JP2018535929A5 - - Google Patents
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- 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
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- 239000008194 pharmaceutical composition Substances 0.000 claims description 46
- 102100026735 Coagulation factor VIII Human genes 0.000 claims description 36
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims description 36
- 241000700605 Viruses Species 0.000 claims description 30
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 24
- 108010054218 Factor VIII Proteins 0.000 claims description 18
- 102000001690 Factor VIII Human genes 0.000 claims description 18
- 239000003246 corticosteroid Substances 0.000 claims description 17
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 claims description 12
- CTKXFMQHOOWWEB-UHFFFAOYSA-N Ethylene oxide/propylene oxide copolymer Chemical compound CCCOC(C)COCCO CTKXFMQHOOWWEB-UHFFFAOYSA-N 0.000 claims description 12
- 229930195725 Mannitol Natural products 0.000 claims description 12
- 210000000234 capsid Anatomy 0.000 claims description 12
- 229940061607 dibasic sodium phosphate Drugs 0.000 claims description 12
- BNIILDVGGAEEIG-UHFFFAOYSA-L disodium hydrogen phosphate Chemical compound [Na+].[Na+].OP([O-])([O-])=O BNIILDVGGAEEIG-UHFFFAOYSA-L 0.000 claims description 12
- 239000000594 mannitol Substances 0.000 claims description 12
- 235000010355 mannitol Nutrition 0.000 claims description 12
- 229920001993 poloxamer 188 Polymers 0.000 claims description 12
- 229940044519 poloxamer 188 Drugs 0.000 claims description 12
- 210000002966 serum Anatomy 0.000 claims description 12
- 239000011780 sodium chloride Substances 0.000 claims description 12
- BBMHARZCALWXSL-UHFFFAOYSA-M sodium dihydrogenphosphate monohydrate Chemical compound O.[Na+].OP(O)([O-])=O BBMHARZCALWXSL-UHFFFAOYSA-M 0.000 claims description 12
- 201000003542 Factor VIII deficiency Diseases 0.000 claims description 10
- 208000009292 Hemophilia A Diseases 0.000 claims description 10
- 208000032843 Hemorrhage Diseases 0.000 claims description 7
- 208000034158 bleeding Diseases 0.000 claims description 7
- 230000000740 bleeding effect Effects 0.000 claims description 7
- 239000000825 pharmaceutical preparation Substances 0.000 claims description 7
- 230000000694 effects Effects 0.000 claims description 4
- 239000004067 bulking agent Substances 0.000 claims description 2
- 239000007951 isotonicity adjuster Substances 0.000 claims description 2
- 239000007788 liquid Substances 0.000 claims description 2
- 238000004904 shortening Methods 0.000 claims description 2
- 239000004094 surface-active agent Substances 0.000 claims description 2
- 229960001334 corticosteroids Drugs 0.000 claims 2
- 150000003431 steroids Chemical class 0.000 claims 2
- 229960000301 factor viii Drugs 0.000 claims 1
- 238000009472 formulation Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 61
- 238000001990 intravenous administration Methods 0.000 description 3
- 208000024891 symptom Diseases 0.000 description 1
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 (cg-RX-API-DMAC7.html) | 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)
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| EP3352787A1 (en) * | 2015-09-24 | 2018-08-01 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same |
| AU2016343887B2 (en) | 2015-10-28 | 2023-04-06 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| KR20230161535A (ko) | 2016-07-26 | 2023-11-27 | 바이오마린 파머수티컬 인크. | 신규 아데노-관련 바이러스 캡시드 단백질 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| AU2018299865A1 (en) * | 2017-07-10 | 2020-01-30 | Uniqure Ip B.V. | Means and methods for AAV gene therapy in humans |
| AU2018312565A1 (en) * | 2017-08-01 | 2020-02-27 | Spark Therapeutics, Inc. | Factor VIII (FVIII) gene therapy methods |
| EP3749756A4 (en) * | 2018-02-05 | 2021-12-08 | Audentes Therapeutics, Inc. | ELEMENTS OF TRANSCRIPTION REGULATION AND ASSOCIATED USES |
| WO2019210187A1 (en) * | 2018-04-26 | 2019-10-31 | The University Of North Carolina At Chapel Hill | Methods and compositions for treatment of hemophilia |
| AU2019265560A1 (en) | 2018-05-09 | 2020-11-26 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| 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 |
| AR117427A1 (es) * | 2018-05-14 | 2021-08-04 | Biomarin Pharm Inc | Expresión estable de vectores de aav en sujetos jóvenes |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| CA3106590A1 (en) * | 2018-07-16 | 2020-01-23 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| MX2021001375A (es) * | 2018-08-03 | 2021-04-19 | Sangamo Therapeutics Inc | Parametros clinicos mejorados por la expresion del factor viii. |
| EP3840775A4 (en) * | 2018-08-24 | 2022-07-06 | Spark Therapeutics, Inc. | OPTIMIZED PROMOTER SEQUENCES, INTRON-FREE EXPRESSION CONSTRUCTS AND METHODS OF USE |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CA3129532A1 (en) | 2019-02-15 | 2020-08-20 | 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 |
| WO2020247814A1 (en) * | 2019-06-05 | 2020-12-10 | Orchard Therapeutics (Europe) Limited | Compositions and methods for modifying eukaryotic cells |
| 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 |
| WO2021084277A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Transcription regulatory elements |
| AU2020395323A1 (en) * | 2019-12-06 | 2022-06-30 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating factor VIII function |
| EP4073106A2 (en) * | 2019-12-12 | 2022-10-19 | Takeda Pharmaceutical Company Limited | 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 |
| MX2023005041A (es) | 2020-11-02 | 2023-05-17 | Biomarin Pharm Inc | Proceso para enriquecer virus adenoasociado. |
| CN115554418B (zh) | 2022-11-22 | 2023-04-14 | 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 |
| 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 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
| DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| CA2329143A1 (en) | 1998-05-27 | 1999-12-02 | Cell Genesys, 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 |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| ES2381796T3 (es) | 2004-09-22 | 2012-05-31 | St. Jude Children's Research Hospital | Expresión mejorada de Factor IX en vectores de terapia génica |
| 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 |
| CN104152489A (zh) | 2007-11-01 | 2014-11-19 | 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 |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| 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 |
| WO2014064277A1 (en) * | 2012-10-26 | 2014-05-01 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| RS61039B1 (sr) * | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| KR102863734B1 (ko) | 2015-02-10 | 2025-09-25 | 젠자임 코포레이션 | 선조체 및 피질로의 바이러스 입자의 향상된 전달 |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| EP3352787A1 (en) * | 2015-09-24 | 2018-08-01 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same |
-
2016
- 2016-09-23 EP EP16778589.8A patent/EP3352787A1/en active Pending
- 2016-09-23 US US15/274,046 patent/US10512675B2/en active Active
- 2016-09-23 CN CN201680056519.9A patent/CN108778323A/zh active Pending
- 2016-09-23 IL IL297353A patent/IL297353B1/en unknown
- 2016-09-23 RU RU2018114888A patent/RU2742352C2/ru active
- 2016-09-23 TW TW105130912A patent/TWI756185B/zh active
- 2016-09-23 TW TW111102320A patent/TWI841908B/zh active
- 2016-09-23 WO PCT/US2016/053269 patent/WO2017053677A1/en not_active Ceased
- 2016-09-23 JP JP2018515603A patent/JP2018535929A/ja not_active Withdrawn
- 2016-09-23 AU AU2016326602A patent/AU2016326602A1/en not_active Abandoned
- 2016-09-23 RU RU2020126034A patent/RU2020126034A/ru unknown
- 2016-09-23 PE PE2023002034A patent/PE20241346A1/es unknown
- 2016-09-23 IL IL257939A patent/IL257939B2/en unknown
- 2016-09-23 MA MA042934A patent/MA42934A/fr unknown
- 2016-09-23 CA CA2999297A patent/CA2999297A1/en active Pending
- 2016-09-23 KR KR1020187009991A patent/KR102805910B1/ko active Active
- 2016-09-23 BR BR112018006074A patent/BR112018006074A2/pt not_active Application Discontinuation
- 2016-09-23 TW TW113113785A patent/TW202446416A/zh unknown
- 2016-09-23 MX MX2018003702A patent/MX2018003702A/es unknown
- 2016-09-23 PE PE2018000419A patent/PE20181338A1/es unknown
-
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
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