DK3740199T3 - Polymerindkapslede virusvektorer til genetisk terapi - Google Patents

Polymerindkapslede virusvektorer til genetisk terapi Download PDF

Info

Publication number
DK3740199T3
DK3740199T3 DK19743691.8T DK19743691T DK3740199T3 DK 3740199 T3 DK3740199 T3 DK 3740199T3 DK 19743691 T DK19743691 T DK 19743691T DK 3740199 T3 DK3740199 T3 DK 3740199T3
Authority
DK
Denmark
Prior art keywords
gene therapy
virus vectors
polymer encapsulated
encapsulated virus
polymer
Prior art date
Application number
DK19743691.8T
Other languages
English (en)
Inventor
Cécile Bauche
Renaud Vaillant
Emeline Sarry
Frederic Mourlane
Philippe Bishop
Original Assignee
Ixaka France
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 Ixaka France filed Critical Ixaka France
Application granted granted Critical
Publication of DK3740199T3 publication Critical patent/DK3740199T3/da

Links

Classifications

    • 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
    • A61K9/5176Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184Virus capsids or envelopes enclosing drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/421Immunoglobulin superfamily
    • A61K40/4211CD19 or B4
    • 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
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • 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
    • A61K47/50Medicinal 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
    • A61K47/69Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A61K47/6931Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
    • A61K47/6935Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol
    • 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
    • A61K9/5146Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A61K9/5153Polyesters, e.g. poly(lactide-co-glycolide)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/7051T-cell receptor (TcR)-CD3 complex
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16045Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Organic Chemistry (AREA)
  • Nanotechnology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Optics & Photonics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Botany (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
DK19743691.8T 2018-01-17 2019-01-17 Polymerindkapslede virusvektorer til genetisk terapi DK3740199T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862618156P 2018-01-17 2018-01-17
US201862661990P 2018-04-24 2018-04-24
US201862715007P 2018-08-06 2018-08-06
PCT/IB2019/000361 WO2019145796A2 (en) 2018-01-17 2019-01-17 Polymer-encapsulated viral vectors for genetic therapy

Publications (1)

Publication Number Publication Date
DK3740199T3 true DK3740199T3 (da) 2024-11-11

Family

ID=67394531

Family Applications (1)

Application Number Title Priority Date Filing Date
DK19743691.8T DK3740199T3 (da) 2018-01-17 2019-01-17 Polymerindkapslede virusvektorer til genetisk terapi

Country Status (14)

Country Link
US (1) US20200360297A1 (da)
EP (1) EP3740199B1 (da)
JP (2) JP2021510548A (da)
KR (1) KR102812046B1 (da)
CN (1) CN111902134A (da)
AU (1) AU2019213290A1 (da)
BR (1) BR112020014650A2 (da)
CA (1) CA3088933A1 (da)
DK (1) DK3740199T3 (da)
ES (1) ES2991922T3 (da)
FI (1) FI3740199T3 (da)
MX (1) MX2021001423A (da)
PL (1) PL3740199T3 (da)
WO (1) WO2019145796A2 (da)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3151983A1 (en) * 2019-09-21 2021-03-25 Ozgul TEZGEL Dmso-free synthesis of oligopeptide-modified poly(beta-amino ester)s and their use in nanoparticle delivery systems
WO2021094831A1 (en) 2019-11-15 2021-05-20 Aratinga.Bio Tnp Polymer-encapsulated viral vectors for in vivo genetic therapy
JP2023525969A (ja) * 2020-05-19 2023-06-20 イグザカ フランス インビトロおよびインビボでのcd3+細胞における遺伝子治療産物の発現のためのプロモータ配列
AU2023398933A1 (en) * 2022-12-16 2025-07-03 Lilium Therapeutics Inc. Viral vectors with engineered function and methods of production thereof
WO2024201067A1 (en) * 2023-03-31 2024-10-03 Dawn Therapeutics Limited Transfection system
EP4709745A1 (en) * 2023-05-11 2026-03-18 Alaya.Bio Inc. Extracellular vesicles for the delivery of payloads to eukaryotic cells

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6502261B2 (ja) * 2012-12-14 2019-04-17 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ターゲティング遺伝子治療のためのウイルスベクターナノカプセル、およびその調製
GB201304245D0 (en) * 2013-03-08 2013-04-24 Inst Quimic De Sarria Chemical compounds
EP2970985A1 (en) 2013-03-14 2016-01-20 Fred Hutchinson Cancer Research Center Compositions and methods to modify cells for therapeutic objectives
US20160251680A1 (en) * 2013-11-05 2016-09-01 Clontech Laboratories, Inc. Dry transfection compositions and methods for making and using the same
US10335500B2 (en) * 2014-05-12 2019-07-02 The Johns Hopkins University Highly stable biodegradable gene vector platforms for overcoming biological barriers
CN113846062B (zh) 2014-07-25 2025-02-21 赛拉福柯蒂斯公司 用于嵌合抗原受体分子的调控表达的慢病毒载体
WO2016019126A1 (en) * 2014-07-30 2016-02-04 The Research Foundation For The State University Of New York System and method for delivering genetic material or protein to cells
WO2016022947A1 (en) * 2014-08-07 2016-02-11 The Johns Hopkins University Nanoparticle modification of human adipose-derived mesenchymal stem cells for treating brain cancer and other neurological diseases
GB201501025D0 (en) 2015-01-21 2015-03-04 Sagetis Biotech Sl Chemical compounds
GB201708203D0 (en) * 2017-05-22 2017-07-05 Sagetis Biotech Sl Chemical compounds

Also Published As

Publication number Publication date
WO2019145796A2 (en) 2019-08-01
AU2019213290A1 (en) 2020-08-06
CN111902134A (zh) 2020-11-06
MX2021001423A (es) 2021-11-17
CA3088933A1 (en) 2019-08-01
WO2019145796A3 (en) 2019-11-07
PL3740199T3 (pl) 2024-12-16
JP2024020452A (ja) 2024-02-14
JP2021510548A (ja) 2021-04-30
KR20200128520A (ko) 2020-11-13
EP3740199A2 (en) 2020-11-25
BR112020014650A2 (pt) 2021-02-17
ES2991922T3 (es) 2024-12-05
FI3740199T3 (fi) 2024-11-11
EP3740199B1 (en) 2024-08-14
KR102812046B1 (ko) 2025-05-23
US20200360297A1 (en) 2020-11-19
AU2024259893A1 (en) 2024-11-28

Similar Documents

Publication Publication Date Title
EP3645021A4 (en) ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
DK3740199T3 (da) Polymerindkapslede virusvektorer til genetisk terapi
IL277666A (en) Virus vector evasion antibody
DK3596222T5 (da) Adenoassocieret virusvektorfremføring af muskelspecifik mikro-dystrophin til behandling af muskeldystrofi
IL259842A (en) Targeting peptides for directing adeno-associated viruses -aavs-
IL270567B (en) Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b
SI3471780T1 (sl) Zdravljenje SDM-ja z uporabo različice AAV2 z afliberceptom
IL277664A (en) Virus vector evasion antibody
EP3861130A4 (en) GENETIC MODULATORS
DK3740222T3 (da) Modificeret RAAV-capsidprotein til genterapi
DK3807413T3 (da) Adenoassocieret virusvektorfremføring af muskelspecifik mikrodystrofin til behandling af muskeldystrofi
HUE071243T2 (hu) Mikro-disztrofin bejuttatása adeno-asszociált vírus vektorral izomdisztrófia kezelésére
EP3558393A4 (en) ADENO-ASSOCIATED VIRAL VECTORS
DK3390429T3 (da) Modificerede kapsid-proteiner til forbedret afgivelse af parvovirus-vektorer
SMT202400506T1 (it) Terapia genica
HUE054967T2 (hu) Új genetikailag módosított Vaccinia vírus
DK3137497T4 (da) Aav-vektorer til retinal- og cns-genterapi
EP3577132A4 (en) ONCOLYTIC VIRUS THERAPY
IL272373A (en) Factor viii (fviii) gene therapy methods
DK3262066T4 (da) Genterapi
EP3735467A4 (en) MODIFIED VACCINE VECTORS
EP4051324A4 (en) Gene therapy vectors
IL279537A (en) Aav compositions
PT3612237T (pt) Terapia genética
IL278668A (en) Virus vector production