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
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- aav
- sequence
- acid variant
- seq
- 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.)
- Granted
Links
- 108020004707 nucleic acids Proteins 0.000 claims 14
- 150000007523 nucleic acids Chemical class 0.000 claims 14
- 102000039446 nucleic acids Human genes 0.000 claims 14
- 239000013607 AAV vector Substances 0.000 claims 12
- CTMZLDSMFCVUNX-VMIOUTBZSA-N cytidylyl-(3'->5')-guanosine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](OP(O)(=O)OC[C@@H]2[C@H]([C@@H](O)[C@@H](O2)N2C3=C(C(N=C(N)N3)=O)N=C2)O)[C@@H](CO)O1 CTMZLDSMFCVUNX-VMIOUTBZSA-N 0.000 claims 10
- 239000008194 pharmaceutical composition Substances 0.000 claims 9
- 210000000234 capsid Anatomy 0.000 claims 6
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 4
- 102000001690 Factor VIII Human genes 0.000 claims 3
- 108010054218 Factor VIII Proteins 0.000 claims 3
- 229960000301 factor viii Drugs 0.000 claims 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
- 229940104302 cytosine Drugs 0.000 claims 2
- 230000037430 deletion Effects 0.000 claims 2
- 238000012217 deletion Methods 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 108091092195 Intron Proteins 0.000 claims 1
- 150000001413 amino acids Chemical class 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 229940125721 immunosuppressive agent Drugs 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000004094 surface-active agent Substances 0.000 claims 1
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) | EP3368560A4 (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 (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| CA2990193A1 (en) | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| 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 |
| EP3491008A2 (en) | 2016-07-26 | 2019-06-05 | BioMarin Pharmaceutical 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) |
| WO2019032898A1 (en) * | 2017-08-09 | 2019-02-14 | Bioverativ Therapeutics Inc. | NUCLEIC ACID MOLECULES AND USES THEREOF |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| EP3790627A2 (en) | 2018-05-09 | 2021-03-17 | BioMarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| JP2021523198A (ja) | 2018-05-14 | 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 | 幼若対象におけるａａｖベクターの安定的発現 |
| 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 |
| SG11202101157VA (en) | 2018-08-09 | 2021-03-30 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| MX2021002198A (es) * | 2018-08-24 | 2021-07-15 | Spark Therapeutics Inc | Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos de uso. |
| EP3880219A4 (en) * | 2018-11-16 | 2022-08-17 | Spark Therapeutics, Inc. | IN VITRO ASSAY FOR DETECTING ENHANCERS AND INHIBITORS OF ADENO-ASSOCIATED VIRUS (AAV) VECTOR TRANSDUCTION AND FOR DETECTING OR QUANTIFYING ANTI-AAV BINDING ANTIBODIES |
| GB201900702D0 (en) * | 2019-01-18 | 2019-03-06 | Univ Bristol | Therapy |
| AU2020221340A1 (en) | 2019-02-15 | 2021-09-16 | Bayer Healthcare Llc | Gene editing for hemophilia A with improved Factor VIII expression |
| AU2020234713A1 (en) | 2019-03-13 | 2021-11-04 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics |
| CN114901686A (zh) * | 2019-11-01 | 2022-08-12 | 自由行疗法有限公司 | 因子viii多肽 |
| PE20231100A1 (es) * | 2020-01-16 | 2023-07-18 | Takeda Pharmaceuticals Co | Terapia genica basada en virus adenoasociados para la fenilcetonuria |
| 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 |
| MA68586A1 (fr) * | 2022-04-28 | 2025-04-30 | Joint Stock Company "Biocad" | Acide nucléique à codons optimisés codant pour fviii-bdd |
| CN120166940A (zh) * | 2022-08-31 | 2025-06-17 | 流体基因治疗有限公司 | 流体动力学基因递送的方法和组合物 |
| 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 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
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| 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 |
| CA2188575A1 (en) | 1995-02-28 | 1996-09-06 | H. Kirk Hammond | Gene transfer-mediated angiogenesis therapy |
| 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 |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| 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 |
| DE60042171D1 (de) * | 1999-12-24 | 2009-06-18 | Chemo Sero Therapeut Res Inst | Faktor viii oder von willebrand faktor zur behandlung von thrombopathie assoziierenden hämorrhagischen erkrankungen |
| 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 |
| KR102110736B1 (ko) | 2011-07-08 | 2020-05-14 | 바이오버라티브 테라퓨틱스 인크. | Viii 인자 키메라 및 하이브리드 폴리펩타이드, 그리고 이들의 사용 방법 |
| EP2748185A1 (en) | 2011-08-24 | 2014-07-02 | The Board of Trustees of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
| WO2013123457A1 (en) * | 2012-02-15 | 2013-08-22 | Biogen Idec Ma Inc. | Recombinant factor viii proteins |
| CA2870736C (en) | 2012-04-18 | 2021-11-02 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| 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 |
| PH12016500162B1 (en) | 2013-07-22 | 2024-02-21 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues |
| CA3178379A1 (en) | 2013-09-12 | 2015-03-19 | Biomarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
| 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 |
| CN107427557B (zh) * | 2014-08-13 | 2022-01-04 | 费城儿童医院 | 用于包装和表达变体因子viii以治疗血友病的改良表达组件 |
| CN115141836B (zh) | 2015-10-28 | 2024-08-27 | 桑格摩生物治疗股份有限公司 | 肝特异性构建体、因子viii表达盒及其使用方法 |
| 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) |
-
2016
- 2016-10-30 PE PE2023001716A patent/PE20231949A1/es unknown
- 2016-10-31 KR KR1020187015213A patent/KR20180090795A/ko active Pending
- 2016-10-31 AU AU2016344030A patent/AU2016344030B2/en active Active
- 2016-10-31 SG SG11201802972UA patent/SG11201802972UA/en unknown
- 2016-10-31 MX MX2018005490A patent/MX2018005490A/es unknown
- 2016-10-31 EP EP16861065.7A patent/EP3368560A4/en active Pending
- 2016-10-31 CN CN201680063183.9A patent/CN108368164A/zh active Pending
- 2016-10-31 BR BR112018008766-1A patent/BR112018008766A2/en active Search and Examination
- 2016-10-31 JP JP2018521281A patent/JP7088831B2/ja active Active
- 2016-10-31 CA CA3002689A patent/CA3002689A1/en active Pending
- 2016-10-31 PE PE2018000713A patent/PE20181168A1/es unknown
- 2016-10-31 IL IL258959A patent/IL258959B2/en unknown
- 2016-10-31 US US15/772,025 patent/US20180312571A1/en not_active Abandoned
- 2016-10-31 MY MYPI2018000612A patent/MY190926A/en unknown
- 2016-10-31 RU RU2018119710A patent/RU2745506C2/ru active
- 2016-10-31 WO PCT/US2016/059793 patent/WO2017075619A1/en not_active Ceased
-
2017
- 2017-03-17 US US15/462,660 patent/US11168124B2/en active Active
-
2018
- 2018-04-26 CL CL2018001107A patent/CL2018001107A1/es unknown
- 2018-04-29 SA SA518391463A patent/SA518391463B1/ar unknown
- 2018-04-30 PH PH12018501055A patent/PH12018501055A1/en unknown
- 2018-04-30 MX MX2023001600A patent/MX2023001600A/es unknown
- 2018-05-21 CO CONC2018/0005306A patent/CO2018005306A2/es unknown
-
2022
- 2022-06-09 JP JP2022093877A patent/JP2022137029A/ja active Pending
-
2024
- 2024-08-08 JP JP2024131729A patent/JP2024170416A/ja active Pending
- 2024-11-26 US US18/960,905 patent/US20250171521A1/en active Pending
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