NL300963I2 - inotersen of een zout daarvan - Google Patents

inotersen of een zout daarvan Download PDF

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Publication number
NL300963I2
NL300963I2 NL300963C NL300963C NL300963I2 NL 300963 I2 NL300963 I2 NL 300963I2 NL 300963 C NL300963 C NL 300963C NL 300963 C NL300963 C NL 300963C NL 300963 I2 NL300963 I2 NL 300963I2
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NL
Netherlands
Prior art keywords
inotersen
salt
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NL300963C
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English (en)
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NL300963I1 (nl
NL300963I9 (nl
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Ionis Pharmaceuticals Inc
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Publication of NL300963I1 publication Critical patent/NL300963I1/nl
Publication of NL300963I9 publication Critical patent/NL300963I9/nl
Publication of NL300963I2 publication Critical patent/NL300963I2/nl

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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
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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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61P7/10—Antioedematous agents; Diuretics
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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
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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
    • 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
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/52—Physical structure branched
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Cardiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Endocrinology (AREA)
  • Ophthalmology & Optometry (AREA)
NL300963C 2010-04-29 2019-01-04 inotersen of een zout daarvan NL300963I2 (nl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US32953810P 2010-04-29 2010-04-29
US40516310P 2010-10-20 2010-10-20
PCT/US2011/034661 WO2011139917A1 (en) 2010-04-29 2011-04-29 Modulation of transthyretin expression

Publications (3)

Publication Number Publication Date
NL300963I1 NL300963I1 (nl) 2019-01-08
NL300963I9 NL300963I9 (nl) 2019-01-16
NL300963I2 true NL300963I2 (nl) 2021-03-08

Family

ID=44903998

Family Applications (1)

Application Number Title Priority Date Filing Date
NL300963C NL300963I2 (nl) 2010-04-29 2019-01-04 inotersen of een zout daarvan

Country Status (25)

Country Link
US (9) US20110294868A1 (nl)
EP (1) EP2563920B1 (nl)
JP (1) JP5896175B2 (nl)
KR (2) KR101835386B1 (nl)
CN (1) CN103038345B (nl)
BR (1) BR112012027547B1 (nl)
CA (2) CA2994063A1 (nl)
CY (2) CY1119070T1 (nl)
DK (1) DK2563920T3 (nl)
ES (1) ES2625689T3 (nl)
HR (1) HRP20170737T1 (nl)
HU (2) HUE031909T2 (nl)
IL (1) IL222697A (nl)
LT (2) LT2563920T (nl)
LU (1) LUC00096I2 (nl)
MX (1) MX343559B (nl)
NL (1) NL300963I2 (nl)
NO (1) NO2019001I1 (nl)
NZ (1) NZ603339A (nl)
PL (1) PL2563920T3 (nl)
PT (1) PT2563920T (nl)
RS (1) RS56011B1 (nl)
RU (1) RU2592669C2 (nl)
SI (1) SI2563920T1 (nl)
WO (1) WO2011139917A1 (nl)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
CN103937793B (zh) 2008-10-20 2017-08-11 阿尔尼拉姆医药品有限公司 抑制运甲状腺素蛋白表达的组合物和方法
EP2496238A4 (en) 2009-11-03 2013-10-02 Alnylam Pharmaceuticals Inc FORMULATED LIPID COMPOSITIONS AND METHODS FOR INHIBITING TRANSTHYRETIN EXPRESSION (TTR)
PT2563920T (pt) * 2010-04-29 2017-05-26 Ionis Pharmaceuticals Inc Modulação da expressão de transtirretina
JP6129844B2 (ja) 2011-09-14 2017-05-17 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物
DK3301177T3 (da) 2011-11-18 2020-06-15 Alnylam Pharmaceuticals Inc Rnai-midler, sammensætninger og fremgangsmåder til anvendelse deraf til behandling af transthyretin (ttr)-forbundne sygdomme
CA2873779A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating mecp2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
WO2014043544A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
EP2920304B1 (en) 2012-11-15 2019-03-06 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
EP2951305B1 (en) 2013-01-30 2018-08-15 F.Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
EP2992098B1 (en) * 2013-05-01 2019-03-27 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating hbv and ttr expression
KR102369736B1 (ko) 2014-05-01 2022-03-02 아이오니스 파마수티컬즈, 인코포레이티드 보체 인자 b 발현을 조절하기 위한 조성물 및 방법
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
KR102631505B1 (ko) * 2014-08-29 2024-02-01 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
EP3256591A4 (en) 2015-02-13 2018-08-08 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
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