JP2022502055A - 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 - Google Patents
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 Download PDFInfo
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- JP2022502055A JP2022502055A JP2021517350A JP2021517350A JP2022502055A JP 2022502055 A JP2022502055 A JP 2022502055A JP 2021517350 A JP2021517350 A JP 2021517350A JP 2021517350 A JP2021517350 A JP 2021517350A JP 2022502055 A JP2022502055 A JP 2022502055A
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
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- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0006—Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
- C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
- C12Y101/01027—L-Lactate dehydrogenase (1.1.1.27)
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- Chemical & Material Sciences (AREA)
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- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
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- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
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- Plant Pathology (AREA)
- Public Health (AREA)
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- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Urology & Nephrology (AREA)
- Mycology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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 true JP2022502055A (ja) | 2022-01-11 |
| JPWO2020069296A5 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 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150376587A1 (en) * | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| WO2017192437A1 (en) * | 2016-05-02 | 2017-11-09 | The Regents Of The University Of California | Mammalian cells devoid of lactate dehydrogenase activity |
Family Cites Families (14)
| 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 |
| 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 |
| 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 |
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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
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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
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150376587A1 (en) * | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| WO2017192437A1 (en) * | 2016-05-02 | 2017-11-09 | The Regents Of The University Of California | Mammalian cells devoid of lactate dehydrogenase activity |
Non-Patent Citations (2)
| Title |
|---|
| "CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary h", NATURE COMMUNICATIONS, vol. 8, JPN6023038006, 21 December 2018 (2018-12-21), pages 1 - 9, ISSN: 0005322569 * |
| "Specific Inhibition of Hepatic Lactate Dehydrogenase Reduces Oxalate Production in Mouse Models of P", MOLECULAR THERAPY, vol. 26, no. 8, JPN6023038008, August 2018 (2018-08-01), pages 1983 - 1995, ISSN: 0005322568 * |
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|---|---|
| EP3856901A1 (en) | 2021-08-04 |
| SG11202102660RA (en) | 2021-04-29 |
| KR20210086621A (ko) | 2021-07-08 |
| US20210222173A1 (en) | 2021-07-22 |
| WO2020069296A1 (en) | 2020-04-02 |
| AU2019347517A1 (en) | 2021-05-06 |
| PH12021550686A1 (en) | 2022-02-28 |
| CN113056559A (zh) | 2021-06-29 |
| BR112021005718A2 (pt) | 2021-06-22 |
| CO2021005231A2 (es) | 2021-06-10 |
| TW202028460A (zh) | 2020-08-01 |
| CA3114425A1 (en) | 2020-04-02 |
| IL281529A (en) | 2021-05-31 |
| MX2021003457A (es) | 2021-07-16 |
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