AR124651A1 - Vectores de aav dirigidos a linfocitos t - Google Patents
Vectores de aav dirigidos a linfocitos tInfo
- Publication number
- AR124651A1 AR124651A1 ARP220100072A ARP220100072A AR124651A1 AR 124651 A1 AR124651 A1 AR 124651A1 AR P220100072 A ARP220100072 A AR P220100072A AR P220100072 A ARP220100072 A AR P220100072A AR 124651 A1 AR124651 A1 AR 124651A1
- Authority
- AR
- Argentina
- Prior art keywords
- seq
- capsid protein
- transduction
- aav
- sequence
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 5
- 108090000565 Capsid Proteins Proteins 0.000 abstract 8
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 8
- 230000026683 transduction Effects 0.000 abstract 5
- 238000010361 transduction Methods 0.000 abstract 5
- 241000700605 Viruses Species 0.000 abstract 4
- 210000000234 capsid Anatomy 0.000 abstract 4
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 4
- 239000013607 AAV vector Substances 0.000 abstract 2
- 150000001413 amino acids Chemical class 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 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 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 210000001744 T-lymphocyte Anatomy 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La descripción proporciona proteínas de la cápside de AAV variantes y cápsides de AAV y vectores de virus que las comprenden. Los vectores de virus descritos en la presente pueden tener una mayor transducción en una célula diana de interés, tal como un linfocito T, en comparación con las secuencias de la cápside de AAV naturales. La descripción también proporciona métodos para administrar los vectores de virus y las cápsides de virus de la descripción a una célula o a un paciente que lo necesita. Reivindicación 1: Un vector de virus adenoasociado recombinante (AAV) que comprende una proteína de la cápside, en donde la proteína de la cápside comprende un péptido asociado a la transducción que tiene la secuencia de cualquiera de las SEQ ID Nº 17 a 23. Reivindicación 5: Un vector de AAV recombinante que comprende una proteína de la cápside, en donde la proteína de la cápside comprende la secuencia de la SEQ ID Nº 1, en donde los aminoácidos 454 - 460 de la SEQ ID Nº 1 se reemplazan con un péptido asociado a la transducción que comprende la secuencia X1-X2-X3-X4-X5-X6-X7 (SEQ ID Nº 24). Reivindicación 24: Un vector de AAV recombinante que comprende una proteína de la cápside, en donde la proteína de la cápside comprende un péptido asociado a la transducción que tiene una secuencia de aminoácidos de la SEQ ID Nº 16, en donde el péptido asociado a la transducción reemplaza los aminoácidos 454 - 460 con respecto a la SEQ ID Nº 1. Reivindicación 26: Un ácido nucleico que codifica una proteína de la cápside de AAV recombinante que tiene la secuencia de cualquiera de las SEQ ID Nº 2, 4, 6, 8, 10, 12 y 14.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163137497P | 2021-01-14 | 2021-01-14 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR124651A1 true AR124651A1 (es) | 2023-04-19 |
Family
ID=81328090
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220100072A AR124651A1 (es) | 2021-01-14 | 2022-01-14 | Vectores de aav dirigidos a linfocitos t |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240123085A1 (es) |
| EP (1) | EP4277920A1 (es) |
| JP (1) | JP2024503091A (es) |
| KR (1) | KR20230135093A (es) |
| CN (1) | CN117203222A (es) |
| AR (1) | AR124651A1 (es) |
| AU (1) | AU2022208037A1 (es) |
| CA (1) | CA3204794A1 (es) |
| TW (1) | TW202242124A (es) |
| WO (1) | WO2022155482A1 (es) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3234664A1 (en) | 2021-10-08 | 2023-04-13 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| WO2024124019A2 (en) * | 2022-12-07 | 2024-06-13 | Ginkgo Bioworks, Inc. | Aav vectors targeting hematopoietic stem cells |
| WO2025179007A1 (en) * | 2024-02-21 | 2025-08-28 | The Regents Of The University Of California | Adeno-associated virus compositions and methods of use thereof for human cells |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0322417A1 (en) | 1986-09-08 | 1989-07-05 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| JPH04501719A (ja) | 1988-11-10 | 1992-03-26 | インペリアル・キヤンサー・リサーチ・テクノロジー・リミテツド | ポリペプチド |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1997022250A1 (en) | 1995-12-15 | 1997-06-26 | Intronn Llc | Therapeutic molecules generated by trans-splicing |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE 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 |
| JP4060531B2 (ja) | 1998-05-28 | 2008-03-12 | アメリカ合衆国 | Ａａｖ５ベクターおよびその使用 |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| 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 |
| DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| ES2327609T3 (es) | 2000-06-01 | 2009-11-02 | University Of North Carolina At Chapel Hill | Procedimientos y compuestos para controlar la liberacion de vectores de parvovirus reconbinantes. |
| WO2001092551A2 (en) | 2000-06-01 | 2001-12-06 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| US7071172B2 (en) | 2002-04-30 | 2006-07-04 | The University Of North Carolina At Chapel Hill | Secretion signal vectors |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| JP4346526B2 (ja) | 2004-08-31 | 2009-10-21 | 株式会社東芝 | 半導体集積回路装置 |
| CA2579519A1 (en) | 2004-09-09 | 2006-03-16 | The General Hospital Corporation | Modulating phosphatase activity in cardiac cells |
| ES2385837T3 (es) | 2004-12-15 | 2012-08-01 | The University Of North Carolina At Chapel Hill | Vectores quiméricos |
| WO2006119432A2 (en) * | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| CN101213203A (zh) | 2005-04-29 | 2008-07-02 | 教堂山北卡罗莱纳州大学 | 在转录后水平调节核酸表达的方法和组合物 |
| AU2007221470B2 (en) | 2006-02-10 | 2013-02-14 | The University Of Cincinnati | Phosphatase inhibitor Protein-1 as a regulator of cardiac function |
| WO2008088895A2 (en) | 2007-01-18 | 2008-07-24 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| CN104937100B (zh) * | 2012-12-25 | 2020-04-03 | 宝生物工程株式会社 | Aav 变体 |
| DK2968605T3 (da) | 2013-03-15 | 2022-09-26 | Univ North Carolina Chapel Hill | Fremgangsmåder og sammensætninger til dobbelte glycanbindende aav-vektorer |
| US11136557B2 (en) * | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| SG11201901757YA (en) * | 2016-09-08 | 2019-03-28 | Bluebird Bio Inc | Pd-1 homing endonuclease variants, compositions, and methods of use |
| EP3589277A4 (en) * | 2017-02-28 | 2020-08-26 | Adverum Biotechnologies, Inc. | MODIFIED AVA CAPSIDES AND USE OF THESE LATEST |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
-
2022
- 2022-01-13 TW TW111101402A patent/TW202242124A/zh unknown
- 2022-01-14 AR ARP220100072A patent/AR124651A1/es unknown
- 2022-01-14 CN CN202280016202.8A patent/CN117203222A/zh active Pending
- 2022-01-14 AU AU2022208037A patent/AU2022208037A1/en active Pending
- 2022-01-14 WO PCT/US2022/012542 patent/WO2022155482A1/en not_active Ceased
- 2022-01-14 CA CA3204794A patent/CA3204794A1/en active Pending
- 2022-01-14 KR KR1020237026711A patent/KR20230135093A/ko active Pending
- 2022-01-14 EP EP22703750.4A patent/EP4277920A1/en not_active Withdrawn
- 2022-01-14 JP JP2023542960A patent/JP2024503091A/ja active Pending
-
2023
- 2023-07-12 US US18/221,211 patent/US20240123085A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20240123085A1 (en) | 2024-04-18 |
| TW202242124A (zh) | 2022-11-01 |
| CA3204794A1 (en) | 2022-07-21 |
| JP2024503091A (ja) | 2024-01-24 |
| WO2022155482A1 (en) | 2022-07-21 |
| CN117203222A (zh) | 2023-12-08 |
| AU2022208037A1 (en) | 2023-07-20 |
| WO2022155482A9 (en) | 2022-12-22 |
| KR20230135093A (ko) | 2023-09-22 |
| EP4277920A1 (en) | 2023-11-22 |
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