MX2017011013A - Terapia de oligonucleotidos para amaurosis congenita de leber. - Google Patents

Terapia de oligonucleotidos para amaurosis congenita de leber.

Info

Publication number
MX2017011013A
MX2017011013A MX2017011013A MX2017011013A MX2017011013A MX 2017011013 A MX2017011013 A MX 2017011013A MX 2017011013 A MX2017011013 A MX 2017011013A MX 2017011013 A MX2017011013 A MX 2017011013A MX 2017011013 A MX2017011013 A MX 2017011013A
Authority
MX
Mexico
Prior art keywords
congenital amaurosis
leber congenital
oligonucleotide therapy
consecutive
cep290
Prior art date
Application number
MX2017011013A
Other languages
English (en)
Inventor
Coromoto Biasutto Patricia
Lam Chan Hee
Original Assignee
Proqr Therapeutics Ii 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 Proqr Therapeutics Ii Bv filed Critical Proqr Therapeutics Ii Bv
Publication of MX2017011013A publication Critical patent/MX2017011013A/es

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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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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

Abstract

Oligonucleótidos antisentído enfocan la mutación en intrón 26 del gene CEP290 y reducen la inclusión del exón aberrante en el mRNA de CEP290. Los oligonucleótidos incluyen no más de 3 guanosinas consecutivas, no tienen más de 60% de nucleobases de guanosina, incluyen cuando mucho una secuencia de CpG y/o no tienen el potencial de formar una horquilla que comprende 3 o más pares de bases complementarios.
MX2017011013A 2015-02-27 2016-02-26 Terapia de oligonucleotidos para amaurosis congenita de leber. MX2017011013A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201503408A GB201503408D0 (en) 2015-02-27 2015-02-27 Oligonucleotides
PCT/EP2016/054164 WO2016135334A1 (en) 2015-02-27 2016-02-26 Oligonucleotide therapy for leber congenital amaurosis

Publications (1)

Publication Number Publication Date
MX2017011013A true MX2017011013A (es) 2018-03-06

Family

ID=52876291

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2017011013A MX2017011013A (es) 2015-02-27 2016-02-26 Terapia de oligonucleotidos para amaurosis congenita de leber.

Country Status (14)

Country Link
US (3) US10421963B2 (es)
EP (1) EP3262170A1 (es)
JP (1) JP6813506B2 (es)
KR (1) KR102368918B1 (es)
CN (1) CN107406852B (es)
AU (1) AU2016223336B2 (es)
CA (1) CA2976601C (es)
EA (1) EA036791B1 (es)
GB (1) GB201503408D0 (es)
IL (1) IL253882B (es)
MX (1) MX2017011013A (es)
NZ (1) NZ735342A (es)
WO (1) WO2016135334A1 (es)
ZA (1) ZA201705331B (es)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
AU2017281497B2 (en) 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
CA3107890A1 (en) 2018-08-21 2020-02-27 Deep Genomics Incorporated Therapeutic splice-switching oligonucleotides
EP3918067A1 (en) 2019-01-28 2021-12-08 ProQR Therapeutics II B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
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
WO2020201144A1 (en) 2019-04-02 2020-10-08 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
WO2020212567A1 (en) 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
EP3959315A1 (en) * 2019-04-26 2022-03-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of retinal dystrophies by exon-skipping strategy
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
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
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS Antisense oligonucleotides for use in the treatment of usher syndrome
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
CN113736812A (zh) * 2021-09-06 2021-12-03 武汉翼康基因科技有限公司 一种pcMINI载体及其构建方法与应用
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia

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EP2718437B1 (en) * 2011-06-10 2018-05-09 Institut National de la Sante et de la Recherche Medicale (INSERM) Methods for the treatment of leber congenital amaurosis
AU2012305053B2 (en) * 2011-09-05 2017-12-21 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for the treatment of Leber congenital amaurosis
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GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides

Also Published As

Publication number Publication date
AU2016223336B2 (en) 2021-12-09
US20210139905A1 (en) 2021-05-13
AU2016223336A1 (en) 2017-09-28
CN107406852A (zh) 2017-11-28
KR20170118755A (ko) 2017-10-25
CA2976601A1 (en) 2016-09-01
KR102368918B1 (ko) 2022-02-28
US10421963B2 (en) 2019-09-24
NZ735342A (en) 2022-07-01
IL253882A0 (en) 2017-10-31
IL253882B (en) 2022-03-01
WO2016135334A1 (en) 2016-09-01
CA2976601C (en) 2023-03-21
EA201791820A1 (ru) 2017-12-29
US10889817B2 (en) 2021-01-12
CN107406852B (zh) 2021-06-11
US20200063134A1 (en) 2020-02-27
EP3262170A1 (en) 2018-01-03
BR112017018335A2 (pt) 2018-04-17
US11920132B2 (en) 2024-03-05
GB201503408D0 (en) 2015-04-15
ZA201705331B (en) 2019-07-31
US20180030439A1 (en) 2018-02-01
JP2018507711A (ja) 2018-03-22
JP6813506B2 (ja) 2021-01-13
EA036791B1 (ru) 2020-12-22

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