JP2019500034A5 - - Google Patents
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- Publication number
- JP2019500034A5 JP2019500034A5 JP2018531240A JP2018531240A JP2019500034A5 JP 2019500034 A5 JP2019500034 A5 JP 2019500034A5 JP 2018531240 A JP2018531240 A JP 2018531240A JP 2018531240 A JP2018531240 A JP 2018531240A JP 2019500034 A5 JP2019500034 A5 JP 2019500034A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- optionally
- raav
- gnptab
- pharmaceutical composition
- 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
- 241000702421 Dependoparvovirus Species 0.000 claims 62
- 230000006870 function Effects 0.000 claims 22
- 239000008194 pharmaceutical composition Substances 0.000 claims 22
- 239000002245 particle Substances 0.000 claims 21
- 108020004707 nucleic acids Proteins 0.000 claims 18
- 102000039446 nucleic acids Human genes 0.000 claims 18
- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 101001072470 Homo sapiens N-acetylglucosamine-1-phosphotransferase subunits alpha/beta Proteins 0.000 claims 17
- 102100036710 N-acetylglucosamine-1-phosphotransferase subunits alpha/beta Human genes 0.000 claims 15
- 239000013608 rAAV vector Substances 0.000 claims 13
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 12
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 10
- 241000700605 Viruses Species 0.000 claims 8
- 210000000234 capsid Anatomy 0.000 claims 8
- 101150060971 GNPTAB gene Proteins 0.000 claims 7
- 208000008955 Mucolipidoses Diseases 0.000 claims 7
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 6
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 6
- 241000124008 Mammalia Species 0.000 claims 6
- 125000003275 alpha amino acid group Chemical group 0.000 claims 6
- 238000012217 deletion Methods 0.000 claims 6
- 230000037430 deletion Effects 0.000 claims 6
- 239000003623 enhancer Substances 0.000 claims 6
- 208000024891 symptom Diseases 0.000 claims 6
- 230000008719 thickening Effects 0.000 claims 6
- 206010072928 Mucolipidosis type II Diseases 0.000 claims 5
- 238000010171 animal model Methods 0.000 claims 5
- 210000002845 virion Anatomy 0.000 claims 5
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 4
- 241001164823 Adeno-associated virus - 7 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
- 108700028369 Alleles Proteins 0.000 claims 4
- 241000283690 Bos taurus Species 0.000 claims 4
- 241000283707 Capra Species 0.000 claims 4
- 241000700584 Simplexvirus Species 0.000 claims 4
- 239000003550 marker Substances 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 230000008488 polyadenylation Effects 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 241000701161 unidentified adenovirus Species 0.000 claims 4
- 241000701447 unidentified baculovirus Species 0.000 claims 4
- 208000028698 Cognitive impairment Diseases 0.000 claims 3
- 206010010774 Constipation Diseases 0.000 claims 3
- 206010011878 Deafness Diseases 0.000 claims 3
- 206010012735 Diarrhoea Diseases 0.000 claims 3
- 241001465754 Metazoa Species 0.000 claims 3
- 206010057190 Respiratory tract infections Diseases 0.000 claims 3
- 102100038413 UDP-N-acetylglucosamine-dolichyl-phosphate N-acetylglucosaminephosphotransferase Human genes 0.000 claims 3
- 108010024501 UDPacetylglucosamine-dolichyl-phosphate acetylglucosamine-1-phosphate transferase Proteins 0.000 claims 3
- 210000000988 bone and bone Anatomy 0.000 claims 3
- 230000037182 bone density Effects 0.000 claims 3
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 208000010877 cognitive disease Diseases 0.000 claims 3
- 230000004064 dysfunction Effects 0.000 claims 3
- 230000010370 hearing loss Effects 0.000 claims 3
- 231100000888 hearing loss Toxicity 0.000 claims 3
- 208000016354 hearing loss disease Diseases 0.000 claims 3
- 229910052500 inorganic mineral Inorganic materials 0.000 claims 3
- 239000011707 mineral Substances 0.000 claims 3
- 210000004115 mitral valve Anatomy 0.000 claims 3
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 claims 3
- 210000003205 muscle Anatomy 0.000 claims 3
- 230000000750 progressive effect Effects 0.000 claims 3
- 231100001055 skeletal defect Toxicity 0.000 claims 3
- 206010045458 umbilical hernia Diseases 0.000 claims 3
- 239000013598 vector Substances 0.000 claims 3
- 206010000060 Abdominal distension Diseases 0.000 claims 2
- 206010072929 Mucolipidosis type III Diseases 0.000 claims 2
- 229930193140 Neomycin Natural products 0.000 claims 2
- 241000283984 Rodentia Species 0.000 claims 2
- 108010006025 bovine growth hormone Proteins 0.000 claims 2
- 230000003247 decreasing effect Effects 0.000 claims 2
- 230000003111 delayed effect Effects 0.000 claims 2
- 230000018109 developmental process Effects 0.000 claims 2
- 230000002068 genetic effect Effects 0.000 claims 2
- 102000044577 human GNPTAB Human genes 0.000 claims 2
- 229960004927 neomycin Drugs 0.000 claims 2
- 239000013646 rAAV2 vector Substances 0.000 claims 2
- 210000001015 abdomen Anatomy 0.000 claims 1
- 230000009286 beneficial effect Effects 0.000 claims 1
- 239000000872 buffer Substances 0.000 claims 1
- 230000001934 delay Effects 0.000 claims 1
- 230000008111 motor development Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 210000002345 respiratory system Anatomy 0.000 claims 1
- 150000003384 small molecules Chemical class 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- 230000004584 weight gain Effects 0.000 claims 1
- 235000019786 weight gain Nutrition 0.000 claims 1
- 230000004580 weight loss Effects 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021210071A JP2022046635A (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2024019140A JP2024056832A (ja) | 2015-12-15 | 2024-02-13 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267502P | 2015-12-15 | 2015-12-15 | |
| US62/267,502 | 2015-12-15 | ||
| PCT/US2016/066611 WO2017106313A1 (en) | 2015-12-15 | 2016-12-14 | Adeno-associated viral vectors for treating mucolipidosis type ii |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021210071A Division JP2022046635A (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500034A JP2019500034A (ja) | 2019-01-10 |
| JP2019500034A5 true JP2019500034A5 (enExample) | 2020-01-30 |
| JP7347932B2 JP7347932B2 (ja) | 2023-09-20 |
Family
ID=57737987
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531240A Active JP7347932B2 (ja) | 2015-12-15 | 2016-12-14 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2021210071A Pending JP2022046635A (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2024019140A Pending JP2024056832A (ja) | 2015-12-15 | 2024-02-13 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021210071A Pending JP2022046635A (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2024019140A Pending JP2024056832A (ja) | 2015-12-15 | 2024-02-13 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200261598A1 (enExample) |
| EP (1) | EP3390644B1 (enExample) |
| JP (3) | JP7347932B2 (enExample) |
| KR (1) | KR102833376B1 (enExample) |
| CN (1) | CN108603199A (enExample) |
| AU (2) | AU2016372035B2 (enExample) |
| CA (1) | CA3008268A1 (enExample) |
| ES (1) | ES2991277T3 (enExample) |
| IL (1) | IL259964B (enExample) |
| MX (1) | MX2018007330A (enExample) |
| RU (1) | RU2742612C2 (enExample) |
| SG (2) | SG11201804994QA (enExample) |
| WO (1) | WO2017106313A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019094061A1 (en) | 2017-11-07 | 2019-05-16 | The University Of North Carolina At Chapel Hill | Optimized aga genes and expression cassettes and their use |
| EA202191418A1 (ru) * | 2018-11-21 | 2021-08-05 | Страйдбайо, Инк. | Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения |
| US20220389453A1 (en) * | 2019-10-18 | 2022-12-08 | Research Institute At Nationwide Children's Hospital | Materials and methods for the treatment of disorders associated with the irf2bpl gene |
| MY209860A (en) * | 2020-02-13 | 2025-08-07 | Tenaya Therapeutics Inc | Gene therapy vectors for treating heart disease |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) * | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| 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 |
| EP1073471A4 (en) * | 1998-04-27 | 2005-06-01 | Univ Temple | METHOD OF TREATING ENDOTOXEMIA |
| US6534300B1 (en) * | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| 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 |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| JP2005523942A (ja) * | 2002-04-30 | 2005-08-11 | アヴィオル セラピューティクス インコーポレイテッド | 免疫療法のためのアデノウイルスベクター |
| US7510872B2 (en) | 2003-02-26 | 2009-03-31 | Nationwide Children's Hospital | Recombinant adeno-associated virus production |
| PT1625210E (pt) | 2003-05-21 | 2011-03-15 | Genzyme Corp | Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| PT3252161T (pt) * | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| CN102803478B (zh) | 2009-06-16 | 2016-04-27 | 建新公司 | 用于纯化重组aav载体的改进方法 |
| DK2588130T3 (en) * | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
| 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 |
| JP6224459B2 (ja) | 2011-02-17 | 2017-11-01 | 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 | 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法 |
| EP2970943A4 (en) * | 2013-03-15 | 2017-04-19 | The Children's Hospital of Philadelphia | Vectors comprising stuffer/filler polynucleotide sequences and methods of use |
| CA2927366A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| EP4600255A3 (en) | 2014-05-02 | 2025-10-22 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
-
2016
- 2016-12-14 MX MX2018007330A patent/MX2018007330A/es unknown
- 2016-12-14 CN CN201680081705.8A patent/CN108603199A/zh active Pending
- 2016-12-14 AU AU2016372035A patent/AU2016372035B2/en active Active
- 2016-12-14 JP JP2018531240A patent/JP7347932B2/ja active Active
- 2016-12-14 WO PCT/US2016/066611 patent/WO2017106313A1/en not_active Ceased
- 2016-12-14 SG SG11201804994QA patent/SG11201804994QA/en unknown
- 2016-12-14 SG SG10201912935WA patent/SG10201912935WA/en unknown
- 2016-12-14 US US16/061,579 patent/US20200261598A1/en active Pending
- 2016-12-14 EP EP16822578.7A patent/EP3390644B1/en active Active
- 2016-12-14 ES ES16822578T patent/ES2991277T3/es active Active
- 2016-12-14 RU RU2018124448A patent/RU2742612C2/ru active
- 2016-12-14 KR KR1020187019970A patent/KR102833376B1/ko active Active
- 2016-12-14 CA CA3008268A patent/CA3008268A1/en active Pending
- 2016-12-14 IL IL259964A patent/IL259964B/en unknown
-
2021
- 2021-12-24 JP JP2021210071A patent/JP2022046635A/ja active Pending
-
2023
- 2023-06-28 AU AU2023204116A patent/AU2023204116A1/en active Pending
-
2024
- 2024-02-13 JP JP2024019140A patent/JP2024056832A/ja active Pending