TW202446416A - 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 - Google Patents

腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 Download PDF

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TW202446416A
TW202446416A TW113113785A TW113113785A TW202446416A TW 202446416 A TW202446416 A TW 202446416A TW 113113785 A TW113113785 A TW 113113785A TW 113113785 A TW113113785 A TW 113113785A TW 202446416 A TW202446416 A TW 202446416A
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fviii
concentration
aav
bases
administration
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TW113113785A
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Chinese (zh)
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史都華 邦廷
彼得 卡麥隆 克羅希
爾諾 龐格
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美商拜奧馬林製藥公司
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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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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    • G01N33/48—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
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    • C12N2710/16011—Herpesviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • G01N2333/015—Parvoviridae, e.g. feline panleukopenia virus, human Parvovirus

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TW113113785A 2015-09-24 2016-09-23 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 TW202446416A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
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

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TW202446416A true TW202446416A (zh) 2024-12-01

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TW113113785A TW202446416A (zh) 2015-09-24 2016-09-23 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
TW105130912A TWI756185B (zh) 2015-09-24 2016-09-23 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
TW111102320A TWI841908B (zh) 2015-09-24 2016-09-23 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物

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TW105130912A TWI756185B (zh) 2015-09-24 2016-09-23 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
TW111102320A TWI841908B (zh) 2015-09-24 2016-09-23 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物

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US (3) US10512675B2 (https=)
EP (1) EP3352787A1 (https=)
JP (3) JP2018535929A (https=)
KR (1) KR102805910B1 (https=)
CN (1) CN108778323A (https=)
AU (2) AU2016326602A1 (https=)
BR (1) BR112018006074A2 (https=)
CA (1) CA2999297A1 (https=)
CL (1) CL2018000740A1 (https=)
IL (2) IL297353B2 (https=)
MA (1) MA42934A (https=)
MX (1) MX2018003702A (https=)
PE (2) PE20241346A1 (https=)
PH (1) PH12018500594A1 (https=)
RU (2) RU2742352C2 (https=)
TW (3) TW202446416A (https=)
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Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
TW202446416A (zh) * 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7764129B2 (ja) 2017-07-10 2025-11-05 ユニキュアー アイピー ビー．ブイ． ヒトにおけるａａｖ遺伝子療法のための手段及び方法
PE20200722A1 (es) * 2017-08-01 2020-07-21 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii)
CN111936621B (zh) * 2018-02-05 2025-05-13 奥登茨治疗公司 转录调控元件和它的用途
US20210093735A1 (en) * 2018-04-26 2021-04-01 The University Of North Carolina At Chapel Hill Methods and compositions for treatment of hemophilia
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
TW202016298A (zh) * 2018-05-14 2020-05-01 美商拜奧馬林製藥公司 Aav 載體於幼年受試者中之穩定表現
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
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
US12091675B2 (en) * 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
US12497632B2 (en) 2018-08-03 2025-12-16 Sangamo Therapeutics, Inc. Clinical parameters by expression of factor VIII
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
SG11202108357PA (en) 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
SG11202109850SA (en) 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
EP3980549A4 (en) * 2019-06-05 2023-07-26 Orchard Therapeutics (Europe) Limited COMPOSITIONS AND METHODS FOR MODIFYING EUKARYOT CELLS
CN114375205A (zh) * 2019-06-20 2022-04-19 武田药品工业株式会社 用基于病毒的基因疗法进行治疗的方法
CN114929735A (zh) 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
AU2020395323A1 (en) * 2019-12-06 2022-06-30 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor VIII function
WO2021119357A2 (en) * 2019-12-12 2021-06-17 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
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
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
MX2023005074A (es) * 2020-11-02 2023-07-17 Regeneron Pharma Caracterizacion de impurezas en muestras de virus adenoasociado (aav) y composiciones de formulacion para estabilizar el aav.
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
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
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
ZA848495B (en) 1984-01-31 1985-09-25 Idaho Res Found Production of polypeptides in insect cells
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
AU4185699A (en) 1998-05-27 1999-12-13 Avigen, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
WO2006036502A2 (en) 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
CN101965409A (zh) 2007-11-01 2011-02-02 罗切斯特大学 具有增加的稳定性的重组因子viii
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
KR101337797B1 (ko) * 2010-07-14 2013-12-06 한미사이언스 주식회사 지속형 인간 성장 호르몬 결합체 액상 제제
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
US20140003880A1 (en) * 2012-06-30 2014-01-02 General Electric Company Ceramic matrix composite and metal attachment configurations
AU2013336601B2 (en) * 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
CN107530447A (zh) 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
TW202446416A (zh) * 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物

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