JP2022525428A5 - - Google Patents
Info
- Publication number
- JP2022525428A5 JP2022525428A5 JP2021557397A JP2021557397A JP2022525428A5 JP 2022525428 A5 JP2022525428 A5 JP 2022525428A5 JP 2021557397 A JP2021557397 A JP 2021557397A JP 2021557397 A JP2021557397 A JP 2021557397A JP 2022525428 A5 JP2022525428 A5 JP 2022525428A5
- Authority
- JP
- Japan
- Prior art keywords
- lipid
- mol
- composition
- composition according
- lnp
- 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 |
|---|---|---|---|
| JP2025016673A JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962825637P | 2019-03-28 | 2019-03-28 | |
| US62/825,637 | 2019-03-28 | ||
| PCT/US2020/025513 WO2020198697A1 (en) | 2019-03-28 | 2020-03-27 | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025016673A Division JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022525428A JP2022525428A (ja) | 2022-05-13 |
| JP2022525428A5 true JP2022525428A5 (https=) | 2023-12-15 |
| JP7631215B2 JP7631215B2 (ja) | 2025-02-18 |
Family
ID=70296147
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021557397A Active JP7631215B2 (ja) | 2019-03-28 | 2020-03-27 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
| JP2025016673A Pending JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025016673A Pending JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20230044994A1 (https=) |
| EP (1) | EP3946598A1 (https=) |
| JP (2) | JP7631215B2 (https=) |
| KR (1) | KR20220004648A (https=) |
| CN (1) | CN113874076A (https=) |
| AU (1) | AU2020244887A1 (https=) |
| BR (1) | BR112021019196A2 (https=) |
| CA (1) | CA3134271A1 (https=) |
| CO (1) | CO2021014559A2 (https=) |
| EA (1) | EA202192636A1 (https=) |
| IL (1) | IL286524A (https=) |
| MX (1) | MX2021011565A (https=) |
| PH (1) | PH12021552301A1 (https=) |
| SG (1) | SG11202110434PA (https=) |
| WO (1) | WO2020198697A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3018046A1 (en) * | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| CA3116576A1 (en) | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| WO2022115645A1 (en) | 2020-11-25 | 2022-06-02 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids, and related methods of use |
| WO2022246266A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| WO2022256619A2 (en) * | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof |
| KR20240038705A (ko) * | 2021-06-22 | 2024-03-25 | 인텔리아 테라퓨틱스, 인크. | 간 유전자의 생체 내 편집 방법 |
| WO2023185697A2 (en) | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| CA3256897A1 (en) | 2022-05-25 | 2023-11-30 | Akagera Medicines, Inc. | Lipid nanoparticles for the administration of nucleic acids and their methods of use |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| WO2024061296A2 (en) | 2022-09-22 | 2024-03-28 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| EP4627083A1 (en) * | 2022-11-30 | 2025-10-08 | Arbor Biotechnologies, Inc. | Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof |
| 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 |
| WO2026003582A2 (en) * | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
| WO2026061971A1 (en) | 2024-09-17 | 2026-03-26 | Lonza Ltd | Nucleic acid untranslated regions |
Family Cites Families (23)
| 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 |
| JP3131222B2 (ja) | 1991-12-24 | 2001-01-31 | アイシス・ファーマシューティカルス・インコーポレーテッド | ギャップを有する２′修飾オリゴヌクレオチド |
| JPH10500310A (ja) | 1994-05-19 | 1998-01-13 | ダコ アクティーゼルスカブ | 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ |
| US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
| US20060051405A1 (en) | 2004-07-19 | 2006-03-09 | Protiva Biotherapeutics, Inc. | Compositions for the delivery of therapeutic agents and uses thereof |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| CN107879960B (zh) | 2013-03-08 | 2021-06-22 | 诺华股份有限公司 | 用于传递活性成分的脂质和脂质组合物 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| 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 |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| WO2017019867A1 (en) * | 2015-07-28 | 2017-02-02 | Danisco Us Inc | Genome editing systems and methods of use |
| CN120842296A (zh) | 2015-09-21 | 2025-10-28 | 垂林克生物技术有限公司 | 用于合成5’-加帽rna的组合物和方法 |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| WO2018007871A1 (en) | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| WO2018067447A1 (en) | 2016-10-03 | 2018-04-12 | Itellia Therapeutics, Inc. | Improved methods for identifying double strand break sites |
| WO2019067910A1 (en) * | 2017-09-29 | 2019-04-04 | Intellia Therapeutics, Inc. | POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION |
| US20230140670A1 (en) | 2017-09-29 | 2023-05-04 | Intellia Therapeutics, Inc. | Formulations |
| EP3688161A1 (en) * | 2017-09-29 | 2020-08-05 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis |
| AU2019282824C1 (en) * | 2018-06-08 | 2026-04-23 | Intellia Therapeutics, Inc. | Modified guide RNAS for gene editing |
| TW202028460A (zh) * | 2018-09-28 | 2020-08-01 | 美商英特利亞醫療公司 | 用於乳酸脫氫酶(ldha)基因編輯之組合物及方法 |
-
2020
- 2020-03-27 JP JP2021557397A patent/JP7631215B2/ja active Active
- 2020-03-27 EA EA202192636A patent/EA202192636A1/ru unknown
- 2020-03-27 AU AU2020244887A patent/AU2020244887A1/en active Pending
- 2020-03-27 MX MX2021011565A patent/MX2021011565A/es unknown
- 2020-03-27 CA CA3134271A patent/CA3134271A1/en active Pending
- 2020-03-27 PH PH1/2021/552301A patent/PH12021552301A1/en unknown
- 2020-03-27 CN CN202080038208.6A patent/CN113874076A/zh active Pending
- 2020-03-27 BR BR112021019196A patent/BR112021019196A2/pt unknown
- 2020-03-27 KR KR1020217035004A patent/KR20220004648A/ko active Pending
- 2020-03-27 WO PCT/US2020/025513 patent/WO2020198697A1/en not_active Ceased
- 2020-03-27 EP EP20720292.0A patent/EP3946598A1/en active Pending
- 2020-03-27 SG SG11202110434PA patent/SG11202110434PA/en unknown
-
2021
- 2021-09-19 IL IL286524A patent/IL286524A/en unknown
- 2021-09-27 US US17/486,735 patent/US20230044994A1/en not_active Abandoned
- 2021-10-28 CO CONC2021/0014559A patent/CO2021014559A2/es unknown
-
2023
- 2023-07-31 US US18/362,867 patent/US20240124897A1/en active Pending
-
2025
- 2025-02-04 JP JP2025016673A patent/JP2025072483A/ja active Pending
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