MX2018013003A - Oligonucleotidos para tratar una enfermedad ocular. - Google Patents

Oligonucleotidos para tratar una enfermedad ocular.

Info

Publication number
MX2018013003A
MX2018013003A MX2018013003A MX2018013003A MX2018013003A MX 2018013003 A MX2018013003 A MX 2018013003A MX 2018013003 A MX2018013003 A MX 2018013003A MX 2018013003 A MX2018013003 A MX 2018013003A MX 2018013003 A MX2018013003 A MX 2018013003A
Authority
MX
Mexico
Prior art keywords
oligonucleotides
eye disease
treat eye
relates
exon
Prior art date
Application number
MX2018013003A
Other languages
English (en)
Inventor
Lam Chan Hee
Catharina VAN DIEPEN Hester
Juha Turunen Janne
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 MX2018013003A publication Critical patent/MX2018013003A/es

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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • 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
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    • C12N15/09Recombinant DNA-technology
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3521Methyl
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Organic Chemistry (AREA)
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  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (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

La invención se refiere a los campos de la medicina y la inmunología. En particular, se refiere a oligonucleótidos antisentido novedosos que se pueden usar en el tratamiento, prevención o retraso del síndrome de Usher de tipo II y/o la degeneración de la retina no sindrómica asociada con USH2A, especialmente mediante el salto de un pseudoexón (PE40) entre los exones 40 y 41 del gen USH2A humano.
MX2018013003A 2016-04-25 2017-04-25 Oligonucleotidos para tratar una enfermedad ocular. MX2018013003A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201607141 2016-04-25
PCT/EP2017/059830 WO2017186739A1 (en) 2016-04-25 2017-04-25 Oligonucleotides to treat eye disease

Publications (1)

Publication Number Publication Date
MX2018013003A true MX2018013003A (es) 2019-01-28

Family

ID=58672573

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018013003A MX2018013003A (es) 2016-04-25 2017-04-25 Oligonucleotidos para tratar una enfermedad ocular.

Country Status (15)

Country Link
US (3) US10617707B2 (es)
EP (1) EP3448999B1 (es)
JP (1) JP7043082B2 (es)
KR (1) KR102368920B1 (es)
CN (1) CN109072239A (es)
AU (1) AU2017257292A1 (es)
CA (1) CA3021899A1 (es)
DK (1) DK3448999T3 (es)
EA (1) EA201892431A1 (es)
ES (1) ES2801823T3 (es)
IL (1) IL262199B (es)
MX (1) MX2018013003A (es)
PL (1) PL3448999T3 (es)
WO (1) WO2017186739A1 (es)
ZA (1) ZA201806628B (es)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021899A1 (en) 2016-04-25 2017-11-02 Proqr Therapeutics Ii B.V. Oligonucleotides to treat eye disease
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
CA3035293A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified 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
EP3728595A1 (en) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
EP3728594A1 (en) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2020157008A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
US20220145305A1 (en) * 2019-02-12 2022-05-12 Universita' Degli Studi Di Trento CAS12a GUIDE RNA MOLECULES AND USES THEREOF
CA3132178A1 (en) 2019-04-02 2020-10-08 Aliye Seda Yilmaz-Elis Antisense oligonucleotides for immunotherapy
PH12021552613A1 (en) 2019-04-15 2022-06-27 Edigene Therapeutics Beijing 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
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
CN114269919B (zh) 2019-07-12 2023-08-18 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑
WO2021018750A1 (en) 2019-07-26 2021-02-04 Proqr Therapeutics Ii B.V. Ophthalmic compositions comprising viscosifying polymers and nucleic acids
EP4010475A1 (en) * 2019-08-08 2022-06-15 UCL Business Ltd Antisense oligonucleotides rescue aberrant splicing of abca4
JP7765837B2 (ja) 2019-12-23 2025-11-07 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 シュタルガルト病の処置におけるヌクレオチド脱アミノ化のためのアンチセンスオリゴヌクレオチド
EP4086346A4 (en) * 2019-12-30 2024-03-20 EdiGene Therapeutics (Beijing) Inc. METHOD FOR TREATMENT OF USHER SYNDROME AND ASSOCIATED COMPOSITION
CN113122577A (zh) * 2019-12-30 2021-07-16 博雅辑因(北京)生物科技有限公司 一种治疗Usher综合征的方法和其组合物
US20230134677A1 (en) 2020-03-04 2023-05-04 Proqr Therapeutics Ii B.V. 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
TR2022001648A2 (tr) * 2022-02-09 2022-02-21 T C Ueskuedar Ueniversitesi Ush2a kaynaklı retinitis pigmentosa hastalığının genetik tedavisi için ekzon 13 atlama işlevi gören kseno nükleik asit antisens-oligonükleotit (xna-aso) dizileri.
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4599060A1 (en) * 2022-10-06 2025-08-13 Stichting Radboud universitair medisch centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
WO2024074670A1 (en) * 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
EP4603585A1 (en) * 2022-10-11 2025-08-20 Guangzhou Reforgene Medicine Co., Ltd. Snrna nucleic acid molecule and application thereof
WO2024105063A1 (en) * 2022-11-15 2024-05-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 39-40
WO2025007937A1 (zh) * 2023-07-04 2025-01-09 广州瑞风生物科技有限公司 靶向USH2A pre-mRNA假外显子PE40的snRNA及其应用

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US8093000B2 (en) 2008-05-09 2012-01-10 The Regents Of The University Of California Methods for predicting and treating tumors resistant to drug, immunotherapy, and radiation
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DK2425814T3 (da) 2010-09-03 2013-09-08 Novagali Pharma Sa Vand-i-olieemulsion til behandling af en øjensygdom
US10131910B2 (en) * 2014-07-10 2018-11-20 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Usher syndrome type 2
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CA3021899A1 (en) 2016-04-25 2017-11-02 Proqr Therapeutics Ii B.V. Oligonucleotides to treat eye disease

Also Published As

Publication number Publication date
DK3448999T3 (da) 2020-06-08
US20190381089A1 (en) 2019-12-19
WO2017186739A1 (en) 2017-11-02
US20200237802A1 (en) 2020-07-30
US10617707B2 (en) 2020-04-14
KR102368920B1 (ko) 2022-02-28
PL3448999T3 (pl) 2020-11-30
AU2017257292A1 (en) 2018-12-06
US20210401871A1 (en) 2021-12-30
IL262199B (en) 2022-03-01
ZA201806628B (en) 2019-07-31
CA3021899A1 (en) 2017-11-02
EP3448999B1 (en) 2020-05-13
EA201892431A1 (ru) 2019-04-30
JP7043082B2 (ja) 2022-03-29
KR20180134931A (ko) 2018-12-19
EP3448999A1 (en) 2019-03-06
US11123360B2 (en) 2021-09-21
ES2801823T3 (es) 2021-01-14
JP2019515688A (ja) 2019-06-13
IL262199A (en) 2018-12-31
CN109072239A (zh) 2018-12-21

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