JP2019531068A5 - - Google Patents
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- JP2019531068A5 JP2019531068A5 JP2019512750A JP2019512750A JP2019531068A5 JP 2019531068 A5 JP2019531068 A5 JP 2019531068A5 JP 2019512750 A JP2019512750 A JP 2019512750A JP 2019512750 A JP2019512750 A JP 2019512750A JP 2019531068 A5 JP2019531068 A5 JP 2019531068A5
- Authority
- JP
- Japan
- Prior art keywords
- domain
- fusion molecule
- gene
- hepatocyte
- dna binding
- 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.)
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- 210000003494 hepatocyte Anatomy 0.000 claims description 16
- 108090000623 proteins and genes Proteins 0.000 claims description 16
- 230000004927 fusion Effects 0.000 claims description 12
- 230000004568 DNA-binding Effects 0.000 claims description 10
- 108091033319 polynucleotide Proteins 0.000 claims description 6
- 102000040430 polynucleotide Human genes 0.000 claims description 6
- 239000002157 polynucleotide Substances 0.000 claims description 6
- 102100022712 Alpha-1-antitrypsin Human genes 0.000 claims description 5
- 102100034869 Plasma kallikrein Human genes 0.000 claims description 4
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 claims description 4
- 108091023040 Transcription factor Proteins 0.000 claims description 4
- 102000040945 Transcription factor Human genes 0.000 claims description 4
- 238000003776 cleavage reaction Methods 0.000 claims description 4
- 230000035772 mutation Effects 0.000 claims description 4
- 239000008194 pharmaceutical composition Substances 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 230000007017 scission Effects 0.000 claims description 4
- 230000002103 transcriptional effect Effects 0.000 claims description 4
- 101000823116 Homo sapiens Alpha-1-antitrypsin Proteins 0.000 claims description 3
- 108020005004 Guide RNA Proteins 0.000 claims description 2
- 101150105486 HAO1 gene Proteins 0.000 claims description 2
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims description 2
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 2
- 101001031589 Homo sapiens 2-Hydroxyacid oxidase 1 Proteins 0.000 claims description 2
- 101001091365 Homo sapiens Plasma kallikrein Proteins 0.000 claims description 2
- 101001098868 Homo sapiens Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 claims description 2
- 208000035150 Hypercholesterolemia Diseases 0.000 claims description 2
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims description 2
- 208000008852 Hyperoxaluria Diseases 0.000 claims description 2
- 101150038962 KLKB1 gene Proteins 0.000 claims description 2
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 101150094724 PCSK9 gene Proteins 0.000 claims description 2
- 101150086419 Serpina11 gene Proteins 0.000 claims description 2
- -1 TTR Proteins 0.000 claims description 2
- 101150091380 TTR gene Proteins 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims description 2
- 101710185494 Zinc finger protein Proteins 0.000 claims description 2
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 2
- 206010002022 amyloidosis Diseases 0.000 claims description 2
- 230000007812 deficiency Effects 0.000 claims description 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims description 2
- 230000001404 mediated effect Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 230000003612 virological effect Effects 0.000 claims description 2
- 210000005229 liver cell Anatomy 0.000 claims 2
- HYIMSNHJOBLJNT-UHFFFAOYSA-N nifedipine Chemical compound COC(=O)C1=C(C)NC(C)=C(C(=O)OC)C1C1=CC=CC=C1[N+]([O-])=O HYIMSNHJOBLJNT-UHFFFAOYSA-N 0.000 claims 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023057789A JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662384428P | 2016-09-07 | 2016-09-07 | |
| US62/384,428 | 2016-09-07 | ||
| PCT/US2017/050446 WO2018049009A2 (en) | 2016-09-07 | 2017-09-07 | Modulation of liver genes |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023057789A Division JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019531068A JP2019531068A (ja) | 2019-10-31 |
| JP2019531068A5 true JP2019531068A5 (cg-RX-API-DMAC7.html) | 2020-10-15 |
| JP7256739B2 JP7256739B2 (ja) | 2023-04-12 |
Family
ID=61281860
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019512750A Active JP7256739B2 (ja) | 2016-09-07 | 2017-09-07 | 肝臓遺伝子のモジュレーション |
| JP2023057789A Withdrawn JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023057789A Withdrawn JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US10960085B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3509645A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7256739B2 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017324462B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3035534A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018049009A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11504389B2 (en) | 2016-12-01 | 2022-11-22 | Sangamo Therapeutics, Inc. | Tau modulators and methods and compositions for delivery thereof |
| EP3615510B1 (en) | 2017-04-28 | 2024-03-27 | Acuitas Therapeutics, Inc. | Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| WO2019067875A1 (en) | 2017-09-29 | 2019-04-04 | Regeneron Pharmaceuticals, Inc. | NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE |
| WO2020010186A1 (en) * | 2018-07-06 | 2020-01-09 | Derek Klarin | Pcsk9 variants |
| CN112867795A (zh) * | 2018-07-31 | 2021-05-28 | 因特利亚治疗公司 | 用于执行羟基酸氧化酶1（hao1）基因编辑以治疗1型原发性高草酸尿症（ph1）的组合物和方法 |
| CA3113449A1 (en) | 2018-09-21 | 2020-03-26 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| CN113226333A (zh) | 2018-10-02 | 2021-08-06 | 桑格摩生物治疗股份有限公司 | 用于调控Tau蛋白的方法和组合物 |
| CA3115158A1 (en) * | 2018-10-02 | 2020-04-09 | Sangamo Therapeutics, Inc. | Engineered genetic modulators |
| WO2020082046A2 (en) | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| EP3898661A1 (en) * | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| IL284535B2 (en) | 2019-01-11 | 2025-03-01 | Acuitas Therapeutics Inc | Lipids for lipid nanoparticle delivery of active ingredients |
| AU2020228028A1 (en) * | 2019-02-25 | 2021-09-30 | University Of Massachusetts | DNA-binding domain transactivators and uses thereof |
| WO2020247452A1 (en) | 2019-06-04 | 2020-12-10 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use |
| US20230081547A1 (en) * | 2020-02-07 | 2023-03-16 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized klkb1 locus and methods of use |
| KR20230051172A (ko) | 2020-07-16 | 2023-04-17 | 아퀴타스 테라퓨틱스 인크. | 지질 나노 입자에 사용하기 위한 양이온성 지질 |
| AR123041A1 (es) * | 2020-07-24 | 2022-10-26 | Univ Massachusetts | Transactivadores del dominio de unión al adn y usos de los mismos |
| KR102826438B1 (ko) | 2020-11-12 | 2025-07-04 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| WO2022256655A2 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| WO2023039447A2 (en) * | 2021-09-08 | 2023-03-16 | Flagship Pioneering Innovations Vi, Llc | Serpina-modulating compositions and methods |
| KR20240123832A (ko) | 2021-12-16 | 2024-08-14 | 아퀴타스 테라퓨틱스 인크. | 지질 나노입자 제형에 사용하기 위한 지질 |
| US20250360224A1 (en) * | 2022-03-11 | 2025-11-27 | Epicrispr Biotechnologies, Inc. | Systems and methods for genetic modulation to treat liver disease |
| JP2025524360A (ja) | 2022-06-07 | 2025-07-30 | スクライブ・セラピューティクス・インコーポレイテッド | Ｐｃｓｋ９の標的化のための組成物及び方法 |
| AU2024248139A1 (en) | 2023-03-29 | 2025-09-25 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| WO2024229020A2 (en) * | 2023-05-01 | 2024-11-07 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of pcsk9 expression |
Family Cites Families (89)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2181548C (en) | 1994-01-18 | 2009-11-03 | Carlos F. Barbas, Iii | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| USRE45721E1 (en) | 1994-08-20 | 2015-10-06 | Gendaq, Ltd. | Relating to binding proteins for recognition of DNA |
| US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| ATE466952T1 (de) | 1998-03-02 | 2010-05-15 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| US6503717B2 (en) | 1999-12-06 | 2003-01-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| ATE353361T1 (de) | 2000-04-28 | 2007-02-15 | Sangamo Biosciences Inc | Gezielten modifikation der chromatinstruktur |
| AU2001257331A1 (en) | 2000-04-28 | 2001-11-12 | Sangamo Biosciences, Inc. | Methods for designing exogenous regulatory molecules |
| US7029847B2 (en) | 2000-05-16 | 2006-04-18 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| US20030044787A1 (en) | 2000-05-16 | 2003-03-06 | Joung J. Keith | Methods and compositions for interaction trap assays |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| US7053264B2 (en) | 2000-09-28 | 2006-05-30 | Sangamo Biosciences, Inc. | Nuclear reprogramming using IWSI and related chromatin remodeling ATPases |
| US6919204B2 (en) | 2000-09-29 | 2005-07-19 | Sangamo Biosciences, Inc. | Modulation of gene expression using localization domains |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| WO2003016496A2 (en) | 2001-08-20 | 2003-02-27 | The Scripps Research Institute | Zinc finger binding domains for cnn |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| CN100575485C (zh) | 2002-01-23 | 2009-12-30 | 犹他大学研究基金会 | 使用锌指核酸酶的定向染色体诱变 |
| EP2368982A3 (en) | 2002-03-21 | 2011-10-12 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| JP2006502748A (ja) | 2002-09-05 | 2006-01-26 | カリフォルニア インスティテュート オブ テクノロジー | 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法 |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| AU2005233550B2 (en) | 2004-04-08 | 2010-11-18 | Sangamo Therapeutics, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| EP1789095A2 (en) | 2004-09-16 | 2007-05-30 | Sangamo Biosciences Inc. | Compositions and methods for protein production |
| CA2651494C (en) | 2006-05-25 | 2015-09-29 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
| EP2019839B1 (en) | 2006-05-25 | 2011-12-07 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| CA2680832A1 (en) * | 2007-03-27 | 2008-10-02 | Merck & Co., Inc. | Method for detecting autoprocessed, secreted pcsk9 |
| EP2137310B1 (en) | 2007-04-26 | 2010-11-24 | Sangamo BioSciences, Inc. | Targeted integration into the ppp1r12c locus |
| US8563314B2 (en) | 2007-09-27 | 2013-10-22 | Sangamo Biosciences, Inc. | Methods and compositions for modulating PD1 |
| JP2011518555A (ja) | 2008-04-14 | 2011-06-30 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 標的組込みのための線形ドナーコンストラクト |
| US9617548B2 (en) | 2008-04-22 | 2017-04-11 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| KR20160015400A (ko) | 2008-08-22 | 2016-02-12 | 상가모 바이오사이언스 인코포레이티드 | 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물 |
| CA2745031C (en) * | 2008-12-04 | 2018-08-14 | Sangamo Biosciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| EP2727600B1 (en) * | 2009-07-28 | 2019-03-27 | Sangamo Therapeutics, Inc. | Zinc finger fusion proteins for repressing a huntington gene |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| HUE041436T2 (hu) | 2009-12-10 | 2019-05-28 | Univ Minnesota | Tal-effektor-közvetített DNS-módosítás |
| AU2011213242B2 (en) | 2010-02-08 | 2015-01-29 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| JP2013518602A (ja) | 2010-02-09 | 2013-05-23 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 部分的に一本鎖のドナー分子による標的化ゲノム改変 |
| KR101880536B1 (ko) | 2010-04-26 | 2018-07-23 | 상가모 테라퓨틱스, 인코포레이티드 | 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집 |
| WO2011146121A1 (en) | 2010-05-17 | 2011-11-24 | Sangamo Biosciences, Inc. | Novel dna-binding proteins and uses thereof |
| CA2805442C (en) | 2010-07-21 | 2020-05-12 | Sangamo Biosciences, Inc. | Methods and compositions for modification of an hla locus |
| CN103270046B (zh) | 2010-10-12 | 2016-05-11 | 费城儿童医院 | 治疗b型血友病的方法和组合物 |
| EP2758529B1 (en) | 2011-09-21 | 2019-03-20 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| AU2012328682B2 (en) | 2011-10-27 | 2017-09-21 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of the HPRT locus |
| HK1200871A1 (en) | 2011-11-16 | 2015-08-14 | Sangamo Therapeutics, Inc. | Modified dna-binding proteins and uses thereof |
| CN108285491B (zh) * | 2012-02-29 | 2021-08-10 | 桑格摩生物科学股份有限公司 | 治疗亨廷顿氏病的方法和组合物 |
| AU2013259647B2 (en) * | 2012-05-07 | 2018-11-08 | Corteva Agriscience Llc | Methods and compositions for nuclease-mediated targeted integration of transgenes |
| EP2872625B1 (en) | 2012-07-11 | 2016-11-16 | Sangamo BioSciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| AU2013308770B2 (en) | 2012-08-29 | 2019-01-17 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| HK1215873A1 (zh) | 2012-12-05 | 2016-09-23 | 桑格摩治疗股份有限公司 | 調整代謝紊亂的方法及組合物 |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| SG10201704932UA (en) * | 2012-12-17 | 2017-07-28 | Harvard College | Rna-guided human genome engineering |
| HK1214920A1 (zh) * | 2013-04-05 | 2016-08-12 | 美国陶氏益农公司 | 用於在植物基因組內整合外源序列的方法和組合物 |
| CA2910489A1 (en) | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| US20140356956A1 (en) | 2013-06-04 | 2014-12-04 | President And Fellows Of Harvard College | RNA-Guided Transcriptional Regulation |
| EP3825406A1 (en) * | 2013-06-17 | 2021-05-26 | The Broad Institute Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
| HRP20200163T1 (hr) * | 2013-06-27 | 2020-08-07 | Roche Innovation Center Copenhagen A/S | Antisens oligomeri i konjugati koji ciljno djeluju na pcsk9 |
| CA2920899C (en) | 2013-08-28 | 2023-02-28 | Sangamo Biosciences, Inc. | Compositions for linking dna-binding domains and cleavage domains |
| MX362066B (es) * | 2013-11-04 | 2019-01-07 | Dow Agrosciences Llc | Loci óptimos de soya. |
| JP6633532B2 (ja) * | 2013-11-04 | 2020-01-22 | ダウ アグロサイエンシィズ エルエルシー | 最適なトウモロコシ遺伝子座 |
| HUE056436T2 (hu) * | 2013-11-13 | 2022-02-28 | Childrens Medical Center | Nukleáz közvetítette génexpresszió-szabályozás |
| HUE051628T2 (hu) | 2013-12-09 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és kompozíciók genom-manipulálására |
| JP2017515819A (ja) | 2014-05-08 | 2017-06-15 | サンガモ セラピューティクス， インコーポレイテッド | ハンチントン病を処置するための方法および組成物 |
| US9574211B2 (en) | 2014-05-13 | 2017-02-21 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
| KR102766526B1 (ko) | 2015-10-28 | 2025-02-13 | 상가모 테라퓨틱스, 인코포레이티드 | 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법 |
| CN108778297B (zh) | 2015-12-18 | 2024-02-09 | 桑格摩生物治疗股份有限公司 | T细胞受体的靶向破坏 |
| CN109069568B (zh) | 2016-02-02 | 2023-07-07 | 桑格摩生物治疗股份有限公司 | 用于连接dna结合结构域和切割结构域的组合物 |
| KR20240144493A (ko) | 2016-08-24 | 2024-10-02 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
-
2017
- 2017-09-07 CA CA3035534A patent/CA3035534A1/en active Pending
- 2017-09-07 US US15/697,917 patent/US10960085B2/en not_active Expired - Fee Related
- 2017-09-07 AU AU2017324462A patent/AU2017324462B2/en not_active Expired - Fee Related
- 2017-09-07 WO PCT/US2017/050446 patent/WO2018049009A2/en not_active Ceased
- 2017-09-07 EP EP17849517.2A patent/EP3509645A4/en not_active Withdrawn
- 2017-09-07 JP JP2019512750A patent/JP7256739B2/ja active Active
-
2020
- 2020-11-12 US US17/096,502 patent/US20210060179A1/en not_active Abandoned
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2023
- 2023-03-31 JP JP2023057789A patent/JP2023082133A/ja not_active Withdrawn
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