MX2021009404A - Administracion del virus adenoasociado de polinucleotido de cln6. - Google Patents

Administracion del virus adenoasociado de polinucleotido de cln6.

Info

Publication number
MX2021009404A
MX2021009404A MX2021009404A MX2021009404A MX2021009404A MX 2021009404 A MX2021009404 A MX 2021009404A MX 2021009404 A MX2021009404 A MX 2021009404A MX 2021009404 A MX2021009404 A MX 2021009404A MX 2021009404 A MX2021009404 A MX 2021009404A
Authority
MX
Mexico
Prior art keywords
cln6
polynucleotide
associated virus
adeno
raav
Prior art date
Application number
MX2021009404A
Other languages
English (en)
Inventor
Brian K Kaspar
Kathrin Meyer
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of MX2021009404A publication Critical patent/MX2021009404A/es

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

La presente divulgación se refiere a la administración del virus adenoasociado recombinante (rAAV) de un polinucleótido de lipofuscinosis ceroide neuronal 6 (CLN6). La divulgación proporciona el rAAV y métodos para utilizar el rAAV en el tratamiento génico de CLN6 de la lipofuscinosis ceroide neuronal o la enfermedad de Batten CLN6.
MX2021009404A 2019-02-04 2020-02-04 Administracion del virus adenoasociado de polinucleotido de cln6. MX2021009404A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201962800915P 2019-02-04 2019-02-04
US201962880641P 2019-07-30 2019-07-30
US201962881151P 2019-07-31 2019-07-31
US201962912977P 2019-10-09 2019-10-09
US201962923125P 2019-10-18 2019-10-18
PCT/US2020/016541 WO2020163299A1 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of cln6 polynucleotide

Publications (1)

Publication Number Publication Date
MX2021009404A true MX2021009404A (es) 2021-11-12

Family

ID=69771088

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021009404A MX2021009404A (es) 2019-02-04 2020-02-04 Administracion del virus adenoasociado de polinucleotido de cln6.

Country Status (14)

Country Link
US (1) US20220202956A1 (es)
EP (1) EP3921429A1 (es)
JP (1) JP2022519597A (es)
KR (1) KR20210124299A (es)
CN (1) CN113574176A (es)
AU (1) AU2020218501A1 (es)
BR (1) BR112021015150A2 (es)
CA (1) CA3127801A1 (es)
CL (1) CL2021002051A1 (es)
IL (1) IL285329A (es)
MX (1) MX2021009404A (es)
SG (1) SG11202107983TA (es)
TW (1) TW202033768A (es)
WO (1) WO2020163299A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital, Inc. Intrathecal delivery of recombinant adeno-associated virus 9
WO2023018674A1 (en) * 2021-08-09 2023-02-16 Amicus Therapeutics, Inc. Determination of gene transduction potency in neuron-like cells

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
US6143548A (en) 1995-08-30 2000-11-07 Genzyme Corporation Chromatographic purification of adeno-associated virus (AAV)
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
KR20000068501A (ko) 1996-09-06 2000-11-25 트러스티스 오브 더 유니버시티 오브 펜실바니아 재조합 아데노-관련 바이러스 지정 유전자 요법을 위한 방법
ES2557997T3 (es) 1997-09-05 2016-02-01 Genzyme Corporation Métodos para generar preparados de vectores AAV de título elevado sin virus auxiliar
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
JP6754361B2 (ja) * 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP3411484B1 (en) * 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

Also Published As

Publication number Publication date
JP2022519597A (ja) 2022-03-24
IL285329A (en) 2021-09-30
CL2021002051A1 (es) 2022-04-08
EP3921429A1 (en) 2021-12-15
TW202033768A (zh) 2020-09-16
WO2020163299A1 (en) 2020-08-13
SG11202107983TA (en) 2021-08-30
CA3127801A1 (en) 2020-08-13
KR20210124299A (ko) 2021-10-14
AU2020218501A1 (en) 2021-08-19
BR112021015150A2 (pt) 2021-09-28
CN113574176A (zh) 2021-10-29
US20220202956A1 (en) 2022-06-30

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