JP2019506445A5 - - Google Patents
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- JP2019506445A5 JP2019506445A5 JP2018549419A JP2018549419A JP2019506445A5 JP 2019506445 A5 JP2019506445 A5 JP 2019506445A5 JP 2018549419 A JP2018549419 A JP 2018549419A JP 2018549419 A JP2018549419 A JP 2018549419A JP 2019506445 A5 JP2019506445 A5 JP 2019506445A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- amino acid
- disease
- viral vector
- parvovirus
- 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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- 108090000565 Capsid Proteins Proteins 0.000 claims 37
- 102100023321 Ceruloplasmin Human genes 0.000 claims 37
- 239000013603 viral vector Substances 0.000 claims 14
- 241000700605 Viruses Species 0.000 claims 12
- 125000000539 amino acid group Chemical group 0.000 claims 12
- 239000013598 vector Substances 0.000 claims 11
- 241000125945 Protoparvovirus Species 0.000 claims 10
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 229920002971 Heparan sulfate Polymers 0.000 claims 4
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 claims 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 210000000234 capsid Anatomy 0.000 claims 4
- 208000011580 syndromic disease Diseases 0.000 claims 4
- 230000010415 tropism Effects 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 206010019280 Heart failures Diseases 0.000 claims 2
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 2
- 206010003246 arthritis Diseases 0.000 claims 2
- 230000007812 deficiency Effects 0.000 claims 2
- 208000019622 heart disease Diseases 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 238000012986 modification Methods 0.000 claims 2
- 230000004048 modification Effects 0.000 claims 2
- 210000003205 muscle Anatomy 0.000 claims 2
- 201000006938 muscular dystrophy Diseases 0.000 claims 2
- 210000002027 skeletal muscle Anatomy 0.000 claims 2
- 230000009885 systemic effect Effects 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 206010003591 Ataxia Diseases 0.000 claims 1
- 208000023275 Autoimmune disease Diseases 0.000 claims 1
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 1
- 206010007559 Cardiac failure congestive Diseases 0.000 claims 1
- 102100022641 Coagulation factor IX Human genes 0.000 claims 1
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims 1
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 208000015178 Hurler syndrome Diseases 0.000 claims 1
- 206010020575 Hyperammonaemia Diseases 0.000 claims 1
- 208000028226 Krabbe disease Diseases 0.000 claims 1
- 108010001831 LDL receptors Proteins 0.000 claims 1
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims 1
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 claims 1
- 206010028289 Muscle atrophy Diseases 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000005764 Peripheral Arterial Disease Diseases 0.000 claims 1
- 208000030831 Peripheral arterial occlusive disease Diseases 0.000 claims 1
- 201000011252 Phenylketonuria Diseases 0.000 claims 1
- 201000001828 Sly syndrome Diseases 0.000 claims 1
- 208000002903 Thalassemia Diseases 0.000 claims 1
- 229960005305 adenosine Drugs 0.000 claims 1
- 208000007502 anemia Diseases 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 206010012601 diabetes mellitus Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 206010015037 epilepsy Diseases 0.000 claims 1
- 210000001508 eye Anatomy 0.000 claims 1
- 235000013922 glutamic acid Nutrition 0.000 claims 1
- 239000004220 glutamic acid Substances 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 229910052739 hydrogen Inorganic materials 0.000 claims 1
- 239000001257 hydrogen Substances 0.000 claims 1
- 206010020718 hyperplasia Diseases 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 208000030159 metabolic disease Diseases 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims 1
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims 1
- 201000006417 multiple sclerosis Diseases 0.000 claims 1
- 201000000585 muscular atrophy Diseases 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 208000015122 neurodegenerative disease Diseases 0.000 claims 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 1
- 201000001119 neuropathy Diseases 0.000 claims 1
- 230000007823 neuropathy Effects 0.000 claims 1
- 208000030613 peripheral artery disease Diseases 0.000 claims 1
- 208000033808 peripheral neuropathy Diseases 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 230000026683 transduction Effects 0.000 claims 1
- 238000010361 transduction Methods 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022016322A JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2024003324A JP2024045227A (ja) | 2015-12-14 | 2024-01-12 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266941P | 2015-12-14 | 2015-12-14 | |
| US62/266,941 | 2015-12-14 | ||
| PCT/US2016/066466 WO2017106236A1 (en) | 2015-12-14 | 2016-12-14 | Modified capsid proteins for enhanced delivery of parvovirus vectors |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022016322A Division JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019506445A JP2019506445A (ja) | 2019-03-07 |
| JP2019506445A5 true JP2019506445A5 (https=) | 2020-01-30 |
| JP7406783B2 JP7406783B2 (ja) | 2023-12-28 |
Family
ID=59057532
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018549419A Active JP7406783B2 (ja) | 2015-12-14 | 2016-12-14 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2022016322A Pending JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2024003324A Pending JP2024045227A (ja) | 2015-12-14 | 2024-01-12 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022016322A Pending JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2024003324A Pending JP2024045227A (ja) | 2015-12-14 | 2024-01-12 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US11124546B2 (https=) |
| EP (2) | EP3390429B1 (https=) |
| JP (3) | JP7406783B2 (https=) |
| CN (2) | CN116478254A (https=) |
| AU (2) | AU2016371779C1 (https=) |
| BR (1) | BR112018011881A2 (https=) |
| CA (1) | CA3008013A1 (https=) |
| DK (1) | DK3390429T3 (https=) |
| ES (1) | ES3040934T3 (https=) |
| MX (2) | MX395524B (https=) |
| PL (1) | PL3390429T3 (https=) |
| RU (1) | RU2751953C2 (https=) |
| WO (1) | WO2017106236A1 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
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| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| EP3384035A4 (en) | 2015-12-02 | 2019-08-07 | Voyager Therapeutics, Inc. | ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES |
| JP7406783B2 (ja) * | 2015-12-14 | 2023-12-28 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| EP3585883A4 (en) * | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| JP2021519612A (ja) * | 2018-03-29 | 2021-08-12 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | 中和を回避する肝臓向性組換えａａｖ６ベクター |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
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| EP4169535A1 (en) * | 2019-04-26 | 2023-04-26 | The University of North Carolina at Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
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| WO2010093784A2 (en) * | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| EP3492597A3 (en) * | 2013-05-21 | 2019-08-28 | University of Florida Research Foundation, Inc. | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
| WO2015048534A1 (en) * | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| US10081659B2 (en) * | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| ES2865487T3 (es) * | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| JP7406783B2 (ja) * | 2015-12-14 | 2023-12-28 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
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2016
- 2016-12-14 JP JP2018549419A patent/JP7406783B2/ja active Active
- 2016-12-14 ES ES16876517T patent/ES3040934T3/es active Active
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- 2016-12-14 WO PCT/US2016/066466 patent/WO2017106236A1/en not_active Ceased
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