JP7547201B2 - アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用 - Google Patents

アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用 Download PDF

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JP7547201B2
JP7547201B2 JP2020538629A JP2020538629A JP7547201B2 JP 7547201 B2 JP7547201 B2 JP 7547201B2 JP 2020538629 A JP2020538629 A JP 2020538629A JP 2020538629 A JP2020538629 A JP 2020538629A JP 7547201 B2 JP7547201 B2 JP 7547201B2
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aso
snca
sequence
antisense oligonucleotide
nucleotide
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Japanese (ja)
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JP2021510511A5 (https=
JP2021510511A (ja
JPWO2019140236A5 (https=
Inventor
リチャード・イー・オルソン
アンジェラ・エム・カケース
ジェリィ・イー・メレディス・ジュニア
ニノ・デヴィゼ
ジェイムズ・ケイ・ロイ
カール・ジェイ・バルディック
アンナプルナ・ペンドリ
アイバー・エム・マクドナルド
ピーター・ヘーイドーン
マリアンネ・レアベック・イェンセン
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Roche Innovation Center Copenhagen AS
Bristol Myers Squibb Co
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Roche Innovation Center Copenhagen AS
Bristol Myers Squibb Co
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Publication of JPWO2019140236A5 publication Critical patent/JPWO2019140236A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
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    • C12N2310/11—Antisense
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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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    • C12N2310/30—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
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JP2020538629A 2018-01-12 2019-01-11 アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用 Active JP7547201B2 (ja)

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JP2024076354A JP2024122973A (ja) 2018-01-12 2024-05-09 アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862616994P 2018-01-12 2018-01-12
US62/616,994 2018-01-12
PCT/US2019/013255 WO2019140236A1 (en) 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof

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Publications (4)

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JP2021510511A JP2021510511A (ja) 2021-04-30
JP2021510511A5 JP2021510511A5 (https=) 2022-01-19
JPWO2019140236A5 JPWO2019140236A5 (https=) 2022-01-19
JP7547201B2 true JP7547201B2 (ja) 2024-09-09

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JP2024076354A Withdrawn JP2024122973A (ja) 2018-01-12 2024-05-09 アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用

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US (2) US12241065B2 (https=)
EP (1) EP3737761A1 (https=)
JP (2) JP7547201B2 (https=)
KR (1) KR102839166B1 (https=)
CN (1) CN111819283A (https=)
AU (1) AU2019208011B2 (https=)
BR (1) BR112020014011A2 (https=)
CA (1) CA3087966A1 (https=)
CO (1) CO2020009715A2 (https=)
EA (1) EA202091693A1 (https=)
IL (1) IL275903B2 (https=)
MX (1) MX2020007439A (https=)
PE (1) PE20211749A1 (https=)
SG (1) SG11202006528XA (https=)
WO (1) WO2019140236A1 (https=)

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CN110506057B (zh) 2017-02-17 2023-09-29 百时美施贵宝公司 Alpha突触核蛋白抗体及其应用
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
BR112020013994A2 (pt) * 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CA3163789A1 (en) * 2020-01-06 2021-07-15 Veenu AISHWARYA Antisense oligonucleotides for treatment of neurological disorders
PE20240696A1 (es) * 2021-03-08 2024-04-10 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina
KR102680316B1 (ko) * 2021-11-01 2024-07-01 오토텔릭바이오 주식회사 안티센스 올리고뉴클레오타이드
WO2024173759A2 (en) * 2023-02-17 2024-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating alpha-synuclein expression
WO2024196752A2 (en) * 2023-03-17 2024-09-26 Duke University Compositions for cell specific snca-targeted gene therapy and methods of making and using same

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JP2014501507A (ja) 2010-11-17 2014-01-23 アイシス ファーマシューティカルズ， インコーポレーテッド α−シヌクレイン発現の調節
JP2021510512A (ja) 2018-01-12 2021-04-30 ブリストル−マイヤーズ スクイブ カンパニーＢｒｉｓｔｏｌ−Ｍｙｅｒｓ Ｓｑｕｉｂｂ Ｃｏｍｐａｎｙ アルファ−シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用

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