JP2022505402A5 - - Google Patents
Info
- Publication number
- JP2022505402A5 JP2022505402A5 JP2021521406A JP2021521406A JP2022505402A5 JP 2022505402 A5 JP2022505402 A5 JP 2022505402A5 JP 2021521406 A JP2021521406 A JP 2021521406A JP 2021521406 A JP2021521406 A JP 2021521406A JP 2022505402 A5 JP2022505402 A5 JP 2022505402A5
- Authority
- JP
- Japan
- Prior art keywords
- sgrna
- use according
- nucleic acid
- acid encoding
- item
- 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 |
|---|---|---|---|
| JP2024063210A JP7820434B2 (ja) | 2018-10-18 | 2024-04-10 | アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862747402P | 2018-10-18 | 2018-10-18 | |
| US62/747,402 | 2018-10-18 | ||
| US201962840346P | 2019-04-29 | 2019-04-29 | |
| US62/840,346 | 2019-04-29 | ||
| PCT/US2019/057086 WO2020082042A2 (en) | 2018-10-18 | 2019-10-18 | Compositions and methods for transgene expression from an albumin locus |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024063210A Division JP7820434B2 (ja) | 2018-10-18 | 2024-04-10 | アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022505402A JP2022505402A (ja) | 2022-01-14 |
| JP2022505402A5 true JP2022505402A5 (enExample) | 2022-10-26 |
| JP7472121B2 JP7472121B2 (ja) | 2024-04-22 |
Family
ID=68733595
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021521406A Active JP7472121B2 (ja) | 2018-10-18 | 2019-10-18 | アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 |
| JP2024063210A Active JP7820434B2 (ja) | 2018-10-18 | 2024-04-10 | アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024063210A Active JP7820434B2 (ja) | 2018-10-18 | 2024-04-10 | アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US20200270617A1 (enExample) |
| EP (1) | EP3867381A2 (enExample) |
| JP (2) | JP7472121B2 (enExample) |
| KR (2) | KR20260048369A (enExample) |
| CN (1) | CN114207130A (enExample) |
| AU (2) | AU2019361203B2 (enExample) |
| BR (1) | BR112021007343A2 (enExample) |
| CA (1) | CA3116918A1 (enExample) |
| CO (1) | CO2021006363A2 (enExample) |
| IL (2) | IL282236B1 (enExample) |
| MX (1) | MX2021004278A (enExample) |
| MY (1) | MY208564A (enExample) |
| PH (1) | PH12021550844A1 (enExample) |
| SA (1) | SA521421766B1 (enExample) |
| SG (1) | SG11202103733SA (enExample) |
| TW (2) | TW202507011A (enExample) |
| WO (1) | WO2020082042A2 (enExample) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| IL326835A (en) | 2018-10-16 | 2026-04-01 | Blueallele Llc | Methods for targeted insertion of dna genes |
| 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 |
| CN113939595A (zh) | 2019-06-07 | 2022-01-14 | 瑞泽恩制药公司 | 包括人源化白蛋白基因座的非人动物 |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| KR20240099358A (ko) | 2021-10-27 | 2024-06-28 | 리제너론 파마슈티칼스 인코포레이티드 | B형 혈우병 치료를 위한 인자 ix를 발현하기 위한 조성물 및 방법 |
| EP4423271A2 (en) | 2021-10-28 | 2024-09-04 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-related methods and compositions for knocking out c5 |
| IL314482A (en) | 2022-02-02 | 2024-09-01 | Regeneron Pharma | 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 |
| AU2023269134A1 (en) | 2022-05-09 | 2024-12-12 | 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 |
| WO2023235726A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr interference 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 |
| AU2023371614A1 (en) | 2022-11-04 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | 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 |
| US20250002946A1 (en) | 2023-06-30 | 2025-01-02 | Regeneron Pharmaceuticals, Inc. | Methods And Compositions For Increasing Homology-Directed Repair |
| 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 |
| AR133384A1 (es) | 2023-07-28 | 2025-09-24 | Regeneron Pharma | Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida |
| 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 |
| EP4771049A1 (en) | 2023-08-28 | 2026-07-08 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
| 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 |
| EP4574975A1 (en) | 2023-12-20 | 2025-06-25 | Bayer AG | Compositions and methods for delivering transgenes |
| 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 (41)
| 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 |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| CA2186118C (en) | 1994-03-23 | 2010-10-19 | Richard W. Hanson | Compacted nucleic acids and their delivery to cells |
| EP0760008A1 (en) | 1994-05-19 | 1997-03-05 | Dako A/S | Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis |
| CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| JP5908725B2 (ja) | 2008-08-22 | 2016-04-26 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 標的一本鎖開裂および標的組込みのための方法、並びに組成物 |
| US9255259B2 (en) | 2010-02-09 | 2016-02-09 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| EP2758529B1 (en) | 2011-09-21 | 2019-03-20 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| ES2728782T3 (es) | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| 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 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| ES2887254T3 (es) | 2013-03-08 | 2021-12-22 | Novartis Ag | Lípidos y composiciones lipídicas para el suministro de agentes activos |
| EP2997146A4 (en) * | 2013-05-15 | 2017-04-26 | Sangamo BioSciences, Inc. | Methods and compositions for treatment of a genetic condition |
| WO2014204725A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| CA3229275A1 (en) | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| US10059655B2 (en) | 2013-12-19 | 2018-08-28 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| WO2015200555A2 (en) | 2014-06-25 | 2015-12-30 | 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 |
| CN113337533A (zh) | 2014-12-23 | 2021-09-03 | 先正达参股股份有限公司 | 用于鉴定和富集包含位点特异性基因组修饰的细胞的方法和组合物 |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| EP3380622A4 (en) | 2015-11-23 | 2019-08-07 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING IMMUNITY |
| WO2017093804A2 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| CA3009308A1 (en) | 2015-12-23 | 2017-06-29 | Chad Albert COWAN | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| US11083799B2 (en) | 2016-03-16 | 2021-08-10 | Crispr Therapeutics Ag | Materials and methods for treatment of hereditary haemochromatosis |
| ES3030658T3 (en) | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| CN105950626B (zh) | 2016-06-17 | 2018-09-28 | 新疆畜牧科学院生物技术研究所 | 基于CRISPR/Cas9获得不同毛色绵羊的方法及靶向ASIP基因的sgRNA |
| WO2018007871A1 (en) | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| KR102712926B1 (ko) | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| EP3551757A1 (en) | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| JP7383478B2 (ja) * | 2016-12-22 | 2023-11-20 | インテリア セラピューティクス，インコーポレイテッド | 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および方法 |
| US11597947B2 (en) * | 2016-12-29 | 2023-03-07 | Asc Therapeutics Inc. | Gene editing method using virus |
| IL311278B2 (en) | 2017-09-29 | 2026-05-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing |
| US20220080055A9 (en) * | 2017-10-17 | 2022-03-17 | Crispr Therapeutics Ag | Compositions and methods for gene editing for hemophilia a |
| IL326835A (en) * | 2018-10-16 | 2026-04-01 | Blueallele Llc | Methods for targeted insertion of dna genes |
-
2019
- 2019-10-18 BR BR112021007343-4A patent/BR112021007343A2/pt unknown
- 2019-10-18 AU AU2019361203A patent/AU2019361203B2/en active Active
- 2019-10-18 TW TW113136358A patent/TW202507011A/zh unknown
- 2019-10-18 MX MX2021004278A patent/MX2021004278A/es unknown
- 2019-10-18 CA CA3116918A patent/CA3116918A1/en active Pending
- 2019-10-18 JP JP2021521406A patent/JP7472121B2/ja active Active
- 2019-10-18 TW TW108137785A patent/TWI860300B/zh active
- 2019-10-18 US US16/657,944 patent/US20200270617A1/en not_active Abandoned
- 2019-10-18 CN CN201980083672.4A patent/CN114207130A/zh active Pending
- 2019-10-18 SG SG11202103733SA patent/SG11202103733SA/en unknown
- 2019-10-18 EP EP19813206.0A patent/EP3867381A2/en active Pending
- 2019-10-18 KR KR1020267010209A patent/KR20260048369A/ko active Pending
- 2019-10-18 WO PCT/US2019/057086 patent/WO2020082042A2/en not_active Ceased
- 2019-10-18 MY MYPI2021002128A patent/MY208564A/en unknown
- 2019-10-18 IL IL282236A patent/IL282236B1/en unknown
- 2019-10-18 IL IL328888A patent/IL328888A/en unknown
- 2019-10-18 KR KR1020217014887A patent/KR102949204B1/ko active Active
-
2021
- 2021-04-14 SA SA521421766A patent/SA521421766B1/ar unknown
- 2021-04-15 PH PH12021550844A patent/PH12021550844A1/en unknown
- 2021-04-16 US US17/233,373 patent/US20220354967A1/en not_active Abandoned
- 2021-05-18 CO CONC2021/0006363A patent/CO2021006363A2/es unknown
-
2024
- 2024-04-10 JP JP2024063210A patent/JP7820434B2/ja active Active
-
2025
- 2025-04-09 US US19/174,328 patent/US20260132408A1/en active Pending
-
2026
- 2026-06-25 AU AU2026204954A patent/AU2026204954A1/en active Pending
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