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, Regeneron PharmafiledCriticalAlnylam Pharmaceuticals Inc
Publication of AR120712A1publicationCriticalpatent/AR120712A1/en
Pharmaceuticals Containing Other Organic And Inorganic Compounds
(AREA)
Abstract
Agentes y composiciones de ácido ribonucleico de hebra doble (ARNhdi) dirigidos al gen del marco de lectura abierto 72 del cromosoma 9 humano (C9orf72), así como métodos para inhibir la expresión de un gen C9orf72 y métodos de tratamiento de sujetos que padecen una enfermedad o trastorno asociado a C9orf72, por ejemplo, esclerosis lateral amiotrófica / demencia frontotemporal por C9orf72 o el síndrome tipo Huntington debido a expansiones del C9orf72, usando dichos agentes de ARNhdi y composiciones con los mismos.Double-stranded ribonucleic acid (dsRNA) agents and compositions targeting the human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods for inhibiting the expression of a C9orf72 gene and methods of treating subjects suffering from a disease or C9orf72-associated disorder, eg, C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-like syndrome due to C9orf72 expansions, using such shRNA agents and compositions therewith.
ARP200103433A2019-12-132020-12-10
HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF
AR120712A1
(en)
RNAi COMPOSITIONS AGAINST Factor XII (Hageman FACTOR) (F12), CALICREINE B, PLASMATIC (FLETCHER FACTOR) 1 (KLKB1), AND FIFTH KINOGEN 1 (KNG1) AND METHODS OF USE