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 (enExample) | 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 (enExample) |
| EP (1) | EP3374387A1 (enExample) |
| JP (4) | JP6768798B2 (enExample) |
| KR (2) | KR102484553B1 (enExample) |
| CN (3) | CN116479001A (enExample) |
| AU (4) | AU2016353353B2 (enExample) |
| BR (1) | BR112018009717B1 (enExample) |
| CA (1) | CA3005334A1 (enExample) |
| CL (1) | CL2018001299A1 (enExample) |
| CO (1) | CO2018005377A2 (enExample) |
| EA (2) | EA202190827A1 (enExample) |
| IL (3) | IL281038B2 (enExample) |
| MX (1) | MX2018005982A (enExample) |
| MY (2) | MY190067A (enExample) |
| SG (2) | SG10202106307UA (enExample) |
| TW (3) | TWI695069B (enExample) |
| WO (1) | WO2017083762A1 (enExample) |
| ZA (3) | ZA201803443B (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2016343887B2 (en) | 2015-10-28 | 2023-04-06 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| SG11201804064WA (en) * | 2015-11-13 | 2018-06-28 | Baxalta Inc | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| US10189888B2 (en) | 2015-11-13 | 2019-01-29 | Baxalta Incorporated | Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| PE20250736A1 (es) * | 2016-04-15 | 2025-03-10 | Univ Pennsylvania | Terapia genica para tratar la hemofilia a |
| ES2981519T3 (es) | 2016-11-04 | 2024-10-09 | Takeda Pharmaceuticals Co | Formulaciones de virus adeno-asociados |
| CA3077380A1 (en) * | 2017-09-27 | 2019-04-04 | Sigilon Therapeutics, Inc. | Methods, compositions, and implantable elements comprising active cells |
| EP3668997A4 (en) * | 2017-10-02 | 2021-05-19 | Research Institute at Nationwide Children's Hospital | SYSTEM FOR MIRNA DETARGETING 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 |
| 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 |
| AU2019314513A1 (en) * | 2018-08-03 | 2021-01-28 | 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 |
| EP3923992A1 (en) | 2019-02-15 | 2021-12-22 | CRISPR Therapeutics AG | 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 |
| US20200405883A1 (en) * | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| JP2023501262A (ja) * | 2019-11-01 | 2023-01-18 | フリーライン セラピューティクス リミテッド | 転写調節エレメント |
| 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 |
| CN116710554B (zh) * | 2021-11-25 | 2024-06-07 | 四川至善唯新生物科技有限公司 | 基因工程改造获得的分泌能力和凝血活性增强的人类八因子 |
| KR20250007615A (ko) * | 2022-04-28 | 2025-01-14 | 조인트 스탁 컴퍼니 "바이오케드" | Fviii-bdd를 인코딩하는 코돈 최적화된 핵산 |
| 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 |
| TW202435846A (zh) | 2023-03-14 | 2024-09-16 | 美商希吉隆醫療公司 | 包含共價交聯多醣及胰島細胞之水膠膠囊 |
| TW202517669A (zh) * | 2023-07-05 | 2025-05-01 | 日商武田藥品工業股份有限公司 | 用於A型血友病之基因療法的編碼表現增加之重組FVlll變異體的病毒載體 |
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| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| DE3683980D1 (de) | 1985-04-12 | 1992-04-02 | Genetics Inst | Neue prokoagulierungsproteine. |
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| US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| US5610278A (en) | 1986-06-24 | 1997-03-11 | Novo Nordisk A/S | Process for producing a coagulation active complex of factor VIII fragments |
| US6060447A (en) | 1987-05-19 | 2000-05-09 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| US6346513B1 (en) | 1987-06-12 | 2002-02-12 | Baxter Trading Gmbh | Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them |
| IE69026B1 (en) | 1987-06-12 | 1996-08-07 | Immuno Ag | Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them |
| FR2619314B1 (fr) | 1987-08-11 | 1990-06-15 | Transgene Sa | Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant |
| ATE322547T1 (de) | 1993-06-10 | 2006-04-15 | Genetic Therapy Inc | Adenovirale vektoren für die behandlung der hämophilie |
| SE504074C2 (sv) | 1993-07-05 | 1996-11-04 | Pharmacia Ab | Proteinberedning för subkutan, intramuskulär eller intradermal administrering |
| 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 |
| US5952198A (en) * | 1995-05-04 | 1999-09-14 | Bayer Corporation | Production of recombinant Factor VIII in the presence of liposome-like substances of mixed composition |
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| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| US6458563B1 (en) | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
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| JP4634036B2 (ja) | 2001-10-05 | 2011-02-16 | エクスプレッション セラピューティクス, エルエルシー | 高レベルエキスプレッサー第viii因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法 |
| PT2359869T (pt) | 2001-12-17 | 2019-04-16 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
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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 |
| DK2911687T3 (da) | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| ES2959747T3 (es) | 2013-02-15 | 2024-02-28 | Bioverativ Therapeutics Inc | Gen del factor VIII optimizado |
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| SI3013855T1 (sl) | 2013-06-24 | 2021-06-30 | Xiao, Weidong | Mutantne sestave faktorja VIII in metode |
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| ES2993915T3 (en) | 2014-08-13 | 2025-01-13 | Childrens Hospital Philadelphia | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
| EP3253786A4 (en) * | 2015-02-06 | 2018-10-17 | The University of North Carolina at Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
| EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| US10189888B2 (en) | 2015-11-13 | 2019-01-29 | Baxalta Incorporated | Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A |
-
2016
- 2016-11-11 US US15/349,930 patent/US10189888B2/en active Active
- 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 BR BR112018009717-9A patent/BR112018009717B1/pt active IP Right Grant
- 2016-11-11 TW TW105136965A patent/TWI695069B/zh active
- 2016-11-11 TW TW109118505A patent/TWI777175B/zh active
- 2016-11-11 SG SG10202106307UA patent/SG10202106307UA/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 SG SG11201804070XA patent/SG11201804070XA/en unknown
- 2016-11-11 KR KR1020187015189A patent/KR102484553B1/ko active Active
- 2016-11-11 IL IL281038A patent/IL281038B2/en unknown
- 2016-11-11 WO PCT/US2016/061684 patent/WO2017083762A1/en not_active Ceased
- 2016-11-11 IL IL305526A patent/IL305526A/en unknown
- 2016-11-11 KR KR1020227046419A patent/KR20230008256A/ko not_active Ceased
- 2016-11-11 MX MX2018005982A patent/MX2018005982A/es unknown
- 2016-11-11 TW TW111137316A patent/TW202302627A/zh unknown
- 2016-11-11 EA EA201891137A patent/EA038288B1/ru unknown
- 2016-11-11 CN CN201680067846.4A patent/CN108884145B/zh active Active
- 2016-11-11 MY MYPI2018000735A patent/MY190067A/en unknown
- 2016-11-11 MY MYPI2020005892A patent/MY198589A/en unknown
- 2016-11-11 EP EP16809573.5A patent/EP3374387A1/en active Pending
- 2016-11-11 CN CN202310964403.7A patent/CN116949052A/zh active Pending
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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 US16/211,202 patent/US11254731B2/en active Active
- 2018-12-05 US US16/211,201 patent/US11492388B2/en active Active
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2019
- 2019-07-23 ZA ZA2019/04814A patent/ZA201904814B/en unknown
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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
-
2022
- 2022-01-06 US US17/570,187 patent/US20220213172A1/en active Pending
- 2022-02-25 AU AU2022201319A patent/AU2022201319B2/en active Active
- 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
- 2023-06-30 JP JP2023108132A patent/JP7631425B2/ja active Active
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