CY1122850T1 - Υπερδιακλαδισμενος πολυ (βητα-αμινοεστερας) για γονιδιακη θεραπεια - Google Patents

Υπερδιακλαδισμενος πολυ (βητα-αμινοεστερας) για γονιδιακη θεραπεια

Info

Publication number
CY1122850T1
CY1122850T1 CY20201100361T CY201100361T CY1122850T1 CY 1122850 T1 CY1122850 T1 CY 1122850T1 CY 20201100361 T CY20201100361 T CY 20201100361T CY 201100361 T CY201100361 T CY 201100361T CY 1122850 T1 CY1122850 T1 CY 1122850T1
Authority
CY
Cyprus
Prior art keywords
gene therapy
aminoester
beta
hyperbranched poly
hyperbranched
Prior art date
Application number
CY20201100361T
Other languages
English (en)
Inventor
Wenxin Wang
Dezhong ZHOU
Original Assignee
University College Dublin, National University Of Ireland Dublin
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 University College Dublin, National University Of Ireland Dublin filed Critical University College Dublin, National University Of Ireland Dublin
Publication of CY1122850T1 publication Critical patent/CY1122850T1/el

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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
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G63/00—Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
    • C08G63/68—Polyesters containing atoms other than carbon, hydrogen and oxygen
    • C08G63/685—Polyesters containing atoms other than carbon, hydrogen and oxygen containing nitrogen
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G73/00—Macromolecular compounds obtained by reactions forming a linkage containing nitrogen with or without oxygen or carbon in the main chain of the macromolecule, not provided for in groups C08G12/00 - C08G71/00
    • C08G73/02—Polyamines
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G83/00—Macromolecular compounds not provided for in groups C08G2/00 - C08G81/00
    • C08G83/002—Dendritic macromolecules
    • C08G83/005—Hyperbranched macromolecules
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Polymers & Plastics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Polyesters Or Polycarbonates (AREA)
  • Biological Depolymerization Polymers (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Η εφεύρεση σχετίζεται με διακλαδισμένα πολυμερή, τα οποία χρησιμοποιούνται σε εφαρμογές γονιδιακής θεραπείας ως παράγοντες διαμόλυνσης νουκλεϊκού οξέος. Πιο συγκεκριμένα, η εφεύρεση παρέχει βιοαποικοδομήσιμα, υπέρ διακλαδισμένα πολυμερή, τα οποία μπορούν να χρησιμοποιηθούν στη χορήγηση των γονιδίων και παρέχουν βελτιωμένες αποδόσεις διαμόλυνσης, οι οποίες είναι ταυτόχρονα ασφαλείς και μη τοξικές.
CY20201100361T 2014-08-06 2020-04-16 Υπερδιακλαδισμενος πολυ (βητα-αμινοεστερας) για γονιδιακη θεραπεια CY1122850T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1413907.5A GB201413907D0 (en) 2014-08-06 2014-08-06 Hyberbranched poly(beta-amino ester) for gene therapy
PCT/EP2015/068153 WO2016020474A1 (en) 2014-08-06 2015-08-06 HYPERBRANCHED POLY( ß -AMINO ESTER) FOR GENE THERAPY

Publications (1)

Publication Number Publication Date
CY1122850T1 true CY1122850T1 (el) 2021-05-05

Family

ID=51587799

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20201100361T CY1122850T1 (el) 2014-08-06 2020-04-16 Υπερδιακλαδισμενος πολυ (βητα-αμινοεστερας) για γονιδιακη θεραπεια

Country Status (14)

Country Link
US (1) US20170216455A1 (el)
EP (2) EP3738992A1 (el)
CY (1) CY1122850T1 (el)
DK (1) DK3177670T3 (el)
ES (1) ES2785248T3 (el)
GB (1) GB201413907D0 (el)
HR (1) HRP20200633T1 (el)
HU (1) HUE049704T2 (el)
LT (1) LT3177670T (el)
PL (1) PL3177670T3 (el)
PT (1) PT3177670T (el)
RS (1) RS60335B1 (el)
SI (1) SI3177670T1 (el)
WO (1) WO2016020474A1 (el)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3700965A1 (en) * 2017-10-27 2020-09-02 Massachusetts Institute of Technology Poly (beta-amino esters) and uses thereof
MX2021004169A (es) 2018-10-12 2021-08-05 Amryt Genetics Ltd Composiciones y metodos para la transfeccion de celulas.
EP3870629A1 (en) 2018-10-26 2021-09-01 Massachusetts Institute of Technology Polymer-lipids and compositions
CA3119794A1 (en) * 2018-12-28 2020-07-02 Amryt Genetics Ltd. Methods for forming polyplexes
EP3798250A1 (en) 2019-09-25 2021-03-31 University College Dublin Hyperbranched cationic polymers useful as nucleic acid delivery vectors for transfecting
CN114650811A (zh) 2019-09-25 2022-06-21 爱尔兰国立都柏林大学 用于基因疗法的纳米粒子组合物
CN110746599B (zh) * 2019-09-30 2021-06-18 苏州大学 具有高效基因递送能力的UV光响应性超支化聚β-氨基酯及其制备方法与应用
CN111135306B (zh) * 2020-01-19 2023-03-10 安阳师范学院 基于叶酸靶向聚乙二醇修饰型超支化聚胺制备叶酸和磁性双靶向型非病毒基因载体的方法
EP4305088A1 (en) * 2021-03-09 2024-01-17 Massachusetts Institute of Technology Branched poly(-amino esters) for the delivery of nucleic acids
CN114106348B (zh) * 2021-11-12 2023-07-07 西安交通大学 苯硼酸修饰的胞内可降解超支化聚（β-氨基酯）及其制备方法和蛋白递送应用
EP4183884A1 (en) 2021-11-19 2023-05-24 Branca Bunus Limited A method of preparing a fraction of a starting polymer vector for gene therapy
WO2023131648A1 (en) 2022-01-05 2023-07-13 Branca Bunus Limited Nanoparticulate compositions for gene therapy
EP4324485A1 (en) 2022-08-15 2024-02-21 Branca Banus Ltd A method for the production of pegylated hyperbranched poly( -amino ester)s, pegylated hyperbranched poly( -amino ester)s produced by those methods and their use in enhanced nucleic acid delivery

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5180424A (en) * 1991-10-07 1993-01-19 Westvaco Corporation Michael addition aminopolyester resins as dilution extenders for zinc-containing metal resinate inks
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
EP1874844A4 (en) * 2005-04-29 2010-03-24 Agency Science Tech & Res HYPERRAMIFIED POLYMERS AND THEIR APPLICATIONS
EP2046266A4 (en) * 2006-07-21 2009-11-04 Massachusetts Inst Technology ENDMODICIFIED POLY (BETA AMINO ESTER) AND ITS USE

Also Published As

Publication number Publication date
RS60335B1 (sr) 2020-07-31
WO2016020474A1 (en) 2016-02-11
DK3177670T3 (da) 2020-04-27
EP3738992A1 (en) 2020-11-18
HRP20200633T1 (hr) 2020-08-07
GB201413907D0 (en) 2014-09-17
US20170216455A1 (en) 2017-08-03
PT3177670T (pt) 2020-04-24
ES2785248T3 (es) 2020-10-06
LT3177670T (lt) 2020-06-25
EP3177670B1 (en) 2020-03-18
HUE049704T2 (hu) 2020-10-28
EP3177670A1 (en) 2017-06-14
SI3177670T1 (sl) 2020-07-31
PL3177670T3 (pl) 2020-07-13

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