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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- Japan
- Prior art keywords
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- pharmaceutical formulation
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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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3352787A1 (OSRAM) |
| JP (3) | JP2018535929A (OSRAM) |
| KR (1) | KR102805910B1 (OSRAM) |
| CN (1) | CN108778323A (OSRAM) |
| AU (2) | AU2016326602A1 (OSRAM) |
| BR (1) | BR112018006074A2 (OSRAM) |
| CA (1) | CA2999297A1 (OSRAM) |
| CL (1) | CL2018000740A1 (OSRAM) |
| IL (2) | IL257939B2 (OSRAM) |
| MA (1) | MA42934A (OSRAM) |
| MX (1) | MX2018003702A (OSRAM) |
| PE (2) | PE20241346A1 (OSRAM) |
| PH (1) | PH12018500594A1 (OSRAM) |
| RU (2) | RU2020126034A (OSRAM) |
| TW (3) | TWI841908B (OSRAM) |
| WO (1) | WO2017053677A1 (OSRAM) |
| ZA (1) | ZA201801657B (OSRAM) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3368664A4 (en) | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| PL3652326T3 (pl) | 2017-07-10 | 2025-02-24 | Uniqure Ip B.V. | Środki i sposoby terapii genowej aav u ludzi |
| MY208862A (en) * | 2017-08-01 | 2025-06-04 | Spark Therapeutics Inc | Factor viii (fviii) gene therapy methods |
| US12296026B2 (en) * | 2018-02-05 | 2025-05-13 | Astellas Gene Therapies, Inc. | Transcription regulatory elements and uses thereof |
| EP3810647A4 (en) * | 2018-04-26 | 2022-08-17 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR THE TREATMENT OF HEMOPHILIA |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | 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 |
| KR20210008491A (ko) * | 2018-05-14 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 청소년 대상체에서 aav 벡터의 안정한 발현 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| 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 |
| MX2021001375A (es) * | 2018-08-03 | 2021-04-19 | Sangamo Therapeutics Inc | Parametros clinicos mejorados por la expresion del factor viii. |
| WO2020041773A1 (en) * | 2018-08-24 | 2020-02-27 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| MA54951A (fr) | 2019-02-15 | 2021-12-22 | Bayer Healthcare Llc | Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée |
| MA55313A (fr) | 2019-03-13 | 2022-01-19 | Generation Bio Co | Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii |
| JP2022534798A (ja) * | 2019-06-05 | 2022-08-03 | オーチャード セラピューティクス（ヨーロッパ）リミテッド | 真核細胞を修飾するための組成物及び方法 |
| US20200405883A1 (en) * | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| WO2021084277A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Transcription regulatory elements |
| US20220403005A1 (en) * | 2019-12-06 | 2022-12-22 | 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 |
| WO2025045082A1 (zh) * | 2023-09-01 | 2025-03-06 | 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 |
|---|---|---|---|---|
| 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 |
| 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 |
| AU4185699A (en) | 1998-05-27 | 1999-12-13 | Avigen, Inc. | Adeno-associated viral vector-mediated expression of factor viii activity |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus 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 |
| ES2436741T3 (es) | 2004-09-22 | 2014-01-07 | 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 |
| KR20100113478A (ko) | 2007-11-01 | 2010-10-21 | 유니버시티 오브 로체스터 | 증가된 안정성을 갖는 재조합 인자 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 |
| JP6373763B2 (ja) | 2012-02-17 | 2018-08-22 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| 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 |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| SI3044231T1 (sl) * | 2013-09-12 | 2020-12-31 | Biomarin Pharmaceutical Inc. | Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII |
| SG11201706445SA (en) | 2015-02-10 | 2017-09-28 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
-
2016
- 2016-09-23 RU RU2020126034A patent/RU2020126034A/ru unknown
- 2016-09-23 MX MX2018003702A patent/MX2018003702A/es unknown
- 2016-09-23 WO PCT/US2016/053269 patent/WO2017053677A1/en not_active Ceased
- 2016-09-23 CA CA2999297A patent/CA2999297A1/en active Pending
- 2016-09-23 AU AU2016326602A patent/AU2016326602A1/en not_active Abandoned
- 2016-09-23 PE PE2023002034A patent/PE20241346A1/es unknown
- 2016-09-23 KR KR1020187009991A patent/KR102805910B1/ko active Active
- 2016-09-23 RU RU2018114888A patent/RU2742352C2/ru active
- 2016-09-23 IL IL257939A patent/IL257939B2/en unknown
- 2016-09-23 MA MA042934A patent/MA42934A/fr unknown
- 2016-09-23 IL IL297353A patent/IL297353B1/en unknown
- 2016-09-23 TW TW111102320A patent/TWI841908B/zh active
- 2016-09-23 TW TW105130912A patent/TWI756185B/zh active
- 2016-09-23 CN CN201680056519.9A patent/CN108778323A/zh active Pending
- 2016-09-23 TW TW113113785A patent/TW202446416A/zh unknown
- 2016-09-23 PE PE2018000419A patent/PE20181338A1/es unknown
- 2016-09-23 US US15/274,046 patent/US10512675B2/en active Active
- 2016-09-23 BR BR112018006074A patent/BR112018006074A2/pt not_active Application Discontinuation
- 2016-09-23 EP EP16778589.8A patent/EP3352787A1/en active Pending
- 2016-09-23 JP JP2018515603A patent/JP2018535929A/ja not_active Withdrawn
-
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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