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 (3) | US11549107B2 (enExample) |
| EP (1) | EP3559232A1 (enExample) |
| JP (2) | JP7383478B2 (enExample) |
| KR (1) | KR102551664B1 (enExample) |
| CN (2) | CN117737062A (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 (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7383478B2 (ja) * | 2016-12-22 | 2023-11-20 | インテリア セラピューティクス，インコーポレイテッド | 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 |
| EP3688162B1 (en) * | 2017-09-29 | 2024-03-06 | Intellia Therapeutics, Inc. | Formulations |
| WO2019217942A1 (en) | 2018-05-11 | 2019-11-14 | 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 |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| 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 |
| MX2021004276A (es) | 2018-10-18 | 2021-09-08 | Intellia Therapeutics Inc | Composiciones y metodos para tratar deficiencia de alfa-1 antitripsina. |
| BR112021007343A2 (pt) * | 2018-10-18 | 2021-08-03 | Intellia Therapeutics, Inc. | composições e métodos para expressão de transgene a partir de um lócus de albumina |
| US12600971B2 (en) | 2019-02-13 | 2026-04-14 | Beam Therapeutics Inc. | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| WO2020168135A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| US12594301B2 (en) | 2019-09-27 | 2026-04-07 | Beam Therapeutics Inc. | Compositions and methods for treatment of liquid cancers |
| WO2022060707A1 (en) * | 2020-09-15 | 2022-03-24 | Rutgers, The State University Of New Jersey | Systems for gene editing and methods of use thereof |
| AU2021347359A1 (en) | 2020-09-25 | 2023-05-18 | 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 |
| EP4259792A1 (en) | 2020-12-11 | 2023-10-18 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing involving deamination |
| AU2022216614B2 (en) | 2021-02-05 | 2026-03-05 | 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 |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| EP0760008A1 (en) | 1994-05-19 | 1997-03-05 | 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 |
| DK3363902T3 (da) * | 2012-12-06 | 2020-01-02 | Sigma Aldrich Co Llc | Crispr-baseret genommodifikation og -regulering |
| ES2887254T3 (es) * | 2013-03-08 | 2021-12-22 | Novartis Ag | Lípidos y composiciones lipídicas para el suministro de agentes activos |
| CN105408483A (zh) * | 2013-03-15 | 2016-03-16 | 通用医疗公司 | 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向 |
| JP6738728B2 (ja) * | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用 |
| CA2916499A1 (en) | 2013-07-03 | 2015-01-08 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna |
| CN106133138A (zh) * | 2013-08-22 | 2016-11-16 | 先锋国际良种公司 | 使用引导多核苷酸/cas内切核酸酶系统的基因组修饰及其使用方法 |
| CN111218447B (zh) * | 2013-11-07 | 2024-10-11 | 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 |
| US20150166984A1 (en) * | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| KR20160102056A (ko) | 2013-12-26 | 2016-08-26 | 더 제너럴 하스피탈 코포레이션 | 멀티플렉스 가이드 ｒｎａ |
| CA2969619A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| CN107787367B (zh) * | 2015-04-06 | 2021-10-26 | 里兰斯坦福初级大学理事会 | 用于crispr/cas介导的基因调控的化学修饰的引导rna |
| JP7011590B2 (ja) * | 2015-10-12 | 2022-02-10 | イー・アイ・デュポン・ドウ・ヌムール・アンド・カンパニー | 細胞内での遺伝子組換えおよび相同組換えの増加のための保護ｄｎａ鋳型および使用方法 |
| US11866727B2 (en) * | 2015-11-06 | 2024-01-09 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1A |
| WO2017093804A2 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| AU2016365720B2 (en) * | 2015-12-07 | 2020-11-26 | Arc Bio, Llc | Methods and compositions for the making and using of guide nucleic acids |
| EP3433364A1 (en) * | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| ES3030658T3 (en) * | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| JP2019515914A (ja) | 2016-04-22 | 2019-06-13 | インテリア セラピューティクス，インコーポレイテッド | 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 |
| EP3551757A1 (en) * | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| JP7383478B2 (ja) * | 2016-12-22 | 2023-11-20 | インテリア セラピューティクス，インコーポレイテッド | 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 |
| MX2021004276A (es) * | 2018-10-18 | 2021-09-08 | Intellia Therapeutics Inc | Composiciones y metodos para tratar deficiencia de alfa-1 antitripsina. |
-
2017
- 2017-12-21 JP JP2019533577A patent/JP7383478B2/ja active Active
- 2017-12-21 WO PCT/US2017/067800 patent/WO2018119182A1/en not_active Ceased
- 2017-12-21 SG SG10202106412RA patent/SG10202106412RA/en unknown
- 2017-12-21 KR KR1020197020995A patent/KR102551664B1/ko active Active
- 2017-12-21 AU AU2017379073A patent/AU2017379073B2/en active Active
- 2017-12-21 EP EP17835903.0A patent/EP3559232A1/en active Pending
- 2017-12-21 EA EA201991455A patent/EA201991455A1/ru unknown
- 2017-12-21 TW TW113125536A patent/TW202509233A/zh unknown
- 2017-12-21 MY MYPI2019003394A patent/MY204487A/en unknown
- 2017-12-21 CA CA3047415A patent/CA3047415A1/en active Pending
- 2017-12-21 TW TW106145118A patent/TWI851534B/zh active
- 2017-12-21 BR BR112019012825A patent/BR112019012825A2/pt not_active Application Discontinuation
- 2017-12-21 IL IL267448A patent/IL267448B2/en unknown
- 2017-12-21 MX MX2019007594A patent/MX2019007594A/es unknown
- 2017-12-21 CN CN202311710568.8A patent/CN117737062A/zh active Pending
- 2017-12-21 CN CN201780086243.3A patent/CN110382697B/zh active Active
-
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 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 not_active Abandoned
-
2025
- 2025-10-08 US US19/353,412 patent/US20260125677A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021500864A5 (enExample) | ||
| US20240141336A1 (en) | Targeted rna editing | |
| JP2023029966A5 (enExample) | ||
| US11851656B2 (en) | Chemically modified single-stranded RNA-editing oligonucleotides | |
| Keeler et al. | Gene therapy 2017: progress and future directions | |
| JP2022525428A5 (enExample) | ||
| US10174320B2 (en) | Downregulating miR-132 for the treatment of lipid related disorders | |
| EP3159407A1 (en) | Guide rnas, methods and uses | |
| EP3433364A1 (en) | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency | |
| AU2017254718A1 (en) | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four | |
| JP2023512758A5 (enExample) | ||
| JP7553036B2 (ja) | ｓｎｏＲＮＡの発現抑制剤を有効成分とするがん増殖抑制剤 | |
| 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 | |
| JP7762651B2 (ja) | 修飾二重鎖ドナー鋳型 | |
| Haussecker | Stacking up CRISPR against RNA i for therapeutic gene inhibition | |
| CN119546758A (zh) | 治疗、改善和/或预防多囊肾病和多囊肝病的方法 | |
| JP2022500047A5 (enExample) | ||
| WO2025007951A1 (zh) | 一种靶向ttr的sacas9 sgrna及其修饰方式 | |
| 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 | |
| Pérez et al. | MicroRNA interference | |
| JP2024529400A5 (enExample) | ||
| WO2026008077A1 (en) | Modified grna for gene editing using crispr/cas system | |
| 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 |