JP2023544385A - Snca関連神経変性疾患を治療又は予防するためのsnca irna組成物及びその使用方法 - Google Patents

Snca関連神経変性疾患を治療又は予防するためのsnca irna組成物及びその使用方法 Download PDF

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JP2023544385A
JP2023544385A JP2023520289A JP2023520289A JP2023544385A JP 2023544385 A JP2023544385 A JP 2023544385A JP 2023520289 A JP2023520289 A JP 2023520289A JP 2023520289 A JP2023520289 A JP 2023520289A JP 2023544385 A JP2023544385 A JP 2023544385A
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nucleotides
strand
dsrna agent
nucleotide
snca
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JP2023544385A5 (https=
JPWO2022072447A5 (https=
Inventor
マンガラ ミーナクシー サウンダラパンディアン,
ラン ティ ホアン ダン,
ジェイムズ ディ. マキニンチ,
マーク ケイ. シュリーゲル,
アダム カストレーノ,
チャラランボス カイタニス,
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Alnylam Pharmaceuticals Inc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • C12N2310/3533Halogen

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JP2023520289A 2020-10-01 2021-09-29 Snca関連神経変性疾患を治療又は予防するためのsnca irna組成物及びその使用方法 Pending JP2023544385A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063086495P 2020-10-01 2020-10-01
US63/086,495 2020-10-01
PCT/US2021/052580 WO2022072447A1 (en) 2020-10-01 2021-09-29 Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases

Publications (3)

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JP2023544385A true JP2023544385A (ja) 2023-10-23
JP2023544385A5 JP2023544385A5 (https=) 2024-10-07
JPWO2022072447A5 JPWO2022072447A5 (https=) 2024-10-07

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JP2023520289A Pending JP2023544385A (ja) 2020-10-01 2021-09-29 Snca関連神経変性疾患を治療又は予防するためのsnca irna組成物及びその使用方法

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US (1) US20240011032A1 (https=)
EP (1) EP4221838A4 (https=)
JP (1) JP2023544385A (https=)
KR (1) KR20230079405A (https=)
CN (1) CN116710107A (https=)
AR (1) AR123679A1 (https=)
AU (1) AU2021353935A1 (https=)
CA (1) CA3193830A1 (https=)
IL (1) IL301712A (https=)
MX (1) MX2023003810A (https=)
TW (1) TW202229551A (https=)
WO (1) WO2022072447A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025170027A1 (ja) * 2024-02-08 2025-08-14 テルモ株式会社 オリゴヌクレオチド

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119630791A (zh) * 2022-05-13 2025-03-14 迪克纳制药公司 用于抑制snca表达的组合物和方法
US20260103703A1 (en) * 2022-09-29 2026-04-16 Atalanta Therapeutics, Inc. siRNA COMPOSITIONS AND METHODS TARGETING ALPHA-SYNUCLEIN NUCLEIC ACIDS
WO2024166019A1 (en) * 2023-02-08 2024-08-15 Biorchestra Co., Ltd. Sirna targeting alpha-synuclein and uses thereof
AU2024335806A1 (en) * 2023-09-07 2026-04-23 Janssen Pharmaceutica Nv SNCA siRNA AND USES THEREOF
WO2025162214A1 (en) * 2024-01-30 2025-08-07 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of synuclein alpha (snca) gene

Citations (4)

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JP2007528367A (ja) * 2003-06-09 2007-10-11 アルナイラム ファーマシューティカルズ インコーポレイテッド 神経変性疾患を治療する方法
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CN108728533A (zh) * 2017-04-20 2018-11-02 常青 用于髓母细胞瘤分子分型的基因群以及snca基因作为4型髓母细胞瘤的生物标志物的用途
WO2020028816A1 (en) * 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein

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WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
WO2005045034A2 (en) * 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
US20080003570A1 (en) * 2004-12-22 2008-01-03 The General Hospital Corporation Translation enhancer elements of genes encoding human Tau protein and human alpha-synuclein protein
US20100105134A1 (en) * 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
WO2009079399A2 (en) * 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
CA3077910A1 (en) * 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
AU2019207859A1 (en) * 2018-01-12 2020-07-02 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
WO2019217459A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery

Patent Citations (4)

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Publication number Priority date Publication date Assignee Title
JP2007528367A (ja) * 2003-06-09 2007-10-11 アルナイラム ファーマシューティカルズ インコーポレイテッド 神経変性疾患を治療する方法
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CN108728533A (zh) * 2017-04-20 2018-11-02 常青 用于髓母细胞瘤分子分型的基因群以及snca基因作为4型髓母细胞瘤的生物标志物的用途
WO2020028816A1 (en) * 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025170027A1 (ja) * 2024-02-08 2025-08-14 テルモ株式会社 オリゴヌクレオチド

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TW202229551A (zh) 2022-08-01
CA3193830A1 (en) 2022-04-07
CN116710107A (zh) 2023-09-05
KR20230079405A (ko) 2023-06-07
AU2021353935A1 (en) 2023-05-25
WO2022072447A1 (en) 2022-04-07
AR123679A1 (es) 2023-01-04
EP4221838A1 (en) 2023-08-09
MX2023003810A (es) 2023-06-12
US20240011032A1 (en) 2024-01-11
EP4221838A4 (en) 2025-04-16
IL301712A (en) 2023-05-01

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