CN107735079A - 核酸‑阳离子聚合物组合物及其制备和使用方法 - Google Patents

核酸‑阳离子聚合物组合物及其制备和使用方法 Download PDF

Info

Publication number
CN107735079A
CN107735079A CN201680039770.4A CN201680039770A CN107735079A CN 107735079 A CN107735079 A CN 107735079A CN 201680039770 A CN201680039770 A CN 201680039770A CN 107735079 A CN107735079 A CN 107735079A
Authority
CN
China
Prior art keywords
carbohydrate
composition
nucleic acid
cationic polymer
poly
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
CN201680039770.4A
Other languages
English (en)
Chinese (zh)
Inventor
A·霍克伯格
詹妮弗·加吕拉
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.)
Yissum Research Development Co of Hebrew University of Jerusalem
Original Assignee
Yissum Research Development Co of Hebrew University of Jerusalem
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 Yissum Research Development Co of Hebrew University of Jerusalem filed Critical Yissum Research Development Co of Hebrew University of Jerusalem
Publication of CN107735079A publication Critical patent/CN107735079A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K48/0008—Medicinal 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
    • A61K48/0025—Medicinal 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
    • A61K48/0041—Medicinal 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
    • 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
    • A61K48/005—Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/005—Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Dermatology (AREA)
  • Manufacturing & Machinery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201680039770.4A 2015-05-05 2016-05-05 核酸‑阳离子聚合物组合物及其制备和使用方法 Pending CN107735079A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
IL23864315 2015-05-05
IL238643 2015-05-05
PCT/IL2016/050477 WO2016178233A1 (en) 2015-05-05 2016-05-05 Nucleic acid-cationic polymer compositions and methods of making and using the same

Publications (1)

Publication Number Publication Date
CN107735079A true CN107735079A (zh) 2018-02-23

Family

ID=56134418

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680039770.4A Pending CN107735079A (zh) 2015-05-05 2016-05-05 核酸‑阳离子聚合物组合物及其制备和使用方法

Country Status (14)

Country Link
US (2) US20180154023A1 (enExample)
EP (1) EP3291799A1 (enExample)
JP (2) JP2018515479A (enExample)
KR (1) KR20180015145A (enExample)
CN (1) CN107735079A (enExample)
AU (1) AU2016258326A1 (enExample)
BR (1) BR112017023788A2 (enExample)
CA (1) CA2984879A1 (enExample)
IL (1) IL255254A0 (enExample)
MX (1) MX2017014060A (enExample)
PH (1) PH12017502216A1 (enExample)
RU (1) RU2017140065A (enExample)
SG (2) SG11201708760PA (enExample)
WO (1) WO2016178233A1 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114762731A (zh) * 2022-05-16 2022-07-19 常州大学 一种延长基因药物在温和条件下储存时间的方法
CN116036043A (zh) * 2023-01-10 2023-05-02 深圳市乐土生物医药有限公司 一种核酸药物制剂、冻干粉及制备方法
CN116194081A (zh) * 2020-10-01 2023-05-30 生物技术公司 适用于治疗的脂质体rna制剂的制备和储存
WO2023142284A1 (zh) * 2022-01-27 2023-08-03 深圳市瑞吉生物科技有限公司 一种载核酸脂质纳米颗粒冻干制剂及其制备方法与应用

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018515479A (ja) * 2015-05-05 2018-06-14 イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッドＹｉｓｓｕｍ Ｒｅｓｅａｒｃｈ Ｄｅｖｅｌｏｐｍｅｎｔ Ｃｏｍｐａｎｙ Ｏｆ Ｔｈｅ Ｈｅｂｒｅｗ Ｕｎｉｖｅｒｓｔｙ Ｏｆ Ｊｅｒｕｓａｌｅｍ Ｌｔｄ． 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法
DE102017003004A1 (de) 2017-03-23 2018-09-27 Friedrich-Schiller-Universität Jena Kationische Polymere mit D-Fructose-Substituenten
US10849921B2 (en) * 2017-05-17 2020-12-01 Bioncotech Therapeutics Sl Pharmaceutical composition comprising particles comprising a complex of a double-stranded polyribonucleotide and a polyalkyleneimine
EP3924463A1 (en) 2019-02-11 2021-12-22 Excellgene SA Novel eukaryotic cell transfection systems and related methods
CN111363761A (zh) * 2020-03-17 2020-07-03 苏州吉恒基因科技有限公司 利用阳离子多聚体dna复合物促进aav介导的基因表达方法
CA3181339A1 (en) * 2020-04-27 2021-11-04 Juno Therapeutics, Inc. Stabilization of polyethyleneimine-deoxyribonucleic acid complex size and activity
WO2022249180A1 (en) 2021-05-25 2022-12-01 Ramot At Tel-Aviv University Ltd. Compositions comprising extracellular vesicles and an active agent and uses thereof
CN113501889A (zh) * 2021-07-06 2021-10-15 郑州大学 一种三七多糖阳离子衍生物的制备方法及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1280616A (zh) * 1997-10-03 2001-01-17 耶路撒冷希伯来大学伊森姆研究发展公司 诱导肿瘤特异性细胞毒性的方法和组合物
CN101820864A (zh) * 2007-08-06 2010-09-01 艾根股份有限公司 核酸-脂聚合物组合物

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US20040142474A1 (en) * 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
WO2007007317A1 (en) 2005-07-07 2007-01-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Nucleic acid agents for downregulating h19, and methods of using same
JP2009508516A (ja) 2005-09-22 2009-03-05 イッサム リサーチ ディベロプメント カンパニー オブ ザ ヘブリュー ユニバーシティ オブ エルサレム 核酸構築物、薬剤組成物、及び癌治療のためのその使用方法
CA2675967A1 (en) 2007-01-16 2008-07-24 Yissum Research Development Company Of The Hebrew University Of Jerusale M Nucleic acid constructs and methods for specific silencing of h19
US20090042825A1 (en) * 2007-08-06 2009-02-12 Majed Matar Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer
BRPI0818937B8 (pt) 2007-10-25 2021-05-25 Yissum Res Dev Co Of Hebrew Univ Jerusalem Ltd constructos contendo múltiplos cassetes de expressão para terapia de câncer
WO2010028093A2 (en) * 2008-09-03 2010-03-11 Senesco Technologies, Inc. Use of a truncated elf-5a1 polynucleotide to induce apoptosis in cancer cells
DE102009006606A1 (de) * 2009-01-29 2010-08-05 Philipps-Universität Marburg Nicht-virales Transfektionsmittel
JP2018515479A (ja) * 2015-05-05 2018-06-14 イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッドＹｉｓｓｕｍ Ｒｅｓｅａｒｃｈ Ｄｅｖｅｌｏｐｍｅｎｔ Ｃｏｍｐａｎｙ Ｏｆ Ｔｈｅ Ｈｅｂｒｅｗ Ｕｎｉｖｅｒｓｔｙ Ｏｆ Ｊｅｒｕｓａｌｅｍ Ｌｔｄ． 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1280616A (zh) * 1997-10-03 2001-01-17 耶路撒冷希伯来大学伊森姆研究发展公司 诱导肿瘤特异性细胞毒性的方法和组合物
CN101820864A (zh) * 2007-08-06 2010-09-01 艾根股份有限公司 核酸-脂聚合物组合物

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CAROLA BRUS，等: "Stabilizat ion of oligonucleotide – polyethyle nimine complexes by freeze-drying: physicochemical and biological chara cterization", 《JOURNAL OF CONTROLLED RELEASE》 *
SABRINA HÖBEL，等: "Polyethylenimines for siRNA and miRNA delivery in vivo", 《WILEY INTERDISCIPLINARY REVIEWS-NANOMEDICINE AND NANOBIOTECHNOLOGY》 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116194081A (zh) * 2020-10-01 2023-05-30 生物技术公司 适用于治疗的脂质体rna制剂的制备和储存
WO2023142284A1 (zh) * 2022-01-27 2023-08-03 深圳市瑞吉生物科技有限公司 一种载核酸脂质纳米颗粒冻干制剂及其制备方法与应用
CN114762731A (zh) * 2022-05-16 2022-07-19 常州大学 一种延长基因药物在温和条件下储存时间的方法
CN114762731B (zh) * 2022-05-16 2024-08-23 常州大学 一种延长基因药物在温和条件下储存时间的方法
CN116036043A (zh) * 2023-01-10 2023-05-02 深圳市乐土生物医药有限公司 一种核酸药物制剂、冻干粉及制备方法

Also Published As

Publication number Publication date
IL255254A0 (en) 2017-12-31
EP3291799A1 (en) 2018-03-14
US20200138977A1 (en) 2020-05-07
US20180154023A1 (en) 2018-06-07
RU2017140065A (ru) 2019-06-05
BR112017023788A2 (pt) 2018-10-16
KR20180015145A (ko) 2018-02-12
MX2017014060A (es) 2018-07-06
CA2984879A1 (en) 2016-11-10
JP2021130671A (ja) 2021-09-09
AU2016258326A1 (en) 2017-11-23
WO2016178233A1 (en) 2016-11-10
RU2017140065A3 (enExample) 2019-09-27
SG11201708760PA (en) 2017-11-29
PH12017502216A1 (en) 2018-06-11
JP2018515479A (ja) 2018-06-14
SG10201909209RA (en) 2019-11-28

Similar Documents

Publication Publication Date Title
US20200138977A1 (en) Nucleic acid-cationic polymer compositions and methods of making and using the same
Chow et al. Inhaled RNA therapy: from promise to reality
Lai et al. Nucleic acid delivery with chitosan and its derivatives
Aranda-Barradas et al. Effect of molecular weight of chitosan on the physicochemical, morphological, and biological properties of polyplex nanoparticles intended for gene delivery
CA2695901C (en) Nucleic acid-lipopolymer compositions
US9827331B2 (en) Nucleic acid-lipopolymer compositions
Ito et al. DNA/polyethyleneimine/hyaluronic acid small complex particles and tumor suppression in mice
CA2915131A1 (en) Freeze-dried polyelectrolyte complexes that maintain size and biological activity
CN114206906A (zh) Peg化的合成kl4肽、其组合物和方法
Zhou et al. Polyplex nanovesicles of single strand oligonucleotides for efficient cytosolic delivery of biomacromolecules
Duskunovic et al. Effective mRNA delivery by condensation with cationic nanogels incorporated into liposomes
CN109890393A (zh) 功能rna和小分子药物治疗性复合物和纳米颗粒递送媒介
JP6763780B2 (ja) 合成脳浸透遺伝子ベクターの操作
Liu et al. PH-sensitive self-assembled carboxymethyl chitosan-modified DNA/polyethylenimine complexes for efficient gene delivery
CN106474472B (zh) 肿瘤曲妥珠单抗靶向anti-miR-21纳米粒子及其制备方法
Trenkenschuh Freeze-drying of nanoparticles: impact of particle properties on formulation and process development
HK1247839A1 (en) Nucleic acid-cationic polymer compositions and methods of making and using the same
Singh et al. The Junction of Biomaterials and Gene Therapy–Current Strategies and Future Directions
Jiang et al. Poly (amido amine)-based nanogels for delivery of mRNA
WO2025166259A1 (en) Mineral binding mrna lipid nanoparticles
WO2024163503A1 (en) Process for producing dna formulations with lipopolymer delivery systems
Subsantisuk Development of gene carriers using liposomes with surfactants coated with cationic polymers
Vitro Chemical and Molecular Conjugates I

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 1247839

Country of ref document: HK

WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20180223

REG Reference to a national code

Ref country code: HK

Ref legal event code: WD

Ref document number: 1247839

Country of ref document: HK