P38 kinase inhibitors reduce DUX4 and downstream gene expression for the treatment of FSHD

US10342786B2 · kind B2 · utility

Assignee

• Fulcrum Therapeutics, Inc. · US

Inventors

• Angela Cacace · Haddam Neck, US
• Luis Gustavo Alejandro Rojas Soto · Cambridge, US
• Lorin A. Thompson, III · Higganum, US
• Owen Brendan Wallace · Zionsville, US
• Lucienne V. Ronco · Wellesley, US
• Ning Shen · Cambridge, US
• Alan Scott Robertson · Cambridge, US
• Aaron Nakwon Chang · Lexington, US

Key dates

• Filing date: Oct 19, 2018
• Grant date: Jul 9, 2019
• Expiry date: Oct 19, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Q2600/156
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The disclosure relates to methods and compositions including p38 kinase inhibitors and agents that regulate expression of DUX4 and downstream genes including but not restricted to ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDC1L, RFPL2, CCNA1, SLC34A2, TPRX1, PRAMEF20, TRIM49, PRAMEF4, PRAME6, PRAMEF15, or ZNF280A. Methods useful for treating a disease associated with abnormal DUX4 and downstream gene expression (e.g., Fascioscapulohumeral muscular dystrophy) are disclosed.