JPWO2020028327A5 - - Google Patents
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- JPWO2020028327A5 JPWO2020028327A5 JP2021504770A JP2021504770A JPWO2020028327A5 JP WO2020028327 A5 JPWO2020028327 A5 JP WO2020028327A5 JP 2021504770 A JP2021504770 A JP 2021504770A JP 2021504770 A JP2021504770 A JP 2021504770A JP WO2020028327 A5 JPWO2020028327 A5 JP WO2020028327A5
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- 239000000203 mixture Substances 0.000 claims description 540
- 238000000034 method Methods 0.000 claims description 267
- 125000003729 nucleotide group Chemical group 0.000 claims description 78
- 239000002773 nucleotide Substances 0.000 claims description 69
- 230000004048 modification Effects 0.000 claims description 68
- 238000012986 modification Methods 0.000 claims description 68
- 108020005004 Guide RNA Proteins 0.000 claims description 66
- MUBZPKHOEPUJKR-UHFFFAOYSA-N Oxalic acid Chemical compound OC(=O)C(O)=O MUBZPKHOEPUJKR-UHFFFAOYSA-N 0.000 claims description 37
- 230000004568 DNA-binding Effects 0.000 claims description 34
- 239000011230 binding agent Substances 0.000 claims description 34
- 101150105486 HAO1 gene Proteins 0.000 claims description 17
- 150000002632 lipids Chemical class 0.000 claims description 16
- 101000629622 Homo sapiens Serine-pyruvate aminotransferase Proteins 0.000 claims description 14
- 102100026842 Serine-pyruvate aminotransferase Human genes 0.000 claims description 14
- 108020004707 nucleic acids Proteins 0.000 claims description 14
- 150000007523 nucleic acids Chemical class 0.000 claims description 14
- 102000039446 nucleic acids Human genes 0.000 claims description 14
- 208000000891 primary hyperoxaluria type 1 Diseases 0.000 claims description 14
- 230000005782 double-strand break Effects 0.000 claims description 11
- AEMRFAOFKBGASW-UHFFFAOYSA-M Glycolate Chemical compound OCC([O-])=O AEMRFAOFKBGASW-UHFFFAOYSA-M 0.000 claims description 9
- 210000002966 serum Anatomy 0.000 claims description 9
- 230000002485 urinary effect Effects 0.000 claims description 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 8
- 208000008852 Hyperoxaluria Diseases 0.000 claims description 8
- QXDMQSPYEZFLGF-UHFFFAOYSA-L calcium oxalate Chemical compound [Ca+2].[O-]C(=O)C([O-])=O QXDMQSPYEZFLGF-UHFFFAOYSA-L 0.000 claims description 8
- 235000006408 oxalic acid Nutrition 0.000 claims description 8
- 208000020832 chronic kidney disease Diseases 0.000 claims description 7
- 230000008021 deposition Effects 0.000 claims description 7
- 201000000523 end stage renal failure Diseases 0.000 claims description 7
- 208000006750 hematuria Diseases 0.000 claims description 7
- 238000004519 manufacturing process Methods 0.000 claims description 7
- 230000014509 gene expression Effects 0.000 claims description 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
- 230000001939 inductive effect Effects 0.000 claims description 5
- 230000005783 single-strand break Effects 0.000 claims description 5
- -1 3-((4,4-bis(octyloxy)butanoyl)oxy)-2-((((3-(diethylamino)propoxy)carbonyl)oxy)methyl)propyl Chemical group 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 4
- 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 description 4
- 210000003734 kidney Anatomy 0.000 claims description 4
- 210000004185 liver Anatomy 0.000 claims description 4
- 230000007935 neutral effect Effects 0.000 claims description 4
- 101000991410 Homo sapiens Nucleolar and spindle-associated protein 1 Proteins 0.000 claims description 3
- 102100030991 Nucleolar and spindle-associated protein 1 Human genes 0.000 claims description 3
- 210000000056 organ Anatomy 0.000 claims description 3
- 230000009885 systemic effect Effects 0.000 claims description 3
- 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 description 2
- CHTXXFZHKGGQGX-UHFFFAOYSA-N [2-[3-(diethylamino)propoxycarbonyloxymethyl]-3-(4,4-dioctoxybutanoyloxy)propyl] (9Z,12Z)-octadeca-9,12-dienoate Chemical compound C(CCCCCCCC=C/CC=C/CCCCC)(=O)OCC(COC(CCC(OCCCCCCCC)OCCCCCCCC)=O)COC(=O)OCCCN(CC)CC CHTXXFZHKGGQGX-UHFFFAOYSA-N 0.000 claims description 2
- 235000012000 cholesterol Nutrition 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 239000002105 nanoparticle Substances 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims 4
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 101001031589 Homo sapiens 2-Hydroxyacid oxidase 1 Proteins 0.000 description 8
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 description 5
- 230000000295 complement effect Effects 0.000 description 4
- 102100039856 Histone H1.1 Human genes 0.000 description 3
- 101001035402 Homo sapiens Histone H1.1 Proteins 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 210000002700 urine Anatomy 0.000 description 3
- 102100038837 2-Hydroxyacid oxidase 1 Human genes 0.000 description 2
- 208000028208 end stage renal disease Diseases 0.000 description 2
- 238000011191 terminal modification Methods 0.000 description 2
- 238000002054 transplantation Methods 0.000 description 2
- 239000001149 (9Z,12Z)-octadeca-9,12-dienoate Substances 0.000 description 1
- OYHQOLUKZRVURQ-HZJYTTRNSA-M 9-cis,12-cis-Octadecadienoate Chemical compound CCCCC\C=C/C\C=C/CCCCCCCC([O-])=O OYHQOLUKZRVURQ-HZJYTTRNSA-M 0.000 description 1
- 108700024394 Exon Proteins 0.000 description 1
- 102100023917 Histone H1.10 Human genes 0.000 description 1
- 102100039855 Histone H1.2 Human genes 0.000 description 1
- 102100027368 Histone H1.3 Human genes 0.000 description 1
- 102100027369 Histone H1.4 Human genes 0.000 description 1
- 102100022653 Histone H1.5 Human genes 0.000 description 1
- 102100033558 Histone H1.8 Human genes 0.000 description 1
- 102100023920 Histone H1t Human genes 0.000 description 1
- 101000905024 Homo sapiens Histone H1.10 Proteins 0.000 description 1
- 101001035375 Homo sapiens Histone H1.2 Proteins 0.000 description 1
- 101001009450 Homo sapiens Histone H1.3 Proteins 0.000 description 1
- 101001009443 Homo sapiens Histone H1.4 Proteins 0.000 description 1
- 101000899879 Homo sapiens Histone H1.5 Proteins 0.000 description 1
- 101000872218 Homo sapiens Histone H1.8 Proteins 0.000 description 1
- 101000905044 Homo sapiens Histone H1t Proteins 0.000 description 1
- 101000897979 Homo sapiens Putative spermatid-specific linker histone H1-like protein Proteins 0.000 description 1
- 101000843236 Homo sapiens Testis-specific H1 histone Proteins 0.000 description 1
- 208000000913 Kidney Calculi Diseases 0.000 description 1
- 206010029148 Nephrolithiasis Diseases 0.000 description 1
- 102100021861 Putative spermatid-specific linker histone H1-like protein Human genes 0.000 description 1
- 102100031010 Testis-specific H1 histone Human genes 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 210000001508 eye Anatomy 0.000 description 1
- 238000009472 formulation Methods 0.000 description 1
- 210000002216 heart Anatomy 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 230000000979 retarding effect Effects 0.000 description 1
- 210000003491 skin Anatomy 0.000 description 1
- 238000003860 storage Methods 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 210000003932 urinary bladder Anatomy 0.000 description 1
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862712904P | 2018-07-31 | 2018-07-31 | |
| US62/712,904 | 2018-07-31 | ||
| US201862738936P | 2018-09-28 | 2018-09-28 | |
| US62/738,936 | 2018-09-28 | ||
| US201962834328P | 2019-04-15 | 2019-04-15 | |
| US62/834,328 | 2019-04-15 | ||
| US201962841734P | 2019-05-01 | 2019-05-01 | |
| US62/841,734 | 2019-05-01 | ||
| PCT/US2019/044080 WO2020028327A1 (en) | 2018-07-31 | 2019-07-30 | Compositions and methods for hydroxyacid oxidase 1 ( hao1) gene editing for treating primary hyperoxaluria type 1 (ph1) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021531804A JP2021531804A (ja) | 2021-11-25 |
| JPWO2020028327A5 true JPWO2020028327A5 (https=) | 2022-09-05 |
| JP2021531804A5 JP2021531804A5 (https=) | 2022-09-05 |
Family
ID=67810993
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021504770A Pending JP2021531804A (ja) | 2018-07-31 | 2019-07-30 | 原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20210163943A1 (https=) |
| EP (1) | EP3830267A1 (https=) |
| JP (1) | JP2021531804A (https=) |
| KR (1) | KR20210053888A (https=) |
| CN (1) | CN112867795A (https=) |
| AU (1) | AU2019313348A1 (https=) |
| BR (1) | BR112021001546A2 (https=) |
| CA (1) | CA3113190A1 (https=) |
| CO (1) | CO2021002691A2 (https=) |
| MX (1) | MX2021001070A (https=) |
| TW (1) | TW202020156A (https=) |
| WO (1) | WO2020028327A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220090047A1 (en) * | 2018-12-21 | 2022-03-24 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| CA3172481A1 (en) | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| CR20230305A (es) | 2020-12-11 | 2023-11-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación |
| WO2022256655A2 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| CA3222159A1 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof |
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| 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 |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| CN118086297B (zh) * | 2024-02-28 | 2025-10-03 | 复旦大学附属儿科医院 | 用于人HAO1基因编辑的sgRNA、组合物及其应用 |
| CN117925623B (zh) * | 2024-03-20 | 2024-06-07 | 北京引正基因科技有限公司 | 用于hao1基因编辑和治疗ph1的组合物 |
Family Cites Families (21)
| 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 |
| 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 |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| 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 |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| WO2015100436A1 (en) * | 2013-12-27 | 2015-07-02 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| ES3047792T3 (en) * | 2014-07-14 | 2025-12-04 | Univ California | Crispr/cas transcriptional modulation |
| 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 |
| WO2016205323A1 (en) * | 2015-06-18 | 2016-12-22 | Alnylam Pharmaceuticals, Inc. | Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof |
| CN109310784B (zh) * | 2015-12-07 | 2022-08-19 | 阿克生物公司 | 用于制备和使用指导核酸的方法和组合物 |
| US11845933B2 (en) | 2016-02-03 | 2023-12-19 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide RNA and its applications |
| 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 |
| WO2018049009A2 (en) * | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| AU2017374044B2 (en) | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
-
2019
- 2019-07-30 BR BR112021001546-9A patent/BR112021001546A2/pt not_active IP Right Cessation
- 2019-07-30 AU AU2019313348A patent/AU2019313348A1/en not_active Withdrawn
- 2019-07-30 CA CA3113190A patent/CA3113190A1/en active Pending
- 2019-07-30 MX MX2021001070A patent/MX2021001070A/es unknown
- 2019-07-30 KR KR1020217005361A patent/KR20210053888A/ko not_active Withdrawn
- 2019-07-30 CN CN201980064321.9A patent/CN112867795A/zh active Pending
- 2019-07-30 EP EP19762240.0A patent/EP3830267A1/en active Pending
- 2019-07-30 TW TW108127073A patent/TW202020156A/zh unknown
- 2019-07-30 WO PCT/US2019/044080 patent/WO2020028327A1/en not_active Ceased
- 2019-07-30 JP JP2021504770A patent/JP2021531804A/ja active Pending
-
2021
- 2021-01-29 US US17/162,377 patent/US20210163943A1/en not_active Abandoned
- 2021-02-26 CO CONC2021/0002691A patent/CO2021002691A2/es unknown
-
2025
- 2025-06-23 US US19/246,289 patent/US20260043026A1/en active Pending
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