JPWO2020069296A5 - - Google Patents
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- JPWO2020069296A5 JPWO2020069296A5 JP2021517350A JP2021517350A JPWO2020069296A5 JP WO2020069296 A5 JPWO2020069296 A5 JP WO2020069296A5 JP 2021517350 A JP2021517350 A JP 2021517350A JP 2021517350 A JP2021517350 A JP 2021517350A JP WO2020069296 A5 JPWO2020069296 A5 JP WO2020069296A5
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- 239000000203 mixture Substances 0.000 claims description 285
- 238000000034 method Methods 0.000 claims description 217
- 125000003729 nucleotide group Chemical group 0.000 claims description 82
- 239000002773 nucleotide Substances 0.000 claims description 72
- 230000004048 modification Effects 0.000 claims description 71
- 238000012986 modification Methods 0.000 claims description 71
- 108020005004 Guide RNA Proteins 0.000 claims description 58
- MUBZPKHOEPUJKR-UHFFFAOYSA-N Oxalic acid Chemical compound OC(=O)C(O)=O MUBZPKHOEPUJKR-UHFFFAOYSA-N 0.000 claims description 45
- 230000004568 DNA-binding Effects 0.000 claims description 34
- 239000011230 binding agent Substances 0.000 claims description 34
- 208000008852 Hyperoxaluria Diseases 0.000 claims description 20
- 101150041530 ldha gene Proteins 0.000 claims description 17
- 150000002632 lipids Chemical class 0.000 claims description 16
- 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
- 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
- 235000006408 oxalic acid Nutrition 0.000 claims description 9
- 210000002966 serum Anatomy 0.000 claims description 9
- 230000002485 urinary effect Effects 0.000 claims description 9
- QXDMQSPYEZFLGF-UHFFFAOYSA-L calcium oxalate Chemical compound [Ca+2].[O-]C(=O)C([O-])=O QXDMQSPYEZFLGF-UHFFFAOYSA-L 0.000 claims description 8
- 210000003734 kidney Anatomy 0.000 claims description 8
- 210000004185 liver Anatomy 0.000 claims description 8
- 208000004777 Primary Hyperoxaluria Diseases 0.000 claims description 7
- 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
- 238000002054 transplantation Methods 0.000 claims description 7
- 230000005783 single-strand break 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
- -1 (3-(diethylamino)propoxy)carbonyl 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
- 230000007935 neutral effect Effects 0.000 claims description 4
- 235000013305 food Nutrition 0.000 claims description 3
- 230000000968 intestinal effect Effects 0.000 claims description 3
- 210000000056 organ Anatomy 0.000 claims description 3
- 230000009885 systemic effect Effects 0.000 claims description 3
- 210000002700 urine Anatomy 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
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims 4
- 230000002265 prevention Effects 0.000 claims 2
- 230000003247 decreasing effect Effects 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- AJAMRCUNWLZBDF-MURFETPASA-N propyl linoleate Chemical compound CCCCC\C=C/C\C=C/CCCCCCCC(=O)OCCC AJAMRCUNWLZBDF-MURFETPASA-N 0.000 claims 1
- 101100288581 Homo sapiens LDHA gene Proteins 0.000 description 6
- 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
- 230000002459 sustained effect Effects 0.000 description 3
- 108700024394 Exon Proteins 0.000 description 2
- 101001090713 Homo sapiens L-lactate dehydrogenase A chain Proteins 0.000 description 2
- 102100034671 L-lactate dehydrogenase A chain Human genes 0.000 description 2
- 208000028208 end stage renal disease Diseases 0.000 description 2
- 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
- 101000991410 Homo sapiens Nucleolar and spindle-associated protein 1 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
- 102100030991 Nucleolar and spindle-associated protein 1 Human genes 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
- 210000003491 skin Anatomy 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 210000003932 urinary bladder Anatomy 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862738956P | 2018-09-28 | 2018-09-28 | |
| US62/738,956 | 2018-09-28 | ||
| US201962834334P | 2019-04-15 | 2019-04-15 | |
| US62/834,334 | 2019-04-15 | ||
| US201962841740P | 2019-05-01 | 2019-05-01 | |
| US62/841,740 | 2019-05-01 | ||
| PCT/US2019/053423 WO2020069296A1 (en) | 2018-09-28 | 2019-09-27 | Compositions and methods for lactate dehydrogenase (ldha) gene editing |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022502055A JP2022502055A (ja) | 2022-01-11 |
| JPWO2020069296A5 true JPWO2020069296A5 (enExample) | 2022-09-30 |
Family
ID=68296656
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021517350A Pending JP2022502055A (ja) | 2018-09-28 | 2019-09-27 | 乳酸デヒドロゲナーゼ（ｌｄｈａ）遺伝子編集のための組成物及び方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20210222173A1 (enExample) |
| EP (1) | EP3856901A1 (enExample) |
| JP (1) | JP2022502055A (enExample) |
| KR (1) | KR20210086621A (enExample) |
| CN (1) | CN113056559A (enExample) |
| AU (1) | AU2019347517A1 (enExample) |
| BR (1) | BR112021005718A2 (enExample) |
| CA (1) | CA3114425A1 (enExample) |
| CO (1) | CO2021005231A2 (enExample) |
| IL (1) | IL281529A (enExample) |
| MX (1) | MX2021003457A (enExample) |
| PH (1) | PH12021550686A1 (enExample) |
| SG (1) | SG11202102660RA (enExample) |
| TW (1) | TW202028460A (enExample) |
| WO (1) | WO2020069296A1 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3946598A1 (en) * | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
| AU2021360494A1 (en) | 2020-10-14 | 2023-05-18 | George Mason Research Foundation, Inc. | Ionizable lipids and methods of manufacture and use thereof |
| US20230034581A1 (en) | 2021-06-04 | 2023-02-02 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| CN118251491A (zh) | 2021-10-28 | 2024-06-25 | 瑞泽恩制药公司 | 用于敲除C5的CRISPR/Cas相关方法及组合物 |
| WO2023122433A1 (en) * | 2021-12-22 | 2023-06-29 | Arbor Biotechnologies, Inc. | Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha) |
| EP4473103A2 (en) | 2022-02-02 | 2024-12-11 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| CN114657181B (zh) * | 2022-04-01 | 2023-08-25 | 安徽大学 | 一种靶向H1.4的sgRNA以及H1.4基因编辑方法 |
| JP2025514304A (ja) | 2022-04-29 | 2025-05-02 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定 |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| JP2025521154A (ja) | 2022-05-31 | 2025-07-08 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法 |
| CN120659627A (zh) | 2022-07-29 | 2025-09-16 | 瑞泽恩制药公司 | 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法 |
| CN120265314A (zh) | 2022-09-28 | 2025-07-04 | 瑞泽恩制药公司 | 抗体抗性修饰受体以增强基于细胞的疗法 |
| US20240182561A1 (en) | 2022-11-04 | 2024-06-06 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
| JP2025538220A (ja) | 2022-11-14 | 2025-11-26 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | アストロサイトへの線維芽細胞増殖因子受容体３媒介送達のための組成物および方法 |
| US20250049896A1 (en) | 2023-07-28 | 2025-02-13 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency |
| WO2025029654A2 (en) | 2023-07-28 | 2025-02-06 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
| WO2025029657A2 (en) | 2023-07-28 | 2025-02-06 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease |
| WO2025049524A1 (en) | 2023-08-28 | 2025-03-06 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
| WO2025045247A1 (en) * | 2023-08-31 | 2025-03-06 | Geneditbio Limited | Nucleic acids encoding crispr-associated proteins and uses thereof |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | 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 |
| WO2025162435A1 (en) * | 2024-02-02 | 2025-08-07 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of liver disease |
| 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 |
| WO2025184567A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
| WO2025235388A1 (en) | 2024-05-06 | 2025-11-13 | Regeneron Pharmaceuticals, Inc. | Transgene genomic identification by nuclease-mediated long read sequencing |
Family Cites Families (16)
| 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 |
| EP2931898B1 (en) | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| DK3553174T3 (da) | 2012-12-17 | 2025-08-04 | Harvard College | Rna-guided modificering af humant genom |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| US20150376587A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| US10351854B2 (en) * | 2014-10-10 | 2019-07-16 | Dicerna Pharmaceuticals, Inc. | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| WO2016141224A1 (en) | 2015-03-03 | 2016-09-09 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| 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 |
| EP3452080A4 (en) * | 2016-05-02 | 2019-10-09 | The Regents of the University of California | MAMMALIAN CELLS WITHOUT LACTATE DEHYDROGENASE ACTIVITY |
| 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 |
-
2019
- 2019-09-27 CN CN201980076179.XA patent/CN113056559A/zh active Pending
- 2019-09-27 AU AU2019347517A patent/AU2019347517A1/en not_active Withdrawn
- 2019-09-27 KR KR1020217012025A patent/KR20210086621A/ko not_active Withdrawn
- 2019-09-27 MX MX2021003457A patent/MX2021003457A/es unknown
- 2019-09-27 TW TW108135340A patent/TW202028460A/zh unknown
- 2019-09-27 EP EP19790921.1A patent/EP3856901A1/en active Pending
- 2019-09-27 JP JP2021517350A patent/JP2022502055A/ja active Pending
- 2019-09-27 WO PCT/US2019/053423 patent/WO2020069296A1/en not_active Ceased
- 2019-09-27 BR BR112021005718-8A patent/BR112021005718A2/pt not_active IP Right Cessation
- 2019-09-27 CA CA3114425A patent/CA3114425A1/en active Pending
- 2019-09-27 SG SG11202102660RA patent/SG11202102660RA/en unknown
-
2021
- 2021-03-16 IL IL281529A patent/IL281529A/en unknown
- 2021-03-25 US US17/212,901 patent/US20210222173A1/en not_active Abandoned
- 2021-03-26 PH PH12021550686A patent/PH12021550686A1/en unknown
- 2021-04-23 CO CONC2021/0005231A patent/CO2021005231A2/es unknown
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