JP2019513794A5 - - Google Patents
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- JP2019513794A5 JP2019513794A5 JP2018554331A JP2018554331A JP2019513794A5 JP 2019513794 A5 JP2019513794 A5 JP 2019513794A5 JP 2018554331 A JP2018554331 A JP 2018554331A JP 2018554331 A JP2018554331 A JP 2018554331A JP 2019513794 A5 JP2019513794 A5 JP 2019513794A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- suspension
- aqueous suspension
- enhancer
- aav
- 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.)
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- 239000007900 aqueous suspension Substances 0.000 claims 7
- 241000702421 Dependoparvovirus Species 0.000 claims 6
- 108010071690 Prealbumin Proteins 0.000 claims 5
- 102000009190 Transthyretin Human genes 0.000 claims 5
- 239000003623 enhancer Substances 0.000 claims 5
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 4
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 3
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 3
- 208000009292 Hemophilia A Diseases 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 239000000725 suspension Substances 0.000 claims 3
- 108091026890 Coding region Proteins 0.000 claims 2
- 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 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 230000001112 coagulating effect Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 230000003494 hepatotrophic effect Effects 0.000 claims 1
- 102000057593 human F8 Human genes 0.000 claims 1
- 229960000900 human factor viii Drugs 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 239000003755 preservative agent Substances 0.000 claims 1
- 230000002335 preservative effect Effects 0.000 claims 1
- 239000004094 surface-active agent Substances 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022011052A JP2022062121A (ja) | 2016-04-15 | 2022-01-27 | 血友病ａ治療に対する遺伝子療法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323336P | 2016-04-15 | 2016-04-15 | |
| US62/323,336 | 2016-04-15 | ||
| US201662331807P | 2016-05-04 | 2016-05-04 | |
| US62/331,807 | 2016-05-04 | ||
| US201662428866P | 2016-12-01 | 2016-12-01 | |
| US62/428,866 | 2016-12-01 | ||
| PCT/US2017/027396 WO2017180857A1 (en) | 2016-04-15 | 2017-04-13 | Gene therapy for treating hemophilia a |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022011052A Division JP2022062121A (ja) | 2016-04-15 | 2022-01-27 | 血友病ａ治療に対する遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019513794A JP2019513794A (ja) | 2019-05-30 |
| JP2019513794A5 true JP2019513794A5 (https=) | 2020-05-21 |
| JP7347933B2 JP7347933B2 (ja) | 2023-09-20 |
Family
ID=59253992
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018554331A Active JP7347933B2 (ja) | 2016-04-15 | 2017-04-13 | 血友病ａ治療に対する遺伝子療法 |
| JP2022011052A Pending JP2022062121A (ja) | 2016-04-15 | 2022-01-27 | 血友病ａ治療に対する遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022011052A Pending JP2022062121A (ja) | 2016-04-15 | 2022-01-27 | 血友病ａ治療に対する遺伝子療法 |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US11779656B2 (https=) |
| EP (1) | EP3452102B9 (https=) |
| JP (2) | JP7347933B2 (https=) |
| KR (1) | KR102450833B1 (https=) |
| CN (1) | CN109562191A (https=) |
| AU (1) | AU2017248659B2 (https=) |
| BR (1) | BR112018071200A2 (https=) |
| CA (1) | CA3019425A1 (https=) |
| IL (1) | IL262215B2 (https=) |
| MX (1) | MX2018012537A (https=) |
| PE (2) | PE20181922A1 (https=) |
| RU (1) | RU2762257C2 (https=) |
| SA (1) | SA518400233B1 (https=) |
| SG (2) | SG10202009849WA (https=) |
| TW (1) | TWI791433B (https=) |
| WO (1) | WO2017180857A1 (https=) |
| ZA (1) | ZA201806477B (https=) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
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| EP3442597A1 (en) | 2016-04-15 | 2019-02-20 | The Trustees Of The University Of Pennsylvania | 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 |
| MX2020007876A (es) | 2018-01-31 | 2020-12-03 | Res Inst Nationwide Childrens Hospital | Terapia genica para la distrofia muscular de cinturas tipo 2c. |
| MX2020014119A (es) | 2018-06-29 | 2021-06-18 | Res Inst Nationwide Childrens Hospital | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a. |
| TW202028225A (zh) * | 2018-09-21 | 2020-08-01 | 英商夜星克有限公司 | 用於治療色素性視網膜炎之組合物及方法 |
| EP4461814A3 (en) * | 2018-10-12 | 2025-02-26 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
| JP2022514112A (ja) * | 2018-10-15 | 2022-02-09 | リジェネクスバイオ インコーポレイテッド | 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法 |
| JP7460643B2 (ja) | 2018-10-16 | 2024-04-02 | ブルーアレル， エルエルシー | 遺伝子へのｄｎａの標的化挿入のための方法 |
| GB201818816D0 (en) * | 2018-11-19 | 2019-01-02 | Synpromics Ltd | Regulatory nucleic acid sequences |
| 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 |
| MX2021010356A (es) * | 2019-02-26 | 2022-03-04 | Res Inst Nationwide Childrens Hospital | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. |
| WO2020186207A2 (en) | 2019-03-13 | 2020-09-17 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| PT4017871T (pt) | 2019-08-21 | 2024-04-22 | Res Inst Nationwide Childrens Hospital | Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular |
| EP4051704A2 (en) * | 2019-11-01 | 2022-09-07 | Freeline Therapeutics Limited | Factor viii construct |
| CN111218446B (zh) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技（武汉）有限公司 | 一种肝脏特异性启动子及其应用 |
| IL294713A (en) * | 2020-01-16 | 2022-09-01 | Takeda Pharmaceuticals Co | Adeno associated virus based gene therapy for phenylketonuria |
| WO2021222238A1 (en) * | 2020-04-27 | 2021-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP4118200B1 (en) * | 2020-07-29 | 2025-10-08 | Spur Therapeutics Limited | Mutated beta-glucocerebrosidase with improved stability |
| WO2022165246A1 (en) * | 2021-01-29 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and method of use of mutant ace2 decoy variants |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| JP2024506860A (ja) * | 2021-02-01 | 2024-02-15 | ザ・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 | ニーマンピック病ａ型を治療するための組成物及び方法 |
| JP2023059858A (ja) | 2021-10-15 | 2023-04-27 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 |
| US20240335564A1 (en) * | 2021-11-02 | 2024-10-10 | Gritgen Therapeutics Limited | Isolated nucleic acid molecule and use thereof |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023205300A2 (en) * | 2022-04-20 | 2023-10-26 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
| US20250352626A1 (en) * | 2022-06-10 | 2025-11-20 | University Of Florida Research Foundation, Incorporated | Immune tolerance induction to viral capsids |
| WO2025180508A1 (en) * | 2024-02-29 | 2025-09-04 | Shanghai Vitalgen Biopharma Co., Ltd. | Human factor viii variant expression cassettes and uses thereof for treating hemophilia a |
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| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
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2017
- 2017-04-13 KR KR1020187032617A patent/KR102450833B1/ko active Active
- 2017-04-13 CN CN201780023787.5A patent/CN109562191A/zh active Pending
- 2017-04-13 EP EP17734159.1A patent/EP3452102B9/en active Active
- 2017-04-13 MX MX2018012537A patent/MX2018012537A/es unknown
- 2017-04-13 SG SG10202009849WA patent/SG10202009849WA/en unknown
- 2017-04-13 WO PCT/US2017/027396 patent/WO2017180857A1/en not_active Ceased
- 2017-04-13 US US16/093,798 patent/US11779656B2/en active Active
- 2017-04-13 PE PE2018001994A patent/PE20181922A1/es unknown
- 2017-04-13 BR BR112018071200-0A patent/BR112018071200A2/pt not_active Application Discontinuation
- 2017-04-13 JP JP2018554331A patent/JP7347933B2/ja active Active
- 2017-04-13 CA CA3019425A patent/CA3019425A1/en active Pending
- 2017-04-13 TW TW106112302A patent/TWI791433B/zh not_active IP Right Cessation
- 2017-04-13 AU AU2017248659A patent/AU2017248659B2/en not_active Ceased
- 2017-04-13 PE PE2023002954A patent/PE20250736A1/es unknown
- 2017-04-13 SG SG11201808422QA patent/SG11201808422QA/en unknown
- 2017-04-13 RU RU2018136611A patent/RU2762257C2/ru active
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2018
- 2018-09-28 ZA ZA2018/06477A patent/ZA201806477B/en unknown
- 2018-10-08 IL IL262215A patent/IL262215B2/en unknown
- 2018-10-14 SA SA518400233A patent/SA518400233B1/ar unknown
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2019
- 2019-11-22 US US16/691,822 patent/US10888628B2/en active Active
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2022
- 2022-01-27 JP JP2022011052A patent/JP2022062121A/ja active Pending
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2023
- 2023-08-08 US US18/446,243 patent/US20240000974A1/en not_active Abandoned
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