SV2018005619A - Composiciones de oligonucleotidos y metodos de los mismos - Google Patents

Composiciones de oligonucleotidos y metodos de los mismos

Info

Publication number
SV2018005619A
SV2018005619A SV2018005619A SV2018005619A SV2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A
Authority
SV
El Salvador
Prior art keywords
quirally
controlled
oligonucleotydic
forms
composition
Prior art date
Application number
SV2018005619A
Other languages
English (en)
Inventor
Chandra Vargeese
Nenad Svrzikapa
Susovan Mohapatra
Christopher J Francis
Gregory L Verdine
Anna Sokolovska
Meena
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 SV2018005619A publication Critical patent/SV2018005619A/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
    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

Abstract

ENTRE OTRAS COSAS, LA PRESENTE DIVULGACIÓN SE REFIERE A OLIGONUCLEÓTIDOS QUIRALMENTE CONTROLADOS DE DISEÑOS SELECCIONADOS, COMPOSICIONES OLIGONUCLEOTÍDICAS QUIRALMENTE CONTROLADAS Y MÉTODOS DE PREPARACIÓN Y USO DE ELLOS. EN ALGUNAS FORMAS DE REALIZACIÓN, UNA COMPOSICIÓN OLIGONUCLEOTÍDICA QUIRALMENTE CONTROLADA SUMINISTRADA PROPORCIONA DIFERENTES PATRONES DE ESCISIÓN DE UN POLÍMERO DE ÁCIDO NUCLEICO QUE UNA COMPOSICIÓN OLIGONUCLEOTÍDICA DE REFERENCIA. EN ALGUNAS FORMAS DE REALIZACIÓN, UNA COMPOSICIÓN OLIGONUCLEOTÍDICA QUIRALMENTE CONTROLADA SUMINISTRADA PROPORCIONA ESCISIÓN DE SITIO ÚNICO DENTRO DE UNA SECUENCIA COMPLEMENTARIA DE UN POLÍMERO DE ÁCIDO NUCLEICO. EN ALGUNAS FORMAS DE REALIZACIÓN, UNA COMPOSICIÓN OLIGONUCLEOTÍDICA QUIRALMENTE CONTROLADA TIENE CUALQUIER SECUENCIA DE BASES, Y/O PATRÓN O MODIFICACIONES DE BASE, MODIFICACIONES DE AZÚCAR, MODIFICACIONES ESTRUCTURALES Y/O ESTEREOQUÍMICA O COMBINACIÓN DE ESTOS ELEMENTOS DESCRITOS EN LA PRESENTE
SV2018005619A 2015-07-22 2018-01-22 Composiciones de oligonucleotidos y metodos de los mismos SV2018005619A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562195779P 2015-07-22 2015-07-22
US201562236847P 2015-10-02 2015-10-02
US201662331960P 2016-05-04 2016-05-04

Publications (1)

Publication Number Publication Date
SV2018005619A true SV2018005619A (es) 2018-07-20

Family

ID=57835219

Family Applications (1)

Application Number Title Priority Date Filing Date
SV2018005619A SV2018005619A (es) 2015-07-22 2018-01-22 Composiciones de oligonucleotidos y metodos de los mismos

Country Status (24)

Country Link
US (5) US20180216107A1 (es)
EP (3) EP4344744A2 (es)
JP (5) JP2018525357A (es)
KR (1) KR20180028516A (es)
CN (2) CN108135921B (es)
AU (2) AU2016297155B2 (es)
BR (1) BR112017028636A2 (es)
CA (1) CA2989682A1 (es)
CL (1) CL2018000145A1 (es)
CO (1) CO2018001484A2 (es)
CR (1) CR20180107A (es)
EC (1) ECSP18010463A (es)
HK (2) HK1255369A1 (es)
IL (1) IL256603A (es)
MA (1) MA43072A (es)
MX (1) MX2018000796A (es)
NI (1) NI201800009A (es)
PE (1) PE20181174A1 (es)
PH (1) PH12018500145A1 (es)
RU (1) RU2017146349A (es)
SG (1) SG10201912900XA (es)
SV (1) SV2018005619A (es)
TW (1) TW201707711A (es)
WO (2) WO2017015575A1 (es)

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EP3891284A4 (en) 2018-12-06 2023-04-12 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
CA3126845A1 (en) 2019-02-01 2020-08-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
AU2020241559A1 (en) 2019-03-20 2021-11-11 Wave Life Sciences Ltd. Technologies useful for oligonucleotide preparation
JPWO2020196662A1 (es) 2019-03-25 2020-10-01
SG11202111387YA (en) 2019-04-25 2021-11-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
EP3958872A2 (en) 2019-04-25 2022-03-02 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2020227691A2 (en) 2019-05-09 2020-11-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
BR112022006207A2 (pt) 2019-10-06 2022-07-26 Wave Life Sciences Ltd Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição
US20230220384A1 (en) 2019-10-06 2023-07-13 Prashant Monian Oligonucleotide compositions and methods of use thereof
WO2021178237A2 (en) 2020-03-01 2021-09-10 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
AU2021277407A1 (en) 2020-05-22 2022-11-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

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