CA2708354C - Means for delivery of nucleic acids active for gene silencing using synthetic polymers - Google Patents

Means for delivery of nucleic acids active for gene silencing using synthetic polymers Download PDF

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Publication number
CA2708354C
CA2708354C CA2708354A CA2708354A CA2708354C CA 2708354 C CA2708354 C CA 2708354C CA 2708354 A CA2708354 A CA 2708354A CA 2708354 A CA2708354 A CA 2708354A CA 2708354 C CA2708354 C CA 2708354C
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CA
Canada
Prior art keywords
sirna
tyrosine
rna
tryptophan
polyethylenimine
Prior art date
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Active
Application number
CA2708354A
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English (en)
French (fr)
Other versions
CA2708354A1 (en
Inventor
Abdennaji Adib
Patrick Erbacher
Fabrice Stock
Nadia Hafdi
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Polyplus Transfection SA
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Polyplus Transfection SA
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Publication date
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Publication of CA2708354A1 publication Critical patent/CA2708354A1/en
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Publication of CA2708354C publication Critical patent/CA2708354C/en
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Classifications

    • 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
    • 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
    • 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
    • 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
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Polyamides (AREA)
CA2708354A 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers Active CA2708354C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1335807P 2007-12-13 2007-12-13
US61/013,358 2007-12-13
PCT/IB2008/055256 WO2009074970A2 (en) 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers

Publications (2)

Publication Number Publication Date
CA2708354A1 CA2708354A1 (en) 2009-06-18
CA2708354C true CA2708354C (en) 2018-12-11

Family

ID=40561891

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2708354A Active CA2708354C (en) 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers

Country Status (8)

Country Link
US (1) US20100297756A1 (enExample)
EP (1) EP2229445B1 (enExample)
JP (1) JP5802389B2 (enExample)
KR (1) KR101590652B1 (enExample)
CN (1) CN101952434B (enExample)
CA (1) CA2708354C (enExample)
IL (1) IL206310A (enExample)
WO (1) WO2009074970A2 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2552460A1 (en) 2010-03-29 2013-02-06 Universite De Strasbourg Polymers for delivering molecules of interest
KR101223483B1 (ko) * 2010-09-10 2013-01-17 한국과학기술연구원 전하결합 및 생분해성 공유결합으로 동시에 연결된 고분자―siRNA 나노입자 전달체
KR101497668B1 (ko) * 2012-09-28 2015-03-03 한양대학교 산학협력단 전신 순환을 위한 짧은 간섭 rna 유전자 전달체
WO2014051318A2 (ko) * 2012-09-28 2014-04-03 한양대학교 산학협력단 전신 순환을 위한 짧은 간섭 rna 유전자 전달체
EP4085930A1 (en) 2012-10-05 2022-11-09 BioNTech Delivery Technologies GmbH Hydroxylated polyamine derivatives as transfection reagents for rna
CN104703629B (zh) 2012-10-08 2020-12-01 生物技术传送科技有限责任公司 作为转染试剂的羧化多胺衍生物
CN103087314B (zh) * 2013-02-01 2015-02-18 同济大学 生物可降解聚氨酯基因转染试剂的制备方法
CN107805643B (zh) * 2017-09-15 2020-12-08 四川大学 靶向抑制沙门氏菌耐药外排泵基因acrA的siRNA-DNA纳米系统及其制备方法
BR112022002149A2 (pt) * 2019-08-05 2022-04-19 Polyplus Transfection Composição adequada para transfectar uma molécula de ácido nucleico em uma célula, métodos para transfecção in vitro ou ex vivo de células vivas e para a produção e usos in vitro ou ex vivo da composição
DE102020114183B4 (de) 2020-05-27 2022-07-14 Universität Leipzig Dezernat Forschung und Transfer Mittel zur transfektion von nukleinsäuren in zellen
CN112195193A (zh) * 2020-09-30 2021-01-08 上海交通大学 单链dna在基因转染中的应用
US20240327830A1 (en) * 2022-02-28 2024-10-03 Northwestern University Dise-inducing srna-polyplexes and srna-lipopolyplexes and methods of using the same to treat cancer

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7427394B2 (en) * 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US7601367B2 (en) * 2002-05-28 2009-10-13 Mirus Bio Llc Compositions and processes using siRNA, amphipathic compounds and polycations
EP1546173A4 (en) * 2002-08-06 2006-05-31 Intradigm Corp METHOD FOR DOWNSTREGULATING THE EXPRESSION OF TARGET GENES IN VIVO BY INCLUDING INTERFERING RNA
JP2006517793A (ja) * 2003-01-31 2006-08-03 ノバルティス アクチエンゲゼルシャフト スクリーニングアッセイ
JP2007502129A (ja) * 2003-05-09 2007-02-08 ユニヴァーシティ オヴ ピッツバーグ オヴ ザ コモンウェルス システム オヴ ハイアー エデュケーション 短鎖干渉rnaライブラリーならびに合成および使用の方法
SG190613A1 (en) * 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
WO2005059111A2 (en) * 2003-12-17 2005-06-30 The Trustees Of Columbia University In The City Of New York DELIVERY OF DNA OR RNA VIA GAP JUNCTIONS FROM HOST CELLS TO TARGET CELLS AND A CELL-BASED DELIVERY SYSTEM FOR ANTISENSE OR SiRNA
JP2008527993A (ja) * 2005-01-24 2008-07-31 アヴァリス・アクチエボラーグ 特異性及び送達の改善のためのsiRNA分子、shRNA分子又はアンチセンス分子及び機能的実体を含む複合体
US20100310670A1 (en) * 2005-09-02 2010-12-09 Takeda Pharmaceutical Company Limited Sustained-release microsphere containing short chain deoxyribonucleic acid or short chain ribonucleic acid and method of producing the same
KR100863405B1 (ko) * 2005-09-12 2008-10-14 주식회사 대웅 암 진단 마커 및 이의 이용
JP2007119396A (ja) * 2005-10-28 2007-05-17 Hosokawa Funtai Gijutsu Kenkyusho:Kk 核酸化合物封入ナノ粒子を含む経肺投与用医薬製剤
GB0613753D0 (en) * 2006-07-11 2006-08-23 Norwegian Radium Hospital Res Method
CN101085356A (zh) * 2007-06-21 2007-12-12 中国科学院长春应用化学研究所 一种非病毒基因转染载体、其与质粒dna复合物颗粒及制备方法和用法

Also Published As

Publication number Publication date
IL206310A (en) 2013-07-31
KR101590652B1 (ko) 2016-02-18
CN101952434A (zh) 2011-01-19
US20100297756A1 (en) 2010-11-25
KR20100105674A (ko) 2010-09-29
JP5802389B2 (ja) 2015-10-28
WO2009074970A3 (en) 2009-10-29
EP2229445B1 (en) 2012-12-05
JP2011505826A (ja) 2011-03-03
EP2229445A2 (en) 2010-09-22
WO2009074970A2 (en) 2009-06-18
IL206310A0 (en) 2010-12-30
CA2708354A1 (en) 2009-06-18
CN101952434B (zh) 2016-11-16

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