EA201990644A1 - Антисмысловые олигонуклеотиды для лечения глазного заболевания - Google Patents

Антисмысловые олигонуклеотиды для лечения глазного заболевания

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Publication number
EA201990644A1
EA201990644A1 EA201990644A EA201990644A EA201990644A1 EA 201990644 A1 EA201990644 A1 EA 201990644A1 EA 201990644 A EA201990644 A EA 201990644A EA 201990644 A EA201990644 A EA 201990644A EA 201990644 A1 EA201990644 A1 EA 201990644A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
eye disease
sense oligonucleotides
relates
extraindromic
Prior art date
Application number
EA201990644A
Other languages
English (en)
Inventor
Хестер Катарина Ван Дипен
Джанне Джуха Турунен
Хээ Лам Чань
Original Assignee
Прокр Терапеутикс Ii Б.В.
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 Прокр Терапеутикс Ii Б.В. filed Critical Прокр Терапеутикс Ii Б.В.
Publication of EA201990644A1 publication Critical patent/EA201990644A1/ru

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Настоящее изобретение относится к областям медицины и иммунологии. В частности, оно относится к новым антисмысловым олигонуклеотидам (AON), которые могут быть использованы в лечении, предупреждении и/или задержке развития синдрома Ушера II типа и/или связанной с USH2A внесиндромной дегенерации сетчатки.
EA201990644A 2016-09-23 2017-09-22 Антисмысловые олигонуклеотиды для лечения глазного заболевания EA201990644A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1616202.6A GB201616202D0 (en) 2016-09-23 2016-09-23 Antisense oligonucleotides for the treatment of eye deisease
PCT/EP2017/074133 WO2018055134A1 (en) 2016-09-23 2017-09-22 Antisense oligonucleotides for the treatment of eye disease

Publications (1)

Publication Number Publication Date
EA201990644A1 true EA201990644A1 (ru) 2019-08-30

Family

ID=57539952

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201990644A EA201990644A1 (ru) 2016-09-23 2017-09-22 Антисмысловые олигонуклеотиды для лечения глазного заболевания

Country Status (19)

Country Link
US (2) US10612025B2 (ru)
EP (2) EP3985117A1 (ru)
JP (1) JP7141123B2 (ru)
KR (1) KR102450757B1 (ru)
CN (1) CN109804069A (ru)
AU (1) AU2017330062B2 (ru)
BR (1) BR112019005586A2 (ru)
CA (1) CA3035627A1 (ru)
DK (1) DK3516060T3 (ru)
EA (1) EA201990644A1 (ru)
ES (1) ES2886118T3 (ru)
GB (1) GB201616202D0 (ru)
IL (1) IL265206B2 (ru)
MX (1) MX2019003361A (ru)
NZ (1) NZ752572A (ru)
PL (1) PL3516060T3 (ru)
PT (1) PT3516060T (ru)
WO (1) WO2018055134A1 (ru)
ZA (1) ZA201901247B (ru)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
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AU2017281497B2 (en) 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
JP2022518808A (ja) * 2019-01-28 2022-03-16 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ レーバー先天黒内障の処置のためのアンチセンスオリゴヌクレオチド
EP3918067A1 (en) 2019-01-28 2021-12-08 ProQR Therapeutics II B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
WO2020201144A1 (en) 2019-04-02 2020-10-08 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
SG11202111401RA (en) 2019-04-15 2021-11-29 Edigene Inc Methods and compositions for editing rnas
WO2020212567A1 (en) 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
EP3956452A4 (en) * 2019-04-19 2023-03-15 University of Massachusetts GENE THERAPIES FOR USHER SYNDROME (USH2A)
SG11202111386UA (en) * 2019-04-25 2021-11-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
WO2021008447A1 (en) 2019-07-12 2021-01-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas
US20220265695A1 (en) 2019-07-26 2022-08-25 Proqr Therapeutics Ii B.V. Opthalmic compositions comprising viscosifying polymers and nucleic acids
JP2023504314A (ja) 2019-12-02 2023-02-02 シェイプ セラピューティクス インコーポレイテッド 治療的編集
US20230039928A1 (en) 2019-12-23 2023-02-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease
TW202138561A (zh) * 2019-12-30 2021-10-16 大陸商博雅輯因（北京）生物科技有限公司 治療Usher綜合症的方法和其組合物
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS Antisense oligonucleotides for use in the treatment of usher syndrome
WO2022184888A2 (en) 2021-03-05 2022-09-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
WO2023134560A1 (zh) * 2022-01-11 2023-07-20 广州瑞风生物科技有限公司 一种核苷酸及其应用
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2023183825A2 (en) * 2022-03-21 2023-09-28 Ptc Therapeutics Gt, Inc. Gene therapy of ush2a-associated diseases
WO2024078345A1 (zh) * 2022-10-11 2024-04-18 广州瑞风生物科技有限公司 snRNA核酸分子及其应用

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US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
PT2425814E (pt) 2010-09-03 2013-09-02 Novagali Pharma Sa Emulsão de tipo água em óleo para tratamento de uma doença ocular
WO2012151324A1 (en) 2011-05-02 2012-11-08 Isis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
AU2012305053B2 (en) * 2011-09-05 2017-12-21 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for the treatment of Leber congenital amaurosis
CN103710340B (zh) * 2013-10-12 2016-01-27 中国人民解放军总医院 一种i型usher综合征相关基因突变及应用此突变基因的耳聋分子病因学诊断试剂
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GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease

Also Published As

Publication number Publication date
IL265206B2 (en) 2023-08-01
CA3035627A1 (en) 2018-03-29
IL265206B1 (en) 2023-04-01
KR102450757B1 (ko) 2022-10-04
US20200181616A1 (en) 2020-06-11
PT3516060T (pt) 2021-08-27
EP3516060B1 (en) 2021-07-21
US11479771B2 (en) 2022-10-25
BR112019005586A2 (pt) 2019-06-11
CN109804069A (zh) 2019-05-24
EP3516060A1 (en) 2019-07-31
NZ752572A (en) 2023-01-27
ES2886118T3 (es) 2021-12-16
KR20190051020A (ko) 2019-05-14
IL265206A (en) 2019-05-30
WO2018055134A1 (en) 2018-03-29
PL3516060T3 (pl) 2021-12-13
ZA201901247B (en) 2020-10-28
JP2019528747A (ja) 2019-10-17
MX2019003361A (es) 2019-07-18
AU2017330062B2 (en) 2024-04-04
JP7141123B2 (ja) 2022-09-22
GB201616202D0 (en) 2016-11-09
AU2017330062A1 (en) 2019-05-02
US20190256847A1 (en) 2019-08-22
US10612025B2 (en) 2020-04-07
EP3985117A1 (en) 2022-04-20
DK3516060T3 (da) 2021-09-27

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