[go: up one dir, main page]

MX2020002148A - Vectores adenoasociados recombinantes. - Google Patents

Vectores adenoasociados recombinantes.

Info

Publication number
MX2020002148A
MX2020002148A MX2020002148A MX2020002148A MX2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A
Authority
MX
Mexico
Prior art keywords
vectors
recombinant adeno
aav
associated vectors
adeno
Prior art date
Application number
MX2020002148A
Other languages
English (en)
Inventor
Matthew During
Original Assignee
Ovid Therapeutics Inc
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 Ovid Therapeutics Inc filed Critical Ovid Therapeutics Inc
Publication of MX2020002148A publication Critical patent/MX2020002148A/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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Se proporcionan vectores de virus adenoasociados (AAV) y usos de los mismos. Más específicamente, se proporcionan vectores AAV que muestran tropismo específico para ciertos tejidos blanco, tales como sistema nervioso central (SNC) y tejido adiposo y que se puede usar para transducir las células para introducción de genes de interés en los tejidos blanco. Asimismo se proporcionan composiciones farmacéuticas que incluyen vectores AAV y un excipiente, diluyente o vehículo farmacéuticamente aceptable.
MX2020002148A 2017-08-25 2018-08-22 Vectores adenoasociados recombinantes. MX2020002148A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762550458P 2017-08-25 2017-08-25
PCT/US2018/047466 WO2019040586A1 (en) 2017-08-25 2018-08-22 RECOMBINANT ADENO-ASSOCIATE VECTORS

Publications (1)

Publication Number Publication Date
MX2020002148A true MX2020002148A (es) 2020-07-20

Family

ID=65439655

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020002148A MX2020002148A (es) 2017-08-25 2018-08-22 Vectores adenoasociados recombinantes.

Country Status (10)

Country Link
US (2) US20190085358A1 (es)
EP (1) EP3655041A4 (es)
JP (1) JP2020533968A (es)
KR (1) KR20200042935A (es)
CN (1) CN111163811A (es)
AU (1) AU2018320849A1 (es)
CA (1) CA3073937A1 (es)
IL (1) IL272655A (es)
MX (1) MX2020002148A (es)
WO (1) WO2019040586A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3883566A4 (en) 2018-11-21 2022-09-07 Certego Therapeutics Inc. GABOXADOL FOR REDUCE RISK OF SUICIDE AND RAPID RELIEF OF DEPRESSION
AU2020341451A1 (en) * 2019-09-03 2022-03-24 Krystal Biotech, Inc. Compositions and methods for the treatment of congenital ichthyoses
US11597726B2 (en) 2020-05-20 2023-03-07 Certego Therapeutics Inc. Ring deuterated gaboxadol and its use for the treatment of psychiatric disorders
AU2021335601A1 (en) * 2020-09-04 2023-04-06 Ohio State Innovation Foundation Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2329060C (en) * 1998-05-28 2011-09-13 John A. Chiorini Aav5 vector and uses thereof
CA2376400A1 (en) * 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
JP2005535332A (ja) * 2002-08-12 2005-11-24 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 結節性硬化症の診断法および治療法
US7427396B2 (en) * 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
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
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2692868A1 (en) * 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
AU2013388083B2 (en) * 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
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
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015171547A1 (en) * 2014-05-05 2015-11-12 Ovid Therapeutics Inc. Methods of treating cognitive impairment associated with neurodegenerative disorders
CA2949713A1 (en) * 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
EP3795580A1 (en) * 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
EP3387137B1 (en) * 2015-12-11 2021-02-03 California Institute of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
JP7082050B2 (ja) * 2015-12-14 2022-06-07 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 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター
AU2017261812B2 (en) * 2016-05-13 2019-04-18 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
IL268534B2 (en) * 2017-02-15 2024-10-01 Univ North Carolina Chapel Hill Methods and compositions for gene transfer across the vasculature
KR20190124309A (ko) * 2017-03-15 2019-11-04 오비드 테라퓨틱스 인크. 발작 장애들의 치료에 있어 디자이너 약물들에 의하여 배타적으로 활성화되는 디자이너 수용체들의 용도
CN111867595A (zh) * 2017-12-20 2020-10-30 奥维德医疗公司 hM4Di在治疗癫痫发作紊乱中的用途
CA3103267A1 (en) * 2018-06-14 2019-12-19 Ovid Therapeutics Inc. Use of mir-92a or mir-145 in the treatment of angelman syndrome
CA3114199A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
WO2021102107A1 (en) * 2019-11-19 2021-05-27 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders
WO2021113634A1 (en) * 2019-12-05 2021-06-10 The Board Of Regents Of The University Of Texas Transgene cassettes designed to express a human mecp2 gene
CA3116391A1 (en) * 2020-09-14 2022-03-14 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
AU2022216272A1 (en) * 2021-02-03 2023-08-17 The University Of North Carolina At Chapel Hill Protein m analogs and fusion proteins and their use for inhibiting antibody function
US20240197919A1 (en) * 2021-05-04 2024-06-20 California Institute Of Technology Recombinant aavs for delivery to central nervous system and brain vasculature
CN114259502A (zh) * 2021-12-24 2022-04-01 南京鼓楼医院 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用
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

Also Published As

Publication number Publication date
CN111163811A (zh) 2020-05-15
AU2018320849A1 (en) 2020-03-05
US20190085358A1 (en) 2019-03-21
WO2019040586A1 (en) 2019-02-28
CA3073937A1 (en) 2019-02-28
KR20200042935A (ko) 2020-04-24
EP3655041A1 (en) 2020-05-27
US20210371880A1 (en) 2021-12-02
JP2020533968A (ja) 2020-11-26
IL272655A (en) 2020-03-31
EP3655041A4 (en) 2021-04-21

Similar Documents

Publication Publication Date Title
MX2020002148A (es) Vectores adenoasociados recombinantes.
PH12020551744A1 (en) Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2020028751A3 (en) Aav variants with enhanced tropism
PH12018500594A1 (en) Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
SA517390590B1 (ar) Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة
ZA202002094B (en) Trispecific proteins and methods of use
WO2017058892A3 (en) Methods and compositions for antibody-evading virus vectors
WO2018191502A3 (en) Anti-cd137 antibodies and methods of use thereof
MX2018006840A (es) Focalizacion de peptidos para dirigir virus adenoasociados.
MX2021006615A (es) Proteinas de union triespecificas y metodos de uso.
MX2021004397A (es) Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos.
MX388331B (es) Variantes de virus adenoasociado y métodos de uso de estas
EA201792236A1 (ru) Получение увеличенных в размере векторов на основе аденоассоциированного вируса
MX367100B (es) Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
AU2017248121A1 (en) T cell receptors
AU2017248120A1 (en) T cell receptors
WO2021226267A3 (en) Cross-species compatible adeno-associated virus compositions and methods of use thereof
WO2012142434A3 (en) Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
MX2018010483A (es) Inmunogenos para vacunacion contra vih.
WO2021025995A9 (en) Aav variants with enhanced tropism
MX2025008949A (es) Arni variante
CO2018003960A2 (es) Virus adeno-asociado recombinante que comprende un ácido nucleico galgt2
MX2019008105A (es) Virus.
MX2021004688A (es) Distrofinas miniaturizadas y usos de las mismas.
EA202090700A1 (ru) Компетентные по репликации аденовирусные векторы