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SMT201800341T1 - Sistema di trasferimento in vivo ottimizzato con agenti endosomolitici per coniugati di acidi nucleici - Google Patents

Sistema di trasferimento in vivo ottimizzato con agenti endosomolitici per coniugati di acidi nucleici

Info

Publication number
SMT201800341T1
SMT201800341T1 SM20180341T SMT201800341T SMT201800341T1 SM T201800341 T1 SMT201800341 T1 SM T201800341T1 SM 20180341 T SM20180341 T SM 20180341T SM T201800341 T SMT201800341 T SM T201800341T SM T201800341 T1 SMT201800341 T1 SM T201800341T1
Authority
SM
San Marino
Prior art keywords
optimized
nucleic acid
delivery system
vivo delivery
acid conjugates
Prior art date
Application number
SM20180341T
Other languages
English (en)
Inventor
Jian-Sheng Sun
Marie Dutreix
Maria Quanz-Schoeffel
Original Assignee
Onxeo
Centre Nat Rech Scient
Inst Curie
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 Onxeo, Centre Nat Rech Scient, Inst Curie filed Critical Onxeo
Publication of SMT201800341T1 publication Critical patent/SMT201800341T1/it

Links

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/4706—4-Aminoquinolines; 8-Aminoquinolines, e.g. chloroquine, primaquine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • 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
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K47/55—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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • A61K47/551—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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
    • 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
    • A61K47/554—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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Botany (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
SM20180341T 2010-06-22 2011-06-21 Sistema di trasferimento in vivo ottimizzato con agenti endosomolitici per coniugati di acidi nucleici SMT201800341T1 (it)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP10166936 2010-06-22
EP16183451.0A EP3135301B1 (en) 2010-06-22 2011-06-21 Optimized in vivo delivery system with endosomolytic agents for nucleic acid conjugates

Publications (1)

Publication Number Publication Date
SMT201800341T1 true SMT201800341T1 (it) 2018-09-13

Family

ID=42727428

Family Applications (1)

Application Number Title Priority Date Filing Date
SM20180341T SMT201800341T1 (it) 2010-06-22 2011-06-21 Sistema di trasferimento in vivo ottimizzato con agenti endosomolitici per coniugati di acidi nucleici

Country Status (21)

Country Link
US (5) US9205099B2 (it)
EP (4) EP3689381A1 (it)
JP (1) JP5860874B2 (it)
CN (2) CN103025356B (it)
AU (1) AU2011269041B2 (it)
CA (1) CA2802463C (it)
CY (1) CY1120560T1 (it)
DK (2) DK3372248T3 (it)
EA (2) EA023927B1 (it)
ES (2) ES2674412T3 (it)
HR (1) HRP20180934T1 (it)
HU (1) HUE037926T2 (it)
IL (2) IL223467A (it)
LT (1) LT3135301T (it)
PL (1) PL3135301T3 (it)
PT (1) PT3135301T (it)
RS (1) RS57439B1 (it)
SI (1) SI3135301T1 (it)
SM (1) SMT201800341T1 (it)
TR (1) TR201807294T4 (it)
WO (1) WO2011161075A1 (it)

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EP2527440A1 (en) * 2011-05-27 2012-11-28 Institut Curie Cancer treatment by combining DNA molecules mimicking double strand breaks with hyperthermia
CN103550783A (zh) * 2013-04-27 2014-02-05 中国人民解放军军事医学科学院放射与辐射医学研究所 一种核酸类药物靶向递送系统及其制备方法
LT3325623T (lt) * 2015-07-23 2019-10-25 Inst Curie Dbait molekulės ir parp inhibitorių derinio panaudojimas vėžiui gydyti
US20210052631A1 (en) * 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
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WO2017186882A1 (en) 2016-04-29 2017-11-02 Onxeo A method of predicting a response to an anti-tumor treatment by means of signal interfering dna molecules
JP7107503B2 (ja) * 2016-12-01 2022-07-27 国立大学法人埼玉大学 薬物送達系用エンドサイトーシス増強剤
EP3570814B1 (en) 2017-01-19 2023-11-29 Universiteit Gent Molecular adjuvants for enhanced cytosolic delivery of active agents
WO2018162439A1 (en) 2017-03-08 2018-09-13 Onxeo New predictive biomarker for the sensitivity to a treatment of cancer with a dbait molecule
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US20200407720A1 (en) 2018-03-13 2020-12-31 Onxeo A dbait molecule against acquired resistance in the treatment of cancer
CA3114299A1 (en) * 2018-11-01 2020-05-07 Alpha Tau Medical Ltd. Intratumoral alpha-emitter radiation and activation of cytoplasmatic sensors for intracellular pathogen
IL282838B2 (en) * 2018-12-21 2025-05-01 Valerio Therapeutics Conjugated nucleic acid molecules and their uses
SG11202109426YA (en) * 2019-03-06 2021-09-29 Generation Bio Co Non-active lipid nanoparticles with non-viral, capsid free dna
KR20210142154A (ko) 2019-03-21 2021-11-24 옹쎄오 암 치료를 위한 키나제 억제제와 조합된 dbait 분자
WO2021148581A1 (en) 2020-01-22 2021-07-29 Onxeo Novel dbait molecule and its use
TW202210633A (zh) 2020-06-05 2022-03-16 法商昂席歐公司 用於治療癌症之dbait分子與kras抑制劑的組合
TW202214857A (zh) 2020-06-19 2022-04-16 法商昂席歐公司 新型結合核酸分子及其用途
US20240294926A1 (en) 2021-12-16 2024-09-05 Valerio Therapeutics New conjugated nucleic acid molecules and their uses
EP4626477A1 (en) * 2022-12-01 2025-10-08 Yale University Stimuli-responsive traceless engineering platform for intracellular payload delivery

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EP2527440A1 (en) * 2011-05-27 2012-11-28 Institut Curie Cancer treatment by combining DNA molecules mimicking double strand breaks with hyperthermia

Also Published As

Publication number Publication date
HUE037926T2 (hu) 2018-09-28
HRP20180934T1 (hr) 2018-10-05
SI3135301T1 (en) 2018-08-31
EA023927B1 (ru) 2016-07-29
US10363317B2 (en) 2019-07-30
CN104788523A (zh) 2015-07-22
CY1120560T1 (el) 2019-07-10
IL223467A (en) 2017-09-28
US10765758B2 (en) 2020-09-08
LT3135301T (lt) 2018-07-10
US9428538B2 (en) 2016-08-30
EP3372248A1 (en) 2018-09-12
EP2585113A1 (en) 2013-05-01
CA2802463C (en) 2019-02-26
AU2011269041A1 (en) 2013-01-31
JP5860874B2 (ja) 2016-02-16
EP3135301A1 (en) 2017-03-01
EP3372248B1 (en) 2020-03-11
PT3135301T (pt) 2018-07-16
US20130131155A1 (en) 2013-05-23
EP3689381A1 (en) 2020-08-05
RS57439B1 (sr) 2018-09-28
CN103025356A (zh) 2013-04-03
ES2674412T3 (es) 2018-06-29
AU2011269041A2 (en) 2013-07-11
TR201807294T4 (tr) 2018-06-21
AU2011269041B2 (en) 2015-05-07
EA032867B1 (ru) 2019-07-31
EP3135301B1 (en) 2018-05-02
PL3135301T3 (pl) 2018-11-30
DK3372248T3 (da) 2020-04-06
US20170298089A1 (en) 2017-10-19
IL252241A0 (en) 2017-08-31
US20160339107A1 (en) 2016-11-24
US9687557B2 (en) 2017-06-27
CA2802463A1 (en) 2011-12-29
CN103025356B (zh) 2015-02-18
ES2784793T3 (es) 2020-09-30
EA201300039A1 (ru) 2013-05-30
JP2013534521A (ja) 2013-09-05
EA201690782A1 (ru) 2016-08-31
US20160046662A1 (en) 2016-02-18
WO2011161075A1 (en) 2011-12-29
US20190314511A1 (en) 2019-10-17
EA032867B9 (ru) 2020-01-22
US9205099B2 (en) 2015-12-08
DK3135301T3 (en) 2018-06-25
CN104788523B (zh) 2017-09-08

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