JP2018535929A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018535929A5 JP2018535929A5 JP2018515603A JP2018515603A JP2018535929A5 JP 2018535929 A5 JP2018535929 A5 JP 2018535929A5 JP 2018515603 A JP2018515603 A JP 2018515603A JP 2018515603 A JP2018515603 A JP 2018515603A JP 2018535929 A5 JP2018535929 A5 JP 2018535929A5
- Authority
- JP
- Japan
- Prior art keywords
- subject
- concentration
- pharmaceutical formulation
- item
- administered
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Withdrawn
Links
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 |
Applications Claiming Priority (7)
| 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 | ||
| 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021181755A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018535929A JP2018535929A (ja) | 2018-12-06 |
| JP2018535929A5 true JP2018535929A5 (https=) | 2019-10-31 |
Family
ID=57113749
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018515603A Withdrawn JP2018535929A (ja) | 2015-09-24 | 2016-09-23 | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 |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 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (18)
| Country | Link |
|---|---|
| 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=) |
| WO (1) | WO2017053677A1 (https=) |
| ZA (1) | ZA201801657B (https=) |
Families Citing this family (31)
| 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)
| 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 |
-
2016
- 2016-09-23 TW TW113113785A patent/TW202446416A/zh unknown
- 2016-09-23 RU RU2018114888A patent/RU2742352C2/ru active
- 2016-09-23 US US15/274,046 patent/US10512675B2/en active Active
- 2016-09-23 IL IL297353A patent/IL297353B2/en unknown
- 2016-09-23 JP JP2018515603A patent/JP2018535929A/ja not_active Withdrawn
- 2016-09-23 WO PCT/US2016/053269 patent/WO2017053677A1/en not_active Ceased
- 2016-09-23 MX MX2018003702A patent/MX2018003702A/es unknown
- 2016-09-23 IL IL257939A patent/IL257939B2/en unknown
- 2016-09-23 CA CA2999297A patent/CA2999297A1/en active Pending
- 2016-09-23 TW TW105130912A patent/TWI756185B/zh active
- 2016-09-23 BR BR112018006074A patent/BR112018006074A2/pt not_active Application Discontinuation
- 2016-09-23 MA MA042934A patent/MA42934A/fr unknown
- 2016-09-23 PE PE2023002034A patent/PE20241346A1/es unknown
- 2016-09-23 PE PE2018000419A patent/PE20181338A1/es unknown
- 2016-09-23 KR KR1020187009991A patent/KR102805910B1/ko active Active
- 2016-09-23 CN CN201680056519.9A patent/CN108778323A/zh active Pending
- 2016-09-23 EP EP16778589.8A patent/EP3352787A1/en active Pending
- 2016-09-23 RU RU2020126034A patent/RU2020126034A/ru unknown
- 2016-09-23 TW TW111102320A patent/TWI841908B/zh active
- 2016-09-23 AU AU2016326602A patent/AU2016326602A1/en not_active Abandoned
-
2018
- 2018-03-09 ZA ZA2018/01657A patent/ZA201801657B/en unknown
- 2018-03-19 PH PH12018500594A patent/PH12018500594A1/en unknown
- 2018-03-21 CL CL2018000740A patent/CL2018000740A1/es unknown
-
2019
- 2019-11-06 US US16/675,841 patent/US11690898B2/en active Active
-
2021
- 2021-11-01 AU AU2021261832A patent/AU2021261832B2/en active Active
- 2021-11-08 JP JP2021181755A patent/JP2022010141A/ja active Pending
-
2023
- 2023-05-26 US US18/324,594 patent/US20240058423A1/en active Pending
-
2024
- 2024-07-26 JP JP2024120967A patent/JP2024147801A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018535929A5 (https=) | ||
| RU2018114888A (ru) | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции | |
| US20230047111A1 (en) | Pharmaceutical formulations of tnf-alpha antibodies | |
| JP2021523198A5 (https=) | ||
| Lyden et al. | Phase 1 safety, tolerability and pharmacokinetics of 3K3A-APC in healthy adult volunteers | |
| JP2018138578A5 (https=) | ||
| JP2015519382A5 (https=) | ||
| JP2019515899A5 (https=) | ||
| JP2020537637A5 (https=) | ||
| Rodriguez et al. | Umbilical cord: an allogenic tissue for potential treatment of COVID-19 | |
| TWI745114B (zh) | 用於後天性血友病a之預防及/或治療之醫藥組成物、及包含該醫藥組成物之製品 | |
| JP6836696B1 (ja) | 後天性血友病ａの予防および／または治療に用いられる医薬組成物、および当該医薬組成物を含む製品 | |
| CN101199845B (zh) | 一种稳定的抗IgE人源化单抗制剂 | |
| JP2017533252A (ja) | Ｃ型肝炎のための長時間作用型医薬組成物 | |
| NZ740521B2 (en) | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same | |
| RU2023105585A (ru) | Стабильная экспрессия векторов на основе аденоассоциированного вируса у несовершеннолетних пациентов | |
| RU2020140634A (ru) | Стабильная экспрессия векторов на основе аденоассоциированного вируса у несовершеннолетних пациентов | |
| JP2022518354A5 (https=) | ||
| OA17126A (en) | Pharmaceutical formulations of TNF-alpha antibodies | |
| Jennings et al. | Effect of actinomycin D on the production of acute phase protein in the rabbit |