JP2018510648A5 - - Google Patents
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- Publication number
- JP2018510648A5 JP2018510648A5 JP2017553005A JP2017553005A JP2018510648A5 JP 2018510648 A5 JP2018510648 A5 JP 2018510648A5 JP 2017553005 A JP2017553005 A JP 2017553005A JP 2017553005 A JP2017553005 A JP 2017553005A JP 2018510648 A5 JP2018510648 A5 JP 2018510648A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- optionally
- aav2
- raav
- oversized
- 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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- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 45
- 239000002245 particle Substances 0.000 claims 42
- 210000000234 capsid Anatomy 0.000 claims 25
- 108700019146 Transgenes Proteins 0.000 claims 24
- 230000008488 polyadenylation Effects 0.000 claims 17
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 12
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 12
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 12
- 101000772176 Mus musculus Transthyretin Proteins 0.000 claims 11
- 238000000034 method Methods 0.000 claims 11
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 claims 9
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 9
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 8
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 8
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 8
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 8
- 241000283690 Bos taurus Species 0.000 claims 8
- 108091092195 Intron Proteins 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical class 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 241000283707 Capra Species 0.000 claims 7
- 239000000203 mixture Substances 0.000 claims 7
- 101150044789 Cap gene Proteins 0.000 claims 6
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 6
- 108010006025 bovine growth hormone Proteins 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 6
- 208000035475 disorder Diseases 0.000 claims 6
- 101150066583 rep gene Proteins 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 108010069091 Dystrophin Proteins 0.000 claims 5
- 102000001039 Dystrophin Human genes 0.000 claims 5
- 108010054218 Factor VIII Proteins 0.000 claims 5
- 102000001690 Factor VIII Human genes 0.000 claims 5
- 229960000301 factor viii Drugs 0.000 claims 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 4
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 4
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 4
- -1 dysferrin Proteins 0.000 claims 4
- 239000013646 rAAV2 vector Substances 0.000 claims 4
- 230000001225 therapeutic effect Effects 0.000 claims 4
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 3
- 241000288906 Primates Species 0.000 claims 3
- 101100409194 Rattus norvegicus Ppargc1b gene Proteins 0.000 claims 3
- 102000057593 human F8 Human genes 0.000 claims 3
- 239000000725 suspension Substances 0.000 claims 3
- 238000003146 transient transfection Methods 0.000 claims 3
- 241000701161 unidentified adenovirus Species 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 238000012217 deletion Methods 0.000 claims 2
- 230000037430 deletion Effects 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 238000000746 purification Methods 0.000 claims 2
- 239000013608 rAAV vector Substances 0.000 claims 2
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 102000004168 Dysferlin Human genes 0.000 claims 1
- 108090000620 Dysferlin Proteins 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 208000022583 Qualitative or quantitative defects of dysferlin Diseases 0.000 claims 1
- 238000011210 chromatographic step Methods 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021075526A JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2023097370A JP2023116678A (ja) | 2015-04-08 | 2023-06-14 | オーバーサイズアデノ随伴ベクターの産生 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562144862P | 2015-04-08 | 2015-04-08 | |
| US62/144,862 | 2015-04-08 | ||
| US201562220067P | 2015-09-17 | 2015-09-17 | |
| US62/220,067 | 2015-09-17 | ||
| PCT/US2016/026486 WO2016164609A2 (en) | 2015-04-08 | 2016-04-07 | Production of oversized adeno-associated vectors |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021075526A Division JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018510648A JP2018510648A (ja) | 2018-04-19 |
| JP2018510648A5 true JP2018510648A5 (cg-RX-API-DMAC7.html) | 2019-05-09 |
| JP6878299B2 JP6878299B2 (ja) | 2021-05-26 |
Family
ID=56943906
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017553005A Active JP6878299B2 (ja) | 2015-04-08 | 2016-04-07 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2021075526A Withdrawn JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2023097370A Pending JP2023116678A (ja) | 2015-04-08 | 2023-06-14 | オーバーサイズアデノ随伴ベクターの産生 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021075526A Withdrawn JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2023097370A Pending JP2023116678A (ja) | 2015-04-08 | 2023-06-14 | オーバーサイズアデノ随伴ベクターの産生 |
Country Status (27)
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2956477T5 (pl) | 2013-02-15 | 2024-05-27 | Bioverativ Therapeutics Inc. | Zoptymalizowany gen czynnika viii |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| EP3352787A1 (en) | 2015-09-24 | 2018-08-01 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same |
| RS63548B1 (sr) | 2016-02-01 | 2022-09-30 | Bioverativ Therapeutics Inc | Optimizovani geni faktora viii |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| WO2018154096A1 (en) | 2017-02-24 | 2018-08-30 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| CN118956965A (zh) * | 2017-05-19 | 2024-11-15 | 编码治疗公司 | 高活性调控元件 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| EP3654860A1 (en) | 2017-07-17 | 2020-05-27 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| CN111448308A (zh) | 2017-08-03 | 2020-07-24 | 沃雅戈治疗公司 | 递送aav的组合物和方法 |
| KR20250040746A (ko) | 2017-08-09 | 2025-03-24 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 이의 용도 |
| WO2019067840A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| AU2018375192B2 (en) | 2017-12-01 | 2023-11-09 | Encoded Therapeutics, Inc. | Engineered DNA binding proteins |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US20210338838A1 (en) * | 2018-07-20 | 2021-11-04 | University Of Utah Research Foundation | Gene therapy for macular degeneration |
| JP7602454B2 (ja) | 2018-08-09 | 2024-12-18 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| JP2022504740A (ja) * | 2018-10-12 | 2022-01-13 | ジェンザイム・コーポレーション | 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成 |
| CN119061198A (zh) * | 2019-08-29 | 2024-12-03 | 美国西门子医学诊断股份有限公司 | 用于检测aav脱落的试剂和方法 |
| US12403164B2 (en) | 2019-09-30 | 2025-09-02 | Bioverativ Therapeutics Inc. | Lentiviral vector formulations |
| 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 |
| CN115552037A (zh) * | 2020-03-16 | 2022-12-30 | 北卡罗来纳大学教堂山分校 | 用于选择性检测肿瘤源性病毒dna的组合物和方法 |
| BR112022026127A2 (pt) | 2020-06-24 | 2023-01-17 | Bioverativ Therapeutics Inc | Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína |
| CN116323953A (zh) | 2020-07-30 | 2023-06-23 | 沙普治疗股份有限公司 | 诱导产生raav病毒粒子的稳定细胞系 |
| AU2021330936A1 (en) | 2020-08-23 | 2023-05-04 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| JP2024534123A (ja) | 2021-08-23 | 2024-09-18 | バイオベラティブ セラピューティクス インコーポレイテッド | バキュロウイルス発現システム |
| TW202323274A (zh) | 2021-08-23 | 2023-06-16 | 美商百歐維拉提夫治療公司 | 優化因子viii基因 |
| WO2023028455A1 (en) | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Closed-end dna production with inverted terminal repeat sequences |
| AU2022355088A1 (en) | 2021-09-30 | 2024-05-16 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
| WO2023069926A1 (en) | 2021-10-18 | 2023-04-27 | Regeneron Pharmaceuticals, Inc. | Eukaryotic cells comprising adenovirus-associated virus polynucleotides |
| WO2024224361A1 (en) * | 2023-04-26 | 2024-10-31 | Sanofi | Host cell lines and methods for identifying and using such host cell lines |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| CA2387484A1 (en) * | 1999-10-12 | 2001-04-19 | Haim Burstein | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| CA2410828C (en) | 2000-06-01 | 2012-01-24 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| US20040092008A1 (en) * | 2002-11-12 | 2004-05-13 | Snyder Richard O. | Recombinant AAV vectors for gene therapy of hemophilia A |
| WO2004075861A2 (en) | 2003-02-26 | 2004-09-10 | Children's Hospital, Inc. | Recombinant adeno-associated virus production |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| SG10201403290SA (en) | 2009-06-16 | 2014-10-30 | Genzyme Corp | Improved methods for purification of recombinant aav vectors |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| WO2014064277A1 (en) * | 2012-10-26 | 2014-05-01 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| RS61039B1 (sr) * | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| KR102863734B1 (ko) | 2015-02-10 | 2025-09-25 | 젠자임 코포레이션 | 선조체 및 피질로의 바이러스 입자의 향상된 전달 |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
-
2016
- 2016-04-06 TW TW105110751A patent/TWI707951B/zh active
- 2016-04-07 TN TNP/2017/000431A patent/TN2017000431A1/en unknown
- 2016-04-07 IL IL294965A patent/IL294965A/en unknown
- 2016-04-07 SG SG11201708203UA patent/SG11201708203UA/en unknown
- 2016-04-07 CA CA2982123A patent/CA2982123A1/en active Pending
- 2016-04-07 KR KR1020177032062A patent/KR102848324B1/ko active Active
- 2016-04-07 IL IL283291A patent/IL283291B/en unknown
- 2016-04-07 SG SG10201912912QA patent/SG10201912912QA/en unknown
- 2016-04-07 CR CR20170505A patent/CR20170505A/es unknown
- 2016-04-07 KR KR1020257027291A patent/KR20250126875A/ko active Pending
- 2016-04-07 EA EA201792236A patent/EA201792236A1/ru unknown
- 2016-04-07 CN CN201680033119.6A patent/CN107864657A/zh active Pending
- 2016-04-07 BR BR112017021505A patent/BR112017021505A2/pt not_active Application Discontinuation
- 2016-04-07 WO PCT/US2016/026486 patent/WO2016164609A2/en not_active Ceased
- 2016-04-07 EP EP24207442.5A patent/EP4512429A3/en active Pending
- 2016-04-07 HU HUE16767050A patent/HUE069779T2/hu unknown
- 2016-04-07 SG SG10201909381W patent/SG10201909381WA/en unknown
- 2016-04-07 MX MX2017012935A patent/MX2017012935A/es unknown
- 2016-04-07 US US15/565,020 patent/US10815497B2/en active Active
- 2016-04-07 JP JP2017553005A patent/JP6878299B2/ja active Active
- 2016-04-07 DK DK16767050.4T patent/DK3280799T3/da active
- 2016-04-07 AU AU2016245806A patent/AU2016245806A1/en not_active Abandoned
- 2016-04-07 PL PL16767050.4T patent/PL3280799T3/pl unknown
- 2016-04-07 PE PE2017001997A patent/PE20171800A1/es unknown
- 2016-04-07 ES ES16767050T patent/ES3008382T3/es active Active
- 2016-04-07 EP EP16767050.4A patent/EP3280799B1/en active Active
- 2016-04-08 UY UY0001036611A patent/UY36611A/es not_active Application Discontinuation
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2017
- 2017-10-06 CL CL2017002537A patent/CL2017002537A1/es unknown
- 2017-10-06 PH PH12017501836A patent/PH12017501836A1/en unknown
- 2017-10-06 GT GT201700214A patent/GT201700214A/es unknown
- 2017-10-08 IL IL254933A patent/IL254933B/en active IP Right Grant
- 2017-11-03 CO CONC2017/0011344A patent/CO2017011344A2/es unknown
- 2017-11-08 EC ECIEPI201774016A patent/ECSP17074016A/es unknown
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2021
- 2021-04-28 JP JP2021075526A patent/JP2021118724A/ja not_active Withdrawn
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2022
- 2022-06-07 AU AU2022203942A patent/AU2022203942B2/en active Active
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2023
- 2023-06-14 JP JP2023097370A patent/JP2023116678A/ja active Pending
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2024
- 2024-03-12 AU AU2024201619A patent/AU2024201619A1/en active Pending