AU2022226409A1 - Formulations for aerosol formation and aerosols for the delivery of nucleic acid - Google Patents

Formulations for aerosol formation and aerosols for the delivery of nucleic acid Download PDF

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Publication number
AU2022226409A1
AU2022226409A1 AU2022226409A AU2022226409A AU2022226409A1 AU 2022226409 A1 AU2022226409 A1 AU 2022226409A1 AU 2022226409 A AU2022226409 A AU 2022226409A AU 2022226409 A AU2022226409 A AU 2022226409A AU 2022226409 A1 AU2022226409 A1 AU 2022226409A1
Authority
AU
Australia
Prior art keywords
lipid
aerosol
accordance
suspension formulation
nanoparticles
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
AU2022226409A
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English (en)
Other versions
AU2022226409A9 (en
Inventor
Philipp Beck
Christian Dohmen
Christian Plank
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.)
Ethris GmbH
Original Assignee
Ethris GmbH
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 Ethris GmbH filed Critical Ethris GmbH
Publication of AU2022226409A1 publication Critical patent/AU2022226409A1/en
Publication of AU2022226409A9 publication Critical patent/AU2022226409A9/en
Pending legal-status Critical Current

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Classifications

    • 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
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • 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
    • 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
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Genetics & Genomics (AREA)
  • Nanotechnology (AREA)
  • Organic Chemistry (AREA)
  • Pulmonology (AREA)
  • Optics & Photonics (AREA)
  • Molecular Biology (AREA)
  • Dispersion Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Otolaryngology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Inorganic Chemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2022226409A 2021-02-26 2022-02-25 Formulations for aerosol formation and aerosols for the delivery of nucleic acid Pending AU2022226409A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21159455.1 2021-02-26
EP21159455 2021-02-26
PCT/EP2022/054796 WO2022180213A1 (en) 2021-02-26 2022-02-25 Formulations for aerosol formation and aerosols for the delivery of nucleic acid

Publications (2)

Publication Number Publication Date
AU2022226409A1 true AU2022226409A1 (en) 2023-08-10
AU2022226409A9 AU2022226409A9 (en) 2024-07-18

Family

ID=74797735

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022226409A Pending AU2022226409A1 (en) 2021-02-26 2022-02-25 Formulations for aerosol formation and aerosols for the delivery of nucleic acid

Country Status (11)

Country Link
US (1) US20240156729A1 (https=)
EP (1) EP4297722A1 (https=)
JP (1) JP2024507394A (https=)
KR (1) KR20230152014A (https=)
CN (1) CN116887812A (https=)
AU (1) AU2022226409A1 (https=)
BR (1) BR112023016903A2 (https=)
CA (1) CA3209032A1 (https=)
IL (1) IL305353A (https=)
MX (1) MX2023009757A (https=)
WO (1) WO2022180213A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024042236A1 (en) * 2022-08-26 2024-02-29 Ethris Gmbh Stable lipid or lipidoid nanoparticle suspensions
EP4327829A1 (en) * 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
CN115417778B (zh) * 2022-11-01 2023-06-20 北京华芢生物技术有限公司 可离子化的阳离子脂c5及由其组成的纳米脂质体颗粒
CN115708818B (zh) * 2022-11-03 2023-07-18 荣灿生物医药技术(上海)有限公司 一种用于雾化吸入脂质纳米粒的通用型处方
CN115433099B (zh) * 2022-11-03 2023-06-16 北京华芢生物技术有限公司 可离子化的阳离子脂c6及由其组成的纳米脂质体颗粒
CN120752027A (zh) * 2023-03-24 2025-10-03 星锐医药(苏州)有限公司 编码治疗性多肽的核酸及包含所述核酸的脂质纳米颗粒组合物
CN121712488A (zh) * 2023-08-14 2026-03-20 埃泽瑞斯公司 非全身性mRNA施用
CN121729225A (zh) * 2023-08-25 2026-03-24 埃泽瑞斯公司 具有增强药物应用的特定表面活性剂性质的稳定脂质和类脂质纳米颗粒制剂
WO2025088192A1 (en) 2023-10-27 2025-05-01 Xl-Protein Gmbh Methods for the coupling of di(alkyl)amines to polypeptides

Family Cites Families (22)

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Publication number Priority date Publication date Assignee Title
US6017700A (en) 1995-08-04 2000-01-25 Bayer Corporation Cationic oligonucleotides, and related methods of synthesis and use
CA2294334A1 (en) * 1997-06-13 1998-12-17 Adi Eisenberg Compositions for delivery of biological agents and methods for the preparation thereof
DE19834683A1 (de) 1997-08-13 1999-04-01 Biontex Lab Gmbh Neue Lipopolyamine, deren Darstellung und Anwendung
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
WO2006138380A2 (en) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
NZ569138A (en) 2005-12-15 2012-06-29 Centre Nat Rech Scient Cationic oligonucleotides automated methods for preparing same and their uses
US7897737B2 (en) 2006-12-05 2011-03-01 Lasergen, Inc. 3′-OH unblocked, nucleotides and nucleosides, base modified with photocleavable, terminating groups and methods for their use in DNA sequencing
CA2721183C (en) 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
CA2769670C (en) 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
US8648017B2 (en) * 2009-11-04 2014-02-11 Diamir, Llc Methods of using small RNA from bodily fluids for diagnosis and monitoring of neurodegenerative diseases
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
US12064484B2 (en) 2013-06-28 2024-08-20 Ethris Gmbh Compositions for introducing RNA into cells
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Also Published As

Publication number Publication date
CN116887812A (zh) 2023-10-13
MX2023009757A (es) 2023-09-04
BR112023016903A2 (pt) 2023-10-10
CA3209032A1 (en) 2022-09-01
WO2022180213A1 (en) 2022-09-01
IL305353A (en) 2023-10-01
US20240156729A1 (en) 2024-05-16
KR20230152014A (ko) 2023-11-02
JP2024507394A (ja) 2024-02-19
EP4297722A1 (en) 2024-01-03
AU2022226409A9 (en) 2024-07-18

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