JP2023115348A5 - - Google Patents
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- JP2023115348A5 JP2023115348A5 JP2023108132A JP2023108132A JP2023115348A5 JP 2023115348 A5 JP2023115348 A5 JP 2023115348A5 JP 2023108132 A JP2023108132 A JP 2023108132A JP 2023108132 A JP2023108132 A JP 2023108132A JP 2023115348 A5 JP2023115348 A5 JP 2023115348A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- seq
- identity
- nucleotide sequence
- polynucleotide according
- 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
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- 239000002157 polynucleotide Substances 0.000 claims 24
- 102000040430 polynucleotide Human genes 0.000 claims 24
- 108091033319 polynucleotide Proteins 0.000 claims 24
- 239000002773 nucleotide Substances 0.000 claims 13
- 125000003729 nucleotide group Chemical group 0.000 claims 13
- 229920001184 polypeptide Polymers 0.000 claims 7
- 102000004196 processed proteins & peptides Human genes 0.000 claims 7
- 108090000765 processed proteins & peptides Proteins 0.000 claims 7
- 102000001690 Factor VIII Human genes 0.000 claims 5
- 108010054218 Factor VIII Proteins 0.000 claims 5
- 229960000301 factor viii Drugs 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 239000002245 particle Substances 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 208000031220 Hemophilia Diseases 0.000 claims 2
- 208000009292 Hemophilia A Diseases 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 102000004961 Furin Human genes 0.000 claims 1
- 108090001126 Furin Proteins 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562255317P | 2015-11-13 | 2015-11-13 | |
| US62/255,317 | 2015-11-13 | ||
| JP2020132079A JP7069258B2 (ja) | 2015-11-13 | 2020-08-04 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2022075864A JP7307836B2 (ja) | 2015-11-13 | 2022-05-02 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022075864A Division JP7307836B2 (ja) | 2015-11-13 | 2022-05-02 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023115348A JP2023115348A (ja) | 2023-08-18 |
| JP2023115348A5 true JP2023115348A5 (OSRAM) | 2023-11-17 |
| JP7631425B2 JP7631425B2 (ja) | 2025-02-18 |
Family
ID=57539605
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018524747A Active JP6768798B2 (ja) | 2015-11-13 | 2016-11-11 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2020132079A Active JP7069258B2 (ja) | 2015-11-13 | 2020-08-04 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2022075864A Active JP7307836B2 (ja) | 2015-11-13 | 2022-05-02 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2023108132A Active JP7631425B2 (ja) | 2015-11-13 | 2023-06-30 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018524747A Active JP6768798B2 (ja) | 2015-11-13 | 2016-11-11 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2020132079A Active JP7069258B2 (ja) | 2015-11-13 | 2020-08-04 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| JP2022075864A Active JP7307836B2 (ja) | 2015-11-13 | 2022-05-02 | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
Country Status (18)
| Country | Link |
|---|---|
| US (4) | US10189888B2 (OSRAM) |
| EP (1) | EP3374387A1 (OSRAM) |
| JP (4) | JP6768798B2 (OSRAM) |
| KR (2) | KR20230008256A (OSRAM) |
| CN (3) | CN116479001A (OSRAM) |
| AU (4) | AU2016353353B2 (OSRAM) |
| BR (1) | BR112018009717B1 (OSRAM) |
| CA (1) | CA3005334A1 (OSRAM) |
| CL (1) | CL2018001299A1 (OSRAM) |
| CO (1) | CO2018005377A2 (OSRAM) |
| EA (2) | EA202190827A1 (OSRAM) |
| IL (3) | IL281038B2 (OSRAM) |
| MX (1) | MX2018005982A (OSRAM) |
| MY (2) | MY198589A (OSRAM) |
| SG (2) | SG11201804070XA (OSRAM) |
| TW (3) | TWI777175B (OSRAM) |
| WO (1) | WO2017083762A1 (OSRAM) |
| ZA (3) | ZA201803443B (OSRAM) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| MX2018005969A (es) | 2015-11-13 | 2018-11-29 | Baxalta Inc | Vectores virales que codifican variantes del factor fviii recombinantes con mayor expresión para la terapia génica de hemofilia a. |
| EA202190827A1 (ru) | 2015-11-13 | 2021-11-30 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| WO2018128689A1 (en) | 2016-11-04 | 2018-07-12 | Baxalta Incorporated | Adeno-associated virus formulations |
| WO2019067766A1 (en) * | 2017-09-27 | 2019-04-04 | Sigilon Therapeutics, Inc. | METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS |
| US20200248179A1 (en) * | 2017-10-02 | 2020-08-06 | Research Institute At Nationwide Children's Hospital | MiRNA Detargeting System for Tissue Specific Interference |
| KR102105145B1 (ko) * | 2017-10-13 | 2020-05-13 | 영남대학교 산학협력단 | 촬영 장치 및 용기 |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| KR20210034013A (ko) * | 2018-07-16 | 2021-03-29 | 박스알타 인코퍼레이티드 | 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법 |
| WO2020028830A1 (en) * | 2018-08-03 | 2020-02-06 | Sangamo Therapeutics, Inc. | Improved clinical parameters by expression of factor viii |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| TWI851647B (zh) * | 2019-01-16 | 2024-08-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| AU2020221340A1 (en) | 2019-02-15 | 2021-09-16 | Bayer Healthcare Llc | Gene editing for hemophilia A with improved Factor VIII expression |
| BR112021015776A2 (pt) * | 2019-06-05 | 2021-11-09 | Bayer Healthcare Llc | Edição de gene para hemofilia a com expressão de fator viii melhorada |
| EP3986481A2 (en) * | 2019-06-20 | 2022-04-27 | Takeda Pharmaceutical Company Limited | Method of treatment with viral-based gene therapy |
| CN114901686A (zh) * | 2019-11-01 | 2022-08-12 | 自由行疗法有限公司 | 因子viii多肽 |
| 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 |
| WO2022211604A1 (ko) * | 2021-04-02 | 2022-10-06 | 연세대학교 산학협력단 | Fe-fviii 변이 유전자로 교정된 줄기세포, 이로부터 분화된 내피 세포 및 이를 포함하는 혈우병 예방 또는 치료용 약제학적 조성물 |
| KR102421131B1 (ko) * | 2021-05-28 | 2022-07-15 | 알엔에이진(주) | 세포내에서 장시간동안 존재할 수 있는 외부 도입 mRNA를 선별하는 방법 |
| JP2024544499A (ja) * | 2021-11-25 | 2024-12-03 | 四川至善唯新生物科技有限公司 | 遺伝子工学的改変により得られた、分泌能と凝固活性を向上させた組換えヒト第ｖｉｉｉ因子 |
| MA68586A1 (fr) * | 2022-04-28 | 2025-04-30 | Joint Stock Company "Biocad" | Acide nucléique à codons optimisés codant pour fviii-bdd |
| 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 |
| WO2024191428A1 (en) | 2023-03-14 | 2024-09-19 | Sigilon Therapeutics, Inc. | Hydrogel capsules comprising covalently photocrosslinked polysaccharides and islet cells |
| WO2025008774A1 (en) | 2023-07-05 | 2025-01-09 | Takeda Pharmaceutical Company Limited | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
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| US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
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| EP3513802B1 (en) | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
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| KR102554850B1 (ko) * | 2015-02-06 | 2023-07-13 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도 |
| ES2764453T3 (es) | 2015-03-17 | 2020-06-03 | Univ Brussel Vrije | Sistemas de expresión específica de hígado optimizados para FVIII y FIX |
| EA202190827A1 (ru) | 2015-11-13 | 2021-11-30 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
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2016
- 2016-11-11 EA EA202190827A patent/EA202190827A1/ru unknown
- 2016-11-11 CA CA3005334A patent/CA3005334A1/en active Pending
- 2016-11-11 AU AU2016353353A patent/AU2016353353B2/en active Active
- 2016-11-11 EP EP16809573.5A patent/EP3374387A1/en active Pending
- 2016-11-11 KR KR1020227046419A patent/KR20230008256A/ko not_active Ceased
- 2016-11-11 TW TW109118505A patent/TWI777175B/zh active
- 2016-11-11 SG SG11201804070XA patent/SG11201804070XA/en unknown
- 2016-11-11 IL IL281038A patent/IL281038B2/en unknown
- 2016-11-11 CN CN202310490697.4A patent/CN116479001A/zh active Pending
- 2016-11-11 JP JP2018524747A patent/JP6768798B2/ja active Active
- 2016-11-11 IL IL305526A patent/IL305526A/en unknown
- 2016-11-11 EA EA201891137A patent/EA038288B1/ru unknown
- 2016-11-11 SG SG10202106307UA patent/SG10202106307UA/en unknown
- 2016-11-11 CN CN202310964403.7A patent/CN116949052A/zh active Pending
- 2016-11-11 MX MX2018005982A patent/MX2018005982A/es unknown
- 2016-11-11 CN CN201680067846.4A patent/CN108884145B/zh active Active
- 2016-11-11 MY MYPI2020005892A patent/MY198589A/en unknown
- 2016-11-11 US US15/349,930 patent/US10189888B2/en active Active
- 2016-11-11 BR BR112018009717-9A patent/BR112018009717B1/pt active IP Right Grant
- 2016-11-11 TW TW111137316A patent/TW202302627A/zh unknown
- 2016-11-11 TW TW105136965A patent/TWI695069B/zh active
- 2016-11-11 MY MYPI2018000735A patent/MY190067A/en unknown
- 2016-11-11 KR KR1020187015189A patent/KR102484553B1/ko active Active
- 2016-11-11 WO PCT/US2016/061684 patent/WO2017083762A1/en not_active Ceased
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2018
- 2018-05-13 IL IL259302A patent/IL259302B/en active IP Right Grant
- 2018-05-14 CL CL2018001299A patent/CL2018001299A1/es unknown
- 2018-05-23 CO CONC2018/0005377A patent/CO2018005377A2/es unknown
- 2018-05-24 ZA ZA2018/03443A patent/ZA201803443B/en unknown
- 2018-12-05 US US16/211,201 patent/US11492388B2/en active Active
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2019
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2020
- 2020-06-05 AU AU2020203761A patent/AU2020203761B2/en active Active
- 2020-06-05 AU AU2020203760A patent/AU2020203760B2/en active Active
- 2020-08-04 JP JP2020132079A patent/JP7069258B2/ja active Active
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2022
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- 2022-05-02 JP JP2022075864A patent/JP7307836B2/ja active Active
- 2022-12-19 ZA ZA2022/13661A patent/ZA202213661B/en unknown
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2023
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