MX2024011539A - Composiciones de acido ribonucleico de interferencia corto de hebra doble (arnip) contra alfa-sinucleina (snca) para tratar enfermedades asociadas con la misma. - Google Patents

Composiciones de acido ribonucleico de interferencia corto de hebra doble (arnip) contra alfa-sinucleina (snca) para tratar enfermedades asociadas con la misma.

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Publication number
MX2024011539A
MX2024011539A MX2024011539A MX2024011539A MX2024011539A MX 2024011539 A MX2024011539 A MX 2024011539A MX 2024011539 A MX2024011539 A MX 2024011539A MX 2024011539 A MX2024011539 A MX 2024011539A MX 2024011539 A MX2024011539 A MX 2024011539A
Authority
MX
Mexico
Prior art keywords
snca
treating
associated disease
targeting sirna
sirna compositions
Prior art date
Application number
MX2024011539A
Other languages
English (en)
Spanish (es)
Inventor
Tuyen Nguyen
Mark K Schlegel
Adam Castoreno
Joseph Barry
Dang Lan Thi Hoang
Kawai So
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 MX2024011539A publication Critical patent/MX2024011539A/es

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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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • A61P25/28—Drugs 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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    • C12N2310/00—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
    • C12N2310/10—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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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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
    • C12N2310/35—Nature of the modification
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    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • 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
    • C—CHEMISTRY; METALLURGY
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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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  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
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  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Psychiatry (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)
MX2024011539A 2022-04-01 2023-03-31 Composiciones de acido ribonucleico de interferencia corto de hebra doble (arnip) contra alfa-sinucleina (snca) para tratar enfermedades asociadas con la misma. MX2024011539A (es)

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
MX2024011539A true MX2024011539A (es) 2024-09-26

Family

ID=88203639

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024011539A MX2024011539A (es) 2022-04-01 2023-03-31 Composiciones de acido ribonucleico de interferencia corto de hebra doble (arnip) contra alfa-sinucleina (snca) para tratar enfermedades asociadas con la misma.

Country Status (13)

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

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026085164A2 (en) 2024-10-16 2026-04-23 Alnylam Pharmaceuticals, Inc. Snca irna formulations and methods of use thereof

Also Published As

Publication number Publication date
JP2025513757A (ja) 2025-04-30
CO2024012499A2 (es) 2024-10-10
US20250059541A1 (en) 2025-02-20
KR20240166577A (ko) 2024-11-26
EP4504938A2 (en) 2025-02-12
CA3254960A1 (en) 2023-10-05
AU2023241905A1 (en) 2024-09-26
TW202407100A (zh) 2024-02-16
IL315594A (en) 2024-11-01
WO2023192977A2 (en) 2023-10-05
CN119156448A (zh) 2024-12-17
US20250115911A1 (en) 2025-04-10
CL2024002960A1 (es) 2025-03-07

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