CA3030864A1 - Compounds and methods for modulation of smn2 - Google Patents

Compounds and methods for modulation of smn2 Download PDF

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Publication number
CA3030864A1
CA3030864A1 CA3030864A CA3030864A CA3030864A1 CA 3030864 A1 CA3030864 A1 CA 3030864A1 CA 3030864 A CA3030864 A CA 3030864A CA 3030864 A CA3030864 A CA 3030864A CA 3030864 A1 CA3030864 A1 CA 3030864A1
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Prior art keywords
oligomeric compound
modified
modified oligonucleotide
nucleosides
compound
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Legal status (The legal status 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 status listed.)
Pending
Application number
CA3030864A
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English (en)
French (fr)
Inventor
Frank Rigo
Thazha P. Prakash
Punit P. Seth
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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=CA3030864(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 CA3030864A1 publication Critical patent/CA3030864A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • 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
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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
    • 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
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/351—Conjugate
    • 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
    • C—CHEMISTRY; METALLURGY
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Bioinformatics & Cheminformatics (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
CA3030864A 2016-07-15 2017-07-17 Compounds and methods for modulation of smn2 Pending CA3030864A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662363195P 2016-07-15 2016-07-15
US62/363,195 2016-07-15
PCT/US2017/042463 WO2018014041A2 (en) 2016-07-15 2017-07-17 Compounds and methods for modulation of smn2

Publications (1)

Publication Number Publication Date
CA3030864A1 true CA3030864A1 (en) 2018-01-18

Family

ID=60952211

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3030864A Pending CA3030864A1 (en) 2016-07-15 2017-07-17 Compounds and methods for modulation of smn2

Country Status (27)

Country Link
US (3) US20190323006A1 (enExample)
EP (2) EP3484524B1 (enExample)
JP (3) JP7197463B2 (enExample)
KR (1) KR102556825B1 (enExample)
CN (1) CN109844115B (enExample)
AU (2) AU2017297622A1 (enExample)
BR (1) BR112019000356A2 (enExample)
CA (1) CA3030864A1 (enExample)
CL (2) CL2019000115A1 (enExample)
CO (1) CO2019001236A2 (enExample)
DK (1) DK3484524T3 (enExample)
ES (1) ES2935658T3 (enExample)
FI (1) FI3484524T3 (enExample)
HR (1) HRP20221378T1 (enExample)
HU (1) HUE060831T2 (enExample)
IL (2) IL264216B2 (enExample)
LT (1) LT3484524T (enExample)
MX (1) MX2019000619A (enExample)
NZ (1) NZ750713A (enExample)
PE (1) PE20190513A1 (enExample)
PL (1) PL3484524T3 (enExample)
PT (1) PT3484524T (enExample)
RS (1) RS63928B1 (enExample)
SG (1) SG11201900238UA (enExample)
SI (1) SI3484524T1 (enExample)
SM (1) SMT202200489T1 (enExample)
WO (1) WO2018014041A2 (enExample)

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EP3560502B1 (en) 2013-04-12 2025-09-24 The Curators of the University of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
WO2017035366A1 (en) 2015-08-26 2017-03-02 Incyte Corporation Pyrrolopyrimidine derivatives as tam inhibitors
AU2017241524B2 (en) 2016-03-28 2021-07-08 Incyte Corporation Pyrrolotriazine compounds as TAM inhibitors
TWI821200B (zh) 2017-09-27 2023-11-11 美商英塞特公司 Tam抑制劑之鹽
US11241438B2 (en) 2018-06-29 2022-02-08 Incyte Corporation Formulations of an AXL/MER inhibitor
RS64879B1 (sr) * 2019-07-19 2023-12-29 Biogen Ma Inc Postupci za lečenje ili prevenciju spinalne mišićne atrofije
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
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
EP4110919A4 (en) * 2020-02-28 2024-06-05 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
EP4114401A1 (en) 2020-03-06 2023-01-11 Incyte Corporation Combination therapy comprising axl/mer and pd-1/pd-l1 inhibitors
KR20240032184A (ko) * 2021-04-22 2024-03-11 몰레큘라 악시옴, 엘엘씨 Sos 유전자 발현을 조절하기 위한 조성물 및 방법
FR3128873B1 (fr) 2021-11-10 2026-01-16 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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