JP7288282B2 - 標的遺伝子改変用組成物 - Google Patents

標的遺伝子改変用組成物 Download PDF

Info

Publication number
JP7288282B2
JP7288282B2 JP2019562142A JP2019562142A JP7288282B2 JP 7288282 B2 JP7288282 B2 JP 7288282B2 JP 2019562142 A JP2019562142 A JP 2019562142A JP 2019562142 A JP2019562142 A JP 2019562142A JP 7288282 B2 JP7288282 B2 JP 7288282B2
Authority
JP
Japan
Prior art keywords
seq
rna
composition
nucleic acid
cells
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.)
Active
Application number
JP2019562142A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2019131829A5 (https=
JPWO2019131829A1 (ja
Inventor
秋津 堀田
正隆 井福
直子 藤本
久美子 岩渕
江利也 見城
幸正 蒔田
留美子 落合
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Takeda Pharmaceutical Co Ltd
Kyoto University NUC
Original Assignee
Takeda Pharmaceutical Co Ltd
Kyoto University NUC
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Takeda Pharmaceutical Co Ltd, Kyoto University NUC filed Critical Takeda Pharmaceutical Co Ltd
Publication of JPWO2019131829A1 publication Critical patent/JPWO2019131829A1/ja
Publication of JPWO2019131829A5 publication Critical patent/JPWO2019131829A5/ja
Application granted granted Critical
Publication of JP7288282B2 publication Critical patent/JP7288282B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Environmental Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
JP2019562142A 2017-12-28 2018-12-27 標的遺伝子改変用組成物 Active JP7288282B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2017254798 2017-12-28
JP2017254798 2017-12-28
PCT/JP2018/048034 WO2019131829A1 (ja) 2017-12-28 2018-12-27 標的遺伝子改変用組成物

Publications (3)

Publication Number Publication Date
JPWO2019131829A1 JPWO2019131829A1 (ja) 2021-02-04
JPWO2019131829A5 JPWO2019131829A5 (https=) 2022-01-12
JP7288282B2 true JP7288282B2 (ja) 2023-06-07

Family

ID=67067613

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019562142A Active JP7288282B2 (ja) 2017-12-28 2018-12-27 標的遺伝子改変用組成物

Country Status (9)

Country Link
US (1) US12031130B2 (https=)
EP (1) EP3733844B1 (https=)
JP (1) JP7288282B2 (https=)
KR (1) KR102799255B1 (https=)
CN (1) CN111527204B (https=)
AU (1) AU2018397951B2 (https=)
CA (1) CA3086885A1 (https=)
TW (1) TWI825057B (https=)
WO (1) WO2019131829A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
US20200331841A1 (en) 2017-12-28 2020-10-22 Takeda Pharmaceutical Company Limited Cationic lipids
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
EP3842538A4 (en) * 2018-08-10 2022-06-08 Kyoto University METHOD OF TRANSFECTION INTO CARDIOMYOCYTES USING CATIONIC LIPID
US12479791B2 (en) 2018-08-10 2025-11-25 Takeda Pharmaceutical Company Limited Cationic lipid
TW202208626A (zh) * 2020-04-24 2022-03-01 美商生命編輯公司 Rna引導核酸酶及其活性片段與變體，以及使用方法
EP4225907A4 (en) * 2020-10-12 2025-01-22 Duke University CRISPR/CAS-BASED BASE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
CN113528437B (zh) * 2021-07-07 2023-08-25 中国医学科学院血液病医院(中国医学科学院血液学研究所) 一种增强基因编辑效率的试剂盒及其应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003102150A2 (en) 2002-05-31 2003-12-11 Genteric, Inc. Multi-functional polyamines for delivery of biologically-active polynucleotides
WO2016153012A1 (ja) 2015-03-24 2016-09-29 協和発酵キリン株式会社 核酸含有脂質ナノ粒子
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7223887B2 (en) 2001-12-18 2007-05-29 The University Of British Columbia Multivalent cationic lipids and methods of using same in the production of lipid particles
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
KR101773643B1 (ko) 2010-04-28 2017-08-31 교와 핫꼬 기린 가부시키가이샤 양이온성 지질
WO2012028524A2 (en) 2010-08-30 2012-03-08 F. Hoffmann-La Roche Ag Method for producing a lipid particle, the lipid particle itself and its use
JP6108197B2 (ja) 2012-07-02 2017-04-05 日油株式会社 三級アミノ基含有脂質の製造方法
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
EP3093283B1 (en) 2014-01-09 2018-12-19 Eisai R&D Management Co., Ltd. Cationic lipid
US20170015994A1 (en) * 2014-02-24 2017-01-19 Massachusetts Institute Of Technology Methods for in vivo genome editing
CN104116643B (zh) * 2014-07-10 2016-08-24 上海应用技术学院 一种包覆曲酸双棕榈酸酯的纳米固体脂质载体及制备方法
KR101882634B1 (ko) 2014-08-07 2018-07-26 다케다 야쿠힝 고교 가부시키가이샤 양이온성 지질
CN106714845A (zh) 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
CN104873976B (zh) 2015-05-08 2016-04-20 厦门成坤生物技术有限公司 一类阳离子类脂化合物的应用
AU2016344609B2 (en) 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
TWI894123B (zh) 2017-12-27 2025-08-21 日商武田藥品工業有限公司 含核酸之脂質奈米粒子及其用途

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003102150A2 (en) 2002-05-31 2003-12-11 Genteric, Inc. Multi-functional polyamines for delivery of biologically-active polynucleotides
WO2016153012A1 (ja) 2015-03-24 2016-09-29 協和発酵キリン株式会社 核酸含有脂質ナノ粒子
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
CLINICAL CALCIUM，2017年02月28日，Vol.27, No.3，pp.391-399
Nat. Biotechno.，2016年，Vol.34, No.3，pp.328-333
Nat. Biotechnol，2017年11月13日，Vol.35, No.12，pp.1179-1187

Also Published As

Publication number Publication date
WO2019131829A1 (ja) 2019-07-04
CN111527204A (zh) 2020-08-11
AU2018397951B2 (en) 2025-06-26
US20210371854A1 (en) 2021-12-02
TW201938202A (zh) 2019-10-01
KR20200104318A (ko) 2020-09-03
EP3733844A4 (en) 2021-11-03
CN111527204B (zh) 2023-09-01
AU2018397951A1 (en) 2020-07-09
EP3733844A1 (en) 2020-11-04
TWI825057B (zh) 2023-12-11
US12031130B2 (en) 2024-07-09
CA3086885A1 (en) 2019-07-04
JPWO2019131829A1 (ja) 2021-02-04
EP3733844B1 (en) 2025-04-02
KR102799255B1 (ko) 2025-04-24

Similar Documents

Publication Publication Date Title
JP7288282B2 (ja) 標的遺伝子改変用組成物
US11993570B2 (en) Cationic lipids
CN112533893B (zh) 阳离子性脂质
CN115484932A (zh) 用于靶向rna递送的组合物和方法
IL272328B1 (en) Non-human animals containing a human ttr site and methods of use
JP2019521659A (ja) ゲノム編集分子の細胞内送達のためのペプチドおよびナノ粒子
KR20230135060A (ko) 생분해성 특징을 포함하는 나노물질
EP4536293A1 (en) Nucleobase editing system and method of using same for modifying nucleic acid sequences
JP2026505800A (ja) Ｈｓｃ特異的抗体結合脂質ナノ粒子及びその使用
EP4712948A2 (en) Gene editing systems and compositions for treatment of hemoglobinopathies and methods of using the same
WO2025080867A1 (en) Ionizable lipids for improved mrna delivery
RU2782218C2 (ru) Катионные липиды
BR112020012424B1 (pt) Composto, partícula lipídica, e, composição para introdução de ácido nucleico
WO2023085299A1 (ja) カチオン性脂質

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211223

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20211223

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221129

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230126

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230214

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230412

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230425

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230518

R150 Certificate of patent or registration of utility model

Ref document number: 7288282

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150