CO2017007335A2 - Supresión del gen de la huntingtina inducida por la arni - Google Patents

Supresión del gen de la huntingtina inducida por la arni

Info

Publication number
CO2017007335A2
CO2017007335A2 CONC2017/0007335A CO2017007335A CO2017007335A2 CO 2017007335 A2 CO2017007335 A2 CO 2017007335A2 CO 2017007335 A CO2017007335 A CO 2017007335A CO 2017007335 A2 CO2017007335 A2 CO 2017007335A2
Authority
CO
Colombia
Prior art keywords
rna
arni
suppression
double stranded
huntingtin gene
Prior art date
Application number
CONC2017/0007335A
Other languages
English (en)
Inventor
Pavlina Stefanova Konstantinova
Jana MINIARIKOVÁ
Original Assignee
Uniqure Ip Bv
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 Uniqure Ip Bv filed Critical Uniqure Ip Bv
Publication of CO2017007335A2 publication Critical patent/CO2017007335A2/es

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Classifications

    • 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
    • C12N15/11—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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

Abstract

RESUMEN La presente invención provee un ARN de doble cadena que comprende una primera secuencia de ARN y una segunda secuencia de ARN, en donde la primera y segunda secuencias de ARN son sustancialmente complementarias, en donde la primera secuencia de ARN tiene una longitud de secuencia de por lo menos 19 nucleótidos y es sustancialmente complementaria con SEQ ID NO. 1; dicho ARN de doble cadena es para su uso en la inducción de ARNi contra las secuencias del exón 1 de la huntingtina; el ARN de doble cadena de conformidad con la invención fue capaz de reducir la muerte de las células neuronales y los agregados de la huntingtina en un modelo animal.
CONC2017/0007335A 2014-12-24 2017-07-24 Supresión del gen de la huntingtina inducida por la arni CO2017007335A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP14200308 2014-12-24
PCT/EP2015/081157 WO2016102664A1 (en) 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression

Publications (1)

Publication Number Publication Date
CO2017007335A2 true CO2017007335A2 (es) 2018-01-05

Family

ID=52282540

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2017/0007335A CO2017007335A2 (es) 2014-12-24 2017-07-24 Supresión del gen de la huntingtina inducida por la arni

Country Status (25)

Country Link
US (4) US10174321B2 (es)
EP (2) EP3237618B1 (es)
JP (2) JP6839094B2 (es)
KR (1) KR102636633B1 (es)
CN (1) CN108064292B (es)
AU (1) AU2015370903B2 (es)
BR (1) BR112017013573A2 (es)
CA (1) CA2971920A1 (es)
CO (1) CO2017007335A2 (es)
CY (1) CY1121733T1 (es)
DK (1) DK3237618T3 (es)
EA (1) EA037696B1 (es)
ES (1) ES2732023T3 (es)
HK (1) HK1246344B (es)
HR (1) HRP20190992T1 (es)
HU (1) HUE043842T2 (es)
IL (1) IL252990B (es)
LT (1) LT3237618T (es)
MX (1) MX2017008500A (es)
PL (1) PL3237618T3 (es)
PT (1) PT3237618T (es)
RS (1) RS58862B1 (es)
SI (1) SI3237618T1 (es)
TR (1) TR201908969T4 (es)
WO (1) WO2016102664A1 (es)

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Also Published As

Publication number Publication date
EP3237618B1 (en) 2019-05-22
KR102636633B1 (ko) 2024-02-13
AU2015370903B2 (en) 2021-06-17
TR201908969T4 (tr) 2019-07-22
JP2021000136A (ja) 2021-01-07
DK3237618T3 (da) 2019-06-24
CA2971920A1 (en) 2016-06-30
ES2732023T3 (es) 2019-11-20
WO2016102664A1 (en) 2016-06-30
US20200339992A1 (en) 2020-10-29
CY1121733T1 (el) 2020-07-31
PL3237618T3 (pl) 2019-09-30
HRP20190992T1 (hr) 2019-09-20
EP3237618A1 (en) 2017-11-01
BR112017013573A2 (pt) 2018-03-06
US10174321B2 (en) 2019-01-08
EA037696B1 (ru) 2021-05-12
CN108064292A (zh) 2018-05-22
LT3237618T (lt) 2019-07-10
IL252990A0 (en) 2017-08-31
RS58862B1 (sr) 2019-07-31
US20230119344A1 (en) 2023-04-20
JP6839094B2 (ja) 2021-03-03
IL252990B (en) 2021-09-30
KR20170120572A (ko) 2017-10-31
US10767180B2 (en) 2020-09-08
MX2017008500A (es) 2018-02-01
US20170355989A1 (en) 2017-12-14
EA201791417A1 (ru) 2017-12-29
US11371044B2 (en) 2022-06-28
AU2015370903A1 (en) 2017-07-20
PT3237618T (pt) 2019-07-04
HK1246344B (zh) 2020-01-10
SI3237618T1 (sl) 2019-09-30
EP3540063A1 (en) 2019-09-18
CN108064292B (zh) 2021-05-04
JP2018502601A (ja) 2018-02-01
HUE043842T4 (hu) 2019-08-28
US20190144860A1 (en) 2019-05-16
NZ733296A (en) 2021-10-29
HUE043842T2 (hu) 2019-09-30

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