MA35635B1 - Composés pour la modulation de l'épissage de smn2 - Google Patents

Composés pour la modulation de l'épissage de smn2

Info

Publication number
MA35635B1
MA35635B1 MA36990A MA36990A MA35635B1 MA 35635 B1 MA35635 B1 MA 35635B1 MA 36990 A MA36990 A MA 36990A MA 36990 A MA36990 A MA 36990A MA 35635 B1 MA35635 B1 MA 35635B1
Authority
MA
Morocco
Prior art keywords
smn2
arnm
modulation
compounds
delta7
Prior art date
Application number
MA36990A
Other languages
English (en)
Inventor
Susanne Kammler
Original Assignee
Santaris Pharma As
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 Santaris Pharma As filed Critical Santaris Pharma As
Publication of MA35635B1 publication Critical patent/MA35635B1/fr

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • 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
    • C12N2310/323—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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3235—Chemical structure of the sugar modified ring structure having the O of the ribose replaced by another atom
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La présente invention concerne des composés oligomériques (oligomères) qui ciblent des acides nucléiques codant pour le smn2 humain dans une cellule, conduisant à la modulation de l'épissage d'arnm de smn2 qui favorise l'arnm de smn2 de longueur totale plutôt qu'un transcrit tronqué faiblement fonctionnel, smn2 delta7. La réduction de l'expression d'arnm de smna7 et/ou l'augmentation de l'expression d'arnm de smn2 de longueur totale sont bénéfiques pour le traitement de maladies ou de troubles associés à la surexpression ou des niveaux élevés indésirables de formes aberrantes de smn2, en particulier smn2 delta7, telles que la maladie d'aran-duchenne (amyotrophie spinale, sma).
MA36990A 2011-11-11 2014-05-07 Composés pour la modulation de l'épissage de smn2 MA35635B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161558462P 2011-11-11 2011-11-11
PCT/EP2012/072100 WO2013068441A1 (fr) 2011-11-11 2012-11-08 Composés pour la modulation de l'épissage de smn2

Publications (1)

Publication Number Publication Date
MA35635B1 true MA35635B1 (fr) 2014-11-01

Family

ID=47178001

Family Applications (1)

Application Number Title Priority Date Filing Date
MA36990A MA35635B1 (fr) 2011-11-11 2014-05-07 Composés pour la modulation de l'épissage de smn2

Country Status (13)

Country Link
US (1) US20140343127A1 (fr)
EP (1) EP2776563A1 (fr)
JP (1) JP2014533944A (fr)
KR (1) KR20140091587A (fr)
CN (1) CN103946380A (fr)
AU (1) AU2012334045A1 (fr)
BR (1) BR112014011018A2 (fr)
CA (1) CA2855241A1 (fr)
EA (1) EA201400566A1 (fr)
IL (1) IL232380A0 (fr)
MA (1) MA35635B1 (fr)
TN (1) TN2014000200A1 (fr)
WO (1) WO2013068441A1 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
ES2932304T3 (es) * 2014-04-17 2023-01-17 Biogen Ma Inc Composiciones y métodos para la modulación del empalme de SMN2 en un sujeto
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016040748A1 (fr) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions et procédés de détection d'une protéine smn chez un patient et traitement d'un patient
EP3201339A4 (fr) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Augmentation ciblée de la production de gènes nucléaires
SG11201802870RA (en) 2015-10-09 2018-05-30 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
SG11201804443UA (en) 2015-12-14 2018-06-28 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
RS65031B1 (sr) 2017-08-25 2024-02-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje stanja i bolesti
WO2019213525A1 (fr) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Méthodes et compositions pour le traitement d'une maladie de stockage d'ester de cholestéryle
EP4013387A4 (fr) * 2019-08-15 2023-09-27 Biogen MA Inc. Polythérapie pour atrophie musculaire spinale
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
MX2022014151A (es) 2020-05-11 2022-11-30 Stoke Therapeutics Inc Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4914210A (en) 1987-10-02 1990-04-03 Cetus Corporation Oligonucleotide functionalizing reagents
US4962029A (en) 1987-10-02 1990-10-09 Cetus Corporation Covalent oligonucleotide-horseradish peroxidase conjugate
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US7087229B2 (en) 2003-05-30 2006-08-08 Enzon Pharmaceuticals, Inc. Releasable polymeric conjugates based on aliphatic biodegradable linkers
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
DK3308788T3 (da) 2005-06-23 2019-01-02 Biogen Ma Inc Sammensætninger og fremgangsmåder til modulering af smn2-splejsning
WO2007031091A2 (fr) 2005-09-15 2007-03-22 Santaris Pharma A/S Composes antagonistes d'arn de modulation de l'expression de p21 ras
ES2516815T3 (es) 2006-01-27 2014-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6
WO2007134181A2 (fr) 2006-05-11 2007-11-22 Isis Pharmaceuticals, Inc. Analogues d'acides nucléiques bicycliques modifiés en 5'
CA2662520A1 (fr) 2006-09-15 2008-03-20 Enzon Pharmaceuticals, Inc. Conjugues polymeres contenant des fragments charges positivement
MX2009002859A (es) 2006-09-15 2009-03-30 Enzon Pharmaceuticals Inc Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos.
EP2090127A2 (fr) 2006-10-30 2009-08-19 Nokia Corporation Procédé, appareil et système fournissant une mobilité commandée par un opérateur pour un équipement utilisateur
EP2170917B1 (fr) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. Analogues d'acides nucléiques bicycliques pontés par aminométhylène n-substitué
EP2173760B2 (fr) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Analogues d'acide nucléique bicyclique carbocylique
EP2176280B2 (fr) 2007-07-05 2015-06-24 Isis Pharmaceuticals, Inc. Analogues d'acides nucléiques bicycliques disubstitués en position 6
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
ES2573981T3 (es) * 2009-04-10 2016-06-13 Association Institut De Myologie Oligonucleótidos antisentido de triciclo-ADN, composiciones, y métodos para el tratamiento de enfermedades
WO2010120820A1 (fr) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions et procédés pour moduler l'épissage de smn2
AU2010262862C1 (en) 2009-06-17 2020-04-30 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject

Also Published As

Publication number Publication date
EP2776563A1 (fr) 2014-09-17
JP2014533944A (ja) 2014-12-18
US20140343127A1 (en) 2014-11-20
KR20140091587A (ko) 2014-07-21
CN103946380A (zh) 2014-07-23
CA2855241A1 (fr) 2013-05-16
WO2013068441A1 (fr) 2013-05-16
TN2014000200A1 (en) 2015-09-30
AU2012334045A1 (en) 2014-04-24
BR112014011018A2 (pt) 2017-05-02
IL232380A0 (en) 2014-06-30
EA201400566A1 (ru) 2014-09-30

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