A drug repurposing project for the ultra-rare mitochondrial disease MEPAN has yielded a clinical candidate and serendipitously illuminated a pathway to designer pharmacological chaperones using GenAI.
Repurposing is a gateway drug
A drug repurposing project for the ultra-rare mitochondrial disease MEPAN has yielded a clinical candidate and serendipitously illuminated a pathway to designer pharmacological chaperones using GenAI.