JP2025509566A - Ａａｖ医薬製品の凍結乾燥製剤 - Google Patents

Ａａｖ医薬製品の凍結乾燥製剤 Download PDF

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Publication number
JP2025509566A
JP2025509566A JP2024554782A JP2024554782A JP2025509566A JP 2025509566 A JP2025509566 A JP 2025509566A JP 2024554782 A JP2024554782 A JP 2024554782A JP 2024554782 A JP2024554782 A JP 2024554782A JP 2025509566 A JP2025509566 A JP 2025509566A
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Japan
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concentration
pharmaceutical
formulation
reconstituted
pharmaceutical formulation
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Pending
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JP2024554782A
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English (en)
Japanese (ja)
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JP2025509566A5 (https=
Inventor
ディンヂィァン・リウ
リー・ジー
ヤオ・チェン
クゥアン－ユー．ライ
ヨナタン・ヴェルト
シャオイエン・ワン
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication of JP2025509566A publication Critical patent/JP2025509566A/ja
Publication of JP2025509566A5 publication Critical patent/JP2025509566A5/ja
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • 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
    • A61K48/0008—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
    • A61K9/0012—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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • 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
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • A61K9/1623—Sugars or sugar alcohols, e.g. lactose; Derivatives thereof; Homeopathic globules
    • 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
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1629—Organic macromolecular compounds
    • A61K9/1641—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poloxamers
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Inorganic Chemistry (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024554782A 2022-03-14 2023-03-13 Ａａｖ医薬製品の凍結乾燥製剤 Pending JP2025509566A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263319657P 2022-03-14 2022-03-14
US63/319,657 2022-03-14
PCT/US2023/015112 WO2023177626A1 (en) 2022-03-14 2023-03-13 Lyophilized formulations of aav drug products

Publications (2)

Publication Number Publication Date
JP2025509566A true JP2025509566A (ja) 2025-04-11
JP2025509566A5 JP2025509566A5 (https=) 2026-03-23

Family

ID=85979703

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024554782A Pending JP2025509566A (ja) 2022-03-14 2023-03-13 Ａａｖ医薬製品の凍結乾燥製剤

Country Status (11)

Country Link
US (1) US20230293724A1 (https=)
EP (1) EP4493162A1 (https=)
JP (1) JP2025509566A (https=)
KR (1) KR20250002229A (https=)
CN (1) CN119072302A (https=)
AR (1) AR128785A1 (https=)
AU (1) AU2023233503A1 (https=)
CA (1) CA3245651A1 (https=)
IL (1) IL315322A (https=)
TW (1) TW202406560A (https=)
WO (1) WO2023177626A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025076729A1 (zh) * 2023-10-11 2025-04-17 朗信启昇(苏州)生物制药有限公司 包含aav的药物组合物及其制备方法
WO2025114524A1 (en) * 2023-11-30 2025-06-05 Uniqure Biopharma B.V. Formulations for viral drug products
WO2025185606A1 (en) * 2024-03-04 2025-09-12 Nanjing Legend Biotech Co., Ltd. Composition for cryoprotection of viral vector or vlp
WO2025188316A1 (en) * 2024-03-08 2025-09-12 Jaguar Gene Therapy, Llc Aav pharmaceutical formulations and methods thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit
SG11202107645RA (en) * 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
TW202332458A (zh) * 2019-04-19 2023-08-16 美商銳進科斯生物股份有限公司 腺相關病毒載體調配物及方法
WO2021071835A1 (en) * 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
TW202144575A (zh) * 2020-04-03 2021-12-01 美商拜奧馬林製藥公司 使用ａａｖ及治療調配物之苯酮尿症治療

Also Published As

Publication number Publication date
US20230293724A1 (en) 2023-09-21
TW202406560A (zh) 2024-02-16
EP4493162A1 (en) 2025-01-22
AR128785A1 (es) 2024-06-12
AU2023233503A1 (en) 2024-09-19
KR20250002229A (ko) 2025-01-07
CA3245651A1 (en) 2023-09-21
WO2023177626A1 (en) 2023-09-21
IL315322A (en) 2024-10-01
CN119072302A (zh) 2024-12-03

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