CN104781402A - siRNA及其在用于治疗和/或预防眼部病症的方法和组合物中的应用 - Google Patents

siRNA及其在用于治疗和/或预防眼部病症的方法和组合物中的应用 Download PDF

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Publication number
CN104781402A
CN104781402A CN201280076706.5A CN201280076706A CN104781402A CN 104781402 A CN104781402 A CN 104781402A CN 201280076706 A CN201280076706 A CN 201280076706A CN 104781402 A CN104781402 A CN 104781402A
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sina
iop
syl040012
glaucoma
sirna
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Pending
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Chinese (zh)
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安娜·伊萨贝尔·希门尼斯安东
维多利亚·冈萨雷斯法哈多
韦罗妮卡·鲁斯帕洛马尔
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Sylentis SA
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Sylentis SA
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Epidemiology (AREA)
  • Medical Preparation Storing Or Oral Administration Devices (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201280076706.5A 2012-09-05 2012-09-05 siRNA及其在用于治疗和/或预防眼部病症的方法和组合物中的应用 Pending CN104781402A (zh)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/GB2012/052177 WO2014037686A1 (en) 2012-09-05 2012-09-05 Sirna and their use in methods and compositions for the treatment and/or prevention of eye conditions

Publications (1)

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CN104781402A true CN104781402A (zh) 2015-07-15

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CN201280076706.5A Pending CN104781402A (zh) 2012-09-05 2012-09-05 siRNA及其在用于治疗和/或预防眼部病症的方法和组合物中的应用

Country Status (18)

Country Link
US (1) US10011832B2 (enExample)
EP (1) EP2893018B1 (enExample)
JP (1) JP2015533792A (enExample)
KR (1) KR102120060B1 (enExample)
CN (1) CN104781402A (enExample)
AU (1) AU2012389270B2 (enExample)
BR (1) BR112015004452A2 (enExample)
CA (1) CA2883007A1 (enExample)
CL (1) CL2015000538A1 (enExample)
EC (1) ECSP15011105A (enExample)
ES (1) ES2750125T3 (enExample)
IL (1) IL237403B (enExample)
IN (1) IN2015DN02699A (enExample)
MX (1) MX368084B (enExample)
RU (1) RU2653766C2 (enExample)
SG (1) SG11201501385UA (enExample)
WO (1) WO2014037686A1 (enExample)
ZA (1) ZA201502181B (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2750125T3 (es) 2012-09-05 2020-03-25 Sylentis Sau ARNip y su uso en métodos y composiciones para el tratamiento y/o la prevención de afecciones oculares
US10011837B2 (en) 2014-03-04 2018-07-03 Sylentis Sau SiRNAs and their use in methods and compositions for the treatment and/or prevention of eye conditions
WO2024230837A1 (zh) * 2023-05-11 2024-11-14 广州瑞风生物科技有限公司 指导rna、基因编辑系统及其应用

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Also Published As

Publication number Publication date
JP2015533792A (ja) 2015-11-26
MX368084B (es) 2019-09-19
RU2015112131A (ru) 2016-10-27
MX2015002800A (es) 2015-07-17
EP2893018A1 (en) 2015-07-15
HK1211983A1 (en) 2016-06-03
SG11201501385UA (en) 2015-03-30
US20150259677A1 (en) 2015-09-17
WO2014037686A1 (en) 2014-03-13
BR112015004452A2 (pt) 2017-08-08
US10011832B2 (en) 2018-07-03
EP2893018B1 (en) 2019-07-24
ES2750125T3 (es) 2020-03-25
IL237403A0 (en) 2015-04-30
CL2015000538A1 (es) 2015-07-31
CA2883007A1 (en) 2014-03-13
RU2653766C2 (ru) 2018-05-14
AU2012389270B2 (en) 2018-11-08
IL237403B (en) 2019-11-28
ECSP15011105A (es) 2015-12-31
KR20150048880A (ko) 2015-05-07
AU2012389270A1 (en) 2015-02-26
KR102120060B1 (ko) 2020-06-09
ZA201502181B (en) 2016-01-27
IN2015DN02699A (enExample) 2015-09-04

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Application publication date: 20150715