Publication number: 20260325653
Abstract: Disclosed is a method and compounds for treating AMPK, cGAS, or ?F508-CFTR mediated diseases, the method including administering one or more AMPK, cGAS, or CFTR DUBTACs to a subject who has an AMPK, cGAS, or ?F508-CFTR-mediated disease, the AMPK, cGAS, or CFTR DUBTACs being heterobifunctional small-molecule compounds (or bivalent compounds in short) including an AMPK, cGAS, or CFTR ligand conjugated to a de-ubiquitination tag (such as a small-molecule ligand of USP7) via a linker, which would stabilize AMPK, cGAS, or CFTR. The AMPK, cGAS, or ?F508-CFTR-mediated disease can be a disease resulting from AMPK, cGAS, or CFTR destabilization. The AMPK, cGAS, or ?F508-CFTR-mediated disease can have reduced AMPK, cGAS, or CFTR expression relative to a wild-type tissue of the same species and tissue type. The disclosed bivalent compounds stabilize AMPK, cGAS, or CFTR by recruiting USP7.
Type:
Application
Filed:
May 2, 2024
Publication date:
October 22, 2026
Inventors:
Jian Jin, Wenyi Wei, Yan Xiong, Jing Liu, Kaixiu Luo, Xiaoping Hu, Zhen Wang, Li Chen