DK3717646T3 - Angelman syndrome antisense treatment - Google Patents

Angelman syndrome antisense treatment Download PDF

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Publication number
DK3717646T3
DK3717646T3 DK18883102.8T DK18883102T DK3717646T3 DK 3717646 T3 DK3717646 T3 DK 3717646T3 DK 18883102 T DK18883102 T DK 18883102T DK 3717646 T3 DK3717646 T3 DK 3717646T3
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angelman syndrome
antisense treatment
antisense
treatment
angelman
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DK18883102.8T
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Danish (da)
English (en)
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Scott Victor Dindot
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Texas A & M Univ Sys
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • 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
    • 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
    • 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
    • C07H21/04—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 deoxyribosyl as saccharide radical
    • 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
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
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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
    • 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
    • C12N2310/323—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
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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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/35—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
    • 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

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  • Health & Medical Sciences (AREA)
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  • Life Sciences & Earth Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
DK18883102.8T 2017-12-01 2018-11-30 Angelman syndrome antisense treatment DK3717646T3 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US201862676034P 2018-05-24 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

Publications (1)

Publication Number Publication Date
DK3717646T3 true DK3717646T3 (en) 2025-05-19

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DK18883102.8T DK3717646T3 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

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US (5) US11198869B2 (enExample)
EP (2) EP3717646B1 (enExample)
JP (3) JP7297320B2 (enExample)
KR (2) KR102723989B1 (enExample)
CN (2) CN111433361B (enExample)
AU (1) AU2018375807B2 (enExample)
BR (1) BR112020009431A2 (enExample)
CA (1) CA3079755A1 (enExample)
CL (1) CL2020001344A1 (enExample)
CO (1) CO2020006361A2 (enExample)
DK (1) DK3717646T3 (enExample)
ES (1) ES3032315T3 (enExample)
FI (1) FI3717646T3 (enExample)
HR (1) HRP20250804T1 (enExample)
IL (1) IL274146B2 (enExample)
LT (1) LT3717646T (enExample)
MX (2) MX2020005680A (enExample)
PE (1) PE20211238A1 (enExample)
PL (1) PL3717646T3 (enExample)
PT (1) PT3717646T (enExample)
RS (1) RS66986B1 (enExample)
RU (1) RU2020121752A (enExample)
SG (1) SG11202003596WA (enExample)
SI (1) SI3717646T1 (enExample)
SM (1) SMT202500334T1 (enExample)
WO (1) WO2019109001A1 (enExample)

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PL3374509T3 (pl) 2015-11-12 2021-05-04 F. Hoffmann-La Roche Ag Oligonukleotydy do indukowania ojcowskiej ekspresji UBE3A
WO2019168950A1 (en) * 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
TWI874376B (zh) 2019-03-29 2025-03-01 美商Ionis製藥公司 用於調節ube3a-ats 之化合物及方法
TW202216996A (zh) * 2020-06-29 2022-05-01 美商Ionis製藥公司 調節ｐｌｐ１之化合物及方法
CA3197664A1 (en) * 2020-10-06 2022-04-14 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
EP4473116A2 (en) * 2022-03-07 2024-12-11 University of Connecticut Shrna targeting snord115 locations to restore paternal ube3a gene expression in angelman syndrome
CN120476156A (zh) 2023-01-31 2025-08-12 富士胶片株式会社 固化性组合物、固化物、光学材料、衍射光学元件及化合物
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025217466A1 (en) * 2024-04-11 2025-10-16 Ultragenyx Pharmaceutical Inc. Dosing regimens for antisense treatment of angelman syndrome

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Publication number Publication date
US20250066786A1 (en) 2025-02-27
WO2019109001A1 (en) 2019-06-06
EP4328306A2 (en) 2024-02-28
CN118291458A (zh) 2024-07-05
EP3717646A4 (en) 2021-12-01
FI3717646T3 (fi) 2025-07-17
US11685920B2 (en) 2023-06-27
JP2021505129A (ja) 2021-02-18
US12180480B2 (en) 2024-12-31
PL3717646T3 (pl) 2025-10-27
CO2020006361A2 (es) 2020-08-10
PT3717646T (pt) 2025-07-28
IL274146A (en) 2020-06-30
US20230332152A1 (en) 2023-10-19
JP2023123517A (ja) 2023-09-05
CN111433361B (zh) 2024-03-29
EP4328306A3 (en) 2025-05-21
AU2018375807B2 (en) 2025-04-10
IL274146B2 (en) 2024-11-01
CL2020001344A1 (es) 2020-09-21
JP2025041632A (ja) 2025-03-26
US20230112629A1 (en) 2023-04-13
KR102723989B1 (ko) 2024-11-01
KR20200094152A (ko) 2020-08-06
US11739328B2 (en) 2023-08-29
US11198869B2 (en) 2021-12-14
KR20240161203A (ko) 2024-11-12
CA3079755A1 (en) 2019-06-06
HRP20250804T1 (hr) 2025-09-12
IL274146B1 (en) 2024-07-01
PE20211238A1 (es) 2021-07-09
SG11202003596WA (en) 2020-05-28
CN111433361A (zh) 2020-07-17
EP3717646A1 (en) 2020-10-07
US20220090075A1 (en) 2022-03-24
JP7601441B2 (ja) 2024-12-17
RS66986B1 (sr) 2025-07-31
SI3717646T1 (sl) 2025-08-29
MX2025009778A (es) 2025-09-02
SMT202500334T1 (it) 2025-11-10
LT3717646T (lt) 2025-06-10
BR112020009431A2 (pt) 2020-10-13
RU2020121752A (ru) 2021-12-30
JP7297320B2 (ja) 2023-06-26
AU2018375807A1 (en) 2020-05-07
US20200370046A1 (en) 2020-11-26
EP3717646B1 (en) 2025-04-23
MX2020005680A (es) 2020-08-20
ES3032315T3 (en) 2025-07-17

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