July 2025

Genexgen Submits Orphan Drug Designation Application to FDA for HLH Therapy

Genexgen has officially submitted its Orphan Drug Designation (ODD) application to the U.S. Food and Drug Administration for the treatment of Hemophagocytic Lymphohistiocytosis (HLH), a rare and severe pediatric hyperinflammatory genetic disorder.

This marks a major scientific and operational milestone in Genexgen’s mission to radically extend human healthspan. By targeting HLH and leveraging the accelerated regulatory pathway for rare diseases, Genexgen aims to rapidly evaluate the safety and efficacy of its epigenetic modulation platform in humans. This first-in-human validation sets the stage for addressing one of the key hallmarks of aging: chronic systemic inflammation, also known as inflammaging.