JP2019503649A5 - - Google Patents

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Publication number
JP2019503649A5
JP2019503649A5 JP2018521281A JP2018521281A JP2019503649A5 JP 2019503649 A5 JP2019503649 A5 JP 2019503649A5 JP 2018521281 A JP2018521281 A JP 2018521281A JP 2018521281 A JP2018521281 A JP 2018521281A JP 2019503649 A5 JP2019503649 A5 JP 2019503649A5
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JP
Japan
Prior art keywords
nucleic acid
aav
sequence
acid variant
seq
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Application number
JP2018521281A
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English (en)
Japanese (ja)
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JP7088831B2 (ja
JP2019503649A (ja
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Priority claimed from PCT/US2016/059793 external-priority patent/WO2017075619A1/en
Publication of JP2019503649A publication Critical patent/JP2019503649A/ja
Publication of JP2019503649A5 publication Critical patent/JP2019503649A5/ja
Priority to JP2022093877A priority Critical patent/JP2022137029A/ja
Application granted granted Critical
Publication of JP7088831B2 publication Critical patent/JP7088831B2/ja
Priority to JP2024131729A priority patent/JP2024170416A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2018521281A 2015-10-30 2016-10-31 核酸変種、アデノ随伴ウイルスベクター及び医薬組成物 Active JP7088831B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022093877A JP2022137029A (ja) 2015-10-30 2022-06-09 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用
JP2024131729A JP2024170416A (ja) 2015-10-30 2024-08-08 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201562249001P 2015-10-30 2015-10-30
US62/249,001 2015-10-30
US201662331872P 2016-05-04 2016-05-04
US62/331,872 2016-05-04
US201662349532P 2016-06-13 2016-06-13
US62/349,532 2016-06-13
US201662357874P 2016-07-01 2016-07-01
US62/357,874 2016-07-01
PCT/US2016/059793 WO2017075619A1 (en) 2015-10-30 2016-10-31 Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022093877A Division JP2022137029A (ja) 2015-10-30 2022-06-09 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

Publications (3)

Publication Number Publication Date
JP2019503649A JP2019503649A (ja) 2019-02-14
JP2019503649A5 true JP2019503649A5 (enExample) 2019-12-05
JP7088831B2 JP7088831B2 (ja) 2022-06-21

Family

ID=58630834

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018521281A Active JP7088831B2 (ja) 2015-10-30 2016-10-31 核酸変種、アデノ随伴ウイルスベクター及び医薬組成物
JP2022093877A Pending JP2022137029A (ja) 2015-10-30 2022-06-09 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用
JP2024131729A Pending JP2024170416A (ja) 2015-10-30 2024-08-08 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022093877A Pending JP2022137029A (ja) 2015-10-30 2022-06-09 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用
JP2024131729A Pending JP2024170416A (ja) 2015-10-30 2024-08-08 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

Country Status (19)

Country Link
US (3) US20180312571A1 (enExample)
EP (1) EP3368560B1 (enExample)
JP (3) JP7088831B2 (enExample)
KR (1) KR20180090795A (enExample)
CN (1) CN108368164A (enExample)
AU (1) AU2016344030B2 (enExample)
BR (1) BR112018008766A2 (enExample)
CA (1) CA3002689A1 (enExample)
CL (1) CL2018001107A1 (enExample)
CO (1) CO2018005306A2 (enExample)
IL (1) IL258959B2 (enExample)
MX (2) MX2018005490A (enExample)
MY (1) MY190926A (enExample)
PE (2) PE20231949A1 (enExample)
PH (1) PH12018501055A1 (enExample)
RU (1) RU2745506C2 (enExample)
SA (1) SA518391463B1 (enExample)
SG (1) SG11201802972UA (enExample)
WO (1) WO2017075619A1 (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
HUE068603T2 (hu) 2015-06-23 2025-01-28 Childrens Hospital Philadelphia Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
BR112020001979A2 (pt) * 2017-08-01 2020-08-18 Spark Therapeutics, Inc. métodos de terapia genética do fator viii (fviii)
TWI904068B (zh) * 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
AR117427A1 (es) 2018-05-14 2021-08-04 Biomarin Pharm Inc Expresión estable de vectores de aav en sujetos jóvenes
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
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
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
BR112021009452A2 (pt) * 2018-11-16 2021-08-17 Spark Therapeutics, Inc. ensaio in vitro para detectar intensificadores e inibidores de transdução de vetor de vírus adeno-associados (aav) e/ou detectar ou quantificar anticorpos de ligação a anti-aav
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
JP7795357B2 (ja) 2019-03-13 2026-01-07 ジェネレーション バイオ カンパニー Ｆｖｉｉｉ治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用
US20220396610A1 (en) * 2019-11-01 2022-12-15 Freeline Therapeutics Limited Factor viii polypeptide
KR20220130174A (ko) * 2020-01-16 2022-09-26 다케다 야쿠힌 고교 가부시키가이샤 페닐케톤뇨증에 대한 아데노 연관 바이러스 기반 유전자 치료법
GB202009039D0 (en) 2020-06-15 2020-07-29 Univ Bristol Therapy
WO2023036054A2 (en) * 2021-09-08 2023-03-16 Inspirar Limited Composition and method for treating hemophilia
US20250282847A1 (en) * 2022-04-28 2025-09-11 Joint Stock Company "Biocad" Codon-optimized nucleic acid encoding the fviii-bdd
AU2023333051A1 (en) * 2022-08-31 2025-03-20 Hydrogene Therapeutics, Inc. Methods and compositions for hydrodynamic gene delivery
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2025164792A1 (ja) * 2024-02-02 2025-08-07 学校法人自治医科大学 高比活性／高分泌発現型の第ｖｉｉｉ因子改変体、高発現型の第ｖｉｉｉ因子コード核酸及びそれらの用途
WO2025180508A1 (en) * 2024-02-29 2025-09-04 Shanghai Vitalgen Biopharma Co., Ltd. Human factor viii variant expression cassettes and uses thereof for treating hemophilia a

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6093699A (en) 1987-07-09 2000-07-25 The University Of Manitoba Method for gene therapy involving suppression of an immune response
WO1994017810A1 (en) 1993-02-12 1994-08-18 The Wistar Institute Of Anatomy And Biology Recombinant cytomegalovirus vaccine
WO1994023744A1 (en) 1993-04-16 1994-10-27 The Wistar Institute Of Anatomy And Biology Recombinant cytomegalovirus vaccine
WO1995005864A1 (en) 1993-08-27 1995-03-02 Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Convection-enhanced drug delivery
AT401270B (de) 1994-09-26 1996-07-25 Immuno Ag Verfahren zur quantifizierung von genomischer dna
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
US6818439B1 (en) 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
EA001616B1 (ru) 1995-02-28 2001-06-25 Зе Риджентс Оф Зи Юнивесити Оф Кэлифоньэ Способ лечения болезни сердца, способ лечения недостаточности периферических сосудов и способ ограничения доставки и экспрессии трансгенной конструкции в определенном органе или структуре
AU2319497A (en) 1996-03-07 1997-09-22 Regents Of The University Of California, The Helper-free, totally defective adenovirus for gene therapy
US6458563B1 (en) * 1996-06-26 2002-10-01 Emory University Modified factor VIII
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
US7033994B2 (en) * 1999-12-24 2006-04-25 Juridical Foundation The Chemo-Sero-Therapeutic Research Institute Medicinal compositions for preventing and treating hemorrhagic diseases associating thrombopathy
MXPA03002256A (es) * 2000-09-19 2003-09-10 Univ Emory Factor viii modificado.
DE102004037611B4 (de) * 2004-08-03 2013-10-02 Geneart Ag Induzierbare Genexpression
GB0908515D0 (en) 2009-05-18 2009-06-24 Apitope Technology Bristol Ltd Peptide
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
DK2729161T3 (en) 2011-07-08 2019-04-08 Bioverativ Therapeutics Inc FACTOR VIII CHEMICAL AND HYBRID POLYPEPTIDES AND PROCEDURES FOR USE THEREOF
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV capsid proteins for nucleic acid transfer
NZ628014A (en) * 2012-02-15 2016-09-30 Biogen Ma Inc Recombinant factor viii proteins
RU2683497C2 (ru) 2012-04-18 2019-03-28 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
CA3209883A1 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
WO2015054439A2 (en) * 2013-10-08 2015-04-16 Haplomics, Inc. Hybrid factor viii polypeptides for use to treat hemophilia a
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
AU2015301598B2 (en) * 2014-08-13 2020-07-16 Liron Elkouby An improved expression cassette for packaging and expression of variant Factor VIII for the treatment of hemostasis disorders
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
BR112020001979A2 (pt) 2017-08-01 2020-08-18 Spark Therapeutics, Inc. métodos de terapia genética do fator viii (fviii)

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