CO2018004758A2 - Composiciones oligonucleotídicas y sus métodos - Google Patents

Composiciones oligonucleotídicas y sus métodos

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Publication number
CO2018004758A2
CO2018004758A2 CONC2018/0004758A CO2018004758A CO2018004758A2 CO 2018004758 A2 CO2018004758 A2 CO 2018004758A2 CO 2018004758 A CO2018004758 A CO 2018004758A CO 2018004758 A2 CO2018004758 A2 CO 2018004758A2
Authority
CO
Colombia
Prior art keywords
oligonucleotide compositions
compositions provided
methods
oligonucleotide
better
Prior art date
Application number
CONC2018/0004758A
Other languages
English (en)
Inventor
Chandra Vargeese
Christopher J Francis
Gregory L Verdine
Meena
David Charles Donnell Butler
Sethumadhavan Divakaramenon
Maria David Frank-Kamenetsky
Naoki Iwamoto
Genliang Lu
Subramanian Marappan
Hailin Yang
Jason Jingxin Zhang
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 CO2018004758A2 publication Critical patent/CO2018004758A2/es

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • 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
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    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/553—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having one nitrogen atom as the only ring hetero atom
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    • C12N15/1138—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 against receptors or cell surface proteins
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Abstract

Entre otras cosas, la presente revelación se refiere a oligonucleótidos, composiciones diseñados y sus métodos. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas suministran un empalme alterado de un transcripto. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas tienen baja toxicidad. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas suministran mejores perfiles de ligación de proteínas. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas tienen mejor suministro. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas tienen mejor captación. En algunas formas de realización, la presente revelación proporciona métodos para el tratamiento de enfermedades usando composiciones proporcionadas oligonucleotídicas.
CONC2018/0004758A 2015-10-09 2018-05-03 Composiciones oligonucleotídicas y sus métodos CO2018004758A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562239839P 2015-10-09 2015-10-09
US201662331966P 2016-05-04 2016-05-04
US201662331961P 2016-05-04 2016-05-04
PCT/US2016/056123 WO2017062862A2 (en) 2015-10-09 2016-10-07 Oligonucleotide compositions and methods thereof

Publications (1)

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CO2018004758A2 true CO2018004758A2 (es) 2018-06-20

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CONC2018/0004758A CO2018004758A2 (es) 2015-10-09 2018-05-03 Composiciones oligonucleotídicas y sus métodos

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US (4) US20190127733A1 (es)
EP (2) EP3359523A4 (es)
JP (3) JP7511326B2 (es)
KR (1) KR20180056766A (es)
CN (3) CN108137492B (es)
AU (3) AU2016334232B2 (es)
BR (1) BR112018006636B1 (es)
CA (2) CA2999772A1 (es)
CL (1) CL2018000818A1 (es)
CO (1) CO2018004758A2 (es)
CR (1) CR20180233A (es)
EC (1) ECSP18032914A (es)
HK (1) HK1256225A1 (es)
IL (2) IL258230B (es)
MA (2) MA46427A (es)
MX (2) MX2018003992A (es)
NI (1) NI201800045A (es)
PE (1) PE20181353A1 (es)
PH (1) PH12018500719A1 (es)
SG (1) SG10201912902TA (es)
SV (1) SV2018005661A (es)
TW (1) TW201722439A (es)
WO (2) WO2017062862A2 (es)

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