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 (15)

* 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
EP3797780B1 (fr) * 2014-04-17 2022-09-14 Biogen MA Inc. Compositions et procédés de modulation de l'épissage du smn2 chez un sujet
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017106377A1 (fr) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Oligomères antisens destinés au traitement du retard mental dominant autosomique 5 et du syndrome de dravet
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
CA3099280A1 (fr) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methodes et compositions pour le traitement d'une maladie de stockage d'ester de cholesteryle
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
EP4013387A4 (fr) * 2019-08-15 2023-09-27 Biogen MA Inc. Polythérapie pour atrophie musculaire spinale
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物

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
EP3470072A1 (fr) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions et procédés pour modulation de l'épissage du smn2
WO2007031091A2 (fr) 2005-09-15 2007-03-22 Santaris Pharma A/S Composes antagonistes d'arn de modulation de l'expression de p21 ras
WO2007090071A2 (fr) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. Analogues d'acides nucleiques bicycliques modifies en position 6
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
MX2009002859A (es) 2006-09-15 2009-03-30 Enzon Pharmaceuticals Inc Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos.
WO2008034123A2 (fr) 2006-09-15 2008-03-20 Enzon Pharmaceuticals, Inc. Conjugués polymères contenant des fragments chargés positivement
CN101554074B (zh) 2006-10-30 2012-08-22 诺基亚公司 为用户设备提供运营商控制的移动性的方法、设备和系统
CA2688321A1 (fr) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. Analogues d'acides nucleiques bicycliques pontes par aminomethylene n-substitue
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009067647A1 (fr) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Analogues d'acide nucléique alpha-l-bicyclique carbocyclique
CA2758189C (fr) * 2009-04-10 2020-12-29 Association Institut De Myologie Oligonucleotides antisens tricyclo-adn, compositions, et methodes de traitement de maladies
WO2010120820A1 (fr) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions et procédés pour moduler l'épissage de smn2
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto

Also Published As

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

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