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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

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
    • 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
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
US201862677474P 2018-05-29 2018-05-29
US201862677471P 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 (2) JP7498665B2 (es)
KR (1) KR20210006327A (es)
CN (1) 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 (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
MA52087A (fr) * 2018-02-27 2021-06-02 Univ Pennsylvania Nouveaux vecteurs de virus adéno-associés (aav), vecteurs aav ayant une déamidation de capside réduite et leurs utilisations
AU2019247746B2 (en) 2018-04-03 2024-08-15 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
US12359181B2 (en) 2018-12-21 2025-07-15 The University Of North Carolina At Chapel Hill Optimized GALC genes and expression cassettes and their use
WO2020191300A1 (en) 2019-03-21 2020-09-24 Stridebio, Inc. Recombinant adeno-associated virus vectors
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
EP4048681A4 (en) * 2019-10-21 2024-03-20 The Trustees of The University of Pennsylvania AAV3B VARIANTS WITH IMPROVED PRODUCTION YIELD AND LIVER TROPISM
IL294230A (en) * 2020-01-03 2022-08-01 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
JP2023531451A (ja) 2020-06-17 2023-07-24 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 遺伝子療法患者の治療のための組成物及び方法
AU2021309645A1 (en) 2020-07-13 2023-02-09 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
IL300717A (en) 2020-08-19 2023-04-01 Sarepta Therapeutics Inc ADENO-related virus vectors for the treatment of RETT syndrome
TW202229315A (zh) * 2020-10-18 2022-08-01 賓州大學委員會 改良的腺相關病毒(aav)載體及其用途
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
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
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2024206226A1 (en) * 2023-03-25 2024-10-03 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004083441A2 (en) * 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
HUE054805T2 (hu) * 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
SG10201913259QA (en) * 2014-09-24 2020-02-27 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016086227A2 (en) * 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
CN109661470A (zh) * 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
JP6994018B2 (ja) * 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
CA3033856A1 (en) * 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav
AU2018227440B2 (en) * 2017-02-28 2024-06-06 The Trustees Of The University Of 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
MA52087A (fr) * 2018-02-27 2021-06-02 Univ Pennsylvania Nouveaux vecteurs de virus adéno-associés (aav), vecteurs aav ayant une déamidation de capside réduite et leurs utilisations

Also Published As

Publication number Publication date
BR112020017278A2 (pt) 2020-12-22
EP3768695A4 (en) 2022-04-06
SG11202007942YA (en) 2020-09-29
CN112236443B (zh) 2025-02-07
CA3091795A1 (en) 2019-09-06
CN112236443A (zh) 2021-01-15
JP2024115558A (ja) 2024-08-26
CL2020002201A1 (es) 2020-12-18
KR20210006327A (ko) 2021-01-18
US20210123073A1 (en) 2021-04-29
WO2019169004A1 (en) 2019-09-06
WO2019169004A9 (en) 2019-10-31
JP7498665B2 (ja) 2024-06-12
EP3768695A1 (en) 2021-01-27
AU2019228504B2 (en) 2025-06-26
WO2019169004A8 (en) 2019-12-05
IL276860A (en) 2020-10-29
MA52087A (fr) 2021-06-02
AU2019228504A1 (en) 2020-09-10
JP2021515548A (ja) 2021-06-24

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