CA3149700A1 - Conjugate and uses thereof - Google Patents

Conjugate and uses thereof Download PDF

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Publication number
CA3149700A1
CA3149700A1 CA3149700A CA3149700A CA3149700A1 CA 3149700 A1 CA3149700 A1 CA 3149700A1 CA 3149700 A CA3149700 A CA 3149700A CA 3149700 A CA3149700 A CA 3149700A CA 3149700 A1 CA3149700 A1 CA 3149700A1
Authority
CA
Canada
Prior art keywords
seq
suitably
peptide
conjugate
cationic
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
CA3149700A
Other languages
English (en)
French (fr)
Inventor
Matthew Wood
Miguel VARELA
Ashling HOLLAND
Richard Raz
Denis Furling
Arnaud KLEIN
Michael Gait
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.)
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Sorbonne Universite
Oxford University Innovation Ltd
United Kingdom Research and Innovation
Original Assignee
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Sorbonne Universite
Oxford University Innovation Ltd
United Kingdom Research and Innovation
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 Institut National de la Sante et de la Recherche Medicale INSERM, Association Institut de Myologie, Sorbonne Universite, Oxford University Innovation Ltd, United Kingdom Research and Innovation filed Critical Institut National de la Sante et de la Recherche Medicale INSERM
Publication of CA3149700A1 publication Critical patent/CA3149700A1/en
Pending legal-status Critical Current

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Classifications

    • 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
    • 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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Genetics & Genomics (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3149700A 2019-08-09 2020-08-07 Conjugate and uses thereof Pending CA3149700A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1911403.2 2019-08-09
GBGB1911403.2A GB201911403D0 (en) 2019-08-09 2019-08-09 Conjugate and uses thereof
PCT/GB2020/051891 WO2021028666A1 (en) 2019-08-09 2020-08-07 Conjugate and uses thereof

Publications (1)

Publication Number Publication Date
CA3149700A1 true CA3149700A1 (en) 2021-02-18

Family

ID=67990936

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3149700A Pending CA3149700A1 (en) 2019-08-09 2020-08-07 Conjugate and uses thereof

Country Status (13)

Country Link
US (1) US20220275372A1 (https=)
EP (1) EP4010030A1 (https=)
JP (1) JP7678654B2 (https=)
KR (1) KR20220079524A (https=)
CN (2) CN114615998B (https=)
AU (1) AU2020327659A1 (https=)
BR (1) BR112022002309A2 (https=)
CA (1) CA3149700A1 (https=)
GB (1) GB201911403D0 (https=)
IL (1) IL290424A (https=)
MX (1) MX2022001712A (https=)
SA (1) SA522431627B1 (https=)
WO (1) WO2021028666A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200131231A1 (en) 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
US12465646B2 (en) 2018-12-07 2025-11-11 Oxford University Innovation Limited Linkers
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
EP4304657A4 (en) * 2021-03-12 2026-04-01 Pepgen Inc METHODS OF TREATMENT OF TYPE 1 MYOTONIC DYSTROPHY USING PEPTIDE-OLIGONUCLEOTIDE CONJUGATES
KR20250116673A (ko) * 2022-11-21 2025-08-01 펩젠 인크. 세포-침투 펩티드, 그의 접합체 및 그의 사용 방법
WO2025033255A1 (ja) * 2023-08-07 2025-02-13 東亞合成株式会社 ペプチドフラグメント及びその利用
WO2025033256A1 (ja) * 2023-08-07 2025-02-13 東亞合成株式会社 ペプチドフラグメント及びその利用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2049664T1 (sl) * 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
WO2009005793A2 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009147368A1 (en) * 2008-06-04 2009-12-10 Medical Research Council Peptides
US20110269665A1 (en) * 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CN103998458B (zh) * 2011-08-30 2018-10-09 医学研究理事会 具有中央疏水域的细胞穿透肽
EP2900821B1 (en) * 2012-09-25 2020-04-01 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения

Also Published As

Publication number Publication date
CN114615998A (zh) 2022-06-10
US20220275372A1 (en) 2022-09-01
JP2022543320A (ja) 2022-10-11
JP7678654B2 (ja) 2025-05-16
KR20220079524A (ko) 2022-06-13
AU2020327659A1 (en) 2022-02-24
EP4010030A1 (en) 2022-06-15
CN114615998B (zh) 2025-05-23
SA522431627B1 (ar) 2024-05-19
GB201911403D0 (en) 2019-09-25
BR112022002309A2 (pt) 2022-04-26
CN120837670A (zh) 2025-10-28
WO2021028666A1 (en) 2021-02-18
MX2022001712A (es) 2022-05-13
IL290424A (en) 2022-04-01

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