SMT202000454T1 - Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina - Google Patents

Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina

Info

Publication number
SMT202000454T1
SMT202000454T1 SM20200454T SMT202000454T SMT202000454T1 SM T202000454 T1 SMT202000454 T1 SM T202000454T1 SM 20200454 T SM20200454 T SM 20200454T SM T202000454 T SMT202000454 T SM T202000454T SM T202000454 T1 SMT202000454 T1 SM T202000454T1
Authority
SM
San Marino
Prior art keywords
huntingtin mrna
oligonucleotide compounds
targeting huntingtin
targeting
oligonucleotide
Prior art date
Application number
SM20200454T
Other languages
English (en)
Inventor
Anastasia Khvorova
Neil Aronin
Julia Alterman
Original Assignee
Univ Massachusetts
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 Univ Massachusetts filed Critical Univ Massachusetts
Publication of SMT202000454T1 publication Critical patent/SMT202000454T1/it

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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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3517—Marker; Tag
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/52—Physical structure branched
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
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  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
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  • Biophysics (AREA)
  • Neurology (AREA)
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  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
SM20200454T 2015-04-03 2016-04-01 Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina SMT202000454T1 (it)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562142731P 2015-04-03 2015-04-03
US201662289274P 2016-01-31 2016-01-31
EP16716412.8A EP3277814B1 (en) 2015-04-03 2016-04-01 Oligonucleotide compounds for targeting huntingtin mrna
PCT/US2016/025722 WO2016161374A1 (en) 2015-04-03 2016-04-01 Oligonucleotide compounds for targeting huntingtin mrna

Publications (1)

Publication Number Publication Date
SMT202000454T1 true SMT202000454T1 (it) 2020-11-10

Family

ID=55752785

Family Applications (1)

Application Number Title Priority Date Filing Date
SM20200454T SMT202000454T1 (it) 2015-04-03 2016-04-01 Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina

Country Status (18)

Country Link
US (5) US9809817B2 (it)
EP (2) EP3277814B1 (it)
JP (5) JP7009356B2 (it)
CN (2) CN107980062B (it)
AU (4) AU2016244027B2 (it)
CA (1) CA2980337A1 (it)
CY (1) CY1123289T1 (it)
DK (1) DK3277814T3 (it)
ES (1) ES2808750T3 (it)
HR (1) HRP20201200T1 (it)
HU (1) HUE050441T2 (it)
LT (1) LT3277814T (it)
PL (1) PL3277814T3 (it)
PT (1) PT3277814T (it)
RS (1) RS60762B1 (it)
SI (1) SI3277814T1 (it)
SM (1) SMT202000454T1 (it)
WO (1) WO2016161374A1 (it)

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