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MX2024013992A - Composiciones y metodos para inhibir la expresion de snca - Google Patents

Composiciones y metodos para inhibir la expresion de snca

Info

Publication number
MX2024013992A
MX2024013992A MX2024013992A MX2024013992A MX2024013992A MX 2024013992 A MX2024013992 A MX 2024013992A MX 2024013992 A MX2024013992 A MX 2024013992A MX 2024013992 A MX2024013992 A MX 2024013992A MX 2024013992 A MX2024013992 A MX 2024013992A
Authority
MX
Mexico
Prior art keywords
snca
compositions
expression
inhibiting
methods
Prior art date
Application number
MX2024013992A
Other languages
English (en)
Inventor
Bob Dale Brown
Henryk T Dudek
Seongmoon Cheong
Shiyu Wang
Travis Grim
Matthew Guese Costales
Maire Jung
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of MX2024013992A publication Critical patent/MX2024013992A/es

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    • 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
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    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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  • Psychology (AREA)
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  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Bakery Products And Manufacturing Methods Therefor (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En el presente documento se proporcionan oligonucleótidos (por ejemplo, oligonucleótidos de ARNi) que inhiben la expresión génica de SNCA, incluyendo oligonucleótidos conjugados con un ligando de direccionamiento (por ejemplo, resto de GalNAC o resto lipídico). También se proporcionan composiciones que incluyen estas y sus usos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociadas con la expresión génica de SNCA.
MX2024013992A 2022-05-13 2024-11-12 Composiciones y metodos para inhibir la expresion de snca MX2024013992A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263364639P 2022-05-13 2022-05-13
PCT/US2023/022007 WO2023220351A1 (en) 2022-05-13 2023-05-12 Compositions and methods for inhibiting snca expression

Publications (1)

Publication Number Publication Date
MX2024013992A true MX2024013992A (es) 2024-12-06

Family

ID=86760635

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024013992A MX2024013992A (es) 2022-05-13 2024-11-12 Composiciones y metodos para inhibir la expresion de snca

Country Status (17)

Country Link
US (1) US20230416743A1 (es)
EP (1) EP4522748A1 (es)
JP (1) JP2025518507A (es)
KR (1) KR20250024534A (es)
CN (1) CN119630791A (es)
AR (1) AR129312A1 (es)
AU (1) AU2023267664A1 (es)
CA (1) CA3250061A1 (es)
CL (2) CL2024003390A1 (es)
CO (1) CO2024015326A2 (es)
CR (1) CR20240494A (es)
DO (1) DOP2024000226A (es)
IL (1) IL316843A (es)
MX (1) MX2024013992A (es)
PE (1) PE20250929A1 (es)
TW (1) TW202409275A (es)
WO (1) WO2023220351A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025151407A1 (en) * 2024-01-08 2025-07-17 Denali Therapeutics Inc. Compositions and methods for modulating alpha-synuclein expression

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US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
DE69431669T2 (de) 1993-09-02 2003-10-23 Ribozyme Pharmaceuticals, Inc. Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
EP1309726B2 (en) 2000-03-30 2018-10-03 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
JP2005508196A (ja) 2001-11-07 2005-03-31 アプレラ コーポレイション 核酸分析の汎用ヌクレオチド
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
WO2009079399A2 (en) * 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
AU2009293636A1 (en) 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsRNA possessing modifications
EP2379083B1 (en) 2008-12-18 2017-08-16 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
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EP3569711B1 (en) 2014-12-15 2021-02-03 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
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AU2021321430A1 (en) * 2020-08-04 2023-03-02 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting PLP1 expression
CA3193830A1 (en) * 2020-10-01 2022-04-07 Mangala Meenakshi Soundarapandian Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases

Also Published As

Publication number Publication date
CR20240494A (es) 2025-03-03
AU2023267664A1 (en) 2024-10-31
WO2023220351A1 (en) 2023-11-16
CL2024003390A1 (es) 2025-03-14
AR129312A1 (es) 2024-08-14
US20230416743A1 (en) 2023-12-28
KR20250024534A (ko) 2025-02-18
CN119630791A (zh) 2025-03-14
DOP2024000226A (es) 2024-12-15
CO2024015326A2 (es) 2024-12-30
CL2025002529A1 (es) 2025-11-28
TW202409275A (zh) 2024-03-01
CA3250061A1 (en) 2023-11-16
IL316843A (en) 2025-01-01
PE20250929A1 (es) 2025-04-02
EP4522748A1 (en) 2025-03-19
JP2025518507A (ja) 2025-06-17

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