RS60762B1 - Oligonukleotidna jedinjenja za ciljane irnk hantingtina - Google Patents

Oligonukleotidna jedinjenja za ciljane irnk hantingtina

Info

Publication number
RS60762B1
RS60762B1 RS20200929A RSP20200929A RS60762B1 RS 60762 B1 RS60762 B1 RS 60762B1 RS 20200929 A RS20200929 A RS 20200929A RS P20200929 A RSP20200929 A RS P20200929A RS 60762 B1 RS60762 B1 RS 60762B1
Authority
RS
Serbia
Prior art keywords
huntingtin mrna
oligonucleotide compounds
targeting huntingtin
targeting
oligonucleotide
Prior art date
Application number
RS20200929A
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 RS60762B1 publication Critical patent/RS60762B1/sr

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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 N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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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
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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
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    • C12N2320/30—Special therapeutic applications
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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
RS20200929A 2015-04-03 2016-04-01 Oligonukleotidna jedinjenja za ciljane irnk hantingtina RS60762B1 (sr)

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
RS60762B1 true RS60762B1 (sr) 2020-10-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
RS20200929A RS60762B1 (sr) 2015-04-03 2016-04-01 Oligonukleotidna jedinjenja za ciljane irnk hantingtina

Country Status (17)

Country Link
US (5) US9809817B2 (sr)
EP (2) EP3277814B1 (sr)
JP (4) JP7009356B2 (sr)
CN (2) CN116004624A (sr)
AU (3) AU2016244027B2 (sr)
CA (1) CA2980337A1 (sr)
CY (1) CY1123289T1 (sr)
DK (1) DK3277814T3 (sr)
ES (1) ES2808750T3 (sr)
HR (1) HRP20201200T1 (sr)
HU (1) HUE050441T2 (sr)
LT (1) LT3277814T (sr)
PL (1) PL3277814T3 (sr)
PT (1) PT3277814T (sr)
RS (1) RS60762B1 (sr)
SI (1) SI3277814T1 (sr)
WO (1) WO2016161374A1 (sr)

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