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.)
- Granted
Links
- 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 (enExample) | 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 (enExample) |
| EP (1) | EP3452102B9 (enExample) |
| JP (2) | JP7347933B2 (enExample) |
| KR (1) | KR102450833B1 (enExample) |
| CN (1) | CN109562191A (enExample) |
| AU (1) | AU2017248659B2 (enExample) |
| BR (1) | BR112018071200A2 (enExample) |
| CA (1) | CA3019425A1 (enExample) |
| IL (1) | IL262215B2 (enExample) |
| MX (1) | MX2018012537A (enExample) |
| PE (2) | PE20250736A1 (enExample) |
| RU (1) | RU2762257C2 (enExample) |
| SA (1) | SA518400233B1 (enExample) |
| SG (2) | SG10202009849WA (enExample) |
| TW (1) | TWI791433B (enExample) |
| WO (1) | WO2017180857A1 (enExample) |
| ZA (1) | ZA201806477B (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2337849B1 (en) | 2008-09-15 | 2018-06-13 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| US11617783B2 (en) | 2015-11-16 | 2023-04-04 | Research Institute At Nationwide Children's Hospital | Repairing a mutant human titin gene using CRISPR technology |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| WO2017180857A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
| US12391928B2 (en) | 2018-06-29 | 2025-08-19 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A |
| JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
| 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 |
| CN112912518A (zh) * | 2018-10-15 | 2021-06-04 | 再生生物股份有限公司 | 用于测量复制缺陷型病毒载体和病毒的感染性的方法 |
| BR112021007229A2 (pt) | 2018-10-16 | 2021-08-10 | Blueallele, Llc | métodos para inserção dirigida de dna em genes |
| 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 |
| CN113766935A (zh) * | 2019-02-26 | 2021-12-07 | 全国儿童医院研究所 | β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗 |
| JP7795357B2 (ja) | 2019-03-13 | 2026-01-07 | ジェネレーション バイオ カンパニー | Ｆｖｉｉｉ治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用 |
| SI4017871T1 (sl) | 2019-08-21 | 2024-06-28 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije |
| US20220396610A1 (en) * | 2019-11-01 | 2022-12-15 | Freeline Therapeutics Limited | Factor viii polypeptide |
| 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 |
| KR20220130174A (ko) * | 2020-01-16 | 2022-09-26 | 다케다 야쿠힌 고교 가부시키가이샤 | 페닐케톤뇨증에 대한 아데노 연관 바이러스 기반 유전자 치료법 |
| EP4143308A4 (en) * | 2020-04-27 | 2024-06-26 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| DK4118200T3 (da) * | 2020-07-29 | 2025-12-08 | Spur Therapeutics Ltd | Muteret beta-glucocerebrosidase med forbedret stabilitet |
| 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 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | 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 |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| US20240335564A1 (en) * | 2021-11-02 | 2024-10-10 | Gritgen Therapeutics Limited | Isolated nucleic acid molecule and use thereof |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20250276093A1 (en) * | 2022-04-20 | 2025-09-04 | AskBio 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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| WO2017180857A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
-
2017
- 2017-04-13 WO PCT/US2017/027396 patent/WO2017180857A1/en not_active Ceased
- 2017-04-13 BR BR112018071200-0A patent/BR112018071200A2/pt not_active Application Discontinuation
- 2017-04-13 KR KR1020187032617A patent/KR102450833B1/ko active Active
- 2017-04-13 SG SG10202009849WA patent/SG10202009849WA/en unknown
- 2017-04-13 PE PE2023002954A patent/PE20250736A1/es unknown
- 2017-04-13 JP JP2018554331A patent/JP7347933B2/ja active Active
- 2017-04-13 US US16/093,798 patent/US11779656B2/en active Active
- 2017-04-13 MX MX2018012537A patent/MX2018012537A/es unknown
- 2017-04-13 RU RU2018136611A patent/RU2762257C2/ru active
- 2017-04-13 CA CA3019425A patent/CA3019425A1/en active Pending
- 2017-04-13 PE PE2018001994A patent/PE20181922A1/es unknown
- 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 AU AU2017248659A patent/AU2017248659B2/en not_active Ceased
- 2017-04-13 TW TW106112302A patent/TWI791433B/zh not_active IP Right Cessation
- 2017-04-13 SG SG11201808422QA patent/SG11201808422QA/en unknown
-
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
-
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
-
2023
- 2023-08-08 US US18/446,243 patent/US20240000974A1/en not_active Abandoned