zh active Pending
- 2018-09-28 KR KR1020207012163A patent/KR20200058509A/ko active Pending
- 2018-09-28 AU AU2018338787A patent/AU2018338787C1/en active Active
- 2018-09-28 CA CA3077251A patent/CA3077251A1/en active Pending
- 2018-09-28 SG SG11202002565YA patent/SG11202002565YA/en unknown
- 2018-09-28 CN CN201880076628.6A patent/CN111417728A/zh active Pending
- 2018-09-28 EP EP18786638.9A patent/EP3688161A1/en active Pending
- 2018-09-28 BR BR112020005287-6A patent/BR112020005287A2/pt unknown
- 2018-09-28 MX MX2020003608A patent/MX2020003608A/es unknown
- 2018-09-28 IL IL311170A patent/IL311170A/en unknown
- 2018-09-28 MY MYPI2020001489A patent/MY203849A/en unknown
- 2018-09-28 JP JP2020517500A patent/JP2021500864A/ja active Pending
- 2018-09-28 WO PCT/US2018/053382 patent/WO2019067872A1/en not_active Ceased
-
2020
- 2020-03-15 IL IL273317A patent/IL273317A/en unknown
- 2020-03-24 US US16/828,573 patent/US20200248180A1/en not_active Abandoned
- 2020-03-27 PH PH12020550364A patent/PH12020550364A1/en unknown
- 2020-04-24 CO CONC2020/0005116A patent/CO2020005116A2/es unknown
-
2022
- 2022-12-06 JP JP2022194564A patent/JP7707140B2/ja active Active
- 2022-12-09 US US18/063,972 patent/US11965165B2/en active Active
- 2022-12-09 US US18/064,086 patent/US11795460B2/en active Active
-
2024
- 2024-11-07 US US18/940,080 patent/US20250207130A1/en active Pending
-
2025
- 2025-04-17 AU AU2025202742A patent/AU2025202742A1/en active Pending
- 2025-07-02 JP JP2025112164A patent/JP2025163016A/ja active Pending
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