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MX2020008933A - 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
MX2020008933A
MX2020008933A MX2020008933A MX2020008933A MX2020008933A MX 2020008933 A MX2020008933 A MX 2020008933A MX 2020008933 A MX2020008933 A MX 2020008933A MX 2020008933 A MX2020008933 A MX 2020008933A MX 2020008933 A MX2020008933 A MX 2020008933A
Authority
MX
Mexico
Prior art keywords
aav
vectors
capsid
associated virus
deamydation
Prior art date
Application number
MX2020008933A
Other languages
English (en)
Inventor
James M Wilson
Kevin Turner
April Tepe
Joshua Joyner Sims
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 MX2020008933A publication Critical patent/MX2020008933A/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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 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.
MX2020008933A 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. MX2020008933A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US201862667585P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862722382P 2018-08-24 2018-08-24
PCT/US2019/019804 WO2019168961A1 (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
MX2020008933A true MX2020008933A (es) 2021-01-15

Family

ID=67805569

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020008933A MX2020008933A (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 (13)

Country Link
US (1) US20200407750A1 (es)
EP (1) EP3758724A4 (es)
JP (3) JP2021516046A (es)
KR (1) KR20210010434A (es)
CN (2) CN112352050A (es)
AU (1) AU2019227726B2 (es)
BR (1) BR112020017348A2 (es)
CA (1) CA3091806A1 (es)
CL (2) CL2020002200A1 (es)
IL (1) IL276859B2 (es)
MX (1) MX2020008933A (es)
SG (1) SG11202008182TA (es)
WO (1) WO2019168961A1 (es)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
AU2019247746B2 (en) 2018-04-03 2024-08-15 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
KR20210006358A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 안 조직을 표적으로 하기 위한 바이러스 벡터
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
US12472268B2 (en) 2018-10-01 2025-11-18 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
WO2020223232A1 (en) 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
WO2021080991A1 (en) * 2019-10-21 2021-04-29 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
KR20220140537A (ko) * 2020-02-14 2022-10-18 울트라제닉스 파마수티컬 인코포레이티드 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법
EP4127189A1 (en) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP4150051A4 (en) * 2020-04-27 2025-05-21 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)
MX2022016528A (es) 2020-06-17 2023-06-02 Univ Pennsylvania Composiciones y métodos para el tratamiento de pacientes de terapia génica.
WO2022015715A1 (en) 2020-07-13 2022-01-20 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
CA3196499A1 (en) * 2020-10-29 2022-05-05 James M. Wilson Aav capsids and compositions containing same
CA3200014A1 (en) 2020-12-01 2022-06-09 James M. Wilson Novel compositions with tissue-specific targeting motifs and compositions containing same
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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)
AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
IL313832A (en) * 2021-12-28 2024-08-01 Chengdu Origen Biotechnology Co Ltd Modified AAV capsid protein and its use
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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
EP4469584A1 (en) 2022-01-25 2024-12-04 The Trustees of The University of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
AU2023232781A1 (en) 2022-03-07 2024-09-26 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
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
CN115011601B (zh) * 2022-06-27 2023-07-21 山东大学齐鲁医院 一种干扰JUND表达的shRNA、重组腺相关病毒载体及其应用
WO2024015966A2 (en) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
CN115554418B (zh) 2022-11-22 2023-04-14 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
JP2026500351A (ja) 2022-12-17 2026-01-06 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 心筋および骨格筋特異的標的化モチーフを有する組換えａａｖ変異体ベクターおよびそれを含む組成物
AU2023412879A1 (en) * 2022-12-23 2025-07-10 Spark Therapeutics, Inc. Adeno-associated virus formulations
CN121127597A (zh) * 2023-05-03 2025-12-12 沃雅戈治疗公司 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法
CN121079310A (zh) * 2023-05-03 2025-12-05 沃雅戈治疗公司 用于治疗与cdkl5缺乏相关的病症的组合物及方法
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102212558B (zh) * 2003-09-30 2016-08-03 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
CN101203613B (zh) * 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
PE20150163A1 (es) * 2012-04-18 2015-02-23 Philadelphia Children Hospital Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
PH12016500162B1 (en) * 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
WO2017100674A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
IL298604A (en) * 2016-04-15 2023-01-01 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
ES2863674T3 (es) * 2016-08-15 2021-10-11 Genzyme Corp Métodos para detectar AAV
SG10201912973XA (en) * 2017-02-28 2020-03-30 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

Also Published As

Publication number Publication date
BR112020017348A2 (pt) 2020-12-29
AU2019227726A1 (en) 2020-09-10
US20200407750A1 (en) 2020-12-31
IL276859B1 (en) 2025-08-01
JP2023159235A (ja) 2023-10-31
SG11202008182TA (en) 2020-09-29
JP2024041967A (ja) 2024-03-27
AU2019227726B2 (en) 2025-09-04
EP3758724A4 (en) 2022-07-06
IL276859B2 (en) 2025-12-01
CN112352050A (zh) 2021-02-09
KR20210010434A (ko) 2021-01-27
CN119842635A (zh) 2025-04-18
EP3758724A1 (en) 2021-01-06
CL2022003757A1 (es) 2023-05-19
JP2021516046A (ja) 2021-07-01
CL2020002200A1 (es) 2021-01-29
WO2019168961A1 (en) 2019-09-06
IL276859A (en) 2020-10-29
CA3091806A1 (en) 2019-09-06

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