TWI904069B - 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法 - Google Patents

用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法

Info

Publication number
TWI904069B
TWI904069B TW107134336A TW107134336A TWI904069B TW I904069 B TWI904069 B TW I904069B TW 107134336 A TW107134336 A TW 107134336A TW 107134336 A TW107134336 A TW 107134336A TW I904069 B TWI904069 B TW I904069B
Authority
TW
Taiwan
Prior art keywords
seq
sequence
rna
ttr
combination
Prior art date
Application number
TW107134336A
Other languages
English (en)
Chinese (zh)
Other versions
TW201932479A (zh
Inventor
阿提 馬亨德拉 布拉科許 康裘莉亞
書布 歐德
潔西卡 林 西賽
雷納德 麥克 雷斯卡里
瓦特 史瑞普斯
Original Assignee
美商英特利亞醫療公司
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 美商英特利亞醫療公司 filed Critical 美商英特利亞醫療公司
Publication of TW201932479A publication Critical patent/TW201932479A/zh
Application granted granted Critical
Publication of TWI904069B publication Critical patent/TWI904069B/zh

Links

Classifications

    • 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
    • A61K48/0041—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 polymeric
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • C12N15/11—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
    • 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
    • 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
    • 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
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
TW107134336A 2017-09-29 2018-09-28 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法 TWI904069B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762566236P 2017-09-29 2017-09-29
US62/566,236 2017-09-29
US201862671902P 2018-05-15 2018-05-15
US62/671,902 2018-05-15

Publications (2)

Publication Number Publication Date
TW201932479A TW201932479A (zh) 2019-08-16
TWI904069B true TWI904069B (zh) 2025-11-11

Family

ID=63858214

Family Applications (1)

Application Number Title Priority Date Filing Date
TW107134336A TWI904069B (zh) 2017-09-29 2018-09-28 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法

Country Status (16)

Country Link
US (4) US20200248180A1 (https=)
EP (1) EP3688161A1 (https=)
JP (3) JP2021500864A (https=)
KR (1) KR20200058509A (https=)
CN (2) CN118530993A (https=)
AU (2) AU2018338787C1 (https=)
BR (1) BR112020005287A2 (https=)
CA (1) CA3077251A1 (https=)
CO (1) CO2020005116A2 (https=)
IL (3) IL311170B1 (https=)
MX (2) MX2020003608A (https=)
MY (1) MY203849A (https=)
PH (1) PH12020550364A1 (https=)
SG (1) SG11202002565YA (https=)
TW (1) TWI904069B (https=)
WO (1) WO2019067872A1 (https=)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
MX2021011757A (es) * 2019-03-28 2021-12-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la expresión de polipéptidos.
JP7631215B2 (ja) * 2019-03-28 2025-02-18 インテリア セラピューティクス，インコーポレイテッド Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法
AU2020248337A1 (en) * 2019-03-28 2021-11-04 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
EP4073249A1 (en) * 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
JP2023548005A (ja) * 2020-10-28 2023-11-15 ノヴォ ノルディスク アー／エス 抗トランスサイレチン抗体およびその使用方法
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
CN112795595A (zh) * 2020-12-24 2021-05-14 中山大学 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统
WO2022246266A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
KR20240038705A (ko) 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
US20250127927A1 (en) * 2021-08-23 2025-04-24 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CN116064598B (zh) * 2021-10-08 2024-03-12 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
WO2023185697A2 (en) * 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
CN118853659A (zh) * 2023-04-14 2024-10-29 深圳瑞吉生物科技有限公司 提高mRNA翻译蛋白的输出量的核酸及其应用
WO2024259135A1 (en) 2023-06-13 2024-12-19 Intellia Therapeutics, Inc. Assays for analysis of ribonucleic acid (rna) molecules
CN120225668A (zh) * 2023-10-25 2025-06-27 石药集团中奇制药技术（石家庄）有限公司 一种高效低脱靶的基因编辑工具
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015183026A1 (ko) * 2014-05-28 2015-12-03 주식회사 툴젠 불활성화된 표적 특이적 뉴클레아제를 이용한 표적 dna의 분리 방법
WO2017053431A2 (en) * 2015-09-21 2017-03-30 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
WO2017077386A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a

Family Cites Families (31)

* Cited by examiner, † Cited by third party
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
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する２′修飾オリゴヌクレオチド
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ
US6974667B2 (en) 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
US7244764B2 (en) * 2003-06-23 2007-07-17 Neurochem (International) Limited Methods and compositions for treating amyloid-related diseases
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
EP2323667A4 (en) 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
MX360460B (es) 2008-10-20 2018-11-05 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
PL2563920T3 (pl) 2010-04-29 2017-08-31 Ionis Pharmaceuticals, Inc. Modulacja ekspresji transtyretyny
EP3730618A1 (en) 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3031921B1 (en) * 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
DK3456831T3 (da) * 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
CN105683379A (zh) * 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
SI3161128T1 (sl) * 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Postopki in sestavki za ciljano genetsko spremembo in postopki uporabe
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
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
CN120842296A (zh) 2015-09-21 2025-10-28 垂林克生物技术有限公司 用于合成5’-加帽rna的组合物和方法
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
EP3429632B1 (en) * 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
LT3436077T (lt) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015183026A1 (ko) * 2014-05-28 2015-12-03 주식회사 툴젠 불활성화된 표적 특이적 뉴클레아제를 이용한 표적 dna의 분리 방법
WO2017053431A2 (en) * 2015-09-21 2017-03-30 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
WO2017077386A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
期刊 Anders, Carolin, Martin Jinek, In vitro enzymology of Cas9, Methods in enzymology, vol. 546, 2014/11/12, pages 1-20;網路文獻 David V. Morrissey, "Robust In Vivo Editing of Hepatocyte Target DNA Mediated by Lipid Nanoparticle Delivery of CRISPR/Cas9 Components", /www.intelliatx.com/, 2017/05/13, chrome-extension://efaidnbmnnnibpcajpcglclefindmkaj/https://ir.intelliatx.com/static-files/c4880575-fbdb-44a3-ad2d-1ce551cec00b *
網路文獻 David V. Morrissey, "Robust In Vivo Editing of Hepatocyte Target DNA Mediated by Lipid Nanoparticle Delivery of CRISPR/Cas9 Components", /www.intelliatx.com/, 2017/05/13, chrome-extension://efaidnbmnnnibpcajpcglclefindmkaj/https://ir.intelliatx.com/static-files/c4880575-fbdb-44a3-ad2d-1ce551cec00b

Also Published As

Publication number Publication date
AU2025202742A1 (en) 2025-05-15
JP2025163016A (ja) 2025-10-28
CA3077251A1 (en) 2019-04-04
EP3688161A1 (en) 2020-08-05
CN118530993A (zh) 2024-08-23
SG11202002565YA (en) 2020-04-29
CN111417728A (zh) 2020-07-14
JP7707140B2 (ja) 2025-07-14
CO2020005116A2 (es) 2020-05-15
IL325460A (en) 2026-02-01
AU2018338787A1 (en) 2020-04-16
AU2018338787B2 (en) 2025-05-08
KR20200058509A (ko) 2020-05-27
US20230257747A1 (en) 2023-08-17
JP2023029966A (ja) 2023-03-07
AU2018338787C1 (en) 2025-10-23
NZ762942A (en) 2024-10-25
IL273317A (en) 2020-04-30
IL311170B1 (en) 2026-02-01
US20230118592A1 (en) 2023-04-20
PH12020550364A1 (en) 2021-02-15
US11795460B2 (en) 2023-10-24
JP2021500864A (ja) 2021-01-14
MY203849A (en) 2024-07-21
WO2019067872A1 (en) 2019-04-04
MX2025014378A (es) 2026-01-07
US20250207130A1 (en) 2025-06-26
US20200248180A1 (en) 2020-08-06
US11965165B2 (en) 2024-04-23
IL311170A (en) 2024-04-01
MX2020003608A (es) 2020-09-25
BR112020005287A2 (pt) 2020-09-24
TW201932479A (zh) 2019-08-16

Similar Documents

Publication Publication Date Title
TWI904069B (zh) 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法
TWI839337B (zh) 用於基因組編輯之多核苷酸、組合物及方法
JP7631215B2 (ja) Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法
JP7636338B2 (ja) コルチコステロイドを含む、ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスを治療するための組成物および方法、またはその使用
HK40110281A (zh) 用於ttr基因编辑及治疗attr淀粉样变性的组合物及方法
HK40032213A (en) Compositions and methods for ttr gene editing and treating attr amyloidosis
HK40065742A (zh) 用於ttr基因编辑和治疗attr淀粉样变性的包括皮质类固醇的组合物和方法或其用途
HK40065872A (zh) 包括ttr向导rna和对rna引导的dna结合剂进行编码的多核苷酸的组合物和方法
EA048813B1 (ru) Композиции и способы редактирования гена ttr и лечения транстиретинового амилоидоза (attr)
HK40067192A (zh) 用於多肽表达的多核苷酸、组合物和方法