[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

MX2021012862A - Composiciones de oligonucleotidos y metodos de uso de las mismas. - Google Patents

Composiciones de oligonucleotidos y metodos de uso de las mismas.

Info

Publication number
MX2021012862A
MX2021012862A MX2021012862A MX2021012862A MX2021012862A MX 2021012862 A MX2021012862 A MX 2021012862A MX 2021012862 A MX2021012862 A MX 2021012862A MX 2021012862 A MX2021012862 A MX 2021012862A MX 2021012862 A MX2021012862 A MX 2021012862A
Authority
MX
Mexico
Prior art keywords
ush2a
methods
oligonucleotides
usher syndrome
compositions
Prior art date
Application number
MX2021012862A
Other languages
English (en)
Inventor
Chandra Vargeese
Naoki Iwamoto
Michael John Byrne
Vinod Vathipadiekal
Lankai Guo
Original Assignee
Wave Life Sciences Ltd
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 Wave Life Sciences Ltd filed Critical Wave Life Sciences Ltd
Publication of MX2021012862A publication Critical patent/MX2021012862A/es

Links

Classifications

    • 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/106Primate
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/40Fish
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Entre otras cosas, la presente divulgación proporciona oligonucleótidos de USH2A, y composiciones y métodos de uso de los mismos, para prevenir y/o tratar diversas afecciones, trastornos o enfermedades. En algunas realizaciones, los oligonucleótidos de USH2A proporcionados comprenden modificaciones de nucleobase, modificaciones de azúcar, modificaciones de unión internucleotídica y/o patrones de las mismas, y tienen propiedades, actividades y/o selectividades mejoradas. En algunas realizaciones, la presente divulgación proporciona oligonucleótidos de USH2A, composiciones y métodos para prevenir y/o tratar afecciones, trastornos o enfermedades relacionados con USH2A, tales como el síndrome de Usher (por ejemplo, el síndrome de Usher de tipo 2A), el síndrome de Usher atípico o la retinitis pigmentosa no sindrómica.
MX2021012862A 2019-04-25 2020-04-24 Composiciones de oligonucleotidos y metodos de uso de las mismas. MX2021012862A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962838701P 2019-04-25 2019-04-25
US201962905323P 2019-09-24 2019-09-24
PCT/US2020/029957 WO2020219981A2 (en) 2019-04-25 2020-04-24 Oligonucleotide compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2021012862A true MX2021012862A (es) 2022-03-04

Family

ID=72941436

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021012862A MX2021012862A (es) 2019-04-25 2020-04-24 Composiciones de oligonucleotidos y metodos de uso de las mismas.

Country Status (7)

Country Link
EP (1) EP3958872A4 (es)
AU (1) AU2020261434A1 (es)
BR (1) BR112021021203A2 (es)
CA (1) CA3137740A1 (es)
MX (1) MX2021012862A (es)
SG (1) SG11202111386UA (es)
WO (1) WO2020219981A2 (es)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101850319B1 (ko) 2012-07-13 2018-04-20 웨이브 라이프 사이언시스 리미티드 비대칭 보조 그룹
EP2872147B1 (en) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US11407775B2 (en) 2016-03-13 2022-08-09 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
US10988763B2 (en) 2016-06-22 2021-04-27 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
JP2020522510A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物及びその使用方法
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018237194A1 (en) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. COMPOUNDS, COMPOSITIONS AND METHODS OF SYNTHESIS
US20200362337A1 (en) 2017-08-08 2020-11-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
SG11202001783YA (en) 2017-10-12 2020-03-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
WO2024121373A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024200472A1 (en) 2023-03-27 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220106232A (ko) * 2014-01-16 2022-07-28 웨이브 라이프 사이언시스 리미티드 키랄 디자인
EP3957735A1 (en) * 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US10131910B2 (en) * 2014-07-10 2018-11-20 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Usher syndrome type 2
MA43072A (fr) * 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JP7511326B2 (ja) * 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
GB201616202D0 (en) * 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
GB201808146D0 (en) * 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides

Also Published As

Publication number Publication date
WO2020219981A3 (en) 2020-11-26
EP3958872A2 (en) 2022-03-02
BR112021021203A2 (pt) 2022-01-04
WO2020219981A2 (en) 2020-10-29
CA3137740A1 (en) 2020-10-29
EP3958872A4 (en) 2024-07-03
AU2020261434A1 (en) 2021-11-11
SG11202111386UA (en) 2021-11-29

Similar Documents

Publication Publication Date Title
MX2021012862A (es) Composiciones de oligonucleotidos y metodos de uso de las mismas.
MX2021009178A (es) Composiciones de oligonucleotidos y metodos de las mismas.
MX2021012981A (es) Composiciones de oligonucleotido y metodos de uso de las mismas.
MX2022004101A (es) Composiciones de oligonucleotidos y metodos de uso de las mismas.
MX2023005326A (es) Composiciones de oligonucleótidos y métodos de las mismas.
WO2021178237A3 (en) Oligonucleotide compositions and methods thereof
MX2022014606A (es) Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
MX2020003965A (es) Terapias genicas para los trastornos lisosomales.
PE20181085A1 (es) Composiciones y metodos para modular la expresion de angiotensinogeno
EP4026835A3 (en) Pd-1/pd-l1 inhibitors
WO2016106400A3 (en) Rna interference agents for gst-pi gene modulation
MX2018008266A (es) Dinucleotidos ciclicos para tratar condiciones asociadas con actividad del estimulador de genes del interferon (sting) tales como cancer.
MX2022006700A (es) Compuestos poliheterociclicos como inhibidores de mettl3.
MX2023001450A (es) Composiciones y metodos para la inhibicion de la expresion de plp1.
MX2020004005A (es) Terapias genicas para los trastornos lisosomales.
EP4371562A3 (en) 2-quinolone derived inhibitors of bcl6
MX2021006745A (es) Constructos de iarn modificados quimicamente y usos de estos.
MX2022011550A (es) Composiciones y métodos para inhibir la expresión de angptl3.
MX2023001538A (es) Composiciones y metodos para inhibir la expresion de lpa.
CR20220205A (es) INHIBICIÓN DE INTEGRINA a4ß7 HUMANA
MX2018001126A (es) Oligonucleotidos terapeuticos.
MX2023012048A (es) Composiciones y metodos para inhibir cetohexoquinasa (khk).
PH12020551641A1 (en) Compositions and methods for treating macular dystrophy
MX2024000996A (es) Oligonucleótido antisentido unc13a.
CA3156667A1 (en) OLIGONUCLEOTIDES INCLUDING NUCLEOSIDE ANALOGS