PE20190513A1 - Compuestos y metodos para la modulacion de smn2 - Google Patents

Compuestos y metodos para la modulacion de smn2

Info

Publication number
PE20190513A1
PE20190513A1 PE2019000045A PE2019000045A PE20190513A1 PE 20190513 A1 PE20190513 A1 PE 20190513A1 PE 2019000045 A PE2019000045 A PE 2019000045A PE 2019000045 A PE2019000045 A PE 2019000045A PE 20190513 A1 PE20190513 A1 PE 20190513A1
Authority
PE
Peru
Prior art keywords
smn2
compounds
modified oligonucleotide
modulation
methods
Prior art date
Application number
PE2019000045A
Other languages
English (en)
Inventor
Thazha P Prakash
Punit P Seth
Original Assignee
Ionis 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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=60952211&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PE20190513(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of PE20190513A1 publication Critical patent/PE20190513A1/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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
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    • C12N2310/11—Antisense
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    • 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
    • C12N2310/31—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
    • C12N2310/3521—Methyl
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
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  • Wood Science & Technology (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Steroid Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

Se refiere a un compuesto oligomerico compuesto de un oligonucleotido modificado que consiste en 14 a 25 nucleosidos enlazados, donde el oligonucleotido modificado es complementario a un pre-ARNm de la neurona motora de supervivencia 2 (SMN2) y el nucleosido del oligonucleotido modificado tiene a formula II, donde Bx es una nucleobase; R1 y R2 son hidrogeno o metilo. tambien esta referida a una composicion farmaceutica. Dichos compuestos son utiles para el tratamiento de la atrofia muscular espinal y sus tipos
PE2019000045A 2016-07-15 2017-07-17 Compuestos y metodos para la modulacion de smn2 PE20190513A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662363195P 2016-07-15 2016-07-15

Publications (1)

Publication Number Publication Date
PE20190513A1 true PE20190513A1 (es) 2019-04-10

Family

ID=60952211

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2019000045A PE20190513A1 (es) 2016-07-15 2017-07-17 Compuestos y metodos para la modulacion de smn2

Country Status (25)

Country Link
US (2) US20190323006A1 (es)
EP (2) EP3484524B1 (es)
JP (2) JP7197463B2 (es)
KR (1) KR102556825B1 (es)
CN (1) CN109844115B (es)
AU (2) AU2017297622A1 (es)
BR (1) BR112019000356A2 (es)
CA (1) CA3030864A1 (es)
CL (2) CL2019000115A1 (es)
CO (1) CO2019001236A2 (es)
DK (1) DK3484524T3 (es)
ES (1) ES2935658T3 (es)
FI (1) FI3484524T3 (es)
HR (1) HRP20221378T1 (es)
HU (1) HUE060831T2 (es)
IL (2) IL264216B2 (es)
LT (1) LT3484524T (es)
MX (1) MX2019000619A (es)
PE (1) PE20190513A1 (es)
PL (1) PL3484524T3 (es)
PT (1) PT3484524T (es)
RS (1) RS63928B1 (es)
SG (1) SG11201900238UA (es)
SI (1) SI3484524T1 (es)
WO (1) WO2018014041A2 (es)

Families Citing this family (11)

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Publication number Priority date Publication date Assignee Title
US9885040B2 (en) 2013-04-12 2018-02-06 The Curators Of The University Of Missouri SMN2 element 1 antisense compositions and methods and uses thereof
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
US10053465B2 (en) 2015-08-26 2018-08-21 Incyte Corporation Pyrrolopyrimidine derivatives as TAM inhibitors
RS65129B1 (sr) 2016-03-28 2024-02-29 Incyte Corp Jedinjenja pirolotriazina kao inhibitori tam
EP3988552A1 (en) 2017-09-27 2022-04-27 Incyte Corporation Salts of pyrrolotriazine derivatives useful as tam inhibitors
AU2019293618A1 (en) 2018-06-29 2021-02-18 Incyte Corporation Formulations of an AXL/MER inhibitor
JOP20220007A1 (ar) * 2019-07-19 2023-01-30 Biogen Ma Inc طرق للعلاج أو الوقاية من ضمور العضلات النخاعي
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
EP4032551A4 (en) * 2019-09-18 2023-12-20 National University Corporation Tokyo Medical and Dental University NUCLEIC ACID COMPLEX
MX2022010602A (es) 2020-02-28 2022-09-09 Ionis Pharmaceuticals Inc Compuestos y metodos para modular smn2.
FR3128873A1 (fr) 2021-11-10 2023-05-12 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale

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