JP2020508667A5 - - Google Patents
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- Publication number
- JP2020508667A5 JP2020508667A5 JP2019546041A JP2019546041A JP2020508667A5 JP 2020508667 A5 JP2020508667 A5 JP 2020508667A5 JP 2019546041 A JP2019546041 A JP 2019546041A JP 2019546041 A JP2019546041 A JP 2019546041A JP 2020508667 A5 JP2020508667 A5 JP 2020508667A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- raav
- amino acid
- serotype
- phenylalanine
- 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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- 150000001413 amino acids Chemical group 0.000 claims 27
- 108090000565 Capsid Proteins Proteins 0.000 claims 24
- 102100023321 Ceruloplasmin Human genes 0.000 claims 24
- 235000001014 amino acid Nutrition 0.000 claims 20
- 238000006467 substitution reaction Methods 0.000 claims 16
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 13
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical group OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 claims 13
- 235000003704 aspartic acid Nutrition 0.000 claims 13
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Chemical group OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 13
- 229940024606 amino acid Drugs 0.000 claims 12
- 239000002245 particle Substances 0.000 claims 11
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Chemical group OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims 10
- 235000008729 phenylalanine Nutrition 0.000 claims 10
- COLNVLDHVKWLRT-QMMMGPOBSA-N phenylalanine group Chemical group N[C@@H](CC1=CC=CC=C1)C(=O)O COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims 10
- 239000000203 mixture Substances 0.000 claims 7
- 210000004027 cell Anatomy 0.000 claims 6
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical group C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims 5
- 235000004279 alanine Nutrition 0.000 claims 5
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical group NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 4
- 230000002207 retinal effect Effects 0.000 claims 4
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Chemical group OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 3
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 claims 3
- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical group OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 claims 3
- AYFVYJQAPQTCCC-GBXIJSLDSA-N L-threonine Chemical compound C[C@@H](O)[C@H](N)C(O)=O AYFVYJQAPQTCCC-GBXIJSLDSA-N 0.000 claims 3
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 claims 3
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims 3
- 239000004473 Threonine Substances 0.000 claims 3
- 235000013922 glutamic acid Nutrition 0.000 claims 3
- 239000004220 glutamic acid Chemical group 0.000 claims 3
- 235000013930 proline Nutrition 0.000 claims 3
- 235000018102 proteins Nutrition 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 235000008521 threonine Nutrition 0.000 claims 3
- 239000004471 Glycine Chemical group 0.000 claims 2
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 claims 2
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 239000004474 valine Substances 0.000 claims 2
- 235000014393 valine Nutrition 0.000 claims 2
- 239000004475 Arginine Substances 0.000 claims 1
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims 1
- 108010001857 Cell Surface Receptors Proteins 0.000 claims 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 1
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 1
- 102000019034 Chemokines Human genes 0.000 claims 1
- 108010012236 Chemokines Proteins 0.000 claims 1
- 102000004127 Cytokines Human genes 0.000 claims 1
- 108090000695 Cytokines Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000008394 Immunoglobulin Fragments Human genes 0.000 claims 1
- 108010021625 Immunoglobulin Fragments Proteins 0.000 claims 1
- 102000004310 Ion Channels Human genes 0.000 claims 1
- 108090000862 Ion Channels Proteins 0.000 claims 1
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims 1
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical group OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims 1
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims 1
- 108010052285 Membrane Proteins Proteins 0.000 claims 1
- 101710163270 Nuclease Proteins 0.000 claims 1
- 230000003213 activating effect Effects 0.000 claims 1
- 210000000411 amacrine cell Anatomy 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- 235000009697 arginine Nutrition 0.000 claims 1
- 235000009582 asparagine Nutrition 0.000 claims 1
- 229960001230 asparagine Drugs 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000010362 genome editing Methods 0.000 claims 1
- 239000003292 glue Substances 0.000 claims 1
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 1
- 235000004554 glutamine Nutrition 0.000 claims 1
- 239000003102 growth factor Substances 0.000 claims 1
- 210000002287 horizontal cell Anatomy 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 229940088597 hormone Drugs 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 230000006525 intracellular process Effects 0.000 claims 1
- 102000006240 membrane receptors Human genes 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 210000000608 photoreceptor cell Anatomy 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 239000013646 rAAV2 vector Substances 0.000 claims 1
- 210000003994 retinal ganglion cell Anatomy 0.000 claims 1
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 210000001585 trabecular meshwork Anatomy 0.000 claims 1
- 230000002463 transducing effect Effects 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762461770P | 2017-02-21 | 2017-02-21 | |
| US62/461,770 | 2017-02-21 | ||
| US201862625486P | 2018-02-02 | 2018-02-02 | |
| US62/625,486 | 2018-02-02 | ||
| PCT/US2018/019050 WO2018156654A1 (en) | 2017-02-21 | 2018-02-21 | Modified aav capsid proteins and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020508667A JP2020508667A (ja) | 2020-03-26 |
| JP2020508667A5 true JP2020508667A5 (OSRAM) | 2021-04-08 |
| JP7237843B2 JP7237843B2 (ja) | 2023-03-13 |
Family
ID=63253351
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019546041A Active JP7237843B2 (ja) | 2017-02-21 | 2018-02-21 | 修飾されたａａｖキャプシドタンパク質およびその使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US10793606B2 (OSRAM) |
| EP (1) | EP3585883A4 (OSRAM) |
| JP (1) | JP7237843B2 (OSRAM) |
| AU (1) | AU2018224044B2 (OSRAM) |
| CA (2) | CA3114549A1 (OSRAM) |
| WO (1) | WO2018156654A1 (OSRAM) |
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| EP3633041A3 (en) | 2013-09-26 | 2020-07-29 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| CA3075656A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| US11723988B2 (en) | 2017-10-20 | 2023-08-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector |
| WO2019158619A1 (en) * | 2018-02-15 | 2019-08-22 | Bjoerklund Tomas | Modified viral capsids |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020072451A1 (en) | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| EP3867359A4 (en) * | 2018-10-17 | 2022-09-21 | Children's Medical Research Institute | NUCLEIC ACID MOLECULES AND AAV VECTOR SELECTION METHODS |
| US20220133909A1 (en) | 2019-01-23 | 2022-05-05 | University Of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| US11141425B2 (en) | 2019-06-27 | 2021-10-12 | University Of Florida Research Foundation, Incorporated | Enhancing AAV-mediated transduction of ocular tissues with hyaluronic acid |
| JP2022550435A (ja) | 2019-10-04 | 2022-12-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えａａｖの改善された治療的使用のための方法 |
| IL294781A (en) | 2020-02-14 | 2022-09-01 | Ultragenyx Pharmaceutical Inc | Gene therapy for treating cdkl5 deficiency disorder |
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| JP2023520402A (ja) | 2020-03-31 | 2023-05-17 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | プロピオン酸血症を処置するための遺伝子治療 |
| US20230233708A1 (en) * | 2020-04-14 | 2023-07-27 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated delivery and transduction with polyvinyl alcohol |
| CN111729078B (zh) * | 2020-07-24 | 2020-11-20 | 苏州世诺生物技术有限公司 | 鸡传染性贫血病毒基因工程疫苗 |
| CA3190214A1 (en) | 2020-07-29 | 2022-02-03 | University Of Florida Research Foundation, Incorporated | Improved aav-mediated x-linked retinoschisis therapies |
| KR20230107285A (ko) * | 2020-11-10 | 2023-07-14 | 에이버맥스 바이오파마 인크. | 조작된 바이러스 캡시드 및 사용 방법 |
| TW202235618A (zh) * | 2021-01-05 | 2022-09-16 | 美國馬友醫藥教育研究基金會 | 治療眼內壓相關疾患 |
| WO2023019168A1 (en) | 2021-08-11 | 2023-02-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| AU2022358779A1 (en) | 2021-10-08 | 2024-04-18 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| TW202342740A (zh) | 2022-03-07 | 2023-11-01 | 美商奧崔基尼克斯製藥公司 | 改良的批量aav生產系統和方法 |
| AU2024234833A1 (en) | 2023-03-10 | 2025-10-23 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024229049A1 (en) | 2023-05-01 | 2024-11-07 | University Of Florida Research Foundation, Incorporated | Dual aav vectors for treating stargardt disease |
| WO2025049993A1 (en) | 2023-08-31 | 2025-03-06 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
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| WO2025235555A1 (en) * | 2024-05-06 | 2025-11-13 | University Of Florida Research Foundation, Incorporated | Rational design of aav vectors with limited liver targeting |
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| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| US11124546B2 (en) * | 2015-12-14 | 2021-09-21 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| EP4010357A2 (en) | 2019-08-09 | 2022-06-15 | University of Florida Research Foundation, Incorporated | Aav capsid variants for targeting human glioblastoma cells |
-
2018
- 2018-02-21 CA CA3114549A patent/CA3114549A1/en active Pending
- 2018-02-21 EP EP18756857.1A patent/EP3585883A4/en active Pending
- 2018-02-21 AU AU2018224044A patent/AU2018224044B2/en active Active
- 2018-02-21 WO PCT/US2018/019050 patent/WO2018156654A1/en not_active Ceased
- 2018-02-21 CA CA3054131A patent/CA3054131C/en active Active
- 2018-02-21 JP JP2019546041A patent/JP7237843B2/ja active Active
-
2019
- 2019-08-21 US US16/547,532 patent/US10793606B2/en active Active
-
2020
- 2020-09-01 US US17/009,536 patent/US11332502B2/en active Active
-
2022
- 2022-03-08 US US17/689,324 patent/US11767346B2/en active Active
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