JP2024528503A - ロイシンリッチリピートキナーゼ２（ＬＲＲＫ２）ｉＲＮＡ剤組成物およびその使用方法 - Google Patents

ロイシンリッチリピートキナーゼ２（ＬＲＲＫ２）ｉＲＮＡ剤組成物およびその使用方法 Download PDF

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JP2024528503A
JP2024528503A JP2023579696A JP2023579696A JP2024528503A JP 2024528503 A JP2024528503 A JP 2024528503A JP 2023579696 A JP2023579696 A JP 2023579696A JP 2023579696 A JP2023579696 A JP 2023579696A JP 2024528503 A JP2024528503 A JP 2024528503A
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nucleotides
dsrna agent
strand
nucleotide
antisense strand
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JP2023579696A
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Japanese (ja)
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JP2024528503A5 (enExample
Inventor
ティ ホアン ダン，ラン
ディ マキニンチ，ジェイムズ
ケイ シュリーゲル，マーク
カストレノ，アダム
エム グエン，トゥエン
バリー，ジョセフ
ストリコス，マシュー
ルブラン，サラ
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of JP2024528503A publication Critical patent/JP2024528503A/ja
Publication of JP2024528503A5 publication Critical patent/JP2024528503A5/ja
Pending legal-status Critical Current

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • G—PHYSICS
    • G01—MEASURING; TESTING
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    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
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    • C12N2310/35—Nature of the modification
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    • C12N2310/35—Nature of the modification
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3527—Other alkyl chain
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/91—Transferases (2.)
    • G01N2333/912—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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JP2023579696A 2021-06-29 2022-06-29 ロイシンリッチリピートキナーゼ２（ＬＲＲＫ２）ｉＲＮＡ剤組成物およびその使用方法 Pending JP2024528503A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163216119P 2021-06-29 2021-06-29
US63/216,119 2021-06-29
US202263353953P 2022-06-21 2022-06-21
US63/353,953 2022-06-21
PCT/US2022/035561 WO2023278607A1 (en) 2021-06-29 2022-06-29 Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof

Publications (2)

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JP2024528503A true JP2024528503A (ja) 2024-07-30
JP2024528503A5 JP2024528503A5 (enExample) 2025-07-07

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JP2023579696A Pending JP2024528503A (ja) 2021-06-29 2022-06-29 ロイシンリッチリピートキナーゼ２（ＬＲＲＫ２）ｉＲＮＡ剤組成物およびその使用方法

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US (1) US20240301426A1 (enExample)
EP (1) EP4363588A4 (enExample)
JP (1) JP2024528503A (enExample)
KR (1) KR20240033237A (enExample)
AU (1) AU2022303358A1 (enExample)
BR (1) BR112023027446A2 (enExample)
CA (1) CA3225740A1 (enExample)
CL (3) CL2023003984A1 (enExample)
CO (1) CO2023018579A2 (enExample)
IL (1) IL309548A (enExample)
MX (1) MX2024000065A (enExample)
TW (1) TW202317149A (enExample)
WO (1) WO2023278607A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024238385A2 (en) * 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
CN121712891A (zh) * 2023-06-20 2026-03-20 阿达尔克斯制药有限公司 Lrrk2调节组合物及其使用方法
CN122074089A (zh) 2023-09-27 2026-05-22 三晟医药公司 新型siRNA构建体、治疗剂和修饰
CN120082552A (zh) * 2023-12-01 2025-06-03 艾码生物科技(南京)有限公司 初级-miRNA及其应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007124096A2 (en) * 2006-04-21 2007-11-01 The Trustees Of Columbia University In The City Of New York Lrrk2 regulaton of neuronal process morphology
GB201105137D0 (en) * 2011-03-28 2011-05-11 Isis Innovation Therapeutic molecules for use in the suppression of Parkinson's disease
WO2014110205A1 (en) * 2013-01-11 2014-07-17 Children's Medical Center Corporation Methods and compositions for the production of sirnas
EP3400300A4 (en) * 2016-01-05 2019-08-07 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING LRRK2 EXPRESSION
CA3098623A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
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
EP3769768A1 (en) * 2019-07-23 2021-01-27 Consejo Superior de Investigaciones Cientificas (CSIC) Compounds useful for the treatment of parkinson s disease

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EP4363588A4 (en) 2026-05-13
TW202317149A (zh) 2023-05-01
AU2022303358A1 (en) 2024-01-18
IL309548A (en) 2024-02-01
KR20240033237A (ko) 2024-03-12
US20240301426A1 (en) 2024-09-12
CL2025000168A1 (es) 2025-05-30
CO2023018579A2 (es) 2024-01-15
CA3225740A1 (en) 2023-01-05
MX2024000065A (es) 2024-02-23
EP4363588A1 (en) 2024-05-08
BR112023027446A2 (pt) 2024-03-12
CL2023003984A1 (es) 2024-08-16
CL2025003326A1 (es) 2026-02-20
WO2023278607A1 (en) 2023-01-05

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