JPWO2020082041A5 - - Google Patents
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- Publication number
- JPWO2020082041A5 JPWO2020082041A5 JP2021521097A JP2021521097A JPWO2020082041A5 JP WO2020082041 A5 JPWO2020082041 A5 JP WO2020082041A5 JP 2021521097 A JP2021521097 A JP 2021521097A JP 2021521097 A JP2021521097 A JP 2021521097A JP WO2020082041 A5 JPWO2020082041 A5 JP WO2020082041A5
- Authority
- JP
- Japan
- Prior art keywords
- construct
- nucleic acid
- vector
- polypeptide
- cell
- 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 (4)
| Publication Number | Publication Date |
|---|---|
| JP2022512726A JP2022512726A (ja) | 2022-02-07 |
| JP2022512726A5 JP2022512726A5 (https=) | 2022-10-26 |
| JPWO2020082041A5 true JPWO2020082041A5 (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 |
|---|---|---|---|---|
| JP7460643B2 (ja) | 2018-10-16 | 2024-04-02 | ブルーアレル， エルエルシー | 遺伝子へのｄｎａの標的化挿入のための方法 |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
| US11622547B2 (en) | 2019-06-07 | 2023-04-11 | Regeneran Pharmaceuticals, Inc. | Genetically modified mouse that expresses human albumin |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| KR20240099164A (ko) * | 2021-09-08 | 2024-06-28 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | Pah-조절 조성물 및 방법 |
| US20230149563A1 (en) | 2021-10-27 | 2023-05-18 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
| EP4423271A2 (en) | 2021-10-28 | 2024-09-04 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-related methods and compositions for knocking out c5 |
| EP4473103A2 (en) | 2022-02-02 | 2024-12-11 | 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 |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| CN119421951A (zh) | 2022-05-31 | 2025-02-11 | 瑞泽恩制药公司 | 用于c9orf72重复序列扩增疾病的crispr干扰疗法 |
| IL318625A (en) | 2022-07-29 | 2025-03-01 | Regeneron Pharma | Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle |
| CN120265314A (zh) | 2022-09-28 | 2025-07-04 | 瑞泽恩制药公司 | 抗体抗性修饰受体以增强基于细胞的疗法 |
| IL320016A (en) | 2022-11-04 | 2025-06-01 | Regeneron Pharma | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
| AU2023379457A1 (en) | 2022-11-14 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes |
| AU2024309884A1 (en) | 2023-06-30 | 2025-12-18 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for increasing homology-directed repair |
| US20250049896A1 (en) | 2023-07-28 | 2025-02-13 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency |
| AU2024315073A1 (en) | 2023-07-28 | 2026-01-22 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
| AU2024317483A1 (en) | 2023-07-28 | 2026-01-29 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease |
| 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 |
| WO2025160340A2 (en) | 2024-01-26 | 2025-07-31 | Regeneron Pharmaceuticals, Inc. | Combination immunosuppression for inhibiting an immune response and enabling immunogen administration and re-administration |
| WO2025184567A1 (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 |
|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| 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 |
| US6169169B1 (en) | 1994-05-19 | 2001-01-02 | Dako A/S | PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis |
| ES2465996T3 (es) | 2006-05-25 | 2014-06-09 | Sangamo Biosciences, Inc. | Métodos y composiciones para la inactivación genética |
| WO2008133938A2 (en) | 2007-04-26 | 2008-11-06 | Sangamo Biosciences, Inc. | Targeted integration into the ppp1r12c locus |
| EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | 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 |
| EP3156494B8 (en) | 2008-12-04 | 2018-09-19 | Sangamo Therapeutics, Inc. | Genome editing in rats using zinc-finger nucleases |
| EP2534163B1 (en) | 2010-02-09 | 2015-11-04 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US8771985B2 (en) | 2010-04-26 | 2014-07-08 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using zinc-finger nucleases |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| AU2012312260B2 (en) | 2011-09-21 | 2017-08-31 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| JP6188703B2 (ja) | 2011-10-27 | 2017-08-30 | サンガモ セラピューティクス， インコーポレイテッド | Ｈｐｒｔ遺伝子座を修飾するための方法および組成物 |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| DK3444342T3 (da) * | 2012-07-11 | 2020-08-24 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme |
| EP4357457B1 (en) | 2012-10-23 | 2024-10-16 | 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 |
| EP3608308B1 (en) | 2013-03-08 | 2021-07-21 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| ES2895651T3 (es) | 2013-12-19 | 2022-02-22 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| CA2969151A1 (en) | 2014-12-23 | 2016-06-30 | Syngenta Participations Ag | Methods and compositions for identifying and enriching for cells comprising site specific genomic modifications |
| EP3265559B1 (en) | 2015-03-03 | 2021-01-06 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| CA3006618A1 (en) | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| TWI773666B (zh) | 2016-03-30 | 2022-08-11 | 美商英特利亞醫療公司 | Crispr/cas 組分之脂質奈米粒子調配物 |
| AR113154A1 (es) | 2017-09-29 | 2020-01-29 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para edición del genoma |
| JP7460643B2 (ja) * | 2018-10-16 | 2024-04-02 | ブルーアレル， エルエルシー | 遺伝子へのｄｎａの標的化挿入のための方法 |
-
2019
- 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 BR BR112021007323-0A patent/BR112021007323A2/pt unknown
- 2019-10-18 SG SG11202103732RA patent/SG11202103732RA/en unknown
- 2019-10-18 EP EP19801449.0A patent/EP3867376A1/en not_active Withdrawn
- 2019-10-18 JP JP2021521097A patent/JP7593920B2/ja active Active
- 2019-10-18 MY MYPI2021002127A patent/MY208708A/en unknown
- 2019-10-18 AU AU2019360269A patent/AU2019360269A1/en not_active Abandoned
- 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 IL IL322336A patent/IL322336A/en unknown
- 2019-10-18 MX MX2021004282A patent/MX2021004282A/es unknown
- 2019-10-18 IL IL282233A patent/IL282233B2/en unknown
- 2019-10-18 CA CA3116580A patent/CA3116580A1/en active Pending
- 2019-10-18 EP EP25192266.2A patent/EP4667576A2/en active Pending
- 2019-10-18 CN CN201980078035.8A patent/CN113272428A/zh active Pending
-
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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