MX2021010134A - Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. - Google Patents

Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.

Info

Publication number
MX2021010134A
MX2021010134A MX2021010134A MX2021010134A MX2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A
Authority
MX
Mexico
Prior art keywords
progranulin
grn
associated virus
raav
aav
Prior art date
Application number
MX2021010134A
Other languages
English (en)
Inventor
Christian Hinderer
James M Wilson
Nimrod Miller
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 MX2021010134A publication Critical patent/MX2021010134A/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
    • C12N15/63—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
    • A61K48/005—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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se proporciona un AAV recombinante (rAAV) adecuado para su uso en el tratamiento de la neurodegeneración de inicio en adultos causada por la haploinsuficiencia de granulina (GRN), tal como la demencia frontotemporal (FTD) relacionada con progranulina (PGRN). El rAAV comprende (a) una cápside del virus adenoasociado 1, y (b) un genoma del vector empaquetado en la cápside de AAV, en donde dicho genoma del vector comprende repeticiones terminales invertidas de AAV, una secuencia codificante para progranulina humana y secuencias reguladoras que dirigen la expresión de la progranulina; también se proporciona un método para tratar a un paciente humano con PGRN-FTD y otra neurodegeneración de inicio en adultos causada por haploinsuficiencias de granulina (GRN), que comprende administrar al sistema nervioso central (SNC) un virus adenoasociado recombinante (rAAV) que tiene una cápside del virus adenoasociado 1 (AAV1), en donde dicho rAAV comprende además un genoma del vector empaquetado en la cápside de AAV, en donde dicho genoma del vector comprende repeticiones terminales invertidas de AAV, una secuencia codificante para progranulina humana y secuencias reguladoras que dirigen la expresión de la progranulina.
MX2021010134A 2019-02-22 2020-02-21 Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. MX2021010134A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962809329P 2019-02-22 2019-02-22
US201962923812P 2019-10-21 2019-10-21
US202062969108P 2020-02-02 2020-02-02
PCT/US2020/019149 WO2020172490A1 (en) 2019-02-22 2020-02-21 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

Publications (1)

Publication Number Publication Date
MX2021010134A true MX2021010134A (es) 2021-09-23

Family

ID=70058449

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021010134A MX2021010134A (es) 2019-02-22 2020-02-21 Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.

Country Status (16)

Country Link
US (1) US20220136008A1 (es)
EP (1) EP3927381A1 (es)
JP (1) JP2022523766A (es)
KR (1) KR20210131370A (es)
CN (1) CN113710281A (es)
AU (1) AU2020225472A1 (es)
BR (1) BR112021015817A2 (es)
CA (1) CA3129672A1 (es)
CL (1) CL2021002172A1 (es)
CO (1) CO2021011040A2 (es)
IL (1) IL285654A (es)
MX (1) MX2021010134A (es)
PE (1) PE20211819A1 (es)
SG (1) SG11202108504YA (es)
TW (1) TW202045730A (es)
WO (1) WO2020172490A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020160458A1 (en) * 2019-02-01 2020-08-06 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
WO2021081201A1 (en) * 2019-10-22 2021-04-29 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders
IL300622A (en) * 2020-08-26 2023-04-01 Univ Pennsylvania Recombinant adeno-associated virus for the treatment of adult-onset GRN-related neurodegeneration
WO2023150506A2 (en) * 2022-02-01 2023-08-10 Shape Therapeutics Inc. STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
ATE270324T1 (de) 1997-04-14 2004-07-15 Cell Genesys Inc Methoden zur erhöhung der effizienz rekombinanter aav produkte
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
NZ522841A (en) 2000-06-01 2004-12-24 Univ North Carolina Surgically implantable matrix with a viral vector dried onto it for controlled release of recombinant parvovirus vectors
IL161827A0 (en) 2001-11-13 2005-11-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
PL2292780T3 (pl) 2003-09-30 2018-05-30 The Trustees Of The University Of Pennsylvania Klady wirusów związanych z adenowirusami (AAV), sekwencje, wektory je zawierające i ich zastosowania
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
CA3139726A1 (en) * 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for motor disorders
WO2009089635A1 (en) * 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
CN102869779A (zh) 2010-03-29 2013-01-09 宾夕法尼亚大学托管会 药理学诱导的转基因消融***
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
CN116019934A (zh) * 2015-02-10 2023-04-28 建新公司 病毒颗粒至纹状体和皮质的增强递送
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
CA3016314A1 (en) * 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia
CA3053399A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) clade f vector and uses therefor

Also Published As

Publication number Publication date
CN113710281A (zh) 2021-11-26
CL2021002172A1 (es) 2022-02-11
EP3927381A1 (en) 2021-12-29
CA3129672A1 (en) 2020-08-27
WO2020172490A1 (en) 2020-08-27
JP2022523766A (ja) 2022-04-26
CO2021011040A2 (es) 2021-09-09
PE20211819A1 (es) 2021-09-14
KR20210131370A (ko) 2021-11-02
TW202045730A (zh) 2020-12-16
SG11202108504YA (en) 2021-09-29
AU2020225472A1 (en) 2021-08-26
BR112021015817A2 (pt) 2021-10-13
IL285654A (en) 2021-09-30
US20220136008A1 (en) 2022-05-05

Similar Documents

Publication Publication Date Title
MX2021010134A (es) Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
PH12021551341A1 (en) Compositions for drg-specific reduction of transgene expression
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
RU2018136611A (ru) Генная терапия для лечения гемофилии a
MX2020001402A (es) Metodos de terapia genica del factor viii (fviii).
PT1204739E (pt) Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
CA2407309A1 (en) Dna sequences encoding dystrophin minigenes and methods of use thereof
HRP20201831T1 (hr) Optimizirani promoter rpe65 i kodirajuće sekvence
MX2021013364A (es) Composiciones utiles para el tratamiento de la enfermedad de pompe.
RU2018132517A (ru) Aav-idua вектор для лечения ассоциированной с mps i слепоты
MX2021013420A (es) Composiciones utiles en el tratamiento de leucodistrofia metacromatica.
MX2022014258A (es) Composiciones para la reducción específica de un fármaco de la expresión de transgén.
JP2018510620A5 (es)
MX2022002961A (es) Metodos para tratar enfermedades neovasculares oculares usando variantes de aav2 que codifican para aflibercept.
MX2023007474A (es) Proteinas de la capside viral con especificidad para las celulas del tejido cardiaco.
WO2022221420A3 (en) Selected aav compositions having preferred brain enrichment
MX2020003042A (es) Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
MX2022007135A (es) Vectores de virus adenoasociados para el tratamiento del síndrome de hunter.
EA202192323A1 (ru) Рекомбинантный аденоассоциированный вирус для лечения grn-ассоциированной нейродегенерации взрослых
MX2023006694A (es) Tratamiento de la enfermedad de danon.
MX2023000995A (es) Vector de genoterapia para eef1a2 y usos del mismo.
AR118177A1 (es) Virus adeno-asociado recombinante para el tratamiento del inicio de la neurodegeneración en adultos asociadas con la grn
WO2022221400A3 (en) Aav compositions having high expression levels in brain
MX2023002695A (es) Virus adeno-asociado para la administración de kh902 (conbercept) y usos del mismo.