JP2024500133A - アデノ随伴ウイルスベクターを含む改善された医薬組成物 - Google Patents

アデノ随伴ウイルスベクターを含む改善された医薬組成物 Download PDF

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JP2024500133A
JP2024500133A JP2023537242A JP2023537242A JP2024500133A JP 2024500133 A JP2024500133 A JP 2024500133A JP 2023537242 A JP2023537242 A JP 2023537242A JP 2023537242 A JP2023537242 A JP 2023537242A JP 2024500133 A JP2024500133 A JP 2024500133A
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pharmaceutical composition
composition according
formulation
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chloride
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JP2024500133A5 (https=
Inventor
アイリーン コナー，ジェシカ
アン クロフォード，リンジー
ダミッツ，ロバート
マイケル デイビス，ブレンダン
マイケル ホッジ，コディ
ザ セカンド，マイケル リーランド キメル
ウィラード，ティハミ クレシ
ラムジー，フィリップ
ジョセフ ソーン，ダニエル
リー ヤング，アンソニー
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Inorganic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2023537242A 2020-12-18 2021-12-17 アデノ随伴ウイルスベクターを含む改善された医薬組成物 Pending JP2024500133A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063127826P 2020-12-18 2020-12-18
US63/127,826 2020-12-18
PCT/US2021/064222 WO2022133324A1 (en) 2020-12-18 2021-12-17 Improved pharmaceutical compositions containing adeno-associated viral vector

Publications (2)

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JP2024500133A true JP2024500133A (ja) 2024-01-04
JP2024500133A5 JP2024500133A5 (https=) 2026-04-02

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JP2023537242A Pending JP2024500133A (ja) 2020-12-18 2021-12-17 アデノ随伴ウイルスベクターを含む改善された医薬組成物

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US (1) US20240066146A1 (https=)
EP (1) EP4262754A1 (https=)
JP (1) JP2024500133A (https=)
KR (1) KR20230122076A (https=)
CN (1) CN116710149A (https=)
AU (1) AU2021403118A1 (https=)
CA (1) CA3204462A1 (https=)
IL (1) IL303737A (https=)
MX (1) MX2023006471A (https=)
WO (1) WO2022133324A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024505060A (ja) * 2021-01-27 2024-02-02 エイラ・セラピューティクス・インコーポレイテッド Ｐｎｍａ２ベースのキャプシド及びその使用
US20240307553A1 (en) * 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CN116350801A (zh) * 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
EP4651883A1 (en) * 2023-01-19 2025-11-26 uniQure biopharma B.V. Pharmaceutical formulations of gene delivery vehicles
WO2025045083A1 (zh) * 2023-09-01 2025-03-06 康霖生物科技（杭州）有限公司 一种液体制剂及其用途
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
WO2025108407A2 (en) * 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) * 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008540277A (ja) * 2005-05-17 2008-11-20 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム 密閉システムおよびバイアルの充填方法
JP2016534739A (ja) * 2013-09-12 2016-11-10 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルス第ｖｉｉｉ因子ベクター
WO2020028830A1 (en) * 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
JP2020072675A (ja) * 2011-09-19 2020-05-14 アクソン ニューロサイエンス エスエー アルツハイマー病におけるタウ媒介性病理のタンパク質ベースの治療および診断
WO2020102369A1 (en) * 2018-11-14 2020-05-22 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2020102434A2 (en) * 2018-11-13 2020-05-22 Sio2 Medical Products, Inc. Polymer vials with substantially flat bottoms and injection stretch blow molding methods for making the same

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE226449T1 (de) * 1998-12-03 2002-11-15 Avigen Inc Hilfsstoffe zur verwendung in adeno-assoziiertes virus enthaltenden pharmazeutischen zubereitungen und damit hergestellte pharmazeutische zubereitungen
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
CN112770812A (zh) * 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
AU2019428629A1 (en) 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
WO2021071835A1 (en) * 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008540277A (ja) * 2005-05-17 2008-11-20 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム 密閉システムおよびバイアルの充填方法
JP2020072675A (ja) * 2011-09-19 2020-05-14 アクソン ニューロサイエンス エスエー アルツハイマー病におけるタウ媒介性病理のタンパク質ベースの治療および診断
JP2016534739A (ja) * 2013-09-12 2016-11-10 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルス第ｖｉｉｉ因子ベクター
WO2020028830A1 (en) * 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
WO2020102434A2 (en) * 2018-11-13 2020-05-22 Sio2 Medical Products, Inc. Polymer vials with substantially flat bottoms and injection stretch blow molding methods for making the same
WO2020102369A1 (en) * 2018-11-14 2020-05-22 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses

Also Published As

Publication number Publication date
MX2023006471A (es) 2023-08-11
AU2021403118A9 (en) 2024-04-18
KR20230122076A (ko) 2023-08-22
CA3204462A1 (en) 2022-06-23
IL303737A (en) 2023-08-01
WO2022133324A1 (en) 2022-06-23
CN116710149A (zh) 2023-09-05
AU2021403118A1 (en) 2023-06-22
US20240066146A1 (en) 2024-02-29
EP4262754A1 (en) 2023-10-25

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