PE20211819A1 - 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

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Publication number
PE20211819A1
PE20211819A1 PE2021001357A PE2021001357A PE20211819A1 PE 20211819 A1 PE20211819 A1 PE 20211819A1 PE 2021001357 A PE2021001357 A PE 2021001357A PE 2021001357 A PE2021001357 A PE 2021001357A PE 20211819 A1 PE20211819 A1 PE 20211819A1
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PE
Peru
Prior art keywords
progranulin
aav
grn
associated virus
capsid
Prior art date
Application number
PE2021001357A
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English (en)
Inventor
Christian Hinderer
Nimrod Miller
James M Wilson
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 PE20211819A1 publication Critical patent/PE20211819A1/es

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    • 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

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  • 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)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (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 neurodegeneracion 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 capside del virus adenoasociado 1, y (b) un genoma del vector empaquetado en la capside 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 expresion de la progranulina. Tambien se proporciona un metodo para tratar a un paciente humano con PGRN-FTD y otra neurodegeneracion 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 capside del virus adenoasociado 1 (AAV1), en donde dicho rAAV comprende ademas un genoma del vector empaquetado en la capside 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 expresion de la progranulina
PE2021001357A 2019-02-22 2020-02-21 Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn PE20211819A1 (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
PE20211819A1 true PE20211819A1 (es) 2021-09-14

Family

ID=70058449

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021001357A PE20211819A1 (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
CA3128003A1 (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
EP4048799A4 (en) * 2019-10-22 2023-11-15 Applied Genetic Technologies Corporation ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR THE TREATMENT OF PROGRANULIN-ASSOCIATED NEURODEGENERATIVE DISEASES OR DISORDERS
TW202227633A (zh) * 2020-08-26 2022-07-16 賓州大學委員會 用於治療grn相關的成年發病神經退化之重組腺相關病毒
WO2023150506A2 (en) * 2022-02-01 2023-08-10 Shape Therapeutics Inc. STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS

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US20220136008A1 (en) 2022-05-05
KR20210131370A (ko) 2021-11-02
CA3129672A1 (en) 2020-08-27
CN113710281A (zh) 2021-11-26
TW202045730A (zh) 2020-12-16
BR112021015817A2 (pt) 2021-10-13
AU2020225472A1 (en) 2021-08-26
SG11202108504YA (en) 2021-09-29
MX2021010134A (es) 2021-09-23
JP2022523766A (ja) 2022-04-26
EP3927381A1 (en) 2021-12-29
CO2021011040A2 (es) 2021-09-09
CL2021002172A1 (es) 2022-02-11
WO2020172490A1 (en) 2020-08-27
IL285654A (en) 2021-09-30

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