JP2018535929A - アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 - Google Patents

アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 Download PDF

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JP2018535929A
JP2018535929A JP2018515603A JP2018515603A JP2018535929A JP 2018535929 A JP2018535929 A JP 2018535929A JP 2018515603 A JP2018515603 A JP 2018515603A JP 2018515603 A JP2018515603 A JP 2018515603A JP 2018535929 A JP2018535929 A JP 2018535929A
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fviii
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concentration
aav
bases
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JP2018535929A5 (cg-RX-API-DMAC7.html
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スチュアート バンティング，
スチュアート バンティング，
ピーター キャメロン コロシ，
ピーター キャメロン コロシ，
エルノ パンゴール，
エルノ パンゴール，
Original Assignee
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
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
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Publication of JP2018535929A publication Critical patent/JP2018535929A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • 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
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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16041—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • G01N2333/01—DNA viruses
    • G01N2333/015—Parvoviridae, e.g. feline panleukopenia virus, human Parvovirus

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JP2018515603A 2015-09-24 2016-09-23 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 Withdrawn JP2018535929A (ja)

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JP2021181755A JP2022010141A (ja) 2015-09-24 2021-11-08 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤
JP2024120967A JP2024147801A (ja) 2015-09-24 2024-07-26 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤

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US201562232242P 2015-09-24 2015-09-24
US62/232,242 2015-09-24
US201662323182P 2016-04-15 2016-04-15
US62/323,182 2016-04-15
US201662365544P 2016-07-22 2016-07-22
US62/365,544 2016-07-22
PCT/US2016/053269 WO2017053677A1 (en) 2015-09-24 2016-09-23 Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same

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JP2018515603A Withdrawn JP2018535929A (ja) 2015-09-24 2016-09-23 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤
JP2021181755A Pending JP2022010141A (ja) 2015-09-24 2021-11-08 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤
JP2024120967A Pending JP2024147801A (ja) 2015-09-24 2024-07-26 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤

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JP2024120967A Pending JP2024147801A (ja) 2015-09-24 2024-07-26 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤

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JP2023505208A (ja) * 2019-12-06 2023-02-08 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 第ｖｉｉｉ因子の機能を調節するための組成物および方法
JP2023506171A (ja) * 2019-12-12 2023-02-15 武田薬品工業株式会社 発現が上昇した組換えｆｖｉｉｉバリアントをコードするウイルスベクターを使用する、血友病ａの遺伝子療法

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TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
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JP2022537555A (ja) * 2019-06-20 2022-08-26 武田薬品工業株式会社 ウイルスベースの遺伝子療法による治療方法
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KR102167829B1 (ko) * 2020-02-10 2020-10-20 주식회사 이노테라피 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
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CN119015447A (zh) * 2023-09-01 2024-11-26 康霖生物科技（杭州）有限公司 一种缓冲系统及其相应液体制剂及其用途

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