JP2020533276A5 - - Google Patents
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- Publication number
- JP2020533276A5 JP2020533276A5 JP2020505478A JP2020505478A JP2020533276A5 JP 2020533276 A5 JP2020533276 A5 JP 2020533276A5 JP 2020505478 A JP2020505478 A JP 2020505478A JP 2020505478 A JP2020505478 A JP 2020505478A JP 2020533276 A5 JP2020533276 A5 JP 2020533276A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- raav vector
- nucleic acid
- aav
- human
- 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
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762540053P | 2017-08-01 | 2017-08-01 | |
| US62/540,053 | 2017-08-01 | ||
| US201762583890P | 2017-11-09 | 2017-11-09 | |
| US62/583,890 | 2017-11-09 | ||
| US201762596535P | 2017-12-08 | 2017-12-08 | |
| US201762596670P | 2017-12-08 | 2017-12-08 | |
| US62/596,535 | 2017-12-08 | ||
| US62/596,670 | 2017-12-08 | ||
| PCT/US2018/044892 WO2019028192A1 (en) | 2017-08-01 | 2018-08-01 | METHODS OF GENE THERAPY TARGETING FACTOR VIII (FVIII) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020533276A JP2020533276A (ja) | 2020-11-19 |
| JP2020533276A5 true JP2020533276A5 (enExample) | 2021-11-25 |
| JP7499174B2 JP7499174B2 (ja) | 2024-06-13 |
Family
ID=65234171
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020505478A Active JP7499174B2 (ja) | 2017-08-01 | 2018-08-01 | 第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法 |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20200237930A1 (enExample) |
| EP (1) | EP3661541A4 (enExample) |
| JP (1) | JP7499174B2 (enExample) |
| KR (2) | KR20200066289A (enExample) |
| CN (1) | CN111163796A (enExample) |
| AU (2) | AU2018312565A1 (enExample) |
| BR (1) | BR112020001979A2 (enExample) |
| CA (1) | CA3071519A1 (enExample) |
| CL (1) | CL2020000295A1 (enExample) |
| CO (1) | CO2020002283A2 (enExample) |
| IL (1) | IL272373A (enExample) |
| MX (1) | MX2020001402A (enExample) |
| MY (1) | MY208862A (enExample) |
| PE (1) | PE20200722A1 (enExample) |
| PH (1) | PH12020500239A1 (enExample) |
| SG (1) | SG11202000650YA (enExample) |
| WO (1) | WO2019028192A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| 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 |
| CA3087344A1 (en) | 2018-01-05 | 2019-07-11 | Platelet Biogenesis, Inc. | Compositions and methods for producing megakaryocytes |
| JP2021529770A (ja) | 2018-06-29 | 2021-11-04 | プレートレット バイオジェネシス， インコーポレイテッド | 薬物送達のための組成物およびその使用方法 |
| AU2019314513B2 (en) * | 2018-08-03 | 2026-04-30 | Sangamo Therapeutics, Inc. | Improved clinical parameters by expression of factor viii |
| SG11202108357PA (en) | 2019-02-15 | 2021-08-30 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| KR20210151785A (ko) | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
| CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
| WO2021084276A2 (en) * | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Factor viii construct |
| AU2020395323A1 (en) * | 2019-12-06 | 2022-06-30 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating factor VIII function |
| CN114989307B (zh) * | 2022-05-11 | 2023-08-01 | 华兰生物工程股份有限公司 | 一种重组人凝血因子Ⅷ-Fc融合蛋白及制备方法 |
| 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 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| JP2003511082A (ja) * | 1999-10-12 | 2003-03-25 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法 |
| ES2813698T3 (es) * | 2013-09-12 | 2021-03-24 | Biomarin Pharm Inc | Vectores de AAV que comprenden un gen que codifica el factor VIII |
| SG11201701033QA (en) * | 2014-08-13 | 2017-03-30 | Philadelphia Children Hospital | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
| CN115094062A (zh) * | 2015-04-16 | 2022-09-23 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| JP2018535929A (ja) * | 2015-09-24 | 2018-12-06 | バイオマリン ファーマシューティカル インコーポレイテッド | 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 |
| US10143760B2 (en) * | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| 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 |
| US20190144524A1 (en) * | 2016-01-14 | 2019-05-16 | The Children's Hospital Of Philadelphia | Factor viii variants, nucleic acid sequences, and methods and uses for treatment of hemostasis disorders |
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2018
- 2018-08-01 US US16/635,957 patent/US20200237930A1/en not_active Abandoned
- 2018-08-01 KR KR1020207005859A patent/KR20200066289A/ko not_active Ceased
- 2018-08-01 AU AU2018312565A patent/AU2018312565A1/en not_active Abandoned
- 2018-08-01 JP JP2020505478A patent/JP7499174B2/ja active Active
- 2018-08-01 MY MYPI2020000557A patent/MY208862A/en unknown
- 2018-08-01 PE PE2020000180A patent/PE20200722A1/es unknown
- 2018-08-01 CN CN201880064058.9A patent/CN111163796A/zh active Pending
- 2018-08-01 CA CA3071519A patent/CA3071519A1/en active Pending
- 2018-08-01 BR BR112020001979-8A patent/BR112020001979A2/pt unknown
- 2018-08-01 KR KR1020247034886A patent/KR20240160648A/ko active Pending
- 2018-08-01 SG SG11202000650YA patent/SG11202000650YA/en unknown
- 2018-08-01 EP EP18840279.6A patent/EP3661541A4/en active Pending
- 2018-08-01 MX MX2020001402A patent/MX2020001402A/es unknown
- 2018-08-01 WO PCT/US2018/044892 patent/WO2019028192A1/en not_active Ceased
-
2020
- 2020-01-30 IL IL272373A patent/IL272373A/en unknown
- 2020-01-31 CL CL2020000295A patent/CL2020000295A1/es unknown
- 2020-01-31 PH PH12020500239A patent/PH12020500239A1/en unknown
- 2020-02-28 CO CONC2020/0002283A patent/CO2020002283A2/es unknown
-
2024
- 2024-09-03 AU AU2024219336A patent/AU2024219336A1/en active Pending