GB2631171A - Compositions and methods for efficient in vivo delivery - Google Patents

Compositions and methods for efficient in vivo delivery Download PDF

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Publication number
GB2631171A
GB2631171A GB2409605.9A GB202409605A GB2631171A GB 2631171 A GB2631171 A GB 2631171A GB 202409605 A GB202409605 A GB 202409605A GB 2631171 A GB2631171 A GB 2631171A
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United Kingdom
Prior art keywords
lipid
protein
ness
containing particle
optionally
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Pending
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GB2409605.9A
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GB202409605D0 (en
Inventor
R Liu David
J Cahill Thomas III
Desouza Philip
Raguram Aditya
Banskota Samagya
An Meirui
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Broad Institute Inc
Harvard University
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Broad Institute Inc
Harvard University
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Publication of GB202409605D0 publication Critical patent/GB202409605D0/en
Publication of GB2631171A publication Critical patent/GB2631171A/en
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C07K14/4713—Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens
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    • 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
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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
    • C12N15/11—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
    • C12N15/1137—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 against enzymes
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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
    • C12N15/1138—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 against receptors or cell surface proteins
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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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]
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C12Y201/01037—DNA (cytosine-5-)-methyltransferase (2.1.1.37)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/033—Fusion polypeptide containing a localisation/targetting motif containing a motif for targeting to the internal surface of the plasma membrane, e.g. containing a myristoylation motif
    • 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
    • C07K2319/095—Fusion polypeptide containing a localisation/targetting motif containing a nuclear export signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/735—Fusion polypeptide containing domain for protein-protein interaction containing a domain for self-assembly, e.g. a viral coat protein (includes phage display)
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    • 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
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13023—Virus like particles [VLP]
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13045—Special targeting system for viral vectors

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  • Diabetes (AREA)
  • Hematology (AREA)
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  • Rehabilitation Therapy (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
GB2409605.9A 2021-12-03 2022-12-02 Compositions and methods for efficient in vivo delivery Pending GB2631171A (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202163285995P 2021-12-03 2021-12-03
US202263298626P 2022-01-11 2022-01-11
US202263298621P 2022-01-11 2022-01-11
US202263298611P 2022-01-11 2022-01-11
US202263423372P 2022-11-07 2022-11-07
PCT/US2022/080856 WO2023102550A2 (en) 2021-12-03 2022-12-02 Compositions and methods for efficient in vivo delivery

Publications (2)

Publication Number Publication Date
GB202409605D0 GB202409605D0 (en) 2024-08-14
GB2631171A true GB2631171A (en) 2024-12-25

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GB2409605.9A Pending GB2631171A (en) 2021-12-03 2022-12-02 Compositions and methods for efficient in vivo delivery

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US (3) US20250223584A2 (enExample)
EP (1) EP4441074A2 (enExample)
JP (1) JP2024544013A (enExample)
KR (1) KR20240130158A (enExample)
AU (1) AU2022402249A1 (enExample)
CA (1) CA3239381A1 (enExample)
GB (1) GB2631171A (enExample)
IL (1) IL313161A (enExample)
WO (1) WO2023102550A2 (enExample)

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CN114258398A (zh) 2019-06-13 2022-03-29 总医院公司 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法
CN116670154A (zh) 2020-07-24 2023-08-29 总医院公司 增强的病毒样颗粒及使用其递送至细胞的方法
US12565647B2 (en) 2021-05-28 2026-03-03 The Regents Of The University Of California Compositions and methods for targeted delivery of CRISPR-Cas effector polypeptides and transgenes
CN120303407A (zh) * 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
WO2024044557A1 (en) * 2022-08-23 2024-02-29 The Regents Of The University Of California Compositions and methods for targeted delivery of crispr-cas effector polypeptides
WO2024254544A2 (en) * 2023-06-07 2024-12-12 Nvelop Therapeutics, Inc. Compositions and methods for promoting payload delivery in cells
CN121646640A (zh) * 2023-06-16 2026-03-10 胡桃夹子治疗公司 纳米颗粒递送媒介物的比较评估
CN116814566A (zh) * 2023-06-27 2023-09-29 上海交通大学 树突状细胞靶向的类病毒体dvlp及其制备方法和应用
KR20260032590A (ko) * 2023-06-29 2026-03-09 세퀴러스 인코포레이티드 재조합 바이러스 유사 입자
CN117187305B (zh) * 2023-07-26 2024-09-24 山东省农业科学院 斑翅果蝇常染色体基因移码突变纯合品系的构建方法
CN121794285A (zh) * 2023-09-09 2026-04-03 正基基因科技有限公司 工程化蛋白质递送载体
WO2025096936A2 (en) 2023-11-03 2025-05-08 The Broad Institute, Inc. Use of prime editing in correcting mutations in cdkl5
CN117717632B (zh) * 2023-12-13 2024-11-22 深圳市眼科医院(深圳市眼病防治研究所) 利用增强的先导编辑阻断异常血管生成的药剂及制备方法
EP4600357A1 (en) * 2024-02-12 2025-08-13 Technische Universität München Delivery system for gene editing tools
WO2025257403A1 (en) * 2024-06-14 2025-12-18 Dotbio Pte. Ltd. Virus-like particles
CN118497196B (zh) * 2024-07-18 2024-11-01 南方医科大学珠江医院 用于递送siRNA的框架核酸机器及其制备方法与应用
WO2026024856A2 (en) 2024-07-23 2026-01-29 The Broad Institute, Inc. Compositions and methods for production of double-stranded dna in cells for programmable gene integration
WO2026083093A1 (en) * 2024-10-17 2026-04-23 Cambridge Enterprise Limited Virus particles
CN120555402B (zh) * 2025-07-30 2025-10-17 西湖凝聚体(杭州)生物科技有限公司 一种在原代细胞中进行基因编辑的方法

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