EA201490563A1 - Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера - Google Patents

Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера

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Publication number
EA201490563A1
EA201490563A1 EA201490563A EA201490563A EA201490563A1 EA 201490563 A1 EA201490563 A1 EA 201490563A1 EA 201490563 A EA201490563 A EA 201490563A EA 201490563 A EA201490563 A EA 201490563A EA 201490563 A1 EA201490563 A1 EA 201490563A1
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EA
Eurasian Patent Office
Prior art keywords
amaurosis
congenital
leaver
treatment
small oligonucleotide
Prior art date
Application number
EA201490563A
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English (en)
Other versions
EA033653B1 (ru
Inventor
Роберт Вильхельмус Йоханна Коллин
Францискус Петер Мария Кремерс
Антония Ингрид Ден Холландер
Original Assignee
Стихтинг Катхолике Юниверситейт
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Publication date
Application filed by Стихтинг Катхолике Юниверситейт filed Critical Стихтинг Катхолике Юниверситейт
Publication of EA201490563A1 publication Critical patent/EA201490563A1/ru
Publication of EA033653B1 publication Critical patent/EA033653B1/ru

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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
    • 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
    • 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
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • 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
    • C12N2310/34—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/34—Allele or polymorphism specific uses

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

Abstract

Изобретение относится к областям медицины и иммунологии. В частности, оно относится к новым антисмысловым олигонуклеотидам, которые можно использовать для лечения, профилактики и/или замедления врожденного амавроза Лебера.
EA201490563A 2011-09-05 2012-04-25 Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера EA033653B1 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
NL2007351 2011-09-05
US201161531137P 2011-09-06 2011-09-06
PCT/NL2012/050275 WO2013036105A1 (en) 2011-09-05 2012-04-25 Antisense oligonucleotides for the treatment of leber congenital amaurosis

Publications (2)

Publication Number Publication Date
EA201490563A1 true EA201490563A1 (ru) 2014-08-29
EA033653B1 EA033653B1 (ru) 2019-11-13

Family

ID=47832421

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201490563A EA033653B1 (ru) 2011-09-05 2012-04-25 Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера

Country Status (11)

Country Link
US (6) US9771580B2 (ru)
EP (1) EP2753694B1 (ru)
AU (1) AU2012305053B2 (ru)
BR (1) BR112014004895B1 (ru)
CA (1) CA2847664C (ru)
DK (1) DK2753694T3 (ru)
EA (1) EA033653B1 (ru)
ES (1) ES2646716T3 (ru)
HK (1) HK1198658A1 (ru)
PL (1) PL2753694T3 (ru)
WO (1) WO2013036105A1 (ru)

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US9012425B2 (en) 2011-06-10 2015-04-21 Inserm (Institute National De La Sante Et De La Recherche Medicale) Methods for the treatment of Leber congenital amaurosis
PL2753694T3 (pl) * 2011-09-05 2018-01-31 Stichting Katholieke Univ Oligonukleotydy antysensowne do leczenia wrodzonej ślepoty Lebera
US9637744B2 (en) * 2013-06-13 2017-05-02 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and pharmaceutical compositions for the treatment of erythropoietic protoporphyria
US11667913B2 (en) 2013-07-08 2023-06-06 Inserm Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof
WO2015009575A1 (en) * 2013-07-16 2015-01-22 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to cep290
WO2016034680A1 (en) * 2014-09-05 2016-03-10 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
GB201503408D0 (en) * 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
IL308174A (en) 2015-10-09 2024-01-01 Univ Southampton Gene expression modulation and dysregulated protein expression scanning
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
US20180010117A1 (en) * 2016-07-07 2018-01-11 Sangamo Therapeutics, Inc. Targeted treatment of leber congenital amourosis
GB201616202D0 (en) * 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
GB2599884B (en) 2017-08-25 2022-08-31 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
MX2020010959A (es) * 2018-04-17 2021-01-15 Univ Pennsylvania Moleculas de empalme trans.
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
EP3840758A1 (en) 2018-08-21 2021-06-30 Deep Genomics Incorporated Therapeutic splice-switching oligonucleotides
CA3126371A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of leber's congenital amaurosis
CA3124493A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
WO2020212567A1 (en) 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
WO2021130313A1 (en) 2019-12-23 2021-07-01 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS Antisense oligonucleotides for use in the treatment of usher syndrome
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
WO2022090256A1 (en) 2020-10-26 2022-05-05 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of stargardt disease
WO2022184888A2 (en) 2021-03-05 2022-09-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia

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US7517644B1 (en) * 1991-08-23 2009-04-14 Larry J. Smith Method and compositions for cellular reprogramming
AU7855900A (en) 1999-10-04 2001-05-10 University Of Medicine And Dentistry Of New Jersey Methods for identifying rna binding compounds
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
WO2003037909A1 (en) * 2001-10-29 2003-05-08 Mcgill University Acyclic linker-containing oligonucleotides and uses thereof
US20050118625A1 (en) * 2003-10-02 2005-06-02 Mounts William M. Nucleic acid arrays for detecting gene expression associated with human osteoarthritis and human proteases
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US20090011040A1 (en) * 2007-05-02 2009-01-08 Naash Muna I Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
EP2262899A1 (en) * 2008-04-02 2010-12-22 Fondazione Telethon Method of treating genetic disorders
US20120108654A1 (en) * 2008-06-30 2012-05-03 The Johns Hopkins University Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa
US9012425B2 (en) * 2011-06-10 2015-04-21 Inserm (Institute National De La Sante Et De La Recherche Medicale) Methods for the treatment of Leber congenital amaurosis
PL2753694T3 (pl) * 2011-09-05 2018-01-31 Stichting Katholieke Univ Oligonukleotydy antysensowne do leczenia wrodzonej ślepoty Lebera

Also Published As

Publication number Publication date
PL2753694T3 (pl) 2018-01-31
US20200255832A1 (en) 2020-08-13
US20220235355A1 (en) 2022-07-28
EP2753694A1 (en) 2014-07-16
US9771580B2 (en) 2017-09-26
ES2646716T3 (es) 2017-12-15
AU2012305053B2 (en) 2017-12-21
US20180305692A1 (en) 2018-10-25
EP2753694B1 (en) 2017-08-23
US20190078091A1 (en) 2019-03-14
CA2847664A1 (en) 2013-03-14
US10647985B2 (en) 2020-05-12
HK1198658A1 (en) 2015-05-22
EA033653B1 (ru) 2019-11-13
US10167470B2 (en) 2019-01-01
BR112014004895B1 (pt) 2022-07-05
US20170342414A1 (en) 2017-11-30
BR112014004895A2 (pt) 2017-07-04
WO2013036105A1 (en) 2013-03-14
US11279933B2 (en) 2022-03-22
CA2847664C (en) 2021-08-31
US20140336238A1 (en) 2014-11-13
AU2012305053A1 (en) 2014-03-20
DK2753694T3 (en) 2017-10-16

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