PT3717646T - Tratamento antisense da síndrome de angelman - Google Patents

Tratamento antisense da síndrome de angelman

Info

Publication number
PT3717646T
PT3717646T PT188831028T PT18883102T PT3717646T PT 3717646 T PT3717646 T PT 3717646T PT 188831028 T PT188831028 T PT 188831028T PT 18883102 T PT18883102 T PT 18883102T PT 3717646 T PT3717646 T PT 3717646T
Authority
PT
Portugal
Prior art keywords
angelman syndrome
antisense treatment
antisense
treatment
angelman
Prior art date
Application number
PT188831028T
Other languages
English (en)
Portuguese (pt)
Original Assignee
Texas A & M Univ Sys
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 Texas A & M Univ Sys filed Critical Texas A & M Univ Sys
Publication of PT3717646T publication Critical patent/PT3717646T/pt

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    • 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
    • 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
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/33—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
    • 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
    • C12N2310/34—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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35—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
    • 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
    • C12N2310/35—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • 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)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
PT188831028T 2017-12-01 2018-11-30 Tratamento antisense da síndrome de angelman PT3717646T (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US201862676034P 2018-05-24 2018-05-24

Publications (1)

Publication Number Publication Date
PT3717646T true PT3717646T (pt) 2025-07-28

Family

ID=66664231

Family Applications (1)

Application Number Title Priority Date Filing Date
PT188831028T PT3717646T (pt) 2017-12-01 2018-11-30 Tratamento antisense da síndrome de angelman

Country Status (27)

Country Link
US (5) US11198869B2 (https=)
EP (2) EP3717646B1 (https=)
JP (3) JP7297320B2 (https=)
KR (2) KR102723989B1 (https=)
CN (2) CN111433361B (https=)
AU (1) AU2018375807B2 (https=)
BR (1) BR112020009431A2 (https=)
CA (1) CA3079755A1 (https=)
CL (1) CL2020001344A1 (https=)
CO (1) CO2020006361A2 (https=)
DK (1) DK3717646T3 (https=)
ES (1) ES3032315T3 (https=)
FI (1) FI3717646T3 (https=)
HR (1) HRP20250804T1 (https=)
HU (1) HUE072645T2 (https=)
IL (1) IL274146B2 (https=)
LT (1) LT3717646T (https=)
MX (2) MX2020005680A (https=)
PE (1) PE20211238A1 (https=)
PL (1) PL3717646T3 (https=)
PT (1) PT3717646T (https=)
RS (1) RS66986B1 (https=)
RU (1) RU2020121752A (https=)
SG (1) SG11202003596WA (https=)
SI (1) SI3717646T1 (https=)
SM (1) SMT202500334T1 (https=)
WO (1) WO2019109001A1 (https=)

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WO2019168950A1 (en) * 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
PT3947684T (pt) 2019-03-29 2025-05-19 Ionis Pharmaceuticals Inc Compostos e métodos para modular ube3a‑ats
EP4172338A4 (en) * 2020-06-29 2025-06-11 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING PLP1
US20230416851A1 (en) * 2020-10-06 2023-12-28 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
CN119384504A (zh) * 2022-03-07 2025-01-28 康涅狄格大学 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA
KR20250107262A (ko) 2023-01-31 2025-07-11 후지필름 가부시키가이샤 경화성 조성물, 경화물, 광학 재료, 회절 광학 소자 및 화합물
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
KR102723989B1 (ko) 2024-11-01
JP2023123517A (ja) 2023-09-05
MX2020005680A (es) 2020-08-20
HUE072645T2 (hu) 2025-12-28
CA3079755A1 (en) 2019-06-06
US20230112629A1 (en) 2023-04-13
WO2019109001A1 (en) 2019-06-06
EP3717646B1 (en) 2025-04-23
DK3717646T3 (en) 2025-05-19
US11739328B2 (en) 2023-08-29
MX2025009778A (es) 2025-09-02
SI3717646T1 (sl) 2025-08-29
RU2020121752A (ru) 2021-12-30
JP7601441B2 (ja) 2024-12-17
ES3032315T3 (en) 2025-07-17
AU2018375807A1 (en) 2020-05-07
CN111433361A (zh) 2020-07-17
PL3717646T3 (pl) 2025-10-27
SMT202500334T1 (it) 2025-11-10
IL274146B1 (en) 2024-07-01
JP2025041632A (ja) 2025-03-26
EP3717646A1 (en) 2020-10-07
CN118291458A (zh) 2024-07-05
CO2020006361A2 (es) 2020-08-10
HRP20250804T1 (hr) 2025-09-12
EP4328306A2 (en) 2024-02-28
KR20240161203A (ko) 2024-11-12
US11198869B2 (en) 2021-12-14
US20230332152A1 (en) 2023-10-19
PE20211238A1 (es) 2021-07-09
IL274146A (en) 2020-06-30
US20220090075A1 (en) 2022-03-24
RS66986B1 (sr) 2025-07-31
EP3717646A4 (en) 2021-12-01
US20250066786A1 (en) 2025-02-27
JP7297320B2 (ja) 2023-06-26
BR112020009431A2 (pt) 2020-10-13
CL2020001344A1 (es) 2020-09-21
IL274146B2 (en) 2024-11-01
KR20200094152A (ko) 2020-08-06
LT3717646T (lt) 2025-06-10
CN111433361B (zh) 2024-03-29
JP2021505129A (ja) 2021-02-18
EP4328306A3 (en) 2025-05-21
US11685920B2 (en) 2023-06-27
US12180480B2 (en) 2024-12-31
US20200370046A1 (en) 2020-11-26
AU2018375807B2 (en) 2025-04-10
SG11202003596WA (en) 2020-05-28
FI3717646T3 (fi) 2025-07-17

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GB201517432D0 (en) Splint
GB201513826D0 (en) Pasteurisation
PL3393415T3 (pl) Bandaż
GB201512582D0 (en) Wound treatment
GB201508841D0 (en) Treatment
GB201813378D0 (en) n
GB201718985D0 (en) Treatment
GB2567679B (en) Curved devices
GB201503008D0 (en) Treatment
GB201713650D0 (en) Treatment
GB201711677D0 (en) Combination treatment
GB201706955D0 (en) Treatment
GB201704949D0 (en) Combination treatment
GB201716645D0 (en) Non-freid monoliths
GB201710601D0 (en) Redcloud BB2
AU201714927S (en) Sterilisation Wrap