JP2020501582A5 - - Google Patents
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- 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
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- 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 (https=) | 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 (https=) |
| EP (1) | EP3559232A1 (https=) |
| JP (2) | JP7383478B2 (https=) |
| KR (1) | KR102551664B1 (https=) |
| CN (2) | CN117737062A (https=) |
| AU (2) | AU2017379073B2 (https=) |
| BR (1) | BR112019012825A2 (https=) |
| CA (1) | CA3047415A1 (https=) |
| CO (1) | CO2019007776A2 (https=) |
| EA (1) | EA201991455A1 (https=) |
| IL (1) | IL267448B2 (https=) |
| MX (2) | MX2019007594A (https=) |
| MY (1) | MY204487A (https=) |
| PH (1) | PH12019501457A1 (https=) |
| SA (1) | SA519402192B1 (https=) |
| SG (1) | SG10202106412RA (https=) |
| TW (2) | TWI851534B (https=) |
| WO (1) | WO2018119182A1 (https=) |
| ZA (1) | ZA202007632B (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20230140670A1 (en) * | 2017-09-29 | 2023-05-04 | Intellia Therapeutics, Inc. | Formulations |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| WO2020006708A1 (en) * | 2018-07-04 | 2020-01-09 | 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 |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| WO2020082047A1 (en) | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
| US20200270617A1 (en) * | 2018-10-18 | 2020-08-27 | Intellia Therapeutics, Inc. | Compositions and methods for transgene expression from an albumin locus |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| US20230416725A1 (en) * | 2020-09-15 | 2023-12-28 | Rutgers, The State University Of New Jersey | Systems for gene editing and methods of use thereof |
| CA3196831A1 (en) | 2020-09-25 | 2022-03-31 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| US20220160847A1 (en) * | 2020-11-20 | 2022-05-26 | Mark Egly | Method of preventing and/or treating a plurality of diseases |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | 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 |
| 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 |
| 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 |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| PL3360964T3 (pl) * | 2012-12-06 | 2020-03-31 | Sigma-Aldrich Co. Llc | Modyfikacja i regulacja genomu oparta na crispr |
| 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 |
| CN112301024A (zh) * | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| RU2716420C2 (ru) * | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| US9458457B2 (en) | 2013-07-03 | 2016-10-04 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA |
| CN120574876A (zh) * | 2013-08-22 | 2025-09-02 | 纳幕尔杜邦公司 | 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法 |
| CN106459995B (zh) * | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| US20150165054A1 (en) * | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| CA2935032C (en) | 2013-12-26 | 2024-01-23 | The General Hospital Corporation | Multiplex guide rnas |
| CA2969619A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| KR102888521B1 (ko) * | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| EP4144844B1 (en) * | 2015-10-12 | 2025-09-10 | DuPont US Holding, LLC | Protected dna templates for gene modification and increased homologous recombination in cells and methods of use |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| CN109715801B (zh) * | 2015-12-01 | 2022-11-01 | 克里斯普治疗股份公司 | 用于治疗α1抗胰蛋白酶缺乏的材料和方法 |
| AU2016365720B2 (en) * | 2015-12-07 | 2020-11-26 | 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 |
| LT3436077T (lt) * | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| KR20180134412A (ko) | 2016-04-22 | 2018-12-18 | 인텔리아 테라퓨틱스, 인크. | 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 |
| 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 |
| WO2020082047A1 (en) * | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
-
2017
- 2017-12-21 TW TW106145118A patent/TWI851534B/zh active
- 2017-12-21 MY MYPI2019003394A patent/MY204487A/en unknown
- 2017-12-21 AU AU2017379073A patent/AU2017379073B2/en active Active
- 2017-12-21 EA EA201991455A patent/EA201991455A1/ru unknown
- 2017-12-21 JP JP2019533577A patent/JP7383478B2/ja active Active
- 2017-12-21 MX MX2019007594A patent/MX2019007594A/es unknown
- 2017-12-21 CN CN202311710568.8A patent/CN117737062A/zh active Pending
- 2017-12-21 KR KR1020197020995A patent/KR102551664B1/ko active Active
- 2017-12-21 TW TW113125536A patent/TW202509233A/zh unknown
- 2017-12-21 CA CA3047415A patent/CA3047415A1/en active Pending
- 2017-12-21 EP EP17835903.0A patent/EP3559232A1/en active Pending
- 2017-12-21 WO PCT/US2017/067800 patent/WO2018119182A1/en not_active Ceased
- 2017-12-21 IL IL267448A patent/IL267448B2/en unknown
- 2017-12-21 BR BR112019012825A patent/BR112019012825A2/pt not_active Application Discontinuation
- 2017-12-21 CN CN201780086243.3A patent/CN110382697B/zh active Active
- 2017-12-21 SG SG10202106412RA patent/SG10202106412RA/en unknown
-
2019
- 2019-06-20 US US16/446,764 patent/US11549107B2/en active Active
- 2019-06-20 SA SA519402192A patent/SA519402192B1/ar unknown
- 2019-06-21 MX MX2025002623A patent/MX2025002623A/es unknown
- 2019-06-21 PH PH12019501457A patent/PH12019501457A1/en 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
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