IL322701A - Compounds and methods for regulating alpha-synuclein expression - Google Patents

Compounds and methods for regulating alpha-synuclein expression

Info

Publication number
IL322701A
IL322701A IL322701A IL32270125A IL322701A IL 322701 A IL322701 A IL 322701A IL 322701 A IL322701 A IL 322701A IL 32270125 A IL32270125 A IL 32270125A IL 322701 A IL322701 A IL 322701A
Authority
IL
Israel
Prior art keywords
modified
oligomeric compound
sugar moiety
certain embodiments
nucleobase
Prior art date
Application number
IL322701A
Other languages
English (en)
Hebrew (he)
Inventor
Hien Thuy Zhao
Susan M Freier
W Brad Wan
Antony Thomas
Michael T Migawa
Punit P Seth
Bethany Fitzsimmons
Original Assignee
Ionis Pharmaceuticals Inc
Hien Thuy Zhao
Susan M Freier
W Brad Wan
Antony Thomas
Michael T Migawa
Punit P Seth
Bethany Fitzsimmons
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, Hien Thuy Zhao, Susan M Freier, W Brad Wan, Antony Thomas, Michael T Migawa, Punit P Seth, Bethany Fitzsimmons filed Critical Ionis Pharmaceuticals Inc
Publication of IL322701A publication Critical patent/IL322701A/en

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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL322701A 2023-02-17 2024-02-16 Compounds and methods for regulating alpha-synuclein expression IL322701A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202363485845P 2023-02-17 2023-02-17
US202363587718P 2023-10-03 2023-10-03
PCT/US2024/016097 WO2024173759A2 (en) 2023-02-17 2024-02-16 Compounds and methods for modulating alpha-synuclein expression

Publications (1)

Publication Number Publication Date
IL322701A true IL322701A (en) 2025-10-01

Family

ID=92420759

Family Applications (1)

Application Number Title Priority Date Filing Date
IL322701A IL322701A (en) 2023-02-17 2024-02-16 Compounds and methods for regulating alpha-synuclein expression

Country Status (8)

Country Link
EP (1) EP4665357A2 (de)
JP (1) JP2026507572A (de)
KR (1) KR20250158088A (de)
CN (1) CN120958135A (de)
AU (1) AU2024220937A1 (de)
IL (1) IL322701A (de)
MX (1) MX2025009645A (de)
WO (1) WO2024173759A2 (de)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220062517A (ko) 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 결합 변형된 올리고머 화합물 및 이의 용도

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2817960C (en) * 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
BR122020018668B1 (pt) * 2012-08-27 2022-05-17 Evogene Ltd. Método para aumentar o rendimento, taxa de crescimento, biomassa, vigor, rendimento da semente, rendimento da fibra, capacidade fotossintética, e/ou tolerância ao estresse abiótico em uma planta
SG11202006528XA (en) * 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
WO2023215863A2 (en) * 2022-05-06 2023-11-09 Ionis Pharmaceuticals, Inc. Rnai agents for modulating snca

Also Published As

Publication number Publication date
WO2024173759A2 (en) 2024-08-22
CN120958135A (zh) 2025-11-14
WO2024173759A3 (en) 2024-10-24
MX2025009645A (es) 2026-01-07
EP4665357A2 (de) 2025-12-24
AU2024220937A1 (en) 2025-10-02
JP2026507572A (ja) 2026-03-04
KR20250158088A (ko) 2025-11-05

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