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 (https=) | 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 (2) | US20200270617A1 (https=) |
| EP (1) | EP3867381A2 (https=) |
| JP (2) | JP7472121B2 (https=) |
| KR (2) | KR102949204B1 (https=) |
| CN (1) | CN114207130A (https=) |
| AU (1) | AU2019361203B2 (https=) |
| BR (1) | BR112021007343A2 (https=) |
| CA (1) | CA3116918A1 (https=) |
| CO (1) | CO2021006363A2 (https=) |
| IL (1) | IL282236A (https=) |
| MX (1) | MX2021004278A (https=) |
| MY (1) | MY208564A (https=) |
| PH (1) | PH12021550844A1 (https=) |
| SA (1) | SA521421766B1 (https=) |
| SG (1) | SG11202103733SA (https=) |
| TW (2) | TWI860300B (https=) |
| WO (1) | WO2020082042A2 (https=) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
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| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| 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 |
| 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 |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| 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 |
| 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 |
| 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 |
| 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 |
| WO2025049524A1 (en) | 2023-08-28 | 2025-03-06 | 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 |
| 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 |
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| 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 |
| 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 |
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| 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 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| 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 |
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| CN105940013B (zh) | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
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| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| EP3429632B1 (en) | 2016-03-16 | 2023-01-04 | CRISPR Therapeutics AG | Materials and methods for treatment of hereditary haemochromatosis |
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| 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 |
| 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 |
| US11597947B2 (en) * | 2016-12-29 | 2023-03-07 | Asc Therapeutics Inc. | Gene editing method using virus |
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| MA50833A (fr) * | 2017-10-17 | 2020-08-26 | Bayer Healthcare Llc | Compositions et méthodes pour l'édition génique pour l'hémophilie a |
| SG11202103917VA (en) * | 2018-10-16 | 2021-05-28 | Blueallele Llc | Methods for targeted insertion of dna in genes |
-
2019
- 2019-10-18 US US16/657,944 patent/US20200270617A1/en not_active Abandoned
- 2019-10-18 CA CA3116918A patent/CA3116918A1/en active Pending
- 2019-10-18 EP EP19813206.0A patent/EP3867381A2/en active Pending
- 2019-10-18 TW TW108137785A patent/TWI860300B/zh active
- 2019-10-18 MX MX2021004278A patent/MX2021004278A/es unknown
- 2019-10-18 JP JP2021521406A patent/JP7472121B2/ja active Active
- 2019-10-18 KR KR1020217014887A patent/KR102949204B1/ko active Active
- 2019-10-18 MY MYPI2021002128A patent/MY208564A/en unknown
- 2019-10-18 CN CN201980083672.4A patent/CN114207130A/zh active Pending
- 2019-10-18 TW TW113136358A patent/TW202507011A/zh unknown
- 2019-10-18 SG SG11202103733SA patent/SG11202103733SA/en unknown
- 2019-10-18 BR BR112021007343-4A patent/BR112021007343A2/pt unknown
- 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 AU AU2019361203A patent/AU2019361203B2/en active Active
-
2021
- 2021-04-11 IL IL282236A patent/IL282236A/en unknown
- 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
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