JP2025505258A - 疾患の治療方法および／または予防方法に使用するための製剤 - Google Patents

疾患の治療方法および／または予防方法に使用するための製剤 Download PDF

Info

Publication number
JP2025505258A
JP2025505258A JP2024547657A JP2024547657A JP2025505258A JP 2025505258 A JP2025505258 A JP 2025505258A JP 2024547657 A JP2024547657 A JP 2024547657A JP 2024547657 A JP2024547657 A JP 2024547657A JP 2025505258 A JP2025505258 A JP 2025505258A
Authority
JP
Japan
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
JP2024547657A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025505258A5 (https=
Inventor
フォルカー フェリング
オリバー キール
イエルク カウフマン
アカンクシャ モガ
Original Assignee
パンテルナ セラピューティクス ゲーエムベーハー
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 パンテルナ セラピューティクス ゲーエムベーハー filed Critical パンテルナ セラピューティクス ゲーエムベーハー
Publication of JP2025505258A publication Critical patent/JP2025505258A/ja
Publication of JP2025505258A5 publication Critical patent/JP2025505258A5/ja
Pending legal-status Critical Current

Links

Images

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
    • A61K9/10—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
    • A61K9/0012—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
    • 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)
JP2024547657A 2022-02-14 2023-02-14 疾患の治療方法および／または予防方法に使用するための製剤 Pending JP2025505258A (ja)

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 (2)

Publication Number Publication Date
JP2025505258A true JP2025505258A (ja) 2025-02-21
JP2025505258A5 JP2025505258A5 (https=) 2026-02-18

Family

ID=80683166

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024547657A Pending JP2025505258A (ja) 2022-02-14 2023-02-14 疾患の治療方法および／または予防方法に使用するための製剤

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
CN118695875A (zh) 2024-09-24
US20240408014A1 (en) 2024-12-12
CA3243105A1 (en) 2023-08-17

Similar Documents

Publication Publication Date Title
JP7753190B2 (ja) 核酸の送達のための改善された脂質ナノ粒子
JP2025065390A (ja) メッセンジャーｒｎａを送達するための組成物及び方法
US20240350662A1 (en) Systems and methods for nucleic acid expression in vivo
WO2024119051A1 (en) Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
KR20220140584A (ko) 내피로의 치료 활성제의 전달을 위한 지질 조성물 및 이의 용도
Xu et al. Ligands located within a cholesterol domain enhance gene delivery to the target tissue
CA3266086A1 (en) IONIZABLE LIPIDS, LIPID NANOPARTICLES AND THEIR USES
JP2025505258A (ja) 疾患の治療方法および／または予防方法に使用するための製剤
JP2024505924A (ja) 核酸送達
WO2025090663A1 (en) Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
TW202530174A (zh) 用於遞送的脂質化合物及脂質奈米顆粒
JP2025540076A (ja) 核酸、イオン化可能な脂質、ステロール、脂質アンカーポリマー、及びヘルパー脂質を含む脂質ナノ粒子、それらの使用
KR20240072931A (ko) 히스티딘을 포함하는 지질 나노입자 및 그 제조방법
CN117460537A (zh) 核酸递送
US20260041785A1 (en) Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2026064512A1 (en) Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
KR20250092049A (ko) mRNA/LNP 백신의 면역원성 향상용 지질
EP4124345A1 (en) Nanoparticles for muscle delivery
TW202502302A (zh) 用於靶向樹突細胞之奈米遞送之方法及組合物
CN120289333A (zh) 一种醇胺多尾脂质及其制备方法和应用

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260209

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20260209