JP2019521659A - 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 - Google Patents

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 Download PDF

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JP2019521659A
JP2019521659A JP2018561967A JP2018561967A JP2019521659A JP 2019521659 A JP2019521659 A JP 2019521659A JP 2018561967 A JP2018561967 A JP 2018561967A JP 2018561967 A JP2018561967 A JP 2018561967A JP 2019521659 A JP2019521659 A JP 2019521659A
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peptide
genome editing
cell membrane
vepep
nucleic acid
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JP2019521659A5 (cg-RX-API-DMAC7.html
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ジル ディビタ，
ジル ディビタ，
ニール デサイ，
ニール デサイ，
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アーディジェン， エルエルシー
アーディジェン， エルエルシー
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Publication of JP2019521659A publication Critical patent/JP2019521659A/ja
Publication of JP2019521659A5 publication Critical patent/JP2019521659A5/ja
Priority to JP2021184056A priority Critical patent/JP7379447B2/ja
Priority to JP2023169638A priority patent/JP2023165986A/ja
Priority to JP2025079827A priority patent/JP2025118829A/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0011—Cancer antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/421—Immunoglobulin superfamily
    • A61K40/4211—CD19 or B4
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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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
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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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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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]
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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JP2018561967A 2016-05-27 2017-05-26 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 Pending JP2019521659A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021184056A JP7379447B2 (ja) 2016-05-27 2021-11-11 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
JP2023169638A JP2023165986A (ja) 2016-05-27 2023-09-29 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
JP2025079827A JP2025118829A (ja) 2016-05-27 2025-05-12 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

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662342823P 2016-05-27 2016-05-27
US62/342,823 2016-05-27
US201662394140P 2016-09-13 2016-09-13
US62/394,140 2016-09-13
US201762477357P 2017-03-27 2017-03-27
US62/477,357 2017-03-27
PCT/US2017/034862 WO2017205846A1 (en) 2016-05-27 2017-05-26 Peptides and nanoparticles for intracellular delivery of genome-editing molecules

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JP2019521659A true JP2019521659A (ja) 2019-08-08
JP2019521659A5 JP2019521659A5 (cg-RX-API-DMAC7.html) 2020-07-02

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JP2018561967A Pending JP2019521659A (ja) 2016-05-27 2017-05-26 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
JP2021184056A Active JP7379447B2 (ja) 2016-05-27 2021-11-11 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
JP2023169638A Withdrawn JP2023165986A (ja) 2016-05-27 2023-09-29 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
JP2025079827A Pending JP2025118829A (ja) 2016-05-27 2025-05-12 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

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JP2021184056A Active JP7379447B2 (ja) 2016-05-27 2021-11-11 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
JP2023169638A Withdrawn JP2023165986A (ja) 2016-05-27 2023-09-29 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
JP2025079827A Pending JP2025118829A (ja) 2016-05-27 2025-05-12 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

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EP (2) EP3910059A1 (cg-RX-API-DMAC7.html)
JP (4) JP2019521659A (cg-RX-API-DMAC7.html)
CN (2) CN109689677B (cg-RX-API-DMAC7.html)
AU (2) AU2017268842B2 (cg-RX-API-DMAC7.html)
CA (1) CA3025523A1 (cg-RX-API-DMAC7.html)
ES (1) ES2901215T3 (cg-RX-API-DMAC7.html)
MX (2) MX388294B (cg-RX-API-DMAC7.html)
WO (1) WO2017205846A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA201807869B (cg-RX-API-DMAC7.html)

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KR20220111883A (ko) * 2021-02-03 2022-08-10 한국과학기술연구원 자연살해세포 특이적 CRISPR/Cas 시스템을 위한 융합 단백질 및 이의 용도
JP2023536436A (ja) * 2020-07-24 2023-08-25 アーディジェン， エルエルシー ウイルス感染を治療するための組成物及び方法

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CN111194351A (zh) * 2017-08-10 2020-05-22 阿迪根有限公司 用于细胞内递送病毒的肽和纳米颗粒
AU2018352221B2 (en) * 2017-10-16 2024-04-18 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of mRNA
FR3081169B1 (fr) * 2018-05-15 2020-06-19 Messenger Biopharma Substitution de la coiffe des arn messagers par deux sequences d'arn introduites a leur extremite 5'
WO2019237387A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 用于敲除人ACT35基因的gRNA序列及其应用
WO2019237384A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 用于敲除人CDw137基因的gRNA序列及其应用
CN111363013B (zh) * 2018-12-25 2021-11-19 深圳先进技术研究院 多组分纳米颗粒簇的构建方法
GB201902648D0 (en) 2019-02-27 2019-04-10 Cyca Oncosolutions Ltd Cell membrane penetrating conjugates
AU2020259449A1 (en) * 2019-04-17 2021-12-09 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of molecules
CN109880891B (zh) * 2019-04-22 2021-07-30 上海交通大学 基于核酸酶偶联pcr原理富集低丰度dna突变的检测技术体系及应用
US11473087B2 (en) * 2019-05-07 2022-10-18 Ecole Polytechnique Federale De Lausanne (Epfl) FMRP and cancer treatment
CN110638789A (zh) * 2019-10-31 2020-01-03 南开大学 一种可实现肿瘤靶向递送核糖核蛋白复合物的纳米粒子的制备方法和应用
WO2021217100A1 (en) * 2020-04-24 2021-10-28 Aadigen, Llc Compositions for treating cancer with kras mutations and uses thereof
WO2023006704A2 (en) * 2021-07-26 2023-02-02 Imperial College Innovations Limited Nanozyme-catalysed detection of nucleic acids
WO2025059215A1 (en) 2023-09-12 2025-03-20 Aadigen, Llc Methods and compositions for treating or preventing cancer
WO2025090926A1 (en) * 2023-10-26 2025-05-01 The Penn State Research Foundation Ultrasound-sensitive peptide particles with surface-bound gene editing tools for spatially resolved molecule delivery
CN119119204B (zh) * 2024-11-11 2025-03-18 南昌大学 温敏特异性多肽和基于温差特异性无损分离外泌体的方法

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