HK1209456A1 - 用於治療肌肉萎縮症患者的寡核苷酸 - Google Patents

用於治療肌肉萎縮症患者的寡核苷酸

Info

Publication number
HK1209456A1
HK1209456A1 HK15110181.1A HK15110181A HK1209456A1 HK 1209456 A1 HK1209456 A1 HK 1209456A1 HK 15110181 A HK15110181 A HK 15110181A HK 1209456 A1 HK1209456 A1 HK 1209456A1
Authority
HK
Hong Kong
Prior art keywords
oligonucleotide
treatment
muscular dystrophy
dystrophy patients
patients
Prior art date
Application number
HK15110181.1A
Other languages
English (en)
Inventor
Judith Christina Theodora Van Deutekom
Original Assignee
Prosensa Technologies Bv
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 Prosensa Technologies Bv filed Critical Prosensa Technologies Bv
Publication of HK1209456A1 publication Critical patent/HK1209456A1/zh

Links

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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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • 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
    • 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
    • C12N2310/351—Conjugate
    • 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
HK15110181.1A 2012-07-03 2015-10-16 用於治療肌肉萎縮症患者的寡核苷酸 HK1209456A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261667517P 2012-07-03 2012-07-03
EP12174781 2012-07-03
PCT/NL2013/050487 WO2014007620A2 (en) 2012-07-03 2013-07-03 Oligonucleotide for the treatment of muscular dystrophy patients

Publications (1)

Publication Number Publication Date
HK1209456A1 true HK1209456A1 (zh) 2016-04-01

Family

ID=49882554

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15110181.1A HK1209456A1 (zh) 2012-07-03 2015-10-16 用於治療肌肉萎縮症患者的寡核苷酸

Country Status (11)

Country Link
US (1) US20150191725A1 (zh)
EP (2) EP2870246B1 (zh)
JP (1) JP6460983B2 (zh)
CN (2) CN104603271B (zh)
AU (1) AU2013285698A1 (zh)
BR (1) BR112014033004B1 (zh)
CA (1) CA2877644A1 (zh)
HK (1) HK1209456A1 (zh)
IL (1) IL236504B (zh)
RU (1) RU2674600C2 (zh)
WO (1) WO2014007620A2 (zh)

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Also Published As

Publication number Publication date
WO2014007620A2 (en) 2014-01-09
BR112014033004A2 (pt) 2017-08-01
IL236504A0 (en) 2015-02-26
US20150191725A1 (en) 2015-07-09
RU2674600C2 (ru) 2018-12-11
AU2013285698A1 (en) 2015-02-19
JP2015522275A (ja) 2015-08-06
RU2015103227A (ru) 2016-08-20
IL236504B (en) 2019-02-28
EP2870246B1 (en) 2019-09-11
EP3608407A1 (en) 2020-02-12
CN110257379A (zh) 2019-09-20
CA2877644A1 (en) 2014-01-09
WO2014007620A3 (en) 2014-02-27
CN104603271A (zh) 2015-05-06
CN110257379B (zh) 2023-08-11
CN104603271B (zh) 2019-07-19
JP6460983B2 (ja) 2019-01-30
RU2018141234A (ru) 2018-12-27
EP2870246A2 (en) 2015-05-13
BR112014033004B1 (pt) 2021-10-19

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