JPWO2019195727A5 - - Google Patents
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- JPWO2019195727A5 JPWO2019195727A5 JP2021504131A JP2021504131A JPWO2019195727A5 JP WO2019195727 A5 JPWO2019195727 A5 JP WO2019195727A5 JP 2021504131 A JP2021504131 A JP 2021504131A JP 2021504131 A JP2021504131 A JP 2021504131A JP WO2019195727 A5 JPWO2019195727 A5 JP WO2019195727A5
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- JP
- Japan
- Prior art keywords
- sequence encoding
- sequence
- cells
- item
- vector
- 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.)
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Links
- 239000000203 mixture Substances 0.000 claims description 55
- 210000004027 cells Anatomy 0.000 claims description 47
- 229920002459 Intron Polymers 0.000 claims description 16
- 230000035772 mutation Effects 0.000 claims description 16
- 102000013810 Bestrophin-1 Human genes 0.000 claims description 15
- 108050003623 Bestrophin-1 Proteins 0.000 claims description 15
- 238000002347 injection Methods 0.000 claims description 14
- 239000007924 injection Substances 0.000 claims description 14
- 150000007523 nucleic acids Chemical group 0.000 claims description 13
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 12
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 11
- 239000008194 pharmaceutical composition Substances 0.000 claims description 11
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 10
- 101700047202 BEST1 Proteins 0.000 claims description 10
- 102100007149 BEST1 Human genes 0.000 claims description 10
- 230000003612 virological Effects 0.000 claims description 8
- 208000002780 Macular Degeneration Diseases 0.000 claims description 7
- 206010025425 Maculopathy Diseases 0.000 claims description 7
- 229920001320 Leader sequence (mRNA) Polymers 0.000 claims description 6
- 210000003583 Retinal Pigment Epithelium Anatomy 0.000 claims description 6
- 239000003937 drug carrier Substances 0.000 claims description 6
- 238000001802 infusion Methods 0.000 claims description 6
- 210000004962 mammalian cells Anatomy 0.000 claims description 6
- 229920001405 Coding region Polymers 0.000 claims description 5
- 230000037361 pathway Effects 0.000 claims description 5
- 101000116121 BEST1 Proteins 0.000 claims description 4
- 241000287828 Gallus gallus Species 0.000 claims description 4
- 102100019126 HBB Human genes 0.000 claims description 4
- 108091005902 Hemoglobin subunit beta Proteins 0.000 claims description 4
- 108020004412 RNA 3' Polyadenylation Signals Proteins 0.000 claims description 4
- 241000700159 Rattus Species 0.000 claims description 4
- 241000283984 Rodentia Species 0.000 claims description 4
- 241000251539 Vertebrata <Metazoa> Species 0.000 claims description 4
- 108010006025 bovine growth hormone Proteins 0.000 claims description 4
- 102000030979 human BEST1 protein Human genes 0.000 claims description 4
- 239000002245 particle Substances 0.000 claims description 4
- 210000001525 Retina Anatomy 0.000 claims description 3
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims description 2
- 108020003589 5' Untranslated Regions Proteins 0.000 claims description 2
- 102100011550 ACTB Human genes 0.000 claims description 2
- 101700033661 ACTB Proteins 0.000 claims description 2
- 101710032514 ACTI Proteins 0.000 claims description 2
- 241000432074 Adeno-associated virus Species 0.000 claims description 2
- 241001553178 Arachis glabrata Species 0.000 claims description 2
- 241000282472 Canis lupus familiaris Species 0.000 claims description 2
- 229920002676 Complementary DNA Polymers 0.000 claims description 2
- 241000282326 Felis catus Species 0.000 claims description 2
- 210000002763 Pyramidal Cells Anatomy 0.000 claims description 2
- 229920002533 WHP Posttrascriptional Response Element Polymers 0.000 claims description 2
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 2
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 230000004927 fusion Effects 0.000 claims description 2
- 210000004255 neuroglia Anatomy 0.000 claims description 2
- 210000002569 neurons Anatomy 0.000 claims description 2
- 230000001402 polyadenylating Effects 0.000 claims description 2
- 230000001124 posttranscriptional Effects 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000001105 regulatory Effects 0.000 claims description 2
- 230000002207 retinal Effects 0.000 claims description 2
- 235000020945 retinal Nutrition 0.000 claims description 2
- 235000013601 eggs Nutrition 0.000 claims 2
- 210000003161 Choroid Anatomy 0.000 claims 1
- 241000584079 Cubus Species 0.000 claims 1
- 206010015150 Erythema Diseases 0.000 claims 1
- 241000282412 Homo Species 0.000 claims 1
- 241000699670 Mus sp. Species 0.000 claims 1
- 229930002945 all-trans-retinaldehyde Natural products 0.000 claims 1
- 231100000321 erythema Toxicity 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 239000011604 retinal Substances 0.000 claims 1
- 241000700605 Viruses Species 0.000 description 5
- 241000699666 Mus <mouse, genus> Species 0.000 description 1
- 210000000608 Photoreceptor Cells Anatomy 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000011065 in-situ storage Methods 0.000 description 1
- 210000003867 nerve cell Anatomy 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862653131P | 2018-04-05 | 2018-04-05 | |
| US62/653,131 | 2018-04-05 | ||
| PCT/US2019/026062 WO2019195727A1 (en) | 2018-04-05 | 2019-04-05 | Compositions and methods for treating macular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021520232A JP2021520232A (ja) | 2021-08-19 |
| JPWO2019195727A5 true JPWO2019195727A5 (zh) | 2022-04-12 |
Family
ID=66223872
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021504131A Withdrawn JP2021520232A (ja) | 2018-04-05 | 2019-04-05 | 黄斑ジストロフィーを処置するための組成物及び方法 |
Country Status (21)
| Country | Link |
|---|---|
| US (2) | US20190307900A1 (zh) |
| EP (1) | EP3775233A1 (zh) |
| JP (1) | JP2021520232A (zh) |
| KR (1) | KR20210005040A (zh) |
| CN (1) | CN113056561A (zh) |
| AU (1) | AU2019247864A1 (zh) |
| BR (1) | BR112020020204A2 (zh) |
| CA (1) | CA3096088A1 (zh) |
| CL (1) | CL2020002561A1 (zh) |
| CO (1) | CO2020013690A2 (zh) |
| EA (1) | EA202092069A1 (zh) |
| IL (1) | IL277779A (zh) |
| JO (1) | JOP20200253A1 (zh) |
| MA (1) | MA52199A (zh) |
| MX (1) | MX2020010477A (zh) |
| PE (1) | PE20210918A1 (zh) |
| PH (1) | PH12020551641A1 (zh) |
| RU (1) | RU2020132890A (zh) |
| SG (1) | SG11202009759SA (zh) |
| TW (1) | TW202003052A (zh) |
| WO (1) | WO2019195727A1 (zh) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020086881A1 (en) * | 2018-10-25 | 2020-04-30 | Baxalta Incorporated | Aav triple-plasmid system |
| US20220089670A1 (en) * | 2018-12-28 | 2022-03-24 | University Of Rochester | Gene Therapy for BEST1 Dominant Mutations |
| WO2021174175A1 (en) * | 2020-02-28 | 2021-09-02 | The Trustees Of The University Of Pennsylvania | Treating autosomal recessive bestrophinopathies and methods for evaluating same |
| CN111849998A (zh) * | 2020-07-29 | 2020-10-30 | 武汉纽福斯生物科技有限公司 | 编码人卵黄状黄斑病蛋白1的核酸分子及其应用 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009153563A1 (en) * | 2008-06-18 | 2009-12-23 | Oxford Biomedica (Uk) Limited | Virus purification |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| WO2015075154A2 (en) * | 2013-11-20 | 2015-05-28 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
| KR102281881B1 (ko) * | 2013-12-06 | 2021-07-27 | 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) | 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물 |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| EP3265571B1 (en) * | 2015-03-03 | 2022-04-13 | Fondazione Telethon | Multiple vector system and uses thereof |
| WO2017083722A1 (en) * | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
-
2019
- 2019-04-05 PE PE2020001530A patent/PE20210918A1/es unknown
- 2019-04-05 EA EA202092069A patent/EA202092069A1/ru unknown
- 2019-04-05 RU RU2020132890A patent/RU2020132890A/ru unknown
- 2019-04-05 BR BR112020020204-5A patent/BR112020020204A2/pt not_active Application Discontinuation
- 2019-04-05 US US16/376,808 patent/US20190307900A1/en not_active Abandoned
- 2019-04-05 AU AU2019247864A patent/AU2019247864A1/en not_active Abandoned
- 2019-04-05 KR KR1020207031540A patent/KR20210005040A/ko unknown
- 2019-04-05 EP EP19718557.2A patent/EP3775233A1/en not_active Withdrawn
- 2019-04-05 SG SG11202009759SA patent/SG11202009759SA/en unknown
- 2019-04-05 WO PCT/US2019/026062 patent/WO2019195727A1/en active Application Filing
- 2019-04-05 CA CA3096088A patent/CA3096088A1/en not_active Withdrawn
- 2019-04-05 MA MA052199A patent/MA52199A/fr unknown
- 2019-04-05 JP JP2021504131A patent/JP2021520232A/ja not_active Withdrawn
- 2019-04-05 JO JOP/2020/0253A patent/JOP20200253A1/ar unknown
- 2019-04-05 MX MX2020010477A patent/MX2020010477A/es unknown
- 2019-04-05 CN CN201980031769.0A patent/CN113056561A/zh not_active Withdrawn
- 2019-04-08 TW TW108112207A patent/TW202003052A/zh unknown
-
2020
- 2020-10-02 CL CL2020002561A patent/CL2020002561A1/es unknown
- 2020-10-04 IL IL277779A patent/IL277779A/en unknown
- 2020-10-05 PH PH12020551641A patent/PH12020551641A1/en unknown
- 2020-10-30 CO CONC2020/0013690A patent/CO2020013690A2/es unknown
-
2022
- 2022-09-15 US US17/945,344 patent/US20230149566A1/en not_active Abandoned
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