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
associated virus
uses therefor
heterogeneous population
Prior art date
Application number
MX2020008932A
Other languages
English (en)
Inventor
James M Wilson
Kevin Turner
April Tepe
Joshua Joyner Sims
Qiang Wang
Original Assignee
Univ Pennsylvania
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2020008932A publication Critical patent/MX2020008932A/es

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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
    • 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)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (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.
MX2020008932A 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. MX2020008932A (es)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US201862635968P 2018-02-27 2018-02-27
US201862663797P 2018-04-27 2018-04-27
US201862663788P 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
US201862677471P 2018-05-29 2018-05-29
US201862677474P 2018-05-29 2018-05-29
US201862667585P 2018-05-29 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862703673P 2018-07-26 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US201862722388P 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 (1) US20210123073A1 (es)
EP (1) EP3768695A4 (es)
JP (1) JP7498665B2 (es)
KR (1) KR20210006327A (es)
CN (1) CN112236443A (es)
AU (1) AU2019228504A1 (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 (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
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
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
US20230002788A1 (en) * 2019-10-21 2023-01-05 The Trustees Of The University Of Pennsylvania Aav3b variants with improved production yield and liver tropism
WO2021138381A1 (en) * 2020-01-03 2021-07-08 Sarepta Therapeutics, Inc. Methods for analyzing aav capsid proteins
EP4117735A4 (en) * 2020-03-09 2024-04-10 University of Massachusetts GENE REPLACEMENT THERAPY FOR FOXG1 SYNDROME
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
KR20230038503A (ko) 2020-07-13 2023-03-20 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 샤르코-마리-투스 질환의 치료에 유용한 조성물
AR123838A1 (es) * 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
AU2022258312A1 (en) 2021-04-12 2023-10-26 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (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
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023133584A1 (en) 2022-01-10 2023-07-13 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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070172460A1 (en) * 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
EP2298926A1 (en) * 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP3409296A1 (en) * 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
ES2856090T3 (es) * 2014-09-24 2021-09-27 Hope City Variantes de vectores de virus adenoasociados para la edición genómica de alta eficacia y sus métodos
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
CA3019423A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
TN2019000047A1 (en) * 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
SG11201907714UA (en) * 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي

Also Published As

Publication number Publication date
CN112236443A (zh) 2021-01-15
MA52087A (fr) 2021-06-02
EP3768695A1 (en) 2021-01-27
CA3091795A1 (en) 2019-09-06
IL276860A (en) 2020-10-29
BR112020017278A2 (pt) 2020-12-22
CL2020002201A1 (es) 2020-12-18
WO2019169004A8 (en) 2019-12-05
US20210123073A1 (en) 2021-04-29
JP7498665B2 (ja) 2024-06-12
EP3768695A4 (en) 2022-04-06
JP2021515548A (ja) 2021-06-24
KR20210006327A (ko) 2021-01-18
SG11202007942YA (en) 2020-09-29
AU2019228504A1 (en) 2020-09-10
WO2019169004A9 (en) 2019-10-31
WO2019169004A1 (en) 2019-09-06

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