MX2020003095A - Arni variante. - Google Patents

Arni variante.

Info

Publication number
MX2020003095A
MX2020003095A MX2020003095A MX2020003095A MX2020003095A MX 2020003095 A MX2020003095 A MX 2020003095A MX 2020003095 A MX2020003095 A MX 2020003095A MX 2020003095 A MX2020003095 A MX 2020003095A MX 2020003095 A MX2020003095 A MX 2020003095A
Authority
MX
Mexico
Prior art keywords
rnai
recombinant
disease
further provided
vectors
Prior art date
Application number
MX2020003095A
Other languages
English (en)
Inventor
Lisa M Stanek
Adam Palermo
Brenda Richards
Catherine R O'riordan
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of MX2020003095A publication Critical patent/MX2020003095A/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • 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
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    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
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    • C12N2710/10011—Adenoviridae
    • C12N2710/10023—Virus like particles [VLP]
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2710/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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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    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

En la presente se proporcionan moléculas de ARNi para tratar la enfermedad de Huntington. En la presente se proporcionan adicionalmente casetes de expresión, vectores (por ejemplo, vectores de rAAV, adenovirales recombinantes, lentivirales recombinantes, y HSV recombinantes), células, partículas virales, y composiciones farmacéuticas que contienen el ARNi. Aún en la presente se proporcionan adicionalmente métodos y kits relacionados con el uso del ARNi, por ejemplo, para tratar la enfermedad de Huntington.
MX2020003095A 2017-09-22 2018-09-21 Arni variante. MX2020003095A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762561843P 2017-09-22 2017-09-22
PCT/US2018/052221 WO2019060726A1 (en) 2017-09-22 2018-09-21 VARIANT OF ARNI

Publications (1)

Publication Number Publication Date
MX2020003095A true MX2020003095A (es) 2020-10-15

Family

ID=64017428

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020003095A MX2020003095A (es) 2017-09-22 2018-09-21 Arni variante.

Country Status (18)

Country Link
US (2) US11603529B2 (es)
EP (1) EP3684932A1 (es)
JP (1) JP7248658B2 (es)
KR (1) KR20200056428A (es)
CN (1) CN111356763B (es)
AR (1) AR113134A1 (es)
AU (1) AU2018335410A1 (es)
BR (1) BR112020005569A2 (es)
CA (1) CA3076191A1 (es)
CO (1) CO2020003187A2 (es)
IL (1) IL273394A (es)
MX (1) MX2020003095A (es)
PH (1) PH12020550117A1 (es)
SG (1) SG11202002488UA (es)
TW (1) TW201923079A (es)
UY (1) UY37897A (es)
WO (1) WO2019060726A1 (es)
ZA (1) ZA202001586B (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2017010369A (es) 2015-02-10 2017-12-14 Genzyme Corp Arni variante.
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
US11603529B2 (en) 2017-09-22 2023-03-14 Genzyme Corporation Variant RNAi
CN112980837B (zh) * 2019-12-13 2023-07-04 深圳艾码生物科技有限公司 一种抑制HTT基因表达的siRNA及其前体和应用
CN115141848A (zh) * 2021-03-30 2022-10-04 南京大学 一种用于治疗亨廷顿病的rna递送系统

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MX2017010369A (es) 2015-02-10 2017-12-14 Genzyme Corp Arni variante.
EP3458589A4 (en) * 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
US11603529B2 (en) 2017-09-22 2023-03-14 Genzyme Corporation Variant RNAi
AR114540A1 (es) * 2018-08-03 2020-09-16 Genzyme Corp VARIANTE DE ARNi CONTRA a-SINUCLEÍNA

Also Published As

Publication number Publication date
CN111356763B (zh) 2024-03-12
CO2020003187A2 (es) 2020-04-13
US20230392149A1 (en) 2023-12-07
TW201923079A (zh) 2019-06-16
RU2020114229A (ru) 2021-10-22
JP2020535803A (ja) 2020-12-10
CN111356763A (zh) 2020-06-30
AU2018335410A1 (en) 2020-05-07
KR20200056428A (ko) 2020-05-22
SG11202002488UA (en) 2020-04-29
RU2020114229A3 (es) 2022-02-01
PH12020550117A1 (en) 2020-12-07
AR113134A1 (es) 2020-01-29
WO2019060726A1 (en) 2019-03-28
CA3076191A1 (en) 2019-03-28
BR112020005569A2 (pt) 2020-10-06
ZA202001586B (en) 2024-01-31
UY37897A (es) 2019-04-30
JP7248658B2 (ja) 2023-03-29
IL273394A (en) 2020-05-31
EP3684932A1 (en) 2020-07-29
US11603529B2 (en) 2023-03-14
US20200216848A1 (en) 2020-07-09

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