AR123524A1 - Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1 - Google Patents

Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1

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Publication number
AR123524A1
AR123524A1 ARP210102563A ARP210102563A AR123524A1 AR 123524 A1 AR123524 A1 AR 123524A1 AR P210102563 A ARP210102563 A AR P210102563A AR P210102563 A ARP210102563 A AR P210102563A AR 123524 A1 AR123524 A1 AR 123524A1
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AR
Argentina
Prior art keywords
sequence
promoter
mirna
raav
als
Prior art date
Application number
ARP210102563A
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English (en)
Inventor
Joyce Shih Lo
- Mcampbell Alex Ching
Maria Zavodszky
Edward Guilmette
Barret Pfeiffer
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Biogen Ma Inc
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Filing date
Publication date
Application filed by Biogen Ma Inc filed Critical Biogen Ma Inc
Publication of AR123524A1 publication Critical patent/AR123524A1/es

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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (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 That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente descripción proporciona composiciones y métodos para tratar la esclerosis lateral amiotrófica (ELA). Entre otras cosas, la presente descripción proporciona ácidos nucleicos inhibidores que inhiben la expresión de genes que causan o están implicados en la patogénesis de la ELA. La presente descripción proporciona además vectores de virus adenoasociados recombinantes (rAAV) que comprenden ácidos nucleicos inhibidores que inhiben la expresión de genes que causan o están implicados en la patogénesis de la ELA. Reivindicación 1: Un vector de virus adenoasociado recombinante (rAAV) que comprende: a) un genoma de AAV modificado que comprende: (i) un promotor; y (ii) al menos dos o más secuencias de miARN diferentes; y b) una cápside; en el que cada una de las dos o más secuencias de miARN comprenden una secuencia de hebra guía que se dirige a la superóxido dismutasa 1 (SOD1), y una secuencia de andamiaje, y en el que cada una de las dos o más secuencias de miARN están unidas de forma operativa al promotor. Reivindicación 49: Un vector de virus adenoasociado recombinante (rAAV) que comprende: a) un genoma de AAV modificado que comprende: (i) un promotor; (ii) al menos una secuencia de miARN; y b) una cápside; en el que al menos una secuencia de miARN comprende una secuencia de hebra guía que comprende la SEQ ID Nº 2 y una secuencia de andamiaje de miR-155, y en el que la secuencia de miARN está unida de forma operativa al promotor. Reivindicación 50: Un vector de virus adenoasociado recombinante (rAAV) que comprende: a) un genoma de AAV modificado que comprende: (i) un promotor; (ii) al menos una secuencia de miARN; y b) una cápside; en el que al menos una secuencia de miARN comprende una secuencia de hebra guía que comprende la SEQ ID Nº 5 y una secuencia de andamiaje, y en el que la secuencia de miARN está unida de forma operativa al promotor. Reivindicación 52: Un vector de virus adenoasociado recombinante (rAAV) que comprende: a) un genoma de AAV modificado que comprende: (i) un promotor; (ii) al menos una secuencia de miARN; y b) una cápside; en el que al menos una secuencia de miARN comprende una secuencia de hebra guía que comprende la SEQ ID Nº 7 y una secuencia de andamiaje, y en el que la secuencia de miARN está unida de forma operativa al promotor.
ARP210102563A 2020-09-16 2021-09-15 Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1 AR123524A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063079459P 2020-09-16 2020-09-16

Publications (1)

Publication Number Publication Date
AR123524A1 true AR123524A1 (es) 2022-12-14

Family

ID=80775574

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210102563A AR123524A1 (es) 2020-09-16 2021-09-15 Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1

Country Status (8)

Country Link
US (1) US20230340489A1 (es)
EP (1) EP4214324A1 (es)
JP (1) JP2023542130A (es)
CN (1) CN116507731A (es)
AR (1) AR123524A1 (es)
TW (1) TW202223096A (es)
UY (1) UY39426A (es)
WO (1) WO2022060857A1 (es)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10280418B2 (en) * 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP3121284A1 (en) * 2015-07-22 2017-01-25 Ecole Polytechnique Federale de Lausanne (EPFL) Bicistronic aav vector for rna interference in als
EP3684937A4 (en) * 2017-09-22 2021-06-02 University of Massachusetts DUAL EXPRESSION VECTORS OF SOD1 AND THEIR USES

Also Published As

Publication number Publication date
EP4214324A1 (en) 2023-07-26
WO2022060857A1 (en) 2022-03-24
CN116507731A (zh) 2023-07-28
JP2023542130A (ja) 2023-10-05
UY39426A (es) 2022-04-29
TW202223096A (zh) 2022-06-16
US20230340489A1 (en) 2023-10-26

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