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MX2024000575A - Composiciones de acido nucleico-polipeptido y su uso en la induccion de la omision de exon. - Google Patents

Composiciones de acido nucleico-polipeptido y su uso en la induccion de la omision de exon.

Info

Publication number
MX2024000575A
MX2024000575A MX2024000575A MX2024000575A MX2024000575A MX 2024000575 A MX2024000575 A MX 2024000575A MX 2024000575 A MX2024000575 A MX 2024000575A MX 2024000575 A MX2024000575 A MX 2024000575A MX 2024000575 A MX2024000575 A MX 2024000575A
Authority
MX
Mexico
Prior art keywords
exon skipping
methods
nucleic acid
polypeptide compositions
inducing exon
Prior art date
Application number
MX2024000575A
Other languages
English (en)
Inventor
Venkata Ramana Doppalapudi
Michael Caramian COCHRAN
Rob Burke
Arthur A Levin
Hanhua Huang
Andrew John Geall
Original Assignee
Avidity Biosciences Inc
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 Avidity Biosciences Inc filed Critical Avidity Biosciences Inc
Publication of MX2024000575A publication Critical patent/MX2024000575A/es

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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    • A61K47/59—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
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    • A61K48/0025—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 non-active part clearly interacts with the delivered nucleic acid
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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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C—CHEMISTRY; METALLURGY
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    • 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
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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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Abstract

En el presente documento se describe un conjugado de oligonucleótido monocatenario para usarse en el tratamiento de distrofia muscular en un sujeto que lo necesite, en donde el conjugado de oligonucleótido monocatenario está adaptado para ser administrable al sujeto, en donde el conjugado de oligonucleótido monocatenario comprende un anticuerpo anti-receptor de transferrina o fragmento de unión al antígeno del mismo conjugado con un oligonucleótido monocatenario que hibrida con un sitio de empalme aceptor, con un sitio de empalme donante, o con un elemento potenciador de empalme exónico de un transcrito del pre-ARNm del gen DMD; en donde el conjugado de oligonucleótido monocatenario induce la omisión de exón en el transcrito del pre-ARNm para generar un transcrito de ARNm que codifica una proteína distrofina truncada, tratando de este modo la distrofia muscular en el sujeto.
MX2024000575A 2017-01-06 2019-07-05 Composiciones de acido nucleico-polipeptido y su uso en la induccion de la omision de exon. MX2024000575A (es)

Applications Claiming Priority (2)

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US201762443514P 2017-01-06 2017-01-06
US201762561939P 2017-09-22 2017-09-22

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MX2024000575A true MX2024000575A (es) 2024-01-29

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MX2024000575A MX2024000575A (es) 2017-01-06 2019-07-05 Composiciones de acido nucleico-polipeptido y su uso en la induccion de la omision de exon.

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US (7) US10994020B2 (es)
EP (2) EP4556489A3 (es)
JP (3) JP7573966B2 (es)
KR (2) KR20240172236A (es)
CN (1) CN110381980A (es)
AU (2) AU2018205259B2 (es)
CA (1) CA3049424A1 (es)
IL (1) IL267793A (es)
MX (2) MX2019008199A (es)
SG (2) SG11201906200WA (es)
WO (1) WO2018129384A1 (es)

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MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
EP3630117A4 (en) 2017-05-25 2021-02-24 Mingsight Pharmaceuticals, Inc. PKC INHIBITOR SOLID STATE FORMS
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