CA3225740A1 - 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
CA3225740A1
CA3225740A1 CA3225740A CA3225740A CA3225740A1 CA 3225740 A1 CA3225740 A1 CA 3225740A1 CA 3225740 A CA3225740 A CA 3225740A CA 3225740 A CA3225740 A CA 3225740A CA 3225740 A1 CA3225740 A1 CA 3225740A1
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Prior art keywords
dsrna agent
nucleotide
nucleotides
strand
antisense strand
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Pending
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CA3225740A
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English (en)
French (fr)
Inventor
Lan Thi Hoang DANG
James D. Mcininch
Mark K. SCHLEGEL
Adam CASTORENO
Tuyen M. NGUYEN
Joseph Barry
Matthew STRICOS
Sarah LEBLANC
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of CA3225740A1 publication Critical patent/CA3225740A1/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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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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
    • C12N2310/10—Type of nucleic acid
    • 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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    • C12N2310/30—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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    • C—CHEMISTRY; METALLURGY
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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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    • C12N2310/3525—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
    • 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
    • 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
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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)
    • G—PHYSICS
    • G01—MEASURING; TESTING
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CA3225740A 2021-06-29 2022-06-29 Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof Pending CA3225740A1 (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)

Publication Number Publication Date
CA3225740A1 true CA3225740A1 (en) 2023-01-05

Family

ID=84692073

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CA3225740A Pending CA3225740A1 (en) 2021-06-29 2022-06-29 Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof

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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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Publication number Publication date
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
JP2024528503A (ja) 2024-07-30
CL2025000168A1 (es) 2025-05-30
CO2023018579A2 (es) 2024-01-15
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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