WO2024035932A1 - Compositions de nanoparticules lyophilisées et leurs procédés d'utilisation - Google Patents

Compositions de nanoparticules lyophilisées et leurs procédés d'utilisation Download PDF

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Publication number
WO2024035932A1
WO2024035932A1 PCT/US2023/030082 US2023030082W WO2024035932A1 WO 2024035932 A1 WO2024035932 A1 WO 2024035932A1 US 2023030082 W US2023030082 W US 2023030082W WO 2024035932 A1 WO2024035932 A1 WO 2024035932A1
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WO
WIPO (PCT)
Prior art keywords
lipid
containing component
solution
mole
lnp
Prior art date
Application number
PCT/US2023/030082
Other languages
English (en)
Inventor
Evan Michael LEWOCZKO
Dong Shen
Original Assignee
RNAimmune, Inc.
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 RNAimmune, Inc. filed Critical RNAimmune, Inc.
Publication of WO2024035932A1 publication Critical patent/WO2024035932A1/fr

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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
    • A61K9/48—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
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23L—FOODS, FOODSTUFFS, OR NON-ALCOHOLIC BEVERAGES, NOT COVERED BY SUBCLASSES A21D OR A23B-A23J; THEIR PREPARATION OR TREATMENT, e.g. COOKING, MODIFICATION OF NUTRITIVE QUALITIES, PHYSICAL TREATMENT; PRESERVATION OF FOODS OR FOODSTUFFS, IN GENERAL
    • A23L33/00—Modifying nutritive qualities of foods; Dietetic products; Preparation or treatment thereof
    • A23L33/10—Modifying nutritive qualities of foods; Dietetic products; Preparation or treatment thereof using additives
    • A23L33/13—Nucleic acids or derivatives thereof
    • 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
    • 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
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Optics & Photonics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Nanotechnology (AREA)
  • Mycology (AREA)
  • Nutrition Science (AREA)
  • Food Science & Technology (AREA)
  • Polymers & Plastics (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne un procédé de fabrication d'une composition de nanoparticules, comprenant : (a) la congélation d'une solution comprenant une composition de nanoparticules lipidiques (LNP) et un acide nucléique à une température qui est de 2 °C à 10 °C au-dessous d'un point eutectique de la solution pour produire une solution congelée, la composition LNP comprenant un composant contenant des lipides cationiques ou ionisables, un composant contenant des lipides stéroïdiens ou structuraux, et un composant contenant des lipides stabilisants ; (b) le placement de la solution congelée de (a) sous vide pour produire un échantillon ; (c) le réchauffement de l'échantillon de (b) à une température de 20 °C à 35 °C ; et (d) le placement de l'échantillon réchauffé de (c) sous vide.
PCT/US2023/030082 2022-08-11 2023-08-11 Compositions de nanoparticules lyophilisées et leurs procédés d'utilisation WO2024035932A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263371096P 2022-08-11 2022-08-11
US63/371,096 2022-08-11

Publications (1)

Publication Number Publication Date
WO2024035932A1 true WO2024035932A1 (fr) 2024-02-15

Family

ID=88018245

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/030082 WO2024035932A1 (fr) 2022-08-11 2023-08-11 Compositions de nanoparticules lyophilisées et leurs procédés d'utilisation

Country Status (1)

Country Link
WO (1) WO2024035932A1 (fr)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017049245A2 (fr) 2015-09-17 2017-03-23 Modernatx, Inc. Composés et compositions pour l'administration intracellulaire d'agents thérapeutiques
WO2017218704A1 (fr) * 2016-06-14 2017-12-21 Modernatx, Inc. Formulations stabilisées de nanoparticules lipidiques
CN114557971A (zh) * 2022-04-25 2022-05-31 康希诺生物股份公司 一种核酸-脂质纳米颗粒的冷冻干燥保护剂及其制备方法和应用

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017049245A2 (fr) 2015-09-17 2017-03-23 Modernatx, Inc. Composés et compositions pour l'administration intracellulaire d'agents thérapeutiques
WO2017218704A1 (fr) * 2016-06-14 2017-12-21 Modernatx, Inc. Formulations stabilisées de nanoparticules lipidiques
CN114557971A (zh) * 2022-04-25 2022-05-31 康希诺生物股份公司 一种核酸-脂质纳米颗粒的冷冻干燥保护剂及其制备方法和应用

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
"Pharmaceutical Salts: Properties, Selection, and Use", 2008, WILEY-VCH
"Remington's Pharmaceutical Sciences", 1985, MACK PUBLISHING COMPANY, pages: 1418
"Remington's The Science and Practice of Pharmacy", 2006, LIPPINCOTT, WILLIAMS & WILKINS
BERGE ET AL.: "Journal of Pharmaceutical Science", vol. 66, 1977, pages: 1 - 19
BRUNET L ET AL.: "Thermodynamic calculation of n-component eutectic mixtures", INTERNATIONAL JOURNAL OF MODERN PHYSICS C, vol. 15, no. 05, 2004, pages 675 - 687
KON EDO ET AL: "Principles for designing an optimal mRNA lipid nanoparticle vaccine", CURRENT OPINION IN BIOTECHNOLOGY, LONDON, GB, vol. 73, 26 October 2021 (2021-10-26), pages 329 - 336, XP086958490, ISSN: 0958-1669, [retrieved on 20211026], DOI: 10.1016/J.COPBIO.2021.09.016 *
KUMAR NITESH ET AL: "A REVIEW ARTICLE ON LYOPHILIZATION TECHNIQUES USED IN PHARMACEUTICAL MANUFACTURING", NAUTIYAL, 30 December 2017 (2017-12-30), pages 478 - 484, XP093094074, Retrieved from the Internet <URL:https://ijpmr.org/pdf/A-REVIEW-ARTICLE-ON-LYOPHILIZATION-TECHNIQUES-USED-IN-PHARMACEUTICAL-MANUFACTURING%20.pdf> [retrieved on 20231023] *
UDDIN MOHAMMAD N. ET AL: "Challenges of Storage and Stability of mRNA-Based COVID-19 Vaccines", VACCINES, vol. 9, no. 9, 17 September 2021 (2021-09-17), CH, pages 1033, XP055861754, ISSN: 2076-393X, DOI: 10.3390/vaccines9091033 *

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