US20190275071A1 - Particle-mediated delivery of biologics - Google Patents

Particle-mediated delivery of biologics Download PDF

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Publication number
US20190275071A1
US20190275071A1 US16/463,053 US201716463053A US2019275071A1 US 20190275071 A1 US20190275071 A1 US 20190275071A1 US 201716463053 A US201716463053 A US 201716463053A US 2019275071 A1 US2019275071 A1 US 2019275071A1
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United States
Prior art keywords
particle
mir
mrna
alginate
composition
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Abandoned
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US16/463,053
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English (en)
Inventor
Atta Behfar
Andre Terzic
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Mayo Foundation for Medical Education and Research
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Mayo Foundation for Medical Education and Research
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Priority to US16/463,053 priority Critical patent/US20190275071A1/en
Assigned to MAYO FOUNDATION FOR MEDICAL EDUCATION AND RESEARCH reassignment MAYO FOUNDATION FOR MEDICAL EDUCATION AND RESEARCH ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: BEHAR, ATTA, TERZIC, ANDRE
Publication of US20190275071A1 publication Critical patent/US20190275071A1/en
Abandoned legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Liposomes
    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/06Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/513Organic macromolecular compounds; Dendrimers
    • A61K9/5161Polysaccharides, e.g. alginate, chitosan, cellulose derivatives; Cyclodextrin
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds 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
    • 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
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
US16/463,053 2016-11-23 2017-11-22 Particle-mediated delivery of biologics Abandoned US20190275071A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US16/463,053 US20190275071A1 (en) 2016-11-23 2017-11-22 Particle-mediated delivery of biologics

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662426090P 2016-11-23 2016-11-23
US16/463,053 US20190275071A1 (en) 2016-11-23 2017-11-22 Particle-mediated delivery of biologics
PCT/US2017/063060 WO2018098312A2 (en) 2016-11-23 2017-11-22 Particle-mediated delivery of biologics

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2017/063060 A-371-Of-International WO2018098312A2 (en) 2016-11-23 2017-11-22 Particle-mediated delivery of biologics

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US17/567,482 Continuation US20220152084A1 (en) 2016-11-23 2022-01-03 Particle-mediated delivery of biologics

Publications (1)

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US20190275071A1 true US20190275071A1 (en) 2019-09-12

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Family Applications (2)

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US16/463,053 Abandoned US20190275071A1 (en) 2016-11-23 2017-11-22 Particle-mediated delivery of biologics
US17/567,482 Pending US20220152084A1 (en) 2016-11-23 2022-01-03 Particle-mediated delivery of biologics

Family Applications After (1)

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US17/567,482 Pending US20220152084A1 (en) 2016-11-23 2022-01-03 Particle-mediated delivery of biologics

Country Status (6)

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US (2) US20190275071A1 (de)
EP (2) EP3973955A3 (de)
JP (2) JP7121005B2 (de)
CA (1) CA3044703A1 (de)
ES (1) ES2912270T3 (de)
WO (1) WO2018098312A2 (de)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3796944A4 (de) * 2018-05-23 2022-04-06 Mayo Foundation for Medical Education and Research Mikroverkapselte modifizierte polynukleotidzusammensetzungen und verfahren
CN108753726B (zh) * 2018-06-11 2020-10-09 西南医科大学 一种含有ECRG4 mRNA的外泌体及其制备方法和应用
WO2021184020A1 (en) * 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Methods of treating neuroinflammation
CN114984184A (zh) * 2021-03-02 2022-09-02 上海交通大学医学院附属上海儿童医学中心 增强tbx1表达在修复心脏组织损伤中的用途

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US7608262B2 (en) 1996-02-16 2009-10-27 The Kennedy Institute Of Rheumatology Methods of preventing or treating thrombosis with tumor necrosis factor antagonists
EP2292771A3 (de) * 1997-09-19 2011-07-27 Life Technologies Corporation Sense mRNA Therapie
AU2006306228A1 (en) * 2005-10-24 2007-05-03 University Of Massachusetts Compositions and their uses for gene therapy of bone conditions
KR101095943B1 (ko) 2006-02-21 2011-12-19 밀리켈빈 테크놀로지스 엘엘씨 과분극화 방법, 시스템 및 조성물
EP2125030A2 (de) * 2007-01-29 2009-12-02 Medipol S.A. Kolloidale partikel auf chitosan-basis zur rna-abgabe
US9822364B2 (en) * 2008-03-11 2017-11-21 Yale University Compositions and methods for controlled delivery of inhibitory ribonucleic acids
US9241898B2 (en) * 2008-03-11 2016-01-26 Yale University Compositions and methods for controlled delivery of inhibitory ribonucleic acids
PT2506857T (pt) * 2009-12-01 2018-05-14 Translate Bio Inc Entrega de arnm para o acréscimo de proteínas e enzimas em doenças genéticas humanas
EP2600901B1 (de) * 2010-08-06 2019-03-27 ModernaTX, Inc. Pharmazeutische zusammensetzungen enthaltenbearbeitete nukleinsäuren und ihre medizinische verwendung
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US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
TR201910686T4 (tr) 2011-06-08 2019-08-21 Translate Bio Inc Mrna iletimine yönelik lipit nanopartikül bileşimleri ve yöntemler.
EP2791160B1 (de) 2011-12-16 2022-03-02 ModernaTX, Inc. Modifizierte mrna zusammensetzungen
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
EP4074834A1 (de) 2012-11-26 2022-10-19 ModernaTX, Inc. Am kettenende modifizierte rna
EP2929035A1 (de) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidnanopartikel zur freisetzung von mrna
ES2767050T3 (es) 2013-09-03 2020-06-16 Mayo Found Medical Education & Res Reducción del riesgo de eventos cardíacos adversos mayores
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NZ718817A (en) 2013-10-22 2020-07-31 Massachusetts Inst Technology Lipid formulations for delivery of messenger rna
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RU2718590C2 (ru) 2014-11-10 2020-04-08 Этрис Гмбх Индукция остеогенеза путем внедрения рнк, кодирующей костный морфогенетический белок (кмб)
EP3796944A4 (de) * 2018-05-23 2022-04-06 Mayo Foundation for Medical Education and Research Mikroverkapselte modifizierte polynukleotidzusammensetzungen und verfahren

Also Published As

Publication number Publication date
CA3044703A1 (en) 2018-05-31
EP3544591B1 (de) 2022-04-20
JP2020500857A (ja) 2020-01-16
EP3544591A2 (de) 2019-10-02
JP2022140640A (ja) 2022-09-26
JP7469397B2 (ja) 2024-04-16
WO2018098312A3 (en) 2018-07-05
EP3973955A3 (de) 2022-06-15
EP3544591A4 (de) 2020-10-07
WO2018098312A2 (en) 2018-05-31
US20220152084A1 (en) 2022-05-19
ES2912270T3 (es) 2022-05-25
EP3973955A2 (de) 2022-03-30
JP7121005B2 (ja) 2022-08-17

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