JPWO2020082047A5 - - Google Patents
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- JPWO2020082047A5 JPWO2020082047A5 JP2021521383A JP2021521383A JPWO2020082047A5 JP WO2020082047 A5 JPWO2020082047 A5 JP WO2020082047A5 JP 2021521383 A JP2021521383 A JP 2021521383A JP 2021521383 A JP2021521383 A JP 2021521383A JP WO2020082047 A5 JPWO2020082047 A5 JP WO2020082047A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid construct
- bidirectional
- bidirectional nucleic
- cell
- 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
Links
- 238000000034 method Methods 0.000 claims description 107
- 108020004707 nucleic acids Proteins 0.000 claims description 97
- 102000039446 nucleic acids Human genes 0.000 claims description 97
- 150000007523 nucleic acids Chemical class 0.000 claims description 97
- 230000002457 bidirectional effect Effects 0.000 claims description 72
- 108020005004 Guide RNA Proteins 0.000 claims description 66
- 210000004027 cell Anatomy 0.000 claims description 63
- 108091026890 Coding region Proteins 0.000 claims description 37
- 239000002773 nucleotide Substances 0.000 claims description 35
- 125000003729 nucleotide group Chemical group 0.000 claims description 35
- 239000013598 vector Substances 0.000 claims description 35
- 102000009027 Albumins Human genes 0.000 claims description 33
- 108010088751 Albumins Proteins 0.000 claims description 33
- 239000011230 binding agent Substances 0.000 claims description 33
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 26
- 230000004568 DNA-binding Effects 0.000 claims description 26
- 230000000295 complement effect Effects 0.000 claims description 25
- 102100022712 Alpha-1-antitrypsin Human genes 0.000 claims description 22
- 101000823116 Homo sapiens Alpha-1-antitrypsin Proteins 0.000 claims description 22
- 101710163270 Nuclease Proteins 0.000 claims description 18
- 108090000623 proteins and genes Proteins 0.000 claims description 18
- 102000004169 proteins and genes Human genes 0.000 claims description 18
- 229920001184 polypeptide Polymers 0.000 claims description 17
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 17
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 17
- 101150069374 Serpina1 gene Proteins 0.000 claims description 14
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims description 14
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 10
- 210000005229 liver cell Anatomy 0.000 claims description 10
- 108020004414 DNA Proteins 0.000 claims description 9
- 230000005782 double-strand break Effects 0.000 claims description 9
- 150000002632 lipids Chemical class 0.000 claims description 9
- 239000002105 nanoparticle Substances 0.000 claims description 9
- 239000013603 viral vector Substances 0.000 claims description 9
- 108091033409 CRISPR Proteins 0.000 claims description 8
- 230000001965 increasing effect Effects 0.000 claims description 7
- 239000013607 AAV vector Substances 0.000 claims description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 5
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 5
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 5
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 5
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 5
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 5
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 5
- 241000958487 Adeno-associated virus 3B Species 0.000 claims description 5
- 210000003494 hepatocyte Anatomy 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 210000004185 liver Anatomy 0.000 claims description 4
- 108700024394 Exon Proteins 0.000 claims description 3
- 101100268523 Homo sapiens SERPINA1 gene Proteins 0.000 claims description 3
- 238000010362 genome editing Methods 0.000 claims description 3
- 210000002381 plasma Anatomy 0.000 claims description 3
- 230000001177 retroviral effect Effects 0.000 claims description 3
- 210000002966 serum Anatomy 0.000 claims description 3
- 108700010070 Codon Usage Proteins 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 230000004075 alteration Effects 0.000 claims 1
- 239000008280 blood Substances 0.000 claims 1
- 210000004369 blood Anatomy 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 12
- 102000053602 DNA Human genes 0.000 description 6
- 230000006780 non-homologous end joining Effects 0.000 description 6
- 230000037430 deletion Effects 0.000 description 4
- 238000012217 deletion Methods 0.000 description 4
- 239000012634 fragment Substances 0.000 description 4
- 230000037431 insertion Effects 0.000 description 4
- 238000003780 insertion Methods 0.000 description 4
- 230000028327 secretion Effects 0.000 description 4
- 108020005067 RNA Splice Sites Proteins 0.000 description 3
- 108020004682 Single-Stranded DNA Proteins 0.000 description 3
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 2
- 108020004485 Nonsense Codon Proteins 0.000 description 2
- 206010036790 Productive cough Diseases 0.000 description 2
- 238000009825 accumulation Methods 0.000 description 2
- 210000001175 cerebrospinal fluid Anatomy 0.000 description 2
- 230000037433 frameshift Effects 0.000 description 2
- 238000000338 in vitro Methods 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- 230000035772 mutation Effects 0.000 description 2
- 230000037434 nonsense mutation Effects 0.000 description 2
- 210000003802 sputum Anatomy 0.000 description 2
- 208000024794 sputum Diseases 0.000 description 2
- 108020004705 Codon Proteins 0.000 description 1
- 210000004100 adrenal gland Anatomy 0.000 description 1
- 150000001413 amino acids Chemical class 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 210000004556 brain Anatomy 0.000 description 1
- 230000002496 gastric effect Effects 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 210000005265 lung cell Anatomy 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 210000000813 small intestine Anatomy 0.000 description 1
- 210000002784 stomach Anatomy 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862747522P | 2018-10-18 | 2018-10-18 | |
| US62/747,522 | 2018-10-18 | ||
| PCT/US2019/057092 WO2020082047A1 (en) | 2018-10-18 | 2019-10-18 | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022505381A JP2022505381A (ja) | 2022-01-14 |
| JP2022505381A5 JP2022505381A5 (https=) | 2022-10-27 |
| JPWO2020082047A5 true JPWO2020082047A5 (https=) | 2022-10-27 |
Family
ID=68468858
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021521383A Pending JP2022505381A (ja) | 2018-10-18 | 2019-10-18 | 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及び方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20200270618A1 (https=) |
| EP (1) | EP3867378A1 (https=) |
| JP (1) | JP2022505381A (https=) |
| KR (1) | KR20210102209A (https=) |
| CN (1) | CN113195721A (https=) |
| AU (1) | AU2019361204A1 (https=) |
| BR (1) | BR112021007289A2 (https=) |
| CA (1) | CA3116739A1 (https=) |
| CO (1) | CO2021006367A2 (https=) |
| EA (1) | EA202191067A1 (https=) |
| IL (1) | IL282237A (https=) |
| MX (1) | MX2021004276A (https=) |
| PH (1) | PH12021550842A1 (https=) |
| SG (1) | SG11202103735TA (https=) |
| TW (1) | TW202027797A (https=) |
| WO (1) | WO2020082047A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017379073B2 (en) * | 2016-12-22 | 2023-12-14 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| JP7460643B2 (ja) | 2018-10-16 | 2024-04-02 | ブルーアレル， エルエルシー | 遺伝子へのｄｎａの標的化挿入のための方法 |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
| WO2020163703A1 (en) | 2019-02-08 | 2020-08-13 | Krystal Biotech, Inc. | Compositions and methods for delivering cftr polypeptides |
| US11622547B2 (en) | 2019-06-07 | 2023-04-11 | Regeneran Pharmaceuticals, Inc. | Genetically modified mouse that expresses human albumin |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| KR102366490B1 (ko) * | 2020-10-20 | 2022-02-23 | 에스티팜 주식회사 | 5'-캡핑된 rna 합성용 올리고뉴클레오티드 |
| US20220160847A1 (en) * | 2020-11-20 | 2022-05-26 | Mark Egly | Method of preventing and/or treating a plurality of diseases |
| WO2023064918A1 (en) * | 2021-10-15 | 2023-04-20 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| EP4419111A1 (en) * | 2021-10-19 | 2024-08-28 | Precision Biosciences, Inc. | Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency |
| WO2024254519A2 (en) * | 2023-06-08 | 2024-12-12 | Mammoth Biosciences, Inc. | Compositions and methods for the modification of human genes expressed by liver cells |
| 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 (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| 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) |
| 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 |
| US6169169B1 (en) | 1994-05-19 | 2001-01-02 | Dako A/S | PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis |
| EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | 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 |
| EP2534163B1 (en) | 2010-02-09 | 2015-11-04 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| EP4357457B1 (en) | 2012-10-23 | 2024-10-16 | 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 |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| EP3608308B1 (en) | 2013-03-08 | 2021-07-21 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| 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 |
| ES2895651T3 (es) | 2013-12-19 | 2022-02-22 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| CA2969151A1 (en) | 2014-12-23 | 2016-06-30 | Syngenta Participations Ag | Methods and compositions for identifying and enriching for cells comprising site specific genomic modifications |
| EP3265559B1 (en) | 2015-03-03 | 2021-01-06 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| US11279928B2 (en) | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| CA3006618A1 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| US11845933B2 (en) | 2016-02-03 | 2023-12-19 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide RNA and its applications |
| WO2017165862A1 (en) * | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| 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 |
| AU2017374044B2 (en) | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
| AU2017379073B2 (en) | 2016-12-22 | 2023-12-14 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| AR113154A1 (es) | 2017-09-29 | 2020-01-29 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para edición del genoma |
| JP7460643B2 (ja) * | 2018-10-16 | 2024-04-02 | ブルーアレル， エルエルシー | 遺伝子へのｄｎａの標的化挿入のための方法 |
-
2019
- 2019-10-18 AU AU2019361204A patent/AU2019361204A1/en active Pending
- 2019-10-18 CN CN201980082770.6A patent/CN113195721A/zh active Pending
- 2019-10-18 BR BR112021007289-6A patent/BR112021007289A2/pt unknown
- 2019-10-18 JP JP2021521383A patent/JP2022505381A/ja active Pending
- 2019-10-18 SG SG11202103735TA patent/SG11202103735TA/en unknown
- 2019-10-18 MX MX2021004276A patent/MX2021004276A/es unknown
- 2019-10-18 US US16/657,967 patent/US20200270618A1/en active Pending
- 2019-10-18 EP EP19798480.0A patent/EP3867378A1/en active Pending
- 2019-10-18 WO PCT/US2019/057092 patent/WO2020082047A1/en not_active Ceased
- 2019-10-18 KR KR1020217014893A patent/KR20210102209A/ko not_active Ceased
- 2019-10-18 CA CA3116739A patent/CA3116739A1/en active Pending
- 2019-10-18 TW TW108137783A patent/TW202027797A/zh unknown
- 2019-10-18 EA EA202191067A patent/EA202191067A1/ru unknown
-
2021
- 2021-04-11 IL IL282237A patent/IL282237A/en unknown
- 2021-04-15 PH PH12021550842A patent/PH12021550842A1/en unknown
- 2021-05-18 CO CONC2021/0006367A patent/CO2021006367A2/es unknown
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