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AR118087A2 - Dosificaciones y métodos para administrar moléculas de ácido nucleico formuladas en lípidos - Google Patents

Dosificaciones y métodos para administrar moléculas de ácido nucleico formuladas en lípidos

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Publication number
AR118087A2
AR118087A2 ARP200100401A ARP200100401A AR118087A2 AR 118087 A2 AR118087 A2 AR 118087A2 AR P200100401 A ARP200100401 A AR P200100401A AR P200100401 A ARP200100401 A AR P200100401A AR 118087 A2 AR118087 A2 AR 118087A2
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AR
Argentina
Prior art keywords
dose
composition
administered
administration
nucleic acid
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Application number
ARP200100401A
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English (en)
Inventor
Jared Gollob
Pia Kasperkovitz
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Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR118087A2 publication Critical patent/AR118087A2/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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Abstract

Métodos, conjuntos de elementos y dispositivos de dosificación de un sujeto para reducir una respuesta de hipersensibilidad a una molécula de ácido nucleico (por ejemplo, ARN) formulada en lípidos. Reivindicación 1: Un método para reducir una respuesta relacionada con la infusión (lRR) y/o una reacción de hipersensibilidad, en un sujeto, a una composición que comprende una formulación de lípidos y una molécula de ácido nucleico, caracterizado porque dicho método comprende administrar a un sujeto: una primera dosis de dicha composición; una segunda dosis de dicha composición; y en donde se cumple uno, dos, tres, cuatro o todas las siguientes condiciones de a) - e): a) la cantidad de dicha composición administrada en dicha primera dosis no es más que 1/X, en donde X es 2, 3, 4, 5, 6, 7, 8, 9, 10, 15, 20, 30, 40 ó 50, de la cantidad de dicha composición administrada en dicha segunda dosis; b) la cantidad de dicha composición administrada en dicha primera dosis no es más que 1/X, en donde X es 2, 3, 4, 5, 6, 7, 8, 9, 10, 15, 20, 30, 40 ó 50, de la cantidad total de dicha composición administrada; c) la primera dosis se administra sobre un período de tiempo que no es mayor que 1/X, en donde X es 2, 3, 4, 5, 6, 7, 8, 9 ó 10, del período de tiempo sobre el cual se administra la segunda dosis; d) la frecuencia de administración, por ejemplo, en mg/min o ml/min, de dicha primera dosis no es mayor que 1/X, en donde X es 2, 3, 4, 5, 6, 7, 8, 9, 10, 15, 20, 30, 40 ó 50, de la frecuencia de administración de dicha segunda dosis; o e) las dosificaciones y los períodos de administración de dichas primera y segunda dosis se seleccionan de manera tal que no hay una IRR y/o una reacción de hipersensibilidad sustancial en dicho sujeto. Reivindicación 81: El método de cualquiera de las reivindicaciones 72 - 80, caracterizado porque la formulación de lípidos comprende aproximadamente 50% de lípidos catiónicos de la fórmula I/MC3, aproximadamente 10% del lípido neutro (por ejemplo, DSPC), aproximadamente 38,5% del esterol (por ejemplo, colesterol) y aproximadamente 1,5% del PEG o del lípido modificado con PEG (por ejemplo, PEG-DMG) (también denominado LNP-11 en la presente). Reivindicación 94: El método de cualquiera de las reivindicaciones 1 - 93, caracterizado porque la molécula de ácido nucleico se selecciona entre: moléculas de ARN de cadena doble (ARNcd), moléculas de ARNi de cadena simple, microARN (miARN), ARN antisentido, ARN en horquilla corta (ARNhc), antagómiros, ARNm, ARN señuelo, ADN, plásmidos o aptámeros. Reivindicación 117: Un dispositivo, por ejemplo, un dispositivo para administración intravenosa, caracterizado porque comprende: un reservorio, por ejemplo, una bolsa, que contiene una composición (por ejemplo, una composición que se puede administrar como una primera y/o una segunda dosis según se describe en la presente), donde dicha composición comprende una formulación de lípidos y una molécula de ácido nucleico (por ejemplo, ARN) como se describe en la presente; un conducto (por ejemplo, tubos); un medio (por ejemplo, una válvula) para ajustar la frecuencia de administración de la composición; y (opcionalmente) una aguja; en donde dicho conducto comunica dicho reservorio con el medio (por ejemplo, una válvula) (y opcionalmente, hay un segundo conducto que lo conecta con la aguja), y en donde: a) la composición se administra a una primera dosis (por ejemplo, una primera dosis como la descrita en la presente) sobre un período de tiempo que no es mayor que 1/X, en donde X es 2, 3, 4, 5, 6, 7, 8, 9 ó 10, del período de tiempo sobre el cual se administra la segunda dosis; o b) la frecuencia de administración, por ejemplo, en mg/min o ml/min, de dicha primera dosis no es mayor que 1/X, en donde X es 2, 3, 4, 5, 6, 7, 8, 9, 10, 15, 20, 30 40 ó 50, de la frecuencia de administración de dicha segunda dosis.
ARP200100401A 2013-05-06 2020-02-14 Dosificaciones y métodos para administrar moléculas de ácido nucleico formuladas en lípidos AR118087A2 (es)

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US (4) US10246708B2 (es)
EP (1) EP2994167B1 (es)
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