The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Dec. 19, 2023
Filed:
Mar. 09, 2023
Arrowhead Pharmaceuticals, Inc., Pasadena, CA (US);
Zhi-Ming Ding, Waunakee, WI (US);
Jonathan Van Dyke, Burke, WI (US);
Xiaokai Li, Middleton, WI (US);
Anthony Nicholas, Oregon, WI (US);
Casi M. Schienebeck, Deerfield, WI (US);
Tao Pei, Middleton, WI (US);
Zhao Xu, Brookfield, WI (US);
Teng Ai, Middleton, WI (US);
Susan Phan, Fitchburg, WI (US);
Susan Ramos-Hunter, Hendersonville, TN (US);
Arrowhead Pharmaceuticals, Inc., Pasadena, CA (US);
Abstract
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a double homeobox 4 (DUX4) gene. The DUX4 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a DUX4 gene. Pharmaceutical compositions that include one or more DUX4 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described DUX4 RNAi agents to skeletal muscle cells in vivo, provides for inhibition of DUX4 gene expression and a reduction in DUX4 levels, which can provide a therapeutic benefit to subjects, including human subjects, suffering from certain skeletal muscle-related diseases or disorders including Facioscapulohumeral Muscular Dystrophy (FSHD).