CN118265522A - 适用于治疗的基于脂质的rna制剂 - Google Patents

适用于治疗的基于脂质的rna制剂 Download PDF

Info

Publication number
CN118265522A
CN118265522A CN202280074040.3A CN202280074040A CN118265522A CN 118265522 A CN118265522 A CN 118265522A CN 202280074040 A CN202280074040 A CN 202280074040A CN 118265522 A CN118265522 A CN 118265522A
Authority
CN
China
Prior art keywords
rna
particle
lipid
composition
particles
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
CN202280074040.3A
Other languages
English (en)
Chinese (zh)
Inventor
H·黑费莎
H·哈斯
F·贝茨
C·霍茨
K·卡里科
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.)
Biotechnology Europe Inc
Original Assignee
Biotechnology Europe 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 Biotechnology Europe Inc filed Critical Biotechnology Europe Inc
Publication of CN118265522A publication Critical patent/CN118265522A/zh
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/193Colony stimulating factors [CSF]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/20Interleukins [IL]
    • A61K38/208IL-12
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/21Interferons [IFN]
    • A61K38/212IFN-alpha
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • 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
    • A61K9/5192Processes

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Optics & Photonics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Zoology (AREA)
  • Dermatology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280074040.3A 2021-09-10 2022-09-09 适用于治疗的基于脂质的rna制剂 Pending CN118265522A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EPPCT/EP2021/074976 2021-09-10
EP2021074976 2021-09-10
PCT/EP2022/075168 WO2023036960A1 (en) 2021-09-10 2022-09-09 Lipid-based rna formulations suitable for therapy

Publications (1)

Publication Number Publication Date
CN118265522A true CN118265522A (zh) 2024-06-28

Family

ID=83594138

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280074040.3A Pending CN118265522A (zh) 2021-09-10 2022-09-09 适用于治疗的基于脂质的rna制剂

Country Status (5)

Country Link
US (1) US20240408032A1 (https=)
EP (1) EP4398882A1 (https=)
JP (1) JP2024535766A (https=)
CN (1) CN118265522A (https=)
WO (1) WO2023036960A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
IL321425A (en) 2022-12-23 2025-08-01 Biontech Delivery Tech Gmbh compound
WO2024193827A1 (en) * 2023-03-23 2024-09-26 BioNTech SE Stabilized nucleic acid compositions and methods for preparing, storing and using the same
WO2025021277A1 (en) * 2023-07-21 2025-01-30 BioNTech SE Agents and methods for targeted delivery of cytokines to immune cells
CN118001254B (zh) * 2024-04-09 2024-07-26 北京悦康科创医药科技股份有限公司 靶向抗原呈递细胞的脂质组合物及其应用
WO2026017917A1 (en) * 2024-07-19 2026-01-22 Ethris Gmbh Rnas with reduced ribosomal frameshift expression products

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP2186528B1 (en) * 2007-08-06 2014-03-19 Senju Pharmaceutical Co., Ltd. Pharmaceutical containing hif-1 alpha and hif-2 alpha expression inhibitor
CA2816925C (en) * 2009-11-04 2023-01-10 The University Of British Columbia Nucleic acid-containing lipid particles and related methods
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
BR112018069417A2 (pt) 2016-04-22 2019-01-22 Biontech Rna Pharmaceuticals Gmbh método para fornecer rna fita simples e ssrna
IL303195A (en) * 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use

Also Published As

Publication number Publication date
JP2024535766A (ja) 2024-10-02
US20240408032A1 (en) 2024-12-12
EP4398882A1 (en) 2024-07-17
WO2023036960A1 (en) 2023-03-16

Similar Documents

Publication Publication Date Title
JP2023549266A (ja) Rnaを含むlnp組成物ならびにそれを調製、貯蔵および使用する方法
JP2023552678A (ja) 粒子およびmRNAを含む医薬組成物ならびにそれを調製および貯蔵する方法
CN118265522A (zh) 适用于治疗的基于脂质的rna制剂
JP7446527B2 (ja) コード核酸の治療可能性についての効力アッセイ
CN119156205A (zh) 用于施用rna的基于脂质的制剂
CN116829134A (zh) 包含rna的lnp组合物及其制备、储存和使用方法
WO2022218503A1 (en) Lnp compositions comprising rna and methods for preparing, storing and using the same
WO2023193892A1 (en) Nucleic acid compositions comprising an inorganic polyphosphate and methods for preparing, storing and using the same
JP2026508330A (ja) 多重コーディング核酸のリンカー配列効力アッセイ
US20240226132A1 (en) Rna compositions comprising a buffer substance and methods for preparing, storing and using the same
CN120712350A (zh) 用于药物用途的rna制剂
CN116669709A (zh) 包含颗粒和mRNA的药物组合物及其制备和储存的方法
WO2025223684A1 (en) Methods and compositions for stimulating immune response
WO2026041793A1 (en) Chimeric proteins comprising non-human protein domains for enhancing antigenicity
EP4408886A1 (en) Treatment involving non-immunogenic rna for antigen vaccination and pd-1 axis binding antagonists
CN117615752A (zh) 包含缓冲物质的rna组合物及其制备、储存和使用方法
HK40099059A (zh) 包含颗粒和mrna的药物组合物及其制备和储存的方法
HK40100852A (zh) 包含rna的lnp组合物及其制备、储存和使用方法

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination