JP2021500864A5 - - Google Patents
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- JP2021500864A5 JP2021500864A5 JP2020517500A JP2020517500A JP2021500864A5 JP 2021500864 A5 JP2021500864 A5 JP 2021500864A5 JP 2020517500 A JP2020517500 A JP 2020517500A JP 2020517500 A JP2020517500 A JP 2020517500A JP 2021500864 A5 JP2021500864 A5 JP 2021500864A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- seq
- guide
- ttr
- subject
- 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.)
- Pending
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- 239000000203 mixture Substances 0.000 claims 52
- 238000000034 method Methods 0.000 claims 27
- 125000003729 nucleotide group Chemical group 0.000 claims 24
- 108020005004 Guide RNA Proteins 0.000 claims 21
- 239000002773 nucleotide Substances 0.000 claims 19
- 101150091380 TTR gene Proteins 0.000 claims 11
- 239000011230 binding agent Substances 0.000 claims 10
- 230000035772 mutation Effects 0.000 claims 9
- 230000004568 DNA-binding Effects 0.000 claims 8
- 238000012986 modification Methods 0.000 claims 7
- 230000004048 modification Effects 0.000 claims 7
- 230000005782 double-strand break Effects 0.000 claims 6
- 108091027544 Subgenomic mRNA Proteins 0.000 claims 5
- 150000002632 lipids Chemical class 0.000 claims 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 4
- 102000001049 Amyloid Human genes 0.000 claims 4
- 108010094108 Amyloid Proteins 0.000 claims 4
- 230000027455 binding Effects 0.000 claims 4
- 210000004027 cell Anatomy 0.000 claims 4
- 210000002966 serum Anatomy 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 4
- 210000003494 hepatocyte Anatomy 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 2
- 206010060860 Neurological symptom Diseases 0.000 claims 2
- 238000009825 accumulation Methods 0.000 claims 2
- 206010002022 amyloidosis Diseases 0.000 claims 2
- 230000000747 cardiac effect Effects 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 230000037430 deletion Effects 0.000 claims 2
- 238000012217 deletion Methods 0.000 claims 2
- 230000011559 double-strand break repair via nonhomologous end joining Effects 0.000 claims 2
- 230000009977 dual effect Effects 0.000 claims 2
- 230000037431 insertion Effects 0.000 claims 2
- 238000003780 insertion Methods 0.000 claims 2
- 239000002105 nanoparticle Substances 0.000 claims 2
- 230000002265 prevention Effects 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
- 102220470698 BUD13 homolog_V30P_mutation Human genes 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 claims 1
- 206010007509 Cardiac amyloidosis Diseases 0.000 claims 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 1
- 208000034846 Familial Amyloid Neuropathies Diseases 0.000 claims 1
- 206010019889 Hereditary neuropathic amyloidosis Diseases 0.000 claims 1
- 108020004485 Nonsense Codon Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 102220558130 Olfactory receptor 2A1/2A42_V30L_mutation Human genes 0.000 claims 1
- 108700026244 Open Reading Frames Proteins 0.000 claims 1
- 230000003941 amyloidogenesis Effects 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000037433 frameshift Effects 0.000 claims 1
- 230000002496 gastric effect Effects 0.000 claims 1
- 230000006698 induction Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- GZQKNULLWNGMCW-PWQABINMSA-N lipid A (E. coli) Chemical compound O1[C@H](CO)[C@@H](OP(O)(O)=O)[C@H](OC(=O)C[C@@H](CCCCCCCCCCC)OC(=O)CCCCCCCCCCCCC)[C@@H](NC(=O)C[C@@H](CCCCCCCCCCC)OC(=O)CCCCCCCCCCC)[C@@H]1OC[C@@H]1[C@@H](O)[C@H](OC(=O)C[C@H](O)CCCCCCCCCCC)[C@@H](NC(=O)C[C@H](O)CCCCCCCCCCC)[C@@H](OP(O)(O)=O)O1 GZQKNULLWNGMCW-PWQABINMSA-N 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 210000005036 nerve Anatomy 0.000 claims 1
- 230000037434 nonsense mutation Effects 0.000 claims 1
- 230000001575 pathological effect Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 102200150628 rs151220873 Human genes 0.000 claims 1
- 102200017272 rs28931576 Human genes 0.000 claims 1
- 102220128569 rs777178486 Human genes 0.000 claims 1
- 201000007905 transthyretin amyloidosis Diseases 0.000 claims 1
- 150000003626 triacylglycerols Chemical class 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022194564A JP7707140B2 (ja) | 2017-09-29 | 2022-12-06 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2025112164A JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566236P | 2017-09-29 | 2017-09-29 | |
| US62/566,236 | 2017-09-29 | ||
| US201862671902P | 2018-05-15 | 2018-05-15 | |
| US62/671,902 | 2018-05-15 | ||
| PCT/US2018/053382 WO2019067872A1 (en) | 2017-09-29 | 2018-09-28 | COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022194564A Division JP7707140B2 (ja) | 2017-09-29 | 2022-12-06 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021500864A JP2021500864A (ja) | 2021-01-14 |
| JP2021500864A5 true JP2021500864A5 (https=) | 2021-11-04 |
Family
ID=63858214
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Pending JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2022194564A Active JP7707140B2 (ja) | 2017-09-29 | 2022-12-06 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2025112164A Pending JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022194564A Active JP7707140B2 (ja) | 2017-09-29 | 2022-12-06 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2025112164A Pending JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (4) | US20200248180A1 (https=) |
| EP (1) | EP3688161A1 (https=) |
| JP (3) | JP2021500864A (https=) |
| KR (1) | KR20200058509A (https=) |
| CN (2) | CN118530993A (https=) |
| AU (2) | AU2018338787C1 (https=) |
| BR (1) | BR112020005287A2 (https=) |
| CA (1) | CA3077251A1 (https=) |
| CO (1) | CO2020005116A2 (https=) |
| IL (3) | IL311170B1 (https=) |
| MX (2) | MX2020003608A (https=) |
| MY (1) | MY203849A (https=) |
| PH (1) | PH12020550364A1 (https=) |
| SG (1) | SG11202002565YA (https=) |
| TW (1) | TWI904069B (https=) |
| WO (1) | WO2019067872A1 (https=) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| MX394999B (es) | 2017-09-29 | 2025-03-24 | Regeneron Pharma | Animales no humanos que comprenden un locus ttr humanizado y metodos de uso |
| JP7543259B2 (ja) | 2018-10-18 | 2024-09-02 | アクイタス セラピューティクス インコーポレイテッド | 活性剤の脂質ナノ粒子送達のための脂質 |
| AU2020248337A1 (en) * | 2019-03-28 | 2021-11-04 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof |
| EP3946598A1 (en) * | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
| BR112021019224A2 (pt) * | 2019-03-28 | 2021-11-30 | Intellia Therapeutics Inc | Polinucleotídeos, composições e métodos para expressão de polipeptídeo |
| US11891618B2 (en) | 2019-06-04 | 2024-02-06 | Regeneron Pharmaceuticals, Inc. | Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| MX2022006950A (es) * | 2019-12-11 | 2022-11-07 | Intellia Therapeutics Inc | Arn guía modificados para edición de genes. |
| EP4237005A4 (en) * | 2020-10-28 | 2024-10-09 | Novo Nordisk A/S | ANTI-TRANSTHYRETIN ANTIBODIES AND METHODS OF USE THEREOF |
| CR20230305A (es) | 2020-12-11 | 2023-11-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación |
| CN112795595A (zh) * | 2020-12-24 | 2021-05-14 | 中山大学 | 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统 |
| WO2022246266A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| KR20240038705A (ko) | 2021-06-22 | 2024-03-25 | 인텔리아 테라퓨틱스, 인크. | 간 유전자의 생체 내 편집 방법 |
| US20250127927A1 (en) * | 2021-08-23 | 2025-04-24 | The Board Of Trustees Of The Leland Stanford Junior University | Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells |
| AU2022345881A1 (en) | 2021-09-20 | 2024-03-21 | Alnylam Pharmaceuticals, Inc. | Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof |
| CN116064598B (zh) * | 2021-10-08 | 2024-03-12 | 苏州艾博生物科技有限公司 | 冠状病毒的核酸疫苗 |
| WO2023185697A2 (en) * | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| TW202417017A (zh) * | 2022-06-30 | 2024-05-01 | 香港商正基基因科技有限公司 | 具有化學修飾的導引rna |
| CN118853659A (zh) * | 2023-04-14 | 2024-10-29 | 深圳瑞吉生物科技有限公司 | 提高mRNA翻译蛋白的输出量的核酸及其应用 |
| WO2024259135A1 (en) | 2023-06-13 | 2024-12-19 | Intellia Therapeutics, Inc. | Assays for analysis of ribonucleic acid (rna) molecules |
| CN120265763A (zh) * | 2023-10-25 | 2025-07-04 | 上海津曼特生物科技有限公司 | 靶向ttr的基因编辑组合物 |
| 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 |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| US6169169B1 (en) | 1994-05-19 | 2001-01-02 | Dako A/S | PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis |
| US6974667B2 (en) | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
| US7244764B2 (en) * | 2003-06-23 | 2007-07-17 | Neurochem (International) Limited | Methods and compositions for treating amyloid-related diseases |
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| WO2010017509A1 (en) | 2008-08-07 | 2010-02-11 | Isis Pharmaceuticals, Inc. | Modulation of transthyretin expression for the treatment of cns related disorders |
| EA036772B1 (ru) | 2008-10-20 | 2020-12-18 | Элнилэм Фармасьютикалз, Инк. | Композиции и способы для ингибирования экспрессии транстиретина |
| US9101643B2 (en) | 2009-11-03 | 2015-08-11 | Alnylam Pharmaceuticals, Inc. | Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR) |
| RS56011B1 (sr) | 2010-04-29 | 2017-09-29 | Ionis Pharmaceuticals Inc | Modulacija ekspresije transtiretina |
| HUE048622T2 (hu) | 2011-11-18 | 2020-08-28 | Alnylam Pharmaceuticals Inc | RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| DK2931897T3 (en) * | 2012-12-12 | 2018-02-05 | Broad Inst Inc | CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS |
| DK2931898T3 (en) | 2012-12-12 | 2016-06-20 | Massachusetts Inst Technology | CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS |
| CA2895155C (en) | 2012-12-17 | 2021-07-06 | President And Fellows Of Harvard College | Rna-guided human genome engineering |
| EP3608308B1 (en) | 2013-03-08 | 2021-07-21 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| SI2986729T1 (sl) | 2013-04-16 | 2019-02-28 | Regeneron Pharmaceuticals, Inc. | Ciljana sprememba genoma podgane |
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| KR20160019553A (ko) * | 2013-06-17 | 2016-02-19 | 더 브로드 인스티튜트, 인코퍼레이티드 | 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| ES2895651T3 (es) | 2013-12-19 | 2022-02-22 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| CN106687601A (zh) * | 2014-05-28 | 2017-05-17 | 株式会社图尔金 | 使用靶特异性核酸酶灵敏检测靶dna的方法 |
| KR102386101B1 (ko) * | 2014-06-26 | 2022-04-14 | 리제너론 파마슈티칼스 인코포레이티드 | 표적화된 유전자 변형을 위한 방법 및 그 조성물, 및 사용 방법 |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| EP3265559B1 (en) | 2015-03-03 | 2021-01-06 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| WO2017053431A2 (en) * | 2015-09-21 | 2017-03-30 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
| ES2964690T3 (es) | 2015-09-21 | 2024-04-09 | Trilink Biotechnologies Llc | Método para sintetizar ARN con caperuza 5' |
| CA2999649A1 (en) * | 2015-11-06 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1a |
| CA3006618A1 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| US11083799B2 (en) * | 2016-03-16 | 2021-08-10 | Crispr Therapeutics Ag | Materials and methods for treatment of hereditary haemochromatosis |
| 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 |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| AU2017374044B2 (en) | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
-
2018
- 2018-09-28 EP EP18786638.9A patent/EP3688161A1/en active Pending
- 2018-09-28 IL IL311170A patent/IL311170B1/en unknown
- 2018-09-28 AU AU2018338787A patent/AU2018338787C1/en active Active
- 2018-09-28 MY MYPI2020001489A patent/MY203849A/en unknown
- 2018-09-28 WO PCT/US2018/053382 patent/WO2019067872A1/en not_active Ceased
- 2018-09-28 BR BR112020005287-6A patent/BR112020005287A2/pt unknown
- 2018-09-28 CN CN202410646325.0A patent/CN118530993A/zh active Pending
- 2018-09-28 CA CA3077251A patent/CA3077251A1/en active Pending
- 2018-09-28 JP JP2020517500A patent/JP2021500864A/ja active Pending
- 2018-09-28 CN CN201880076628.6A patent/CN111417728A/zh active Pending
- 2018-09-28 TW TW107134336A patent/TWI904069B/zh active
- 2018-09-28 SG SG11202002565YA patent/SG11202002565YA/en unknown
- 2018-09-28 KR KR1020207012163A patent/KR20200058509A/ko active Pending
- 2018-09-28 MX MX2020003608A patent/MX2020003608A/es unknown
-
2020
- 2020-03-15 IL IL273317A patent/IL273317A/en unknown
- 2020-03-24 US US16/828,573 patent/US20200248180A1/en not_active Abandoned
- 2020-03-27 PH PH12020550364A patent/PH12020550364A1/en unknown
- 2020-04-24 CO CONC2020/0005116A patent/CO2020005116A2/es unknown
- 2020-07-13 MX MX2025014378A patent/MX2025014378A/es unknown
-
2022
- 2022-12-06 JP JP2022194564A patent/JP7707140B2/ja active Active
- 2022-12-09 US US18/063,972 patent/US11965165B2/en active Active
- 2022-12-09 US US18/064,086 patent/US11795460B2/en active Active
-
2024
- 2024-11-07 US US18/940,080 patent/US20250207130A1/en active Pending
-
2025
- 2025-04-17 AU AU2025202742A patent/AU2025202742A1/en active Pending
- 2025-07-02 JP JP2025112164A patent/JP2025163016A/ja active Pending
- 2025-12-18 IL IL325460A patent/IL325460A/en unknown
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