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

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020106927A1 (en) 2018-11-21 2020-05-28 Certego Therapeutics Gaboxadol for reducing risk of suicide and rapid relief of depression
IL298334A (en) 2020-05-20 2023-01-01 Certego Therapeutics Inc A canceled gaboxadol ring and its use for the treatment of psychiatric disorders
JP2023540756A (ja) * 2020-09-04 2023-09-26 オハイオ・ステイト・イノベーション・ファウンデーション 向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE402254T1 (de) * 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
WO2000075365A2 (en) * 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
WO2004014222A2 (en) * 2002-08-12 2004-02-19 The Regents Of The University Of Michigan Diagnosis and treatment of tuberous sclerosis
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
US8999678B2 (en) * 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
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
WO2014178863A1 (en) * 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
EP3024498B1 (en) * 2013-07-22 2019-12-04 The Children's Hospital of Philadelphia Variant aav and compositions, methods and uses for gene transfer 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
JP2017518372A (ja) * 2014-05-30 2017-07-06 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 潜伏性ウイルス感染用の処置剤を送達するための組成物および方法
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10711270B2 (en) * 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
WO2017100671A1 (en) * 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
KR20180086266A (ko) * 2015-12-14 2018-07-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 척수성 근위축증의 치료에 유용한 아데노-관련 바이러스 벡터
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
EA038695B1 (ru) * 2017-02-15 2021-10-06 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Способы и композиции для переноса генов по сосудистой сети
CN110621318A (zh) * 2017-03-15 2019-12-27 奥维德医疗公司 排他地由设计者药物激活的设计者受体在治疗癫痫发作紊乱中的用途
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
MX2020013667A (es) * 2018-06-14 2021-03-02 Ovid Therapeutics Inc Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.
WO2020068990A1 (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
IL293068A (en) * 2019-11-19 2022-07-01 Asklepios Biopharmaceutical Inc A therapeutic adeno-associated virus containing liver-specific promoters for the treatment of Pompe disease and lysosomal disorders
JP2023504735A (ja) * 2019-12-05 2023-02-06 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ヒトmecp2遺伝子を発現するように設計されたトランスジーンカセット
CA3116391A1 (en) * 2020-09-14 2022-03-14 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
KR20230152008A (ko) * 2021-02-03 2023-11-02 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 단백질 m 유사체 및 융합 단백질 및 이들의 항체 기능 억제 용도
WO2022235702A1 (en) * 2021-05-04 2022-11-10 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
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

Also Published As

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

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