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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JP2021510511A (ja
JP2021510511A5 (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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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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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
    • 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
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
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    • 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
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C07—ORGANIC CHEMISTRY
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    • 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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    • C12N15/09—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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/30—Chemical structure
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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

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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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JP2021510511A JP2021510511A (ja) 2021-04-30
JP2021510511A5 JP2021510511A5 (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=)

Families Citing this family (10)

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Publication number Priority date Publication date Assignee Title
CA3051839A1 (en) 2017-02-17 2018-08-23 Bristol-Myers Squibb Company Antibodies to alpha-synuclein and uses thereof
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
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
WO2019157531A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
JP2023509477A (ja) * 2020-01-06 2023-03-08 オーム ライフテック インコーポレイテッド 神経障害の処置のためのアンチセンスオリゴヌクレオチド
WO2022189363A1 (en) * 2021-03-08 2022-09-15 Les Laboratoires Servier Antisense oligonucleotides for inhibiting alpha-synuclein expression
KR102680316B1 (ko) * 2021-11-01 2024-07-01 오토텔릭바이오 주식회사 안티센스 올리고뉴클레오타이드
JP2026507572A (ja) * 2023-02-17 2026-03-04 アイオーニス ファーマシューティカルズ， インコーポレーテッド アルファ－シヌクレイン発現を調節するための化合物および方法
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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