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

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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
US201862677471P 2018-05-29 2018-05-29
US201862667585P 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 (4) 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)

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BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
EP3861131A4 (en) 2018-10-01 2022-06-15 Ultragenyx Pharmaceutical Inc. GENE THERAPY FOR THE TREATMENT OF PROPIONIC ACIDosis
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
PE20220014A1 (es) 2019-04-29 2022-01-11 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen
US12544437B2 (en) 2019-10-04 2026-02-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant AAV
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
CA3155016A1 (en) * 2019-10-21 2021-04-29 James M. Wilson 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
MX2022009982A (es) * 2020-02-14 2022-09-12 Ultragenyx Pharmaceutical Inc Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
IL297635A (en) * 2020-04-27 2022-12-01 Univ Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
IL302127A (en) 2020-10-18 2023-06-01 Univ Pennsylvania An improved adeno-associated virus (AAV) vector and its uses
AU2021369793A1 (en) * 2020-10-29 2023-06-08 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
IL313832A (en) * 2021-12-28 2024-08-01 Chengdu Origen Biotechnology Co Ltd Modified AAV capsid protein and its use
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
US20250144243A1 (en) 2022-01-25 2025-05-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
CN119137263A (zh) 2022-03-07 2024-12-13 奥特吉尼克斯制药公司 改进的分批aav产生系统和方法
US20250250326A1 (en) 2022-04-06 2025-08-07 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
CN116350801A (zh) 2022-11-22 2023-06-30 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
KR20250135916A (ko) 2022-12-17 2025-09-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물
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
CN120584187A (zh) * 2022-12-23 2025-09-02 斯帕克医疗有限公司 腺相关病毒制剂
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
PE20260124A1 (es) * 2023-05-03 2026-01-16 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de trastornos relacionados con la deficiencia de cdkl5
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
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US11015173B2 (en) * 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
WO2017180854A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
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Also Published As

Publication number Publication date
CA3091806A1 (en) 2019-09-06
US20200407750A1 (en) 2020-12-31
JP2023159235A (ja) 2023-10-31
EP3758724A1 (en) 2021-01-06
CL2022003757A1 (es) 2023-05-19
AU2019227726A1 (en) 2020-09-10
IL276859B2 (en) 2025-12-01
JP2026027394A (ja) 2026-02-18
IL276859A (en) 2020-10-29
SG11202008182TA (en) 2020-09-29
CL2020002200A1 (es) 2021-01-29
AU2019227726B2 (en) 2025-09-04
JP2024041967A (ja) 2024-03-27
EP3758724A4 (en) 2022-07-06
WO2019168961A1 (en) 2019-09-06
KR20210010434A (ko) 2021-01-27
BR112020017348A2 (pt) 2020-12-29
JP2021516046A (ja) 2021-07-01
CN112352050A (zh) 2021-02-09
CN119842635A (zh) 2025-04-18
IL276859B1 (en) 2025-08-01

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