CY1121733T1 - Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης - Google Patents

Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης

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Publication number
CY1121733T1
CY1121733T1 CY20191100616T CY191100616T CY1121733T1 CY 1121733 T1 CY1121733 T1 CY 1121733T1 CY 20191100616 T CY20191100616 T CY 20191100616T CY 191100616 T CY191100616 T CY 191100616T CY 1121733 T1 CY1121733 T1 CY 1121733T1
Authority
CY
Cyprus
Prior art keywords
rna
rnai
double
sequence
huntingtin gene
Prior art date
Application number
CY20191100616T
Other languages
English (en)
Inventor
Pavlina Stefanova KONSTANTINOVA
Jana Miniarikova
Original Assignee
Uniqure Ip B.V.
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 B.V. filed Critical Uniqure Ip B.V.
Publication of CY1121733T1 publication Critical patent/CY1121733T1/el

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    • C—CHEMISTRY; METALLURGY
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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
    • A61K31/713—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
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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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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Η παρούσα εφεύρεση εξασφαλίζει ένα δίκλωνο RNA που περιλαμβάνει μια πρώτη αλληλουχία RNA και μια δεύτερη αλληλουχία RNA όπου η πρώτη και δεύτερη αλληλουχία RNA είναι ουσιαστικά συμπληρωματικές, όπου η πρώτη αλληλουχία RNA έχει ένα μήκος αλληλουχίας τουλάχιστον 19 νουκλεοτίδια και είναι ουσιαστικά συμπληρωματική στην SEQ ID NO. 1. Το ρηθέν δίκλωνο RNA είναι προς χρήση στην επαγωγή RNAi κατά αλληλουχιών του εξονίου 1 της Χαντινγκτίνης. Το δίκλωνο RNA της εφεύρεσης ήταν ικανό μείωσης του νευρωνικού κυτταρικού θανάτου και συσσωματωμάτων της χαντινγκτίνης σε ένα ζωικό μοντέλο.
CY20191100616T 2014-12-24 2019-06-13 Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης CY1121733T1 (el)

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
CY1121733T1 true CY1121733T1 (el) 2020-07-31

Family

ID=52282540

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20191100616T CY1121733T1 (el) 2014-12-24 2019-06-13 Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης

Country Status (24)

Country Link
US (4) US10174321B2 (el)
EP (2) EP3237618B1 (el)
JP (2) JP6839094B2 (el)
KR (1) KR102636633B1 (el)
CN (1) CN108064292B (el)
AU (1) AU2015370903B2 (el)
CA (1) CA2971920C (el)
CO (1) CO2017007335A2 (el)
CY (1) CY1121733T1 (el)
DK (1) DK3237618T3 (el)
EA (1) EA037696B1 (el)
ES (1) ES2732023T3 (el)
HK (1) HK1246344B (el)
HR (1) HRP20190992T1 (el)
HU (1) HUE043842T2 (el)
IL (1) IL252990B (el)
LT (1) LT3237618T (el)
MX (1) MX2017008500A (el)
PL (1) PL3237618T3 (el)
PT (1) PT3237618T (el)
RS (1) RS58862B1 (el)
SI (1) SI3237618T1 (el)
TR (1) TR201908969T4 (el)
WO (1) WO2016102664A1 (el)

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Also Published As

Publication number Publication date
HK1246344B (zh) 2020-01-10
PT3237618T (pt) 2019-07-04
JP6839094B2 (ja) 2021-03-03
US20200339992A1 (en) 2020-10-29
US10174321B2 (en) 2019-01-08
IL252990B (en) 2021-09-30
EA201791417A1 (ru) 2017-12-29
KR20170120572A (ko) 2017-10-31
EP3540063A1 (en) 2019-09-18
EA037696B1 (ru) 2021-05-12
JP2021000136A (ja) 2021-01-07
US20190144860A1 (en) 2019-05-16
BR112017013573A2 (pt) 2018-03-06
KR102636633B1 (ko) 2024-02-13
TR201908969T4 (tr) 2019-07-22
AU2015370903B2 (en) 2021-06-17
EP3237618A1 (en) 2017-11-01
RS58862B1 (sr) 2019-07-31
HRP20190992T1 (hr) 2019-09-20
WO2016102664A1 (en) 2016-06-30
HUE043842T2 (hu) 2019-09-30
MX2017008500A (es) 2018-02-01
US11371044B2 (en) 2022-06-28
LT3237618T (lt) 2019-07-10
CA2971920A1 (en) 2016-06-30
CA2971920C (en) 2024-05-07
AU2015370903A1 (en) 2017-07-20
DK3237618T3 (da) 2019-06-24
US10767180B2 (en) 2020-09-08
US20230119344A1 (en) 2023-04-20
CN108064292A (zh) 2018-05-22
US20170355989A1 (en) 2017-12-14
ES2732023T3 (es) 2019-11-20
JP2018502601A (ja) 2018-02-01
NZ733296A (en) 2021-10-29
IL252990A0 (en) 2017-08-31
PL3237618T3 (pl) 2019-09-30
SI3237618T1 (sl) 2019-09-30
CN108064292B (zh) 2021-05-04
CO2017007335A2 (es) 2018-01-05
HUE043842T4 (hu) 2019-08-28
EP3237618B1 (en) 2019-05-22

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