MX2016012123A - Composiciones para modular la expresion de ataxina 2. - Google Patents

Composiciones para modular la expresion de ataxina 2.

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Publication number
MX2016012123A
MX2016012123A MX2016012123A MX2016012123A MX2016012123A MX 2016012123 A MX2016012123 A MX 2016012123A MX 2016012123 A MX2016012123 A MX 2016012123A MX 2016012123 A MX2016012123 A MX 2016012123A MX 2016012123 A MX2016012123 A MX 2016012123A
Authority
MX
Mexico
Prior art keywords
ataxin
compositions
expression
modulating
methods
Prior art date
Application number
MX2016012123A
Other languages
English (en)
Inventor
M Freier Susan
Frank Bennett C
Hung Gene
Original Assignee
Ionis Pharmaceuticals Inc
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.)
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Publication date
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=54145357&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX2016012123(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of MX2016012123A publication Critical patent/MX2016012123A/es

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    • C—CHEMISTRY; METALLURGY
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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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 ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom

Abstract

La presente invención se refiere a compuestos antisentido y métodos para disminuir la expresión de ARNm y proteínas de Ataxina 2. Tales métodos, compuestos y composiciones son útiles para tratar, prevenir, o aliviar enfermedades, trastornos y afecciones asociadas a la Ataxina 2. Tales enfermedades asociadas a la Ataxina 2 incluyen ataxia espinocerebelosa tipo 2 (SCA2), esclerosis amiotrófica (ALS) y parkinsonismo.
MX2016012123A 2014-03-19 2015-03-19 Composiciones para modular la expresion de ataxina 2. MX2016012123A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461955705P 2014-03-19 2014-03-19
US201461982131P 2014-04-21 2014-04-21
PCT/US2015/021608 WO2015143246A1 (en) 2014-03-19 2015-03-19 Compositions for modulating ataxin 2 expression

Publications (1)

Publication Number Publication Date
MX2016012123A true MX2016012123A (es) 2017-01-19

Family

ID=54145357

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016012123A MX2016012123A (es) 2014-03-19 2015-03-19 Composiciones para modular la expresion de ataxina 2.

Country Status (25)

Country Link
US (3) US10308934B2 (es)
EP (2) EP3119888B1 (es)
JP (3) JP6902869B2 (es)
KR (1) KR102352589B1 (es)
CN (2) CN116970607A (es)
AU (2) AU2015231130C1 (es)
BR (1) BR112016020472B1 (es)
CA (1) CA2942340A1 (es)
CL (1) CL2016002346A1 (es)
CY (1) CY1124526T1 (es)
DK (1) DK3119888T3 (es)
ES (1) ES2890677T3 (es)
HK (1) HK1232251A1 (es)
HR (1) HRP20211397T1 (es)
HU (1) HUE055877T2 (es)
IL (1) IL247600B (es)
LT (1) LT3119888T (es)
MX (1) MX2016012123A (es)
PL (1) PL3119888T3 (es)
PT (1) PT3119888T (es)
RS (1) RS62526B1 (es)
RU (2) RU2702838C2 (es)
SG (2) SG10201808063PA (es)
SI (1) SI3119888T1 (es)
WO (1) WO2015143246A1 (es)

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WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
WO2017053781A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
EP3377629B1 (en) 2015-11-18 2023-07-05 Rosalind Franklin University of Medicine and Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
US20190002887A1 (en) * 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
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CA3098144A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
JP7379387B2 (ja) * 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
TW202020154A (zh) * 2018-07-25 2020-06-01 美商Ionis製藥公司 用於減少atxn2表現之化合物及方法
CA3121749A1 (en) 2018-12-06 2020-06-11 Biogen Ma Inc. Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis
TW202035694A (zh) * 2018-12-14 2020-10-01 美商百健Ｍａ公司 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法
CN109529018B (zh) * 2019-01-10 2021-11-30 南方医科大学南方医院 Atxn2l用于制备逆转胃癌奥沙利铂耐药的药物的用途
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EP4188390A1 (en) * 2020-07-29 2023-06-07 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
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