WO2023025899A3 - Delivery system for targeting genes of the interferon pathway - Google Patents

Delivery system for targeting genes of the interferon pathway Download PDF

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Publication number
WO2023025899A3
WO2023025899A3 PCT/EP2022/073703 EP2022073703W WO2023025899A3 WO 2023025899 A3 WO2023025899 A3 WO 2023025899A3 EP 2022073703 W EP2022073703 W EP 2022073703W WO 2023025899 A3 WO2023025899 A3 WO 2023025899A3
Authority
WO
WIPO (PCT)
Prior art keywords
targeting genes
interferon pathway
delivery system
reverse micelle
micelle system
Prior art date
Application number
PCT/EP2022/073703
Other languages
French (fr)
Other versions
WO2023025899A2 (en
Inventor
Eric Quemeneur
Philippe Erbs
Abdelkader MOURI
Jean-Claude Maurel
Hervé SEITZ
Original Assignee
Transgene
Medesis Pharma
Centre National De La Recherche Scientifique
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 Transgene, Medesis Pharma, Centre National De La Recherche Scientifique filed Critical Transgene
Publication of WO2023025899A2 publication Critical patent/WO2023025899A2/en
Publication of WO2023025899A3 publication Critical patent/WO2023025899A3/en

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    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/768—Oncolytic viruses not provided for in groups A61K35/761 - A61K35/766
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • 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
    • A61K9/0014—Skin, i.e. galenical aspects of topical 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1138—Non-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 against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • C07K14/7156—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interferons [IFN]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/24011—Poxviridae
    • C12N2710/24111—Orthopoxvirus, e.g. vaccinia virus, variola
    • C12N2710/24132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

Abstract

The present invention relates to specific reverse micelle system which allows the administration and intracellular delivery of unmodified oligonucleotide, such as siRNA, targeting genes linked to the interferon pathway, with a preference for the IFNAR1 gene. The reverse micelle system of the invention is thus particularly useful for the treatment of pathologies related to an overexpression of IFNAR1 gene. The reverse micelle system of the invention is also for use in combination with an oncolytic virus or composition thereof for the treatment of cancers. In particular embodiments, said cancers are resistant to oncolytic virus-based treatments and/or are characterized by an overexpression of IFNAR1 gene.
PCT/EP2022/073703 2021-08-26 2022-08-25 Delivery system for targeting genes of the interferon pathway WO2023025899A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP21306158 2021-08-26
EP21306158.3 2021-08-26

Publications (2)

Publication Number Publication Date
WO2023025899A2 WO2023025899A2 (en) 2023-03-02
WO2023025899A3 true WO2023025899A3 (en) 2023-04-20

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2022/073703 WO2023025899A2 (en) 2021-08-26 2022-08-25 Delivery system for targeting genes of the interferon pathway

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WO (1) WO2023025899A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1652513A1 (en) * 2004-11-02 2006-05-03 Denderah Pharm Sa Reverse micelles based on sterols and acylglycerols and therapeutic uses thereof
WO2011117333A2 (en) * 2010-03-24 2011-09-29 Medesis Pharma Reverse micelle system comprising metal ions and use thereof
KR20120098351A (en) * 2011-02-28 2012-09-05 성균관대학교산학협력단 A composition for preventing bird flu comprising interferon receptor gene
CN112063622A (en) * 2020-09-04 2020-12-11 华农（肇庆）生物产业技术研究院有限公司 Construction method and application of I-type IFNAR function-deficient cell line

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
FR2751343B1 (en) 1996-07-16 1998-12-18 Transgene Sa PROCESS FOR THE PRESERVATION OF INFECTIOUS RECOMBINANT VIRUSES, AQUEOUS VIRAL SUSPENSION, AND USE AS A MEDICAMENT
NZ533302A (en) 2001-12-10 2005-11-25 Bavarian Nordic As Poxvirus containing formulations and process for preparing stable, poxvirus containing compositions containing a disaaccharide and a pharmaceutically acceptable polymer i.e. dextran or polyvinylpyrrolidon (PVP)
EP1528101A1 (en) 2003-11-03 2005-05-04 ProBioGen AG Immortalized avian cell lines for virus production
FR2884255B1 (en) 2005-04-11 2010-11-05 Vivalis USE OF EBX AVIATION STEM CELL LINES FOR THE PRODUCTION OF INFLUENZA VACCINE
JP5138601B2 (en) 2005-11-21 2013-02-06 サノフィ パストゥール リミテッド Recombinant virus stabilization formulation
DK2029756T3 (en) 2006-06-20 2017-02-13 Transgene Sa PROCEDURE FOR PREPARING POXVIRA AND POXVIRUS COMPOSITIONS
GB0705245D0 (en) 2007-03-19 2007-04-25 Stabilitech Ltd Stable biological products
EP1985305A1 (en) 2007-04-24 2008-10-29 Vivalis Duck embryonic derived stem cell lines for the production of viral vaccines
JP2010526546A (en) 2007-05-14 2010-08-05 バヴァリアン・ノルディック・アクティーゼルスカブ Purification of vaccinia virus and recombinant vaccinia virus vaccines
US8357531B2 (en) 2007-07-03 2013-01-22 Transgene S.A. Immortalized avian cell lines
NZ584199A (en) 2007-11-19 2012-07-27 Transgene Sa Poxviral oncolytic vectors comprising a defective 14l and/or f4l gene
CN102257134B (en) 2008-02-12 2014-03-05 赛诺菲巴斯德有限公司 Methods using ion exchange and gel filtration chromatography for poxvirus purification
CA2760465A1 (en) 2009-05-12 2010-11-18 Transgene Sa Method for orthopoxvirus production and purification
PL2739293T3 (en) 2011-08-05 2020-11-16 Sillajen Biotherapeutics, Inc. Methods and compositions for production of vaccina virus

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1652513A1 (en) * 2004-11-02 2006-05-03 Denderah Pharm Sa Reverse micelles based on sterols and acylglycerols and therapeutic uses thereof
WO2011117333A2 (en) * 2010-03-24 2011-09-29 Medesis Pharma Reverse micelle system comprising metal ions and use thereof
KR20120098351A (en) * 2011-02-28 2012-09-05 성균관대학교산학협력단 A composition for preventing bird flu comprising interferon receptor gene
CN112063622A (en) * 2020-09-04 2020-12-11 华农（肇庆）生物产业技术研究院有限公司 Construction method and application of I-type IFNAR function-deficient cell line

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Publication number Publication date
WO2023025899A2 (en) 2023-03-02

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