JP2022512726A5 - - Google Patents
Info
- Publication number
- JP2022512726A5 JP2022512726A5 JP2021521097A JP2021521097A JP2022512726A5 JP 2022512726 A5 JP2022512726 A5 JP 2022512726A5 JP 2021521097 A JP2021521097 A JP 2021521097A JP 2021521097 A JP2021521097 A JP 2021521097A JP 2022512726 A5 JP2022512726 A5 JP 2022512726A5
- Authority
- JP
- Japan
- Prior art keywords
- construct
- nucleic acid
- polypeptide
- vector
- acid construct
- 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 |
|---|---|---|---|
| JP2024203045A JP2025028917A (ja) | 2018-10-18 | 2024-11-21 | 核酸構築物及び使用方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862747393P | 2018-10-18 | 2018-10-18 | |
| US62/747,393 | 2018-10-18 | ||
| US201962840343P | 2019-04-29 | 2019-04-29 | |
| US62/840,343 | 2019-04-29 | ||
| PCT/US2019/057084 WO2020082041A1 (en) | 2018-10-18 | 2019-10-18 | Nucleic acid constructs and methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024203045A Division JP2025028917A (ja) | 2018-10-18 | 2024-11-21 | 核酸構築物及び使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022512726A JP2022512726A (ja) | 2022-02-07 |
| JP2022512726A5 true JP2022512726A5 (https=) | 2022-10-26 |
| JP7593920B2 JP7593920B2 (ja) | 2024-12-03 |
Family
ID=68503214
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021521097A Active JP7593920B2 (ja) | 2018-10-18 | 2019-10-18 | 核酸構築物及び使用方法 |
| JP2024203045A Pending JP2025028917A (ja) | 2018-10-18 | 2024-11-21 | 核酸構築物及び使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024203045A Pending JP2025028917A (ja) | 2018-10-18 | 2024-11-21 | 核酸構築物及び使用方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US20200268906A1 (https=) |
| EP (2) | EP3867376A1 (https=) |
| JP (2) | JP7593920B2 (https=) |
| CN (1) | CN113272428A (https=) |
| AU (2) | AU2019360269A1 (https=) |
| BR (1) | BR112021007323A2 (https=) |
| CA (1) | CA3116580A1 (https=) |
| CO (1) | CO2021006362A2 (https=) |
| IL (2) | IL322336A (https=) |
| MA (1) | MA53919A (https=) |
| MX (1) | MX2021004282A (https=) |
| MY (1) | MY208708A (https=) |
| PH (1) | PH12021550843A1 (https=) |
| SG (1) | SG11202103732RA (https=) |
| TW (1) | TW202028461A (https=) |
| WO (1) | WO2020082041A1 (https=) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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. |
| 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 |
| CA3231712A1 (en) | 2021-09-08 | 2023-03-16 | Flagship Pioneering Innovations Vi, Llc | Pah-modulating compositions and methods |
| US20230149563A1 (en) | 2021-10-27 | 2023-05-18 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
| US20240415980A1 (en) | 2021-10-28 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-related methods and compositions for knocking out c5 |
| CA3242731A1 (en) | 2022-02-02 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| WO2023212677A2 (en) | 2022-04-29 | 2023-11-02 | Regeneron Pharmaceuticals, Inc. | Identification of tissue-specific extragenic safe harbors for gene therapy approaches |
| US20250302998A1 (en) | 2022-05-09 | 2025-10-02 | Regeneron Pharmaceuticals, Inc. | Vectors and methods for in vivo antibody production |
| JP2025521154A (ja) | 2022-05-31 | 2025-07-08 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法 |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| CN120659627A (zh) | 2022-07-29 | 2025-09-16 | 瑞泽恩制药公司 | 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法 |
| KR20250075694A (ko) | 2022-09-28 | 2025-05-28 | 리제너론 파마슈티칼스 인코포레이티드 | 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체 |
| EP4612184A1 (en) | 2022-11-04 | 2025-09-10 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
| KR20250116795A (ko) | 2022-11-14 | 2025-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 별아교세포로의 섬유아세포 성장 인자 수용체 3-매개된 전달을 위한 조성물 및 방법 |
| KR20260032927A (ko) | 2023-06-30 | 2026-03-10 | 리제너론 파마슈티칼스 인코포레이티드 | 상동성 지향 복구를 증가시키는 방법 및 조성물 |
| IL326018A (en) | 2023-07-28 | 2026-03-01 | Regeneron Pharma | Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease |
| US20250049896A1 (en) | 2023-07-28 | 2025-02-13 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency |
| CN121693566A (zh) | 2023-07-28 | 2026-03-17 | 瑞泽恩制药公司 | 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达 |
| WO2025049524A1 (en) | 2023-08-28 | 2025-03-06 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
| AU2024349204A1 (en) * | 2023-09-29 | 2026-04-09 | The Board Of Regents Of The University Of Texas System | Endogenous nucleus targeting signal peptides 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 |
| US20250242018A1 (en) | 2024-01-26 | 2025-07-31 | Regeneron Pharmaceuticals, Inc. | Combination immunosuppression for inhibiting an immune response and enabling immunogen administration and re-administration |
| US20250276092A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
| WO2025235388A1 (en) | 2024-05-06 | 2025-11-13 | Regeneron Pharmaceuticals, Inc. | Transgene genomic identification by nuclease-mediated long read sequencing |
| WO2025265017A1 (en) | 2024-06-20 | 2025-12-26 | Regeneron Pharmaceuticals, Inc. | Ass1 gene insertion for the treatment of citrullinemia type i |
| WO2026072529A1 (en) | 2024-09-24 | 2026-04-02 | University Of Florida Research Foundation, Incorporated | Mettl7a for improved embryo competence |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| 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 |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| 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 |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| 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 |
| ES2465996T3 (es) | 2006-05-25 | 2014-06-09 | Sangamo Biosciences, Inc. | Métodos y composiciones para la inactivación genética |
| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| 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 |
| EP2352369B1 (en) | 2008-12-04 | 2017-04-26 | Sangamo BioSciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US9567573B2 (en) | 2010-04-26 | 2017-02-14 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using nucleases |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| CA2848417C (en) | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA3099582A1 (en) | 2011-10-27 | 2013-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| 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 |
| EP3196301B1 (en) * | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic diseases |
| 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 |
| 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 |
| 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 |
| 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 |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| 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 CN CN201980078035.8A patent/CN113272428A/zh active Pending
- 2019-10-18 MY MYPI2021002127A patent/MY208708A/en unknown
- 2019-10-18 SG SG11202103732RA patent/SG11202103732RA/en unknown
- 2019-10-18 WO PCT/US2019/057084 patent/WO2020082041A1/en not_active Ceased
- 2019-10-18 MA MA053919A patent/MA53919A/fr unknown
- 2019-10-18 BR BR112021007323-0A patent/BR112021007323A2/pt unknown
- 2019-10-18 EP EP19801449.0A patent/EP3867376A1/en not_active Withdrawn
- 2019-10-18 EP EP25192266.2A patent/EP4667576A2/en active Pending
- 2019-10-18 IL IL322336A patent/IL322336A/en unknown
- 2019-10-18 AU AU2019360269A patent/AU2019360269A1/en not_active Abandoned
- 2019-10-18 CA CA3116580A patent/CA3116580A1/en active Pending
- 2019-10-18 IL IL282233A patent/IL282233B2/en unknown
- 2019-10-18 US US16/657,939 patent/US20200268906A1/en not_active Abandoned
- 2019-10-18 TW TW108137784A patent/TW202028461A/zh unknown
- 2019-10-18 JP JP2021521097A patent/JP7593920B2/ja active Active
- 2019-10-18 MX MX2021004282A patent/MX2021004282A/es unknown
-
2021
- 2021-04-15 PH PH12021550843A patent/PH12021550843A1/en unknown
- 2021-04-16 US US17/233,376 patent/US20210316014A1/en not_active Abandoned
- 2021-05-18 CO CONC2021/0006362A patent/CO2021006362A2/es unknown
-
2024
- 2024-11-21 JP JP2024203045A patent/JP2025028917A/ja active Pending
-
2026
- 2026-03-23 AU AU2026202230A patent/AU2026202230A1/en active Pending
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