Our Leigh Syndrome research is dedicated to understanding the genetic mutations and metabolic dysfunctions that lead to this progressive neurological disorder. We aim to identify potential therapeutic targets and develop interventions to slow or halt disease progression.
[We are excited to announce the discovery of a novel therapeutic agent, KIT-20, which has demonstrated remarkable efficacy in our advanced research using the Ndufs4 knockout mouse model of Leigh syndrome. KIT-20 significantly improves clinical symptoms and enhances neural activity, offering promising therapeutic potential. Moreover, KIT-20 has been granted Orphan Drug Designation by the FDA, a milestone that underscores its promise in addressing critical unmet needs in rare neurological disorders and accelerates its pathway toward clinical development. This achievement marks a significant step forward in our commitment to pioneering innovative treatments for complex conditions like Leigh syndrome. ]
