AR114435A1 - RNAi COMPOSITIONS OF 17b-HYDROXYESTEROID DEHYDROGENASES TYPE 13 (HSD17B13) AND METHODS OF USE OF THE SAME - Google Patents

RNAi COMPOSITIONS OF 17b-HYDROXYESTEROID DEHYDROGENASES TYPE 13 (HSD17B13) AND METHODS OF USE OF THE SAME

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Publication number
AR114435A1
AR114435A1 ARP190100717A ARP190100717A AR114435A1 AR 114435 A1 AR114435 A1 AR 114435A1 AR P190100717 A ARP190100717 A AR P190100717A AR P190100717 A ARP190100717 A AR P190100717A AR 114435 A1 AR114435 A1 AR 114435A1
Authority
AR
Argentina
Prior art keywords
hsd17b13
hdrna
agent
methods
nucleotides
Prior art date
Application number
ARP190100717A
Other languages
Spanish (es)
Inventor
Aris Baras
Frederick E Dewey
Jesper Gromada
Yurong Xin
Xiping Cheng
Alexander Li
Omri Gottesman
Noura S Abul-Husn
Stuart Milstein
Frederic Tremblay
Alan Shuldiner
Gregory Hinkle
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of AR114435A1 publication Critical patent/AR114435A1/en

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Abstract

Composiciones de ácidos ribonucleicos de hebra doble (ARNhd) dirigidos al gen HSD17B13, así como métodos para inhibir la expresión de HSD17B13, y métodos para tratar a sujetos que pudieran verse beneficiados con una reducción de la expresión de HSD17B13, tales como sujetos que padecen una enfermedad, trastorno o condición asociada con HSD17B13, mediante el uso de dichas composiciones de ARNhd. Reivindicación 1: Un agente de ácido ribonucleico de hebra doble (ARNhd) para inhibir la expresión de 17b-hidroxiesteroide deshidrogenasas tipo 13 (HSD17B13) en una célula, caracterizado porque el agente de ARNhd comprende una hebra orientada en el sentido del marco de lectura y una hebra antisentido, en donde la hebra orientada en el sentido del marco de lectura comprende por lo menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de la secuencia de nucleótidos de la SEQ ID Nº 1 ó 2 y la hebra antisentido comprende por lo menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de la secuencia de nucleótidos de la SEQ ID Nº 8 ó 9. Reivindicación 23: El agente de ARNhd de cualquiera de las reivindicaciones 1 - 6 y 11 - 22, caracterizado porque además comprende un ligando. Reivindicación 27: El agente de ARNhd de la reivindicación 26, caracterizado porque el agente de ARNhd está conjugado al ligando como se muestra en el esquema de fórmula (1) y, en donde X es O ó S.Compositions of double-stranded ribonucleic acids (hdRNA) targeting the HSD17B13 gene, as well as methods for inhibiting the expression of HSD17B13, and methods for treating subjects who could benefit from reduced expression of HSD17B13, such as subjects suffering from a disease, disorder or condition associated with HSD17B13, through the use of said hRNA compositions. Claim 1: A double-stranded ribonucleic acid (hdRNA) agent for inhibiting the expression of 17b-hydroxysteroid dehydrogenases type 13 (HSD17B13) in a cell, characterized in that the hdRNA agent comprises a strand oriented in the reading frame and an antisense strand, wherein the frame oriented strand comprises at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from the nucleotide sequence of SEQ ID No. 1 or 2 and the antisense strand comprises by at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from the nucleotide sequence of SEQ ID No. 8 or 9. Claim 23: The hdRNA agent of any of claims 1-6 and 11-22, characterized in that further comprises a ligand. Claim 27: The hdRNA agent of claim 26, characterized in that the hdRNA agent is conjugated to the ligand as shown in the scheme of formula (1) and, wherein X is O or S.

ARP190100717A 2018-03-21 2019-03-21 RNAi COMPOSITIONS OF 17b-HYDROXYESTEROID DEHYDROGENASES TYPE 13 (HSD17B13) AND METHODS OF USE OF THE SAME AR114435A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862645941P 2018-03-21 2018-03-21

Publications (1)

Publication Number Publication Date
AR114435A1 true AR114435A1 (en) 2020-09-02

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Family Applications (1)

Application Number Title Priority Date Filing Date
ARP190100717A AR114435A1 (en) 2018-03-21 2019-03-21 RNAi COMPOSITIONS OF 17b-HYDROXYESTEROID DEHYDROGENASES TYPE 13 (HSD17B13) AND METHODS OF USE OF THE SAME

Country Status (1)

Country Link
AR (1) AR114435A1 (en)

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