MX2020008932A - Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. - Google Patents
Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.Info
- Publication number
- MX2020008932A MX2020008932A MX2020008932A MX2020008932A MX2020008932A MX 2020008932 A MX2020008932 A MX 2020008932A MX 2020008932 A MX2020008932 A MX 2020008932A MX 2020008932 A MX2020008932 A MX 2020008932A MX 2020008932 A MX2020008932 A MX 2020008932A
- Authority
- MX
- Mexico
- Prior art keywords
- aav
- vectors
- capsid
- associated virus
- deamydation
- Prior art date
Links
Classifications
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- 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
-
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Un vector de virus adenoasociado recombinante (rAAV) que comprende una capside de AAV que tiene una población heterogénea de proteinas vp1, una población heterogénea de proteinas vp2 y una población heterogénea de proteinas vp3; la capside contiene aminoácidos modificados en comparación con la secuencia de aminoácidos de VP1 codificada, en donde la capside contiene residuos de asparagina altamente desamidada en el par asparagina - glicina y comprende ademas muchos otros residuos de asparagina menos desamidada y, opcionalmente, residuos de glutamina.
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862635968P | 2018-02-27 | 2018-02-27 | |
| US201862635964P | 2018-02-27 | 2018-02-27 | |
| US201862663788P | 2018-04-27 | 2018-04-27 | |
| US201862663797P | 2018-04-27 | 2018-04-27 | |
| US201862667587P | 2018-05-06 | 2018-05-06 | |
| US201862667888P | 2018-05-07 | 2018-05-07 | |
| US201862667881P | 2018-05-07 | 2018-05-07 | |
| US201862667585P | 2018-05-29 | 2018-05-29 | |
| US201862677474P | 2018-05-29 | 2018-05-29 | |
| US201862677471P | 2018-05-29 | 2018-05-29 | |
| US201862703670P | 2018-07-26 | 2018-07-26 | |
| US201862703673P | 2018-07-26 | 2018-07-26 | |
| US201862722388P | 2018-08-24 | 2018-08-24 | |
| US201862722382P | 2018-08-24 | 2018-08-24 | |
| PCT/US2019/019861 WO2019169004A1 (en) | 2018-02-27 | 2019-02-27 | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020008932A true MX2020008932A (es) | 2020-10-01 |
Family
ID=67809202
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020008932A MX2020008932A (es) | 2018-02-27 | 2019-02-27 | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US12416016B2 (es) |
| EP (1) | EP3768695A4 (es) |
| JP (2) | JP7498665B2 (es) |
| KR (1) | KR102848518B1 (es) |
| CN (2) | CN112236443B (es) |
| AU (1) | AU2019228504B2 (es) |
| BR (1) | BR112020017278A2 (es) |
| CA (1) | CA3091795A1 (es) |
| CL (1) | CL2020002201A1 (es) |
| IL (1) | IL276860A (es) |
| MA (1) | MA52087A (es) |
| MX (1) | MX2020008932A (es) |
| SG (1) | SG11202007942YA (es) |
| WO (1) | WO2019169004A1 (es) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7064214B2 (ja) | 2015-09-28 | 2022-05-10 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 抗体回避性ウイルスベクターのための方法および組成物 |
| MX2020008932A (es) * | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| MX2020010464A (es) | 2018-04-03 | 2021-01-29 | Vectores de virus que evitan anticuerpos. | |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| WO2020132385A1 (en) | 2018-12-21 | 2020-06-25 | The University Of North Carolina At Chapel Hill | Optimized galc genes and expression cassettes and their use |
| JP7698583B2 (ja) | 2019-03-21 | 2025-06-25 | ギンコ バイオワークス インコーポレイテッド | 組換えアデノ随伴ウイルスベクター |
| US12516351B2 (en) | 2019-04-29 | 2026-01-06 | The Trustees Of The University Of Pennsylvania | AAV capsids and compositions containing same |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| US20230002788A1 (en) * | 2019-10-21 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Aav3b variants with improved production yield and liver tropism |
| CA3165911A1 (en) * | 2020-01-03 | 2021-07-08 | Sarepta Therapeutics, Inc. | Methods for analyzing aav capsid proteins |
| US12497612B2 (en) | 2020-03-09 | 2025-12-16 | University Of Massachusetts | Gene replacement therapy for FOXG1 syndrome |
| BR112022021134A2 (pt) | 2020-04-20 | 2023-02-14 | Tenaya Therapeutics Inc | Vírus adeno-associado com capsídeo projetado |
| EP4143308A4 (en) * | 2020-04-27 | 2024-06-26 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY |
| IL299167A (en) | 2020-06-17 | 2023-02-01 | Univ Pennsylvania | Compositions and methods for treating patients with gene therapy |
| BR112023000578A2 (pt) | 2020-07-13 | 2023-05-09 | Univ Pennsylvania | Composições úteis para tratamento de doença de charcot-marie-tooth |
| CA3189878A1 (en) | 2020-08-19 | 2022-02-24 | Colin O'BANION | Adeno-associated virus vectors for treatment of rett syndrome |
| US20230383313A1 (en) * | 2020-10-18 | 2023-11-30 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| MX2023012052A (es) | 2021-04-12 | 2024-03-15 | Univ Pennsylvania | Composiciones útiles para tratar la atrofia muscular espinal y bulbar (sbma). |
| WO2023081807A1 (en) * | 2021-11-04 | 2023-05-11 | The Trustees of the University of Pennsylvania Penn Center for Innovation | Compositions and methods for reducing pcsk9 levels in a subject |
| AR128239A1 (es) | 2022-01-10 | 2024-04-10 | Univ Pennsylvania | Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72 |
| US20250177495A1 (en) | 2022-01-10 | 2025-06-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| AU2024245299A1 (en) * | 2023-03-25 | 2025-09-25 | Duke University | Compositions comprising kidney-tropic aavs and methods of use thereof |
| WO2025250683A1 (en) * | 2024-05-29 | 2025-12-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
Family Cites Families (38)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| 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 |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| WO2004083441A2 (en) * | 2003-03-19 | 2004-09-30 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Random peptide library displayed on aav vectors |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US20060246079A1 (en) | 2003-11-14 | 2006-11-02 | Morrow Phillip R | Neutralizing human antibodies to anthrax toxin |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| KR20230067694A (ko) * | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| US20180057839A1 (en) * | 2014-11-26 | 2018-03-01 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| CA3003435A1 (en) | 2015-10-28 | 2017-05-04 | The Trustees Of The University Of Pennsylvania | Intrathecal administration of adeno-associated-viral vectors for gene therapy |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| AU2017248656B2 (en) * | 2016-04-15 | 2023-07-27 | The Trustees Of The University Of Pennsylvania | Novel AAV8 mutant capsids and compositions containing same |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| IL263801B2 (en) * | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| US11698377B2 (en) * | 2016-08-15 | 2023-07-11 | Genzyme Corporation | Methods for detecting AAV |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
| MX2020008932A (es) * | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
-
2019
- 2019-02-27 MX MX2020008932A patent/MX2020008932A/es unknown
- 2019-02-27 JP JP2020545159A patent/JP7498665B2/ja active Active
- 2019-02-27 KR KR1020207027462A patent/KR102848518B1/ko active Active
- 2019-02-27 MA MA052087A patent/MA52087A/fr unknown
- 2019-02-27 CA CA3091795A patent/CA3091795A1/en active Pending
- 2019-02-27 CN CN201980029022.1A patent/CN112236443B/zh active Active
- 2019-02-27 CN CN202510015875.7A patent/CN120519403A/zh active Pending
- 2019-02-27 SG SG11202007942YA patent/SG11202007942YA/en unknown
- 2019-02-27 BR BR112020017278-2A patent/BR112020017278A2/pt unknown
- 2019-02-27 EP EP19760152.9A patent/EP3768695A4/en active Pending
- 2019-02-27 AU AU2019228504A patent/AU2019228504B2/en active Active
- 2019-02-27 US US16/975,545 patent/US12416016B2/en active Active
- 2019-02-27 WO PCT/US2019/019861 patent/WO2019169004A1/en not_active Ceased
-
2020
- 2020-08-23 IL IL276860A patent/IL276860A/en unknown
- 2020-08-26 CL CL2020002201A patent/CL2020002201A1/es unknown
-
2024
- 2024-05-31 JP JP2024088641A patent/JP2024115558A/ja active Pending
-
2025
- 2025-08-12 US US19/297,827 patent/US20260049334A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2019169004A9 (en) | 2019-10-31 |
| US20210123073A1 (en) | 2021-04-29 |
| US20260049334A1 (en) | 2026-02-19 |
| CL2020002201A1 (es) | 2020-12-18 |
| JP2024115558A (ja) | 2024-08-26 |
| EP3768695A4 (en) | 2022-04-06 |
| CN112236443A (zh) | 2021-01-15 |
| MA52087A (fr) | 2021-06-02 |
| BR112020017278A2 (pt) | 2020-12-22 |
| JP2021515548A (ja) | 2021-06-24 |
| CN120519403A (zh) | 2025-08-22 |
| EP3768695A1 (en) | 2021-01-27 |
| WO2019169004A1 (en) | 2019-09-06 |
| WO2019169004A8 (en) | 2019-12-05 |
| SG11202007942YA (en) | 2020-09-29 |
| AU2019228504A1 (en) | 2020-09-10 |
| IL276860A (en) | 2020-10-29 |
| CN112236443B (zh) | 2025-02-07 |
| US12416016B2 (en) | 2025-09-16 |
| KR20210006327A (ko) | 2021-01-18 |
| JP7498665B2 (ja) | 2024-06-12 |
| AU2019228504B2 (en) | 2025-06-26 |
| KR102848518B1 (ko) | 2025-08-22 |
| CA3091795A1 (en) | 2019-09-06 |
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