[go: up one dir, main page]

PE20250929A1 - Composiciones y metodos para inhibir la expresion de snca - Google Patents

Composiciones y metodos para inhibir la expresion de snca

Info

Publication number
PE20250929A1
PE20250929A1 PE2024002524A PE2024002524A PE20250929A1 PE 20250929 A1 PE20250929 A1 PE 20250929A1 PE 2024002524 A PE2024002524 A PE 2024002524A PE 2024002524 A PE2024002524 A PE 2024002524A PE 20250929 A1 PE20250929 A1 PE 20250929A1
Authority
PE
Peru
Prior art keywords
snca
gene expression
oligonucleotides
region
antisense strand
Prior art date
Application number
PE2024002524A
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 PE20250929A1 publication Critical patent/PE20250929A1/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (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)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Bakery Products And Manufacturing Methods Therefor (AREA)

Abstract

Se proporcionan oligonucleotidos como oligonucleotidos de ARNi que inhiben la expresion genica de SNCA, incluyendo oligonucleotidos conjugados con un ligando de direccionamiento como resto de GalNAC o resto lipidico. Tambien se proporcionan composiciones que incluyen estas y sus usos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociadas con la expresion genica de SNCA como atrofia sistemica multiple, demencia con cuerpos de Lewy y enfermedad de Parkinson. En particular, se trata de un oligonucleotido de ARNi para reducir la expresion genica de SNCA, donde el oligonucleotido comprende una hebra de sentido y una hebra de antisentido, en donde la hebra de sentido y la hebra de antisentido forman una region duplex, en donde la hebra de antisentido comprende una region de complementariedad con una secuencia diana de ARNm de SNCA de una cualquiera de las SEQ ID NO: 1846, 1683-1845, and 1847-2066, y en donde la region de complementariedad tiene al menos 15 nucleotidos contiguos de longitud.
PE2024002524A 2022-05-13 2023-05-12 Composiciones y metodos para inhibir la expresion de snca PE20250929A1 (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
PE20250929A1 true PE20250929A1 (es) 2025-04-02

Family

ID=86760635

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2024002524A PE20250929A1 (es) 2022-05-13 2023-05-12 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

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. 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
PT748382E (pt) 1993-09-02 2003-03-31 Ribozyme Pharm Inc Acidos nucleicos enzimaticos contendo nao-nucleotidos
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
NZ522045A (en) 2000-03-30 2007-05-31 Whitehead Biomedical Inst RNA sequence-specific mediators of RNA interference
IL155991A0 (en) 2000-12-01 2003-12-23 Max Planck Gesellschaft Rna interference mediating small rna molecules
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
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)
EP1442137A4 (en) 2001-11-07 2005-08-31 Applera Corp UNIVERSAL NUCLEOTIDES FOR NUCLEIC ACID ANALYSIS
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
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, 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
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
BR112014009790A2 (pt) 2011-10-25 2018-05-15 Isis Pharmaceuticals Inc composto para modulação antisense da expressão de gccr, seu uso e composição
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
AU2017321892A1 (en) 2016-09-02 2019-02-28 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
JP2023538284A (ja) * 2020-08-04 2023-09-07 ダイセルナ ファーマシューティカルズ， インコーポレイテッド Ｐｌｐ１発現を阻害するための組成物及び方法
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
CA3250061A1 (en) 2023-11-16
WO2023220351A1 (en) 2023-11-16
CL2025002529A1 (es) 2025-11-28
DOP2024000226A (es) 2024-12-15
JP2025518507A (ja) 2025-06-17
KR20250024534A (ko) 2025-02-18
IL316843A (en) 2025-01-01
CO2024015326A2 (es) 2024-12-30
CL2024003390A1 (es) 2025-03-14
AR129312A1 (es) 2024-08-14
MX2024013992A (es) 2024-12-06
CN119630791A (zh) 2025-03-14
EP4522748A1 (en) 2025-03-19
US20230416743A1 (en) 2023-12-28
TW202409275A (zh) 2024-03-01
CR20240494A (es) 2025-03-03
AU2023267664A1 (en) 2024-10-31

Similar Documents

Publication Publication Date Title
US20230020192A1 (en) Compounds and methods for modulating c90rf72
CL2021002585A1 (es) Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central
PE20201499A1 (es) Oligonucleotidos para reducir la expresion de pd-l1
Lai et al. Synergistic inhibition of lung cancer cell invasion, tumor growth and angiogenesis using aptamer-siRNA chimeras
MX2021006745A (es) Constructos de iarn modificados quimicamente y usos de estos.
PE20211749A1 (es) Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
AR051895A1 (es) Metodo para disparar la interferencia de arn
PE20241933A1 (es) Composiciones y metodos para inhibir la expresion de la proteina angiotensinogeno (agt)
BR112022002691A2 (pt) Construtos de vesícula-aso extracelular visando stat6
PE20250400A1 (es) Composiciones y metodos para inhibir cetohexoquinasa
MX2023008469A (es) Oligonucleotidos de doble hebra modificados.
PE20250156A1 (es) Composiciones y metodos para inhibir el factor b del complemento
PE20250353A1 (es) Terapeutica con arn novedosos y usos de estos
AR124713A1 (es) Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central
PE20250929A1 (es) Composiciones y metodos para inhibir la expresion de snca
AR123679A1 (es) COMPOSICIONES DE ARNi CONTRA EL GEN SNCA Y MÉTODOS DE USO DE LAS MISMAS PARA TRATAR O PREVENIR ENFERMEDADES NEURODEGENERATIVAS ASOCIADAS CON SNCA
AR125992A1 (es) Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1)
AR125230A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
AR129710A1 (es) Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6)
PE20212248A1 (es) Molecula oligomerica de acido nucleico y uso de esta
AR124302A1 (es) Gápmeros de oligonucleótidos de objetivo tau
PE20201501A1 (es) Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
JPWO2019044974A1 (ja) スモールガイドアンチセンス核酸とその使用
PE20250834A1 (es) Composiciones y metodos para inhibir la expresion de mapt
AR131419A1 (es) TERAPÉUTICOS NOVEDOSOS DE iARN DE FAS Y USOS DE ESTOS