JP2018522529A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018522529A5 JP2018522529A5 JP2017518931A JP2017518931A JP2018522529A5 JP 2018522529 A5 JP2018522529 A5 JP 2018522529A5 JP 2017518931 A JP2017518931 A JP 2017518931A JP 2017518931 A JP2017518931 A JP 2017518931A JP 2018522529 A5 JP2018522529 A5 JP 2018522529A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- recombinant aav
- aav vector
- sequence
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims 46
- 239000013607 AAV vector Substances 0.000 claims 37
- 108020004707 nucleic acids Proteins 0.000 claims 28
- 102000039446 nucleic acids Human genes 0.000 claims 28
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 17
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 11
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 11
- 241000702421 Dependoparvovirus Species 0.000 claims 7
- 239000003623 enhancer Substances 0.000 claims 7
- 229960000027 human factor ix Drugs 0.000 claims 7
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 210000000234 capsid Anatomy 0.000 claims 6
- 238000000034 method Methods 0.000 claims 5
- 101710095339 Apolipoprotein E Proteins 0.000 claims 4
- 102100029470 Apolipoprotein E Human genes 0.000 claims 4
- 238000004806 packaging method and process Methods 0.000 claims 4
- 102100022641 Coagulation factor IX Human genes 0.000 claims 3
- 108091092195 Intron Proteins 0.000 claims 3
- 101100172173 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) hcr-1 gene Proteins 0.000 claims 3
- 208000009429 hemophilia B Diseases 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 108091033319 polynucleotide Proteins 0.000 claims 3
- 102000040430 polynucleotide Human genes 0.000 claims 3
- 239000002157 polynucleotide Substances 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 108010013935 factor IX-Padua Proteins 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 239000002245 particle Substances 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 238000013518 transcription Methods 0.000 claims 2
- 230000035897 transcription Effects 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 102000053602 DNA Human genes 0.000 claims 1
- 101000823116 Homo sapiens Alpha-1-antitrypsin Proteins 0.000 claims 1
- 208000026578 Severe hemophilia B Diseases 0.000 claims 1
- 108020004682 Single-Stranded DNA Proteins 0.000 claims 1
- 102000051631 human SERPINA1 Human genes 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021012811A JP7361737B2 (ja) | 2015-06-23 | 2021-01-29 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
| JP2023171763A JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562183599P | 2015-06-23 | 2015-06-23 | |
| US62/183,599 | 2015-06-23 | ||
| US201662315453P | 2016-03-30 | 2016-03-30 | |
| US62/315,453 | 2016-03-30 | ||
| US201662338315P | 2016-05-18 | 2016-05-18 | |
| US62/338,315 | 2016-05-18 | ||
| US201662348781P | 2016-06-10 | 2016-06-10 | |
| US62/348,781 | 2016-06-10 | ||
| US201662349572P | 2016-06-13 | 2016-06-13 | |
| US62/349,572 | 2016-06-13 | ||
| PCT/US2016/039075 WO2016210170A1 (en) | 2015-06-23 | 2016-06-23 | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021012811A Division JP7361737B2 (ja) | 2015-06-23 | 2021-01-29 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018522529A JP2018522529A (ja) | 2018-08-16 |
| JP2018522529A5 true JP2018522529A5 (enExample) | 2019-07-25 |
| JP6831779B2 JP6831779B2 (ja) | 2021-02-17 |
Family
ID=57586246
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017518931A Active JP6831779B2 (ja) | 2015-06-23 | 2016-06-23 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
| JP2021012811A Active JP7361737B2 (ja) | 2015-06-23 | 2021-01-29 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
| JP2023171763A Pending JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021012811A Active JP7361737B2 (ja) | 2015-06-23 | 2021-01-29 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
| JP2023171763A Pending JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Country Status (25)
| Country | Link |
|---|---|
| US (6) | US10799566B2 (enExample) |
| EP (2) | EP4378487A3 (enExample) |
| JP (3) | JP6831779B2 (enExample) |
| KR (2) | KR102415896B1 (enExample) |
| CN (1) | CN108138159A (enExample) |
| AU (2) | AU2016282781A1 (enExample) |
| CA (1) | CA2990193A1 (enExample) |
| CO (1) | CO2018000134A2 (enExample) |
| DK (1) | DK3313991T3 (enExample) |
| ES (1) | ES2989774T3 (enExample) |
| FI (2) | FI3313991T3 (enExample) |
| FR (1) | FR25C1002I2 (enExample) |
| HR (1) | HRP20241180T1 (enExample) |
| HU (2) | HUE068603T2 (enExample) |
| IL (2) | IL309741A (enExample) |
| MX (2) | MX390848B (enExample) |
| NO (1) | NO2025003I1 (enExample) |
| PE (1) | PE20180675A1 (enExample) |
| PH (1) | PH12017502424A1 (enExample) |
| PL (1) | PL3313991T3 (enExample) |
| PT (1) | PT3313991T (enExample) |
| SA (1) | SA517390590B1 (enExample) |
| SG (1) | SG10202100131WA (enExample) |
| SI (1) | SI3313991T1 (enExample) |
| WO (1) | WO2016210170A1 (enExample) |
Families Citing this family (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101203613B (zh) | 2005-04-07 | 2012-12-12 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
| HRP20230259T1 (hr) | 2008-09-15 | 2023-04-28 | Uniqure Biopharma B.V. | Mutant polipeptida faktora ix, njegove primjene i postupak za njegovu proizvodnju |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| MX2016001044A (es) | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| CN108138159A (zh) * | 2015-06-23 | 2018-06-08 | 费城儿童医院 | 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 |
| CA3039673A1 (en) | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| CN117801075A (zh) * | 2017-03-15 | 2024-04-02 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
| CN118581121A (zh) * | 2017-05-22 | 2024-09-03 | 武田药品工业株式会社 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| KR20240010098A (ko) * | 2017-05-31 | 2024-01-23 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| EP3652326B1 (en) * | 2017-07-10 | 2024-10-09 | uniQure IP B.V. | Means and methods for aav gene therapy in humans |
| IL319982A (en) | 2017-12-19 | 2025-05-01 | Akouos Inc | AAV virus-mediated delivery of therapeutic antibodies to the inner ear |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| CN112955174A (zh) | 2018-07-09 | 2021-06-11 | 旗舰先锋创新V股份有限公司 | 融合剂脂质体组合物和其用途 |
| WO2020020475A1 (en) * | 2018-07-27 | 2020-01-30 | Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| MX2021001901A (es) * | 2018-08-20 | 2021-05-27 | Ucl Business Ltd | Nucleotidos de codificacion del factor ix. |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB2576508A (en) * | 2018-08-20 | 2020-02-26 | Ucl Business Plc | Factor IX encoding nucleotides |
| WO2020041773A1 (en) | 2018-08-24 | 2020-02-27 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020082046A2 (en) | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| WO2020097417A1 (en) * | 2018-11-09 | 2020-05-14 | Generation Bio Co. | Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats |
| WO2020142752A1 (en) * | 2019-01-04 | 2020-07-09 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| US11596671B2 (en) | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
| EP3826664A4 (en) * | 2019-02-06 | 2022-10-26 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| CN110205297A (zh) * | 2019-05-15 | 2019-09-06 | 华北理工大学 | 一种重组脂肪干细胞及其重组方法 |
| EP4022097A4 (en) * | 2019-08-29 | 2022-12-14 | Siemens Healthcare Diagnostics Inc. | REAGENTS AND METHODS FOR DETECTING AAV EXCRETION |
| CN111647625A (zh) * | 2019-12-25 | 2020-09-11 | 深圳三智医学科技有限公司 | 一种提高人凝血因子ix表达水平的方法 |
| MX2022011805A (es) * | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| WO2021207415A1 (en) * | 2020-04-07 | 2021-10-14 | The Curators Of The University Of Missouri | CpG-FREE ITRs FOR AAV GENE THERAPY |
| CN113817759B (zh) * | 2020-07-10 | 2023-06-02 | 南京吉迈生物技术有限公司 | 修饰的因子ix、组合物、方法及其在基因治疗中的应用 |
| WO2022097008A1 (en) | 2020-11-03 | 2022-05-12 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| CA3197936A1 (en) | 2020-12-01 | 2022-06-09 | Akouos, Inc. | Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms |
| WO2022130172A1 (en) | 2020-12-15 | 2022-06-23 | Pfizer Inc. | Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins |
| AU2021404944A1 (en) | 2020-12-23 | 2023-07-06 | Pfizer Inc. | Methods for purification of aav vectors by affinity chromatography |
| TW202246505A (zh) * | 2021-03-05 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途 |
| WO2022208342A1 (en) | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| EP4079860A1 (en) * | 2021-04-23 | 2022-10-26 | uniQure biopharma B.V. | Methods and means for the prevention and/or treatment of joint damage in hemophilia |
| WO2022229807A1 (en) | 2021-04-26 | 2022-11-03 | Pfizer Inc. | Adeno-associated viral vector capsids with improved tissue tropism |
| EP4334454A2 (en) * | 2021-05-04 | 2024-03-13 | RegenxBio Inc. | Novel aav vectors and methods and uses thereof |
| US20240271160A1 (en) | 2021-06-22 | 2024-08-15 | Pfizer Inc. | Production of adeno-associated virus vector in insect cells |
| WO2022268110A1 (zh) * | 2021-06-23 | 2022-12-29 | 英斯培瑞有限公司 | 用于治疗b型血友病的组合物和方法 |
| WO2023069979A1 (en) | 2021-10-20 | 2023-04-27 | University Of Rochester | Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss |
| EP4423119A1 (en) * | 2021-10-27 | 2024-09-04 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
| EP4426331A1 (en) | 2021-11-02 | 2024-09-11 | University of Rochester | Tcf7l2 mediated remyelination in the brain |
| EP4198134A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Gamma-sarcoglycan gene transfer increase using modified itr sequences |
| WO2023202469A1 (zh) * | 2022-04-19 | 2023-10-26 | 康霖生物科技（杭州）有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途 |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024038365A1 (en) | 2022-08-16 | 2024-02-22 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| TW202426479A (zh) * | 2022-10-10 | 2024-07-01 | 美商星火治療公司 | Apoe基因療法 |
| WO2024091824A1 (en) | 2022-10-26 | 2024-05-02 | Ada Forsyth Institute, Inc. | Differentiation and reprogramming of chondrocyte |
| EP4658294A2 (en) | 2023-02-02 | 2025-12-10 | University of Rochester | Competitive replacement of glial cells |
| WO2025090427A1 (en) | 2023-10-23 | 2025-05-01 | University Of Rochester | Glial-targeted relief of hyperexcitability in neurodegenerative diseases |
| WO2025143742A1 (ko) * | 2023-12-26 | 2025-07-03 | 삼성바이오에피스 주식회사 | 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드 |
| WO2025250457A1 (en) | 2024-05-28 | 2025-12-04 | University Of Rochester | Enhanced brain transduction by gene therapeutics |
Family Cites Families (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6468524B1 (en) | 2000-03-22 | 2002-10-22 | The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | AAV4 vector and uses thereof |
| US7351813B2 (en) | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
| US6943012B2 (en) | 2001-03-26 | 2005-09-13 | The Board Of Trustees Of The Leland Stanford Junor University | Helper dependent adenoviral vector system and methods for using the same |
| US20080220015A1 (en) | 2001-08-03 | 2008-09-11 | Nokad, S.A. | Method of modulating neutralizing antibodies formation in mammals, and uses thereof in gene therapy, animal trangenesis and in functional inactivation of endogenous proteins |
| KR101014207B1 (ko) | 2001-11-13 | 2011-02-14 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 아데노-결합 바이러스 (ａａｖ) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법 |
| CA2469785C (en) | 2001-12-17 | 2014-04-22 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| WO2003082195A2 (en) | 2002-03-22 | 2003-10-09 | Board Of Regents, The University Of Texas System | Protamine-adenoviral vector complexes and methods of use |
| SI1496944T1 (sl) | 2002-05-01 | 2009-02-28 | Univ Florida | Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov |
| WO2005037226A2 (en) * | 2003-10-17 | 2005-04-28 | Georgia Tech Research Corporation | Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders |
| DK1804839T3 (da) | 2004-09-22 | 2012-04-10 | St Jude Childrens Res Hospital | Forbedret ekspression af faktor ix i genterapivektorer |
| EP1799826B1 (en) | 2004-09-29 | 2009-08-12 | Children's Memorial Hospital | siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN |
| CN101203613B (zh) | 2005-04-07 | 2012-12-12 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| WO2008027084A2 (en) | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
| WO2007149852A2 (en) * | 2006-06-19 | 2007-12-27 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| CA2722238C (en) | 2008-04-22 | 2017-11-28 | Life Sciences Research Partners Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| HRP20230259T1 (hr) | 2008-09-15 | 2023-04-28 | Uniqure Biopharma B.V. | Mutant polipeptida faktora ix, njegove primjene i postupak za njegovu proizvodnju |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| SG178408A1 (en) * | 2009-08-18 | 2012-03-29 | Baxter Int | Aptamers to tissue factor pathway inhibitor and their use as bleeding disorder therapeutics |
| EP2561067B1 (en) | 2010-04-23 | 2018-11-14 | University of Florida Research Foundation, Inc. | Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1) |
| WO2011133890A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| KR101853405B1 (ko) | 2010-10-20 | 2018-05-02 | 주식회사 티움바이오 | 인자 ｉｘ 활성을 갖는 융합 단백질 |
| WO2013078400A1 (en) | 2011-11-22 | 2013-05-30 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| WO2013078316A1 (en) * | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| CA2864126A1 (en) | 2012-02-15 | 2013-08-22 | Biogen Idec Ma Inc. | Recombinant factor viii proteins |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| KR102063483B1 (ko) | 2012-04-18 | 2020-01-08 | 더 칠드런스 호스피탈 오브 필라델피아 | Ａａｖ 캡시드 변이체를 이용한 고효율 유전자 전달을 위한 조성물 및 방법 |
| CA2876293C (en) * | 2012-06-27 | 2023-10-10 | Arthrogen B.V. | Combination for treating an inflammatory disorder |
| GB201213117D0 (en) | 2012-07-24 | 2012-09-05 | Ucl Business Plc | Transgene expression |
| US10398787B2 (en) | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
| US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| US9700486B2 (en) * | 2013-04-24 | 2017-07-11 | Corning Incorporated | Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients |
| EP3019620A4 (en) | 2013-07-12 | 2017-01-18 | The Children's Hospital of Philadelphia | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
| MX2016001044A (es) * | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| NL2012981B1 (en) * | 2014-06-11 | 2017-01-17 | Heineken Supply Chain Bv | Beverage dispensing system, beverage container and pressurizing system for use in a beverage dispensing system or container. |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| US11007280B2 (en) | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| CN108138159A (zh) | 2015-06-23 | 2018-06-08 | 费城儿童医院 | 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
-
2016
- 2016-06-23 CN CN201680043108.6A patent/CN108138159A/zh active Pending
- 2016-06-23 PT PT168153310T patent/PT3313991T/pt unknown
- 2016-06-23 HU HUE16815331A patent/HUE068603T2/hu unknown
- 2016-06-23 EP EP24165196.7A patent/EP4378487A3/en active Pending
- 2016-06-23 KR KR1020207032060A patent/KR102415896B1/ko active Active
- 2016-06-23 MX MX2018000307A patent/MX390848B/es unknown
- 2016-06-23 PL PL16815331.0T patent/PL3313991T3/pl unknown
- 2016-06-23 SI SI201631854T patent/SI3313991T1/sl unknown
- 2016-06-23 HR HRP20241180TT patent/HRP20241180T1/hr unknown
- 2016-06-23 ES ES16815331T patent/ES2989774T3/es active Active
- 2016-06-23 WO PCT/US2016/039075 patent/WO2016210170A1/en not_active Ceased
- 2016-06-23 KR KR1020187002040A patent/KR102178322B1/ko active Active
- 2016-06-23 EP EP16815331.0A patent/EP3313991B1/en active Active
- 2016-06-23 US US15/191,357 patent/US10799566B2/en active Active
- 2016-06-23 IL IL309741A patent/IL309741A/en unknown
- 2016-06-23 FI FIEP16815331.0T patent/FI3313991T3/fi active
- 2016-06-23 PE PE2017002835A patent/PE20180675A1/es unknown
- 2016-06-23 JP JP2017518931A patent/JP6831779B2/ja active Active
- 2016-06-23 DK DK16815331.0T patent/DK3313991T3/da active
- 2016-06-23 IL IL256517A patent/IL256517B2/en unknown
- 2016-06-23 AU AU2016282781A patent/AU2016282781A1/en not_active Abandoned
- 2016-06-23 CA CA2990193A patent/CA2990193A1/en active Pending
- 2016-06-23 SG SG10202100131WA patent/SG10202100131WA/en unknown
-
2017
- 2017-12-21 SA SA517390590A patent/SA517390590B1/ar unknown
- 2017-12-22 PH PH12017502424A patent/PH12017502424A1/en unknown
-
2018
- 2018-01-08 MX MX2022003116A patent/MX2022003116A/es unknown
- 2018-01-09 CO CONC2018/0000134A patent/CO2018000134A2/es unknown
-
2019
- 2019-12-09 US US16/707,984 patent/US20200129598A1/en not_active Abandoned
-
2020
- 2020-09-08 US US17/014,782 patent/US11110153B2/en active Active
-
2021
- 2021-01-29 JP JP2021012811A patent/JP7361737B2/ja active Active
- 2021-07-12 US US17/305,649 patent/US11491213B2/en active Active
-
2022
- 2022-10-28 US US18/050,982 patent/US11896652B2/en active Active
- 2022-11-09 AU AU2022268345A patent/AU2022268345B2/en active Active
-
2023
- 2023-10-03 JP JP2023171763A patent/JP2024009857A/ja active Pending
- 2023-12-19 US US18/389,662 patent/US20240131126A1/en active Pending
-
2025
- 2025-01-03 FI FIC20250001C patent/FIC20250001I1/fi unknown
- 2025-01-07 HU HUS2500003C patent/HUS2500003I1/hu unknown
- 2025-01-07 NO NO2025003C patent/NO2025003I1/no unknown
- 2025-01-09 FR FR25C1002C patent/FR25C1002I2/fr active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018522529A5 (enExample) | ||
| JP2024009857A5 (enExample) | ||
| JP2021106619A5 (enExample) | ||
| JP2021087431A5 (enExample) | ||
| JP2017509632A5 (enExample) | ||
| JP2019116492A5 (enExample) | ||
| JP2020522269A5 (enExample) | ||
| JP2018510648A5 (enExample) | ||
| JP2021003120A5 (enExample) | ||
| JP2020114235A5 (enExample) | ||
| US12084654B2 (en) | Methods for using transcription-dependent directed evolution of AAV capsids | |
| JP2019503649A5 (enExample) | ||
| RU2018119710A (ru) | Кодирующие фактор viii варианты с пониженным содержанием cpg, композиции и способы, и применение в лечении нарушений гемостаза | |
| JP2007507223A5 (enExample) | ||
| JPWO2020106916A5 (enExample) | ||
| JP2020519284A5 (enExample) | ||
| JP2018506261A5 (enExample) | ||
| JP2024099770A5 (enExample) | ||
| JP2020054367A (ja) | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 | |
| JP2020533276A5 (enExample) | ||
| JP2017529395A5 (enExample) | ||
| IL292264B2 (en) | aav transfer cassette | |
| JPWO2021067448A5 (enExample) | ||
| JPWO2020214609A5 (enExample) | ||
| CN120548368A (zh) | 用于正确包装重复元件的腺相关病毒载体 |