JP6993966B2 - ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 - Google Patents

ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 Download PDF

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JP6993966B2
JP6993966B2 JP2018520385A JP2018520385A JP6993966B2 JP 6993966 B2 JP6993966 B2 JP 6993966B2 JP 2018520385 A JP2018520385 A JP 2018520385A JP 2018520385 A JP2018520385 A JP 2018520385A JP 6993966 B2 JP6993966 B2 JP 6993966B2
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dna
gene editing
rna
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ピー. ピーター ゴローチャン,
ジバン ナンデオ イェウレ,
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ポセイダ セラピューティクス, インコーポレイテッド
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/5005Wall or coating material
    • A61K9/5021Organic macromolecular compounds
    • A61K9/5031Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poly(lactide-co-glycolide)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0041Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1273Polymersomes; Liposomes with polymerisable or polymerised bilayer-forming substances
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
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  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Dispersion Chemistry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Polyesters Or Polycarbonates (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2018520385A 2015-07-02 2016-07-01 ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 Active JP6993966B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562187942P 2015-07-02 2015-07-02
US62/187,942 2015-07-02
US201662322346P 2016-04-14 2016-04-14
US62/322,346 2016-04-14
US15/199,021 US20170000743A1 (en) 2015-07-02 2016-06-30 Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
US15/199,021 2016-06-30
PCT/US2016/040673 WO2017004509A1 (en) 2015-07-02 2016-07-01 Compositions and methods for delivery of gene editing tools using polymeric vesicles

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JP2019105213A Division JP2019165745A (ja) 2015-07-02 2019-06-05 ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法

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JP2018532408A JP2018532408A (ja) 2018-11-08
JP2018532408A5 JP2018532408A5 (https=) 2019-07-11
JP6993966B2 true JP6993966B2 (ja) 2022-02-04

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JP2019105213A Pending JP2019165745A (ja) 2015-07-02 2019-06-05 ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法

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US (1) US20170000743A1 (https=)
EP (1) EP3317414A4 (https=)
JP (2) JP6993966B2 (https=)
CN (1) CN108699563A (https=)
AU (1) AU2016288237B2 (https=)
CA (1) CA2991109A1 (https=)
HK (1) HK1255209A1 (https=)
WO (1) WO2017004509A1 (https=)

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CA3036926C (en) 2016-09-30 2022-05-31 Poseida Therapeutics, Inc. Modified stem cell memory t cells, methods of making and methods of using same
US20190119636A1 (en) 2017-10-23 2019-04-25 Poseida Therapeutics, Inc. Modified stem cell memory t cells, methods of making and methods of using same
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JP2020523383A (ja) 2017-06-14 2020-08-06 ダナ−ファーバー キャンサー インスティテュート,インコーポレイテッド B細胞成熟抗原(bcma)指向性ナノ粒子
WO2018232356A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
US10329543B2 (en) 2017-10-23 2019-06-25 Poseida Therapeutics, Inc. Modified stem cell memory T cells, methods of making and methods of using same
JP7101419B2 (ja) 2017-10-27 2022-07-15 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 内因性t細胞受容体の標的置換
CA3124103A1 (en) 2018-12-20 2020-06-25 Poseida Therapeutics, Inc. Nanotransposon compositions and methods of use
US20220372105A1 (en) 2019-09-05 2022-11-24 Poseida Therapeutics, Inc. Allogeneic cell compositions and methods of use
AU2020407235A1 (en) 2019-12-20 2022-07-07 Poseida Therapeutics, Inc. Anti-MUC1 compositions and methods of use
WO2021183795A1 (en) 2020-03-11 2021-09-16 Poseida Therapeutics, Inc. Chimeric stimulatory receptors and methods of use in t cell activation and differentiation
GB202004254D0 (en) 2020-03-24 2020-05-06 Puridify Ltd Characterization of gene therapy vectors
EP4298205A1 (en) 2021-02-23 2024-01-03 Poseida Therapeutics, Inc. Genetically modified induced pluripotent stem cells and methods of use thereof
CN117355607A (zh) * 2021-04-13 2024-01-05 阿森纳生物科学公司 非病毒同源性介导的末端连接
EP4413141A1 (en) 2021-10-04 2024-08-14 Poseida Therapeutics, Inc. Transposon compositions and methods of use thereof
JP2024537991A (ja) 2021-10-14 2024-10-18 アーセナル バイオサイエンシズ インコーポレイテッド 共発現されるshRNAと論理ゲートシステムとを有する免疫細胞
EP4482967A1 (en) 2022-02-23 2025-01-01 Poseida Therapeutics, Inc. Genetically modified cells and methods of use thereof
WO2023193665A1 (zh) * 2022-04-07 2023-10-12 威海纽兰生物科技有限公司 基于细胞外囊泡酯酶响应药物递送载体及其制备方法和用途
EP4646189A1 (en) * 2023-01-06 2025-11-12 Vianautis Bio Limited Polymersomes for delivery of nucleic acid cargoes
EP4669746A1 (en) 2023-02-21 2025-12-31 Poseida Therapeutics, Inc. COMPOSITIONS AND GENOMIC EDITING PROCESSES
KR20250166122A (ko) 2023-02-21 2025-11-27 포세이다 테라퓨틱스, 인크. 게놈 편집을 위한 조성물 및 방법
CN121335979A (zh) 2023-05-17 2026-01-13 波西达治疗公司 靶向hbg1和hbg2的组合物及其使用方法
WO2025096980A1 (en) 2023-11-02 2025-05-08 Poseida Therapeutics, Inc. Compositions targeting klkb1 and methods of use thereof
WO2025221789A1 (en) 2024-04-16 2025-10-23 Poseida Therapeutics, Inc. Compositions and methods for use in car/tcr cell therapies
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EP3317414A4 (en) 2019-03-13
EP3317414A1 (en) 2018-05-09
CN108699563A (zh) 2018-10-23
CA2991109A1 (en) 2017-01-05
AU2016288237A1 (en) 2018-01-25
WO2017004509A1 (en) 2017-01-05
JP2019165745A (ja) 2019-10-03
US20170000743A1 (en) 2017-01-05
JP2018532408A (ja) 2018-11-08
HK1255209A1 (zh) 2019-08-09
AU2016288237B2 (en) 2022-04-14

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