AU2022303358A1 - Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof - Google Patents

Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof Download PDF

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Publication number
AU2022303358A1
AU2022303358A1 AU2022303358A AU2022303358A AU2022303358A1 AU 2022303358 A1 AU2022303358 A1 AU 2022303358A1 AU 2022303358 A AU2022303358 A AU 2022303358A AU 2022303358 A AU2022303358 A AU 2022303358A AU 2022303358 A1 AU2022303358 A1 AU 2022303358A1
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Australia
Prior art keywords
dsrna agent
nucleotide
nucleotides
strand
antisense strand
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Pending
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AU2022303358A
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English (en)
Inventor
Joseph Barry
Adam CASTORENO
Lan Thi Hoang DANG
Sarah LEBLANC
James D. Mcininch
Tuyen M. NGUYEN
Mark K. SCHLEGEL
Matthew STRICOS
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2022303358A1 publication Critical patent/AU2022303358A1/en
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    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
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    • C12Y—ENZYMES
    • 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
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • 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
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3527—Other alkyl chain
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/91—Transferases (2.)
    • G01N2333/912—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2835—Movement disorders, e.g. Parkinson, Huntington, Tourette

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AU2022303358A 2021-06-29 2022-06-29 Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof Pending AU2022303358A1 (en)

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 (1)

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AU2022303358A1 true AU2022303358A1 (en) 2024-01-18

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Country Link
US (1) US20240301426A1 (https=)
EP (1) EP4363588A1 (https=)
JP (1) JP2024528503A (https=)
KR (1) KR20240033237A (https=)
AU (1) AU2022303358A1 (https=)
BR (1) BR112023027446A2 (https=)
CA (1) CA3225740A1 (https=)
CL (3) CL2023003984A1 (https=)
CO (1) CO2023018579A2 (https=)
IL (1) IL309548A (https=)
MX (1) MX2024000065A (https=)
TW (1) TW202317149A (https=)
WO (1) WO2023278607A1 (https=)

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
AU2024353921A1 (en) 2023-09-27 2026-04-16 Neuro3 Therapeutics (Suzhou) Ltd. Novel sirna constructs, therapeutics, and modifications
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014110205A1 (en) * 2013-01-11 2014-07-17 Children's Medical Center Corporation Methods and compositions for the production of sirnas
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

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CL2025000168A1 (es) 2025-05-30
KR20240033237A (ko) 2024-03-12
JP2024528503A (ja) 2024-07-30
WO2023278607A1 (en) 2023-01-05
MX2024000065A (es) 2024-02-23
CL2025003326A1 (es) 2026-02-20
IL309548A (en) 2024-02-01
TW202317149A (zh) 2023-05-01
BR112023027446A2 (pt) 2024-03-12
CL2023003984A1 (es) 2024-08-16
EP4363588A1 (en) 2024-05-08
CA3225740A1 (en) 2023-01-05
US20240301426A1 (en) 2024-09-12
CO2023018579A2 (es) 2024-01-15

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