IL315594A - SNCA-targeted siRNA constructs for the treatment of SNCA-related diseases - Google Patents

SNCA-targeted siRNA constructs for the treatment of SNCA-related diseases

Info

Publication number
IL315594A
IL315594A IL315594A IL31559424A IL315594A IL 315594 A IL315594 A IL 315594A IL 315594 A IL315594 A IL 315594A IL 31559424 A IL31559424 A IL 31559424A IL 315594 A IL315594 A IL 315594A
Authority
IL
Israel
Prior art keywords
snca
treating
associated disease
targeting sirna
sirna compositions
Prior art date
Application number
IL315594A
Other languages
English (en)
Hebrew (he)
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of IL315594A publication Critical patent/IL315594A/en

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    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • 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
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/312Phosphonates
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    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183Diol linkers, e.g. glycols or propanediols
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    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12N2310/3521Methyl
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    • C12N2310/3527Other alkyl chain
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    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
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    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IL315594A 2022-04-01 2023-03-31 SNCA-targeted siRNA constructs for the treatment of SNCA-related diseases IL315594A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263326770P 2022-04-01 2022-04-01
US202263326813P 2022-04-02 2022-04-02
PCT/US2023/065196 WO2023192977A2 (en) 2022-04-01 2023-03-31 Snca-targeting sirna compositions for treating snca-associated disease

Publications (1)

Publication Number Publication Date
IL315594A true IL315594A (en) 2024-11-01

Family

ID=88203639

Family Applications (1)

Application Number Title Priority Date Filing Date
IL315594A IL315594A (en) 2022-04-01 2023-03-31 SNCA-targeted siRNA constructs for the treatment of SNCA-related diseases

Country Status (13)

Country Link
US (2) US20250059541A1 (https=)
EP (1) EP4504938A2 (https=)
JP (1) JP2025513757A (https=)
KR (1) KR20240166577A (https=)
CN (1) CN119156448A (https=)
AU (1) AU2023241905A1 (https=)
CA (1) CA3254960A1 (https=)
CL (1) CL2024002960A1 (https=)
CO (1) CO2024012499A2 (https=)
IL (1) IL315594A (https=)
MX (1) MX2024011539A (https=)
TW (1) TW202407100A (https=)
WO (1) WO2023192977A2 (https=)

Also Published As

Publication number Publication date
CO2024012499A2 (es) 2024-10-10
CA3254960A1 (en) 2023-10-05
WO2023192977A2 (en) 2023-10-05
MX2024011539A (es) 2024-09-26
CL2024002960A1 (es) 2025-03-07
AU2023241905A1 (en) 2024-09-26
CN119156448A (zh) 2024-12-17
JP2025513757A (ja) 2025-04-30
EP4504938A2 (en) 2025-02-12
KR20240166577A (ko) 2024-11-26
TW202407100A (zh) 2024-02-16
US20250059541A1 (en) 2025-02-20
US20250115911A1 (en) 2025-04-10

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