SG11202011864XA - Compounds and methods for reducing lrrk2 expression - Google Patents

Compounds and methods for reducing lrrk2 expression

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Publication number
SG11202011864XA
SG11202011864XA SG11202011864XA SG11202011864XA SG11202011864XA SG 11202011864X A SG11202011864X A SG 11202011864XA SG 11202011864X A SG11202011864X A SG 11202011864XA SG 11202011864X A SG11202011864X A SG 11202011864XA SG 11202011864X A SG11202011864X A SG 11202011864XA
Authority
SG
Singapore
Prior art keywords
compounds
methods
lrrk2 expression
reducing
reducing lrrk2
Prior art date
Application number
SG11202011864XA
Other languages
English (en)
Inventor
Tracy Cole
Holly Kordasiewicz
Huynh-Hoa Bui
Susan Freier
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of SG11202011864XA publication Critical patent/SG11202011864XA/en

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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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3233—Morpholino-type ring
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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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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
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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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  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
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  • Neurology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • Inorganic Chemistry (AREA)
  • Botany (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)
SG11202011864XA 2018-06-27 2019-06-27 Compounds and methods for reducing lrrk2 expression SG11202011864XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862690790P 2018-06-27 2018-06-27
PCT/US2019/039558 WO2020006267A1 (en) 2018-06-27 2019-06-27 Compounds and methods for reducing lrrk2 expression

Publications (1)

Publication Number Publication Date
SG11202011864XA true SG11202011864XA (en) 2020-12-30

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ID=68987149

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202011864XA SG11202011864XA (en) 2018-06-27 2019-06-27 Compounds and methods for reducing lrrk2 expression

Country Status (22)

Country Link
US (2) US11332746B1 (es)
EP (1) EP3814503A4 (es)
JP (2) JP7315594B2 (es)
KR (1) KR20210025055A (es)
CN (1) CN112400019B (es)
AR (1) AR116102A1 (es)
AU (1) AU2019294838A1 (es)
BR (1) BR112020024564A2 (es)
CA (1) CA3104676A1 (es)
CL (1) CL2020003309A1 (es)
CO (1) CO2020015256A2 (es)
CR (1) CR20210044A (es)
EA (1) EA202190140A1 (es)
IL (1) IL279632A (es)
JO (1) JOP20200334A1 (es)
MX (1) MX2020013653A (es)
PE (1) PE20210518A1 (es)
PH (1) PH12021500003A1 (es)
SG (1) SG11202011864XA (es)
UY (1) UY38282A (es)
WO (1) WO2020006267A1 (es)
ZA (1) ZA202007444B (es)

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Publication number Priority date Publication date Assignee Title
CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
JP7455746B2 (ja) 2018-01-12 2024-03-26 ブリストル－マイヤーズ スクイブ カンパニー アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
CA3104654A1 (en) 2018-06-27 2020-01-02 Bristol-Myers Squibb Company Substituted naphthyridinone compounds useful as t cell activators
CA3104676A1 (en) 2018-06-27 2020-01-02 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing lrrk2 expression
CR20220561A (es) 2020-05-06 2023-02-17 Servier Lab Nuevos inhibidores macrocíclicos de la quinasa de lrrk2
AU2021315992A1 (en) * 2020-07-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
AU2022239815A1 (en) 2021-03-18 2023-09-21 Les Laboratoires Servier Macrocyclic lrrk2 kinase inhibitors
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
BR112023021614A2 (pt) 2021-04-19 2024-01-16 Dicerna Pharmaceuticals Inc Composições e métodos para inibir a expressão da subfamília 1 de receptores nucleares, grupo h, membro 3 (nr1h3)
WO2022226018A1 (en) * 2021-04-20 2022-10-27 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
CA3225740A1 (en) * 2021-06-29 2023-01-05 Alnylam Pharmaceuticals, Inc. Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof
WO2023144263A1 (en) * 2022-01-26 2023-08-03 Syddansk Universitet Allele specific splice switching oligonucleotides targeting pseudoexons

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