MX2023009712A - Composiciones oligonucleotidicas y sus metodos. - Google Patents

Composiciones oligonucleotidicas y sus metodos.

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Publication number
MX2023009712A
MX2023009712A MX2023009712A MX2023009712A MX2023009712A MX 2023009712 A MX2023009712 A MX 2023009712A MX 2023009712 A MX2023009712 A MX 2023009712A MX 2023009712 A MX2023009712 A MX 2023009712A MX 2023009712 A MX2023009712 A MX 2023009712A
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MX
Mexico
Prior art keywords
oligonucleotide compositions
provided oligonucleotide
methods
compositions
present disclosure
Prior art date
Application number
MX2023009712A
Other languages
English (en)
Inventor
Chandra Vargeese
Gregory L Verdine
David Charles Donnell Butler
Naoki Iwamoto
Christopher J Francis
Sethumadhavan Divakaramenon
Maria David Frank-Kamenetsky
Genliang Lu
Subramanian Marappan
Hailin Yang
Jason Jingxin Zhang
Meena 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 MX2023009712A publication Critical patent/MX2023009712A/es

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    • 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
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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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    • 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
    • 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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    • 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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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • 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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    • 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.
MX2023009712A 2015-10-09 2018-03-28 Composiciones oligonucleotidicas y sus metodos. MX2023009712A (es)

Applications Claiming Priority (3)

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

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MX2023009712A true MX2023009712A (es) 2023-09-15

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MX2018003992A MX2018003992A (es) 2015-10-09 2016-10-07 Composiciones oligonucleotidicas y sus metodos.
MX2023009712A MX2023009712A (es) 2015-10-09 2018-03-28 Composiciones oligonucleotidicas y sus metodos.

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US (4) US20190127733A1 (es)
EP (2) EP3359523A4 (es)
JP (3) JP2018537952A (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) IL295755A (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)
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WO (2) WO2017062862A2 (es)

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