JP2018531609A5 - - Google Patents
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- JP2018531609A5 JP2018531609A5 JP2018521395A JP2018521395A JP2018531609A5 JP 2018531609 A5 JP2018531609 A5 JP 2018531609A5 JP 2018521395 A JP2018521395 A JP 2018521395A JP 2018521395 A JP2018521395 A JP 2018521395A JP 2018531609 A5 JP2018531609 A5 JP 2018531609A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- vector
- sequence
- fxn
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562251288P | 2015-11-05 | 2015-11-05 | |
| US62/251,288 | 2015-11-05 | ||
| US201662411980P | 2016-10-24 | 2016-10-24 | |
| US62/411,980 | 2016-10-24 | ||
| PCT/IB2016/056572 WO2017077451A1 (en) | 2015-11-05 | 2016-11-01 | Modified friedreich ataxia genes and vectors for gene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020156320A Division JP2021019591A (ja) | 2015-11-05 | 2020-09-17 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018531609A JP2018531609A (ja) | 2018-11-01 |
| JP2018531609A5 true JP2018531609A5 (enExample) | 2019-12-12 |
| JP6767483B2 JP6767483B2 (ja) | 2020-10-14 |
Family
ID=57288476
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Expired - Fee Related JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
| JP2020156320A Pending JP2021019591A (ja) | 2015-11-05 | 2020-09-17 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020156320A Pending JP2021019591A (ja) | 2015-11-05 | 2020-09-17 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10548947B2 (enExample) |
| EP (1) | EP3370778A1 (enExample) |
| JP (2) | JP6767483B2 (enExample) |
| KR (2) | KR102200642B1 (enExample) |
| CN (1) | CN108348621A (enExample) |
| AU (2) | AU2016347887B2 (enExample) |
| BR (1) | BR112018007453A2 (enExample) |
| CA (1) | CA2947584A1 (enExample) |
| CO (1) | CO2018004564A2 (enExample) |
| HK (1) | HK1250924A1 (enExample) |
| IL (1) | IL259141A (enExample) |
| MX (1) | MX2018005084A (enExample) |
| PE (1) | PE20181156A1 (enExample) |
| PH (1) | PH12018500964A1 (enExample) |
| RU (2) | RU2021102893A (enExample) |
| SG (2) | SG10201912763QA (enExample) |
| TW (2) | TWI654301B (enExample) |
| WO (1) | WO2017077451A1 (enExample) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10617770B2 (en) * | 2015-04-24 | 2020-04-14 | University Of Florida Research Foundation, Incorporated | AAV vector for treatment of Friedreich's ataxia |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| CN108289932A (zh) | 2015-05-22 | 2018-07-17 | D·D·格恩金 | 胞外dna作为神经变性的治疗靶点 |
| SG10201912763QA (en) * | 2015-11-05 | 2020-02-27 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| ES3038656T3 (en) * | 2016-03-23 | 2025-10-14 | Univ California | Methods of treating mitochondrial disorders |
| EP3491440A4 (en) | 2016-09-21 | 2020-02-26 | Commscope Technologies LLC | ADAPTER BLOCK ASSEMBLY |
| BR112019009446A2 (pt) | 2016-11-09 | 2019-07-30 | Intrexon Corp | construtos de expressão da frataxina |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| JP7361037B2 (ja) | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| AU2018346102B2 (en) | 2017-10-03 | 2023-05-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CA3079107A1 (en) * | 2017-10-17 | 2019-04-25 | Fundacio Institut D'investigacio En Ciencies De La Salut Germans Trias I Pujol | Vectors for the treatment of friedreich's ataxia |
| US11905522B2 (en) | 2018-01-16 | 2024-02-20 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity |
| CN112272672A (zh) | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| SG11202009451VA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| US20210308274A1 (en) * | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating friedreich's ataxia |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| CN113166748A (zh) * | 2018-08-10 | 2021-07-23 | 逻辑生物治疗公司 | 用于治疗mma的非破坏性基因疗法 |
| SG11202103151RA (en) * | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3880179A2 (en) * | 2018-11-14 | 2021-09-22 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions for cns delivery |
| US20220054656A1 (en) * | 2018-11-16 | 2022-02-24 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| JP7704998B2 (ja) * | 2018-11-21 | 2025-07-09 | 武田薬品工業株式会社 | 組換えウイルスベクター及びそれの産生のための核酸 |
| KR20210117271A (ko) * | 2018-12-21 | 2021-09-28 | 노쓰웨스턴유니버시티 | 근막 손상 예방 및 치료를 위한 아넥신의 사용근각 손상 예방 및 치료를 위한 아넥신의 사용 |
| SG11202110146VA (en) | 2019-03-21 | 2021-10-28 | Stridebio Inc | Recombinant adeno-associated virus vectors |
| BR112021019109A2 (pt) * | 2019-03-27 | 2021-11-30 | Sigilon Therapeutics Inc | Célula rpe manipulada, composições, molécula isolada de dna de fita dupla, dispositivo implantável, cápsula de hidrogel, e, método para aplicar uma terapia com fvii |
| KR20220004649A (ko) * | 2019-03-28 | 2022-01-11 | 인텔리아 테라퓨틱스, 인크. | 폴리펩티드 발현을 위한 폴리뉴클레오티드, 조성물 및 방법 |
| MX2021012184A (es) | 2019-04-10 | 2022-01-24 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| US20230144548A1 (en) * | 2019-06-30 | 2023-05-11 | John Fraser Wright | Recombinant aav vectors with altered immunogencity and methods of making the same |
| WO2021000024A1 (en) * | 2019-07-04 | 2021-01-07 | Children's Medical Research Institute | Methods and aav vectors for in vivo transduction |
| CA3147034A1 (en) * | 2019-07-12 | 2021-01-21 | Cls Therapeutics Limited | Treatment of diseases associated with protein misfolding by nervous system expression of an enzyme which has a deoxyribonuclease (dnase) activity |
| AU2020346914A1 (en) * | 2019-09-13 | 2022-04-21 | Prevail Therapeutics, Inc. | Compositions and methods for treatment of Friedreich's Ataxia |
| EP4045525A1 (en) | 2019-10-17 | 2022-08-24 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| WO2021127533A1 (en) * | 2019-12-19 | 2021-06-24 | The Trustees Of The University Of Pennsylvania | Compositions for treating friedreich's ataxia |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
| EP4188404A4 (en) | 2020-07-29 | 2025-02-26 | The Board Of Regents Of The University Of Texas System | TRANSGENIC CASSETTES, AAV VECTORS AND AAV VIRUS VECTORS FOR THE EXPRESSION OF HUMAN CODON-OPTIMIZED CSTB |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| CA3200401A1 (en) | 2020-11-03 | 2022-05-12 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| WO2022137076A1 (en) | 2020-12-23 | 2022-06-30 | Pfizer Inc. | Methods for purification of aav vectors by affinity chromatography |
| US20240058477A1 (en) * | 2021-01-04 | 2024-02-22 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
| WO2022150772A1 (en) * | 2021-01-11 | 2022-07-14 | The Trustees Of The University Of Pennsylvania | Compositions for treating friedreich's ataxia |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024011109A2 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies, Inc. | Compositions and methods for treatment of achromotopsia |
| CN117089514A (zh) * | 2023-10-13 | 2023-11-21 | 思鹏生物科技(苏州)有限公司 | 提升hek293细胞系aav生产效率的细胞筛选驯化方法 |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| ATE237352T1 (de) | 1995-06-07 | 2003-05-15 | Univ North Carolina | Helfervirus-freie herstellung von aav |
| IL128779A0 (en) | 1996-09-06 | 2000-01-31 | Univ Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| AU762220B2 (en) | 1998-05-28 | 2003-06-19 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | AAV5 vector and uses thereof |
| ES2288037T3 (es) | 1998-11-05 | 2007-12-16 | The Trustees Of The University Of Pennsylvania | Secuencia de acido nucleico del serotipo 1 de adenovirus asociado, vectores y celulas huesped que las contienen. |
| CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| CA2399576A1 (en) | 2000-12-07 | 2002-06-13 | Universite De Nantes | Inducible highly productive raav packaging cell-lines |
| HUE035567T2 (en) | 2003-09-30 | 2018-05-28 | Univ Pennsylvania | Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them |
| NZ555830A (en) | 2004-12-15 | 2009-01-31 | Univ North Carolina | Chimeric vectors |
| MX2010002003A (es) * | 2007-08-20 | 2010-03-10 | Glaxo Group Ltd | Metodo de produccion. |
| ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
| AU2010340358B2 (en) * | 2009-12-21 | 2014-07-24 | Pharmathene, Inc. | Recombinant butyrylcholinesterases and truncates thereof |
| EP2539354B1 (en) * | 2010-02-08 | 2017-04-05 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecules encoding rantes, and compositions comprising and methods of using the same |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| CA2838588C (en) * | 2011-06-09 | 2021-09-14 | Curna, Inc. | Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn |
| EP2771455B1 (en) | 2011-10-28 | 2016-10-05 | The University of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
| US9066966B2 (en) | 2013-02-01 | 2015-06-30 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia |
| CA2904156C (en) | 2013-03-15 | 2023-01-10 | The Children's Hospital Of Philadelphia | Vectors comprising stuffer/filler polynucleotide sequences and methods of use |
| WO2014144229A1 (en) | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav vectors |
| CA2907799A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| CN116004721A (zh) | 2013-07-12 | 2023-04-25 | 费城儿童医院 | Aav载体和用于抗aav（腺相关病毒）中和抗体的检测 |
| SI3024498T1 (sl) | 2013-07-22 | 2020-07-31 | The Children's Hospital Of Philadelphia | Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva |
| EP3459965B1 (en) | 2013-10-11 | 2020-12-02 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| SG10201912763QA (en) * | 2015-11-05 | 2020-02-27 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
-
2016
- 2016-11-01 SG SG10201912763QA patent/SG10201912763QA/en unknown
- 2016-11-01 RU RU2021102893A patent/RU2021102893A/ru unknown
- 2016-11-01 MX MX2018005084A patent/MX2018005084A/es unknown
- 2016-11-01 BR BR112018007453A patent/BR112018007453A2/pt not_active IP Right Cessation
- 2016-11-01 KR KR1020187015819A patent/KR102200642B1/ko not_active Expired - Fee Related
- 2016-11-01 RU RU2018116076A patent/RU2743792C2/ru active
- 2016-11-01 JP JP2018521395A patent/JP6767483B2/ja not_active Expired - Fee Related
- 2016-11-01 CN CN201680064440.0A patent/CN108348621A/zh active Pending
- 2016-11-01 PE PE2018000726A patent/PE20181156A1/es unknown
- 2016-11-01 US US15/340,721 patent/US10548947B2/en active Active
- 2016-11-01 KR KR1020217000034A patent/KR20210005994A/ko not_active Withdrawn
- 2016-11-01 SG SG11201802408RA patent/SG11201802408RA/en unknown
- 2016-11-01 AU AU2016347887A patent/AU2016347887B2/en not_active Ceased
- 2016-11-01 HK HK18110376.3A patent/HK1250924A1/zh unknown
- 2016-11-01 EP EP16795164.9A patent/EP3370778A1/en not_active Withdrawn
- 2016-11-01 WO PCT/IB2016/056572 patent/WO2017077451A1/en not_active Ceased
- 2016-11-04 CA CA2947584A patent/CA2947584A1/en active Pending
- 2016-11-04 TW TW105135911A patent/TWI654301B/zh not_active IP Right Cessation
- 2016-11-04 TW TW108104049A patent/TWI743442B/zh not_active IP Right Cessation
-
2018
- 2018-04-27 CO CONC2018/0004564A patent/CO2018004564A2/es unknown
- 2018-05-03 IL IL259141A patent/IL259141A/en unknown
- 2018-05-03 PH PH12018500964A patent/PH12018500964A1/en unknown
-
2019
- 2019-12-16 US US16/716,033 patent/US20200384073A1/en not_active Abandoned
-
2020
- 2020-02-11 AU AU2020200976A patent/AU2020200976B2/en not_active Ceased
- 2020-09-17 JP JP2020156320A patent/JP2021019591A/ja active Pending
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