MX2022013005A - Arni variante. - Google Patents

Arni variante.

Info

Publication number
MX2022013005A
MX2022013005A MX2022013005A MX2022013005A MX2022013005A MX 2022013005 A MX2022013005 A MX 2022013005A MX 2022013005 A MX2022013005 A MX 2022013005A MX 2022013005 A MX2022013005 A MX 2022013005A MX 2022013005 A MX2022013005 A MX 2022013005A
Authority
MX
Mexico
Prior art keywords
rnai
guide sequence
recombinant
disease
further provided
Prior art date
Application number
MX2022013005A
Other languages
English (en)
Inventor
Sergio Pablo Sardi
Adam Palermo
Brenda Richards
Antonius Song
Lisa M Stanek
Catherine 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 MX2022013005A publication Critical patent/MX2022013005A/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
    • C12N15/11—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2710/10011—Adenoviridae
    • C12N2710/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • 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

Abstract

En la presente descripción se proporcionan moléculas de ARNi que incluyen una primera cadena que contiene una secuencia guía y una segunda cadena que comprende una secuencia no guía en donde la secuencia no guía contiene una protuberancia opuesta a la región semilla de las secuencias guía; p. ej., opuesta a la secuencia de escisión. En algunos aspectos, la invención proporciona ARNi para el tratamiento de la enfermedad de Huntington. Adicionalmente se proporcionan en la presente descripción casetes de expresión, vectores (p. ej., vectores de rAAV, adenovirales recombinantes, lentivirales recombinantes, y de HSV recombinantes), células, partículas virales y composiciones farmacéuticas que contienen el ARNi. Asimismo se proporcionan adicionalmente en la presente descripción métodos y kits relacionados con el uso del ARNi, por ejemplo, para el tratamiento de la enfermedad de Huntington.
MX2022013005A 2015-02-10 2017-08-10 Arni variante. MX2022013005A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10

Publications (1)

Publication Number Publication Date
MX2022013005A true MX2022013005A (es) 2022-11-09

Family

ID=56614905

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017010369A MX2017010369A (es) 2015-02-10 2016-02-09 Arni variante.
MX2022013005A MX2022013005A (es) 2015-02-10 2017-08-10 Arni variante.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2017010369A MX2017010369A (es) 2015-02-10 2016-02-09 Arni variante.

Country Status (26)

Country Link
US (3) US10450563B2 (es)
EP (1) EP3256588A2 (es)
JP (2) JP2018506304A (es)
KR (1) KR20170110149A (es)
CN (1) CN107438671B (es)
AR (1) AR103646A1 (es)
AU (2) AU2016219396B2 (es)
BR (1) BR112017017028A2 (es)
CA (1) CA2976075A1 (es)
CL (1) CL2017002027A1 (es)
CO (1) CO2017009083A2 (es)
CR (1) CR20170406A (es)
EA (1) EA201791805A1 (es)
EC (1) ECSP17059343A (es)
HK (1) HK1247641A1 (es)
IL (1) IL253893B (es)
MA (1) MA40819B1 (es)
MX (2) MX2017010369A (es)
MY (2) MY181458A (es)
PE (1) PE20171382A1 (es)
PH (1) PH12017501432A1 (es)
SG (3) SG10201907396XA (es)
TN (1) TN2017000354A1 (es)
TW (1) TWI781079B (es)
UY (1) UY36554A (es)
WO (1) WO2016130589A2 (es)

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CN107438671A (zh) 2017-12-05
ECSP17059343A (es) 2019-03-29
CN107438671B (zh) 2021-05-25
WO2016130589A2 (en) 2016-08-18
SG10201912942YA (en) 2020-02-27
SG11201706444TA (en) 2017-09-28
BR112017017028A2 (pt) 2018-04-10
MA40819B1 (fr) 2020-04-30
CR20170406A (es) 2017-11-14
AU2022204274A1 (en) 2022-07-07
AU2016219396B2 (en) 2022-03-17
MY194175A (en) 2022-11-17
EA201791805A1 (ru) 2018-05-31
MX2017010369A (es) 2017-12-14
TN2017000354A1 (en) 2019-01-16
PE20171382A1 (es) 2017-09-15
IL253893A0 (en) 2017-10-31
MA40819A1 (fr) 2018-07-31
CL2017002027A1 (es) 2018-04-20
US20210047641A1 (en) 2021-02-18
JP2021118740A (ja) 2021-08-12
IL253893B (en) 2021-08-31
JP2018506304A (ja) 2018-03-08
AU2016219396A1 (en) 2017-09-28
US11781137B2 (en) 2023-10-10
HK1247641A1 (zh) 2018-09-28
US10760079B2 (en) 2020-09-01
MY181458A (en) 2020-12-22
TW201704470A (zh) 2017-02-01
SG10201907396XA (en) 2019-09-27
WO2016130589A3 (en) 2016-09-15
UY36554A (es) 2016-09-30
AR103646A1 (es) 2017-05-24
CA2976075A1 (en) 2016-08-18
PH12017501432A1 (en) 2018-01-15
CO2017009083A2 (es) 2018-01-31
KR20170110149A (ko) 2017-10-10
TWI781079B (zh) 2022-10-21
US20180023082A1 (en) 2018-01-25
US10450563B2 (en) 2019-10-22
EP3256588A2 (en) 2017-12-20
US20200109401A1 (en) 2020-04-09

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