WO2025038902A3 - Ciblage intracellulaire d'oligonucléotides - Google Patents

Ciblage intracellulaire d'oligonucléotides Download PDF

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Publication number
WO2025038902A3
WO2025038902A3 PCT/US2024/042608 US2024042608W WO2025038902A3 WO 2025038902 A3 WO2025038902 A3 WO 2025038902A3 US 2024042608 W US2024042608 W US 2024042608W WO 2025038902 A3 WO2025038902 A3 WO 2025038902A3
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WO
WIPO (PCT)
Prior art keywords
oligonucleotides
intracellular targeting
peptide
component
compound
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
PCT/US2024/042608
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English (en)
Other versions
WO2025038902A2 (fr
Inventor
Patrick Dougherty
Ziqing QIAN
Mahboubeh KHEIRABADI
Matthew Streeter
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Entrada Therapeutics Inc
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Entrada Therapeutics Inc
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Filing date
Publication date
Application filed by Entrada Therapeutics Inc filed Critical Entrada Therapeutics Inc
Priority to AU2024325879A priority Critical patent/AU2024325879A1/en
Priority to CN202480054073.0A priority patent/CN121772940A/zh
Priority to IL326646A priority patent/IL326646A/en
Publication of WO2025038902A2 publication Critical patent/WO2025038902A2/fr
Publication of WO2025038902A3 publication Critical patent/WO2025038902A3/fr
Priority to MX2026001575A priority patent/MX2026001575A/es
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/02—Linear peptides containing at least one abnormal peptide link
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/64—Cyclic peptides containing only normal peptide links
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic 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
    • 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
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)

Abstract

L'invention concerne des composés comprenant un peptide de pénétration de cellule cyclique (cCPP) et un ou plusieurs lieurs comprenant, un composant d'acide aminé, un composant de polyéthylène glycol, un composant hydrophobe, ou une combinaison de ceux-ci. Dans des modes de réalisation, le composé contient un peptide exocyclique (EP). Dans des modes de réalisation, le composé ne contient pas de peptide exocyclique.
PCT/US2024/042608 2023-08-17 2024-08-16 Ciblage intracellulaire d'oligonucléotides Pending WO2025038902A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2024325879A AU2024325879A1 (en) 2023-08-17 2024-08-16 Intracellular targeting of oligonucleotides
CN202480054073.0A CN121772940A (zh) 2023-08-17 2024-08-16 寡核苷酸的细胞内靶向
IL326646A IL326646A (en) 2023-08-17 2024-08-16 Intracellular targeting of oligonucleotides
MX2026001575A MX2026001575A (es) 2023-08-17 2026-02-09 Direccionamiento intracelular de oligonucleotidos

Applications Claiming Priority (14)

Application Number Priority Date Filing Date Title
US202363520274P 2023-08-17 2023-08-17
US202363520271P 2023-08-17 2023-08-17
US63/520,271 2023-08-17
US63/520,274 2023-08-17
US202463557137P 2024-02-23 2024-02-23
US63/557,137 2024-02-23
US202463561529P 2024-03-05 2024-03-05
US202463561552P 2024-03-05 2024-03-05
US63/561,552 2024-03-05
US63/561,529 2024-03-05
US202463640478P 2024-04-30 2024-04-30
US63/640,478 2024-04-30
US202463665630P 2024-06-28 2024-06-28
US63/665,630 2024-06-28

Publications (2)

Publication Number Publication Date
WO2025038902A2 WO2025038902A2 (fr) 2025-02-20
WO2025038902A3 true WO2025038902A3 (fr) 2025-03-20

Family

ID=92762201

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/042608 Pending WO2025038902A2 (fr) 2023-08-17 2024-08-16 Ciblage intracellulaire d'oligonucléotides

Country Status (5)

Country Link
CN (1) CN121772940A (fr)
AU (1) AU2024325879A1 (fr)
IL (1) IL326646A (fr)
MX (1) MX2026001575A (fr)
WO (1) WO2025038902A2 (fr)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022240757A1 (fr) * 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Constructions de liaison à l'antigène et de dégradation d'antigène
WO2022240758A1 (fr) * 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions et méthodes pour moduler l'expression génique
WO2023081893A1 (fr) * 2021-11-08 2023-05-11 Entrada Therapeutics, Inc. Ciblage intracellulaire d'oligonucléotides

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
JP2002535247A (ja) 1998-11-26 2002-10-22 ペンタファルム アクチェンゲゼルシャフト 輸送系複合体
US7034109B2 (en) 2000-10-13 2006-04-25 Christophe Bonny Intracellular delivery of biological effectors
US7033597B2 (en) 2000-10-13 2006-04-25 Université de Lausanne Intracellular delivery of biological effectors
JP6807831B2 (ja) 2014-05-21 2021-01-06 エントラーダ セラピューティクス，インコーポレイテッド 細胞膜透過性ペプチド、並びにこの作製方法及び使用方法
US10815276B2 (en) 2014-05-21 2020-10-27 Entrada Therapeutics, Inc. Cell penetrating peptides and methods of making and using thereof
EP4058032A4 (fr) 2019-12-19 2024-01-10 Entrada Therapeutics, Inc. Compositions pour administrer des composés antisens
EP4314016A1 (fr) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Peptides de pénétration cellulaire cyclique
AU2022271873A1 (en) 2021-05-10 2024-01-04 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
US20230312653A1 (en) 2022-03-31 2023-10-05 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
CN119156395A (zh) 2022-04-22 2024-12-17 恩特拉达治疗学股份有限公司 用于递送治疗剂的环肽

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022240757A1 (fr) * 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Constructions de liaison à l'antigène et de dégradation d'antigène
WO2022240758A1 (fr) * 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions et méthodes pour moduler l'expression génique
WO2023081893A1 (fr) * 2021-11-08 2023-05-11 Entrada Therapeutics, Inc. Ciblage intracellulaire d'oligonucléotides

Also Published As

Publication number Publication date
IL326646A (en) 2026-04-01
CN121772940A (zh) 2026-03-31
AU2024325879A1 (en) 2026-03-12
MX2026001575A (es) 2026-04-01
WO2025038902A2 (fr) 2025-02-20

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