JP2020501582A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020501582A5 JP2020501582A5 JP2019533577A JP2019533577A JP2020501582A5 JP 2020501582 A5 JP2020501582 A5 JP 2020501582A5 JP 2019533577 A JP2019533577 A JP 2019533577A JP 2019533577 A JP2019533577 A JP 2019533577A JP 2020501582 A5 JP2020501582 A5 JP 2020501582A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- seq
- rna
- guide
- sequence
- 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
- 239000000203 mixture Substances 0.000 claims 38
- 238000000034 method Methods 0.000 claims 23
- 125000003729 nucleotide group Chemical group 0.000 claims 16
- 102100022712 Alpha-1-antitrypsin Human genes 0.000 claims 12
- 239000011230 binding agent Substances 0.000 claims 11
- 239000002773 nucleotide Substances 0.000 claims 11
- 230000004568 DNA-binding Effects 0.000 claims 10
- 108020005004 Guide RNA Proteins 0.000 claims 10
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims 10
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims 10
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims 10
- 101150069374 Serpina1 gene Proteins 0.000 claims 9
- 238000012986 modification Methods 0.000 claims 7
- 230000004048 modification Effects 0.000 claims 7
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 6
- 230000005782 double-strand break Effects 0.000 claims 6
- 150000002632 lipids Chemical class 0.000 claims 5
- 210000004185 liver Anatomy 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 4
- 108700028369 Alleles Proteins 0.000 claims 3
- 108091027544 Subgenomic mRNA Proteins 0.000 claims 3
- 230000027455 binding Effects 0.000 claims 3
- 230000006780 non-homologous end joining Effects 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 101000823116 Homo sapiens Alpha-1-antitrypsin Proteins 0.000 claims 2
- 238000009825 accumulation Methods 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
- 230000037430 deletion Effects 0.000 claims 2
- 238000012217 deletion Methods 0.000 claims 2
- 210000003494 hepatocyte Anatomy 0.000 claims 2
- 230000037431 insertion Effects 0.000 claims 2
- 238000003780 insertion Methods 0.000 claims 2
- 208000016332 liver symptom Diseases 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 239000002105 nanoparticle Substances 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
- 206010003445 Ascites Diseases 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 1
- 108700024394 Exon Proteins 0.000 claims 1
- 101100268523 Homo sapiens SERPINA1 gene Proteins 0.000 claims 1
- 206010023126 Jaundice Diseases 0.000 claims 1
- 108020004485 Nonsense Codon Proteins 0.000 claims 1
- 206010030113 Oedema Diseases 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 108010041758 cleavase Proteins 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000003111 delayed effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000037433 frameshift 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
- 210000005229 liver cell Anatomy 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 230000007935 neutral effect Effects 0.000 claims 1
- 230000037434 nonsense mutation Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 230000006641 stabilisation Effects 0.000 claims 1
- 238000011105 stabilization Methods 0.000 claims 1
- 238000002054 transplantation Methods 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023026853A JP2023075164A (ja) | 2016-12-22 | 2023-02-24 | 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 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662438219P | 2016-12-22 | 2016-12-22 | |
| US62/438,219 | 2016-12-22 | ||
| PCT/US2017/067800 WO2018119182A1 (en) | 2016-12-22 | 2017-12-21 | Compositions and methods for treating alpha-1 antitrypsin deficiency |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023026853A Division JP2023075164A (ja) | 2016-12-22 | 2023-02-24 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020501582A JP2020501582A (ja) | 2020-01-23 |
| JP2020501582A5 true JP2020501582A5 (enExample) | 2021-01-28 |
| JP7383478B2 JP7383478B2 (ja) | 2023-11-20 |
Family
ID=61054487
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019533577A Active JP7383478B2 (ja) | 2016-12-22 | 2017-12-21 | 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 |
| JP2023026853A Pending JP2023075164A (ja) | 2016-12-22 | 2023-02-24 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023026853A Pending JP2023075164A (ja) | 2016-12-22 | 2023-02-24 | 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 |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US11549107B2 (enExample) |
| EP (1) | EP3559232A1 (enExample) |
| JP (2) | JP7383478B2 (enExample) |
| KR (1) | KR102551664B1 (enExample) |
| CN (2) | CN110382697B (enExample) |
| AU (2) | AU2017379073B2 (enExample) |
| BR (1) | BR112019012825A2 (enExample) |
| CA (1) | CA3047415A1 (enExample) |
| CO (1) | CO2019007776A2 (enExample) |
| EA (1) | EA201991455A1 (enExample) |
| IL (1) | IL267448B2 (enExample) |
| MX (2) | MX2019007594A (enExample) |
| MY (1) | MY204487A (enExample) |
| PH (1) | PH12019501457A1 (enExample) |
| SA (1) | SA519402192B1 (enExample) |
| SG (1) | SG10202106412RA (enExample) |
| TW (2) | TW202509233A (enExample) |
| WO (1) | WO2018119182A1 (enExample) |
| ZA (1) | ZA202007632B (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102551664B1 (ko) * | 2016-12-22 | 2023-07-05 | 인텔리아 테라퓨틱스, 인크. | 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 |
| AR113031A1 (es) * | 2017-09-29 | 2020-01-15 | Intellia Therapeutics Inc | Composiciones de nanopartículas lipídicas (lnp) que comprende arn |
| JP7642531B2 (ja) | 2018-05-11 | 2025-03-10 | ビーム セラピューティクス インク． | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
| US20210130818A1 (en) * | 2018-07-04 | 2021-05-06 | Yale University | Compositions and Methods for Enhancement of Homology-Directed Repair Mediated Precise Gene Editing by Programming DNA Repair with a Single RNA-Guided Endonuclease |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| BR112021007289A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para tratar deficiência de alfa-1 antitripsina |
| EP3867380A2 (en) | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| JP7472121B2 (ja) * | 2018-10-18 | 2024-04-22 | インテリア セラピューティクス，インコーポレーテッド | 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 |
| US20220160847A1 (en) * | 2020-11-20 | 2022-05-26 | Mark Egly | Method of preventing and/or treating a plurality of diseases |
| WO2022125968A1 (en) | 2020-12-11 | 2022-06-16 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing involving deamination |
| US12171813B2 (en) | 2021-02-05 | 2024-12-24 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| 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 (26)
| 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) |
| AU2522095A (en) | 1994-05-19 | 1995-12-18 | Dako A/S | Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| EP3138911B1 (en) * | 2012-12-06 | 2018-12-05 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
| EA201891018A1 (ru) * | 2013-03-08 | 2018-09-28 | Новартис Аг | Липиды и липидные композиции для доставки активных агентов |
| US9885033B2 (en) * | 2013-03-15 | 2018-02-06 | The General Hospital Corporation | Increasing specificity for RNA-guided genome editing |
| DK3011031T3 (da) * | 2013-06-17 | 2020-12-21 | Broad Inst Inc | Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi |
| ES2905257T3 (es) * | 2013-07-03 | 2022-04-07 | Dicerna Pharmaceuticals Inc | Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario |
| CA3109801C (en) * | 2013-08-22 | 2024-01-09 | Andrew Cigan | Plant genome modification using guide rna/cas endonuclease systems and methods of use |
| CA2930015A1 (en) * | 2013-11-07 | 2015-05-14 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| US9840699B2 (en) * | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| AU2014370416B2 (en) | 2013-12-26 | 2021-03-11 | The General Hospital Corporation | Multiplex guide RNAs |
| CN107250148B (zh) * | 2014-12-03 | 2021-04-16 | 安捷伦科技有限公司 | 具有化学修饰的指导rna |
| WO2016164356A1 (en) * | 2015-04-06 | 2016-10-13 | The Board Of Trustees Of The Leland Stanford Junior University | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
| DK4144844T3 (da) * | 2015-10-12 | 2025-11-24 | Dupont Us Holding Llc | Beskyttede dna-templates til genmodificering og øget homolog rekombination i celler og fremgangsmåder til anvendelse deraf |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| WO2017093804A2 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| EP3871695A1 (en) * | 2015-12-07 | 2021-09-01 | Arc Bio, LLC | Methods and compositions for the making and using of guide nucleic acids |
| US11512311B2 (en) * | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| JP7245651B2 (ja) * | 2016-03-30 | 2023-03-24 | インテリア セラピューティクス，インコーポレイテッド | Ｃｒｉｓｐｒ／ｃａｓ構成成分のための脂質ナノ粒子製剤 |
| BR112018071439A2 (pt) | 2016-04-22 | 2019-03-19 | Intellia Therapeutics, Inc. | composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro |
| TWI835719B (zh) * | 2016-12-08 | 2024-03-21 | 美商英特利亞醫療公司 | 經修飾之嚮導rna |
| KR102551664B1 (ko) * | 2016-12-22 | 2023-07-05 | 인텔리아 테라퓨틱스, 인크. | 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 |
| BR112021007289A2 (pt) * | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para tratar deficiência de alfa-1 antitripsina |
-
2017
- 2017-12-21 KR KR1020197020995A patent/KR102551664B1/ko active Active
- 2017-12-21 EA EA201991455A patent/EA201991455A1/ru unknown
- 2017-12-21 MX MX2019007594A patent/MX2019007594A/es unknown
- 2017-12-21 MY MYPI2019003394A patent/MY204487A/en unknown
- 2017-12-21 CA CA3047415A patent/CA3047415A1/en active Pending
- 2017-12-21 SG SG10202106412RA patent/SG10202106412RA/en unknown
- 2017-12-21 EP EP17835903.0A patent/EP3559232A1/en active Pending
- 2017-12-21 CN CN201780086243.3A patent/CN110382697B/zh active Active
- 2017-12-21 WO PCT/US2017/067800 patent/WO2018119182A1/en not_active Ceased
- 2017-12-21 JP JP2019533577A patent/JP7383478B2/ja active Active
- 2017-12-21 TW TW113125536A patent/TW202509233A/zh unknown
- 2017-12-21 AU AU2017379073A patent/AU2017379073B2/en active Active
- 2017-12-21 BR BR112019012825A patent/BR112019012825A2/pt not_active Application Discontinuation
- 2017-12-21 TW TW106145118A patent/TWI851534B/zh active
- 2017-12-21 CN CN202311710568.8A patent/CN117737062A/zh active Pending
- 2017-12-21 IL IL267448A patent/IL267448B2/en unknown
-
2019
- 2019-06-20 SA SA519402192A patent/SA519402192B1/ar unknown
- 2019-06-20 US US16/446,764 patent/US11549107B2/en active Active
- 2019-06-21 PH PH12019501457A patent/PH12019501457A1/en unknown
- 2019-06-21 MX MX2025002623A patent/MX2025002623A/es unknown
- 2019-07-19 CO CONC2019/0007776A patent/CO2019007776A2/es unknown
-
2020
- 2020-12-08 ZA ZA2020/07632A patent/ZA202007632B/en unknown
-
2022
- 2022-12-09 US US18/063,817 patent/US12460205B2/en active Active
-
2023
- 2023-02-24 JP JP2023026853A patent/JP2023075164A/ja active Pending
-
2024
- 2024-03-06 AU AU2024201464A patent/AU2024201464A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020501582A5 (enExample) | ||
| JP2021500864A5 (enExample) | ||
| US20240141336A1 (en) | Targeted rna editing | |
| JP2023029966A5 (enExample) | ||
| US12203072B2 (en) | Chemically modified single-stranded rna-editing oligonucleotides | |
| Crooke et al. | Antisense technology: an overview and prospectus | |
| US20250025580A1 (en) | Compositions and Methods for Treatment of Diseases Associated with Trinucleotide Repeats in Transcription Factor Four | |
| Keeler et al. | Gene therapy 2017: progress and future directions | |
| EP3159407A1 (en) | Guide rnas, methods and uses | |
| US10174320B2 (en) | Downregulating miR-132 for the treatment of lipid related disorders | |
| US20220259597A1 (en) | Oligonucleotide antagonists for rna guided genome editing | |
| JP2017510583A (ja) | 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的ｕｎａオリゴマー | |
| Haussecker | Stacking up CRISPR against RNA i for therapeutic gene inhibition | |
| JP7385847B2 (ja) | ｓｎｏＲＮＡの発現抑制剤を有効成分とするがん増殖抑制剤 | |
| JPWO2020198697A5 (enExample) | ||
| JP2022510673A (ja) | アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする | |
| JPWO2021158858A5 (enExample) | ||
| KR102824136B1 (ko) | Pdcd-1 유전자에 상보적인 가이드 rna 및 이의 용도 | |
| US20250367128A1 (en) | Biodegradable lipidoids and compositions and methods of use thereof for liver targeted delivery | |
| Glackin et al. | Antisense oligonucleotides in the treatment of bladder cancer | |
| Baruah et al. | Macular Corneal Dystrophy: Past, Present, and Future | |
| WO2025235552A1 (en) | Systems and methods for treatment of hepatitis b virus (hbv) infection | |
| Witz et al. | CRISPR/Cas9-Mediated Knockdown Of BRCA1/2 Restores Response To Olaparib In Pancreatic Cancer Cell Lines | |
| WO2025007951A1 (zh) | 一种靶向ttr的sacas9 sgrna及其修饰方式 | |
| CN120796267A (zh) | 用于使用CRISPR/Cas系统进行基因编辑的经修饰的gRNA |