MX2019008199A - Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. - Google Patents

Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.

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Publication number
MX2019008199A
MX2019008199A MX2019008199A MX2019008199A MX2019008199A MX 2019008199 A MX2019008199 A MX 2019008199A MX 2019008199 A MX2019008199 A MX 2019008199A MX 2019008199 A MX2019008199 A MX 2019008199A MX 2019008199 A MX2019008199 A MX 2019008199A
Authority
MX
Mexico
Prior art keywords
exon skipping
methods
nucleic acid
polypeptide compositions
inducing exon
Prior art date
Application number
MX2019008199A
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English (en)
Inventor
John Geall Andrew
Ramana Doppalapudi Venkata
Caramian Cochran Michael
BURKE Rob
A Levin Arthur
Huang Hanhua
Original Assignee
Avidity Biosciences Llc
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Filing date
Publication date
Application filed by Avidity Biosciences Llc filed Critical Avidity Biosciences Llc
Publication of MX2019008199A publication Critical patent/MX2019008199A/es

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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
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    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K9/51—Nanocapsules; Nanoparticles
    • A—HUMAN NECESSITIES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003—Peptide-nucleic acids (PNAs)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • C—CHEMISTRY; METALLURGY
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    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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    • 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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    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2320/33—Alteration of splicing

Abstract

En el presente documento se describen moléculas y composiciones farmacéuticas que inducen una inserción, eliminación, duplicación o alteración en un transcrito de ARNm empalmando incorrectamente para inducir la omisión de exones a la inclusión de exón. En el presente documento también se incluyen métodos para tratar una enfermedad o trastorno que comprende un molécula o una composición farmacéutica que induce a una inserción, eliminación, duplicación, o alteración en un transcrito de ARNm mal empalmado, para inducir la omisión de exón o la inclusión del mismo.
MX2019008199A 2017-01-06 2018-01-05 Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. MX2019008199A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762443514P 2017-01-06 2017-01-06
US201762561939P 2017-09-22 2017-09-22
PCT/US2018/012672 WO2018129384A1 (en) 2017-01-06 2018-01-05 Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Publications (1)

Publication Number Publication Date
MX2019008199A true MX2019008199A (es) 2019-11-25

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MX2019008199A MX2019008199A (es) 2017-01-06 2018-01-05 Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.

Country Status (11)

Country Link
US (5) US10994020B2 (es)
EP (1) EP3565577A4 (es)
JP (2) JP2020505330A (es)
KR (1) KR20190104381A (es)
CN (1) CN110381980A (es)
AU (2) AU2018205259B2 (es)
CA (1) CA3049424A1 (es)
IL (1) IL267793A (es)
MX (1) MX2019008199A (es)
SG (2) SG10202107429WA (es)
WO (1) WO2018129384A1 (es)

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