PE20240696A1 - Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina - Google Patents

Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina

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Publication number
PE20240696A1
PE20240696A1 PE2023002509A PE2023002509A PE20240696A1 PE 20240696 A1 PE20240696 A1 PE 20240696A1 PE 2023002509 A PE2023002509 A PE 2023002509A PE 2023002509 A PE2023002509 A PE 2023002509A PE 20240696 A1 PE20240696 A1 PE 20240696A1
Authority
PE
Peru
Prior art keywords
alpha
expression
antisense oligonucleotides
sinuclein
inhibit
Prior art date
Application number
PE2023002509A
Other languages
English (en)
Inventor
Helène Tran
Benjamin Chanrion
Sofia Lotfi
Dores Guillaume Das
Thierry Dorval
Raphael Guignard
Original Assignee
Servier Lab
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 Servier Lab filed Critical Servier Lab
Publication of PE20240696A1 publication Critical patent/PE20240696A1/es

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    • 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
    • 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
    • C12N2310/11—Antisense
    • 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
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    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
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  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invencion se refiere a oligonucleotidos antisentido (ASO) para regular la expresion de la alfa-sinucleina y el uso de los mismos para tratar sinucleinopatias. En particular, la invencion se refiere a un oligonucleotido que comprende una secuencia de nucleotidos de 15 a 30 nucleotidos contiguos, en donde la secuencia de nucleotidos es complementaria a una region de la misma longitud encontrada en los nucleotidos: a) 16350-16450, 5 b) 18926-19030, c) 22250-22471, entre otros; de la SEQ ID NO: 1, opcionalmente en donde la secuencia de nucleotidos comprende no mas 15 de 3 emparejamientos erroneos con dicha region.
PE2023002509A 2021-03-08 2022-03-07 Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina PE20240696A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21315033 2021-03-08
EP21306123 2021-08-17
PCT/EP2022/055770 WO2022189363A1 (en) 2021-03-08 2022-03-07 Antisense oligonucleotides for inhibiting alpha-synuclein expression

Publications (1)

Publication Number Publication Date
PE20240696A1 true PE20240696A1 (es) 2024-04-10

Family

ID=80738805

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2023002509A PE20240696A1 (es) 2021-03-08 2022-03-07 Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina

Country Status (10)

Country Link
EP (1) EP4305168A1 (es)
JP (1) JP2024508956A (es)
AU (1) AU2022234522A1 (es)
CA (1) CA3212650A1 (es)
CL (1) CL2023002644A1 (es)
IL (1) IL305668A (es)
MX (1) MX2023010535A (es)
PE (1) PE20240696A1 (es)
TW (1) TW202305132A (es)
WO (1) WO2022189363A1 (es)

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1331011A3 (en) 1991-10-24 2003-12-17 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
EP2534248B1 (en) * 2010-02-08 2018-08-29 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP3067421B1 (en) * 2011-02-08 2018-10-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP3336189A1 (en) * 2012-04-20 2018-06-20 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
AU2013346767B2 (en) 2012-11-15 2019-04-11 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
NZ631552A (en) 2013-05-01 2017-02-24 Ionis Pharmaceuticals Inc Compositions and methods for modulating hbv expression
PE20160158A1 (es) 2013-06-27 2016-03-18 Roche Innovation Ct Copenhagen As Oligomeros antisentido y conjugados con diana en pcsk9
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
EP3585807A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
TWI809004B (zh) * 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
CA3085964A1 (en) * 2018-01-12 2019-07-18 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
EP3966328A4 (en) 2019-05-06 2023-10-18 University Of Massachusetts ANTI-C9ORF72 OLIGONUCLEOTIDES AND RELATED METHODS

Also Published As

Publication number Publication date
TW202305132A (zh) 2023-02-01
AU2022234522A1 (en) 2023-10-26
IL305668A (en) 2023-11-01
EP4305168A1 (en) 2024-01-17
MX2023010535A (es) 2023-09-19
JP2024508956A (ja) 2024-02-28
CL2023002644A1 (es) 2024-04-01
WO2022189363A1 (en) 2022-09-15
CA3212650A1 (en) 2022-09-15

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