CA3159320A1 - Systemes d'administration de particules - Google Patents

Systemes d'administration de particules Download PDF

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Publication number
CA3159320A1
CA3159320A1 CA3159320A CA3159320A CA3159320A1 CA 3159320 A1 CA3159320 A1 CA 3159320A1 CA 3159320 A CA3159320 A CA 3159320A CA 3159320 A CA3159320 A CA 3159320A CA 3159320 A1 CA3159320 A1 CA 3159320A1
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Prior art keywords
xdp
sequence
seq
protein
components
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Pending
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CA3159320A
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English (en)
Inventor
Benjamin OAKES
Sean Higgins
Hannah SPINNER
Sarah DENNY
Brett T. STAAHL
Kian TAYLOR
Katherine BANEY
Isabel COLIN
Maroof ADIL
Matthew Gardner
Trent GOMBERG
Gayathri VIJAYAKUMAR
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Scribe Therapeutics Inc
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Scribe Therapeutics Inc
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Publication of CA3159320A1 publication Critical patent/CA3159320A1/fr
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • 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 RNAses, DNAses
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
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    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16023Virus like particles [VLP]
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    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16211Human Immunodeficiency Virus, HIV concerning HIV gagpol
    • C12N2740/16222New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Seeds, Soups, And Other Foods (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne des systèmes d'administration de particules (XDP) utiles pour l'administration de charges utiles de tout type. Dans certains modes de réalisation, un système de particules XDP ayant un tropisme pour des cellules cibles d'intérêt est utilisé pour administrer des polypeptides CRISPR/Cas (par exemple, des protéines CasX) et des acides nucléiques de guidage (gNA), pour la modification d'acides nucléiques dans des cellules cibles. L'invention concerne également des procédés de fabrication et d'utilisation d'un tel XDP pour modifier les acides nucléiques dans de telles cellules.
CA3159320A 2019-12-06 2020-12-04 Systemes d'administration de particules Pending CA3159320A1 (fr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201962944982P 2019-12-06 2019-12-06
US62/944,982 2019-12-06
US202062968915P 2020-01-31 2020-01-31
US62/968,915 2020-01-31
US202062983460P 2020-02-28 2020-02-28
US62/983,460 2020-02-28
US202063035576P 2020-06-05 2020-06-05
US63/035,576 2020-06-05
US202063120864P 2020-12-03 2020-12-03
US63/120,864 2020-12-03
PCT/US2020/063488 WO2021113772A1 (fr) 2019-12-06 2020-12-04 Systèmes d'administration de particules

Publications (1)

Publication Number Publication Date
CA3159320A1 true CA3159320A1 (fr) 2021-06-10

Family

ID=74046189

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3159320A Pending CA3159320A1 (fr) 2019-12-06 2020-12-04 Systemes d'administration de particules

Country Status (8)

Country Link
EP (1) EP4069716A1 (fr)
JP (1) JP2023504536A (fr)
KR (1) KR20220113442A (fr)
CN (1) CN115175921A (fr)
AU (1) AU2020398658A1 (fr)
CA (1) CA3159320A1 (fr)
IL (1) IL293591A (fr)
WO (1) WO2021113772A1 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020247882A1 (fr) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Systèmes casx modifiés
GB2616584A (en) * 2020-12-03 2023-09-13 Scribe Therapeutics Inc Engineered class 2 type V CRISPR systems
WO2022120089A1 (fr) 2020-12-03 2022-06-09 Scribe Therapeutics Inc. Compositions et procédés pour le ciblage de ptbp1
WO2022261149A2 (fr) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Systèmes d'administration de particules
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
WO2023212396A1 (fr) * 2022-04-29 2023-11-02 The Board Of Trustees Of The Leland Stanford Junior University Vecteurs lentiviraux de grande capacité
WO2023235818A2 (fr) 2022-06-02 2023-12-07 Scribe Therapeutics Inc. Systèmes crispr de type v, classe 2, modifiés
WO2023235888A2 (fr) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. Compositions et procédés d'appauvrissement de cpg
EP4314267A1 (fr) 2022-06-07 2024-02-07 Scribe Therapeutics Inc. Compositions et procédés pour le ciblage de pcsk9
WO2023240027A1 (fr) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Systèmes d'administration de particules
WO2023240074A1 (fr) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions et procédés pour le ciblage de pcsk9
WO2023240157A2 (fr) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions et méthodes pour le ciblage de la dmd
WO2023240162A1 (fr) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Vecteurs aav pour l'édition de gènes
CN117344061B (zh) * 2023-12-05 2024-02-27 云南省细胞质量检测评价中心有限公司 一种同时检测五种人源病毒ebv、hbv、hcv、hiv、hpv的方法、试剂盒、引物和探针及其应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE302854T1 (de) 1993-01-26 2005-09-15 Univ Pennsylvania Zusammensetzungen und verfahren zur verabreichung von genetischem material
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
WO2010075303A1 (fr) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Facteurs d'épissage avec un domaine de liaison à l'arn de protéine puf et domaine effecteur d'épissage et leurs utilisations
US9580714B2 (en) 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
JP7059179B2 (ja) * 2015-10-20 2022-04-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品
EP3523426A4 (fr) 2016-09-30 2020-01-22 The Regents of The University of California Nouvelles enzymes de modification d'acides nucléiques guidées par arn et leurs méthodes d'utilisation
JP2020518275A (ja) * 2017-05-03 2020-06-25 バイオマリン ファーマシューティカル インコーポレイテッド 造血幹細胞の形質導入のための改善されたレンチウイルス

Also Published As

Publication number Publication date
JP2023504536A (ja) 2023-02-03
EP4069716A1 (fr) 2022-10-12
AU2020398658A1 (en) 2022-07-07
IL293591A (en) 2022-08-01
CN115175921A (zh) 2022-10-11
WO2021113772A1 (fr) 2021-06-10
KR20220113442A (ko) 2022-08-12

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