JP7315594B2 - Ｌｒｒｋ２発現を低減するための化合物及び方法 - Google Patents

Ｌｒｒｋ２発現を低減するための化合物及び方法 Download PDF

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JP7315594B2
JP7315594B2 JP2020571686A JP2020571686A JP7315594B2 JP 7315594 B2 JP7315594 B2 JP 7315594B2 JP 2020571686 A JP2020571686 A JP 2020571686A JP 2020571686 A JP2020571686 A JP 2020571686A JP 7315594 B2 JP7315594 B2 JP 7315594B2
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certain embodiments
modified
lrrk2
seq
oligonucleotide
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JP2021530204A5 (2
JP2021530204A (ja
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コール，トレイシー・エイ
コルダシエビッチ，ホリー
ブイ，フィン・ホア
フレアー，スーザン・エム
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A—HUMAN NECESSITIES
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    • A61K47/46—Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Health & Medical Sciences (AREA)
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  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020571686A 2018-06-27 2019-06-27 Ｌｒｒｋ２発現を低減するための化合物及び方法 Active JP7315594B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023114861A JP7678033B2 (ja) 2018-06-27 2023-07-13 Ｌｒｒｋ２発現を低減するための化合物及び方法
JP2025076009A JP2025121956A (ja) 2018-06-27 2025-05-01 Ｌｒｒｋ２発現を低減するための化合物及び方法

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US201862690790P 2018-06-27 2018-06-27
US62/690,790 2018-06-27
PCT/US2019/039558 WO2020006267A1 (en) 2018-06-27 2019-06-27 Compounds and methods for reducing lrrk2 expression

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JP2021530204A JP2021530204A (ja) 2021-11-11
JP2021530204A5 JP2021530204A5 (2) 2022-07-01
JP7315594B2 true JP7315594B2 (ja) 2023-07-26

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JP2020571686A Active JP7315594B2 (ja) 2018-06-27 2019-06-27 Ｌｒｒｋ２発現を低減するための化合物及び方法
JP2023114861A Active JP7678033B2 (ja) 2018-06-27 2023-07-13 Ｌｒｒｋ２発現を低減するための化合物及び方法
JP2025076009A Pending JP2025121956A (ja) 2018-06-27 2025-05-01 Ｌｒｒｋ２発現を低減するための化合物及び方法

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JP2025076009A Pending JP2025121956A (ja) 2018-06-27 2025-05-01 Ｌｒｒｋ２発現を低減するための化合物及び方法

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US (4) US11332746B1 (2)
EP (1) EP3814503A4 (2)
JP (3) JP7315594B2 (2)
KR (1) KR102768381B1 (2)
CN (1) CN112400019B (2)
AR (1) AR116102A1 (2)
AU (1) AU2019294838B2 (2)
BR (1) BR112020024564A2 (2)
CA (1) CA3104676A1 (2)
CL (1) CL2020003309A1 (2)
CO (1) CO2020015256A2 (2)
CR (1) CR20210044A (2)
EA (1) EA202190140A1 (2)
IL (1) IL279632B1 (2)
JO (1) JOP20200334A1 (2)
MX (1) MX2020013653A (2)
MY (1) MY205775A (2)
PE (1) PE20210518A1 (2)
PH (1) PH12021500003A1 (2)
SG (1) SG11202011864XA (2)
TW (1) TWI833770B (2)
UA (1) UA128406C2 (2)
UY (1) UY38282A (2)
WO (1) WO2020006267A1 (2)
ZA (1) ZA202007444B (2)

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CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
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
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US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
KR102767739B1 (ko) 2018-06-27 2025-02-12 브리스톨-마이어스 스큅 컴퍼니 T 세포 활성화제로서 유용한 치환된 나프티리디논 화합물
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
AU2021211723A1 (en) * 2020-01-24 2022-08-04 Alnylam Pharmaceuticals, Inc. Leucine-rich repeat kinase 2 (LRRK2) iRNA agent compositions and methods of use thereof
BR112022022511A2 (pt) 2020-05-06 2022-12-13 Servier Lab Inibidores macrocíclicos de lrrk2 quinase
CA3189922A1 (en) 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
BR112023017834A2 (pt) 2021-03-18 2023-10-03 Servier Lab Inibidores de lrrk2 cinase macrocíclicos
WO2022206816A1 (zh) * 2021-03-30 2022-10-06 南京大学 一种用于治疗帕金森的rna质粒递送系统
WO2022223515A2 (en) 2021-04-19 2022-10-27 Novo Nordisk A/S Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
US20240191304A1 (en) * 2021-04-20 2024-06-13 Institute For Cancer Research D/B/A The Research Institute Of Fox Chase Cancer Center Malignant Mesothelioma Susceptibility As A Result Of Germline Leucine-Rich Repeat Kinase 2 (LRRK2) Alterations
TW202317149A (zh) * 2021-06-29 2023-05-01 美商艾拉倫製藥公司 富含白胺酸之重複激酶２（ＬＲＲＫ２）ｉＲＮＡ藥劑組合物及其使用方法
CA3247528A1 (en) * 2022-01-26 2023-08-03 Syddansk Universitet SPLIT-SWITCHING ALLELE-SPECIFIC OLIGONUCLEOTIDES TARGETTING PSEUDOEXONS
CN120129790A (zh) 2022-10-28 2025-06-10 日本精工株式会社 旋转支承装置及轴支承装置的支承机构位置调整机构
EP4731763A1 (en) * 2023-06-20 2026-04-29 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
AU2024353921A1 (en) * 2023-09-27 2026-04-16 Neuro3 Therapeutics (Suzhou) Ltd. Novel sirna constructs, therapeutics, and modifications
TW202600826A (zh) * 2024-03-14 2026-01-01 大陸商上海拓界生物醫藥科技有限公司 靶向ＬＲＲＫ２的ＲＮＡｉ劑及其醫藥用途

Citations (3)

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JP2016528891A (ja) 2013-07-19 2016-09-23 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． タウ発現を調節するための組成物
WO2017120365A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
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