MX2022004812A - Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina. - Google Patents

Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.

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Publication number
MX2022004812A
MX2022004812A MX2022004812A MX2022004812A MX2022004812A MX 2022004812 A MX2022004812 A MX 2022004812A MX 2022004812 A MX2022004812 A MX 2022004812A MX 2022004812 A MX2022004812 A MX 2022004812A MX 2022004812 A MX2022004812 A MX 2022004812A
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MX
Mexico
Prior art keywords
progranulin
adeno
aav
disorders
systems
Prior art date
Application number
MX2022004812A
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English (en)
Inventor
Juan Li
Mark Shearman
Adrian Timmers
Judith Newmark
Steven Pennock
Tanaz Farivar
Savitri Mandapadi
Original Assignee
Applied Genetic Tech Corporation
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Filing date
Publication date
Application filed by Applied Genetic Tech Corporation filed Critical Applied Genetic Tech Corporation
Publication of MX2022004812A publication Critical patent/MX2022004812A/es

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    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
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    • 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
    • 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
    • C07K14/575—Hormones
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16051—Methods of production or purification of viral material
    • C12N2710/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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

Abstract

La invención proporciona, en parte, ADNc de progranulina (PGRN) modificado de manera óptima y elementos genéticos asociados para uso en terapia génica a base de virus adenoasociados recombinantes (rAAV) para trastornos neurodegenerativos caracterizados por alteración cognitiva, deterioro del comportamiento y deficiencia de almacenamiento lisosómico, incluyendo la demencia frontotemporal (DFT) familiar, la degeneración lobular frontotemporal (FTLD), la lipofuscinosis ceroide neuronal (NCL) o la enfermedad de Alzheimer (AD).
MX2022004812A 2019-10-22 2020-10-22 Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina. MX2022004812A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962924340P 2019-10-22 2019-10-22
PCT/US2020/056860 WO2021081201A1 (en) 2019-10-22 2020-10-22 Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders

Publications (1)

Publication Number Publication Date
MX2022004812A true MX2022004812A (es) 2023-02-23

Family

ID=75620215

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022004812A MX2022004812A (es) 2019-10-22 2020-10-22 Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.

Country Status (10)

Country Link
US (1) US20210147872A1 (es)
EP (1) EP4048799A4 (es)
JP (1) JP2022553307A (es)
KR (1) KR20230019402A (es)
CN (1) CN115715327A (es)
AU (1) AU2020371662A1 (es)
CA (1) CA3158516A1 (es)
IL (1) IL292382A (es)
MX (1) MX2022004812A (es)
WO (1) WO2021081201A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019315445A1 (en) * 2018-07-31 2021-03-18 Cornell University Gene therapy methods to control organ function
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
WO2024100633A1 (en) * 2022-11-13 2024-05-16 Alexion Pharma International Operations Limited Gene therapy for frontotemporal dementia

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3421603T3 (pl) * 2009-05-02 2022-02-14 Genzyme Corporation Terapia genowa zaburzeń neurodegeneracyjnych
WO2013151667A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides
WO2016125330A1 (ja) * 2015-02-02 2016-08-11 原　英彰 網膜再生促進薬
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
WO2018136680A1 (en) * 2017-01-19 2018-07-26 New York University Progranulin and progranulin derivatives in treating impaired fracture healing
AU2018346104B2 (en) * 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
KR20220015500A (ko) * 2017-10-03 2022-02-08 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3917539A4 (en) * 2019-02-01 2022-11-23 Avrobio, Inc. COMPOSITIONS AND METHODS FOR TREATING NEUROCOGNITIVE DISORDERS
PE20211819A1 (es) * 2019-02-22 2021-09-14 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn

Also Published As

Publication number Publication date
JP2022553307A (ja) 2022-12-22
EP4048799A4 (en) 2023-11-15
CN115715327A (zh) 2023-02-24
WO2021081201A8 (en) 2022-04-14
US20210147872A1 (en) 2021-05-20
WO2021081201A1 (en) 2021-04-29
IL292382A (en) 2022-06-01
EP4048799A1 (en) 2022-08-31
CA3158516A1 (en) 2021-04-29
AU2020371662A1 (en) 2022-05-12
KR20230019402A (ko) 2023-02-08

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