MX2019003361A - Oligonucleotidos antisentido para el tratamiento de enfermedad ocular. - Google Patents

Oligonucleotidos antisentido para el tratamiento de enfermedad ocular.

Info

Publication number
MX2019003361A
MX2019003361A MX2019003361A MX2019003361A MX2019003361A MX 2019003361 A MX2019003361 A MX 2019003361A MX 2019003361 A MX2019003361 A MX 2019003361A MX 2019003361 A MX2019003361 A MX 2019003361A MX 2019003361 A MX2019003361 A MX 2019003361A
Authority
MX
Mexico
Prior art keywords
treatment
antisense oligonucleotides
eye disease
relates
ush2a
Prior art date
Application number
MX2019003361A
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 MX2019003361A publication Critical patent/MX2019003361A/es

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Classifications

    • 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (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

La invención se refiere al campo de la medicina e inmunología. En particular, se refiere a nuevos oligonucleótidos antisentido (AON) que pueden ser usados en el tratamiento, la prevención y/o el retraso del síndrome de Usher tipo II y/o degeneración retiniana no sindrómica asociada a USH2A.
MX2019003361A 2016-09-23 2017-09-22 Oligonucleotidos antisentido para el tratamiento de enfermedad ocular. MX2019003361A (es)

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
MX2019003361A true MX2019003361A (es) 2019-07-18

Family

ID=57539952

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019003361A MX2019003361A (es) 2016-09-23 2017-09-22 Oligonucleotidos antisentido para el tratamiento de enfermedad ocular.

Country Status (19)

Country Link
US (2) US10612025B2 (es)
EP (2) EP3516060B1 (es)
JP (1) JP7141123B2 (es)
KR (1) KR102450757B1 (es)
CN (1) CN109804069A (es)
AU (1) AU2017330062B2 (es)
BR (1) BR112019005586A2 (es)
CA (1) CA3035627A1 (es)
DK (1) DK3516060T3 (es)
EA (1) EA201990644A1 (es)
ES (1) ES2886118T3 (es)
GB (1) GB201616202D0 (es)
IL (1) IL265206B2 (es)
MX (1) MX2019003361A (es)
NZ (1) NZ752572A (es)
PL (1) PL3516060T3 (es)
PT (1) PT3516060T (es)
WO (1) WO2018055134A1 (es)
ZA (1) ZA201901247B (es)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017220751A1 (en) 2016-06-22 2017-12-28 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
WO2020157014A1 (en) * 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of leber's congenital amaurosis
JP2022523302A (ja) 2019-01-28 2022-04-22 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 アッシャー症候群の処置のためのｒｎａ編集オリゴヌクレオチド
CA3132178A1 (en) 2019-04-02 2020-10-08 Aliye Seda Yilmaz-Elis Antisense oligonucleotides for immunotherapy
PE20212214A1 (es) 2019-04-15 2021-11-19 Edigene Inc Metodos y composiciones para editar acidos ribonucleicos (arn)
EP3956447A1 (en) 2019-04-18 2022-02-23 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of usher syndrome
WO2020214796A1 (en) * 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for usher syndrome (ush2a)
CA3137740A1 (en) * 2019-04-25 2020-10-29 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
AU2020313143B2 (en) 2019-07-12 2024-04-04 Peking University Targeted RNA editing by leveraging endogenous adar using engineered RNAs
WO2021018750A1 (en) 2019-07-26 2021-02-04 Proqr Therapeutics Ii B.V. Ophthalmic compositions comprising viscosifying polymers and nucleic acids
EP4069842A1 (en) 2019-12-02 2022-10-12 Shape Therapeutics Inc. Therapeutic editing
AU2020414395A1 (en) 2019-12-23 2022-07-21 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease
WO2021136404A1 (zh) * 2019-12-30 2021-07-08 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
WO2021175904A1 (en) 2020-03-04 2021-09-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
US20240141340A1 (en) 2021-03-05 2024-05-02 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

Family Cites Families (11)

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Publication number Priority date Publication date Assignee Title
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
PL2425814T3 (pl) 2010-09-03 2013-11-29 Santen Sas Emulsja typu woda-w-oleju do leczenia choroby oka
US9353371B2 (en) 2011-05-02 2016-05-31 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
ES2646716T3 (es) * 2011-09-05 2017-12-15 Stichting Katholieke Universiteit Oligonucleótidos antisentido para el tratamiento de amaurosis congénita de Leber
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
KR20160089527A (ko) * 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
AU2015286663B2 (en) * 2014-07-10 2021-09-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for the treatment of usher syndrome type 2
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
IL265206A (en) 2019-05-30
PT3516060T (pt) 2021-08-27
EP3516060B1 (en) 2021-07-21
IL265206B1 (en) 2023-04-01
JP2019528747A (ja) 2019-10-17
ZA201901247B (en) 2020-10-28
AU2017330062B2 (en) 2024-04-04
KR20190051020A (ko) 2019-05-14
NZ752572A (en) 2023-01-27
EP3516060A1 (en) 2019-07-31
CN109804069A (zh) 2019-05-24
AU2017330062A1 (en) 2019-05-02
US10612025B2 (en) 2020-04-07
US20200181616A1 (en) 2020-06-11
US11479771B2 (en) 2022-10-25
WO2018055134A1 (en) 2018-03-29
KR102450757B1 (ko) 2022-10-04
CA3035627A1 (en) 2018-03-29
IL265206B2 (en) 2023-08-01
EP3985117A1 (en) 2022-04-20
GB201616202D0 (en) 2016-11-09
JP7141123B2 (ja) 2022-09-22
PL3516060T3 (pl) 2021-12-13
ES2886118T3 (es) 2021-12-16
DK3516060T3 (da) 2021-09-27
US20190256847A1 (en) 2019-08-22
EA201990644A1 (ru) 2019-08-30
BR112019005586A2 (pt) 2019-06-11

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