CN118695875A - 在用于治疗和/或预防疾病的方法中使用的制剂 - Google Patents

在用于治疗和/或预防疾病的方法中使用的制剂 Download PDF

Info

Publication number
CN118695875A
CN118695875A CN202380021541.XA CN202380021541A CN118695875A CN 118695875 A CN118695875 A CN 118695875A CN 202380021541 A CN202380021541 A CN 202380021541A CN 118695875 A CN118695875 A CN 118695875A
Authority
CN
China
Prior art keywords
lipid
formulation
mol
group
administration
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
CN202380021541.XA
Other languages
English (en)
Chinese (zh)
Inventor
V·菲灵
O·凯尔
J·卡夫曼
A·莫嘎
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.)
Panzelna Therapeutics Ltd
Original Assignee
Panzelna Therapeutics Ltd
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 Panzelna Therapeutics Ltd filed Critical Panzelna Therapeutics Ltd
Publication of CN118695875A publication Critical patent/CN118695875A/zh
Pending legal-status Critical Current

Links

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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
    • 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
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-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
    • 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
    • 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
    • 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
    • A61K9/0012—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Dermatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biomedical Technology (AREA)
  • Dispersion Chemistry (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202380021541.XA 2022-02-14 2023-02-14 在用于治疗和/或预防疾病的方法中使用的制剂 Pending CN118695875A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP22156604.5A EP4226949A1 (en) 2022-02-14 2022-02-14 Preparation for use in a method for the treatment and/or prevention of a disease
EP22156604.5 2022-02-14
PCT/EP2023/053639 WO2023152398A1 (en) 2022-02-14 2023-02-14 Preparation for use in a method for the treatment and/or prevention of a disease

Publications (1)

Publication Number Publication Date
CN118695875A true CN118695875A (zh) 2024-09-24

Family

ID=80683166

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380021541.XA Pending CN118695875A (zh) 2022-02-14 2023-02-14 在用于治疗和/或预防疾病的方法中使用的制剂

Country Status (8)

Country Link
US (1) US20240408014A1 (https=)
EP (2) EP4226949A1 (https=)
JP (1) JP2025505258A (https=)
KR (1) KR20240149887A (https=)
CN (1) CN118695875A (https=)
AU (1) AU2023217931A1 (https=)
CA (1) CA3243105A1 (https=)
WO (1) WO2023152398A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4681739A1 (en) 2024-07-19 2026-01-21 PROVIREX Genome Editing Therapies GmbH Lipid nanoparticles for direct delivery

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011119058A2 (en) * 2010-03-22 2011-09-29 Universidade De Coimbra F3-peptide targeted lipid-based nanoparticles useful for the treatment of angiogenesis-dependent diseases
JP2018509387A (ja) * 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
US20180200186A1 (en) * 2015-07-09 2018-07-19 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
EP3562510A4 (en) * 2016-12-30 2021-01-06 Genevant Sciences GmbH BRANCHED PEG MOLECULES AND ASSOCIATED COMPOSITIONS AND PROCEDURES

Also Published As

Publication number Publication date
EP4226949A1 (en) 2023-08-16
WO2023152398A1 (en) 2023-08-17
EP4479096A1 (en) 2024-12-25
KR20240149887A (ko) 2024-10-15
AU2023217931A1 (en) 2024-07-18
JP2025505258A (ja) 2025-02-21
US20240408014A1 (en) 2024-12-12
CA3243105A1 (en) 2023-08-17

Similar Documents

Publication Publication Date Title
KR102537540B1 (ko) 만노스를 포함하는 지질 나노입자 또는 이의 용도
JP2017165748A (ja) 薬物送達用の脂質ナノ粒子の生成方法
US20240350662A1 (en) Systems and methods for nucleic acid expression in vivo
US20250099393A1 (en) Ionizable lipids, lipid nanoparticles, and uses thereof
JP2025541727A (ja) 新規のポリグリセロールコンジュゲート脂質及びそれを含む脂質ナノ粒子組成物
WO2024086929A1 (en) Lipid nanoparticle formulations for anti-sense oligonucleotide delivery
US20240408014A1 (en) Preparation for use in a method for the treatment and/or prevention of a disease
JP2024505924A (ja) 核酸送達
CN120531894A (zh) 一种脂质纳米载体、药物制剂及制备方法和应用
KR20240072931A (ko) 히스티딘을 포함하는 지질 나노입자 및 그 제조방법
JP7585166B2 (ja) 物質送達キャリア及び組成物
WO2010067617A1 (ja) 脂質膜構造体
CN117460537A (zh) 核酸递送
WO2022229903A1 (en) Mrna delivery constructs and methods of using the same
US20260041785A1 (en) Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
US20230190653A1 (en) Method for reducing off-target uptake or accumulation of agents
Katas et al. Bionanocomposites for RNAi-based delivery
WO2024250095A1 (en) An intelligent charge switchable polymer-lipid hybrid system for in vivo delivery of molecules including rnas
JP2025532856A (ja) がん転移の治療のためのｒｎａ複合体及びナノ構造体

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