JP2022505381A5 - - Google Patents
Info
- Publication number
- JP2022505381A5 JP2022505381A5 JP2021521383A JP2021521383A JP2022505381A5 JP 2022505381 A5 JP2022505381 A5 JP 2022505381A5 JP 2021521383 A JP2021521383 A JP 2021521383A JP 2021521383 A JP2021521383 A JP 2021521383A JP 2022505381 A5 JP2022505381 A5 JP 2022505381A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid construct
- bidirectional nucleic
- bidirectional
- sequence
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862747522P | 2018-10-18 | 2018-10-18 | |
| US62/747,522 | 2018-10-18 | ||
| PCT/US2019/057092 WO2020082047A1 (en) | 2018-10-18 | 2019-10-18 | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022505381A JP2022505381A (ja) | 2022-01-14 |
| JP2022505381A5 true JP2022505381A5 (https=) | 2022-10-27 |
Family
ID=68468858
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021521383A Pending JP2022505381A (ja) | 2018-10-18 | 2019-10-18 | アルファ１アンチトリプシン欠乏症を治療するための組成物及び方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20200270618A1 (https=) |
| EP (1) | EP3867378A1 (https=) |
| JP (1) | JP2022505381A (https=) |
| KR (1) | KR20210102209A (https=) |
| CN (1) | CN113195721A (https=) |
| AU (1) | AU2019361204A1 (https=) |
| BR (1) | BR112021007289A2 (https=) |
| CA (1) | CA3116739A1 (https=) |
| CO (1) | CO2021006367A2 (https=) |
| EA (1) | EA202191067A1 (https=) |
| IL (1) | IL282237A (https=) |
| MX (1) | MX2021004276A (https=) |
| PH (1) | PH12021550842A1 (https=) |
| SG (1) | SG11202103735TA (https=) |
| TW (1) | TW202027797A (https=) |
| WO (1) | WO2020082047A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI851534B (zh) * | 2016-12-22 | 2024-08-11 | 美商英特利亞醫療公司 | 用於治療α-１抗胰蛋白酶缺乏症之組合物及方法 |
| SG11202103917VA (en) | 2018-10-16 | 2021-05-28 | Blueallele Llc | Methods for targeted insertion of dna in genes |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| EP3921334A1 (en) | 2019-02-08 | 2021-12-15 | Krystal Biotech, Inc. | Compositions and methods for delivering cftr polypeptides |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| CA3198727A1 (en) * | 2020-10-20 | 2022-04-28 | St Pharm Co., Ltd. | Oligonucleotide for 5'-capped rna synthesis |
| US20220160847A1 (en) * | 2020-11-20 | 2022-05-26 | Mark Egly | Method of preventing and/or treating a plurality of diseases |
| CA3235312A1 (en) * | 2021-10-15 | 2023-04-20 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| IL312244A (en) * | 2021-10-19 | 2024-06-01 | Prec Biosciences Inc | Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency |
| WO2024254519A2 (en) * | 2023-06-08 | 2024-12-12 | Mammoth Biosciences, Inc. | Compositions and methods for the modification of human genes expressed by liver cells |
| 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 (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| 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 |
| JP4285766B2 (ja) | 1994-03-23 | 2009-06-24 | オハイオ ユニバーシティ | 緻密にした核酸および細胞へのデリバリ |
| 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 |
| CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| SG191561A1 (en) | 2008-08-22 | 2013-07-31 | Sangamo Biosciences Inc | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| AU2013335451C1 (en) | 2012-10-23 | 2024-07-04 | 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 |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| 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 |
| CN105683376A (zh) * | 2013-05-15 | 2016-06-15 | 桑格摩生物科学股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| 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 |
| WO2016106121A1 (en) | 2014-12-23 | 2016-06-30 | Syngenta Participations Ag | Methods and compositions for identifying and enriching for cells comprising site specific genomic modifications |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| US11279928B2 (en) | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| CN109715801B (zh) * | 2015-12-01 | 2022-11-01 | 克里斯普治疗股份公司 | 用于治疗α1抗胰蛋白酶缺乏的材料和方法 |
| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| US11512311B2 (en) * | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| SG10202106058WA (en) | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
| TWI851534B (zh) | 2016-12-22 | 2024-08-11 | 美商英特利亞醫療公司 | 用於治療α-１抗胰蛋白酶缺乏症之組合物及方法 |
| WO2019067910A1 (en) | 2017-09-29 | 2019-04-04 | Intellia Therapeutics, Inc. | POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION |
| SG11202103917VA (en) | 2018-10-16 | 2021-05-28 | Blueallele Llc | Methods for targeted insertion of dna in genes |
-
2019
- 2019-10-18 WO PCT/US2019/057092 patent/WO2020082047A1/en not_active Ceased
- 2019-10-18 TW TW108137783A patent/TW202027797A/zh unknown
- 2019-10-18 JP JP2021521383A patent/JP2022505381A/ja active Pending
- 2019-10-18 BR BR112021007289-6A patent/BR112021007289A2/pt unknown
- 2019-10-18 SG SG11202103735TA patent/SG11202103735TA/en unknown
- 2019-10-18 EP EP19798480.0A patent/EP3867378A1/en active Pending
- 2019-10-18 EA EA202191067A patent/EA202191067A1/ru unknown
- 2019-10-18 CA CA3116739A patent/CA3116739A1/en active Pending
- 2019-10-18 MX MX2021004276A patent/MX2021004276A/es unknown
- 2019-10-18 CN CN201980082770.6A patent/CN113195721A/zh active Pending
- 2019-10-18 KR KR1020217014893A patent/KR20210102209A/ko not_active Ceased
- 2019-10-18 US US16/657,967 patent/US20200270618A1/en active Pending
- 2019-10-18 AU AU2019361204A patent/AU2019361204A1/en active Pending
-
2021
- 2021-04-11 IL IL282237A patent/IL282237A/en unknown
- 2021-04-15 PH PH12021550842A patent/PH12021550842A1/en unknown
- 2021-05-18 CO CONC2021/0006367A patent/CO2021006367A2/es unknown
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