JPWO2022036220A5 - - Google Patents
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- Publication number
- JPWO2022036220A5 JPWO2022036220A5 JP2023511556A JP2023511556A JPWO2022036220A5 JP WO2022036220 A5 JPWO2022036220 A5 JP WO2022036220A5 JP 2023511556 A JP2023511556 A JP 2023511556A JP 2023511556 A JP2023511556 A JP 2023511556A JP WO2022036220 A5 JPWO2022036220 A5 JP WO2022036220A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- acid sequence
- aav
- amino acid
- 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.)
- Pending
Links
- 125000003275 alpha amino acid group Chemical group 0.000 claims 18
- 108090000623 proteins and genes Proteins 0.000 claims 18
- 150000007523 nucleic acids Chemical group 0.000 claims 16
- 235000018102 proteins Nutrition 0.000 claims 16
- 102000004169 proteins and genes Human genes 0.000 claims 16
- 210000004027 cell Anatomy 0.000 claims 13
- 239000013608 rAAV vector Substances 0.000 claims 13
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 12
- 235000001014 amino acid Nutrition 0.000 claims 12
- 108700019146 Transgenes Proteins 0.000 claims 11
- 210000000234 capsid Anatomy 0.000 claims 11
- 108090000565 Capsid Proteins Proteins 0.000 claims 10
- 102100023321 Ceruloplasmin Human genes 0.000 claims 10
- 150000001413 amino acids Chemical class 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 9
- 125000003729 nucleotide group Chemical group 0.000 claims 9
- 238000006467 substitution reaction Methods 0.000 claims 7
- 210000004185 liver Anatomy 0.000 claims 6
- 239000013598 vector Substances 0.000 claims 6
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 5
- 210000003169 central nervous system Anatomy 0.000 claims 5
- 239000013607 AAV vector Substances 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 238000000034 method Methods 0.000 claims 3
- 238000004806 packaging method and process Methods 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 231100000304 hepatotoxicity Toxicity 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 230000007056 liver toxicity Effects 0.000 claims 2
- 210000002569 neuron Anatomy 0.000 claims 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 2
- 238000013322 recombinant adeno-associated virus production system Methods 0.000 claims 2
- 230000026683 transduction Effects 0.000 claims 2
- 238000010361 transduction Methods 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 239000004471 Glycine Substances 0.000 claims 1
- 235000009582 asparagine Nutrition 0.000 claims 1
- 150000001508 asparagines Chemical class 0.000 claims 1
- 210000001130 astrocyte Anatomy 0.000 claims 1
- 230000005101 cell tropism Effects 0.000 claims 1
- 210000002987 choroid plexus Anatomy 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000006240 deamidation Effects 0.000 claims 1
- 210000001320 hippocampus Anatomy 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 210000001577 neostriatum Anatomy 0.000 claims 1
- 210000004738 parenchymal cell Anatomy 0.000 claims 1
- 101150066583 rep gene Proteins 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 230000005100 tissue tropism Effects 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063065616P | 2020-08-14 | 2020-08-14 | |
| US63/065,616 | 2020-08-14 | ||
| US202063109734P | 2020-11-04 | 2020-11-04 | |
| US63/109,734 | 2020-11-04 | ||
| PCT/US2021/045945 WO2022036220A1 (en) | 2020-08-14 | 2021-08-13 | Novel aav capsids and compositions containing same |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023537625A JP2023537625A (ja) | 2023-09-04 |
| JPWO2022036220A5 true JPWO2022036220A5 (es) | 2024-08-13 |
Family
ID=77595666
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023511556A Pending JP2023537625A (ja) | 2020-08-14 | 2021-08-13 | 新規ａａｖカプシド及びそれを含む組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230287451A1 (es) |
| EP (1) | EP4196170A1 (es) |
| JP (1) | JP2023537625A (es) |
| KR (1) | KR20230051208A (es) |
| CN (1) | CN116209769A (es) |
| AU (1) | AU2021325954A1 (es) |
| CA (1) | CA3188956A1 (es) |
| IL (1) | IL300410A (es) |
| MX (1) | MX2023001863A (es) |
| TW (1) | TW202221127A (es) |
| WO (1) | WO2022036220A1 (es) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240207452A1 (en) | 2021-04-23 | 2024-06-27 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| IL315256A (en) * | 2022-03-03 | 2024-10-01 | The Trustees Of The Univ Of Pennsylvania | AAV vectors for delivery of GLP-1 receptor agonist fusions |
| WO2024015966A2 (en) * | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| CN117801116A (zh) * | 2022-09-30 | 2024-04-02 | 上海玮美基因科技有限责任公司 | 一种融合型新型腺相关病毒及其应用 |
| WO2024130067A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6174666B1 (en) | 1992-03-27 | 2001-01-16 | The United States Of America As Represented By The Department Of Health And Human Services | Method of eliminating inhibitory/instability regions from mRNA |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| NZ578982A (en) | 2001-11-13 | 2011-03-31 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| EP1486567A1 (en) * | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP2287323A1 (en) | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
| CA2793633A1 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| WO2013155222A2 (en) | 2012-04-10 | 2013-10-17 | The Regents Of The University Of California | Brain-specific enhancers for cell-based therapy |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| JP6741590B2 (ja) | 2014-04-25 | 2020-08-19 | 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 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
-
2021
- 2021-08-13 AU AU2021325954A patent/AU2021325954A1/en active Pending
- 2021-08-13 TW TW110129927A patent/TW202221127A/zh unknown
- 2021-08-13 EP EP21763485.6A patent/EP4196170A1/en active Pending
- 2021-08-13 US US18/040,648 patent/US20230287451A1/en active Pending
- 2021-08-13 CN CN202180070175.8A patent/CN116209769A/zh active Pending
- 2021-08-13 JP JP2023511556A patent/JP2023537625A/ja active Pending
- 2021-08-13 IL IL300410A patent/IL300410A/en unknown
- 2021-08-13 CA CA3188956A patent/CA3188956A1/en active Pending
- 2021-08-13 KR KR1020237007585A patent/KR20230051208A/ko active Search and Examination
- 2021-08-13 MX MX2023001863A patent/MX2023001863A/es unknown
- 2021-08-13 WO PCT/US2021/045945 patent/WO2022036220A1/en active Application Filing
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