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AR134211A2 - ARNi COMPOSITIONS AGAINST TMPRSS6 AND METHODS FOR THEIR USE - Google Patents

ARNi COMPOSITIONS AGAINST TMPRSS6 AND METHODS FOR THEIR USE

Info

Publication number
AR134211A2
AR134211A2 ARP240103000A ARP240103000A AR134211A2 AR 134211 A2 AR134211 A2 AR 134211A2 AR P240103000 A ARP240103000 A AR P240103000A AR P240103000 A ARP240103000 A AR P240103000A AR 134211 A2 AR134211 A2 AR 134211A2
Authority
AR
Argentina
Prior art keywords
nucleotides
agent
tmprss6
hrna
antisense strand
Prior art date
Application number
ARP240103000A
Other languages
Spanish (es)
Inventor
Jim Butler
Brian Bettencourt
Kallanthottathil G Rajeev
Martin Maier
Klaus Charisse
Original Assignee
Alnylam Pharmaceuticals 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR134211A2 publication Critical patent/AR134211A2/en

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La invención se relaciona con agentes de ARNi, por ejemplo, agentes de ARNi de doble hebra, que se dirigen contra el gen TMPRSS6, y métodos para usar dichos agentes de ARNi para inhibir la expresión de TMPRSS6 y métodos para tratar sujetos que tienen un trastorno asociado con TMPRSS6, por ejemplo, un trastorno asociado a sobrecarga de hierro, tal como b-talasemia o hemocromatosis. Reivindicación 1: Un agente de ácido ribonucleico de doble hebra (ARNhd) o una sal farmacéuticamente aceptable del mismo, caracterizado porque comprende una hebra sentido y una hebra antisentido que forman una región de doble hebra, en donde la hebra antisentido comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de la secuencia de nucleótidos AAGGGCAGCUGAGCUCACCdTdT (SEQ ID Nº 1381), o en donde la hebra antisentido comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de la secuencia de nucleótidos AGAAUGAACCAGAAGAAGCAGGU (SEQ ID Nº 187), en donde todos los nucleótidos de la hebra sentido y todos los nucleótidos de la hebra antisentido son nucleótidos modificados seleccionados del grupo que consiste en un nucleótido modificado con 2’-O-metil (2’-OMe), un nucleótido modificado con 2’-desoxi-2’-fluoro (2’-F) y un nucleótido modificado en 2’-desoxi, en donde el agente de ARNhd comprende 6 - 8 enlaces internucleotídicos de fosforotioato, y en donde el agente de ARNhd está conjugado con un ligando derivado de N-acetilgalactosamina (GalNAc) unido en el extremo 3’, el extremo 5’ o ambos extremos de la hebra sentido. Reivindicación 24: Una composición farmacéutica para inhibir la expresión de un gen que codifica la proteasa transmembrana, serina 6 (TMPRSS6), caracterizada porque comprende el agente de ARNhd o una sal farmacéuticamente aceptable del mismo de cualquiera de las reivindicaciones 1 a 23. Reivindicación 30: Una célula aislada caracterizada porque contiene el agente de ARNhd o una sal farmacéuticamente aceptable del mismo de cualquiera de las reivindicaciones 1 a 23.The invention relates to RNAi agents, for example, double-stranded RNAi agents, that target the TMPRSS6 gene, and methods for using such RNAi agents to inhibit the expression of TMPRSS6 and methods for treating subjects who have a disorder associated with TMPRSS6, for example, an iron overload-associated disorder such as β-thalassemia or hemochromatosis. Claim 1: A double-stranded ribonucleic acid (dRNA) agent or a pharmaceutically acceptable salt thereof, characterized in that it comprises a sense strand and an antisense strand forming a double-stranded region, wherein the antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence AAGGGCAGCUGAGCUCACCdTdT (SEQ ID No. 1381), or wherein the antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence AGAAUGAACCAGAAGAAGCAGGU (SEQ ID No. 187), wherein all nucleotides of the sense strand and all nucleotides of the antisense strand are modified nucleotides selected from the group consisting of a 2-O-methyl modified nucleotide (2-OMe), a nucleotide modified with 2-deoxy-2-fluoro (2-F), and a nucleotide modified at 2-deoxy, wherein the hRNA agent comprises 6-8 internucleotide phosphorothioate linkages, and wherein the hRNA agent is conjugated with an N-acetylgalactosamine (GalNAc) derivative ligand attached to the 3' end, the 5' end, or both ends of the sense strand. Claim 24: A pharmaceutical composition for inhibiting the expression of a gene encoding the transmembrane protease serine 6 (TMPRSS6), characterized in that it comprises the hRNA agent or a pharmaceutically acceptable salt thereof of any one of claims 1 to 23. Claim 30: An isolated cell characterized in that it contains the hRNA agent or a pharmaceutically acceptable salt thereof of any one of claims 1 to 23.

ARP240103000A 2013-05-22 2024-11-01 ARNi COMPOSITIONS AGAINST TMPRSS6 AND METHODS FOR THEIR USE AR134211A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361826178P 2013-05-22 2013-05-22
US202061912988P 2020-12-06 2020-12-06

Publications (1)

Publication Number Publication Date
AR134211A2 true AR134211A2 (en) 2025-12-10

Family

ID=98218444

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP240103000A AR134211A2 (en) 2013-05-22 2024-11-01 ARNi COMPOSITIONS AGAINST TMPRSS6 AND METHODS FOR THEIR USE

Country Status (1)

Country Link
AR (1) AR134211A2 (en)

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