JPWO2020219988A5 - - Google Patents
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- JPWO2020219988A5 JPWO2020219988A5 JP2021563281A JP2021563281A JPWO2020219988A5 JP WO2020219988 A5 JPWO2020219988 A5 JP WO2020219988A5 JP 2021563281 A JP2021563281 A JP 2021563281A JP 2021563281 A JP2021563281 A JP 2021563281A JP WO2020219988 A5 JPWO2020219988 A5 JP WO2020219988A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- item
- aav
- seq
- peptide
- 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
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- 150000007523 nucleic acids Chemical class 0.000 claims description 351
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 329
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 319
- 108020004707 nucleic acids Proteins 0.000 claims description 313
- 102000039446 nucleic acids Human genes 0.000 claims description 313
- 108090000565 Capsid Proteins Proteins 0.000 claims description 299
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 299
- 241000702421 Dependoparvovirus Species 0.000 claims description 238
- 238000000034 method Methods 0.000 claims description 98
- 210000004027 cell Anatomy 0.000 claims description 66
- 108090000623 proteins and genes Proteins 0.000 claims description 59
- 102000004169 proteins and genes Human genes 0.000 claims description 57
- 239000013607 AAV vector Substances 0.000 claims description 51
- 230000004807 localization Effects 0.000 claims description 43
- 210000001519 tissue Anatomy 0.000 claims description 38
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 34
- 150000001413 amino acids Chemical class 0.000 claims description 29
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 25
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 22
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 22
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 22
- 241000958487 Adeno-associated virus 3B Species 0.000 claims description 22
- 230000037431 insertion Effects 0.000 claims description 22
- 238000003780 insertion Methods 0.000 claims description 22
- 230000037432 silent mutation Effects 0.000 claims description 22
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 20
- 102000053171 Glial Fibrillary Acidic Human genes 0.000 claims description 18
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 claims description 18
- 102000009572 RNA Polymerase II Human genes 0.000 claims description 18
- 108010009460 RNA Polymerase II Proteins 0.000 claims description 18
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 claims description 18
- 210000000234 capsid Anatomy 0.000 claims description 17
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 14
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims description 10
- KISWVXRQTGLFGD-UHFFFAOYSA-N 2-[[2-[[6-amino-2-[[2-[[2-[[5-amino-2-[[2-[[1-[2-[[6-amino-2-[(2,5-diamino-5-oxopentanoyl)amino]hexanoyl]amino]-5-(diaminomethylideneamino)pentanoyl]pyrrolidine-2-carbonyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]-5-(diaminomethylideneamino)p Chemical compound C1CCN(C(=O)C(CCCN=C(N)N)NC(=O)C(CCCCN)NC(=O)C(N)CCC(N)=O)C1C(=O)NC(CO)C(=O)NC(CCC(N)=O)C(=O)NC(CCCN=C(N)N)C(=O)NC(CO)C(=O)NC(CCCCN)C(=O)NC(C(=O)NC(CC(C)C)C(O)=O)CC1=CC=C(O)C=C1 KISWVXRQTGLFGD-UHFFFAOYSA-N 0.000 claims description 9
- 101000644085 Homo sapiens Protein unc-45 homolog B Proteins 0.000 claims description 9
- 102100033421 Keratin, type I cytoskeletal 18 Human genes 0.000 claims description 9
- 102100033420 Keratin, type I cytoskeletal 19 Human genes 0.000 claims description 9
- 108010066327 Keratin-18 Proteins 0.000 claims description 9
- 108010066302 Keratin-19 Proteins 0.000 claims description 9
- 101150083522 MECP2 gene Proteins 0.000 claims description 9
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 claims description 9
- 102100039124 Methyl-CpG-binding protein 2 Human genes 0.000 claims description 9
- 108010071690 Prealbumin Proteins 0.000 claims description 9
- 102100021034 Protein unc-45 homolog B Human genes 0.000 claims description 9
- 102000001435 Synapsin Human genes 0.000 claims description 9
- 108050009621 Synapsin Proteins 0.000 claims description 9
- 102000009190 Transthyretin Human genes 0.000 claims description 9
- 101150023087 UNC45B gene Proteins 0.000 claims description 9
- 230000000747 cardiac effect Effects 0.000 claims description 9
- 108010033040 Histones Proteins 0.000 claims description 7
- 210000000633 nuclear envelope Anatomy 0.000 claims description 7
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 6
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 6
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 6
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 6
- 102000004987 Troponin T Human genes 0.000 claims description 6
- 108090001108 Troponin T Proteins 0.000 claims description 6
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 5
- 230000005101 cell tropism Effects 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 4
- 230000005100 tissue tropism Effects 0.000 claims description 4
- 230000002463 transducing effect Effects 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 3
- 239000008194 pharmaceutical composition Substances 0.000 claims description 3
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 174
- 101710197658 Capsid protein VP1 Proteins 0.000 description 9
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 description 6
- 210000004185 liver Anatomy 0.000 description 5
- 125000000539 amino acid group Chemical group 0.000 description 4
- 210000001130 astrocyte Anatomy 0.000 description 4
- 101710197665 Capsid protein VP2 Proteins 0.000 description 3
- 101800001319 Capsid protein VP3 Proteins 0.000 description 3
- 108090000331 Firefly luciferases Proteins 0.000 description 3
- 102000004903 Troponin Human genes 0.000 description 3
- 108090001027 Troponin Proteins 0.000 description 3
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 3
- 108091006047 fluorescent proteins Proteins 0.000 description 3
- 102000034287 fluorescent proteins Human genes 0.000 description 3
- -1 mVenus Proteins 0.000 description 3
- 239000002773 nucleotide Substances 0.000 description 3
- 125000003729 nucleotide group Chemical group 0.000 description 3
- 108010054624 red fluorescent protein Proteins 0.000 description 3
- 210000001744 T-lymphocyte Anatomy 0.000 description 2
- 210000003719 b-lymphocyte Anatomy 0.000 description 2
- 210000004369 blood Anatomy 0.000 description 2
- 239000008280 blood Substances 0.000 description 2
- 210000000988 bone and bone Anatomy 0.000 description 2
- 210000004413 cardiac myocyte Anatomy 0.000 description 2
- 210000000845 cartilage Anatomy 0.000 description 2
- 210000003169 central nervous system Anatomy 0.000 description 2
- 210000001671 embryonic stem cell Anatomy 0.000 description 2
- 210000002889 endothelial cell Anatomy 0.000 description 2
- 210000002919 epithelial cell Anatomy 0.000 description 2
- 210000003238 esophagus Anatomy 0.000 description 2
- 210000001508 eye Anatomy 0.000 description 2
- 210000002216 heart Anatomy 0.000 description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 2
- 210000003494 hepatocyte Anatomy 0.000 description 2
- 210000000936 intestine Anatomy 0.000 description 2
- 210000003734 kidney Anatomy 0.000 description 2
- 210000001865 kupffer cell Anatomy 0.000 description 2
- 210000004072 lung Anatomy 0.000 description 2
- 210000002751 lymph Anatomy 0.000 description 2
- 210000000274 microglia Anatomy 0.000 description 2
- 210000002200 mouth mucosa Anatomy 0.000 description 2
- 210000003205 muscle Anatomy 0.000 description 2
- 210000004498 neuroglial cell Anatomy 0.000 description 2
- 210000002569 neuron Anatomy 0.000 description 2
- 210000004248 oligodendroglia Anatomy 0.000 description 2
- 210000001672 ovary Anatomy 0.000 description 2
- 210000003101 oviduct Anatomy 0.000 description 2
- 210000000496 pancreas Anatomy 0.000 description 2
- 210000002307 prostate Anatomy 0.000 description 2
- 210000004116 schwann cell Anatomy 0.000 description 2
- 238000012163 sequencing technique Methods 0.000 description 2
- 210000000329 smooth muscle myocyte Anatomy 0.000 description 2
- 210000002784 stomach Anatomy 0.000 description 2
- 210000001550 testis Anatomy 0.000 description 2
- 210000003437 trachea Anatomy 0.000 description 2
- 210000000626 ureter Anatomy 0.000 description 2
- 210000003708 urethra Anatomy 0.000 description 2
- 210000003932 urinary bladder Anatomy 0.000 description 2
- 210000004291 uterus Anatomy 0.000 description 2
- 108091081024 Start codon Proteins 0.000 description 1
- 238000000746 purification Methods 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962839421P | 2019-04-26 | 2019-04-26 | |
| US62/839,421 | 2019-04-26 | ||
| US201962915386P | 2019-10-15 | 2019-10-15 | |
| US62/915,386 | 2019-10-15 | ||
| US201962939094P | 2019-11-22 | 2019-11-22 | |
| US62/939,094 | 2019-11-22 | ||
| PCT/US2020/029964 WO2020219988A2 (en) | 2019-04-26 | 2020-04-24 | Engineering aav |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022530457A JP2022530457A (ja) | 2022-06-29 |
| JP2022530457A5 JP2022530457A5 (https=) | 2023-05-01 |
| JPWO2020219988A5 true JPWO2020219988A5 (https=) | 2023-05-01 |
Family
ID=70740764
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021563281A Pending JP2022530457A (ja) | 2019-04-26 | 2020-04-24 | 遺伝子操作ａａｖ |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11981967B2 (https=) |
| EP (1) | EP3959226A2 (https=) |
| JP (1) | JP2022530457A (https=) |
| KR (1) | KR20220012249A (https=) |
| CN (1) | CN114269926A (https=) |
| AU (1) | AU2020261435A1 (https=) |
| CA (1) | CA3137961A1 (https=) |
| IL (1) | IL287406A (https=) |
| SG (1) | SG11202111616RA (https=) |
| TW (1) | TW202106700A (https=) |
| WO (1) | WO2020219988A2 (https=) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| EP3861010A1 (en) | 2018-10-02 | 2021-08-11 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| IL297391A (en) | 2020-04-20 | 2022-12-01 | Tenaya Therapeutics Inc | An adeno-associated virus with an engineered capsid |
| CN116096734A (zh) | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| IL301549A (en) * | 2020-10-01 | 2023-05-01 | Sangamo Therapeutics Inc | Methods of detecting anti-aav antibodies |
| IL301675A (en) | 2020-10-09 | 2023-05-01 | Tenaya Therapeutics Inc | Methods and preparations for placophilin-2 gene therapy |
| EP4259791A1 (en) * | 2020-12-09 | 2023-10-18 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| MX2024004936A (es) | 2021-11-02 | 2024-05-08 | Voyager Therapeutics Inc | Variantes de capsides de aav y sus usos. |
| JP2025500413A (ja) * | 2021-12-23 | 2025-01-09 | プロヴェンション・バイオ・インコーポレイテッド | バース症候群を治療するための方法及び組成物 |
| CN119325517A (zh) * | 2022-04-05 | 2025-01-17 | 生物辐射实验室股份有限公司 | 通过数字试验分析病毒颗粒 |
| WO2024124019A2 (en) * | 2022-12-07 | 2024-06-13 | Ginkgo Bioworks, Inc. | Aav vectors targeting hematopoietic stem cells |
| WO2024120528A1 (en) * | 2022-12-08 | 2024-06-13 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Improved system for producing rna-packaged aav particles |
| CN116789738B (zh) * | 2022-12-08 | 2023-12-19 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2024228938A1 (en) * | 2023-05-01 | 2024-11-07 | St. Jude Children's Research Hospital, Inc. | Modified aav p5 promoter for improved vector titer and purity |
| AU2024274127A1 (en) * | 2023-05-15 | 2025-11-13 | Sangamo Therapeutics, Inc. | Fitness maturation of engineered aav capsid stac-102 |
| AU2024271436A1 (en) * | 2023-05-15 | 2025-11-13 | Sangamo Therapeutics, Inc. | Engineered blood brain barrier penetrant aav capsids |
| WO2025090858A1 (en) * | 2023-10-27 | 2025-05-01 | Biogen Ma Inc. | Methods for identifying aav capsid variants with desired characteristics |
| KR102933125B1 (ko) * | 2024-02-01 | 2026-03-04 | 주식회사 글루진테라퓨틱스 | 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터 |
| WO2025194009A1 (en) * | 2024-03-13 | 2025-09-18 | Sangamo Therapeutics, Inc. | Zinc finger proteins for treating prion disease |
| KR102828335B1 (ko) * | 2024-03-20 | 2025-07-04 | 코오롱인더스트리 주식회사 | Gne 근육병 치료용 aav 기반의 유전자 치료제 |
| WO2025235682A1 (en) * | 2024-05-07 | 2025-11-13 | Sangamo Therapeutics, Inc. | Peptides targeting tfrc for blood-brain barrier crossing |
| WO2025238182A1 (en) * | 2024-05-15 | 2025-11-20 | Stichting Amsterdam UMC | Optimized aav-mediated transduction of cardiomyocytes |
| WO2026017727A1 (en) * | 2024-07-16 | 2026-01-22 | Genethon | Alternative barcoding method to assess transduction efficiency of aav vectors |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| CA2349838C (en) | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| CA2395872A1 (en) * | 2000-01-31 | 2001-08-02 | Human Genome Sciences, Inc. | Nucleic acids, proteins, and antibodies |
| US6916635B2 (en) | 2000-10-02 | 2005-07-12 | The Research Foundation Of State University Of New York | Hybrid adenovirus/adeno-associated virus vectors and methods of use thereof |
| US20050106558A1 (en) | 2001-12-21 | 2005-05-19 | Luca Perabo | Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
| US20080148432A1 (en) * | 2005-12-21 | 2008-06-19 | Mark Scott Abad | Transgenic plants with enhanced agronomic traits |
| AU2006204997B2 (en) * | 2005-01-12 | 2011-09-01 | Monsanto Technology, Llc | Genes and uses for plant improvement |
| JP6208580B2 (ja) | 2010-05-17 | 2017-10-04 | サンガモ セラピューティクス， インコーポレイテッド | 新規のｄｎａ結合タンパク質及びその使用 |
| US20140378362A1 (en) * | 2012-01-18 | 2014-12-25 | Conopco, Inc., D/B/A Unilever | Hair treatment composition |
| US11136557B2 (en) * | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| WO2015048534A1 (en) * | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| WO2017196432A1 (en) * | 2016-05-12 | 2017-11-16 | La Jolla Institute For Allergy And Immunology | Compositions and methods for diagnosing and treating neurodegenerative disease |
| EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| WO2018145111A1 (en) | 2017-02-06 | 2018-08-09 | Children's Medical Center Corporation | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| US20200370039A1 (en) * | 2017-04-11 | 2020-11-26 | Ruprecht-Karls-Universitat Heidelberg | Adeno-associated virus library |
-
2020
- 2020-04-24 EP EP20726605.7A patent/EP3959226A2/en active Pending
- 2020-04-24 CA CA3137961A patent/CA3137961A1/en active Pending
- 2020-04-24 AU AU2020261435A patent/AU2020261435A1/en active Pending
- 2020-04-24 US US16/858,489 patent/US11981967B2/en active Active
- 2020-04-24 JP JP2021563281A patent/JP2022530457A/ja active Pending
- 2020-04-24 WO PCT/US2020/029964 patent/WO2020219988A2/en not_active Ceased
- 2020-04-24 TW TW109113963A patent/TW202106700A/zh unknown
- 2020-04-24 CN CN202080044908.6A patent/CN114269926A/zh active Pending
- 2020-04-24 KR KR1020217038155A patent/KR20220012249A/ko active Pending
- 2020-04-24 SG SG11202111616RA patent/SG11202111616RA/en unknown
-
2021
- 2021-10-19 IL IL287406A patent/IL287406A/en unknown
-
2024
- 2024-04-09 US US18/630,996 patent/US12565685B2/en active Active
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