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 (enExample) | 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 (3) | US20230044994A1 (enExample) |
| EP (1) | EP3946598A1 (enExample) |
| JP (2) | JP7631215B2 (enExample) |
| KR (1) | KR20220004648A (enExample) |
| CN (1) | CN113874076A (enExample) |
| AU (1) | AU2020244887A1 (enExample) |
| BR (1) | BR112021019196A2 (enExample) |
| CA (1) | CA3134271A1 (enExample) |
| CO (1) | CO2021014559A2 (enExample) |
| EA (1) | EA202192636A1 (enExample) |
| IL (1) | IL286524A (enExample) |
| MX (1) | MX2021011565A (enExample) |
| PH (1) | PH12021552301A1 (enExample) |
| SG (1) | SG11202110434PA (enExample) |
| WO (1) | WO2020198697A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2923757T3 (es) * | 2011-12-16 | 2022-09-30 | Modernatx Inc | Composiciones de ARNm modificado |
| US12678515B2 (en) | 2015-12-30 | 2026-07-14 | Acuitas Therapeutics, Inc. | Lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| 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 |
| CN117098541A (zh) | 2020-11-25 | 2023-11-21 | 阿卡格拉医药公司 | 用于递送核酸的脂质纳米粒及相关使用方法 |
| CA3220738A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| US20220396782A1 (en) * | 2021-06-04 | 2022-12-15 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof |
| WO2022271780A1 (en) * | 2021-06-22 | 2022-12-29 | Intellia Therapeutics, Inc. | Methods for in vivo editing of a liver gene |
| CN118974256A (zh) | 2022-03-29 | 2024-11-15 | 锐正基因(苏州)有限公司 | 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法 |
| WO2023230587A2 (en) | 2022-05-25 | 2023-11-30 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| EP4590820A2 (en) | 2022-09-22 | 2025-07-30 | 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 (52)
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|---|---|---|---|---|
| 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 |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| EP0760008A1 (en) | 1994-05-19 | 1997-03-05 | 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 |
| US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
| 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 |
| EP2937418B1 (en) | 2008-10-20 | 2017-10-18 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of transthyretin |
| CA2754043A1 (en) | 2009-03-12 | 2010-09-16 | Alnylam Pharmaceuticals, Inc. | Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes |
| 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 |
| ES2625689T3 (es) | 2010-04-29 | 2017-07-20 | Ionis Pharmaceuticals, Inc. | Modulación de la expresión de transtiretina |
| UA118649C2 (uk) | 2011-11-18 | 2019-02-25 | Елнілем Фармасьютікалз, Інк. | ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR) |
| EP4397759A3 (en) | 2012-10-23 | 2024-10-09 | Toolgen Incorporated | Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| PT2931898E (pt) * | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| IL308158A (en) | 2012-12-17 | 2023-12-01 | Harvard College | Rna-guided human genome engineering |
| ES2887254T3 (es) | 2013-03-08 | 2021-12-22 | Novartis Ag | Lípidos y composiciones lipídicas para el suministro de agentes activos |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| PL2992098T3 (pl) | 2013-05-01 | 2019-09-30 | Ionis Pharmaceuticals, Inc. | Kompozycje i sposoby modulowania ekspresji hbv i ttr |
| SG11201510327TA (en) | 2013-06-17 | 2016-01-28 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| WO2015089419A2 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| KR20160097338A (ko) | 2013-12-12 | 2016-08-17 | 더 브로드 인스티튜트, 인코퍼레이티드 | 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도 |
| US10059655B2 (en) | 2013-12-19 | 2018-08-28 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| WO2015183026A1 (ko) | 2014-05-28 | 2015-12-03 | 주식회사 툴젠 | 불활성화된 표적 특이적 뉴클레아제를 이용한 표적 dna의 분리 방법 |
| DK3161128T3 (en) | 2014-06-26 | 2018-11-05 | Regeneron Pharma | METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF |
| 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 |
| CN105821072A (zh) | 2015-01-23 | 2016-08-03 | 深圳华大基因研究院 | 用于DNA组装的CRISPR-Cas9系统及DNA组装方法 |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| PL3298134T3 (pl) | 2015-05-16 | 2023-09-18 | Genzyme Corporation | Edycja genów mutacji głęboko intronowych |
| US20180208945A1 (en) * | 2015-07-28 | 2018-07-26 | Danisco Us Inc. | Genome editing systems and methods of use |
| DK4276189T3 (da) | 2015-09-21 | 2026-05-26 | Trilink Biotechnologies Llc | Initierende oligonukleotidprimere med kappe til syntesering af 5'-ende RNA'er med kappe |
| US10369232B2 (en) | 2015-09-21 | 2019-08-06 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
| US11866727B2 (en) | 2015-11-06 | 2024-01-09 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 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 |
| WO2017165704A1 (en) | 2016-03-23 | 2017-09-28 | University Of Miami | Treatment of cancer by activating endogenous cryptic amyloidogenic aggregating peptides |
| ES3030658T3 (en) | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| JP2019515914A (ja) | 2016-04-22 | 2019-06-13 | インテリア セラピューティクス，インコーポレイテッド | 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 |
| WO2018007871A1 (en) | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| WO2018058064A1 (en) | 2016-09-23 | 2018-03-29 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
| WO2018067447A1 (en) | 2016-10-03 | 2018-04-12 | Itellia Therapeutics, Inc. | Improved methods for identifying double strand break sites |
| EP3551757A1 (en) | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| WO2018183808A1 (en) | 2017-03-31 | 2018-10-04 | Agenovir Corporation | Antiviral therapeutic |
| TWI904069B (zh) * | 2017-09-29 | 2025-11-11 | 美商英特利亞醫療公司 | 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法 |
| EP3688162B1 (en) * | 2017-09-29 | 2024-03-06 | Intellia Therapeutics, Inc. | Formulations |
| IL311278B2 (en) * | 2017-09-29 | 2026-05-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing |
| JP7832761B2 (ja) * | 2018-06-08 | 2026-03-18 | インテリア セラピューティクス，インコーポレイテッド | 遺伝子編集のための修飾されたガイドｒｎａ |
| BR112021005718A2 (pt) * | 2018-09-28 | 2021-06-22 | Intellia Therapeutics, Inc. | composições e métodos para edição de genes de lactato desidrogenase (ldha) |
-
2020
- 2020-03-27 KR KR1020217035004A patent/KR20220004648A/ko active Pending
- 2020-03-27 EA EA202192636A patent/EA202192636A1/ru unknown
- 2020-03-27 WO PCT/US2020/025513 patent/WO2020198697A1/en not_active Ceased
- 2020-03-27 PH PH1/2021/552301A patent/PH12021552301A1/en unknown
- 2020-03-27 BR BR112021019196A patent/BR112021019196A2/pt unknown
- 2020-03-27 MX MX2021011565A patent/MX2021011565A/es unknown
- 2020-03-27 AU AU2020244887A patent/AU2020244887A1/en active Pending
- 2020-03-27 JP JP2021557397A patent/JP7631215B2/ja active Active
- 2020-03-27 CA CA3134271A patent/CA3134271A1/en active Pending
- 2020-03-27 SG SG11202110434PA patent/SG11202110434PA/en unknown
- 2020-03-27 CN CN202080038208.6A patent/CN113874076A/zh active Pending
- 2020-03-27 EP EP20720292.0A patent/EP3946598A1/en active Pending
-
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/US12686876B2/en active Active
-
2025
- 2025-02-04 JP JP2025016673A patent/JP2025072483A/ja active Pending
-
2026
- 2026-03-26 US US19/630,254 patent/US20260201423A1/en active Pending
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