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AR127158A1 - Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina - Google Patents

Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina

Info

Publication number
AR127158A1
AR127158A1 ARP220102593A ARP220102593A AR127158A1 AR 127158 A1 AR127158 A1 AR 127158A1 AR P220102593 A ARP220102593 A AR P220102593A AR P220102593 A ARP220102593 A AR P220102593A AR 127158 A1 AR127158 A1 AR 127158A1
Authority
AR
Argentina
Prior art keywords
antisense oligonucleotide
rna associated
polyglutamine diseases
editing rna
polyq
Prior art date
Application number
ARP220102593A
Other languages
English (en)
Inventor
Lars Joenson
Tobias Merkle
Anja Moelhart Hoeg
Jonas Vikesaa
Original Assignee
Hoffmann La Roche
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 Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of AR127158A1 publication Critical patent/AR127158A1/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
    • 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
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

Landscapes

  • 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)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención se refiere a un oligonucleótido antisentido para su uso en el tratamiento y/o la prevención de una enfermedad de poliglutamina (poliQ), en donde dicho oligonucleótido antisentido puede unirse específicamente a una región de repetición CAG de un mARN que codifica una proteína relacionada con enfermedad de poliQ de modo que se forme un ARN bicatenario que puede atraer una adenosina desaminasa que actúa sobre el ARN (ADAR) que inserta un intercambio de A a I en al menos un trinucleótido CAG de la región de repetición CAG de dicho mARN. La presente invención también se refiere a una composición farmacéutica que comprende dicha molécula antisentido.
ARP220102593A 2021-09-29 2022-09-27 Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina AR127158A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP21199761 2021-09-29

Publications (1)

Publication Number Publication Date
AR127158A1 true AR127158A1 (es) 2023-12-27

Family

ID=78179173

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220102593A AR127158A1 (es) 2021-09-29 2022-09-27 Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina

Country Status (7)

Country Link
US (1) US20240336919A1 (es)
EP (1) EP4408999A1 (es)
JP (1) JP2024536132A (es)
CN (1) CN118019848A (es)
AR (1) AR127158A1 (es)
TW (1) TW202329983A (es)
WO (1) WO2023052317A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202405143D0 (en) * 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
EP2341058A3 (en) 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
TR200102328T2 (tr) 1999-02-12 2002-01-21 Sankyo Company Limited Yeni nükleosid ve oligonükleotid analogları
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
CA2994089A1 (en) 2002-11-18 2004-06-03 Roche Innovation Center Copenhagen A/S Antisense gapmer oligonucleotides
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
DK2314594T3 (da) 2006-01-27 2014-10-27 Isis Pharmaceuticals Inc 6-modificerede bicykliske nukleinsyreanaloger
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2249874A1 (en) * 2008-02-08 2010-11-17 ProSensa Holding BV Methods and means for treating dna repeat instability associated genetic disorders
EP2356129B1 (en) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
EP3922722B1 (en) 2011-08-11 2023-06-28 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2013033223A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
WO2013138353A2 (en) 2012-03-12 2013-09-19 Shire Human Genetic Therapies, Inc. Compositions and methods for modulation of atxn3 expression
WO2013154798A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
ES2639066T3 (es) * 2012-04-23 2017-10-25 Biomarin Technologies B.V. Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de trastornos neuromusculares
EP3027617A4 (en) 2013-07-31 2017-04-12 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
DK3234134T3 (da) 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
KR102418185B1 (ko) 2016-06-22 2022-07-06 프로큐알 테라퓨틱스 Ⅱ 비.브이. 단일 가닥 rna-편집 올리고뉴클레오타이드
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
WO2018134301A1 (en) 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
WO2019158475A1 (en) 2018-02-14 2019-08-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
AU2020210900B2 (en) * 2019-01-22 2025-04-24 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof

Also Published As

Publication number Publication date
TW202329983A (zh) 2023-08-01
JP2024536132A (ja) 2024-10-04
WO2023052317A1 (en) 2023-04-06
CN118019848A (zh) 2024-05-10
EP4408999A1 (en) 2024-08-07
US20240336919A1 (en) 2024-10-10

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