Earthquake compound

The Earthquake compound is an undisclosed small molecule being developed by Hannah's Hope Fund (HHF) for the treatment of Giant Axonal Neuropathy (GAN). GAN is a rare, progressive neurodegenerative disorder caused by mutations in the *GAN* gene, which encodes gigaxonin, an E3 ubiquitin ligase adapter. In the absence of functional gigaxonin, intermediate filaments (IFs) such as neurofilaments and vimentin fail to be degraded and instead accumulate into toxic aggregates within axons. The Earthquake compound was identified through a high-throughput small molecule screen funded by HHF and is designed to modulate the gigaxonin pathway or directly promote the clearance of these pathological intermediate filament aggregates. It is currently in the preclinical stage of development.

Other names

HHF small moleculeGAN small molecule