JP2023547405A - Ｃａｓ９－ＲＮＰ及び他の核タンパク質カーゴの形質導入のために適合された最小長のシャトル剤ペプチド及びそのバリアント - Google Patents

Ｃａｓ９－ＲＮＰ及び他の核タンパク質カーゴの形質導入のために適合された最小長のシャトル剤ペプチド及びそのバリアント Download PDF

Info

Publication number
JP2023547405A
JP2023547405A JP2023524767A JP2023524767A JP2023547405A JP 2023547405 A JP2023547405 A JP 2023547405A JP 2023524767 A JP2023524767 A JP 2023524767A JP 2023524767 A JP2023524767 A JP 2023524767A JP 2023547405 A JP2023547405 A JP 2023547405A
Authority
JP
Japan
Prior art keywords
shuttle agent
peptide
cargo
residues
shuttle
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2023524767A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023547405A5 (https=
Inventor
ディヴィッド・グアイ
ステファニー・アレ
グザヴィエ・バーボー
トマ・デルジュディーチェ
ジャン－パスカル・レペティット－ストファエス
Original Assignee
フェルダン・バイオ・インコーポレーテッド
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 フェルダン・バイオ・インコーポレーテッド filed Critical フェルダン・バイオ・インコーポレーテッド
Publication of JP2023547405A publication Critical patent/JP2023547405A/ja
Publication of JP2023547405A5 publication Critical patent/JP2023547405A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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]
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/146—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic macromolecular compounds
    • 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
    • C07K14/4723—Cationic antimicrobial peptides, e.g. defensins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Inorganic Chemistry (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2023524767A 2020-10-22 2021-10-22 Ｃａｓ９－ＲＮＰ及び他の核タンパク質カーゴの形質導入のために適合された最小長のシャトル剤ペプチド及びそのバリアント Pending JP2023547405A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063104340P 2020-10-22 2020-10-22
US63/104,340 2020-10-22
PCT/CA2021/051490 WO2022082315A1 (en) 2020-10-22 2021-10-22 Shuttle agent peptides of minimal lengh and variants thereof adapted for transduction of cas9-rnp and other nucleoprotein cargos

Publications (2)

Publication Number Publication Date
JP2023547405A true JP2023547405A (ja) 2023-11-10
JP2023547405A5 JP2023547405A5 (https=) 2024-10-31

Family

ID=81291096

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023524767A Pending JP2023547405A (ja) 2020-10-22 2021-10-22 Ｃａｓ９－ＲＮＰ及び他の核タンパク質カーゴの形質導入のために適合された最小長のシャトル剤ペプチド及びそのバリアント

Country Status (9)

Country Link
US (1) US20230399661A1 (https=)
EP (1) EP4232590A4 (https=)
JP (1) JP2023547405A (https=)
KR (1) KR20230088828A (https=)
CN (1) CN116615227A (https=)
AU (1) AU2021366976A1 (https=)
CA (1) CA3196066A1 (https=)
IL (1) IL302227A (https=)
WO (1) WO2022082315A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110291100B (zh) 2016-10-12 2024-06-11 费尔丹生物公司 用于将多肽负荷从细胞外空间递送至靶真核细胞的胞质溶胶和/或细胞核的合理设计的合成肽穿梭剂，其用途、与其相关的方法和试剂盒
CA3040645A1 (en) 2019-04-18 2020-10-18 Feldan Bio Inc. Peptide-based non-proteinaceous cargo delivery
WO2024206311A1 (en) * 2023-03-31 2024-10-03 The Trustees Of The University Of Pennsylvania Hybrid antimicrobial peptides

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018512891A (ja) * 2015-04-10 2018-05-24 フェルダン・バイオ・インコーポレーテッド 標的真核細胞のサイトゾルへのポリペプチドカーゴの形質導入効率を改善するためのポリペプチドベースのシャトル剤、その使用、それと関連する方法及びキット
JP2019531316A (ja) * 2016-10-12 2019-10-31 フェルダン・バイオ・インコーポレーテッド ポリペプチドカーゴを標的真核細胞の細胞外間隙からサイトゾルおよび／または核に送達するための合理的に設計された合成ペプチドシャトル剤、その使用、それに関連する方法およびキット
WO2020210916A1 (en) * 2019-04-18 2020-10-22 Feldan Bio Inc. Peptide-based non-proteinaceous cargo delivery

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018512891A (ja) * 2015-04-10 2018-05-24 フェルダン・バイオ・インコーポレーテッド 標的真核細胞のサイトゾルへのポリペプチドカーゴの形質導入効率を改善するためのポリペプチドベースのシャトル剤、その使用、それと関連する方法及びキット
JP2019531316A (ja) * 2016-10-12 2019-10-31 フェルダン・バイオ・インコーポレーテッド ポリペプチドカーゴを標的真核細胞の細胞外間隙からサイトゾルおよび／または核に送達するための合理的に設計された合成ペプチドシャトル剤、その使用、それに関連する方法およびキット
WO2020210916A1 (en) * 2019-04-18 2020-10-22 Feldan Bio Inc. Peptide-based non-proteinaceous cargo delivery

Also Published As

Publication number Publication date
CA3196066A1 (en) 2022-04-28
EP4232590A4 (en) 2025-03-05
IL302227A (en) 2023-06-01
EP4232590A1 (en) 2023-08-30
KR20230088828A (ko) 2023-06-20
US20230399661A1 (en) 2023-12-14
WO2022082315A1 (en) 2022-04-28
AU2021366976A1 (en) 2023-06-08
CN116615227A (zh) 2023-08-18
WO2022082315A8 (en) 2022-08-04

Similar Documents

Publication Publication Date Title
CN114008210B (zh) 基于肽的非蛋白质负荷递送
Fischer Cellular uptake mechanisms and potential therapeutic utility of peptidic cell delivery vectors: progress 2001–2006
Fowler et al. Structure–function relationships in peptoids: recent advances toward deciphering the structural requirements for biological function
CA2400410C (en) Antimicrobial compounds and formulations
JP2022529651A5 (https=)
JP2023547405A (ja) Ｃａｓ９－ＲＮＰ及び他の核タンパク質カーゴの形質導入のために適合された最小長のシャトル剤ペプチド及びそのバリアント
JP7846169B2 (ja) 細胞透過性ペプチド
Galdiero et al. Intracellular delivery: exploiting viral membranotropic peptides
US20230383291A1 (en) Peptide-based transduction of non-anionic polynucleotide analogs for gene expression modulation
CN104650211A (zh) 一种多肽、多肽衍生物、多肽的可药用盐、药物组合物及其应用
CN104592380A (zh) 一种多肽、多肽衍生物、多肽的可药用盐、药物组合物及其应用
Futaki Design and Creation of Functional Membrane-Interacting Peptides
JP4686780B2 (ja) 細胞死抑制活性強化タンパク質ｆｎｋを用いた脱毛の防止
CN116615205A (zh) 用于基因表达调节的非阴离子多核苷酸类似物的基于肽的转导
WO2004094462A2 (en) Methods of peptide preparation
CN104045702A (zh) 一种多肽、多肽衍生物、多肽的可药用盐及药物组合物
WO2026085280A1 (en) Cell-penetrating peptides with enhanced solubility
CN104045699A (zh) 一种多肽、多肽衍生物、多肽的可药用盐及药物组合物
CN104045700A (zh) 一种多肽、多肽衍生物、多肽的可药用盐及药物组合物

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241022

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241022

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250924

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251222

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260324

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20260324